THX Pharma and Biocodex have entered into a strategic licensing agreement covering two investigational drug candidates targeting three rare genetic disorders: juvenile Batten disease (CLN3), Gaucher disease type 1, and Niemann-Pick type C disease.

Under the agreement, Biocodex has obtained exclusive worldwide rights to develop and commercialise Batten-1 for juvenile Batten disease, as well as exclusive rights in the United States and Canada for TX01, a reformulated version of an approved molecule intended to treat Gaucher disease and Niemann-Pick type C disease.

THX Pharma will continue to lead the clinical development of both programmes, supported financially and scientifically by Biocodex. Biocodex will oversee compassionate use, market access and commercialisation in the licensed territories.

The total deal value may reach Euro 173 million, including  Euro 12 million upfront and up to  Euro 161 million in development and commercial milestone payments. THX Pharma is also eligible to receive double-digit tiered royalties on net sales.

Batten-1 is preparing to enter Phase III clinical trials in 2026. Juvenile Batten disease is an ultra-rare, progressive neurodegenerative disorder that leads to vision loss, cognitive decline, motor impairment and premature death. Currently, no approved treatment exists for the condition. An international launch of Batten-1 is targeted for 2030.

TX01 is an adapted oral formulation of an already approved active substance designed to improve treatment administration for patients with Gaucher disease type 1 and Niemann-Pick type C disease, both rare lysosomal storage disorders with significant clinical burden.

The agreement aims to advance therapeutic options in areas where treatment choices remain limited or unavailable.