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Sunday, February 10
1:00pm Executive Leadership Course: Improving Negotiations and Decision Making By Applying Game Theory (additional fees apply) Harm-Jan Borgeld , Merck KGaA; Stefanie Schubert, SRH University Heidelberg show more Session Description This workshop on Negotiations and Executive Decision Making is designed for executives who wish to enhance their skills in negotiations, influencing and decision making. The uniqueness of this advanced workshop is to leverage the combination of these skills. You will not only learn to anticipate but also to plan strategically ahead by making use of insights from game theory, with the aim to improve decision making and negotiation outcomes. The learnings from game theory will be applied to real-life pharma/biotech cases. Format: Featuring interactive sessions, facilitated discussions and in-class exercises, this workshop is primarily based on real-life pharma/biotech cases. The cases will be analyzed by uniquely combining the ideas of game theory and advanced business development practice. Learning Objectives By participating in this workshop, you will learn how to: Improve your decision making by acquire a systematic framework for decision making using Game Theory. Achieve better negotiation results by applying advanced negotiation techniques. Enhance your influencing skills by applying learnings from the BD practice. Master new strategies to steer business interactions. Who should attend? The workshop is designed for current and future executives with responsibilities or activities at the C-level or Senior Management who wish to improve their ability to create value through strategic decision making and successful negotiations. Topics covered: Decision making Successful CEOs decision making Parallel option assessment Influencers of rationale decision making Negotiations: First and second offer strategies Multiple Equivalent Simultaneous Offers Know when not to negotiate The Right Rationale Sources of Negotiation Power Group negotiations Influencing Traits of success influencers How to weaken your opponent’s strategic moves Influencing in the business environment Time Sunday, Feb 10 1:00PM - 5:30PM Location Hudson/Empire Speakers Harm-Jan Borgeld Merck KGaA Stefanie Schubert SRH University Heidelberg
Monday, February 11
9:00am OncoSec Medical Dan O'Connor, OncoSec show more Session Description OncoSec is a clinical-stage biotechnology company focused on developing cytokine-based intratumoral immunotherapies to stimulate the body's immune system to target and attack cancer. OncoSec's lead immunotherapy platform – TAVO™ enables the intratumoral delivery of DNA-based interleukin-12 (IL-12), a naturally occurring protein with immune-stimulating functions. The technology, which employs electroporation, is designed to produce a controlled, localized expression of IL-12 in the tumor microenvironment, enabling the immune system to target and attack tumors throughout the body. OncoSec has built a deep and diverse clinical pipeline utilizing TAVO™ as a potential treatment for multiple cancer indications either as a monotherapy or in combination with leading checkpoint inhibitors; with the latter potentially enabling OncoSec to address a great unmet medical need in oncology: anti-PD-1 non-responders. Results from recently completed clinical studies of TAV Time Monday, Feb 11 9:00AM - 9:15AM Location Hudson/Empire Speakers Dan O'Connor OncoSec Reshaping Tumor Microenvironments via Immunotherapies Jotin Marango, ROTH Capital Partners; Lewis H. Bender, Intensity Therapeutics; Sabine Chlosta, Triumvira; Gere diZerega, NanOlogy; Eric Falcand, Servier show more Session Description Current CAR-T therapies have shown significant benefits attacking hematological malignancies, and currently approved checkpoint inhibitors are highly but selectively effective in their impact on solid tumors. This session will examine the next wave of innovation in immunotherapies for leveraging knowledge of how tumor microenvironments develop to create treatments able to demonstrate more durable effects on shrinking tumors across wider ranges of patients. Moderator: Jotin Marango, MD, PhD, Managing Directior, Sr. Research Analyst, Roth Capital Partners, LLC Time Monday, Feb 11 9:00AM - 9:55AM Location Shubert Complex Speakers Jotin Marango ROTH Capital Partners Lewis H. Bender Intensity Therapeutics Sabine Chlosta Triumvira Gere diZerega NanOlogy Eric Falcand Servier
9:15am Arcturus Therapeutics Joseph Payne, Arcturus Therapeutics show more Session Description Founded in 2013 and based in San Diego, California, Arcturus Therapeutics Ltd. (NASDAQ: ARCT) is an RNA medicines company with enabling technologies – UNA Oligomer chemistry and LUNAR® lipid-mediated delivery. Arcturus’ diverse pipeline of RNA therapeutics includes programs pursuing rare diseases, Hepatitis B, non-alcoholic steatohepatitis (NASH), cystic fibrosis, and vaccines. Arcturus’ versatile RNA therapeutics platforms can be applied toward multiple types of RNA medicines including small interfering RNA, messenger RNA, replicon RNA, antisense RNA, microRNA and gene editing therapeutics. Arcturus owns LUNAR lipid-mediated delivery and Unlocked Nucleomonomer Agent (UNA) technology including UNA Oligomers, which are covered by its extensive patent portfolio (140 patents and patent applications, issued in the U.S., Europe, Japan, China and other countries). Arcturus’ proprietary UNA technology can be used to target individual genes in the human genome, as well as viral genes, and other species for therapeutic purposes. Arcturus’ commitment to the development of novel RNA therapeutics has led to partnerships with Janssen Pharmaceuticals, Inc., part of the Janssen Pharmaceutical Companies of Johnson & Johnson, Ultragenyx Pharmaceutical, Inc., Takeda Pharmaceutical Company Limited, Synthetic Genomics Inc., CureVac AG and the Cystic Fibrosis Foundation. For more information, visit www.Arcturusrx.com, the content of which is not incorporated herein by reference. Time Monday, Feb 11 9:15AM - 9:30AM Location Herald/Soho Speakers Joseph Payne Arcturus Therapeutics INmune Bio RJ Tesi, INmune Bio show more Session Description INmune Bio, the innate immunology company is proud to announce that it is now a publicly traded company (NASDAQ: INMB) and will be expanding its development programs. INMB is an immunology company that is focused on cancer with two programs in the clinic. INB03 targets myeloid derived suppressor cells (MDSC)., the cells known to cause resistance to immune checkpoint inhibitors (CPI). Combination of INB03 to inhibit MDSC should reverse resistance to CPI in an important group of patients who have failed CPI monotherapy. INKmune, a NK focused development program, has been given the green light by the MHRA to start clinical trials in women with relapsed/refractory ovarian cancer. INKmune is a biologic that prime the inert NK cells of the cancer patient to attack and kill their cancer. INMB is positioning INKmune to eliminate residual disease, the cancer that causes relapse and ultimately death in patients with cancer. INMB just completed an IPO to fund these programs into Phase II. Time Monday, Feb 11 9:15AM - 9:30AM Location Chelsea Speakers RJ Tesi INmune Bio Serina Therapeutics, Inc. Randall Moreadith, Serina Therapeutics, Inc. show more Session Description Serina Therapeutics, Inc. has developed a proprietary drug delivery polymer technology based upon the polymer poly(2-oxazoline), or POZ™. We are developing a pipeline of proprietary and partnered programs applying this platform to cancer, pain, refractory epilepsy, and movement disorders such as Parkinson’s disease and restless leg syndrome. In this presentation I will present "The Era of Once Weekly Continuous Drug Delivery in Parkinson's Disease is Here". We are launching a subsidiary focused on Parkinson's Disease and wish to meet with investors to raise a mezzanine round of $20-30 M with intent to IPO in 2019-2020. Time Monday, Feb 11 9:15AM - 9:30AM Location Gramercy Speakers Randall Moreadith Serina Therapeutics, Inc.
9:30am Aptorum Group Limited Sabrina Khan, Aptorum Group Limited; Thomas Lee, Aptroum Group Limited show more Session Description Aptorum Group is a Hong Kong based pharmaceutical company currently in the preclinical stage, dedicated to developing and commercializing a broad range of therapeutic and diagnostic technologies to tackle unmet medical needs. We have obtained exclusive licenses for our technologies. In addition, we are also developing certain proprietary technologies as product candidates. We are pursuing therapeutic and diagnostic projects (including projects seeking to use extracts or derivatives from natural substances to treat diseases) in neurology, infectious diseases, gastroenterology, oncology and other disease areas. Time Monday, Feb 11 9:30AM - 9:45AM Location Herald/Soho Speakers Sabrina Khan Aptorum Group Limited Thomas Lee Aptroum Group Limited aTyr Pharma, Inc. Sanjay Shukla, aTyr Pharma, Inc. show more Session Description aTyr is a biotherapeutics company engaged in the discovery and development of innovative medicines based on novel immunological pathways. aTyr’s research and development efforts are concentrated on a newly discovered area of biology, the extracellular functionality of tRNA synthetases. aTyr has built a global intellectual property estate directed to a potential pipeline of protein compositions derived from 20 tRNA synthetase genes. aTyr is focused on the therapeutic translation of the Resokine pathway, comprised of extracellular proteins derived from the histidyl tRNA synthetase gene family. ATYR1923 is a clinical-stage product candidate which binds to the neuropilin-2 receptor and is designed to down-regulate immune engagement in interstitial lung diseases and other immune-mediated diseases. Time Monday, Feb 11 9:30AM - 9:45AM Location Hudson/Empire Speakers Sanjay Shukla aTyr Pharma, Inc. NeuroRx Jonathan Javitt, MD, MPH, NeuroRx show more Session Description NeuroRx is developing NRX-101, an FDA-designated Breakthrough Therapy targeting Suicidal Bipolar Depression. NRX-101 is a fixed dose combination of D-cycloserine and lurasidone, which is believed to raise Glx in the brain, as does ketamine and ECT. NeuroRx draws upon 30 years of basic science and clinical expertise in the role of N-methyl-D-aspartate (NMDA), a receptor that regulates human thought processes, particularly depression and suicidality, as well as PTSD. The company is privately funded and led by former senior executives of Johnson & Johnson, BMS, Pfizer Inc., Eli Lilly, and Sunovion. NeuroRx has entered an FDA phase 2b/3 pivotal trial under a Special Protocol Agreement and a Biomarker Letter of Support for treatment of patients with Severe Bipolar Depression and Acute Suicidal Ideation. Phase 2 data demonstrated an 11 point advantage on MADRS depression scale at day 14 vs. lurasidone alone (P=0.03) and a sustained separation through day 42 (P=0.059). Time Monday, Feb 11 9:30AM - 9:45AM Location Chelsea Speakers Jonathan Javitt, MD, MPH NeuroRx Vallon Pharmaceuticals David Baker, Vallon Pharmaceuticals show more Session Description Vallon Pharmaceuticals Inc., headquartered in Philadelphia, is an emerging pharmaceutical company focused on the development of new medications to help patients with central nervous system (CNS) disorders. The company’s lead product candidate is an abuse deterrent formulation of amphetamine immediate release (ADAIR) for the treatment of ADHD and Narcolepsy. There is substantial unmet need for abuse deterrent psychostimulants, given that 5 million adolescents and adults misuse Rx stimulants annually and over 40% of abusers/misusers do so by non-oral routes of administration, predominately by snorting. The ADAIR program is phase III ready. Vallon will begin conducting human abuse liability clinical trials of ADAIR in 1Q 2019. Time Monday, Feb 11 9:30AM - 9:45AM Location Gramercy Speakers David Baker Vallon Pharmaceuticals
9:45am Cox Biosciences LLC Clark Sullivan, Cox Biosciences LLC show more Session Description Curing Autism and Other Cerebral Folate Deficiencies. Cox is a phase III pharmaceutical company developing proprietary treatments for autism and other cerebral folate deficiencies. Cox has formulated levoleucovorin into a new liquid dosage format for its most advanced clinical program, and is building off of ground-breaking clinical research from Autism Speaks to move directly into phase III clinical development. The company was founded based on pioneering research at Downstate Medical Center and the discovery of an autoimmune response that inhibits folate transport to the brain in conditions such as autism. Several clinical studies and case reports have demonstrated that high dose reduced folates are capable of overcoming the folate block induced by this immune response, and overcoming language and other neurological deficits in autistic children. Optimum folate therapies are, however, not available to treat children when they are first diagnosed with autism. Cox’s first drug product, LL-One, is a liquid solution of levoleucovorin designed for optimum treatment of autistic children of all ages. The formulation and method of treatment are protected by two granted patents and two pending applications in the United States with corresponding protection around the world. Time Monday, Feb 11 9:45AM - 10:00AM Location Chelsea Speakers Clark Sullivan Cox Biosciences LLC CytoDyn Inc. Nader Pourhassan, CytoDyn Inc.; Richard Pestell, CytoDyn show more Session Description CytoDyn is a biotechnology company focused on the clinical development and potential commercialization of humanized monoclonal antibodies for the treatment and prevention of Human Immunodeficiency Virus (HIV) infection. The Company has one of the leading monoclonal antibodies under development for HIV infection, PRO 140, which has finished Phase 2 clinical trials with demonstrated antiviral activity in man. PRO 140 blocks the HIV co-receptor CCR5 on T-cells that prevents viral entry. Clinical trial results thus far indicate that PRO 140 does not negatively affect the normal immune functions that are mediated by CCR5. Results from six Phase 1 and Phase 2 human clinical trials have shown that PRO 140 can significantly reduce viral burden in people infected with HIV. A recent Phase 2b clinical trial has shown that PRO 140 can prevent viral escape in patients during 4-6 weeks of interruption from conventional drug therapy. Time Monday, Feb 11 9:45AM - 10:00AM Location Hudson/Empire Speakers Nader Pourhassan CytoDyn Inc. Richard Pestell CytoDyn Fibrocell Science, Inc. John Maslowski, Fibrocell Science show more Session Description Fibrocell is an autologous cell and gene therapy company translating personalized biologics into medical breakthroughs for diseases affecting the skin and connective tissue. Fibrocell’s most advanced product candidate, FCX-007, is the subject of a Phase 1/2 clinical trial for the treatment of RDEB. Fibrocell is also developing FCX-013, the Company’s clinical stage candidate for the treatment of moderate to severe localized scleroderma. Fibrocell’s gene therapy portfolio is being developed in collaboration with Intrexon Corporation (NASDAQ: XON), a leader in synthetic biology. Time Monday, Feb 11 9:45AM - 10:00AM Location Herald/Soho Speakers John Maslowski Fibrocell Science Immunic AG Daniel Vitt, Immunic AG show more Session Description Immunic is a specialist in selective oral drugs in immunology and focused on developing novel oral therapies with best-in-class potential for chronic inflammatory and autoimmune diseases. The company’s three development programs target inflammatory bowel diseases, multiple sclerosis, and psoriasis and include orally available, small molecule inhibitors of DHODH (IMU-838 program), an inverse agonist of RORγt (IMU-935 program), and IMU-856 (undisclosed novel target). Immunic’s lead development program, IMU-838, an orally available, small molecule inhibitor of DHODH, is currently in phase 2 clinical development for ulcerative colitis, with additional phase 2 trials in Crohn’s disease, multiple sclerosis, and primary sclerosing cholangitis planned for 2019. The company was founded in 2016 withheadquarters in Planegg-Martinsried, Germany. Immunic is privately held and supported by several renowned healthcare investors. Time Monday, Feb 11 9:45AM - 10:00AM Location Gramercy Speakers Daniel Vitt Immunic AG
