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.
AcelRx Pharmaceuticals
NASDAQ:ACRXAcelRx Pharmaceuticals, Inc., a specialty pharmaceutical company, focuses on the development and commercialization of therapies for the treatment of acute pain. Its lead product candidate is DSUVIA and DZUVEO, a 30 mcg sufentanil sublingual tablet for the treatment of moderate-to-severe acute pain. The company develops ZALVISO, a patient-controlled analgesia system that allows hospital patients with moderate-to-severe acute pain to self-dose with sufentanil sublingual tablets to manage their pain; ARX-02, which is in Phase 2 clinical trial for the treatment of cancer breakthrough pain in opioid-tolerant patients; and ARX-03, which is in Phase 2 clinical trial for the treatment of procedural anxiety and acute pain. Its product candidates also include Niyad, a regional anticoagulant for the extracorporeal circuit; and LTX-608, a nafamostat formulation for direct IV infusion being explored as an investigational product for antiviral treatment of COVID, acute respiratory distress syndrome, disseminated intravascular coagulation, and acute pancreatitis. The company also develops Fedsyra, an ephedrine pre-filled syringe that contains 10 ml of a solution of 3 mg/ml ephedrine hydrochloride for injection; and phenylephrine, a phenylephrine pre-filled syringe, which contains 10 ml of a solution of 50 mcg/ml phenylephrine for injection. The company was formerly known as SuRx, Inc. and changed its name to AcelRx Pharmaceuticals, Inc. in August 2006. AcelRx Pharmaceuticals, Inc. was incorporated in 2005 and is headquartered in Hayward, California.
Helix BioPharma
OTCMKTS:HBPCFHelix BioPharma Corp. operates as an immune-oncology company in Canada. It is developing L-DOS47 for the treatment of non-small cell lung cancer; and V-DOS47 that targets the vascular epithelial growth factor receptor 2. Helix BioPharma Corp. has collaboration agreements with Moffitt Cancer Center to investigate the pharmacodynamics of L-DOS47 and determine the benefits of combining L-DOS47 with immune checkpoint inhibitors; and with University Hospital Tuebingen to assess the therapeutic response of L-DOS47 in cancer models expressing CEACAM6, with advanced preclinical metabolic imaging. The company is headquartered in Toronto, Canada.
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's MultiTAA-specific T cell therapies include autologous T cells for the treatment of lymphoma and selected solid tumors; allogeneic T cells for the treatment of acute myeloid leukemia (AML) and acute lymphoblastic leukemia; and off-the-shelf products in various indications. It is also developing MT-401, which is in Phase 2 clinical trial for the treatment of post-transplant AML; MT-401-OTS for the treatment of AML; and MT-601 for the treatment of pancreatic cancer and lymphoma. Marker Therapeutics was founded in 1999 and is headquartered in Houston, Texas.
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.