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.
Mirum Pharmaceuticals
NASDAQ:MIRMMirum Pharmaceuticals, Inc., a biopharmaceutical company, focuses on the development and commercialization of novel therapies for debilitating rare and orphan diseases. Its lead product candidate is LIVMARLI (maralixibat), an orally administered and minimally absorbed ileal bile acid transporter (IBAT) inhibitor that is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome in the United States and internationally. The company is also involved in the commercialization of Cholbam, a cholic acid capsule, which is approved as treatment for pediatric and adult patients with bile acid synthesis disorders due to single enzyme defects and for adjunctive treatment of patients with peroxisomal disorders, including peroxisome biogenesis disorder-Zellweger spectrum disorder and Smith-Lemli-Opitz syndrome; and Chenodal, a tablet, which is approved for the treatment of radiolucent stones in the gallbladder, and under Phase 3 development for the treatment cerebrotendinous xanthomatosis. In addition, it develops Volixibat, an oral and minimally absorbed agent designed to inhibit IBAT, currently under Phase 2b clinical trial for the treatment of adult patients with cholestatic liver diseases. The company was incorporated in 2018 and is headquartered in Foster City, California.
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. The company was founded in 2010 and is headquartered in Los Angeles, California.
Summit Therapeutics
NASDAQ:SMMTSummit Therapeutics Inc., a biopharmaceutical company, focuses on discovery, development, and commercialization of patient, physician, caregiver, and societal friendly medicinal therapies in the United States, and the United Kingdom. The company's lead development candidate is Ivonescimab, a bispecific antibody for immunotherapy through blockade of PD-1 with the anti-angiogenesis; and anti-infectives portfolio includes SMT-738, a novel class of precision antibiotics for the treatment of multidrug resistant infections, which primarily includes carbapenem-resistant Enterobacteriaceae infections. It has a collaboration and license agreement with Akeso, Inc. and its affiliates to develop and commercialize ivonescimab. The company was founded in 2003 and is headquartered in Miami, Florida.
Wave Life Sciences
NASDAQ:WVEWave Life Sciences Ltd., a clinical-stage biotechnology company, designs, develops, and commercializes ribonucleic acid (RNA) medicines through PRISM, a discovery and drug development platform. The company's RNA medicines platform, PRISM, combines multiple modalities, chemistry innovation, and deep insights into human genetics to deliver scientific breakthroughs that treat both rare and prevalent disorders. It is developing WVE-006, a RNA editing oligonucleotide for the treatment of alpha-1 antitrypsin deficiency; siRNA clinical candidate for the treatment of obesity and other metabolic disorders; WVE-N531, a exon skipping oligonucleotide for the treatment of duchenne muscular dystrophy; and WVE-003, an antisense silencing oligonucleotide for the treatment of Huntington's disease (HD). The company has collaboration agreements with GlaxoSmithKline for the research, development, and commercialization of oligonucleotide therapeutics; Takeda Pharmaceutical Company Limited for the research, development, and commercialization of oligonucleotide therapeutics for disorders of the Central Nervous System; and Asuragen, Inc. for the development and potential commercialization of companion diagnostics for investigational allele-selective therapeutic programs targeting HD. Wave Life Sciences Ltd. was incorporated in 2012 and is based in Singapore.