10:00am Azitra Inc. Richard Andrews , Azitra, Inc. show more Session Description Azitra Inc. (Azitra) was established in 2014 to leverage the promise of the microbiome to address both skin disease (e.g. eczema, netherton’s, etc.) and adverse skin conditions (e.g. flaky, dry, rough skin). Skin disease and skin problems afflict millions of individuals worldwide. The Company believes that the microbiome holds the key to addressing these challenges and has assembled an experienced team of drug development specialists, microbiologists, biochemists, dermatologists and experts in industrial microbiology to meet its goals. Azitra has robust technical platform and 3 products entering clinical development in 2019. Time Monday, Feb 11 10:00AM - 10:15AM Location Chelsea Speakers Richard Andrews Azitra, Inc. Fireside Chat: Nancy Thornberry, CEO, Kallyope Nancy Thornberry , Kallyope; Yasmeen Rahimi, ROTH Capital Partners show more Session Description Fireside Chat: Nancy Thornberry, CEO, Kallyope Moderator: Yasmeen Rahimi, PhD, Managing Director and Senior Research Analyst, ROTH Capital Partners Time Monday, Feb 11 10:00AM - 10:55AM Location Majestic Complex Speakers Nancy Thornberry Kallyope Yasmeen Rahimi ROTH Capital Partners Infinity Pharmaceuticals Adelene Perkins, Infinity Pharamaceuticals show more Session Description We are an innovative biopharmaceutical company dedicated to developing novel medicines for people with cancer. We are focusing our efforts on advancing IPI-549, an orally administered, clinical-stage, immuno-oncology product candidate that selectively inhibits the enzyme phosphoinositide-3-kinase-gamma or PI3K-gamma. Time Monday, Feb 11 10:00AM - 10:15AM Location Gramercy Speakers Adelene Perkins Infinity Pharamaceuticals MeiraGTx Zandy Forbes, MeiraGTx show more Session Description MeiraGTx (NASDAQ:MGTX) is a vertically integrated, clinical stage gene therapy company with five programs in clinical development and a broad pipeline of preclinical and research programs. MeiraGTx has core capabilities in viral vector design and optimization and gene therapy manufacturing, as well as a potentially transformative gene regulation technology. Led by an experienced management team, MeiraGTx has taken a portfolio approach by licensing, acquiring and developing technologies that give depth across both product candidates and indications. MeiraGTx’s initial focus is on three distinct areas of unmet medical need: inherited retinal diseases, severe forms of xerostomia and neurodegenerative diseases. Though initially focusing on the eye, salivary gland and central nervous system, MeiraGTx intends to expand its focus in the future to develop additional gene therapy treatments for patients suffering from a range of serious diseases. Time Monday, Feb 11 10:00AM - 10:15AM Location Herald/Soho Speakers Zandy Forbes MeiraGTx Summit Therapeutics Glyn Edwards, Summit Therapeutics show more Session Description Summit Therapeutics is a leader in antibiotic innovation. Our new mechanism antibiotics are designed to become the new standards of care for the benefit of patients and create value for payors and healthcare providers. We are currently developing new mechanism antibiotics for infections caused by C. difficile. N. gonorrhoeae and ESKAPE pathogens and are using our proprietary Discuva Platform to expand our pipeline. Time Monday, Feb 11 10:00AM - 10:15AM Location Hudson/Empire Speakers Glyn Edwards Summit Therapeutics
10:15am Amplyx Pharmaceuticals Inc. Ciara Kennedy, Amplyx Pharmaceuticals, Inc. show more Session Description Amplyx was founded on a mission to save lives by developing a new class of antifungal medicine that would overcome the severe limitations of today’s therapeutic options. Amplyx is developing first-in-class products for the treatment of life-threatening infections, with a near-term focus on deadly fungal pathogens in vulnerable, immune-compromised patients. Amplyx's novel small-molecule therapy, APX001, is in clinical development to treat today’s deadliest fungal pathogens, including Candida auris, which the Centers for Disease Control has classified as a “catastrophic threat.” Intravenous and oral formulations of APX001 have been evaluated in a Phase 1 clinical program, with Phase 2 clinical trials in invasive fungal infections currently underway. Time Monday, Feb 11 10:15AM - 10:30AM Location Chelsea Speakers Ciara Kennedy Amplyx Pharmaceuticals, Inc. BioMarker Strategies, LLC Jerry Parrott, BioMarker Strategies, LLC show more Session Description Our mission is to enable use of live cells to personalize drug development and treatment selection for patients with solid tumor cancers. We have developed the patented SnapPath Cancer Diagnostics System - the only diagnostics system that can generate purified populations of live solid tumor cells from live, unfixed samples in an automated and standardized manner. SnapPath preserves molecular integrity of these living cells for ex vivo exposure to targeted therapies. This enables generation of PathMap Functional Signaling Profiles, which are highly predictive of individual solid tumor response to targeted therapies and combinations, because they are based on the dynamic signaling information available only from live cells. This functional information is required to understand initial response and the acquired resistance that too often develops in the treatment of cancer. Time Monday, Feb 11 10:15AM - 10:30AM Location Gramercy Speakers Jerry Parrott BioMarker Strategies, LLC Brainstorm Cell Therapeutics Chaim Lebovits, Brainstorm Cell Therapeutics show more Session Description BrainStorm Cell Therapeutics Inc. is a leading cellular therapy biotechnology company engaged in the development of first-of-its-kind adult stem cell therapies derived from autologous bone marrow cells for the treatment of neurodegenerative diseases. BrainStorm holds the rights to clinical development and commercialization of the NurOwn® technology platform through an exclusive, worldwide licensing agreement. NurOwn® has received Fast Track designation from the U.S. Food and Drug Administration (U.S. FDA) in ALS and has additionally been granted Orphan Status by the U.S. FDA and the European Medicines Agency (EMA). Time Monday, Feb 11 10:15AM - 10:30AM Location Herald/Soho Speakers Chaim Lebovits Brainstorm Cell Therapeutics Interpace Diagnostics Jack Stover, Interpace Diagnostics show more Session Description Interpace Diagnostics develops molecular diagnostic tests. You can be assured that all of the molecular tests we offer are driven by rigorous, validated science. We power the future of molecular diagnostics, delivering cutting-edge and much-needed mutational analysis that helps risk-stratify for thyroid, pancreatic, and other cancers to better inform treatment decisions. The unique molecular diagnostic tests we offer enable healthcare providers to avoid unnecessary surgeries and better assess the risk of cancer progression in their patients. Time Monday, Feb 11 10:15AM - 10:30AM Location Hudson/Empire Speakers Jack Stover Interpace Diagnostics
10:30am Cesca Therapeutics Inc. Chris Xu, Cesca Therapeutics Inc. show more Session Description Cesca Therapeutics develops, commercializes and markets a range of automated technologies for CAR-T and other cell-based therapies. Its device division, ThermoGenesis, provides a full suite of solutions for automated clinical biobanking, point-of-care applications, and automation for immuno-oncology. The Company has launched an automated, functionally-closed CAR-TXpress platform that addresses the critical unmet need for better cellular manufacturing and controls (CMC) for the emerging CAR-T immunotherapy market. Time Monday, Feb 11 10:30AM - 10:45AM Location Herald/Soho Speakers Chris Xu Cesca Therapeutics Inc. Espero BioPharma Lee Golden, Espero BioPharma show more Session Description Espero is focused on the development of late-stage drugs for unmet needs in thrombosis and cardiac rhythm control. Time Monday, Feb 11 10:30AM - 10:45AM Location Gramercy Speakers Lee Golden Espero BioPharma Femeda Ltd Andrew Tasker, Femeda Ltd show more Session Description Femeda is a UK based healthcare company focused on the development an innovative female health portfolio. With its specialism in female health, it has recently launched its first product Pelviva, a treatment for female urinary incontinence in the UK. Pelviva is a new and unique disposable, clinically effective, discreet and easy-to-use Pelvic Floor muscle re-trainer which treats urinary incontinence in women, demonstrating proven clinical success. Pelviva addresses THE biggest unmet need in female health. The unique development is the result of combining advanced research and patents from University of Manchester with Femeda product development expertise to deliver a product which meets core patient needs. A third of all women suffer from bladder leakage and Pelviva is the first product of its kind that is really easy to use and is clinically proven to treat bladder leaks. Pelviva is a pioneering medical device that will have a life-changing impact on women’s quality of life. Time Monday, Feb 11 10:30AM - 10:45AM Location Chelsea Speakers Andrew Tasker Femeda Ltd OncoCyte William Annett, OncoCyte show more Session Description OncoCyte is focused on the development and commercialization of novel, non-invasive blood (“liquid biopsy”) diagnostic tests for the early detection of cancer. Early detection of cancer can improve health outcomes, reduce the cost of care, and improve patients’ quality of life. Liquid biopsy diagnostic tests like those OncoCyte is developing may reduce the need for costlier and riskier diagnostic procedures such as invasive biopsy procedures. OncoCyte’s is focusing its efforts on developing DetermaVu™ as a non-invasive confirmatory diagnostic test for lung cancer. DetermaVu™ is being developed using proprietary sets of genetic and protein molecular markers to detect the presence of lung cancer. Time Monday, Feb 11 10:30AM - 10:45AM Location Hudson/Empire Speakers William Annett OncoCyte
10:45am CASI Pharmaceuticals, Inc. George Chi, CASI Pharmaceuticals, Inc. show more Session Description CASI Pharmaceuticals (NASDAQ: CASI) is a U.S.-based biopharmaceutical company dedicated to the development and delivery of high quality, cost-effective pharmaceutical products and innovative therapeutics to patients in the U.S., China and throughout the world. CASI’s product pipeline features three FDA-approved drugs in-licensed from Spectrum Pharmaceuticals, Inc. for China regional rights currently in various stages in the regulatory process for market approval in China. CASI also acquired a portfolio of 25 FDA-approved ANDAs and four pipeline ANDAs that are pending FDA approval. CASI is headquartered in Rockville, Maryland and has a wholly owned subsidiary and R&D operations in Beijing, China. Time Monday, Feb 11 10:45AM - 11:00AM Location Hudson/Empire Speakers George Chi CASI Pharmaceuticals, Inc. LifeEDIT Mark Moore, LifeEDIT show more Session Description LifeEDIT is a discovery-phase pharmaceutical development company that has identified the world’s largest and most diverse set of novel CRISPR gene editing systems. We are focused on the development and commercialization of human therapeutics using our unique CRISPR technologies. In a short time LifeEDIT has already developed the world’s largest and most diverse collection of CRISPR based nucleic acid editing proteins with multiple unique PAM recognition elements. These are being deployed to develop novel therapeutics in Immuno/oncology, Neurodegenerative Diseases and select hematopoietic disorders. Time Monday, Feb 11 10:45AM - 11:00AM Location Chelsea Speakers Mark Moore LifeEDIT SERAXIS INC. William Rust, SERAXIS INC. show more Session Description Seraxis, Inc. is a private biotech company with a mission of bringing cell replacement therapies to patients in need. With several products in development, its lead product, SR-01, is a novel combination product for type 1 diabetes with an IND application anticipated for 2020. SR-01 comprises of lab-grown human pancreatic islets encased within a novel bi-layered encapsulation device that protects the cells from cytotoxic alloimmune response. The Seraxis researchers are able to create functionally mature human insulin-secreting islets at a level and purity never before reported through a combination of a proprietary iPS cell line and a novel cell differentiation protocol. The retrievable encapsulation device employs biocompatible components and enables the cells to have close association to the host vasculature to physiologically regulate blood glucose. These proprietary technologies have been developed using GMP-processes and are characterized for clinical use. Time Monday, Feb 11 10:45AM - 11:00AM Location Gramercy Speakers William Rust SERAXIS INC. Synthetic Biologics, Inc. Steven Shallcross, syntheticbiologics.com show more Session Description Synthetic Biologics, Inc. (NYSE American: SYN) is a late-stage clinical company developing therapeutics that preserve the microbiome to protect and restore the health of patients. The Company's lead late-stage candidates are: (1) SYN-004 (ribaxamase) which is designed to protect the gut microbiome from the effects of certain commonly used intravenous (IV) beta-lactam antibiotics to prevent microbiome damage, C. difficile infection (CDI), overgrowth of pathogenic organisms and the emergence of antimicrobial resistance (AMR), and (2) SYN-010 which is intended to reduce the impact of methane producing organisms in the gut microbiome to treat an underlying cause of irritable bowel syndrome with constipation (IBS-C). The Company’s preclinical pursuits include an oral formulation of the enzyme intestinal alkaline phosphatase (IAP) to treat both local GI and systemic diseases as well as monoclonal antibody therapies for the prevention and treatment of pertussis. Time Monday, Feb 11 10:45AM - 11:00AM Location Herald/Soho Speakers Steven Shallcross syntheticbiologics.com
11:00am Advances in Neuromuscular Disease Treatments Lynn O'Connor Vos, Muscular Dystrophy Association (MDA); Murray Aitken, IQVIA; Jay A. Barth, Amicus Therapeutics; Amanda Haidet-Phillips, Muscular Dystrophy Association; Brian K. Kaspar, AveXis; Nader Yaghoubi, PathMaker Neurosystems Inc. show more Session Description After a long era without progress, recent years have seen regulatory approvals of treatments for Duchenne muscular dystrophy (DMD) and spinal muscular atrophy (SMA), among other neuromuscular disorders. In particular, SMA is the most common genetic disorder leading to infant death in the world, and now has a first therapy that can slow disease progression, giving patients and families more time together. This session will examine the exciting pipeline of additional neuromuscular disease therapies, and what the underlying science is teaching across disease indications, amid a complicated reimbursement context where the shortage of effective treatments bears significant healthcare costs over time. Moderator: Lynn O'Connor Vos, President and CEO, Muscular Dystrophy Association (MDA) Time Monday, Feb 11 11:00AM - 11:55AM Location Shubert Complex Speakers Lynn O'Connor Vos Muscular Dystrophy Association (MDA) Murray Aitken IQVIA Jay A. Barth Amicus Therapeutics Amanda Haidet-Phillips Muscular Dystrophy Association Brian K. Kaspar AveXis Nader Yaghoubi PathMaker Neurosystems Inc. Anima Biotech Inc. Yochi Slonim, Anima Biotech Inc. show more Session Description Anima Biotech is advancing Translation Control Therapeutics, the first platform for the discovery of small molecule drugs that specifically control mRNA translation as a new strategy against many diseases. With novel biology that monitors the translation of proteins and proprietary cloud-based software, we identify drug candidates that modulate a target protein’s production. We develop a pipeline across therapeutic areas and partner with Pharma for their targets including our $1B+ collaboration with Lilly for the discovery and development of translation inhibitors of several targets. Our approach was further validated with 5 granted patents, 14 peer reviewed publications and 17 scientific collaborations. Time Monday, Feb 11 11:00AM - 11:15AM Location Chelsea Speakers Yochi Slonim Anima Biotech Inc. Heat Biologics, Inc. Jeffrey Wolf, Heat Biologics, Inc. show more Session Description Heat Biologics is a biopharmaceutical company developing immunotherapies designed to activate a patient's immune system against cancer using of CD8+ ''Killer'' T-cells. Our T-Cell Activation Platform (TCAP) produces therapies designed to turn ''cold'' tumors ''hot'' and be administered in combination with checkpoint inhibitors and other immuno-modulators to increase their effectiveness. HS-110 is our first biologic product candidate in a series of proprietary immunotherapies designed to stimulate a patient's own T-cells to attack cancer. Our ComPACT™ technology is a dual-acting immunotherapy designed to deliver T-cell activation and co-stimulation in a single product. We are currently enrolling patients in our Phase 2 clinical trial for advanced non-small cell lung cancer, in combination with Bristol-Myers Squibb's nivolumab (Opdivo®). Pelican Therapeutics, a subsidiary of Heat, is focused on the development of co-stimulatory monoclonal antibody and fusion protein-based therapies. Time Monday, Feb 11 11:00AM - 11:15AM Location Hudson/Empire Speakers Jeffrey Wolf Heat Biologics, Inc. Landos Biopharma Josep Bassaganya-Riera, Landos Biopharma show more Session Description Landos Biopharma, Inc. is a clinical-stage biopharmaceutical company focused on the discovery and development of first-in-class oral therapeutics for patients with autoimmune diseases. Landos’ lead clinical asset, BT-11, is a novel, oral, gut-restricted small molecule targeting the Lanthionine Synthetase C-Like 2 (LANCL2) pathway in the gastrointestinal tract for treatment of inflammatory bowel disease (IBD), has completed Phase 1 clinical testing and initiated Phase 2 clinical trials for Crohn's and ulcerative colitis. Landos is advancing a robust pipeline of new compounds for other autoimmune diseases, some of which will advance to IND in 2019. Time Monday, Feb 11 11:00AM - 11:15AM Location Gramercy Speakers Josep Bassaganya-Riera Landos Biopharma Salarius Pharmaceuticals David Arthur, Salarius Pharmaceuticals show more Session Description Salarius Pharmaceuticals is a clinical-stage biotechnology company focused on developing effective cancer treatments for patients who need them most. Salarius was formed in 2011 after licensing the technology related to their lead compound from the University of Utah. Following formation, Salarius focused on identifying and progressing their lead therapeutic candidate to IND approval. As part of this process, Salarius was able to generate impressive preclinical data that was instrumental in being selected to receive an $18.7M grant from the Cancer Prevention & Research Institute of Texas (CPRIT). Salarius received IND activation from the FDA in March 2018. In 3Q2018 Salarius initiated a Phase 1 clinical trial in Ewing sarcoma and will be opening an additional Phase 1 trial in Advanced Solid Tumors in 1H2019. Time Monday, Feb 11 11:00AM - 11:15AM Location Herald/Soho Speakers David Arthur Salarius Pharmaceuticals
