Achillion Pharmaceuticals

New Haven, CT

Nasdaq: ACHN

Website

Achillion is an innovative biopharmaceutical company dedicated to bringing important new treatments to patients with infectious disease. The company is developing three clinical candidates for the treatment of chronic hepatitis C infection. ACH-1625 is a proprietary potent once-daily NS3 protease inhibitor that is currently in Phase II clinical development. ACH-2684 is a highly potent pan-genotypic inhibitor of HCV NS3 protease with pico-molar potency that is currently in Phase 1 clinical development. ACH-2928 and ACH-3102 are potent inhibitors of HCV NS5A that demonstrate pan-genotypic activity.

Afferent Pharmaceuticals

Palo Alto, CA

Website

Afferent Pharmaceuticals is focused on developing first-in-class medicines to treat chronic pain by targeting P2X3 receptors in peripheral nerves, where pain-causing stimuli are detected. As a result, it does not expect its drugs to be as prone to central nervous system side effects. The non-opiate mechanism also offers the potential to avoid dependence that limits the use of other pain medications. The company’s lead compound, AF-219, has successfully completed two Phase 1 clinical studies, and is expected to undergo efficacy testing in several pain and pain-related indications.

Anthera Pharmaceuticals

San Mateo, CA

Nasdaq: ANTH

Website

Anthera Pharmaceuticals is a pharmaceutical development company focused on the advancement of promising clinical products for the treatment of serious inflammatory diseases. Anthera's lead products are inhibitors of secretory phospholipase A2. The lead product is in Phase 3 development for acute coronary syndrome and a second program is in Phase 2b trials to treat patients with lupus.

Arena Pharmaceuticals

San Diego, CA

Nasdaq: ARNA

IPO (2000)

Website

Biopharmaceutical company that has developed a technology to accelerate the development of lead compounds targeting G-protein coupled receptors. This core technology, called CART, allows for ligand-independent, direct identification of small molecule compounds that regulate the activity of G-protein coupled receptors.

Athersys

Cleveland, OH

Nasdaq: ATHX

Website

Athersys is a biopharmaceutical and regenerative medicine company that has established a pipeline of therapeutic product development programs in multiple disease areas. The company’s product portfolio consists of MultiStem, a non-embryonic stem cell-based approach for treating certain inflammatory disorders, cardiovascular disorders, stroke, and bone marrow transplantation and oncology support.

Bayhill Therapeutics

San Mateo, CA

Website

Bayhill is a biopharmaceutical company focused on the translation of research into therapeutics for the treatment of autoimmune diseases. The company is developing DNA-antigen vaccines and oligonucleotides that are specifically designed to promote immune intolerance. Bayhill's lead compound, an antigen-specific immunotherapy aimed at treating multiple sclerosis, has completed a Phase 2 trial. The company also has a Type 1 diabetes program in a Phase 1/2 trial.

BrainCells

San Diego, CA

Website

BrainCells (BCI) is a leading-edge neurogenesis-based drug discovery and development company targeting novel therapies for depression, mood disorders and other CNS diseases. The company is developing a neurogenesis platform for profiling and selecting drug candidates. BCI believes that the platform represents a major improvement in the predictive power of pre-clinical models for CNS disorders and will facilitate a paradigm shift in the way CNS drugs are discovered.

Calyx Therapeutics

Hayward, CA

Pharmaceutical company that focused on oral therapies for diabetes and inflammatory diseases, utilizing natural plant extracts with demonstrated clinical activity in humans as the source of its discovery efforts.

CeNeRx BioPharma

Cary, NC

Website

CeNeRx is developing innovative treatments for diseases of the central nervous system. Their lead product, a reversible inhibitor of monoamine oxidase, is in a Phase 2 trial for treatment resistant depression. CeNeRx also has a pipeline of selective cannabinoid compounds for the treatment of pain, glaucoma, and obesity.

Cerexa

Alameda, CA

Acquired by Forest Laboratories in 2007

Website

Cerexa, Inc. is a privately owned biopharmaceutical company focused on acquiring, developing, and commercializing hospital-based anti-infective therapies for the treatment of patients with serious, life-threatening infections. The Company was formed in the Spring of 2005 as a spin-out from Peninsula Pharmaceuticals in preparation for the acquisition of Peninsula by Johnson & Johnson.

