Biocodex and THX Pharma (Theranexus) have entered a strategic licensing agreement aimed at advancing treatments for three rare and serious genetic disorders, marking a significant collaboration in the rare-disease drug development landscape.
Under the deal, Biocodex will obtain two key licenses from THX Pharma. The first is an exclusive worldwide license to develop and commercialize Batten-1, a drug candidate targeting juvenile Batten disease (CLN3). The second is an exclusive license covering the United States and Canada for TX01, a reformulated version of an already approved molecule designed to treat Gaucher disease and Niemann-Pick type C disease.
These inherited conditions are typically diagnosed in childhood and are characterized by progressive neurological, visceral, and hematological deterioration. They significantly shorten life expectancy and drastically reduce quality of life. Treatment options remain extremely limited or nonexistent in many cases, making therapeutic development particularly challenging.
The agreement could bring THX Pharma up to €173 million in total payments. This includes an upfront payment of €12 million, as well as potential milestone payments of up to €161 million tied to development and commercialization achievements. THX Pharma is also eligible for double-digit tiered royalties on net sales.
THX Pharma will retain responsibility for clinical development, supported financially and scientifically by Biocodex. Meanwhile, Biocodex will oversee compassionate access programs, market access strategies, and commercialization activities in licensed territories.
Batten-1 is preparing to enter a Phase 3 clinical trial planned for 2026. Juvenile Batten disease is an ultra-rare neurodegenerative disorder marked by progressive vision loss, cognitive decline, motor impairment, and death typically occurring in early adulthood. No approved treatment currently exists, and Batten-1 is being positioned as a potential first-in-class therapy. An international launch is targeted for 2030. The program originated from research by the Beyond Batten Disease Foundation.
TX01, by contrast, builds on an already approved active substance but introduces an adapted oral formulation. The therapy aims to improve administration and patient suitability for those living with Gaucher disease type 1 and Niemann-Pick type C, both lysosomal storage disorders requiring long-term management.
Both companies said the collaboration reflects a shared commitment to addressing high unmet medical needs in rare diseases through scientific partnership and engagement with clinicians and patient communities.