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.
DURECT
NASDAQ:DRRXDURECT Corporation, a biopharmaceutical company, develops medicines based on its epigenetic regulator program. The company's lead product larsucosterol (DUR-928), an endogenous, orally bioavailable small molecule that is in Phase IIb clinical trial to play a regulatory role in lipid metabolism, stress and inflammatory responses, and cell death and survival to treat alcohol-associated hepatitis, as well as completed Phase Ib clinical trial to treat patients with nonalcoholic steatohepatitis. It also offers ALZET product line that consists of osmotic pumps and accessories used for research in mice, rats, and other laboratory animals. In addition, the company offers POSIMIR, a post-surgical pain product to deliver bupivacaine over three days in adults; and Methydur to treat attention deficit hyperactivity disorder. It markets and sells its ALZET lines through direct sales force in the United States, as well as through a network of distributors in other countries. The company has strategic collaboration and other agreements with Virginia Commonwealth University Intellectual Property Foundation; Indivior UK Ltd.; and Innocoll Pharmaceuticals Limited. DURECT Corporation was incorporated in 1998 and is headquartered in Cupertino, 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.
Protagonist Therapeutics
NASDAQ:PTGXProtagonist Therapeutics, Inc., a biopharmaceutical company, develops peptide-based drugs for hematology and blood disorders, and inflammatory and immunomodulatory diseases. It is developing Rusfertide (PTG-300), an injectable hepcidin mimetic that completed phase 2 clinical trials for the treatment of polycythemia vera and other blood disorders; and JNJ-2113, an orally delivered investigational drug to block biological pathways that completed phase 2b clinical trials for the treatment of moderate-to-severe plaque psoriasis; and PN-943, an orally delivered, gut-restricted alpha 4 beta 7 specific integrin antagonist completed a phase 2 clinical trials in patients with moderate to severe ulcerative colitis. The company has a license and collaboration agreement with Takeda to commercialize rusfertide; and JNJ Innovative Medicines to co-develop Interleukin-23 receptor antagonist compound JNJ-2113. Protagonist Therapeutics, Inc. was incorporated in 2006 and is headquartered in Newark, California.