Abeona Therapeutics
NASDAQ:ABEOAbeona Therapeutics Inc., a clinical-stage biopharmaceutical company, focuses on developing and delivering gene therapy products for severe and life-threatening rare diseases. The company's lead programs are EB-101 (gene-corrected skin grafts) for recessive dystrophic epidermolysis bullosa (RDEB); ABO-102, which are AAV based gene therapies for Sanfilippo syndrome type A; and ABO-101, an adeno-associated virus (AAV) based gene therapies for Sanfilippo syndrome type B. It is also developing ABO-201 gene therapy for juvenile Batten disease; ABO-202 gene therapy for treatment of infantile Batten disease; EB-201 for for epidermolysis bullosa (EB); ABO-301 for Fanconi anemia disorder; and ABO-302 using a novel CRISPR/Cas9-based gene editing approach to gene therapy program for rare blood diseases. Further, it is involved in marketing MuGard, a mucoadhesive oral wound rinse for the management of mucositis, stomatitis, aphthous ulcers, and traumatic ulcers. Abeona Therapeutics Inc. has collaborations with EB Research Partnership and Epidermolysis Bullosa Medical Research Foundation that focus on gene therapy treatments for EB; and Brammer Bio for commercial translation of ABO-102. The company was formerly known as PlasmaTech Biopharmaceuticals, Inc. and changed its name to Abeona Therapeutics Inc. in June 2015. Abeona Therapeutics Inc. was incorporated in 1989 and is based in Dallas, Texas.
Agile Therapeutics
NASDAQ:AGRXAgile Therapeutics, Inc., a women's healthcare company, researches, develops, and commercializes prescription contraceptive products for women in the United States. Its lead product is Twirla, also known as AG200-15, a once-weekly prescription combination hormonal contraceptive patch. The company is also developing a pipeline of Twirla line extensions and other products, including AG200-15 Extended Regimen (ER), a regimen that allows a woman to have four episodes of withdrawal bleeding per year; AG200-15 smaller patch (SmP), which is a regimen designed to provide shorter and lighter withdrawal bleeds, and enhance contraceptive efficacy; AG200-15 ER SmP, a regimen to allow a woman to extend the length of her contraceptive cycle, as well as have shorter and lighter withdrawal bleeding episodes per year; and P-Patch, a progestin-only contraceptive patch intended for use by women who are unable or unwilling to take estrogen. The company was incorporated in 1997 and is headquartered in Princeton, New Jersey.
Hookipa Pharma
NASDAQ:HOOKHOOKIPA Pharma Inc., a clinical-stage biopharmaceutical company, develops immunotherapeutics targeting infectious diseases and cancers based on its proprietary arenavirus platform. The company's lead infectious disease product candidate is HB-101, which is in a randomized double-blinded Phase 2 clinical trial in cytomegalovirus-negative patients awaiting kidney transplantation from cytomegalovirus-positive donors. Its lead oncology product candidates are HB-201 and HB-202 that are in preclinical studies for the treatment of human papillomavirus-positive cancers. The company has a collaboration with Gilead Sciences, Inc. to develop arenavirus based therapeutics to treat hepatitis B virus and human immunodeficiency virus infections. HOOKIPA Pharma Inc. was founded in 2011 and is headquartered in New York, New York.
Milestone Pharmaceuticals
NASDAQ:MISTMilestone Pharmaceuticals Inc., a biopharmaceutical company, focuses on the development and commercialization of cardiovascular medicines. The company's lead product candidate is etripamil, a novel and potent calcium channel blocker, which is in Phase III clinical trial for the treatment of paroxysmal supraventricular tachycardia in the United States and Canada; and Phase II clinical trial for the treatment of atrial fibrillation and rapid ventricular rate. It has a license and collaboration agreement with Ji Xing Pharmaceuticals to develop and commercialize etripamil prophylactic and therapeutic uses in humans, as well as with the Montreal Heart Institute, the WCN network, and other research centers. The company was incorporated in 2003 and is headquartered in Montréal, Canada.
Verastem
NASDAQ:VSTMVerastem, Inc., a development-stage biopharmaceutical company, focuses on developing and commercializing drugs for the treatment of cancer in the United States. Its product candidates are Avutometinib, an orally available small molecule RAF/MEK clamp that inhibits the ras sarcoma RAF/MEK, ERK mitogen activated pathway kinase pathway which is involved in cell proliferation, migration, transformation, and survival of tumor cells; and Defactinib, an oral small molecule inhibitor of FAK and proline-rich tyrosine kinase for various solid tumors. The company is involved in clinical studies, including RAMP 301, a randomized global confirmatory trial to evaluate the combination of Avutometinib and Defactinib for the treatment of patients with recurrent low-grade serous ovarian cancer; RAMP 201, an adaptive two-part multicenter, parallel cohort, randomized open label trial to evaluate the efficacy and safety of Avutometinib and in combination with Defactinib; and FRAME, an investigation of Avutometinib and Defactinib in patients with KRAS mutant cancers and subsequent analyses; and RAMP 204 and 205. It has license agreements with Chugai Pharmaceutical Co., Ltd. for the development, commercialization, and manufacture of products containing Avutometinib; and Pfizer Inc. to research, develop, manufacture, and commercialize products containing Pfizer's inhibitors of FAK for therapeutic, diagnostic, and prophylactic uses in humans. In addition, it has a clinical collaboration agreement with Amgen, Inc. to evaluate the combination of Avutometinib with Amgen's KRAS-G12C inhibitor LUMAKRAS which in Phase 1/2 trial entitled RAMP 203; and a discovery and development collaboration with GenFleet Therapeutics to advance new programs targeting RAS pathway-driven cancers. Verastem, Inc. was incorporated in 2010 and is headquartered in Needham, Massachusetts.