Below please find descriptions provided by company presenters, categorized by therapeutic category.
Autoimmune/Inflammation | Biodefense | Bioprinting Technology | CNS | Drug Discovery | Gastroenterology | Gene/Cell Therapy | Hormone Therapy | Infectious Diseases | Metabolic Diseases | Oncology | Opthamology | Platform Technology | Regenerative Medicine | Urology | Vaccines
Autoimmune/Inflammation
Applied Microarrays AMI develops and manufactures high value medical diagnostics devices, in multiple OEM partnership arranagements, using protein and DNA microarray technologies. AMI owns a high capacity microarray factory acquired from GE in 2007, is staffed with an expert team of scientists, engineers and business developers, and is ISO certified for medical devices. Current OEM partnerships include products use silicon and other technologies for rapid-answer Point-of-Care applications. In other partnerships high capacity microtiter plate arrays for Central Lab use are manufactured, providing a solution that is lower cost and superior performance than Luminex bead arrays.
Asmacure Asmacure Ltée is a clinical-stage biopharmaceutical company focused on the development of molecules that target cholinergic receptors for the treatment of inflammatory diseases.
Aurinia Pharmaceuticals Aurinia Pharmaceuticals is a spin-out from Vifor Pharma, the pharmaceutical arm of Galenica Holdings Inc. The company is primarily made up of the former Aspreva team that executed on one of the largest Lupus Nephritis studies ever conducted. Aurinia has licensed an asset that has utility in treating LN and plan to initiate a phase IIB study in 2013.
Celtaxsys Celtaxsys is a privately-held clinical stage drug discovery and development company with operations in San Francisco, CA and Atlanta, GA. The Company is focused on creating therapeutic treatments for inflammatory diseases by modulating innate immunity, with special emphasis on the neutrophil. Celtaxsys is building a sustainable pipeline of innate immunity drugs. Their lead program, CTX-4430, is currently staged for entry into Phase 1 clinical trials. Two additional lead compounds emanating from Celtaxsys' technology platform are currently undergoing preclinical optimization.
Complexa Complexa is a 4-year-old biopharmaceutical company applying endogenous nitro-fatty-acid technology to the treatment of various inflammatory and metabolic based diseases. The Company expects to have its first IND approved in mid-2013 for the treatment of a form of AKI (Acute Kidney Injury) that currently has no effective treatment. Complexa completed its Series A financing in 2012 which has been applied to the completion of its first pre-clinical program.
Biodefense
Terapio Corporation Terapio is a biopharmaceutical company developing therapeutics based on the membrane-associated RLIP76 protein. Initial biodefense applications include a countermeasure for radiation exposure and chemical threats to civilian, military, and first responder populations. The unique mechanism of action of the RLIP76 protein also supports the Company’s commercial pipeline for oncology supportive care and diseases of the central nervous system.
Bioprinting Technology
Organovo Organovo creates functional living human tissue using its breakthrough 3D bioprinting technology that was selected as one of the "Best Inventions of 2010" by TIME Magazine. Organovo is helping pharmaceutical partners develop human biological disease models in three dimensions that enable therapeutic drug discovery and development. Organovo's bioprinting technology can also be developed to create surgical tissues as direct therapy.
CNS
Allon Therapeutics Allon Therapeutics Inc. is a clinical-stage biotechnology company focused on bringing to market innovative central nervous system therapies. Allon’s lead drug, davunetide, is proceeding in a fully enrolled pivotal Phase 2/3 clinical trial in an orphan indication, progressive supranuclear palsy (PSP), under a Special Protocol Assessment (SPA) with the Food and Drug Administration (FDA). This pivotal trial is based upon statistically significant human efficacy demonstrated in amnestic mild cognitive impairment, cognitive impairment associated with schizophrenia, and positive biomarker data.
BioCrea BioCrea turns concepts into drug candidates. BioCrea is creating new drugs for the treatment of debilitating diseases of the central nervous system. With industry background and an international leadership team BIOCREA provides new, first-in-class drug candidates for pharmaceutical companies.