11:15am Abcuro, Inc. Stefano Gulla, Abcuro show more Session Description Abcuro is developing a new generation of immunomodulatory therapeutics for treating both autoimmunity and cancer. The company uses proprietary analysis of transcriptome data from human disease to target key compartments of the immune system. Abcuro was launched in 2016 and to date it has raised over $10M to advance two functionally differentiated therapeutic programs that target KLRG1, an inhibitory receptor on T and NK cells. Abcuro’s autoimmunity program is under development for inclusion body myositis (IBM), primary biliary cholangitis (PBC) and other T cell driven autoimmunity indications including rare forms of leukemia. Abcuro's immuno-oncology program is being developed for solid tumors via a biomarker based indication selection strategy. Both programs have completed candidate selection and are now in pre-clinical development. Time Monday, Feb 11 11:15AM - 11:30AM Location Gramercy Speakers Stefano Gulla Abcuro Catalyst Pharmaceuticals, Inc. Steven Miller, Catalyst Pharmaceuticals, Inc. ; Patrick McEnany, Catalyst Pharma show more Session Description Catalyst Pharmaceuticals is dedicated to advancing therapies targeting rare neuromuscular and neurological diseases, including Lambert-Eaton Myasthenic Syndrome (LEMS), Congenital Myasthenic Syndromes (CMS), MuSK-Myasthenia Gravis, and Spinal Muscular Atrophy. Catalyst is an integrated pharmaceutical company with the ability to develop and market new products, including recently approved Firdapse. Time Monday, Feb 11 11:15AM - 11:30AM Location Herald/Soho Speakers Steven Miller Catalyst Pharmaceuticals, Inc. Patrick McEnany Catalyst Pharma Exicure David Giljohann, Exicure show more Session Description Exicure is a clinical stage biotechnology company developing a new class of immunomodulatory and gene regulating drugs against validated targets. Exicure’s proprietary 3-dimensional, spherical nucleic acid (SNA™) architecture unlocks the potential of therapeutic oligonucleotides in a wide range of cells and tissues. Exicure’s lead programs address inflammatory diseases, genetic disorders and oncology. Exicure is based outside of Chicago, Ill. Learn more at www.exicuretx.com. Time Monday, Feb 11 11:15AM - 11:30AM Location Hudson/Empire Speakers David Giljohann Exicure Sirnaomics,Inc Patrick(Yang) Lu, Sirnaomics,Inc show more Session Description Sirnaomics is a clinical stage biopharmaceutical company leveraging an outstanding level of knowledge and experience in RNA interference (RNAi) technology to forge a path to high-value creation through discovery and development of therapeutics for human disorders with unmet medical needs. Time Monday, Feb 11 11:15AM - 11:30AM Location Chelsea Speakers Patrick(Yang) Lu Sirnaomics,Inc
12:00pm Fireside Chat: Geoff Meyerson, Managing Partner and Co-Founder, Locust Walk Geoff Meyerson, Locust Walk; Sapna Srivastava show more Session Description Geoff Meyerson is Managing Partner and Co-founder of Locust Walk. He brings a track record of success in investment banking, venture capital and licensing where he has closed >50 transactions of all varieties including playing a role in a majority of Locust Walk’s closed transactions. Prior to Locust Walk, Geoff worked in business development at a venture-backed biotech company, Zelos Therapeutics, where he was on the management team and negotiated a drug delivery collaboration with Aegis Therapeutics. Before Zelos, he worked as a venture capitalist at SR One, the venture capital subsidiary of GlaxoSmithKline, where he closed eight financings in the US and Europe. His first and most active investment where co-lead the due diligence was for Algeta, a Norwegian radiopharma company with a product for bone metastasis. They concluded a $3B sale to Bayer, received FDA approval for their product Xofigo, and achieved a >20x return to investors. Geoff also worked at MedImmune in business development helping with their small molecule strategy, which resulted in three transactions, and where he also assisted with two other transactions. He got his start as an investment banker in UBS’ Global Healthcare Investment Banking Group, where he closed 12 financing and M&A transactions with a variety of life science companies. He holds an MBA in Health Care Management from the Wharton School, a Master of Biotechnology from the University of Pennsylvania, and a BS in Economics from Duke University. Geoff founded and is President of an exclusive relationship building non-profit organization called, BioBreak. BioBreak has grown to six regions internationally (Boston, San Francisco, Philadelphia, New Jersey, UK and Germany) and has a membership base of >3,000 senior life science operating and investing executives. He is a member of the Young Presidents Organization New England Chapter and is on the Board of Overseers for Beth Israel Deaconess Medical Center. Geoff also has served two terms on the board of the Wharton Health Care Management Alumni Association. He was named by Wharton Magazine (The Wharton School’s alumni magazine) and the Philadelphia Business Journal to their 40 Under 40 list for 2017 and 2010, respectively. Geoff lives in Needham, MA with his wife Sasha, three children, Victoria, Jacob and Michelle, and mini schnauzer, Duke. He enjoys golf, skiing, tennis and shooting in his free time. He is a registered rep with FINRA holding his Series 79, 63, and 24 licenses. Moderator: Sapna Srivastava, PhD Time Monday, Feb 11 12:00PM - 12:55PM Location Majestic Complex Speakers Geoff Meyerson Locust Walk Sapna Srivastava
1:15pm Bioasis Technologies Inc. Mark Day, Bioasis Technologies Inc. show more Session Description Bioasis Technologies Inc. is a biopharmaceutical company developing the xB³ ™ platform, a proprietary technology for the delivery of therapeutics across the blood-brain barrier (BBB) and the treatment of CNS disorders in areas of high unmet medical need, including brain cancers and neurodegenerative diseases. Bioasis maintains headquarters in Guilford, Conn., United States. Bioasis trades on the TSX Venture Exchange under the symbol “BTI” and on the OTCQB under the symbol “BIOAF.” Time Monday, Feb 11 1:15PM - 1:30PM Location Hudson/Empire Speakers Mark Day Bioasis Technologies Inc. Fulcrum Therapeutics Robert Gould, Fulcrum Therapeutics show more Session Description Fulcrum is focused on small molecule therapeutics for diseases of gene misregulation. The company is creating therapeutics for a range of genetic disorders including: FSHD, Sickle Cell Disease, as well as other neuromuscular diseases. We plan to file our first IND in FSHD in 1H2019. Fulcrum is founded on a precision medicine approach that integrates human genetics across the spectrum of drug discovery. Our integrated drug discovery approach has been productive by: • focusing on the genetic root cause of the disease; • creating accurate in vitro models using patient derived cellular models; • using a combination of a curated gene regulation compound library, a CRISPR/Cas9 library and computational biology; • and, developing genetic biomarkers to support an efficient path to the patient Time Monday, Feb 11 1:15PM - 1:30PM Location Chelsea Speakers Robert Gould Fulcrum Therapeutics Onconova Therapeutics, Inc. Steven Fruchtman, Onconova Therapeutics, Inc. show more Session Description Onconova Therapeutics, Inc. is a Phase 3-stage biopharmaceutical company focused on discovering and developing novel small molecule drug candidates to treat cancer, with a primary focus on Myelodysplastic Syndromes (MDS). Using a proprietary chemistry platform, Onconova has created a pipeline of targeted agents designed to work against specific cellular pathways that are important in cancer cells. Rigosertib, Onconova's lead candidate, is a proprietary Phase 3 small molecule agent, which the Company believes blocks cellular signaling by targeting RAS effector pathways. Advanced clinical trials with Rigosertib are aimed at the unmet medical needs of patients with MDS. There has not been an FDA-approved treatment for higher-risk MDS in over a decade. Both IV and oral rigosertib are protected by issued patents worldwide (earliest expiry 2026) and have been granted orphan drug designation for MDS in the US, Europe, and Japan. Very promising data from the Phase 2 combination trial of o Time Monday, Feb 11 1:15PM - 1:30PM Location Gramercy Speakers Steven Fruchtman Onconova Therapeutics, Inc. Rockwell Medical,Inc Stuart Paul , Rockwell Medical, Inc show more Session Description Rockwell Medical, Inc (Rockwell Medical), is a specialty pharmaceutical company targeting anemia management in end-stage renal disease (ESRD), chronic kidney disease (CKD) and other anemia related disease areas. Triferic is the ONLY FDA APPROVED product indicated for iron replacement and maintenance of hemoglobin in hemodialysis patients. In addition, Rockwell is an established manufacturer, supplier and leader in delivering high-quality hemodialysis concentrates / dialysates (used to maintain human life by removing toxins and replacing critical nutrients in the dialysis patient's bloodstream, to dialysis providers and distributors in the U.S. and abroad. Rockwell has two Triferic formulation: Triferic dialysate, to be launched in the first half of 2019 and Triferic IV for first half 2019 NDA submission. Rockwell Medical owns global Triferic rights and is seeking partnership ex-USA, including Europe and Japan. We have an established China partner with anticipated 2019 China NDA. Time Monday, Feb 11 1:15PM - 1:30PM Location Herald/Soho Speakers Stuart Paul Rockwell Medical, Inc
1:30pm Alnylam Pharmaceuticals Josh Brodsky, Alnylam Pharmaceuticals, Inc. show more Session Description Alnylam is leading the translation of RNA interference (RNAi) into a whole new class of innovative medicines with the potential to transform the lives of patients who have limited or inadequate treatment options. Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically validated approach for the treatment of a wide range of debilitating diseases with high unmet medical need. Alnylam was founded in 2002 on a bold vision to turn scientific possibility into reality, which is now marked by its robust discovery platform and deep pipeline of investigational medicines, including multiple programs in late-stage clinical development. Time Monday, Feb 11 1:30PM - 1:45PM Location Herald/Soho Speakers Josh Brodsky Alnylam Pharmaceuticals, Inc. Anavex Life Sciences Christopher U. Missling, Anavex Life Sciences Corp. show more Session Description Anavex utilizes precision genetic medicine to treat severe and devastating neurological disorders and is focusing on rare diseases with no available therapy (Rett syndrome) and high risk CNS patient populations (Parkinson's Disease, Alzheimer's Disease) Time Monday, Feb 11 1:30PM - 1:45PM Location Gramercy Speakers Christopher U. Missling Anavex Life Sciences Corp. AUM Biosciences Vishal Doshi, B.Pharm, MSc, AUM Biosciences show more Session Description Founded by 2 ex-Quintiles (Now IQVIA) and industry veterans, AUM Biosciences is an oncology-focused biotechnology company headquartered in Singapore that develops innovative and affordable medicines for patients in Asia and globally. The founders and the management team have over 75 years of combined oncology drug development experience, and track record of developing and executing various virtual drug development models in the Industry. AUM’s pipeline includes a novel, small molecule targeted therapy which has the potential to address high unmet medical needs in various solid and liquid tumours. Additionally, it has an undisclosed pipeline of additional pre-clinical and phase I ready programs. Pipeline focus is on small molecules targeted therapies with a mandate of clear biomarker driven strategies for every asset in the pipeline. AUM harnesses the power of precision medicine and digital technologies to accelerate the drug development timelines to unlock the value of innovation. Time Monday, Feb 11 1:30PM - 1:45PM Location Chelsea Speakers Vishal Doshi, B.Pharm, MSc AUM Biosciences Navigating Payers’ Expectations for Value-Based Agreements Yasmeen Rahimi, ROTH Capital Partners; R. John Glasspool, Massachusetts Institute of Technology; Roger Longman, Real Endpoints; Ron Philip , Spark Therapeutics show more Session Description As biomedical innovation has developed advanced therapeutic modalities and prospective cures for ailments that previously had no comparable courses of treatment, the challenge of crafting sustainable reimbursement models has inspired a variety of value-based agreements. Departure from traditional dosage-based models makes it difficult for therapy developers, payers, patients, and investors to plan for a new therapy’s rollout and breadth of patient access. This session will explore the early lessons from current value-based agreements to explain the payers’ expectations for future models of reimbursing treatments based on outcomes versus delivery. Moderator: Yasmeen Rahimi, Managing Director and Senior Research Analyst Biotechnology, ROTH Capital Partners Time Monday, Feb 11 1:30PM - 2:25PM Location Shubert Complex Speakers Yasmeen Rahimi ROTH Capital Partners R. John Glasspool Massachusetts Institute of Technology Roger Longman Real Endpoints Ron Philip Spark Therapeutics Otsuka Pharmaceutical Ron Newbold, Otsuka America Pharmaceutical, Inc. show more Session Description Otsuka is among the top 5 pharma companies from Japan, with strong global drug development and commercial capabilities in CNS disorders, kidney diseases and oncology. Otsuka aspires to be your strong partner in the CNS and renal space. Our antipsychotic franchise around aripiprazoe and brexpiprazole (Abilify, Rexulti) has made us a global leader in mental health. Based on tolvaptan's recent FDA approval for polycycstic kidney disease (Jynarque for ADPKD), we are now aspiring to become a leader in the renal disease space. We are now searching for opportunities to complement our pipeline with innovative therapeutic solutions to clear unmet needs in psychiatric, neurological and renal disorders - regardless of modality (small molecule, biologic, allogeneic cell therapy, gene therapy or digital therapeutics). Time Monday, Feb 11 1:30PM - 1:45PM Location Hudson/Empire Speakers Ron Newbold Otsuka America Pharmaceutical, Inc.
1:45pm Carmell Therapeutics Randy Hubbell , Carmell Therapeutics show more Session Description 1. Carmell is addressing the burden of healing with its proprietary Plasma-based Bioactive Materials (PBM) technology a. Carmell is focused on improving patients’ quality of life and reducing costs to the healthcare system b. Clear FDA regulatory pathway supported by 16+ patent estate c. Led by strong and seasoned management team backed with category experience in developing and commercializing drug/biologic/drug combination products at leading international pharmaceutical and device companies. Carmell is employing a rigorous FDA pathway with a Biologic License Application, giving the company the credibility of an FDA-vetted process Defined BLA pathway provides opportunity with hospital Value Analysis Committees (VACs), who seek proof of efficacy and safety, as well as with potential partners who seek differentiation Carmell’s novel plasma-based technology is uniquely suited for multiple, high-value indications Time Monday, Feb 11 1:45PM - 2:00PM Location Chelsea Speakers Randy Hubbell Carmell Therapeutics Children's Tumor Foundation Annette Bakker, Children's Tumor Foundation show more Session Description The Children’s Tumor Foundation is a 501(c)(3) not-for-profit organization dedicated to funding and driving innovative research that will result in effective treatments for the millions of people worldwide living with neurofibromatosis (NF), a term for three distinct disorders: NF1, NF2, and schwannomatosis. NF causes tumors to grow on nerves throughout the body and may lead to blindness, deafness, bone abnormalities, disfigurement, learning disabilities, disabling pain, and cancer. NF affects 1 in every 3,000 births across all populations equally. There is no cure yet – but the Children’s Tumor Foundation mission of driving research, expanding knowledge, and advancing care for the NF community fosters our vision of one day ending NF. For more information, please visit www.ctf.org. Time Monday, Feb 11 1:45PM - 2:00PM Location Herald/Soho Speakers Annette Bakker Children's Tumor Foundation Innovate Biopharmaceuticals, Inc. Christopher Prior, Innovate Biopharmaceuticals, Inc show more Session Description Innovate is a clinical stage biotechnology company focused on developing novel medicines for autoimmune and inflammatory diseases with unmet needs. Our pipeline includes drug candidates for celiac disease, NASH, Crohn's, and ulcerative colitis. Innovate is led by a strong management team with extensive drug development experience and a history of bringing novel therapeutics to market. All global rights to the products, which are backed by more than 150 patents worldwide, are owned by the company. Time Monday, Feb 11 1:45PM - 2:00PM Location Gramercy Speakers Christopher Prior Innovate Biopharmaceuticals, Inc VistaGen Therapeutics, Inc. Shawn Singh, VistaGen Therapeutics, Inc. show more Session Description VistaGen Therapeutics, Inc. (Nasdaq: VTGN) is a clinical-stage biopharmaceutical company developing new generation medicines for depression, social anxiety, and other CNS diseases and disorders with high unmet need. Lead candidate, AV-101, is currently in Phase 2 for the treatment of major depressive disorder. PH94B and PH10 are being prepared for Phase 3 for the treatment of social anxiety disorder and MDD, respectively. Time Monday, Feb 11 1:45PM - 2:00PM Location Hudson/Empire Speakers Shawn Singh VistaGen Therapeutics, Inc.
