Acasti Pharma
NASDAQ:ACSTAcasti Pharma Inc. engages in the development and commercialization of pharmaceutical products for rare and orphan diseases in Canada. The company's lead product candidate is the GTX-104, an intravenous infusion to treat subarachnoid hemorrhage. It also develops GTX-102, an oral mucosal spray for the treatment of ataxia-telangiectasia; and GTX-101, a topical bioadhesive film-forming bupivacaine spray for postherpetic neuralgia. The company was incorporated in 2002 and is headquartered in Laval, Canada.
Lipocine
NASDAQ:LPCNLipocine Inc., a clinical-stage biopharmaceutical company, engages in the research and development for the delivery of drugs for the treatment of central nervous system (CNS) disorders. Its lead product candidate is TLANDO, an oral testosterone replacement therapy (TRT) comprising testosterone undecanoate. The company's pipeline candidates also include TLANDO XR a candidate for oral TRT for once daily dosing, which has completed Phase 2b clinical study; LPCN 1148, an oral prodrug of bioidentical testosterone, being developed for the treatment of cirrhosis, currently under Phase 2 clinical studies; LPCN 1154, An oral neurosteroid, being developed for the treatment of postpartum depression, currently under Phase 2 studies; LPCN 2101, a NAS candidate, for women with epilepsy; and LPCN 2203 for essential tremor. It is also involved in the development of LPCN 1144, an oral prodrug of bioidentical testosterone for the treatment of pre-cirrhotic non-alcoholic steatohepatitis, which has completed Phase 2 testing; and LPCN 1107, an oral product candidate of 17-alpha-hydroxy progesterone caproate product, currently under Phase 3 studies for the prevention of recurrent preterm birth. The company was founded in 1997 and is headquartered in Salt Lake City, Utah.
Stealth BioTherapeutics
NASDAQ:MITOStealth BioTherapeutics Corp, a clinical-stage biotechnology company, focuses on the discovery, development, and commercialization of therapies for diseases involving mitochondrial dysfunction. Its lead product candidate is Elamipretide that is in phase 3 clinical trial to treat barth syndrome, phase 2/3 clinical trial for the treatment of duchenne cardiomyopathy, phase 2a clinical trial to treat friedreich's ataxia, phase 2 clinical trial for the treatment of dry age-related macular degeneration, phase 2 clinical trial to treat leber's hereditary optic neuropathy, and phase 3 clinical trial for the treatment of replisome myopathies. The company's product candidates also include SBT-20 to treat complications of diabetes, renal diseases, ocular diseases, neurodegenerative, Parkinson's, Alzheimer's, Huntington's, and ALS diseases; SBT-550 for rare neurological indications; and SBT-272, a peptidomimetic that targets the mitochondria and stabilizing mitochondrial function under conditions of oxidative stress, as well as SBT 550. Stealth BioTherapeutics Corp was incorporated in 2006 and is based in George Town, the Cayman Islands.
Synlogic
NASDAQ:SYBXSynlogic, Inc., a clinical-stage biopharmaceutical company, engages in the discovery and development of synthetic biotics to treat metabolic diseases in the United States. Its pipeline include SYNB1618, an orally administered, non-systemically absorbed drug candidate to treat phenylketonuria; SYNB1934, an orally administered, non-systemically absorbed drug candidate, which is in Phase III clinical trial to treat phenylketonuria; SYNB1353, an orally administered, non-systemically absorbed drug candidate, which is in Phase I clinical to treat homocystinuria; SYNB8802, an orally administered, non-systemically absorbed drug candidate that is in Phase II clinical trial for the treatment of enteric hyperoxaluria; and SYNB2081 to lower uric acid for the potential treatment of gout. The company has a collaboration agreement with F. Hoffmann-La Roche Ltd and Hoffmann-La Roche Inc. for the research and pre-clinical development of a synthetic biotic medicine for the treatment of inflammatory bowel disease; and Ginkgo Bioworks, Inc. Synlogic, Inc. is based in Cambridge, Massachusetts.
Vaccinex
NASDAQ:VCNXVaccinex, Inc., a clinical-stage biotechnology company, engages in the discovery and development of targeted biotherapeutics to treat cancer, neurodegenerative diseases, and autoimmune disorders. The company's lead drug candidate, pepinemab, a humanized monoclonal antibody that binds and blocks the activity of SEMA4D. It is developing Pepinemab, which is in phase 2 study for treatment of Alzheimer's disease and has completed phase 2 study for treatment of Huntington's disease; Pepinemab in combination with Pembrolizumab in phase 2 study for head and neck cancer, Avelumab in phase 2 study for pancreatic cancer, and completed phase 2 study with Avelumab for non-small cell lung cancer; and Pepinemab in combination with Nivolumab completed phase 2 study for melanoma, and trastuzumab and DC vaccine in phase 2 study for breast cancer. The company has also developed ActivMAb, an antibody drug discovery platform based on a novel method for complex targets, such as multi-pass membrane receptors or large and diverse libraries of full-length human monoclonal antibodies on the surface of pox viruses. It has collaborations with Merck Sharp & Dohme; Ares Trading S.A.; The Children's Hospital of Philadelphia; Emory University; Huntington Study Group; H. Lee Moffitt Cancer Center and Research Institute, Inc; Catalent Pharma Solutions, LLC; Surface Oncology, Inc.; and Pharmaceutical and Biotech Co. The company was incorporated in 2001 and is headquartered in Rochester, New York.