Biopharmaceuticals

AcelRx Pharmaceuticals, Inc.
Redwood City, California
AcelRx Pharmaceuticals, Inc. is a specialty pharmaceutical company focused on the development and commercialization of innovative therapies for the treatment of acute pain. The Company's late-stage pipeline includes ARX-04 (sufentanil sublingual tablet, 30 mcg) designed for the treatment of moderate-to-severe acute pain in a medically supervised setting; and Zalviso® (sufentanil sublingual tablet system) designed for the management of moderate-to-severe acute pain in adult patients in the hospital setting. ARX-04 delivers 30 mcg sufentanil, a high therapeutic index opioid, sublingually through a disposable, pre-filled, single-dose applicator. AcelRx has reported positive results from the pivotal Phase 3 SAP301 ambulatory surgery study, and has recently completed enrollment of SAP302 (study in emergency room patients) and SAP303 (study in post-operative patients 40 years and older). Zalviso delivers 15 mcg sufentanil sublingually through a non-invasive delivery route via a pre-programmed, patient-controlled analgesia device. In response to the New Drug Application (NDA) AcelRx submitted to the U.S. Food and Drug Administration (FDA) seeking approval for Zalviso, AcelRx received a Complete Response Letter (CRL) on July 25, 2014. The FDA has requested an additional clinical study (IAP312), which AcelRx is planning to initiate once supply testing is complete in order to support its NDA resubmission. Nasdaq: ACRX
Contact: Guy P. Nohra
Achaogen, Inc.
South San Francisco, California,
Achaogen is a clinical-stage biopharmaceutical company passionately committed to the discovery, development, and commercialization of novel antibacterials to treat MDR gram-negative infections. Achaogen is developing plazomicin, Achaogen's lead product candidate, for the treatment of serious bacterial infections due to MDR Enterobacteriaceae, including carbapenem-resistant Enterobacteriaceae. Achaogen's plazomicin program is funded in part with Federal funds from the Biomedical Advanced Research and Development Authority, Office of the Assistant Secretary for Preparedness and Response, Office of the Secretary, Department of Health and Human Services, under Contract No. HHSO100201000046C. Plazomicin is the first clinical candidate from Achaogen's gram-negative antibiotic discovery engine, and Achaogen has other programs in early and late preclinical stages focused on other MDR gram-negative infections. Nasdaq: AKAO
Contact: Farah Champsi
Aerie Pharmaceuticals, Inc.
Research Triangle Park, NC
Aerie is a clinical-stage pharmaceutical company focused on the discovery, development and commercialization of first-in-class therapies for the treatment of patients with glaucoma and other diseases of the eye. Aerie's two lead product candidates are once-daily IOP-lowering therapies with novel mechanisms of action to treat patients with glaucoma or ocular hypertension. It is expected that the NDA filing for RhopressaTM (netarsudil ophthalmic solution) 0.02% will take place in the third quarter of 2016. The second product candidate, RoclatanTM (netarsudil/latanoprost ophthalmic solution) 0.02%/0.005%, which is a fixed dose combination of RhopressaTM and widely prescribed PGA latanoprost, currently has two Phase 3 registration trials underway, named Mercury 1 and Mercury 2. If these trials are successful, a RoclatanTM NDA filing is expected to take place in the second half of 2017. Nasdaq: AERI
Contact: Dan Janney
Alba Therapeutics Corp.
Baltimore, MD
Alba Therapeutics is focused on the development of novel therapeutics for the treatment of inflammatory and immune mediated diseases caused by the malfunction of tight junctions in body. Initial target diseases are Celiac Disease and Type I Diabetes. The Company is in Phase II studies.
Contact: Dan Janney
Allakos
San Mateo, CA
Allakos is a clinical-stage company developing antibodies that selectively target mast cells and eosinophils, important effector cells in a broad spectrum of allergic and inflammatory conditions and several rare proliferative diseases. The company's lead antibodies are AK001 and AK002 intended for the treatment of severe allergic diseases and other diseases characterized by excess activity of mast cells and eosinophils. The company has raised a total of $47 million to-date in Series A financing from Novo Ventures, Alta Partners, RiverVest Venture Partners and the Roche Venture Fund.