Chimerix

Durham, NC

Website

Chimerix is developing a range of antiviral medicines for the treatment of life-threatening diseases. Its product candidates derive from Phospholipid Intramembrane Microfluidization Conjugate Technology, which can enhance the activity, bioavailability and safety of useful drugs. Chimerix’s lead compound, CMX001, is a broad-spectrum oral antiviral agent initially being developed for the treatment of CMV and AdV5 in transplant patients.

Cognetix

Salt Lake City, UT

Biopharmaceutical company that developed peptide therapeutics for the treatment of epilepsy, pain and anesthesia.

CoLucid Pharmaceuticals

Indianapolis, IN

Website

CoLucid Pharmaceuticals is a clinical stage biopharmaceutical company developing therapeutics to treat neurological dysfunctions. CoLucid’s lead program is based on a potent and selective 5-HT1F receptor agonist that has the potential to be useful in the acute treatment and/or prevention or prophylaxis of migraine headaches. The company also possesses a robust chemistry platform that will produce unique bifunctional neuromodulators to treat indications such as Alzheimer's disease.

Dynogen Pharmaceuticals

Waltham, MA

Clinical-stage pharmaceutical company that developed a portfolio of therapies for gastrointestinal and genitourinary disorders.

Elitra Phramaceuticals

San Diego, CA

Antimicrobial functional genomics company that focused on the identification, development and commercialization of novel antimicrobial compounds that target essential gene products of pathogenic organisms. Assets acquired by Merck in 2004.

LEAD Therapeutics

San Francisco, CA

Website

LEAD Therapeutics is a chemistry-driven small-molecule drug discovery company, pursuing validated drug targets with rapid and efficient lead-optimization strategies. The company is leveraging its highly experienced, U.S.-based scientific team with high-quality, lower-cost chemistry capabilities in China. LEAD’s pipeline in oncology consists of a PARP inhibitor program and a Cyp17 inhibitor program. LEAD also has a glycopeptide program for infectious diseases.

Liquidia Technologies

Research Triangle Park, NC

Website

Liquidia utilizes a proprietary technology platform to design and manufacture highly precise nanoparticles that allow for the delivery of biological and small molecule vaccines and therapeutics. The company’s technology makes it possible for the first time to design vaccines and therapies with precise control of particle size, shape, and composition.

Lumena Pharmaceuticals

Durham, NC

Website

Lumena is focused on developing treatments for metabolic diseases through non-systemically absorbed compounds having targets within the lumen of the gut.

Marina Biotech

Bothell, WA

Nasdaq: MRNA

Website

Marina Biotech is a biotechnology company focused on the development and commercialization of therapeutic products based on RNA interference (RNAi). The company is currently employing two proven RNAi platforms to down-regulate the expression of specific proteins that cause disease: TauRNAi and TransKingdom RNAi. These approaches provide the flexibility to optimize oral, systemic and local delivery of RNAi-based therapies to target a wide range of human diseases based on the unique characteristics of the cells and organs involved in each disease.

MethylGene

Montreal, Quebec

TSX: MYG

Website

MethylGene is a biopharmaceutical company focused on the discovery, development and commercialization of inhibitors for enzymes involved primarily in oncologic and infectious disease indications. The company uses functional genomics to validate enzyme targets and uses rational drug design and medicinal chemistry to design, synthesize and optimize enzyme inhibitors.

Milestone Pharmaceuticals

Montreal, Quebec

Website

Milestone Pharmaceuticals is a drug development company developing novel small molecule therapeutics based on clinically validated mechanisms for cardiovascular diseases. The company’s lead program is focused on developing a novel short-acting treatment for acute episodes of paroxysmal supraventricular tachycardia (PSVT) and its lead product is a derivative of an approved drug for the target indication.

Nereus

San Diego, CA

Website

Drug discovery and development company focusing on the oceans and marine environments as a source of small-molecule compounds for the treatment of inflammation, cancer, and infectious diseases. Nereus is utilizing specialized research out of the Scripps Institution of Oceanography.

