BIO CEO 2013 Presenting Company Descriptions

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Autoimmune/Inflammation

Opexa Therapeutics Opexa Therapeutics, Inc. (NASDAQ:OPXA) is a publicly traded biotechnology company dedicated to the development of patient-specific cellular therapies for the treatment of autoimmune diseases. The company's leading product candidate, TcelnaTM , has the potential to address the significant unmet medical needs of the large multiple sclerosis (MS) patient population.

Cardiovascular

Resverlogix Corp. Resverlogix (TSX: RVX) is a clinical stage cardiovascular company with an epigenetic platform technology that modulates protein production. Resverlogix is developing RVX-208, a first-in-class small molecule for the treatment of atherosclerosis. New compounds arising from the epigenetic drug discovery platform function by inhibiting BET bromodomains and have the potential to impact multiple diseases including autoimmune diseases, cancer, neurodegenerative diseases and diabetes mellitus.

Cell Therapy/Regenerative Medicine

ADVENTRX Pharmaceuticals ADVENTRX Pharmaceuticals is a specialty pharmaceutical company focused on developing proprietary product candidates. The Company’s current lead product candidates are ANX-188, a novel, purified, rheologic and antithrombotic compound initially being developed as a first-in-class treatment for pediatric patients with sickle cell disease in acute crisis, and ANX-514, a novel, detergent-free formulation of the chemotherapy drug docetaxel. 

CNS

Amarantus Bioscience Amarantus BioScience, Inc. (“Amarantus”) is a biotechnology company discovering and developing treatments and diagnostics for diseases associated with neurodegeneration and apoptosis (programmed cell death). The Company’s lead therapeutic program Mesencephalic-Astrocyte-derived Neurotrophic Factor, or  MANF is a targeted therapeutic to address the underlying Programmed Cell Death (Apoptosis) associated with a wide range of devastating human disorders. The Company's lead diagnostic program, NuroPro®, is aimed at developing diagnostic blood tests that are capable of early identification of apoptosis-related medical conditions while tracking their progression over time. Both programs will initially target Parkinson's disease. Amarantus’ Management Team and Board of Advisors, have designed a business strategy to formulate and advance pre-clinical and clinical programs for the Company's product candidates through successive de-risking milestones in order to maximize their commercial potential. The Company is also focused on making the assets attractive partnering targets for product development focused biotechnology, pharmaceutical, medical device and diagnostic companies.

BioLineRX Ltd. BioLineRx Ltd. is a publicly-traded biopharmaceutical development company. BioLineRx’s current portfolio consists of five clinical stage candidates: BL-1020 for schizophrenia is in Phase II/III clinical trials; BL-1040 for prevention of pathological cardiac remodeling following a myocardial infarction has completed a Phase I/II study and has been out-licensed to Ikaria Inc. for a total deal value of $282.5 million, in addition to sales royalties; BL-5010 for non-surgical removal of skin lesions has completed a Phase I/II study; BL-1021 for neuropathic pain is in Phase I trials; and BL-7040 for treating Inflammatory Bowel Disease (IBD) has completed Phase I. In addition, BioLineRx has nine products in various pre-clinical development stages for a variety of indications, including central nervous system diseases, oncology, infectious diseases, cardiovascular and autoimmune diseases.

The Michael J. Fox Foundation As the world’s largest private funder of Parkinson’s research, The Michael J. Fox Foundation is dedicated to accelerating a cure for Parkinson’s disease and improved therapies for those living with the condition today. The Foundation pursues its goals through an aggressively funded, highly targeted research program, and has invested over $313 million in research to date.  www.michaeljfox.org. 

Gastrointestinal Disorders

Lexicon Pharmaceuticals Lexicon Pharmaceuticals is a biopharmaceutical company focused on discovering and developing breakthrough treatments for human disease.  Using its proprietary gene knockout technology, Lexicon has discovered more than 100 promising drug targets, and created a unique, growing clinical pipeline in the fields of cardiology, gastroenterology, immunology, metabolism, and ophthalmology.