Contact: Farah Champsi
Applied Genetic Technologies Corporation
Alachua, FL
AGTC is a clinical-stage biotechnology company that uses its proprietary gene therapy platform to develop products designed to transform the lives of patients with severe diseases, with an initial focus in ophthalmology. AGTC's lead product candidates focus on inherited orphan diseases of the eye, caused by mutations in single genes that significantly affect visual function and currently lack effective medical treatments. Nasdaq: AGTC
Contact: Ed Penhoet, Ph.D.
aTyr Pharma, Inc.
San Diego, CA
aTyr Pharma is engaged in the discovery and clinical development of innovative medicines for patients suffering from severe rare diseases using its knowledge of Physiocrine biology, a newly discovered set of physiological modulators. The Company's lead candidate, Resolaris™, is a potential first-in-class intravenous protein therapeutic for the treatment of rare myopathies with an immune component. Resolaris is currently in a Phase 1b/2 clinical trial in adult patients with facioscapulohumeral muscular dystrophy (FSHD); a Phase 1b/2 trial in adult patients with limb-girdle muscular dystrophy 2B (LGMD2B or dysferlinopathies) or FSHD; and a Phase 1b/2 trial in patients with an early onset form of FSHD. To protect this pipeline, aTyr built an intellectual property estate comprising over 70 issued or allowed patents and over 240 pending patent applications that are owned or exclusively licensed by aTyr, including over 300 potential Physiocrine-based protein compositions. aTyr's key programs are currently focused on severe, rare diseases characterized by immune dysregulation for which there are currently limited or no treatment options. Nasdaq: LIFE
Contact: Ed Penhoet, Ph.D.
Cerenis Therapeutics
Toulouse, France and Ann Arbor, MI
Cerenis Therapeutics is an international biopharmaceutical company dedicated to the discovery and development of innovative HDL therapies for the treatment of cardiovascular and metabolic diseases. HDL is the primary mediator of the reverse lipid transport, or RLT, the only natural pathway by which excess cholesterol is removed from arteries and is transported to the liver for elimination from the body. Cerenis is developing a portfolio of HDL therapies, including HDL mimetics for the rapid regression of atherosclerotic plaque in highrisk patients such as post-ACS patients and patients with HDL deficiency, and drugs which increase HDL for patients with low number of HDL particles to treat atherosclerosis and associated metabolic diseases. Cerenis is well positioned to become one of the leaders in the HDL therapeutic market, with a broad portfolio of programs being developed. Since its inception in 2005, the company has been funded by top tier investors: Sofinnova Partners, HealthCap, Alta Partners, EDF Ventures, Daiwa Corporate Investment, TVM Capital, Orbimed, IRDI/IXO Private Equity and Bpifrance (Fund for Strategic Investment) and last March successfully completed an IPO on Euronext raising €53.4m.
Cytokinetics, Inc.
South San Francisco, CA
Cytokinetics is developing novel pharmaceuticals and technologies based on its expertise in cytoskeletal pharmacology and biology. The Company is currently pursuing multiple therapeutic programs in the areas of cancer and cardiovascular diseases. It is in Phase II studies with small molecule therapeutics for solid tumors and for congestive heart failure.
Nasdaq: CYTK
Contact: Farah Champsi
Esperion Therapeutics, Inc.