Panacos Pharmaceuticals

Watertown, MA

Reverse merger in 2005

Website

Panacos engaged in the discovery, development and commercialization of small molecule drugs to treat or prevent serious infectious disease. Its lead antiviral product is PA-457, the first in a new class of drugs to treat HIV infection.

Peninsula Pharmaceuticals

Alameda, CA

Acquired by Johnson & Johnson in 2005

Pharmaceutical development company focused on in-licensing and developing clinical stage pharmaceutical products, including those aimed at treating infectious diseases.

Plexxikon

Berkeley, CA

Website

Plexxikon is a leader in the discovery and development of novel, small molecule pharmaceuticals. The company has utilized its proprietary discovery platform to successfully develop a portfolio of clinical and preclinical stage programs in a number of therapeutic areas, including cardio-renal disease, CNS, inflammation, metabolic disease and oncology. To date, Plexxikon has signed major collaborations with Roche and Wyeth.

Reprogenesis

Cambridge, MA

Merged with Ontogeny and Creative BioMolecules to form Curis (2000)

Website

Company developing in vivo tissue augmentation/repair products. Reprogenesis merged in July 2000 with Creative Biomolecules and Ontogeny to form Curis, a public company focused on regenerative medicine. Curis is developing products that are designed to recreate the conditions that support the growth of cells and tissue.

Signase

Houston, TX

Cancer therapeutics company that developed novel, small molecule inhibitors of a critical cell replication pathway to inhibit tumor growth. Signase was spun out of the M. D. Anderson Cancer Center. Assets acquired by Systems Medicine Inc. in 2005.

Spherics

Mansfield, MA

Pharmaceutical company focused on applying its oral drug delivery platform to develop products with improved therapeutic profiles.

Syndax Pharmaceuticals

Waltham, MA

Website

Syndax is an oncology-focused biopharmaceutical company that is developing novel therapies in optimized, mechanistically driven combination regimens. The company has completed two randomized/placebo-controlled trials with entinostat, a histone deacetylase inhibitor (HDACi) for breast cancer and lung cancer.

Syntonix Pharmaceuticals

Waltham, MA

Acquired by BiogenIDEC (2007)

Website

Syntonix was formed to discover and develop novel therapeutic proteins for treatment of chronic diseases such as hemophilia, anemia, multiple sclerosis, and autoimmune disorders.

TargeGen

San Diego, CA

Website

TargeGen is a biopharmaceutical company focusing on vascular biology, which includes controlling vascular leakage (responsible for organ damage) and angiogenesis (new blood vessel growth). The company is in a Phase 1 trial with its Jak2 inhibitor for treatment of myeloproliferative disease.

TESARO

Waltham, MA

Website

TESARO is an oncology-focused biopharmaceutical company focused on acquiring and developing promising drug candidates. TESARO is developing rolapitant, a potent, selective neurokinin-1 receptor antagonist that has commenced Phase 3 clinical testing for the prevention of chemotherapy induced nausea and vomiting, and is advancing its ALK inhibitor program for oncology indications. TESARO was co-founded by former executives of MGI PHARMA, an oncology and acute-care focused biopharmaceutical company that Eisai Co., Ltd. acquired in 2008 for $3.9 billion.

UltraGenyx Pharmaceutical

Novato, CA

Website

Ultragenyx is a privately held biotechnology company focused on rare genetic disorders, founded by Emil Kakkis, MD, PhD, the former CMO of BioMarin, who is widely recognized as a leader in the rare disease field. Its mission is to build a first-in-class rare disease company addressing proteins or small molecules with clear disease targets and treatment mechanisms which have been overlooked and are ready for development. Its lead program for a substrate replacement therapy for Hereditary Inclusion Body Myopathy (HIBM) has initiated Phase 1 trials.

Variagenics

Cambridge, MA

Nasdaq: NUVO

IPO (2000)

Website

Pharmacogenomics company with proprietary gene variance detection technology anticipated to streamline drug development.

X-Ceptor Therapeutics

San Diego, CA

Acquired by Exelixis in 2004

Website

Genomics-based drug discovery company focused on the discovery and development of novel therapeutics across various therapy areas, including metabolic and cardiovascular diseases.