2:00pm American Gene Technologies Jeff Galvin, American Gene Technologies show more Session Description AGT is an emerging gene and cell therapeutics company with a proprietary lentiviral platform capable of broad applications including: large and orphan indications, infectious disease, immune-oncology, and monogenic disorders. AGT expects to take its patented, lead candidate for an HIV cure into the clinic in 2019. It has pioneered a novel immuno-oncology approach of stimulating gamma-delta (γδ) T cells to attack a variety of cancers. Four key patents in AGT’s novel immuno-oncology approach have been granted. AGT has a diverse portfolio of patent filings surrounding key tools and components in viral vectors, gene therapy, and regenerative medicine. Time Monday, Feb 11 2:00PM - 2:15PM Location Chelsea Speakers Jeff Galvin American Gene Technologies CEL-SCI Corporation Geert Kersten, CEL-SCI Corporation show more Session Description CEL-SCI is a pioneer in cancer immunotherapy. We believe that boosting a patient's immune system while it is still intact should provide the greatest possible impact on survival. Consequently, in its Phase 3 clinical study CEL-SCI is treating patients who are newly diagnosed with advanced primary head and neck cancer with Multikine BEFORE they receive surgery, radiation and/or chemotherapy. The Phase 3 trial is the largest ever in head and neck cancer with 928 patients enrolled. With the last patient enrolled in the study in September 2016, final data read-out based on 298 events is expected in the near future. Multikine has received Orphan Drug designation from the FDA. Advanced primary head and neck cancer represents an unmet medical need with the last FDA approval about 60 years ago. CEL-SCI's LEAPS technology is being developed as a therapeutic vaccine for rheumatoid arthritis and is supported by grants from the NIH. The Company has operations in Vienna, VA, and Baltimore, MD. Time Monday, Feb 11 2:00PM - 2:15PM Location Herald/Soho Speakers Geert Kersten CEL-SCI Corporation CytoSorbents Phillip Chan, CytoSorbents show more Session Description CytoSorbents Corporation (NASDAQ: CTSO) is a critical care immunotherapy company commercializing its CytoSorb blood purification technology in 53 countries worldwide to treat deadly inflammation in life-threatening conditions in the ICU (such as sepsis, trauma, burn injury, liver disease, lung injury, pancreatitis and many others) and cardiac surgery. CytoSorb has garnered more than 51,000 human treatments cumulatively as a "razorblade disposable" that is compatible with existing blood pumps in the hospital, with $19.1M in trailing 12-month sales and healthy 72% blended gross margins. 4 major partners: Fresenius Medical Care, Terumo Cardiovascular, Biocon and Dr. Reddys. $25M+ in U.S. grant funding. Time Monday, Feb 11 2:00PM - 2:15PM Location Gramercy Speakers Phillip Chan CytoSorbents Zynerba Pharmaceuticals Armando Anido, Zynerba Pharmaceuticals show more Session Description Zynerba Pharmaceuticals is dedicated to developing next-generation transdermally-delivered cannabinoid therapeutics for patients affected by rare and near-rare neuropsychiatric conditions. Often, these diseases have few, if any, treatment options and can leave patients and their families feeling helpless and alone. Zynerba is committed to developing breakthrough therapies to transform the quality of lives of patients and their families as they battle these conditions. Our goal is to advance the science of therapeutic cannabinoids, and to improve the lives of patients battling severe neuropsychiatric conditions including Fragile X syndrome (FXS), adult refractory focal epilepsy, and developmental and epileptic encephalopathies (DEE). Our approach is to utilize our pharmaceutically-manufactured, penetration-enhanced transdermal cannabinoid product candidate to modulate pathways in the central nervous system that, when disrupted, cause certain neurological diseases. Time Monday, Feb 11 2:00PM - 2:15PM Location Hudson/Empire Speakers Armando Anido Zynerba Pharmaceuticals
2:15pm Actinium Pharmaceuticals, Inc. Sandesh Seth, Actinium Pharmaceuticals, Inc. show more Session Description Actinium Pharmaceuticals Inc. is focused on improving patient access and outcomes to cellular therapies such as bone marrow transplant (BMT) and CAR-T with its proprietary, chemotherapy free or sparing, targeted conditioning technology. Actinium is the only company with a multi-disease, multi-target, drug development pipeline focused on targeted conditioning. Its targeted conditioning technology is enabled by ARC’s or Antibody Radiation Conjugates that combine the targeting ability of monoclonal antibodies with the cell killing ability of radioisotopes. Actinium’s pipeline of clinical-stage targeted conditioning ARCs target the antigens CD45 and CD33 for patients with a broad range of hematologic malignancies including acute myeloid leukemia (AML), myelodysplastic syndrome (MDS) and multiple myeloma (MM), acute lymphoblastic leukemia (ALL), Hodgkin’s lymphoma and Non-Hodgkin’s lymphoma. Time Monday, Feb 11 2:15PM - 2:30PM Location Herald/Soho Speakers Sandesh Seth Actinium Pharmaceuticals, Inc. Arch Therapeutics, Inc. Terrence Norchi, Arch Therapeutics, Inc. show more Session Description Arch Therapeutics, Inc. is a biotechnology company developing a novel approach to stop bleeding (hemostasis), control leaking (sealant) and manage wounds during surgery, trauma and interventional care. Arch is developing products based on an innovative self-assembling barrier technology platform with the goal of making care faster and safer for patients. Arch's development stage product candidates include the AC5™ Topical Gel and the AC5™ Surgical Hemostatic Device. Time Monday, Feb 11 2:15PM - 2:30PM Location Gramercy Speakers Terrence Norchi Arch Therapeutics, Inc. Newron Pharmaceuticals SpA Stefan Weber, Newron Pharmaceuticals SpA show more Session Description Newron is a biopharmaceutical company focused on the development of novel therapies for patients with diseases of the central and peripheral nervous system. The company is headquartered in near Milan, Italy. Xadago®/safinamide has received marketing authorization for the treatment of Parkinson’s disease in the EU, Switzerland and the USA, and is commercialized by its Partner Zambon. US WorldMeds holds the commercialization rights in the USA. Meiji Seika has the rights to develop and commercialize the compound in Japan and other key Asian territories. In addition to Xadago®/safinamide for PD, Newron has a strong pipeline of promising treatments for rare disease patients at various stages of clinical development, including Sarizotan for patients with Rett syndrome and ralfinamide for patients with specific rare pain indications. Newron is also developing Evenamide as the potential first add-on therapy for the treatment of patients with positive symptoms of schizophrenia. Time Monday, Feb 11 2:15PM - 2:30PM Location Hudson/Empire Speakers Stefan Weber Newron Pharmaceuticals SpA Tarveda Therapeutics, Inc Drew Fromkin, Tarveda Therapeutics show more Session Description Tarveda Therapeutics, Inc. discovers and develops Pentarins®, a new class of potent and selective miniature drug conjugates with enhanced targeting capabilities for the treatment of a wide range of solid tumor cancers. Tarveda’s lead Pentarin drug candidate, PEN-221, is a miniature drug conjugate that targets the somatostatin receptor 2 (SSTR2) for treatment of patients with neuroendocrine, small cell lung, prostate, and other cancers that express SSTR2. PEN-221 comprises a highly selective peptide for SSTR2 conjugated to the potent cytotoxic payload, DM1, through a tuned cleavable linker. Tarveda is also advancing its Pentarin HSP90 drug conjugate platform with lead drug candidate PEN-866, which is a miniature drug conjugate that selectively binds to the intracellular target, Heat Shock Protein 90 (HSP90), and is linked to the payload SN-38, a potent topoisomerase I inhibitor. Time Monday, Feb 11 2:15PM - 2:30PM Location Chelsea Speakers Drew Fromkin Tarveda Therapeutics
2:30pm Fireside Chat: Jinzi Wu, PhD, Founder, Chairman, and CEO, Ascletis Pharma Inc. Jinzi J. Wu, Ascletis Pharma Inc.; Mark Tang, Good Health Capital, New York show more Session Description Fireside Chat: Jinzi Wu, PhD, Founder, Chairman and CEO, Ascletis Pharma Inc. Moderator: Mark Tang, Managing Director, Good Health Capital, New York Time Monday, Feb 11 2:30PM - 3:25PM Location Majestic Complex Speakers Jinzi J. Wu Ascletis Pharma Inc. Mark Tang Good Health Capital, New York Greenwich LifeSciences Snehal Patel, Greenwich LifeSciences show more Session Description Greenwich LifeSciences (GLS) is a private biopharmaceutical company located in Houston, TX that is developing GP2, a novel peptide immunotherapy, planning to commence a Phase III trial in 2019, pending funding. In a just completed Phase IIb study, no recurrences have been observed in the Her2/neu 3+ adjuvant setting for up to 5 years if the patient is fully vaccinated. GP2 is a 9 amino acid transmembrane peptide of the HER2/neu protein, a cell surface receptor protein that is expressed in a variety of common cancers including 75% of breast cancers. Upcoming Phase III Clinical Trial – FDA has approved the Phase III trial protocol and manufacturing has commenced. Substantial Unmet Need – Following breast cancer surgery, a Her2/neu 3+ patient receives Herceptin in the first year and then hopes that their breast cancer will not recur, with the odds of recurrence slowly decreasing over the first 5 years. Herceptin reduces recurrence rates from 25% to 12%, leaving us with the unmet need and potential to use GP2 to fully address the 50% of recurring patients who do not respond to Herceptin. Compelling Clinical Phase IIb Data – In a just completed randomized single-blinded multi-centered (15 sites) Phase IIb clinical trial of 180 HLA-A02 patients of which 101 patients are Her2/neu (3+) over expressor patients, GP2 displayed outstanding efficacy. At up to 5 years of follow up, there were 0% cancer recurrences in the 51 Her2/neu (3+) patients treated with GP2-GMCSF-Herceptin, when fully vaccinated with the 6 primary injection series, versus a 12% cancer recurrence rate in the 50 patient placebo arm treated with GMCSF-Herceptin (p = 0.0268). Potent Immune Response – GP2 immunotherapy elucidated a potent immune response in HLA-A02 patients as measured by a local skin test and immunological assays. Further, booster injections given every six months prolonged the immune response thereby providing longer term protection. Excellent Safety – In the Phase IIb and three Phase I clinical trials where 138 patients have been treated, there were no reported serious adverse events related to GP2 immunotherapy. Time Monday, Feb 11 2:30PM - 2:45PM Location Gramercy Speakers Snehal Patel Greenwich LifeSciences Oncolytics Biotech Inc. Matt Coffey, Oncolytics Biotech Inc. show more Session Description Oncolytics is a biotechnology company developing pelareorep, an intravenously delivered immuno-oncolytic virus. The compound induces selective tumor lysis and promotes an inflamed tumor phenotype -- turning "cold" tumors "hot" -- through innate and adaptive immune responses to treat a variety of cancers. Oncolytics' clinical development program emphasizes three pillars: chemotherapy combinations to trigger selective tumor lysis and immuno-therapy and immune modulator (IMiD) combinations to produce innate and adaptive immune responses. Oncolytics is currently conducting and planning additional studies in combination with checkpoint inhibitors and targeted and IMiD therapies in solid and hematological malignancies, as it prepares for a phase 3 registration study in metastatic breast cancer. Time Monday, Feb 11 2:30PM - 2:45PM Location Herald/Soho Speakers Matt Coffey Oncolytics Biotech Inc. PAION AG Wolfgang Söhngen, PAION AG show more Session Description PAION AG is a publicly listed specialty pharmaceutical company developing and aiming to commercialize innovative drugs for out-patient and hospital-based sedation, anesthesia and critical care services. PAION’s lead compound is remimazolam, an intravenous, ultra-short-acting and controllable benzodiazepine sedative/anesthetic drug candidate for which PAION has completed the clinical development for use in procedural sedation in the U.S. Outside the U.S., PAION is currently focused on the development of remimazolam for general anesthesia. Development of remimazolam for intensive care unit (ICU) sedation is also part of the longer-term life-cycle plan for remimazolam. Time Monday, Feb 11 2:30PM - 2:45PM Location Chelsea Speakers Wolfgang Söhngen PAION AG Tonix Pharmaceuticals Holding Corp. Seth Lederman, Tonix Pharmaceuticals show more Session Description Tonix is a clinical-stage biopharmaceutical company focused on discovering and developing pharmaceutical products to treat serious neuropsychiatric conditions and biological products to improve biodefense through potential medical counter-measures. Tonix is developing Tonmya, which is in Phase 3 development and has been granted Breakthrough Therapy designation, as a bedtime treatment for PTSD. Tonix is also developing TNX-102 SL as a bedtime treatment for agitation in Alzheimer’s disease under a separate IND to support a Phase 2, potential pivotal, efficacy study and has been designated a Fast Track development program by the FDA. TNX-601 (tianeptine oxalate) is in the pre-IND application stage, also for the treatment of PTSD but by a unique mechanism and designed for daytime dosing. Tonix’s lead biologic candidate, TNX-801, is a potential smallpox-preventing vaccine based on a live synthetic version of horsepox virus, currently in the pre-IND application stage. Time Monday, Feb 11 2:30PM - 2:45PM Location Hudson/Empire Speakers Seth Lederman Tonix Pharmaceuticals
2:45pm IMV Inc. Frederic Ors, IMV Inc. show more Session Description IMV Inc. is a clinical stage biopharmaceutical company dedicated to making immunotherapy more effective, more broadly applicable, and more widely available to people facing cancer and other serious diseases. IMV is pioneering a new class of immunotherapies based on the Company’s proprietary drug delivery platform. This patented technology leverages a novel mechanism of action that enables the programming of immune cells in vivo, which are aimed at generating powerful new synthetic therapeutic capabilities. IMV’s lead candidate, DPX-Survivac, is a T cell-activating immunotherapy that combines the utility of the platform with a target: survivin. IMV is currently assessing DPX-Survivac as a monotherapy in advanced ovarian cancer, as well as a combination therapy in multiple clinical studies with Incyte and Merck. Connect at www.imv-inc.com. Time Monday, Feb 11 2:45PM - 3:00PM Location Herald/Soho Speakers Frederic Ors IMV Inc. InCarda Therapeutics Grace Colón, InCarda Therapeutics show more Session Description InCarda Therapeutics is a privately-held, clinical-stage biopharmaceutical company pioneering a novel approach of treating cardiovascular conditions by the inhalation route. The lead product under development, InRhythm, is an inhaled therapy to treat paroxysmal atrial fibrillation (PAF), a widespread atrial arrhythmia. The company has raised over $50M and is currently conducting a Phase 2 trial to treat acute episodes of AF in Europe. If approved, InRhythm will be the only treatment available for patients to treat and quickly resolve their episodes of atrial fibrillation anytime, anywhere they happen to be. Time Monday, Feb 11 2:45PM - 3:00PM Location Gramercy Speakers Grace Colón InCarda Therapeutics Lumme Inc Akshaya Shanmugam, Lumme Inc show more Session Description Lumme is a digital therapeutics company developing a continuous health monitoring and intervention platform. Using our technology platform, we have clinically demonstrated significant patient behavior change during the treatment period. We have received $1.8M in government grants to use our technology platform to develop products that target the top 3 addictions grappling our nation today: smoking, alcohol, and diet. Our 1st product is a smoking cessation platform that has demonstrated the highest clinically reported quit rates of any commercially available product or service. This work is an outcome of research conducted at UMass Amherst, co-developed with the Yale Tobacco Treatment Clinic, and funded by the National Cancer Institute. Time Monday, Feb 11 2:45PM - 3:00PM Location Chelsea Speakers Akshaya Shanmugam Lumme Inc ORYZON CARLOS BUESA-ARJOL, ORYZON show more Session Description Oryzon (ORY.MC) is a public company listed in the Madrid and part of the IBEX-Small Cap Index. It is now one of the most liquid companies in this segment of the market. Oryzon is clinical stage Biopharmaceutical Company that, with its Epigenetic Platform, develops first-in-class drugs for diseases with strong unmet medical need such as cancer and neurodegenerative diseases. Oryzon’s pipeline includes inhibitors against Histone demethylases, with a strong specialization in KDM1A (LSD1). Oryzon is leading epigenetics in CNS with Vafidemstat, a brain penetrant dual LSD1-MAOB inhibitor being explored on three different Phase IIA trials: in mil-moderate AD; in a basket trial in aggressivity covering three different psychiatric indications like like ASD, ADHD and BLP and a third one in RR/SP-MS. Oryzon has also an oncology program, iadademstat, this is a highly selective LSD1 inhibitor being explored in two Phase IIA trials, one in AML and the other in SCLC in a biomarker guided trial. Time Monday, Feb 11 2:45PM - 3:00PM Location Hudson/Empire Speakers CARLOS BUESA-ARJOL ORYZON
3:00pm BioXcel Therapeutics Vimal Mehta, BioXcel Therapeutics show more Session Description BioXcel Therapeutics is a clinical stage biopharmaceutical company focused on drug development in neuroscience and immuno-oncology utilizing artificial intelligence. Currently the company has two lead clinical development programs: o BXCL501, a sublingual thin film formulation designed for acute treatment of agitation resulting from neurological and psychiatric disorders • Reported positive results from Phase 1b trial of intravenously-dosed Dexmedetomidine supporting BXCL501 development in schizophrenia • Data from Phase 1b PK/PD safety study in mild probable Alzheimer’s patients expected in second half of 2018 o BXCL701, an immuno-oncology agent designed for treatment of a rare form of prostate cancer and for treatment of pancreatic cancer • Announced FDA acceptance of IND for treatment emergent neuroendocrine prostate cancer; study expected to be initiated in fourth quarter of 2018 • Entered clinical partnership with Nektar for study in pancreatic cancer Time Monday, Feb 11 3:00PM - 3:15PM Location Hudson/Empire Speakers Vimal Mehta BioXcel Therapeutics Genes2me LLP "Transforming Preventive health and Women & Child health in 1.2B people" Neeraj Gupta, Genes2me LLP show more Session Description Genes2me is a genomics driven advanced diagnostic company creating a benchmark in the field of personalised healthcare and genetic screening with a focused segments into Preventive health, Oncology, Mother & Child health and Pharmacogenomics. The translational approach to enhance global health even in resource limited areas by being cost effective, technologically driven, with a 360 degree overview of complete diagnostic proposition under one test. Keeping innovation at the forefront and an access to clinically relevant samples having history, has backed Genes2me with insights into complex genetic diseases to ideate, create and evaluate to develop the best in class preventive, diagnostics and genetic based products and transform personalised Health. Having state of the art facility established with all leading genomics and clinical diagnostics technologies installed and team of more than 75 experts in assay development, validation, scientific team, business development. Time Monday, Feb 11 3:00PM - 3:15PM Location Gramercy Speakers Neeraj Gupta Genes2me LLP Immunicum AB Carlos de Sousa, Immunicum AB show more Session Description Immunicum is establishing a unique immuno-oncology approach through the development of allogeneic, off-the-shelf cell-based therapies. The company’s lead product ilixadencel, consisting of pro-inflammatory allogeneic dendritic cells, has the potential to improve survival outcomes and quality of life by priming the patient’s own immune system to fight cancer. Ilixadencel is currently in a Phase 2 controlled study in renal cell carcinoma (kidney cancer), has completed Phase 1/2 studies in kidney cancer and liver cancer, and has initiated a Phase 1b/2 combination study with checkpoint inhibitors in Head and Neck (HNSCC), Lung (NSCLC) and gastric cancer by the end of 2018. Immunicum is a public company at the Nasdaq Stockholm and has recently raised USD 39 million. Time Monday, Feb 11 3:00PM - 3:15PM Location Herald/Soho Speakers Carlos de Sousa Immunicum AB Q BioMed Inc Denis Corin, Q BioMed Inc show more Session Description Q BioMed, Inc. is a biomedical acceleration and development company. We are focused on licensing and acquiring biomedical assets across the healthcare spectrum. Q BioMed is dedicated to providing these target assets the strategic resources, developmental support and expansion capital they need to meet their developmental potential so that they can provide products to patients in need. Time Monday, Feb 11 3:00PM - 3:15PM Location Chelsea Speakers Denis Corin Q BioMed Inc
