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- THX Pharma, Biocodex, and the Beyond Batten Disease Foundation jointly serve as Platinum Sponsors of the 2026 BDSRA Annual Family Conference, marking International Batten Disease Awareness Day on June 9. - The partners will deliver an update on Batten-1, an oral miglustat-based investigational therapy, and the planned initiation of an international pivotal Phase 3 study later in 2026. - Phase 1/2 results demonstrated a favorable safety profile, slowed motor symptom progression, reduced neurofilament light chain levels, and real-world data suggesting clinical benefit on visual acuity. - Biocodex is funding the Phase 3 study under a global licensing agreement, with the ambition of making Batten-1 available to patients by 2029 pending regulatory submissions.
- 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.
