BridgeBio Pharma
NASDAQ:BBIOBridgeBio Pharma, Inc., a commercial-stage biopharmaceutical company, discovers, creates, tests, and delivers transformative medicines to treat patients who suffer from genetic diseases and cancers. Its products in development programs include AG10, a next-generation oral small molecule near-complete TTR stabilizer that is in Phase 3 clinical trial for the treatment of TTR amyloidosis, or transthyretin amyloid cardiomyopathy (ATTR-CM); low-dose infigratinib, an oral FGFR1-3 selective tyrosine kinase inhibitor, which is in Phase 3 double-blinded, placebo-controlled pivotal study for the treatment option for children with achondroplasia; and BBP-631, an AAV5 gene transfer product candidate that is in Phase 1/2 clinical trial for the treatment of congenital adrenal hyperplasia, or CAH, driven by 21-hydroxylase deficiency, or 21OHD. The company also develops Encaleret, a small molecule antagonist of the calcium sensing receptor, or CaSR, which is in phase 3 clinical trial for treating autosomal dominant hypocalcemia type 1, or ADH1; and BBP-418, a glycosylation substrate pro-drug that is in Phase 3 clinical trial for treating limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). In addition, it engages in developing products for mendelian, oncology, and gene therapy diseases. BridgeBio Pharma, Inc. has license and collaboration agreements with the Leland Stanford Junior University; and Leidos Biomedical Research, Inc. The company was founded in 2015 and is headquartered in Palo Alto, California.
MannKind
NASDAQ:MNKDMannKind Corporation, a biopharmaceutical company, focuses on the development and commercialization of inhaled therapeutic products for endocrine and orphan lung diseases in the United States. It offers Afrezza, an inhaled insulin used to improve glycemic control in adults with diabetes, and the V-Go wearable insulin delivery device, which provides continuous subcutaneous infusion of insulin in adults. The company's product pipeline also includes Tyvaso DPI (Treprostinil), an inhalation powder for the treatment of pulmonary arterial hypertension and pulmonary hypertension associated with interstitial lung disease; MNKD-101, a nebulized formulation of clofazimine, for the treatment of severe chronic and recurrent pulmonary infections, including nontuberculous mycobacterial lung disease; MNKD-201, a dry-powder formulation of nintedanib, for the treatment of idiopathic pulmonary fibrosis (IPF). In addition, it has collaboration and license agreement with United Therapeutics Corporation for development, regulatory, and commercial activities of Tyvaso DPI; co-promotion agreement with Vertice Pharma to promote Thyquidity; and collaboration agreement with Thirona to evaluate the therapeutic for the treatment of pulmonary fibrosis. Further, the company has supply and distribution agreement with Biomm S.A. for the commercialization of Afrezza in Brazil; and license and distribution agreement with Cipla Ltd. for the marketing and distribution of Afrezza in India. MannKind Corporation was incorporated in 1991 and is headquartered in Danbury, Connecticut.
Travere Therapeutics
NASDAQ:TVTXTravere Therapeutics, Inc., a biopharmaceutical company, identifies, develops, and delivers therapies to people living with rare kidney and metabolic diseases. Its products include FILSPARI (sparsentan), a once-daily, oral medication designed to target two critical pathways in the disease progression of IgA Nephropathy (endothelin 1 and angiotensin-II); and Thiola and Thiola EC (tiopronin tablets) for the treatment of cystinuria, a rare genetic cystine transport disorder that causes high cystine levels in the urine and the formation of recurring kidney stones. The company's clinical-stage programs consist of Sparsentan, a novel investigational product candidate, which has been granted Orphan Drug Designation for the treatment of focal segmental glomerulosclerosis in the U.S. and Europe; and Pegtibatinase (TVT-058), a novel investigational human enzyme replacement candidate being evaluated for the treatment of classical homocystinuria. It has a cooperative research and development agreement with National Institutes of Health's National Center for Advancing Translational Sciences and Alagille Syndrome Alliance for the identification of potential small molecule therapeutics for Alagille syndrome. The company was formerly known as Retrophin, Inc. and changed its name to Travere Therapeutics, Inc. in November 2020. Travere Therapeutics, Inc. was incorporated in 2008 and is headquartered in San Diego, California.
Verve Therapeutics
NASDAQ:VERVVerve Therapeutics, Inc., a clinical stage genetic medicines company, engages in developing gene editing medicines for patients to treat cardiovascular diseases in the United States. The company's lead product candidate is VERVE-101, a single-course gene editing treatment that permanently turns off the PCSK9 gene in the liver; and VERVE-102, a product candidate that targets the PCSK9 gene for the treatment of HeFH. It engages in the development of ANGPTL3 program to permanently turn off the ANGPTL3 gene in the liver. It has a collaboration and license agreement with Beam Therapeutics Inc.; a development and option agreement with Acuitas Therapeutics, Inc.; and a Cas9 license agreement with The Broad Institute and the President and Fellows of Harvard College. The company was formerly known as Endcadia, Inc. and changed its name to Verve Therapeutics, Inc. in January 2019. Verve Therapeutics, Inc. was incorporated in 2018 and is headquartered in Cambridge, Massachusetts.