Acadia Pharmaceuticals Inc.
ACADIA Pharmaceuticals Inc., a biopharmaceutical company, focuses on the development and commercialization of medicines for 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 which completed Phase III clinical trial to treat the negative symptoms of schizophrenia; ACP-101, which completed phase III clinical trial for the treatment of hyperphagia in Prader-Willi syndrome; ACP-204 that completed phase II clinical trial for the treatment of Alzheimer's disease psychosis and Lewy Body Dementia with psychosis; ACP-711, which is in phase I clinical trial for the treatment of essential tremor; ACP-211 that is in phase I clinical trial for the treatment of treatment-resistant depression; ACP-2591 that is in Phase I development for Rett syndrome and Fragile X syndrome; and ACP-271, a GPR88 agonist used in evaluation of neurology and is in the IND-enabling stage. The company has a license agreement with Neuren Pharmaceuticals Limited to 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.
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BioMarin Pharmaceutical Inc., a biotechnology company, engages in the development and commercialization of therapies for life-threatening rare diseases and medical conditions in the United States, Europe, Latin America, the Middle East, the Asia Pacific, and internationally. The company's products include VIMIZIM, an enzyme replacement therapy for the treatment of mucopolysaccharidosis (MPS) IV type A, a lysosomal storage disorder; VOXZOGO, a once daily injection analog of c-type natriuretic peptide (CNP) for the treatment of achondroplasia; NAGLAZYME, a recombinant form of N- acetylgalactosamine 4-sulfatase for patients with MPS VI; and PALYNZIQ, a PEGylated recombinant phenylalanine (Phe) ammonia lyase enzyme delivered through subcutaneous injection to reduce blood Phe concentrations. It also develops BRINEURA, a recombinant human tripeptidyl peptidase 1 for the treatment of patients with ceroid lipofuscinosis type 2, a form of Batten disease; ALDURAZYME, a purified protein designed to be identical to a naturally occurring form of the human enzyme alpha-L-iduronidase; KUVAN, a proprietary synthetic oral form of 6R-BH4 that is used to treat patients with phenylketonuria, an inherited metabolic disease; and ROCTAVIAN, an adeno associated virus vector for the treatment of severe hemophilia A. The company's products under development include BMN 333, a longer-acting CNP for the treatment of multiple growth disorders, such as achondroplasia and hypochondroplasia; BMN 349, an oral therapeutic for the treatment of liver disease associated with alpha-1 antitrypsin deficiency; and BMN 351, an oligonucleotide for the treatment of duchenne muscular dystrophy. It serves specialty pharmacies, hospitals, non-U.S. government agencies, distributors, and pharmaceutical wholesalers. The company has license and collaboration agreements with Catalyst Pharmaceutical Partners, Inc., and Ares Trading S.A. The company was incorporated in 1996 and is based in San Rafael, California.
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