AC Immune
NASDAQ:ACIUAC Immune SA, a clinical stage biopharmaceutical company, discovers, designs, and develops medicines and diagnostic products for the prevention and treatment of neurodegenerative diseases associated with protein misfolding. Its SupraAntigen and Morphomer platforms are designed to generate vaccines, antibodies, and small molecules, which selectively interact with misfolded proteins that are common in a range of neurodegenerative diseases. The company is developing Crenezumab, a humanized, conformation-specific monoclonal antibody, which is in Phase II clinical studies trial for the treatment of Alzheimer's disease (AD). It is also developing ACI-24, an anti-Abeta vaccine candidate that is in Phase II clinical study for AD, as well as completed Phase Ib clinical study for Down syndrome; ACI-35, an anti-Tau vaccine candidate that is in Phase Ib/2a clinical study; and Tau- positron emission tomography (PET) imaging tracer, which is in Phase II clinical study. In addition, the company is researching and developing small molecule Tau aggregation inhibitors for AD and NeuroOrphan indications. Further, it has discovery and preclinical stage molecules targeting range of neurodegenerative diseases, which include diagnostics targeting TDP-43, alpha-synuclein, and NLRP3. AC Immune SA has license agreements and collaborations with Genentech, Inc.; Biogen International GmbH; Janssen Pharmaceuticals, Inc.; Life Molecular Imaging SA; Eli Lilly and Company; and WuXi Biologics. The company was incorporated in 2003 and is headquartered in Lausanne, Switzerland.
Catalyst Pharmaceuticals
NASDAQ:CPRXCatalyst Pharmaceuticals, Inc., a commercial-stage biopharmaceutical company, focuses on developing and commercializing therapies for people with rare debilitating, chronic neuromuscular, and neurological diseases in the United States. It offers Firdapse, an amifampridine phosphate tablets for the treatment of patients with lambert-eaton myasthenic syndrome (LEMS); and Ruzurgi for the treatment of pediatric LEMS patients. The company develops Firdapse for the treatment of MuSK antibody positive myasthenia gravis and spinal muscular atrophy type. It has license agreements with BioMarin Pharmaceutical Inc.; and collaboration and license agreement with Endo Ventures Limited for the development and commercialization of generic Sabril tablets. The company was founded in 2002 and is based in Coral Gables, Florida.
Flexion Therapeutics
NASDAQ:FLXNFlexion Therapeutics, Inc. operates as a biopharmaceutical company, which engages in the development and commercialization of novel and local therapies. It specializes in the treatment of patients with musculoskeletal conditions including osteoarthritis. It offers products under the Zilretta brand, an extended-release corticosteroid approved to manage osteoarthritis knee pain. The company was founded by Michael D. Clayman and Neil Bodick on November 5, 2007 and is headquartered in Burlington, MA.
Fulcrum Therapeutics
NASDAQ:FULCFulcrum Therapeutics, Inc., a clinical-stage biopharmaceutical company, focuses on developing products for improving the lives of patients with genetically defined diseases in the areas of high unmet medical need in the United States. Its product candidates are losmapimod, a small molecule for the treatment of facioscapulohumeral muscular dystrophy is under phase III clinical trial; and FTX-6058, an investigational oral fetal hemoglobin inducer for the treatment of sickle cell disease and other hemoglobinopathies, including beta-thalassemia is under phase I clinical trial. The company is also discovering drug targets for the treatments of rare neuromuscular, muscular, central nervous system, and hematologic disorders, as well as cardiomyopathies and pulmonary diseases. Fulcrum Therapeutics, Inc. has collaboration and license agreement with Acceleron Pharma Inc. to identify biological targets to modulate specific pathways associated with a targeted indication within the pulmonary disease space; and MyoKardia, Inc. to discover, develop, and commercialize novel targeted therapies for the treatment of genetic cardiomyopathies. Fulcrum Therapeutics, Inc. was Incorporated in 2015 and is headquartered in Cambridge, Massachusetts.
Prothena
NASDAQ:PRTAProthena Corporation plc, a late-stage clinical biotechnology company, focuses on discovery and development of novel therapies to treat diseases caused by protein dysregulation in the United States. The company is involved in developing birtamimab, an investigational humanized antibody that is in Phase III clinical trial for the treatment of AL amyloidosis; Prasinezumab, a humanized monoclonal antibody, for the treatment of Parkinson's disease and other related synucleinopathies which is in Phase IIb clinical trial; NNC6019 that is in Phase lI clinical trial for the treatment of ATTR amyloidosis; and BMS-986446 and PRX012, which is in Phase I clinical trial for the treatment of Alzheimer's disease. Its discovery and preclinical programs include PRX123, a dual Aß-Tau vaccine for the treatment and prevention of Alzheimer's disease; and PRX019 for the treatment of neurogenerative diseases, as well as TDP-43 for the treatment of amyotrophic lateral sclerosis. Prothena Corporation plc has a license, development, and commercialization agreement with F. Hoffmann-La Roche Ltd. and Hoffmann-La Roche Inc. to develop and commercialize antibodies that target a-synuclein, including prasinezumab; and a collaboration agreement with Bristol-Myers Squibb to develop and commercialize antibodies targeting tau, TDP-43. The company was incorporated in 2012 and is based in Dublin, Ireland.