3:15pm Concarlo Holdings, LLC Arikha Moses, Concarlo Holdings, LLC show more Session Description CONCARLO Holdings, LLC (Concarlo) is a precision medicine oncology company developing targeted solutions to diagnose and treat metastatic solid tumor cancers. Experienced team with four decades of biotechnology and commercialization experience. Robust IP portfolio. Seeking to raise $7 MM in 2 trenches to fund IND-enabling studies, reach FIH Phase I & complete a corporate partnership. Approximately 37,000 U.S. women will be diagnosed with metastatic ER/PR+, Her2- breast cancer this year, and despite recent advances, the median survival for these patients remains at ~2–3 y. Concarlo is developing a new class of drugs as well as a complementary diagnostic that target p27, a key on-off switch in many metastatic tumor types. Concarlo's therapeutics exhibit more potency against sensitive and resistant tumor types in vivo than CDK4 inhibitors, which are currently first-line therapy for metastatic breast cancer patients. Time Monday, Feb 11 3:15PM - 3:30PM Location Chelsea Speakers Arikha Moses Concarlo Holdings, LLC Eloxx Pharmaceuticals Robert Ward, Eloxx Pharmaceuticals show more Session Description COMMITTED TO TREATING Rare and Ultra-Rare Genetic Diseases Eloxx Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company developing novel RNA-modulating drug candidates (designed to be eukaryotic ribosomal selective glycosides) that are designed to treat rare and ultra-rare premature stop codon diseases. Premature stop codons are point mutations that disrupt protein synthesis from messenger RNA. As a consequence, patients with premature stop codon diseases have reduced or eliminated protein production from the mutation bearing allele accounting for some of the most severe phenotypes in these genetic diseases. These premature stop codons have been identified in over 1,800 rare and ultra-rare diseases. Read-through therapeutic development is focused on extending mRNA half-life and increasing protein synthesis by enabling the cytoplasmic ribosome to read through premature stop codons to produce full-length proteins. Eloxx’s lead investigational product candidate, ELX-02. Time Monday, Feb 11 3:15PM - 3:30PM Location Hudson/Empire Speakers Robert Ward Eloxx Pharmaceuticals Mirum Pharmaceuticals Christopher Peetz, Mirum Pharmaceuticals, Inc. show more Session Description Mirum is a clinical stage company focused on difficult to treat liver diseases. Mirum's lead program, maralixibat, is planned to initiate Phase 3 in Alagille Syndrome and progressive familial intrahepatic cholestasis (PFIC) in 2019. Time Monday, Feb 11 3:15PM - 3:30PM Location Gramercy Speakers Christopher Peetz Mirum Pharmaceuticals, Inc. OxSonics Limited Colin Story, OxSonics Limited show more Session Description As a global leader in ultrasound-based drug delivery, OxSonics’ misssion is to maximise the potential of existing and emerging cancer therapies for the benefit of cancer patients world-wide. OxSonics Therapeutics proprietary platform, SonoTran, has been designed by clinicians for clinicians. Time Monday, Feb 11 3:15PM - 3:30PM Location Herald/Soho Speakers Colin Story OxSonics Limited
3:30pm Attacking Biological Mechanisms of Aging to Extend Healthspan Susan Peschin, Alliance for Aging Research; Philippe Calais, CohBar, Inc.; Grace Colón, InCarda Therapeutics; Evripidis Gavathiotis, Albert Einstein College of Medicine;Selphagy Therapeutics, Inc.; Charles S. Ryan, Neurotrope; Salim Syed, Mizuho show more Session Description A growing number of biopharma companies have responded to the emerging medical consensus that many of the individual ailments experienced by older people have underlying, connective biological causes. These companies are developing drugs in areas such as neurodegeneration, macular degeneration, arthritis, and similar highly prevalent diseases by pursuing novel mechanisms versus existing treatments. This session will discuss the frontier of science in extending the years of healthy aging and in which indications patients might first see progressive new therapies in clinical trials. Moderator: Susan Peschin, MHS, President and Chief Executive Officer, Alliance for Aging Research Time Monday, Feb 11 3:30PM - 4:25PM Location Shubert Complex Speakers Susan Peschin Alliance for Aging Research Philippe Calais CohBar, Inc. Grace Colón InCarda Therapeutics Evripidis Gavathiotis Albert Einstein College of Medicine;Selphagy Therapeutics, Inc. Charles S. Ryan Neurotrope Salim Syed Mizuho Kleo Pharmaceuticals Doug Manion, Kleo Pharmaceuticals show more Session Description Kleo Pharmaceuticals is a unique immuno-oncology company developing next-generation bispecific compounds designed to emulate or enhance the activity of biologics. Similar to complex biologic drugs, Kleo’s compounds recruit the immune system to destroy cancer cells, but unlike biologics, Kleo’s compounds are smaller and more versatile, leading to potentially improved safety and efficacy. They are also much faster and less costly to design and produce, particularly against novel targets. The company is advancing several drug candidates based on its proprietary technology platforms, all of which are modular in design and enable rapid generation of novel immunotherapies that can be optimized against certain cancers, or enhance the properties of existing immunotherapies. These include Antibody Recruiting Molecules (ARMs), Synthetic Antibody Mimics (SyAMs) and Monoclonal Antibody Therapy Enhancers (MATEs). For more information visit http://kleopharmaceuticals.com. Time Monday, Feb 11 3:30PM - 3:45PM Location Herald/Soho Speakers Doug Manion Kleo Pharmaceuticals Scynexis Inc. Marco Taglietti, Scynexis show more Session Description SCYNEXIS, Inc. (NASDAQ:SCYX) is a biotechnology company committed to positively impacting the lives of patients suffering from difficult-to-treat and often life-threatening infections by developing innovative therapies. The SCYNEXIS team has extensive experience in the life sciences industry, having discovered and developed more than 30 innovative medicines over a broad range of therapeutic areas. The Company's lead product candidate, ibrexafungerp (formerly known as SCY-078), is a novel IV/oral antifungal agent in Phase 3 clinical and preclinical development for the treatment of multiple serious and life-threatening invasive fungal infections caused by Candida and Aspergillus species. For more information, visit www.scynexis.com. Time Monday, Feb 11 3:30PM - 3:45PM Location Chelsea Speakers Marco Taglietti Scynexis Trovagene Oncology Thomas Adams, Trovagene Oncology show more Session Description Trovagene is a clinical-stage oncology therapeutics company, developing drugs that target mitosis (cell division) and integrating a biomarker approach to identifying patient most likely to respond to treatment. Currently, 3 clinical trials are underway with lead drug candidate, Onvansertib, a first-in-class, 3rd generation, oral and highly-selective Polo-like Kinase 1(PLK1) inhibitor: a Phase 1b/2 trial of Onvansertib in combination with standard-of-care chemo in Acute Myeloid Leukemia (AML); a Phase 2 trial of Onvansertib in combination with Zytiga in metastatic Castration-Resistant Prostate Cancer (mCRPC; and a Phase 1b/2 trial of Onvansertib in combination with FOLFIRI and Avastin in metastatic Colorectal Cancer (mCRC). Time Monday, Feb 11 3:30PM - 3:45PM Location Hudson/Empire Speakers Thomas Adams Trovagene Oncology VenatoRx Pharmaceuticals Tony Meehan, VenatoRx Pharmaceuticals show more Session Description VenatoRx is a private pharmaceutical company that is focused on the discovery and development of novel anti-infectives to treat multi-drug-resistant (MDR) bacterial infections and hard-to-treat viral infections. VenatoRx’s lead product, VNRX-5133, is an injectable broad-spectrum beta-lactamase inhibitor (BLI) that directly inhibits all four Ambler classes of beta-lactamases. In addition, VenatoRx has a broad pipeline of preclinical programs, including a broad-spectrum orally bioavailable BLI, a novel class of Penicillin-Binding Protein (PBP) inhibitors that are impervious to beta-lactamase-driven resistance, and novel antiviral agents. Time Monday, Feb 11 3:30PM - 3:45PM Location Gramercy Speakers Tony Meehan VenatoRx Pharmaceuticals
3:45pm Marker Therapeutics, Inc. Anthony Kim, Marker Therapeutics, Inc. show more Session Description Marker Therapeutics, Inc is a clinical-stage immuno-oncology company specializing in the development of next-generation T cell-based immunotherapies for the treatment of hematological malignancies and solid tumor indications. Marker’s cell therapy technology is based on the selective expansion of non-engineered, tumor-specific T cells that recognize tumor associated antigens (i.e. tumor targets) and kill tumor cells expressing those targets. Once infused into patients, this population of T cells attacks multiple tumor targets and acts to activate the patient’s immune system to produce broad spectrum anti-tumor activity. Because Marker does not genetically engineer its T cells, when compared to current engineered CAR-T and TCR-based approaches, its products (i) are significantly less expensive and easier to manufacture, (ii) appear to be markedly less toxic, and (iii) are associated with meaningful clinical benefit. Time Monday, Feb 11 3:45PM - 4:00PM Location Hudson/Empire Speakers Anthony Kim Marker Therapeutics, Inc. RubrYc Therapeutics Isaac Bright, RubrYc Therapeutics, Inc. show more Session Description RubrYc Therapeutics is a discovery-stage therapeutic antibody business focused on addressing unmet needs in Oncology and Autoimmune Diseases. The Company was founded in 2017 and launched in April, 2018 with a $10M Series A Preferred financing. RubrYc has developed the Interface Discovery Engine – the operating system the Company employs, leveraging machine learning to realize critical efficiency gains in drug discovery. Team RubrYc maintains intense focus on advancing its proprietary pipeline programs, while engaging in target-specific Platform Partnerships with industry incumbents. The Company will complete platform feasibility, demonstrate non-GLP PoC with its molecules, and raise Series B in 2019. Time Monday, Feb 11 3:45PM - 4:00PM Location Gramercy Speakers Isaac Bright RubrYc Therapeutics, Inc. Sensei Biotherapeutics John Celebi, Sensei Biotherapeutics, Inc. show more Session Description Sensei Biotherapeutics is an innovator in the field of next-generation cancer immunotherapies, including cancer vaccines and T-cell therapies. We are pioneering the development of precision therapeutics that target ASPH, a powerful, uniquely human, tumor-specific antigen to treat cancer. SNS-301, our lead program, is a first-in-class cancer immunotherapy and has successfully completed a Phase 1 clinical study with plans for further clinical development in hematological malignancies and solid tumors. Time Monday, Feb 11 3:45PM - 4:00PM Location Herald/Soho Speakers John Celebi Sensei Biotherapeutics, Inc.
4:30pm Market Outlook—Leveraging Market Corrections for M&A or IPOs Sara Michelmore, MacDougall; Katherine Andersen, Silicon Valley Bank (SVB); Noël Brown, Cantor Fitzgerald; Andrew Gitkin , Piper Jaffray; Geoffrey Goodman, Wells Fargo Securities; Philip Ross, J.P. Morgan; Jennifer Sheng, Citi; Yaron Werber , Cowen show more Session Description After a multi-year expansion of company formation and in biopharma valuations, 2018 was a year of heightened market volatility. Investors’ eagerness to support new modalities enabled more than 50 biotech IPOs, but with much less mature clinical development progress than traditionally demonstrated. More than half those companies are trading below their IPO prices, however, complicating conditions for the next new offering. As interest rate worries, international trade disputes, and regulatory reimbursement uncertainty have increased, biopharma stock indexes have seen significant corrections. With historically high valuations experiencing sudden dips, M&A deal-making seems to have reignited with notable, multi-billion dollar deals in recent weeks. This session will assemble market experts to discuss what forces continue to shape performance within the sector and what opportunities the market correction can reveal for investors and companies seeking resources to deliver the next wave of innovative therapies for patients. Moderator: Sara Michelmore, Senior Vice President, Investor Relations, MacDougall Time Monday, Feb 11 4:30PM - 5:45PM Location Shubert Complex Speakers Sara Michelmore MacDougall Katherine Andersen Silicon Valley Bank (SVB) Noël Brown Cantor Fitzgerald Andrew Gitkin Piper Jaffray Geoffrey Goodman Wells Fargo Securities Philip Ross J.P. Morgan Jennifer Sheng Citi Yaron Werber Cowen
Tuesday, February 12
9:00am New Treatments for Diabetes and Obesity: Innovations for Chronic Diseases Jason Butler, JMP Securities; Vered Caplan, Orgenesis; Steve Daly, Adocia; Dara Schuster, T1D Exchange; David Thomas, Biotechnology Innovation Organization (BIO) show more Session Description BIO’s latest report on highly prevalent chronic diseases shows a slowdown in overall investment for new treatments for diabetes and obesity (available at http://www.bio.org/iareports). However, there remain signs of hope in the early-stage pipeline. This session will detail the drug pipeline, which research approaches are convincing skeptical investors, as well as opportunities for overcoming high-cost clinical trials and reimbursement barriers. Moderator: Jason Butler, PhD, Managing Director and Senior Analyst, JMP Securities Time Tuesday, Feb 12 9:00AM - 9:55AM Location Shubert Complex Speakers Jason Butler JMP Securities Vered Caplan Orgenesis Steve Daly Adocia Dara Schuster T1D Exchange David Thomas Biotechnology Innovation Organization (BIO)
9:15am Entera Bio Phillip Schwartz, Entera Bio show more Session Description Company Profile Entera Bio is a product-focused biotechnology company specializing in the oral delivery of large molecules and biologics. Entera Bio is currently conducting clinical trials on its proprietary technology which allows oral delivery of large molecules and biologics while enhancing their absorption in the GI tract. Entera Bio's technology addresses the two major problems of oral drug delivery of large molecule APIs (active pharmaceutical ingredients). Most large molecules cannot be administered orally as they are not readily absorbed by the gastrointestinal tract and are rapidly degraded by a myriad of enzymes and acids in the gastrointestinal tract. Entera’s Proprietary Technologies Act Synergistically to Transport and Protect Large Molecules. Time Tuesday, Feb 12 9:15AM - 9:30AM Location Chelsea Speakers Phillip Schwartz Entera Bio Second Genome, Inc Karim Dabbagh, Second Genome show more Session Description Second Genome has assembled a one-of-a-kind drug discovery platform that integrates microbiome and host biology for the identification of novel targets and drugs. This approach is based on understanding microbial community composition and its function in healthy and diseased conditions. Our powerful data analysis techniques enable the identification of microbes and microbial biomarkers that influence a given disease state. Combined with our expertise in drug development, our team has built the foundation for the rapid discovery of new therapeutic options for patients in a numerous microbiome-related diseases. Second Genome’s microbiome modulating discovery platform allows us to generate and evaluate small molecules, peptide biologic and bacterial strains that modulate microbe-microbe and microbe-human interactions. By integrating our in vivo models with our in silico analyses we are able to understand how these compounds improve health and eliminate disease. Time Tuesday, Feb 12 9:15AM - 9:30AM Location Gramercy Speakers Karim Dabbagh Second Genome Trevena, Inc Mark A. Demitrack, Trevena show more Session Description Trevena, Inc. is a biopharmaceutical company focused on the development and commercialization of new and innovative treatment options for patients in pain. The Company has three novel and differentiated investigational drug candidates, including IV oliceridine, for the management of moderate to severe acute pain in hospitals, TRV250 for the treatment of acute migraine, and TRV734 for pain and/or management of opioid dependence. In its preclinical programs, Trevena is evaluating a set of novel S1P receptor modulators that may offer a new, non-opioid approach to managing chronic pain. Time Tuesday, Feb 12 9:15AM - 9:30AM Location Hudson/Empire Speakers Mark A. Demitrack Trevena Verseon Adityo Prakash, Verseon show more Session Description Verseon is a clinical stage company that uses a computer-driven drug discovery platform to design, atom by atom, new drug candidates that are unlikely to be found by conventional methods. Our current drug programs include precision oral anticoagulants, oral drugs for diabetic macular edema and hereditary angioedema, and next-generation chemotherapy agents. As part of our mission to develop disruptive technologies and products that advance global health, we are rethinking how drug development is funded. Security token offerings (STOs) provide a new, attractive way for companies to raise money and share their successes with a global investor community. However, the major obstacle to widespread adoption of securities transactions n the blockchain has been the enforcement of regulations of all countries involved. Through our subsidiary BlockRules Ltd., we are building the key missing piece of technology that allows the transition of securities issuance and trading on the blockchain. Time Tuesday, Feb 12 9:15AM - 9:30AM Location Herald/Soho Speakers Adityo Prakash Verseon
9:30am Aspect Biosystems Tamer Mohamed, Aspect Biosystems show more Session Description Aspect Biosystems Ltd. is a privately held biotechnology company operating at the leading edge of 3D bioprinting and tissue engineering. The company’s proprietary microfluidic 3D bioprinting technology is enabling advances in understanding fundamental biology, disease research, development of novel therapeutics, and regenerative medicine. In addition to its internal programs, Aspect is focused on strategically partnering with pharmaceutical and biotechnology companies, as well as academic researchers, to enable the creation of living, human tissues for medical research, therapeutic discovery, and regenerative medicine products. Time Tuesday, Feb 12 9:30AM - 9:45AM Location Chelsea Speakers Tamer Mohamed Aspect Biosystems Caladrius Biosciences, Inc David Mazzo, Caladrius Biosciences, Inc. show more Session Description Caladrius is a clinical-stage biopharmaceutical company committed to the development of innovative products that have the potential to restore the health of people with chronic illnesses. Our current product candidates include two clinical-stage treatments for cardiovascular diseases based on our CD34 cell therapy platform: CLBS12, recipient of SAKIGAKE designation, in Phase 2 testing in Japan and eligible for early conditional approval for the treatment of critical limb ischemia; and CLBS14, in Phase 2 testing for the treatment of coronary microvascular dysfunction and in late-stage clinical development for refractory angina for which it has received RMAT designation. Caladrius’ autoimmune product candidate in Phase 2 testing, CLBS03, is an ex vivo expanded polyclonal T regulatory cell therapy for the treatment of recent-onset type 1 diabetes. CLBS03 has been awarded Fast Track and Orphan designations by the FDA. Time Tuesday, Feb 12 9:30AM - 9:45AM Location Herald/Soho Speakers David Mazzo Caladrius Biosciences, Inc. SELLAS Life Sciences Group, Inc. Angelos Stergiou, SELLAS Life Sciences Group show more Session Description SELLAS Life Sciences is a late-stage biopharmaceutical company focused on the development of novel cancer immunotherapies for a broad range of cancer indications. Time Tuesday, Feb 12 9:30AM - 9:45AM Location Hudson/Empire Speakers Angelos Stergiou SELLAS Life Sciences Group Urigen Pharmaceuticals, Inc. Dan Vickery, Urigen Pharmaceuticals, Inc. show more Session Description Urigen Pharmaceuticals, Inc. is a private clinical stage urology focused specialty pharmaceutical company. Our team is focused on developing innovative products to amerliorate the cause and symtoms associated with Urological ailments, specifically Interstitial Cystitis / Bladder Pain Syndrome (IC/BPS). Our leading phase 2 program is URG101 for treating acute pain associated with IC/BPS flares. Urigen is raising $15M to fund End of Phase 2 meeting with FDA and IPO/exit activities. Time Tuesday, Feb 12 9:30AM - 9:45AM Location Gramercy Speakers Dan Vickery Urigen Pharmaceuticals, Inc.