NPS Pharmaceuticals NPS Pharmaceuticals is a biopharmaceutical company focused on bringing orphan products to patients with rare disorders and few, if any, therapeutic options. The company’s lead product, Gattex® 0.05 mg/kg/d (Teduglutide [rDNA origin]) for Injection is FDA-approved for the treatment of adult patients with short bowel syndrome (SBS) who are dependent on parenteral support. NPS is also developing Natpara® (rhPTH[1-84]) for the treatment of adult hypoparathyroidism and expects to submit its Biologic License Application (BLA) to the FDA in mid-2013.  NPS's earlier stage pipeline includes two calcilytic compounds, NPSP790 and NPSP795, with potential application in rare disorders involving increased calcium receptor activity, such as autosomal dominant hypocalcemia with hypercalciuria (ADHH). NPS complements its proprietary programs with a royalty-based portfolio of products and product candidates. 

Sucampo Pharmaceuticals Sucampo Pharmaceuticals, Inc. is an international biopharmaceutical company focused on developing and commercializing prostone-based medicines to meet the major unmet medical needs of patients on a global basis. 

Tranzyme Tranzyme Pharma is a late-stage biopharmaceutical company focused on discovering, developing and commercializing novel, mechanism-based therapeutics for the treatment of upper gastrointestinal (GI) motility disorders. Tranzyme is developing an intravenous drug, ulimorelin, for patients in acute (hospital-based) settings, as well as an oral drug (TZP-102) for chronic conditions. Ulimorelin is currently in Phase 3 clinical trials and TZP-102 is entering Phase 2b. Together these product candidates target a significant underserved market. 

Oncology

Aduro Biotech Aduro BioTech, Inc. is a clinical-stage immunotherapy company.  Our lead vaccine is currently under evaluation in a randomized, controlled Phase 2 clinical trial, in which the formal interim analysis is expected in Q1 2013.  In addition, we have a pipeline of other vaccines in clinical and pre-clinical development.

Cyclacel Pharmaceuticals Cyclacel is  a biopharmaceutical company developing oral therapies that target various phases of cell cycle control for the treatment of cancer and other serious diseases. 

Mirna Therapeutics  Mirna Therapeutics is focused on the development of microRNA-based therapeutics. The company intends to submit its first IND to FDA early 2013 and enter first clinical testing of the first miRNA mimic in cancer  shortly thereafter. To date, Mirna has raised > $35 M in private financing and > $ 15M in Federal and State funding.

Novelos Therapeutics Novelos Therapeutics, Inc. is a pharmaceutical company developing novel drugs for treatment and diagnosis of cancer. Novelos' three cancer-targeted compounds, which are selectively taken up and retained in cancer cells and cancer stem cells, versus normal cells, are being developed to find, treat and follow cancer anywhere in the body. 

OncoSec Medical OncoSec Medical Inc. is an advance stage biomedical company developing therapeutics to selectively destroy cancer tumors. OMI’s core technology is a novel delivery system using a form of cell membrane stimulation known as electroporation. Using this system to deliver a proven Chemotherapy or Immunotherapy, these therapies can effectively treat cancerous tumors while reducing or eliminating detrimental treatment outcomes associated with other “standard of care” therapies.

Sutro Biopharma Sutro Biopharma is developing a new generation of antibody drug conjugate therapeutics and bifunctional antibody-based therapeutics for targeted cancer therapies. Sutro has established a Good Manufacturing Practice (cGMP) facility for the production of clinical supplies of materials using its biochemical protein synthesis platform and is collaborating with pharmaceutical and biotech companies in the discovery and development of novel protein therapeutics.

Opthamology

pSiVida Corp. pSivida Corp., headquartered in Watertown, MA, develops tiny, sustained release, drug delivery products designed to deliver drugs at a controlled and steady rate for months or years. pSivida is currently focused on treatment of chronic diseases of the back of the eye utilizing its core technology systems, Durasert(TM) and BioSilicon(TM). 

Platform/Discovery

KineMed KineMed’s decision relevant information guides pharma R&D. Its Translational Biomarker Platform pin-points the function of thousands of proteins, all in a drop of blood,  capturing causal process  driving disease progression, and predicting clinical outcomes. KineMed successfully collaborates with leading investigators to enable better medicines and power personalized medicine.

Renal

Affymax Affymax is a biopharmaceutical company committed to developing novel drugs to improve the treatment of serious and often life-threatening conditions. The company’s first product candidate, peginesatide (formerly known as Hematide™), has been investigated in Phase 3 trials for the potential treatment of anemia associated with chronic kidney disease (CKD). A New Drug Application (NDA) for peginesatide for the treatment of anemia associated with CKD in adult patients on dialysis is currently being reviewed by the U.S. Food and Drug Administration (FDA).