Cognition Therapeutics Cognition Therapeutics has pioneered the discovery and development of new drugs acting at new targets to halt disease progression and improve symptoms in neurodegenerative disorders. Their disease-relevant screening and novel chemistry platforms have produced a pipeline of new approaches for protein-based nervous system diseases. CogRx’s leading preclinical program is a highly brain-penetrant orally bioavailable small molecule receptor antagonist that stops memory deficits in Alzheimer’s disease.
FPRT Bio FPRT Bio is a clinical stage company formed to develop XPro1595 to treat neurodegenerative diseases including ALS, Parkinson's disease and Alzheimer's disease. XPro1595 is a selective inhibitor of soluble TNF that is anti-inflammatory, neuroprotective and promotes remyelination – a combination of therapeutic attributes that is not found in any drug on the market or in development. Funds from the Series A will be used to complete the Phase I clinical program in ALS and Parkinson’s disease.
Vallinex Vallinex Inc. is a privately-held private biopharmaceutical company focused on the development of VLN-4975 (capsaicin for injection) for the treatment of chronic pain. Initial focus is treatment of knee osteoarthritis (knee OA).
Drug Discovery
Inception Sciences Inception Sciences is a small molecule pharmaceutical incubator translating breakthrough scientific insights into novel therapies. The Inception Sciences team consists of seasoned drug discoverers who have produced marketed drugs and high-value liquidity events at Merck and at Amira Pharmaceuticals. In collaboration with pioneering academic researchers, Versant Ventures and pharmaceutical companies, Inception Sciences creates new start-up companies dedicated to each novel drug discovery program.
Gastroenterology
Tranzyme Pharma 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. While approximately 40 percent of people in the U.S. are affected by these persistent and recurring conditions which disrupt the normal movement of food throughout the GI tract, currently there are a limited number of safe and effective treatment options. Tranzyme is developing TZP-102, an oral ghrelin agonist for treating the symptoms associated with chronic upper GI motility disorders. This product candidate targets a significant underserved market. Enrollment in a multinational, Phase 2b trial is ongoing; top-line data are expected by year-end 2012. By leveraging its proprietary drug discovery technology, Tranzyme is committed to pursuing first-in-class medicines to address areas of significant unmet medical needs.
Gene/Cell Therapy
Senesco Senesco is a cancer therapeutics company and a leader in eukaryotic translation initiation Factor 5A (eIF5A) technology to regulate cell death and survival. The Company's lead therapeutic candidate SNS01-T incorporates recent developments in siRNA, DNA regulation and nanotechnology. Senesco is building its portfolio with other potential eIF5A applications in cancer, inflammation and ischemia.
Hormone Therapy
BioSante Pharmaceuticals BioSante is a specialty pharmaceutical company focused on developing products for female sexual health and oncology. BioSante´s products include LibiGel® (transdermal testosterone gel) for the treatment of female sexual dysfunction (FSD), specifically hypoactive sexual desire disorder (HSDD), which is in Phase III clinical development. BioSante also is developing a portfolio of cancer vaccines, with 17 Phase I and Phase II clinical trials currently on-going. Four of these vaccines have been granted Orphan Drug designation by the U.S. Food and Drug Administration (FDA). BioSante´s other products include an FDA-approved testosterone gel for male hypogonadism, which is licensed to Teva Pharmaceuticals USA, Inc., and the Pill-Plus™, an oral contraceptive in Phase II clinical development by Pantarhei Bioscience B.V. BioSante´s first FDA-approved product, Elestrin™ (estradiol gel) indicated for the treatment of hot flashes associated with menopause, is marketed in the U.S. by Jazz Pharmaceuticals, BioSante's licensee. Additional information is available online at: www.biosantepharma.com.
Infectious Diseases
Durata Therapeutics Durata Therapeutics is a pharmaceutical company focused on the development and commercialization of novel therapeutics for patients with infectious diseases and acute illnesses. Durata is currently enrolling and dosing patients in two global Phase 3 clinical trials with its lead product candidate, dalbavancin, for the treatment of patients with acute bacterial skin and skin structure infections, or abSSSI.
