Anavex Life Sciences
Anavex Life Sciences Corp. operates as a biopharmaceutical company. It engages in the development of novel therapeutics for the treatment of neurodegenerative, neurodevelopmental, and neuropsychiatric disorders, including Alzheimer's disease, Parkinson's disease, schizophrenia, Rett syndrome, and other central nervous system (CNS) diseases, pain, and various types of cancer. Its lead drug candidate, ANAVEX 2-73 (blarcamesine), which has completed a Phase 2a and a Phase 2b/3 clinical trial for Alzheimer's disease; a Phase 2 proof-of-concept study in Parkinson's disease dementia; and a Phase 2 and a Phase 3 study in adult patients and one Phase 2/3 study in pediatric patients with Rett syndrome. Its ANAVEX 2-73 is an orally available drug candidate designed to restore cellular homeostasis by targeting SIGMAR1 and muscarinic receptors. The ANAVEX 2-73 also exhibited anticonvulsant, anti-amnesic, neuroprotective, and anti-depressant properties in animal models, indicating its potential to treat additional CNS disorders, including epilepsy. The company's ANAVEX 3-71, which targets SIGMAR1 and M1 muscarinic receptors, is a clinical stage drug candidate demonstrating disease-modifying activity against the Alzheimer's disease in transgenic (3xTg-AD) mice, including cognitive deficits, amyloid, and tau pathologies. Anavex Life Sciences Corp. was incorporated in 2004 and is headquartered in New York, New York.
Open AVXL dashboard ↗BioMarin Pharmaceuticals Inc
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.
Open BMRN dashboard ↗