9:45am AbMax Biotechnology CO., LTD Le Sun, AbMax Biotechnology CO., LTD show more Session Description AbMax Biotechnology Co., Ltd. was established in 2006 and now is a high-tech enterprise and training base for the national life science and technical personnel. AbMax has the world’s most advanced one-stop technology platform for antibodies and kits researching, which has the monoclonal antibody developed in an average of 46 days with a high success rate of 90%. AbMax, as a member of biomedical field in the capital scientific and technological condition platform, is the training base of BeijingNormalUniversity and also joins the Alliance of Bio-Box Outsourcing, China (ABO). AbMax also undertook a dozen government major research projects and established the largest pathogen antibody library for emerging/outbreaking infectious diseases in China. Time Tuesday, Feb 12 9:45AM - 10:00AM Location Chelsea Speakers Le Sun AbMax Biotechnology CO., LTD Daré Bioscience Sabrina Johnson, Daré Bioscience, Inc. show more Session Description Daré Bioscience is a clinical-stage biopharmaceutical company committed to the advancement of innovative products for women’s sexual health, vaginal health, fertility, and contraception. The company’s mission is to identify, develop and bring to market a portfolio of novel, differentiated therapies that expand treatment options, improve outcomes and facilitate convenience for women in the areas of contraception, vaginal health, sexual health, and fertility. Daré’s product portfolio includes potential first-in-class candidates in clinical development: Ovaprene®, a non-hormonal, monthly contraceptive vaginal ring; and Sildenafil Cream, 3.6%, a potential treatment for female sexual arousal disorder utilizing the active ingredient in Viagra®, as well as a proprietary solution-to-gel formulation of clindamycin to treat bacterial vaginosis via a single application, DARE-BV1. Time Tuesday, Feb 12 9:45AM - 10:00AM Location Herald/Soho Speakers Sabrina Johnson Daré Bioscience, Inc. E-Scape Bio Julie Anne Smith, E-Scape Bio show more Session Description E-Scape Bio is a biopharmaceutical company developing novel small-molecule therapeutics for patients with inherited forms of neurodegenerative diseases. The company's drug development pipeline includes small molecules targeting known genetic drivers of CNS disorders including an S1P5 agonist for the treatment of CNS lysosomal storage disorders, a Parkinson's disease program targeting LRRK2, and an Alzheimer's disease program targeting ApoE4. Time Tuesday, Feb 12 9:45AM - 10:00AM Location Gramercy Speakers Julie Anne Smith E-Scape Bio Propanc Biopharma Inc. James Nathanielsz, Propanc Biopharma Inc. show more Session Description Propanc Biopharma is a biopharmaceutical company developing new cancer treatments for solid tumors. The company has developed a formulation of anti-cancer compounds designed to control or prevent tumors from recurring and spreading throughout the body by using proenzymes, which are inactive precursors of enzymes. Time Tuesday, Feb 12 9:45AM - 10:00AM Location Hudson/Empire Speakers James Nathanielsz Propanc Biopharma Inc.
10:00am CohBar, Inc. Philippe Calais, CohBar, Inc. show more Session Description CohBar is a clinical stage biotechnology company whose mission is to increase healthy lifespan by developing treatments for the underlying metabolic dysfunction driving the diseases of aging including NASH, obesity, cancer, Type 2 diabetes, and cardiovascular and neurodegenerative diseases. Time Tuesday, Feb 12 10:00AM - 10:15AM Location Herald/Soho Speakers Philippe Calais CohBar, Inc. Cue Biopharma Daniel Passeri, Cue Biopharma show more Session Description Cue Biopharma is dedicated to developing a robust pipeline of novel immunotherapies focused on oncology, autoimmunity and chronic infections. We are building upon fundamental immunological principles to directly engage the immune system with an unprecedented level of specificity and sophistication. Our Immuno-STAT (Selective Targeting and Alteration of T cells) Biologics platform is designed to directly engage with and modulate the activity of antigen-specific T cells in the patient’s body, offering the potential for enhanced efficacy with minimal toxicity. Time Tuesday, Feb 12 10:00AM - 10:15AM Location Hudson/Empire Speakers Daniel Passeri Cue Biopharma EIP Pharma, Inc. John Alam, EIP Pharma show more Session Description EIP Pharma Inc. is a clinical-stage biopharmaceutical company focused on the development & commercialization of innovative drug treatments for Alzheimer’s disease (AD) and other central neurologic disorders. Our lead product candidate, neflamapimod, is a novel oral small molecule drug that inhibits the intra-cellular enzyme p38 mitogen-activated protein kinase alpha. The company was launched in 2014 by industry R&D leaders and has assembled a highly experienced executive management team, board of directors and scientific advisory board to execute on its mission. Based on positive preclinical and phase 2a clinical trial results, in March 2018 the company initiated a Phase 2b randomized double-blind placebo-controlled clinical study of neflamapimod in subjects with early AD, which is called REVERSE-SD; the results of which are expected in the second half of 2019. The REVERSE-SD study is fully funded through a $20.5M Series B financing round completed in April 2018. Time Tuesday, Feb 12 10:00AM - 10:15AM Location Gramercy Speakers John Alam EIP Pharma Fireside Chat: William Slattery, Partner, Deerfield; and Annette Grimaldi, Managing Director & Head of Life Sciences Investment Banking, BMO Capital Markets William Slattery, Deerfield; Annette Grimaldi, BMO Capital Markets show more Session Description Fireside Chat: William Slattery, Partner, Deerfield Moderator: Annette Grimaldi, Managing Director & Head of Life Sciences Investment Banking, BMO Capital Markets Time Tuesday, Feb 12 10:00AM - 10:55AM Location Majestic Complex Speakers William Slattery Deerfield Annette Grimaldi BMO Capital Markets Precision NanoSystems James Taylor, Precision NanoSystems show more Session Description Precision NanoSystems Inc. (“PNI”) provides comprehensive solutions for the development and manufacture of nanoparticle based precision medicines. This includes in the areas of gene therapy, gene editing and CRISPR, immunooncology, vaccines, rare disease and others. PNI’s core technology includes its NanoAssemblr microfluidic-based continuous flow platform for nanomedicine development and manufacture, and its GenVoy nanoparticle delivery technology for nucleic acid delivery. PNI provides its technology to leading pharmaceutical companies, biotechnology companies, and academic institutions in over 20 countries worldwide. Please find more information at https://www.precisionnanosystems.com/. Time Tuesday, Feb 12 10:00AM - 10:15AM Location Chelsea Speakers James Taylor Precision NanoSystems
10:15am Congenica Ltd David Atkins, Congenica show more Session Description Congenica aspires to fully enable the routine use of genomic medicine to support the diagnosis and characterisation of disease like rare disease and cancer using complex genomic data. The reduction DNA sequencing cost has made analysis of a patient’s genome a reality and the challenge now is the data interpretation. Congenica a provider of a diagnostic decision support software platform, Sapientia, which enable clinicians to interrogate the human genome to identify disease-causing variants. A diagnosis removes the fear of the unknown, allowing a patient’s family to deal with the known and make informed decisions. There is also the potential for healthcare savings by avoiding unnecessary appointments, tests and interventions. HQ in Cambridge, UK founded by the Wellcome Trust Sanger Institute and leaders from the NHS. Congenica was recently named as the genomic software partner for the NHS. Congenica is currently expanding into markets in US, China and beyond. Time Tuesday, Feb 12 10:15AM - 10:30AM Location Herald/Soho Speakers David Atkins Congenica Cyclacel Pharmaceuticals, Inc. Spiro Rombotis, Cyclacel Pharmaceuticals, Inc. show more Session Description Cyclacel Pharmaceuticals is a clinical-stage biopharmaceutical company developing innovative medicines targeting cell cycle, transcriptional regulation and DNA damage response molecules critical in oncogenesis/cancer progression. Cyclacel's lead transcriptional regulation program is evaluating a CDK inhibitor, CYC065 in CLL patients in combination with venetoclax. The DNA damage response program is evaluating a sequential regimen of sapacitabine and seliciclib, a CDK inhibitor, in patients with BRCA positive solid cancers. The safety/tolerability profile of CYC140, a Polo-like-kinase 1 inhibitor is being assessed in advanced cancer patients. Cyclacel envisions building a healthcare business focusing on an unmet medical need for patients in the hematology-oncology area. Time Tuesday, Feb 12 10:15AM - 10:30AM Location Hudson/Empire Speakers Spiro Rombotis Cyclacel Pharmaceuticals, Inc. Nemus Biosciences Brian Murphy, Nemus Bioscience show more Session Description NEMUS Bioscience Inc. is a biopharmaceutical company focused on discovering, developing and commercializing new chemical entities from a class of chemically diverse compounds called cannabinoids. These molecules display pleiotropic activity by virtue of selectively binding to two types of cannabinoid receptors (CB1 and CB2 receptors) located throughout multiple organ systems in the body. NEMUS Bioscience Inc. is developing novel and proprietary classes of product candidates that are designed to improve therapeutic options through enhanced chemical engineering that allow drug candidates to have more predictable bioavailability and pharmacokinetics leading to optimized efficacy and safety. Time Tuesday, Feb 12 10:15AM - 10:30AM Location Gramercy Speakers Brian Murphy Nemus Bioscience OcuMedic, Inc. Keith D. Ignotz, OcuMedic, Inc. show more Session Description OcuMedic’s solution is continuous dosing using contact lens with visual correction or a plano contact bandage replacing eye drops providing more efficacious and convenient therapy DECREASING COMPLICATIONS and reducing HEALTH CARE COSTS. Time Tuesday, Feb 12 10:15AM - 10:30AM Location Chelsea Speakers Keith D. Ignotz OcuMedic, Inc.
10:30am Agile Therapeutics, Inc. Al Altomari, Agile Therapeutics, Inc. show more Session Description Agile Therapeutics, Inc. is a women's healthcare company dedicated to fulfilling the unmet health needs of today's women. The Company's lead product candidate, Twirla, is a once-weekly prescription contraceptive patch that is at the end of Phase 3 clinical development. Time Tuesday, Feb 12 10:30AM - 10:45AM Location Gramercy Speakers Al Altomari Agile Therapeutics, Inc. Curis, Inc Jim Dentzer, Curis, Inc show more Session Description Curis is a biotechnology company focused on the development and commercialization of innovative cancer therapeutics including fimepinostat, which is being investigated in clinical studies in patients with DLBCL and solid tumors. Curis also collaborates with Aurigene in the areas of immuno-oncology and precision oncology. As part of this collaboration, Curis has exclusive licenses to oral small molecule antagonists of immune checkpoints including the VISTA/PDL1 antagonist CA-170, the TIM3/PDL1 antagonist CA-327, and the IRAK4 kinase inhibitor, CA-4948. CA-170 is undergoing Phase 1 testing in patients with advanced solid tumors and lymphomas, and Phase 2 testing in India conducted by Aurigene. CA-4948 is currently in a Phase 1 trial in patients with NHL. Curis is also party to a collaboration with Genentech, a member of the Roche Group, under which Genentech and Roche are commercializing Erivedge® for the treatment of advanced basal cell carcinoma. Time Tuesday, Feb 12 10:30AM - 10:45AM Location Hudson/Empire Speakers Jim Dentzer Curis, Inc Lung Therapeutics Brian Windsor, Lung Therapeutics show more Session Description Lung Therapeutics (LTI) is a biopharmaceutical company developing therapeutics for orphan, unmet needs in lung injury and disease. The Company’s lead drug, LTI-01, is a pro-enzyme for the treatment of Loculated Pleural Effusion (LPE), a severe consequence of pneumonia for which there is no approved pharmacotherapy. In a completed ex-US clinical trial in patients, LTI-01 was demonstrated safe and effective in clearing LPE, resulting in avoidance of surgery and decreased hospitalization. There are no approved drugs for the treatment of LPE. LTI has received US and EU Orphan Drug Designation for LTI-01. The Company’s second drug, LTI-03, is in development for the treatment of idiopathic pulmonary fibrosis (IPF). LTI-03 is a novel peptide addressing a unique mechanism which both promotes epithelial cell survival as well as destruction of fibroblasts. LTI-03 has been proven safe in GLP tox and is ready to enter clinical trials. LTI is raising funding for clinical trials of both drugs. Time Tuesday, Feb 12 10:30AM - 10:45AM Location Chelsea Speakers Brian Windsor Lung Therapeutics Oyster Point Pharma, Inc. Jeffrey Nau, Oyster Point Pharma, Inc. show more Session Description Oyster Point is a clinical-stage pharmaceutical company leveraging neuroscience to develop and commercialize novel therapies for ophthalmic diseases. The lead candidate has completed phase 2 and is an innovative therapeutic with the potential become the standard of care for Dry Eye Disease (DED) with $2B+ annual global revenue potential. DED affects over 30 million adults in the US and over 340 million people globally. The company’s leadership team has extensive experience developing and commercializing ophthalmology therapies at companies including Genentech, Allergan, OptiMedica, Oculeve, Ophthotech, Guidant, and Eyetech. Oyster Point was founded in 2015 by neuroscientist and entrepreneur Dr. Michael Ackermann, past CEO of Oculeve (acquired by Allergan). The company is preparing to initiate Phase 3 trials in 2019. The current investors are NEA and Versant Ventures. Oyster Point is headquartered in Princeton, NJ. Time Tuesday, Feb 12 10:30AM - 10:45AM Location Herald/Soho Speakers Jeffrey Nau Oyster Point Pharma, Inc.
