Kronos Bio
NASDAQ:KRONKronos Bio, Inc., a clinical-stage biopharmaceutical company, focuses on the discovery and development of therapeutics for various cancer and other serious diseases in the United States. The company's lead product candidates are KB-0742, an oral cyclin dependent kinase 9 inhibitor for the treatment of MYC-amplified solid tumors, which is in phase 2 clinical trial; and KB-9558, a core oncogenic transcription factor that drives multiple myeloma. Kronos Bio, Inc. was incorporated in 2017 and is headquartered in San Mateo, California.
Ocular Therapeutix
NASDAQ:OCULOcular Therapeutix, Inc., a biopharmaceutical company, focuses on the formulation, development, and commercialization of therapies for diseases and conditions of the eye using its bioresorbable hydrogel-based formulation technology in the United States. The company markets DEXTENZA, a dexamethasone ophthalmic insert to treat post-surgical ocular inflammation and pain following ophthalmic surgery, as well as allergic conjunctivitis. It is developing AXPAXLI, an axitinib intravitreal implant that is in phase 3 trials for the treatment of wet age-related macular degeneration and other retinal diseases; PAXTRAVA, a travoprost intracameral implant, which is in phase 2 clinical trials for the treatment of open-angle glaucoma or ocular hypertension; OTX-CSI, a cyclosporine intracanalicular insert that has completed phase 2 clinical trials for the treatment of dry eye disease; and OTX-DED, a dexamethasone intracanalicular insert, which is in phase 2 clinical trials for the short-term treatment of the signs and symptoms of dry eye disease. In addition, the company offers modulator for intermediate and late dry age-related macular degeneration; and gene delivery for inherited retinal degenerations and protein biofactory indications. The company has a strategic collaboration with Regeneron Pharmaceuticals, Inc. (Regeneron) for the development and commercialization of products using the company's sustained-release hydrogel in combination with Regeneron's large molecule VEGF-targeting compounds for the treatment of retinal diseases; and AffaMed Therapeutics Limited for the development and commercialization of DEXTENZA and OTX-TIC. Ocular Therapeutix, Inc. was incorporated in 2006 and is headquartered in Bedford, Massachusetts.
Phathom Pharmaceuticals
NASDAQ:PHATPhathom Pharmaceuticals, Inc., biopharmaceutical company, focuses on developing and commercializing treatments for gastrointestinal diseases. The company has the rights in the United States, Europe, and Canada for an investigational potassium-competitive acid blocker (P-CAB) that blocks acid secretion in the stomach. It is also developing vonoprazan for the treatment of erosive gastroesophageal reflux disease; and in combination with antibiotics for the treatment of Helicobacter pylori infection. The company was incorporated in 2018 and is headquartered in Florham Park, New Jersey.
Repare Therapeutics
NASDAQ:RPTXRepare Therapeutics Inc., a clinical-stage precision oncology company, engages in the discovery and development of therapeutics by using its synthetic lethality approach in Canada and the United States. It uses its SNIPRx, a proprietary, genome-wide, and CRISPR-enabled platform, to discover, validate, and build a pipeline of SL-based therapeutics that focuses on genomic instability, including DNA damage repair. The company's lead product candidate is Camonsertib (RP-3500), an oral small molecule inhibitor under Phase ½ development for the treatment of solid tumors with specific DNA damage repair-related genomic alterations. It is also developing Lunresertib (RP-6306), a PKMYT1 Inhibitor, which is under Phase 1 clinical trial for tumors with genetic alterations characterized by CCNE1 amplification; RP-1664, an oral PLK4 inhibitor, under Phase 1 clinical trial designed to harness the synthetic lethal relationship with TRIM37 amplification or overexpression in solid tumors; and RP-3467, a polymerase theta adenosinetriphosphatase (ATPase) inhibitor, a SL target associated with BRCA mutations and other genomic alterations. The company has license and collaboration agreement with Hoffmann-La Roche Inc. and F. Hoffmann-La Roche Ltd; Bristol-Myers Squibb Company; New York University; and Ono Pharmaceutical Co., as well as a clinical study and collaboration agreement with Debiopharm to explore the synthetic lethal combination of PKMYT1 and WEE1 inhibition in cancer. Repare Therapeutics Inc. was incorporated in 2016 and is headquartered in Montréal, Canada.
Stoke Therapeutics
NASDAQ:STOKStoke Therapeutics, Inc., an early-stage biopharmaceutical company, develops medicines to treat the underlying causes of severe genetic diseases in the United States. The company utilizes its proprietary targeted augmentation of nuclear gene output to develop antisense oligonucleotides to selectively restore protein levels. Its lead clinical candidate is STK-002, which is in preclinical stage for the treatment of autosomal dominant optic atrophy. The company also develops STK-001, which is in phase I/II clinical trial to treat Dravet syndrome; and programs focused on multiple targets, including haploinsufficiency diseases of the central nervous system and eye. The company has a license and collaboration agreement with Acadia Pharmaceuticals Inc. for the discovery, development, and commercialization of novel RNA-based medicines for the treatment of severe and rare genetic neurodevelopmental diseases of the central nervous system. The company was formerly known as ASOthera Pharmaceuticals, Inc. and changed its name to Stoke Therapeutics, Inc. in May 2016. Stoke Therapeutics, Inc. was incorporated in 2014 and is headquartered in Bedford, Massachusetts.