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- THX Pharma and Biocodex have signed a strategic licensing agreement worth up to €173 million to develop treatments for three rare neurological diseases: Batten disease, Gaucher disease, and Niemann-Pick disease type C. - Biocodex acquired exclusive global rights to Batten-1, a drug candidate preparing for Phase 3 trials in 2026 for juvenile Batten disease, which currently has no approved treatments. - The deal includes a €12 million upfront payment, up to €161 million in milestone payments, and tiered double-digit royalties, with THX Pharma leading clinical development while Biocodex handles commercialization. - TX01, based on an adapted oral formulation of an already approved compound, received regional licensing for the US and Canada to treat Gaucher disease and Niemann-Pick disease type C.
- THX Pharma announced a capital increase of approximately €8 million with preferential subscription rights to finance development and commercialization of rare neurological disease treatments. - The company secured €5.92 million in subscription and guarantee commitments representing 75.5% of the offering, with shares priced at €1.80 each. - Proceeds will support TX01 for Gaucher and Niemann-Pick diseases targeting 2027 commercialization, and Batten-1 for juvenile Batten disease with expected 2028 launch. - THX Pharma projects first commercial sales in 2027, financial profitability by 2028, and revenues of €250 million by 2032.
- Treatment with Batten-1 (miglustat) for 18 months showed no signs of deteriorating vision in patients with juvenile Batten disease. - The Phase 1/2 study demonstrated that Batten-1 was safe and well-tolerated over two years, with potential stabilization of visual acuity. - A Phase 3 trial, with visual acuity as the primary endpoint, is planned to support drug registration in the U.S. and Europe. - Batten-1 acts by blocking glucosylceramide synthase, aiming to reduce fatty aggregate buildup in cells.
