ACADIA Pharmaceuticals
NASDAQ:ACADACADIA Pharmaceuticals Inc., a biopharmaceutical company, focuses on the development and commercialization innovative medicines that address unmet medical needs in central nervous system (CNS) disorders and rare diseases in the United States. The company offers NUPLAZID (pimavanserin) for the treatment of hallucinations and delusions associated with Parkinson's disease psychosis; and DAYBUE, a novel synthetic analog of the amino-terminal tripeptide of insulin-like growth factor 1 for treatment of Rett Syndrome. It also develops Pimavanserin that is in Phase III ADVANCE-2 study to treat the negative symptoms of schizophrenia; ACP-101 whixh is in Phase III for the treatment of hyperphagia in Prader-Willi syndrome; ACP-204 which is in Phase II for the treatment of Alzheimer's disease psychosis; ACP-2591 that is in Phase I for Rett syndrome and Fragile X syndrome; preclinical antisense oligonucleotide programs; and other programs for neuropsychiatric symptoms. It has a license agreement with Neuren Pharmaceuticals Limited to develop and commercialize trofinetide for Rett syndrome and other indications; and a license and collaboration agreement with Stoke Therapeutics, Inc. to discover, develop and commercialize novel RNA-based medicines for the potential treatment of severe and rare genetic neurodevelopmental diseases of the CNS. The company was formerly known as Receptor Technologies, Inc. and changed its name ACADIA Pharmaceuticals Inc. in 1997. ACADIA Pharmaceuticals Inc. was incorporated in 1993 and is headquartered in San Diego, California.
Blueprint Medicines
NASDAQ:BPMCBlueprint Medicines Corporation, a precision therapy company, develops medicines for genomically defined cancers and blood disorders in the United States and internationally. The company is developing AYVAKIT for the treatment of systemic mastocytosis (SM) and gastrointestinal stromal tumors; BLU-263, an orally available, potent, and KIT inhibitor for the treatment of indolent SM, and other mast cell disorders. It is also developing GAVRETO for the treatment of RET fusion-positive non-small cell lung cancer, altered thyroid carcinoma, and medullary thyroid carcinoma; BLU-945 for the treatment of epidermal growth factor receptor driven non-small-cell lung carcinoma (NSCLC); and BLU-451 to treat NSCLC in patients with epidermal growth factor receptor gene (EGFR) exon 20 insertion mutations. In addition, the company is developing BLU-782, for the treatment of fibrodysplasia ossificans progressive; BLU- 222 to treat patients with cyclin E aberrant cancers; and BLU-852 for the treatment of advanced cancers. It has collaboration and license agreements with Clementia Pharmaceuticals, Inc.; Proteovant Therapeutics; CStone Pharmaceuticals; Genentech, Inc.; Hoffmann-La Roche Inc.; and Zai Lab (Shanghai) Co., Ltd. The company was formerly known as Hoyle Pharmaceuticals, Inc. and changed its name to Blueprint Medicines Corporation in June 2011. Blueprint Medicines Corporation was incorporated in 2008 and is headquartered in Cambridge, Massachusetts.
Madrigal Pharmaceuticals
NASDAQ:MDGLMadrigal Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company, focuses on the development of therapeutics for the treatment of non-alcoholic steatohepatitis (NASH) in the United States. Its lead product candidate is resmetirom, a liver-directed thyroid hormone receptor beta agonist, which is in Phase 3 clinical trials for treating NASH. The company is headquartered in West Conshohocken, Pennsylvania.
Nuvalent
NASDAQ:NUVLNuvalent, Inc., a clinical stage biopharmaceutical company, engages in the development of therapies for patients with cancer. Its lead product candidates are NVL-520, a novel ROS1-selective inhibitor to address the clinical challenges of emergent treatment resistance, central nervous system (CNS)-related adverse events, and brain metastases that may limit the use of ROS1 tyrosine kinase inhibitors (TKIs) for patients with ROS proto-oncogene 1 (ROS1)-positive non-small cell lung cancer (NSCLC) which is under the phase 2 portion of the ARROS-1 Phase 1/2 clinical trial; NVL-655, a brain-penetrant ALK-selective inhibitor, to address the clinical challenges of emergent treatment resistance, CNS-related adverse events, and brain metastases that might limit the use of first-, second-, and third-generation ALK inhibitors that is under the phase 2 portion of the ALKOVE-1 Phase 1/2 clinical trial; and NVL-330, a brain-penetrant human epidermal growth factor receptor 2 (HER2)-selective inhibitor designed to treat tumors driven by HER2ex20, brain metastases, and avoiding treatment-limiting adverse events including due to off-target inhibition of wild-type EGFR, which is expected to initiate phase 1 trial. The company was incorporated in 2017 and is headquartered in Cambridge, Massachusetts.