Companies in #Tuberous Sclerosis Complex
Found 2 companies operating in this sector.
Filana Therapeutics is a clinical-stage biopharmaceutical company developing simufilam, a small-molecule modulator of filamin A, for tuberous sclerosis complex (TSC)-related epilepsy. Formerly known as Cassava Sciences and, before that, Pain Therapeutics, the company rebranded and refocused on refractory TSC-associated seizures in March 2026 after an earlier Alzheimer's disease development programme. In September 2026 the FDA lifted a full clinical hold on the company's investigational new drug application for simufilam, clearing the way for a planned 16-week, multicenter, randomized, double-blind Phase 2a proof-of-concept study evaluating two doses in 40 patients aged 12 to 55 at 13 U.S. sites, with patient screening expected to begin in the first quarter of 2027.
Quoin Pharmaceuticals, Ltd. (NASDAQ: QNRX) is a late-stage specialty pharmaceutical company founded in 2018 and based in Ashburn, Virginia, focused exclusively on rare and orphan dermatologic diseases. Its lead program, QRX003 — a topical serine protease inhibitor lotion for Netherton syndrome — holds Orphan Drug Designation in the US, EU, and Japan plus FDA Fast Track and Rare Pediatric Disease designations, and received FDA conditional approval of QYLEKI as its proposed brand name in June 2026. The company submitted the first-ever IND for Peeling Skin Syndrome (FDA-cleared July 2026) and is advancing a topical rapamycin platform (QRX009) toward clinical testing across Pachyonychia Congenita, Gorlin Syndrome, and Tuberous Sclerosis Complex. As of June 30, 2026 the company held approximately $10.8 million in cash and marketable securities, which management stated would fund operations into 2027.