HeatFlow Technologies HeatFlow Technologies created, patented, and owns the next generation of quantitative real-time PCR (qPCR) technology. Heat Flow Polymerase Chain Reaction (HF-PCR™) is a novel, user-friendly, and cost-effective method of qPCR analysis, free of fluorescent dyes and optical equipment. The Company is developing its HF-PCR device, FireCycler™, and assays, including an HIV viral load assay suitable for point of care settings.
Metabolic Diseases
Profil Institute for Translational Medicine Profil Institute for Translational Medicine is a drug development company focused on transitioning compounds from preclinical through early phase clinical trials by way of risk-sharing collaborations and licensing agreements. The company is a wholly owned subsidiary of privately held Profil Institute for Clinical Research, a highly regarded Center of Excellence for early phase clinical research of drugs and devices for metabolic diseases.
Oncology
Affimed Therapeutics AG Affimed is a clinical stage company with a portfolio of antibody candidates for treatment of diseases with high unmet medical need in oncology and autoimmune diseases. These candidates are derived from Affimed´s proprietary TandAb platform. Tand Abs are tetravalent, bispecific antibody formats with improved efficacy and safety profile.
CytRx Corporation CytRx is an oncology-focused biopharmaceutical company with three drug candidates: aldoxorubicin (formerly INNO-206), bafetinib and tamibarotene. Each candidate is either in or completed phase 2 clinical trials.
Epizyme Epizyme is leading the creation of small molecule histone methyltransferase inhibitors (HMTi), a new class of personalized therapeutics for the treatment of patients with genetically defined cancers, based on breakthroughs in the field of epigenetics. Genetic alterations in HMTs, a family of epigenetic enzymes, drive multiple human diseases. Epizyme's approach represents the future of healthcare by matching better medicines with the right patients.
MabVax Therapeutics MabVax is a clinical stage biotechnology company focused on the development of vaccines and fully human antibodies to address the unmet medical need of preventing recurrent cancer. MabVax has two separate vaccines in POC Phase II clinical trials aimed at preventing recurrent sarcoma and ovarian cancer. The Company's fully human antibody 5B1 has demonstrated very positive results in preclinical studies aimed at treating metastatic pancreatic and colon cancer and is headed for GMP production of Phase I clinical material early next year.
MEI Pharma MEI Pharma, Inc. (Nasdaq:MEIP) is a San Diego-based oncology company focused on the clinical development of novel therapeutics targeting cancer metabolism. The Company's lead drug candidates, ME-143 & ME-344, have been shown in pre-clinical stuides to interact with specific enzyme targets resulting in inhibition of tumor cell metabolism, a function critical for cancer survival. ME-143 & ME-344 are currently being investigated in Phase 1 clinical trials in patients with solid refractory tumors.
Novelos Therapeutics Novelos Therapeutics is a pharmaceutical company developing novel drugs for the treatment and diagnosis of cancer. Novelos Therapeutics' cancer-targeted compounds are selectively taken up and retained in cancer cells, including cancer stem cells, versus normal cells. Thus, their therapeutic compounds appear to directly kill cancer cells while minimizing harm to normal cells. This offers the potential for a paradigm shift in cancer therapy by providing efficacy versus all three major drivers of mortality in cancer: primary tumors, metastases and stem cell-based relapse.
Vaccinogen Vaccinogen, Inc. is a cancer vaccine company developing OncoVAX® as a treatment to prevent the recurrence of colon cancer and potentially other solid tumors. Vaccinogen has developed proprietary technology to use the patient’s own live tumor cells as the source of the vaccine. OncoVAX has completed five dose and regimen finding clinical studies, including one Phase III trial with the optimum dose and regimen and will begin a pivotal phase III trial under an FDA Special Protocol Acceptance (SPA) classification, by the end of 2012. More information is available at www.vaccinogeninc.com.
