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.
Marker Therapeutics
NASDAQ:MRKRMarker Therapeutics, Inc., a clinical-stage immuno-oncology company, engages in the development and commercialization of various T cell-based immunotherapies for the treatment of hematological malignancies and solid tumor indications in the United States. Its MultiTAA-specific T cell technology is based on the manufacture of non-engineered tumor-specific T cells that recognize multiple tumor-associated antigens and kill tumor cells. The company also develops MT-401-OTS, which is in phase 1 clinical trial for the treatment of acute myeloid leukemia; and MT-601 which is in phase 1 clinical trial for the treatment of lymphoma, as well as in phase 1/2 clinical trial for the treatment of pancreatic cancer. Marker Therapeutics is headquartered in Houston, Texas.
Savara
NASDAQ:SVRASavara Inc., formerly Mast Therapeutics, Inc., is a clinical-stage pharmaceutical company. The Company is focused on the development and commercialization of novel therapies for the treatment of patients with rare respiratory diseases. Its pipeline includes AeroVanc, Molgradex and AIR001. AeroVanc is an inhaled formulation of vancomycin, which the Company is developing for the treatment of persistent methicillin-resistant Staphylococcus aureus, lung infection in cystic fibrosis patients. Molgradex is an inhaled formulation of recombinant human granulocyte-macrophage colony-stimulating factor. It is developing Molgradex for the treatment of autoimmune pulmonary alveolar proteinosis, a rare lung disease. AIR001 is a sodium nitrite solution for inhalation via nebulization. AIR001 is in Phase II clinical development for the treatment of heart failure with preserved ejection fraction, also known as diastolic heart failure or heart failure with preserved systolic function.
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.
vTv Therapeutics
NASDAQ:VTVTvTv Therapeutics Inc., a clinical stage biopharmaceutical company, focuses on the development of orally administered treatments for metabolic and inflammatory diseases. The company's lead drug candidate is TTP399, an orally administered, small molecule, and liver-selective glucokinase activator for the treatment of type 1 diabetes that is in Phase III clinical trial; and HPP737, an orally administered non-CNS penetrant phosphodiesterase type 4 (PDE4) inhibitor that addresses inflammatory diseases, psoriasis, COPD, and Atopic Dermatitis that is in Phase III clinical trial. It also develops TTP273, an orally available, small molecule glucagon-like peptide 1 receptor agonists for the treatment of cystic fibrosis related diabetes that is in Phase I clinical trial. In addition, the company is involved in the clinical development of other programs, including HPP3033, a non-electrophilic therapeutic approach to activating the nuclear factor erythroid 2related factor 2 (Nrf2) pathway for the treatment of chronic diseases associated with oxidative stress; and Azeliragon, a RAGE antagonist for inflammatory lung diseases, including severe COVID-19, as well as for glioblastoma, other cancers, and cancer treatment-related conditions. vTv Therapeutics Inc. has a license agreement with Reneo Pharmaceuticals, Inc. to develop and commercialize peroxisome proliferation activated receptor delta agonist program, including the compound HPP593 for therapeutic, prophylactic, and diagnostic application in humans; and with Anteris Bio, Inc. to develop and commercialize HPP971, a Nrf2 activator; with Cantex Pharmaceuticals, Inc.; Hangzhou Zhongmei Huadong Pharmaceutical Co., Ltd.; Newsoara Biopharma Co., Ltd.; JDRF International; and Novo Nordisk A/S. The company was incorporated in 2015 and is headquartered in High Point, North Carolina. vTv Therapeutics Inc. is a subsidiary of MacAndrews & Forbes Incorporated.