10:45am EyeGate Pharmaceuticals, Inc. Stephen From, EyeGate Pharmaceuticals, Inc. show more Session Description EyeGate is a clinical-stage specialty pharmaceutical company developing and commercializing products for treating diseases and disorders of the eye. The Company has two proprietary platform technologies. Our most advanced platform is based on cross-linked thiolated carboxymethyl hyaluronic acid (“CMHA-S”), a modified form of the natural polymer hyaluronic acid (“HA”), which is a gel that possesses unique physical and chemical properties such as hydrating and healing when applied to the ocular surface. We recently announced positive data from two studies for our lead CMHA-S product, EyeGate Ocular Bandage Gel (“OBG”), which is being developed for corneal re-epithelialization following photorefractive keratectomy (“PRK”) surgery, as well as for better management of patients with punctate epitheliopathies (“PE”) due to pathologies such as dry. EyeGate OBG is classified as a Class 2 medical device, which we plan to pursue U.S. regulatory clearance via the FDA’s De Novo 510(k) pathway. Time Tuesday, Feb 12 10:45AM - 11:00AM Location Herald/Soho Speakers Stephen From EyeGate Pharmaceuticals, Inc. MaxCyte, Inc. Douglas Doerfler, MaxCyte, Inc. show more Session Description MaxCyte is a global cell-based medicines and life sciences company applying its patented cell engineering technology to help patients with high unmet medical needs in a broad range of conditions. MaxCyte is developing novel CARMA™ therapies for its own pipeline. CARMA is MaxCyte’s mRNA-based proprietary platform for autologous cell therapy. In addition, through its core business, MaxCyte leverages its Flow Electroporation® Technology platform to enable its biopharma partners to advance the development of innovative, cutting-edge medicines, particularly in cell therapy, including the use of gene editing tools in the treatment of inherited genetic diseases and immuno-oncology approaches. The Company has placed its cutting-edge instruments worldwide, including with 9 of the top 10 global biopharma companies, and has 55+ partnered program licenses in cell therapy including 25+ licensed for clinical use. MaxCyte helps its partners to unlock the full potential of their products. Time Tuesday, Feb 12 10:45AM - 11:00AM Location Hudson/Empire Speakers Douglas Doerfler MaxCyte, Inc. NoNO Inc. Michael Tymianski, NoNO Inc. show more Session Description NoNO Inc. is a privately held, Canadian biotech developing neurotherapeutics for acute and chronic CNS disease. Our lead product is in two Phase 3 acute stroke studies – ESCAPE NA-1 for endovascular stroke therapy patients in the hospital and FRONTIER for treatment of suspected stroke patients pre-hospital by paramedics. We have imminent pivotal data with our lead indication reading out in Q4 2019 followed by anticipated commercial launch in Q1 2021. NA-1 is a first-in-class drug against a first-in-class target, PSD95, for the treatment of the leading cause of global neurological disability. The USFDA has granted Fast Track and Orphan designations. NoNO has a strong balance sheet and cash flow to achieve NDA clearance and to support R&D efforts for label expansion from acute to chronic CNS. We anticipate entering clinic for our first chronic CNS indication by the end of 2019. Time Tuesday, Feb 12 10:45AM - 11:00AM Location Gramercy Speakers Michael Tymianski NoNO Inc.
11:00am Cellectar Biosciences James Caruso, Cellectar Biosciences show more Session Description Cellectar Biosciences is focused on the discovery, development, and commercialization of drugs for the treatment of cancer. The company plans to develop proprietary drugs independently and through research and development (R&D) collaborations. The core drug development strategy is to leverage our PDC platform to develop therapeutics that specifically target treatment to cancer cells. Through R&D collaborations, the company’s strategy is to generate near-term capital, supplement internal resources, gain access to novel molecules or payloads, accelerate product candidate development and to broaden our proprietary and partnered product pipelines. The company’s lead PDC therapeutic, CLR 131, is in a Phase 1 clinical study in patients with R/R MM and a Phase 2 clinical study in R/R MM and a range of B-cell malignancies. The company plans to initiate a Phase 1 study with CLR 131 in pediatric solid tumors and lymphomas. Time Tuesday, Feb 12 11:00AM - 11:15AM Location Hudson/Empire Speakers James Caruso Cellectar Biosciences Galimedix Therapeutics Andrew Pearlman, Galimedix Therapeutics show more Session Description Galimedix is a Phase 2 US/Israel clinical stage ophthalmic biopharma company developing truly novel and transformative treatments based on a novel mechanism of action directed to the common cause of many neurodegenerative diseases: toxic oligomers of amyloid beta. Lead product, GAL-101, is an eye drop for treatment to slow progression of dry macular degeneration of the retina, and glaucoma: two of the leading causes of blindness. Led by a highly experienced biopharma team including many ex-Pharma executives, Galimedix has an exclusive worldwide license from Tel Aviv University and German-based Merz Pharmaceuticals, where the compound was developed for seven years through Phase I. A next generation, potentially superior compound, GAL-201, is in preclinical development for oral administration, with the potential to treat CNS diseases such as Alzheimer’s. The Board of Directors and scientific advisors include many leading names in the field of glaucoma and retinal disease. Time Tuesday, Feb 12 11:00AM - 11:15AM Location Chelsea Speakers Andrew Pearlman Galimedix Therapeutics National Health Council Patient-Centered Health Care Accelerator Eleanor Perfetto, National Health Council show more Session Description The National Health Council (NHC) and Boomtown Accelerator, have established the FIRST PATIENT-CENTERED HEALTH CARE ACCELERATOR which will identify and invest in 20 EARLY STAGE, PATIENT-CENTRIC HEALTH STARTUPS over 3 years. As research shows, the key to successful health care product and service development is patient-centric design, leading to increased uptake and improvements in the outcomes patients care most about. Patient-centric products and services have become the leading sector for health care investments. NHC is widely regarded as the industry thought leader in patient-centricity research and advocates on patients’ behalf at the highest public-policy levels. Its’ members include the nation’s leading patient organizations, as well as leading pharmaceutical, device, and insurance companies. Our members will be involved in mentoring roles as new accelerator participants are enrolled (see www.nationalhealthcouncil.org). Time Tuesday, Feb 12 11:00AM - 11:15AM Location Gramercy Speakers Eleanor Perfetto National Health Council Nicox SA Michele GARUFI , Nicox show more Session Description Nicox is an international ophthalmology company developing innovative solutions to help maintain vision and improve ocular health. Our portfolio includes three programs in development all of which are in, or preparing to, enter Phase 2 clinical studies: NCX 470 for glaucoma, NCX 4251 for blepharitis and NCX 4280 for morning ocular congestion. Our pipeline also includes future generation stand alone NO donors in the research stage and other exploratory novel NO donating compounds targeting ophthalmic conditions. We have two ophthalmology assets approved by the U.S. FDA; VYZULTA exclusively licensed worldwide to Bausch + Lomb and commercialized in the U.S. since December 2017 as well as ZERVIATE exclusively licensed in the U.S. to Eyevance with a commercial launch in the U.S planned for spring 2019 Time Tuesday, Feb 12 11:00AM - 11:15AM Location Herald/Soho Speakers Michele GARUFI Nicox Policy Outlook—How Could the Trump Administration’s Drug Reimbursement Proposals Affect Medical Innovation? James C. Greenwood, Biotechnology Innovation Organization (BIO); Paul Lammers, Triumvira Immunologics; Susan Peschin, Alliance for Aging Research; Peter Pitts, Center for Medicine in the Public Interest; Duane Schulthess, Vital Transformation; Erica Whittaker, UCB Ventures; Peter Young, Pappas Capital show more Session Description The U.S. Department of Health and Human Services has proposed piloting an “International Pricing Index” (IPI) payment model to reduce reimbursement levels for the Centers for Medicare & Medicaid Services (CMS), as well as proposing other reforms to long-standing policies regarding commercialized medicines. In a year with a record number of new medicines being approved to reach patients in the United States, such reforms introduce significant uncertainty for many investors’ valuation models. This session will feature drug developers, investors, and policy experts to explain the resource allocation implications if these reforms proceed and risks of shrinking the pipelines of new medicines for years to come unless alternative approaches can be introduced. Moderator: James C. Greenwood, President and CEO, Biotechnology Innovation Organization (BIO) Time Tuesday, Feb 12 11:00AM - 11:55AM Location Shubert Complex Speakers James C. Greenwood Biotechnology Innovation Organization (BIO) Paul Lammers Triumvira Immunologics Susan Peschin Alliance for Aging Research Peter Pitts Center for Medicine in the Public Interest Duane Schulthess Vital Transformation Erica Whittaker UCB Ventures Peter Young Pappas Capital
11:15am Knopp Biosciences Michael Bozik, Knopp Biosciences show more Session Description Knopp Biosciences is a privately held drug discovery and development company focused on delivering breakthrough treatments for inflammatory and neurological diseases of high unmet need. Knopp’s clinical-stage small molecule, dexpramipexole, is entering Phase 3 clinical studies in hypereosinophilic syndrome and Phase 2 clinical studies in eosinophilic asthma. Knopp’s preclinical Kv7 platform is directed to small molecule treatments for neonatal epileptic encephalopathy, other rare epilepsies, tinnitus, and neuropathic pain. The Company is looking to complete a crossover financing in the first quarter of 2019. Time Tuesday, Feb 12 11:15AM - 11:30AM Location Gramercy Speakers Michael Bozik Knopp Biosciences Oxurion NV Patrik De Haes, OXURION NV show more Session Description Oxurion NV is a leading ophthalmology biopharmaceutical company developing treatments to preserve vision for patients with diseases affecting the back of the eye. It has engineered a diverse portfolio of best-in-class disease-modifying drug candidates, including treatments for diabetic eye disease, a leading cause of blindness in people of working age worldwide. Oxurion’s clinical pipeline consists of THR-317, an anti- PlGF antibody in phase 2 for the treatment of DME, and in a phase 2 for MacTel 1; THR-149, a plasma kallikrein inhibitor for the treatment of DME; and THR-687, a pan-RGD integrin antagonist for the treatment of DR, DME, and wet AMD. Time Tuesday, Feb 12 11:15AM - 11:30AM Location Herald/Soho Speakers Patrik De Haes OXURION NV Sense Diagnostics, Inc. Dan Kincaid, Sense Diagnostics,Inc show more Session Description Sense Diagnostics team is skilled in the clinical care of severe brain injuries, with three practicing physicians among the founders.The SENSE scanner transmits low power radio waves through the patient’s brain every few minutes. It detects scattering by the blood cells and plasma when the signal encounters a hemorrhage. This fact allows our scanner to detect bleeding in minutes, not hours. The SENSE device was invented and designed by practicing clinicians and researchers. It will fit easily into the workflow and footprint of the ambulance and the ICU. It also doesn’t interfere with other treatments or monitors used the patient. Current funding initiatives: FDA Approval (1Q18 -2Q20): $3.5-5 million. During this phase, the company will be conducting a pivotal clinical trials on Traumatic Brain Injury and Stroke patients. Targeting preparation and submission of required documentation to the FDA by June 2019. Initiating sales, production capabilities for product launch in mid 2020. Time Tuesday, Feb 12 11:15AM - 11:30AM Location Chelsea Speakers Dan Kincaid Sense Diagnostics,Inc Sutro Biopharma, Inc. William Newell, Sutro Biopharma show more Session Description We have pioneered a compelling and unique way of discovering, developing and manufacturing therapeutics. Our focus is aimed primarily on next generation cancer and autoimmune therapeutics — Antibody Conjugates, Bispecific Antibodies and Cytokine derivatives. Unconstrained by traditional methods of cell-based discovery, we can design and develop targeted medicines by innovating outside the constraints of the cell. Our technology enables us to iteratively discover and test molecules in a rapid cycle of weeks rather than months, to rapidly identify the optimal molecule designed for safety and potency. Our approach to discovery, without the cell, is also transcending the limitations of biologics manufacturing. Sutro has the world’s only cGMP cell-free manufacturing facility located in San Carlos, California. Time Tuesday, Feb 12 11:15AM - 11:30AM Location Hudson/Empire Speakers William Newell Sutro Biopharma
12:00pm Fireside Chat: Roger Crystal, MD, Chief Executive Officer, Opiant Pharmaceuticals Roger Crystal, Opiant Pharmaceuticals; Brandon Folkes, Cantor Fitzgerald show more Session Description Fireside Chat: Roger Crystal, MD, Chief Executive Officer, Opiant Pharmaceuticals Moderator: Brandon Folkes, Director, Cantor Fitzgerald Time Tuesday, Feb 12 12:00PM - 12:55PM Location Majestic Complex Speakers Roger Crystal Opiant Pharmaceuticals Brandon Folkes Cantor Fitzgerald
1:00pm Regulatory Changes to Cross-Border Investments: CFIUS and Biotech Deals Lisa Schaefer , Biotechnology Innovation Organization (BIO); Jun Bao, Impact Therapeutics; Joseph Benkert , Morrison & Foerster LLP; Jeff Farrah, National Venture Capital Association (NVCA); Kimberly Nearing, Cedrus Group show more Session Description New national security regulations under the Foreign Investment Risk Review Modernization Act (FIRRMA) are changing the landscape for cross-border investments in biotechnology. In November 2018, new rules for a “pilot program” apply to foreign investors into U.S. companies who now need to file with the Committee on Foreign Investment in the United States (CFIUS) to approve covered transactions before entering licensing deals or other equity investments. This session will update market participants on the evolving national security review process and its impact on the biotechnology industry, as well as consider how additional rules may impact future business development and investment opportunities. Moderator: Lisa Schaefer, Director, Tax & Financial Services Policy, Biotechnology Innovation Organization (BIO) Time Tuesday, Feb 12 1:00PM - 1:55PM Location Shubert Complex Speakers Lisa Schaefer Biotechnology Innovation Organization (BIO) Jun Bao Impact Therapeutics Joseph Benkert Morrison & Foerster LLP Jeff Farrah National Venture Capital Association (NVCA) Kimberly Nearing Cedrus Group
1:15pm Catalyst Biosciences Nassim Usman, Catalyst Biosciences show more Session Description Catalyst is focused on developing novel medicines to address serious medical conditions for patients who need new or better treatment options. We used a scientific approach focused on protease-based therapeutic candidates to build a clinical-stage biopharmaceutical company whose current mission is to develop valuable therapies for patients with hemophilia. Time Tuesday, Feb 12 1:15PM - 1:30PM Location Herald/Soho Speakers Nassim Usman Catalyst Biosciences ImmunSYS, Inc. Joseph Gerardi, ImmunSYS, Inc. show more Session Description ImmunSYS is a clinical stage biopharmaceutical company focused on the development of innovative cancer immunotherapy products to improve the lives of patients with late-stage metastatic cancers. Immunotherapy, also called biologic therapy, is a type of cancer treatment that boosts the body's natural defenses to fight cancer. It uses substances made by the body or in a laboratory to improve or restore immune system function. Immunotherapy may work by: *Stopping or slowing the growth of cancer cells *Stopping cancer from spreading to other parts of the body *Helping the immune system work better at destroying cancer cells. Our platform technology, YourVaccx™ utilizes a novel combination of the proprietary ANTIGENerator™ energy delivery device system, a proprietary branded formulation of immunotherapeutic drugs, and a proprietary procedure developed by the focal prostate cancer therapy pioneer Gary Onik, M.D. Time Tuesday, Feb 12 1:15PM - 1:30PM Location Chelsea Speakers Joseph Gerardi ImmunSYS, Inc. Sapience Therapeutics Barry Kappel, Sapience Therapeutics show more Session Description Sapience Therapeutics is a privately held company focused on discovering and developing peptide-based therapeutics to previously ‘undruggable’ targets for major unmet medical needs, particularly high mortality cancers. Sapience identifies promising targets through a deep understanding of the transcriptional regulation of oncogenic or immune-modulatory proteins. Our targets may be non-traditional in drug discovery as they do not demonstrate enzymatic activities or act as ligand-binding receptors. Sapience has multiple approaches to generate initial hits, which are then optimized to therapeutic candidates based on decades of experience working with peptide and protein therapeutics. Sapience is looking to raise a series B financing in the first half of 2019. Time Tuesday, Feb 12 1:15PM - 1:30PM Location Hudson/Empire Speakers Barry Kappel Sapience Therapeutics SpinalCyte, LLC Pete OHeeron, SpinalCyte, LLC show more Session Description Based in Houston, Texas, SpinalCyte, LLC is a regenerative medicine company developing an innovative solution for spinal disc replacement using human dermal fibroblasts. Currently, SpinalCyte holds 36 U.S. and international issued patents and has filed for an additional 40 patents pending. SpinalCyte holds 117 U.S. and International Patents pending and issued across a variety of clinical pathways, including disc degeneration, cancer, diabetes, liver failure and heart failure. Funded entirely by angel investors, SpinalCyte represents the next generation of medical advancement in cell therapy. Time Tuesday, Feb 12 1:15PM - 1:30PM Location Gramercy Speakers Pete OHeeron SpinalCyte, LLC