Plymouth, MI
Esperion Therapeutics, Inc. is a late-stage pharmaceutical company focused on developing and commercializing oral therapies for the treatment of patients with elevated LDL-C. Through scientific and clinical excellence, and a deep understanding of cholesterol biology, the team at Esperion is committed to developing new LDL-C lowering therapies that will make a substantial impact on reducing global cardiovascular disease; the leading cause of death around the world. Bempedoic acid, the Company's lead product candidate, dramatically lowers LDL-C levels and could potentially reduce elevated levels of LDL-C in patients with hypercholesterolemia, especially those that can't tolerate statins. Nasdaq: ESPR
Contact: Dan Janney
Genedata AG
Basel, Switzerland
GeneData develops and commercializes bioinformatics systems and related services in the functional genomics arena. The Company delivers computational solutions for the management and analysis of large quantities of structurally complex biological data, generated by genome sequencing, microarrays, protein expression profiling, metabolic profiling and high-throughput screening.
Immune Design Corp.
Seattle, Washington
Immune Design is a clinical-stage immunotherapy company with next-generation in vivo approaches designed to enable the body’s immune system to fight disease. The Company’s technologies are engineered to activate the immune system’s natural ability to create and/or expand antigen-specific cytotoxic T cells, while enhancing other immune effectors, to fight cancer and other chronic diseases. Immune Design's clinical programs are the product of its two synergistic discovery platforms: ZVexTM and GLAASTM. Immune Design went public in July 2014. Nasdaq: IMDZ.
Contact: Ed Penhoet, Ph.D.
Intarcia Therapeutics, Inc.
Hayward, CA
Intarcia Therapeutics, Inc. is a privately held biopharmaceutical company developing therapies to ensure enhanced treatment outcome by optimizing patient adherence and improving the efficacy, convenience and tolerability of drug therapies. Intarcia's drug development expertise and competitive edge are demonstrated by its abilities to stabilize proteins and peptides at above body temperature and to deliver them in a constant and consistent manner via the proprietary DUROS drug delivery platform. Intarcia is pursuing a clinical stage development program for type 2 diabetes and has other programs for weight regulation to control obesity. The Company’s headquarters and manufacturing facilities are in Hayward, CA.
Contact: Farah Champsi
Kiadis Pharma
Amsterdam, The Netherlands
Kiadis Pharma is focused on cell-based immunotherapy products for the treatment of blood cancers and inherited blood disorders. The Company’s products have the potential to address the risks and limitations connected with allogeneic hematopoietic stem cell transplantation (HSCT), namely Graft-versus-Host-Disease (GVHD), cancer relapse, opportunistic infections and limited matched donor availability. The Company believes that HSCT could become a first-choice treatment for blood cancers, inherited blood disorders and possibly autoimmune diseases and solid organ transplantations. In April 2016, the Company reported positive Phase II results with its lead product ATIR101™ in patients with blood cancer. The data showed that ATIR101™ significantly reduced Transplant Related Mortality and significantly improved Overall Survival. In addition, ATIR101™ did not elicit grade III-IV GVHD in any patient. ATIR101™ has been granted Orphan Drug Designations both in the US and Europe. The Company’s second product candidate, ATIR201™, addresses inherited blood disorders with an initial focus on thalassemia, a disease which results in destruction of red blood cells in patients. ATIR201™ is expected to enter Phase I/II clinical development in the second half of 2016. Kiadis Pharma, based in Amsterdam, The Netherlands, was granted an Advanced Therapy Medicinal Product (ATMP) certificate for manufacturing quality and non-clinical data by the European Medicines Agency (EMA). The Company’s shares are listed on Euronext Amsterdam and Euronext Brussels.
Contact: Dan Janney
Kite Pharma, Inc.
Los Angeles, California
Kite Pharma, Inc., is a clinical-stage biopharmaceutical company engaged in the development of novel cancer immunotherapy products, with a primary focus on engineered autologous cell therapy (eACT™) designed to restore the immune system's ability to recognize and eradicate tumors. Nasdaq: KITE
Contact: Farah Champsi
Neothetics, Inc.
San Diego, California,
Neothetics is a clinical-stage specialty pharmaceutical company focused on development and commercialization of therapeutics for the aesthetic market. Our current focus is on localized fat reduction and body contouring. Neothetics product candidate, LIPO-202, is a potential injectable treatment for undesirable, localized areas of fat that requires no pain management before or after treatment, no downtime, and causes no damage to nearby tissues. Nasdaq: NEOT
Contact: Dan Janney
Prolacta Bioscience, Inc.