Opthamology
Ohr Pharmaceutical Ohr Pharmaceutical Inc. (www.ohrpharmaceutical.com) is a pharmaceutical company dedicated to the clinical development of new drugs for underserved therapeutic needs in large and growing markets. The company is focused on two lead compounds currently being investigated in clinical phase II trials: Squalamine Eye Drops for the treatment of the wet form of age-related macular degeneration, and OHR/AVR118 for the treatment of cancer cachexia.
Platform Technology
Broadvector Limited Broadvector Limited is an Australian biotech developing clinical stage minimally invasive, vector-based therapies. Broadvector's lead therapy, BDV102 is a Phase II stage, minimally invasive gene directed enzyme prodrug therapy (GDEPT) that eliminates synovial-like tissue allowing in situ repair of prosthetic implants, without the need for general anesthesia.
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.
RXi Pharmaceuticals RXi Pharmaceuticals Corporation is a biotechnology company focused on discovering, developing and commercializing innovative therapies based on its proprietary, next-generation RNAi platform. Therapeutics that use RNA interference, or “RNAi,” have great promise because of their ability to “silence,” or down-regulate, the expression of a specific gene that may be overexpressed in a disease condition. Building on the pioneering work of scientific founder and Nobel Laureate Dr. Craig Mello, RXi’s first RNAi product candidate, RXI-109, entered into RXi’s first human clinical trial in June 2012. RXI-109 targets connective tissue growth factor (CTGF), a key regulatory component of fibrosis and scar formation, and is initially being developed to reduce or inhibit scar formation in the skin following surgery.
Serina Therapeutics Serina Therapeutics, Inc. has developed a novel, proprietary polymer technology based on polyoxazoline (POZ). Drugs can be attached to these polymers to create POZ-conjugates for a host of unmet diseases. The company has focused on two lead clinical programs – Parkinson’s disease and oncology. The company is advancing these through IND-enabling studies now with plans to introduce these into the clinic in 2013-2014. Proof-of-concept studies in pre-clinical models will be presented at the BIO Investor Forum meeting during the week of October 9-12, 2012.
Regenerative Medicine
TissueGene TissueGene is a Maryland-based biopharmaceutical company focused on developing noninvasive regenerative therapies for the treatment of various orthopedic diseases. The Company’s core technology employs a form of cell therapy to deliver therapeutic proteins to localized areas of damaged tissue and is currently developing four products for the regeneration of cartilage, bone, disc and nerve tissue. TissueGene's lead product candidate, TG-C, has been developed for cartilage regeneration in patients with severe osteoarthritis of the knee and is currently in Phase II clinical trials in both the U.S. and Korea.
Urology
TheraVida TheraVida is developing combination drug products for the treatment of overactive bladder. Their lead product, THVD-201, is patent-protected and has completed Phase 2 studies.
Wellesley Pharmaceuticals Wellesley has developed a novel combination therapy for the treatment of nocturia, or excessive urination at night. Wellesley has discovered that certain off-patent, FDA-approved very safe drugs can be combined and reformulated at low dosages to significantly reduce dangerous nocturia symptoms, which afflict close to 2 billion people on a nightly basis. The first two of the Company's 18 patents pending were issued in August.
Vaccines
GeoVax Labs Inc. GeoVax Labs, Inc. (OTCQB: GOVX) is a clinical-stage biotechnology company developing vaccines for HIV/AIDS. Their vaccines are being evaluated to determine their potential both to (a) prevent HIV infection and (b) to serve as a treatment for individuals who are already infected with HIV. GeoVax Labs' vaccine technology was developed in collaboration with researchers at Emory University, the National Institutes of Health (NIH), and the Centers for Disease Control and Prevention (CDC).
NT Pharma LLC NT Pharma is a Russian biopharmaceutical company developing several exclusive genetic vaccines and biopharmaceuticals on the basis of adenoviral vectors. The new patented technology provides delivery of target genes and a specific molecule into human cells. The production facility is located in Moscow, Russia, and a large state-of the-art pharmaceutical plant is under construction 85 miles away from Moscow.
Jan M. Lundberg, Ph.D, Eli Lilly
Simone Fishburn, PhD