1:30pm Aquestive Therapeutics Keith Kendall, Aquestive Therapeutics show more Session Description Aquestive Therapeutics is a specialty pharmaceutical company committed to identifying, developing and commercializing differentiated products to address unmet medical needs. Aquestive Therapeutics has a late-stage proprietary product pipeline focused on the treatment of CNS diseases, and is working to advance orally-administered complex molecules that it believes can be alternatives to invasively-administered standard of care therapies. As the leader in developing and delivering drugs via its PharmFilm® technology, Aquestive Therapeutics also collaborates with pharmaceutical partners to bring new molecules to market in differentiated and highly-marketable dosage forms. Time Tuesday, Feb 12 1:30PM - 1:45PM Location Herald/Soho Speakers Keith Kendall Aquestive Therapeutics ImmunoMet Therapeutics Benjamin Cowen, ImmunoMet Therapeutics show more Session Description ImmunoMet is a clinical stage biotech utilizing cellular metabolism to develop novel anti-tumor and immuno-metabolism therapies. ImmunoMet was founded in 2015 as a is Spin-off from HanAll Biopharma, a mid-sized Korean pharma co, and is headquartered at Houston JLABs near MD Anderson. We have raised $20 M post spin-off including $10 M from VCs. Our lead molecule, IM156, is an OXPHOS inhibitor which has demonstrated impressive in vivo efficacy in resistant solid tumors and entered Phase 1 in 4Q17. ImmunoMet owns a large biguanide library which forms an engine we are leveraging to unlock value and develop products in immuno-metabolism. Time Tuesday, Feb 12 1:30PM - 1:45PM Location Hudson/Empire Speakers Benjamin Cowen ImmunoMet Therapeutics Senti Biosciences Timothy Lu, Senti Biosciences show more Session Description Founded in 2016 by Drs. Timothy Lu, Philip Lee, Jim Collins and Wilson Wong, Senti Biosciences is the next generation therapeutics company that designs gene circuits and programs cells for tremendous therapeutic value. Senti's mission is to engineer an entirely new class of medicine that will transform people’s lives by curing the most complex diseases. Backed by a syndicate of leading biotech and tech investors including NEA, 8VC, Lux, Pear, Menlo Ventures, and Amgen Ventures, and led by an experienced team with deep translational knowledge of advanced cell therapies, the company is leveraging its proprietary gene circuit repertoire to solve the most pressing unmet needs in oncology and other difficult disease areas. Time Tuesday, Feb 12 1:30PM - 1:45PM Location Gramercy Speakers Timothy Lu Senti Biosciences
1:45pm AIVITA Biomedical Hans Keirstead, AIVITA Biomedical, Inc. show more Session Description AIVITA Biomedical is a clinical stage biotech developing next generation immunotherapies. We leverage our expertise with stem cells to develop therapies to treat solid tumor cancers. Our platform technology isolates and propagates tumor-initiating cells (or cancer stem cells), responsible for metastases of cancers, to target the full patient-specific antigenic profile of that patient's tumor. When presented by the patient's dendritic cells, these antigens are recognized by the immune system and targeted for termination. Exposing the full antigenic load of the tumor means the immune system may be able to identify mutated or dormant tumor-initiating cells, improving the patient's ability to fight the disease. In addition to two phase 2 studies in the US, we have a pivotal trial in Japan in metastatic melanoma starting Q1 2019. Our platform technology is autologous cell therapy. We have robust data in efficacy and mechanism of action and a low-cost, efficient manufacturing proc Time Tuesday, Feb 12 1:45PM - 2:00PM Location Hudson/Empire Speakers Hans Keirstead AIVITA Biomedical, Inc. Oramed Pharmaceuticals Nadav Kidron , Oramed Pharmaceuticals show more Session Description Oramed Pharmaceuticals is a platform technology pioneer in the field of oral delivery solutions for drugs currently delivered via injection. Established in 2006, Oramed's Protein Oral Delivery (PODTM) technology is based on over 30 years of research by scientists at Jerusalem's Hadassah Medical Center. Oramed is seeking to revolutionize the treatment of diabetes through its proprietary flagship product, an orally ingestible insulin capsule (ORMD-0801). The Company completed multiple Phase II clinical trials under an Investigational New Drug application with the U.S. Food and Drug Administration. In addition, Oramed is developing an oral GLP-1 analog capsule (ORMD-0901). Time Tuesday, Feb 12 1:45PM - 2:00PM Location Herald/Soho Speakers Nadav Kidron Oramed Pharmaceuticals ProterixBio, Inc Michael Miller, ProterixBio, Inc show more Session Description ProterixBio is a provider of molecular assessments to transform the care of patients with chronic respiratory disease. Our initial focus is COPD, which is the 3rd leading cause of death worldwide and contributes >$40B of direct healthcare spending annually in the U.S. alone. ProterixBio’s proprietary technology uses multiple blood-based biomarkers combined in an algorithm to characterize a patient’s disease state. Results from multiple clinical studies presented major respiratory conferences demonstrate improvement over symptoms and history alone for assessing propensity for future exacerbations. We are starting pilot studies with COPD management programs to target patient enrollment with the ultimate goal of reducing the number of ER visits and hospitalizations. The rapid expansion of value-based care programs managing chronically ill populations and the emergence of new targeted therapies within respiratory provide substantial opportunity for growth. Time Tuesday, Feb 12 1:45PM - 2:00PM Location Gramercy Speakers Michael Miller ProterixBio, Inc
2:00pm Immunomic Therapeutics, Inc. Bill Hearl, Immunomic Therapeutics, Inc. show more Session Description Immunomic Therapeutics, Inc. (ITI) is a privately-held, clinical stage biotechnology company pioneering the development of vaccines through its proprietary technology platform, UNiversal Intracellular Targeted Expression (UNITE), which is designed to utilize the body’s natural biochemistry to generate broad immune responses. ITI’s UNITE platform has broad therapeutic applications in oncology, including viral antigens, cancer antigens, neoantigens and antigen-derived antibodies as biologics and ITI has built a large pipeline from UNITE with six oncology programs and two allergy programs. ITI's technology has been deployed in over 135 oncology patients and 210 allergy patients and in oncology, has demonstrated survival benefit and immune system activation in the clinic. Due to its expertise in the field, ITI entered into a significant allergy partnership with Astellas Pharma and has formed several academic collaborations with leading Immuno-oncology researchers. Time Tuesday, Feb 12 2:00PM - 2:15PM Location Hudson/Empire Speakers Bill Hearl Immunomic Therapeutics, Inc. Removing Commercialization Barriers for New Antimicrobial Drugs Gregory Frank, Biotechnology Innovation Organization; Heather Behanna, Entasis Therapeutics; John H. Johnson, Melinta; Ciara Kennedy, Amplyx Pharmaceuticals, Inc.; Kevin Outterson, Boston University;CARB-X; Manos Perros, Entasis Therapeutics show more Session Description After years of sparse development of new antimicrobials, R&D incentive initiatives such as CARB-X have helped grow late-stage pipelines and new approvals. Unfortunately, difficult commercialization experiences of leaders in this space threaten to derail the momentum of the sector. In response, the FDA has proposed the health care system consider a subscription model for the purchase of antibiotics by hospitals in an effort to increase the reliability of a return on investment in new antibiotics and avoid high-volume prescribing practices that would accelerate antimicrobial resistance. This session will include policy, antimicrobial developer, and investor perspectives on this issue to discuss the opportunities and challenges of business model changes and what it will take to avoid a crisis described by the World Health Organization as a “post-antibiotic era where common infections and minor injuries can once again kill.” Moderator: Gregory Frank, PhD, Director, Infectious Disease Policy, Biotechnology Innovation Organization (BIO) Time Tuesday, Feb 12 2:00PM - 2:55PM Location Shubert Complex Speakers Gregory Frank Biotechnology Innovation Organization Heather Behanna Entasis Therapeutics John H. Johnson Melinta Ciara Kennedy Amplyx Pharmaceuticals, Inc. Kevin Outterson Boston University;CARB-X Manos Perros Entasis Therapeutics Resverlogix Corp. Donald McCaffrey, Resverlogix Corp. show more Session Description Resverlogix is developing apabetalone (RVX-208), a first-in-class, small molecule that is a selective BET (bromodomain and extra-terminal) inhibitor. BET bromodomain inhibition is an epigenetic mechanism that can regulate disease-causing genes. Apabetalone is a BET inhibitor selective for the second bromodomain (BD2) within the BET proteins. This selective inhibition of apabetalone on BD2 produces a specific set of biological effects with potentially important benefits for patients with high-risk cardiovascular disease, diabetes mellitus, chronic kidney disease, end-stage renal disease treated with hemodialysis, neurodegenerative disease, Fabry disease, peripheral artery disease and other orphan diseases, while maintaining a well described safety profile. Time Tuesday, Feb 12 2:00PM - 2:15PM Location Herald/Soho Speakers Donald McCaffrey Resverlogix Corp. SATOR Therapeutics LLC Stephen Charles, SATOR Therapeutics LLC show more Session Description THE SATOR PLATFORM for the Comprehensive Treatment of Metastatic Cancers: PLATFORM concept of PANELS of nonengineered, nonpathogenic SATOR viruses proven in a variety of human cancers in cell culture, mouse xenograft models, and via human compassionate use studies. Pretreatment testing with efficacy–predicting COMPANION DIAGNOSTICS minimizes treatment of non–responders. No serious adverse events reported; substantial life extension achieved. Efficient US regulatory pathway due to choice of orphan indication ovarian cancer. Comprehensive worldwide patent protection applied for. Upcoming Milestones: Complete $40 million Series A private financing. INTERACT meeting with FDA. Orphan drug designation submission to US FDA and European EMA. cGMP manufacturing and preclinical testing of 3 lead candidate SATOR viruses; development and validation of 3 efficacy–predicting companion diagnostics. IND preparation and filing for 3 SATOR viruses. Conduct Phase 1b patient study in ovarian cancer. Time Tuesday, Feb 12 2:00PM - 2:15PM Location Gramercy Speakers Stephen Charles SATOR Therapeutics LLC
2:15pm Lyndra Inc Amy Schulman, Lyndra Inc show more Session Description At Lyndra, we are working to realize a healthier world by transforming the way we take medicine. Our team is developing pills that can last a week or more, freeing patients and caregivers of the burden of daily medications. Expected benefits of our long-acting oral therapies include: better health outcomes and quality of life, reduced side effects, improved drug efficacy, slowed spread of disease, and lower health costs. Time Tuesday, Feb 12 2:15PM - 2:30PM Location Gramercy Speakers Amy Schulman Lyndra Inc Tenax Therapeutics, Inc. Anthony DiTonno, Tenax Therapeutics, Inc. show more Session Description Tenax Therapeutics, Inc. (TENX), a specialty pharmaceutical company focused on identifying, developing and commercializing of drugs that address diseases with high unmet medical need. Tenax has secured the North American rights to develop and commercialize levosimendan from the originator company, Orion Corporation of Espoo Finland. Levosimendan is a calcium sensitizer and K-ATP channel opener that works through a unique triple mechanism of action. It is approved in over 60 countries as an intravenous treatment for acute decompensated heart failure. However, levosimendan is not available in the United States or in Canada. Tenax plans to advance the development of Levosimendan for the treatment of pulmonary hypertension associated with heart failure and preserved ejection fraction (PH-HFpEF). Tenax plans to initiate a Phase 2 trial in PH-HFpEF patients in the next few months. Time Tuesday, Feb 12 2:15PM - 2:30PM Location Herald/Soho Speakers Anthony DiTonno Tenax Therapeutics, Inc. Vyriad, Inc. Kah-Whye Peng, Vyriad show more Session Description Vyriad is a clinical stage biopharmaceutical company established to realize the full potential of oncolytic viruses to impact cancer, alone or in combination with immune-oncology agents. The company is developing multiple unique viral platforms, focusing on systemic delivery, single cycle therapy and noninvasive tracking of the infection in treated patients using proprietary reporter gene technologies. The company has a broad technology base, dominating intellectual property position, and a superior lead clinical asset with proven clinical trials execution capability and in-house GMP manufacturing coming on stream in 2019. The early clinical data sets from ongoing Phase 1 trials are compelling and will be summarized in the presentation. Time Tuesday, Feb 12 2:15PM - 2:30PM Location Hudson/Empire Speakers Kah-Whye Peng Vyriad
2:30pm NanoViricides, Inc. Anil Diwan, NanoViricides, Inc. show more Session Description NanoViricides, Inc. is pioneering a unique platform of nanomedicines to treat viral diseases that we call nanoviricides®. We are advancing our first drug candidate in the HerpeCide™ program towards clinical trials. The initial indication would be for topical treatment of shingles rash. Additional drugs against HSV-1 cold sores and HSV-2 genital ulcers would follow. The Company has previously demonstrated drug candidates with strong efficacy in animal studies against a wide range of viruses including Influenzas, HIV, Dengue, Ebola, among others, attesting to robustness of the platform. The Company is also unique in that it has its own flexible multi-product c-GMP-capable manufacturing facility that can scale to several kg API per batch, enabling rapid translation to the clinic. Our platform is designed to mimic host cell receptors to the virus, thereby trapping & destroying the virus particle. This approach makes viral resistance unlikely as the resistant virus would be incompetent. Time Tuesday, Feb 12 2:30PM - 2:45PM Location Gramercy Speakers Anil Diwan NanoViricides, Inc. Verona Pharma Piers Morgan, Verona Pharma show more Session Description Verona Pharma is a clinical-stage biopharmaceutical company focused on developing and commercializing innovative therapies for the treatment of respiratory diseases with significant unmet medical needs. Verona Pharma’s product candidate, ensifentrine, is an investigational first-in-class, inhaled, dual inhibitor of the enzymes phosphodiesterase 3 and 4 that is designed to act as both a bronchodilator and an anti-inflammatory agent in a single compound. In previous clinical trials, the nebulized formulation of ensifentrine has been observed to result in bronchodilator effects when used alone or as an add-on treatment to other COPD bronchodilators. It has shown clinically meaningful and statistically significant improvements in lung function when administered in addition to frequently used short- and long-acting bronchodilators, such as tiotropium (Spiriva®), compared with such bronchodilators administered as a single agent. Time Tuesday, Feb 12 2:30PM - 2:45PM Location Herald/Soho Speakers Piers Morgan Verona Pharma Zenith Epigenetics Donald McCaffrey, Zenith Epigenetics show more Session Description Zenith Epigenetics Ltd. is a clinical stage biotechnology company developing best in class bromodomain (BET) inhibitors for the treatment of cancer and other disorders with significant unmet medical need. Our goal is to be a leading epigenetic company translating bromodomain biology into impactful therapies. Time Tuesday, Feb 12 2:30PM - 2:45PM Location Hudson/Empire Speakers Donald McCaffrey Zenith Epigenetics
3:00pm Fireside Chat: Dr. Aoife M. Brennan, President and Chief Executive Officer and Chief Medical Officer, Synlogic, Inc. Aoife M. Brennan, Synlogic; Yi Chen, H.C. Wainwright & Co., LLC show more Session Description Fireside Chat: Dr. Aoife M. Brennan, President and Chief Executive Officer and Chief Medical Officer, Synlogic, Inc. Moderator: Yi Chen, PhD, Managing Director of Equity Research, H.C. Wainwright & Co, LLC (A) The equity research department of H.C. Wainwright & Co. covers Synlogic, Inc. with a Buy rating and $20 price target as of February 12, 2019. (B) None of the research analysts or the research analyst’s household has a financial interest in the securities of Synlogic, Inc. (C) As of January 31, 2019, neither H.C. Wainwright & Co. nor its affiliates beneficially own 1% or more of any class of common equity securities of Synlogic, Inc. (D) H.C. Wainwright & Co. or its affiliates did not receive compensation from Synlogic, Inc. in the previous twelve months. (E) None of the research analysts have received compensation from Synlogic, Inc. in the previous twelve months. (F) Synlogic, Inc. was not a client of H.C. Wainwright & Co. or its affiliates during the previous twelve months. (G) H.C. Wainwright & Co. does not make a market in Synlogic, Inc. as of February 12, 2019. Time Tuesday, Feb 12 3:00PM - 3:55PM Location Majestic Complex Speakers Aoife M. Brennan Synlogic Yi Chen H.C. Wainwright & Co., LLC
4:00pm Are Record Levels of IPOs Still Disrupting M&A Deals? Lydia Ramsey , Business Insider; John Chambers, H.C. Wainwright & Co., LLC; Jie D'Elia, Bristol-Myers Squibb; Craig Farrell , AlphaSense; Chris Garabedian, Xontogeny; Perceptive Advisors; Jim Healy, Sofinnova; Dennis J. Purcell, Aisling Capital LLC show more Session Description In 2018, biotechs delivered the second-best year for public issuances ever. The attractive IPO market has however resulted in fewer M&A deals than prior years, with high pre-money IPO valuations for later-stage companies making it less enticing for them to accept early-exit offers. This has led to an M&A shift to focus on early-stage companies. How are changing valuations and exit options affecting venture investors? How are prospective buyers changing their deal terms? The session will examine the recent deal and market trends while highlighting the path forward amid a market with greatly increased volatility and reimbursement policy uncertainty. Moderator: Lydia Ramsey, Senior Reporter, Business Insider Time Tuesday, Feb 12 4:00PM - 4:55PM Location Shubert Complex Speakers Lydia Ramsey Business Insider John Chambers H.C. Wainwright & Co., LLC Jie D'Elia Bristol-Myers Squibb Craig Farrell AlphaSense Chris Garabedian Xontogeny; Perceptive Advisors Jim Healy Sofinnova Dennis J. Purcell Aisling Capital LLC
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Development Phase of Primary Product