Monrovia, CA
Prolacta Bioscience, Inc. (Prolacta) is the pioneer in standardized human milk-based nutritional products for premature infants in the neonatal intensive care unit (NICU). Prolacta believes that there is no adequate replacement for human breast milk and, as such, we believe infant nutritional products should be human milk-based. As a privately held, for-profit and scientifically driven company, committed to improving premature infant nutrition, we are using human milk to change the standard of care in the NICU. Mothers who have a surplus of breast milk can help save the country’s most fragile infants by donating their extra breast milk through one of Prolacta’s affiliate milk banks.
Contact: Dan Janney
Scynexis, Inc.
Research Triangle Park, NC
SCYNEXIS is a pharmaceutical company committed to the development and commercialization of novel anti-infectives to address significant unmet therapeutic needs. We are developing our lead product candidate, SCY-078, as an oral and IV drug for the treatment of several fungal infections, including serious and life-threatening invasive fungal infections. Nasdaq: SCYX
Contact: Ed Penhoet, Ph.D.
Sunesis Pharmaceuticals, Inc.
South San Francisco, CA
Sunesis is a biopharmaceutical company focused on the development and commercialization of new oncology therapeutics for the potential treatment of solid and hematologic cancers. Sunesis has built a highly experienced cancer drug development organization committed to improving the lives of people with cancer and is currently pursuing regulatory approval in Europe for its lead product candidate, vosaroxin, for the treatment of relapsed or refractory acute myeloid leukemia in patients aged 60 and older. In addition, the company is advancing its kinase-inhibitor pipeline of novel targeted therapies into the clinic. Nasdaq: SNSS
Contact: Ed Penhoet, Ph.D.
Sutro Biopharma
South San Francisco, California
Sutro Biopharma, Inc. develops best-in-class antibody drug conjugate and multi-specific antibody-based therapeutics for cancer therapy, including Immuno-Oncology therapies. Sutro’s discovery and development efforts are driven by our proprietary Xpress CF™ and Xpress CF+™ platforms, a biochemical synthesis system that enables rapid and systematic evaluation of protein structure-activity relationships, as well as rapid and predictable scalability for manufacturing in Sutro’s cGMP facility. In addition to developing its own drug candidate pipeline, which is focused on mono- and bi-specific ADCs, Sutro Biopharma is collaborating with select pharmaceutical and biotech companies to discover and develop novel therapeutics.
Contact: Dan Janney
Threshold Pharmaceuticals, Inc.
Redwood City, CA
Threshold is a clinical-stage biopharmaceutical company focused on the discovery and development of drugs and diagnostic agents targeting tumor hypoxia, the low oxygen condition found in microenvironments of most solid tumors as well as the bone marrows of some hematologic malignancies. This approach offers broad potential to treat a variety of cancers. By selectively targeting tumor cells, they are building a pipeline of drugs that hold promise to be more effective and less toxic to healthy tissues than conventional anticancer drugs. Nasdaq: THLD
Trevena, Inc.
King of Prussia, Pennsylvania
Trevena, Inc. is a clinical stage biopharmaceutical company that discovers, develops and intends to commercialize therapeutics that use a novel approach to target G protein coupled receptors, or GPCRs. Using its proprietary product platform, Trevena has identified three biased ligand product candidates – oliceridine (TRV130) to treat moderate to severe acute pain intravenously (Phase 3), TRV734 to treat moderate to severe acute and chronic pain orally (Phase 1), and TRV250 for acute episodic migraine and other CNS disorders (preclinical). Nasdaq: TRVN
Contact: Farah Champsi
ViroBay, Inc.
Menlo Park, California
ViroBay is a biotechnology company focused on developing novel HCV protease and polymerase inhibitors.
Contact: Dan Janney