Crinetics Pharmaceuticals
NASDAQ:CRNXCrinetics Pharmaceuticals, Inc., a clinical-stage pharmaceutical company, focuses on the discovery, development, and commercialization of novel therapeutics for rare endocrine diseases and endocrine-related tumors. The company's lead product candidate is paltusotine, an oral selective nonpeptide somatostatin receptor type 2 agonist, which is in Phase 3 trial for the treatment of acromegaly; and Phase 2 trial for treating carcinoid syndrome associated with neuroendocrine tumors. It is also developing CRN04894, an investigational oral nonpeptide product candidate to antagonize the adrenocorticotrophic hormone (ACTH) receptor that has completed a Phase 1 study for the treatment of diseases caused by excess ACTH, including congenital adrenal hyperplasia and Cushing's disease. In addition, the company is developing antagonists of the parathyroid hormone (PTH) receptor for the treatment of primary hyperparathyroidism and humoral hypercalcemia of malignancy, and other diseases of excess PTH; identified investigational orally available somatostatin receptor type 3 targeted nonpeptide agonists for the treatment of autosomal dominant polycystic kidney disease; and developing thyroid-stimulating hormone receptor antagonists for the treatment of graves' disease and thyroid eye disease, as well as Oral GLP-1 and GIP nonpeptides for the treatment of diabetes and obesity. Crinetics Pharmaceuticals, Inc. was incorporated in 2008 and is headquartered in San Diego, California.
Homology Medicines
NASDAQ:FIXXHomology Medicines, Inc., a genetic medicines company, focuses on transforming the lives of patients suffering from rare genetic diseases in the United States. The company offers proprietary platform is designed to utilize its human hematopoietic stem cell derived adeno-associated virus vectors (AAVHSCs) to deliver genetic medicines in vivo either through a nuclease-free gene editing mobility, gene therapy or for various genetic disorders. Its earlier stage product candidates comprising HMI-202, an investigational gene therapy which completed IND-enabling studies for the treatment of metachromatic leukodystrophy; and HMI-104 which completed IND-enabling studies. Homology Medicines, Inc. was incorporated in 2015 and is headquartered in Bedford, Massachusetts.
IMARA
NASDAQ:IMRAIMARA Inc., a clinical-stage biopharmaceutical company, develops and commercializes therapeutics for patients with rare genetic disorders of hemoglobin. Its lead product candidate is IMR-687, an oral and once-a-day therapeutic that is in Phase 2b clinical trials for the treatment of sickle cell disease and ß-thalassemia. The company also develops IMR-261, an oral and clinical-ready activator of nuclear factor erythroid 2-related factor 2 for the treatment of hemoglobinopathies, iron disorders, and potentially other areas. IMARA Inc. was incorporated in 2016 and is headquartered in Boston, Massachusetts.
Molecular Templates
NASDAQ:MTEMMolecular Templates, Inc., a clinical stage biopharmaceutical company, focuses on the discovery and development of biologic therapeutics for the treatment of cancer and other serious diseases in the United States. The company primarily develops therapies through its proprietary biologic engineered toxin body (ETB) drug platform. The company is developing MT- 8421, an ETB candidate that is in Phase I clinical trial for the treatment of dismantling TME through direct cell-kill of tumor and immune cell; MT-0169, which is in Phase I clinical trial to treat relapsed/refractory multiple myeloma; and MT-6402 in that is in Phase I clinical trial for relapsed/refractory patients with PD-L1 expressing tumors. The company has a collaboration agreement with Bristol Myers Squibb to discover and develop novel products containing ETBs directed to multiple targets. Molecular Templates, Inc. was founded in 2001 and is headquartered in Austin, Texas.
Puma Biotechnology
NASDAQ:PBYIPuma Biotechnology, Inc., a biopharmaceutical company, focuses on the development and commercialization of products to enhance cancer care in the United States and internationally. The company offers NERLYNX, an oral version of neratinib that is used to treat adult patients with early stage HER2-overexpressed/amplified breast cancer; and advanced or metastatic HER2-positive breast cancer when combined with capecitabine. It also develops alisertib, a small molecule inhibitor of aurora kinase A for the treatment of hormone receptor positive breast cancer, triple negative breast cancer, small cell lung cancer, and head and neck cancer. The company sells its products through specialty pharmacy and distributor networks. It has license agreements with Pfizer Inc. for the development, manufacture, and commercialization of neratinib (oral), neratinib (intravenous), PB357, and related compounds; and Takeda Pharmaceutical Company Limited for the research, development, and commercialization of alisertib, as well as sub-license agreements with Specialised Therapeutics Asia Pte Ltd., Medison Pharma Ltd., Pint Pharma International SA, Knight Therapeutics, Inc., Pierre Fabre Medicament SAS, and Bixink Therapeutics Co., Ltd. Puma Biotechnology, Inc. was founded in 2010 and is headquartered in Los Angeles, California.