Albireo Pharma
NASDAQ:ALBOAlbireo Pharma, Inc. is a clinical stage biopharmaceutical company, which engages in the development and commercialization of novel bile acid modulators for the treatment of orphan pediatric liver diseases and gastrointestinal disorders. Its pipeline products include Odevixibat, A3384, and Elobixibat. The company was founded by Jan Peter Mattsson and Per-Göran Gillberg on December 8, 2003 and is headquartered in Boston, MA.
Amryt Pharma
NASDAQ:AMYTAmryt Pharma plc, a commercial-stage biopharmaceutical company, focuses on acquiring, developing, and commercializing various treatments to help improve the lives of patients with rare and orphan diseases. The company provides metreleptin, an adjunct to diet as replacement therapy to treat the complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy; oral octreotide, a long-term maintenance therapy in acromegaly patients; and lomitapide, an adjunct to a low-fat diet and other lipid-lowering medicinal products for adults with the Homozygous familial Hypercholesteraemia. Its development candidates include Oleogel-S10 for the cutaneous manifestations of Junctional and Dystrophic Epidermolysis Bullosa (EB), a rare and distressing genetic skin disorder affecting young children and adults; and AP103, a pre-clinical gene therapy for patients with dystrophic EB. The company sells its products in the Americas, Europe, and the Middle East. Amryt Pharma plc was founded in 2015 and is headquartered in Dublin, Ireland.
Kura Oncology
NASDAQ:KURAKura Oncology, Inc., a clinical-stage biopharmaceutical company, develops medicines for the treatment of cancer. The company's pipeline consists of small molecule product candidates that target cancer. Its lead product candidates are ziftomenib, an orally bioavailable small molecule inhibitor of the menin-KMT2A interaction for the treatment of genetically defined subsets of acute leukemias, including acute myeloid leukemia and acute lymphoblastic leukemia; tipifarnib, an orally bioavailable farnesyl transferase inhibitor combination with alpelisib for patients with PIK3CA-dependent HNSCC; and KO-2806, a farnesyl transferase inhibitor for the treatment of solid tumors. It has a clinical collaboration with Novartis Pharma AG to evaluate the combination of tipifarnib and alpelisib in patients with head and neck squamous cell carcinoma whose tumors have HRAS overexpression and/or PIK3CA mutation and/or amplification. The company was founded in 2014 and is headquartered in San Diego, California.
PureTech Health
NASDAQ:PRTCPureTech Health plc, a clinical stage biotherapeutics company, engages in the discovery, development, and commercialization of biotechnology and pharmaceutical solutions in the United States. The company is developing LYT-100, currently under Phase 3 stage, to treat idiopathic pulmonary fibrosis (IPF); and LYT-200, a IgG4 monoclonal antibody, currently under Phase 1/2 stage, to inhibit the activity of galectin-9 for treating solid tumors and hematological malignancies. It is also developing LYT-300, an oral allopregnanolone, currently completed Phase 1 stage, to treat depression, anxiety, and related indications; and LYT-310, an oral cannabidiol, currently under preclinical stage, to treat epilepsies and other neurological indications. In addition, the company is developing LYT-503/IMB-150, a non-opioid inflammation targeted disease immunomodulation for the potential treatment of interstitial cystitis, or bladder pain syndrome; and Glyph technology platform, a lymphatic-targeting chemistry platform, which leverages the body's natural lipid absorption and transports the process to orally administer drugs via the lymphatic system. The company was incorporated in 2015 and is headquartered in Boston, Massachusetts.