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.
BeyondSpring
NASDAQ:BYSIBeyondSpring Inc., a clinical stage biopharmaceutical company, together with its subsidiaries, focuses on the development of cancer therapies. The company's lead asset is the Plinabulin, a selective immune-modulating microtubule-binding agent that has completed Phase III clinical trials for the prevention of chemotherapy-induced neutropenia; and for treatment of later-stage non-small cell lung cancer. It is also developing Plinabulin in combination with various immuno-oncology agents, including nivolumab, a PD-1 antibody for the treatment of non-small cell lung cancer; ipilimumab, a CTLA-4 antibody for the treatment of extensive-stage small cell lung cancer; and in combination with PD-1 or PD-L1 antibodies and radiation for the treatment of various cancers. In addition, the company engages in the development of three small molecule immune agents in preclinical stages; and a drug development platform. The company was founded in 2010 and is headquartered in New York, New York.
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.
Foghorn Therapeutics
NASDAQ:FHTXFoghorn Therapeutics Inc., a clinical-stage biopharmaceutical company, engages in the discovery and development of medicines targeting genetically determined dependencies within the chromatin regulatory system in the United States. The company uses its proprietary Gene Traffic Control platform to identify, validate, and potentially drug targets within the system. It develops FHD-286, a small molecule inhibitor of the enzymatic activity of BRG1 and BRM that is in phase I for the treatment of relapsed and/or refractory acute myeloid leukemia/myelodysplastic syndrome. The company is also developing therapies for mutant cancers, such as Non-Small Cell Lung, bladder, endometrial, colorectal, and melanoma cancers; and dependent cancers, including prostate cancer and diffuse large b-cell lymphoma. It has a research collaboration and license agreement with Merck Sharp & Dohme Corp. to discover and develop novel oncology therapeutics against a transcription factor target; and Eli Lilly and Company for developing FHD-909, a selective ATPase inhibitor of BRM. Foghorn Therapeutics Inc. was incorporated in 2015 and is headquartered in Cambridge, Massachusetts.
Viking Therapeutics
NASDAQ:VKTXViking Therapeutics, Inc., a clinical-stage biopharmaceutical company, focuses on the development of novel therapies for metabolic and endocrine disorders. The company's lead drug candidate is VK2809, an orally available tissue and receptor-subtype selective agonist of the thyroid hormone receptor beta (TRß), which is in Phase IIb clinical trials to treat patients with biopsy-confirmed non-alcoholic steatohepatitis, as well as NAFLD. It also develops VK5211, an orally available non-steroidal selective androgen receptor modulator that is in Phase II clinical trials for the treatment of patients recovering from non-elective hip fracture surgery; VK0612, an orally available Phase IIb-ready drug candidate for type 2 diabetes; VK2735, a novel dual agonist of the glucagon-like peptide, which is in Phase 1 SAD/MAD clinical trial, and VK0214, an orally available tissue and receptor-subtype selective agonist of the TRß for X-linked adrenoleukodystrophy. The company was incorporated in 2012 and is headquartered in San Diego, California.