THX Pharma, also known as Theranexus, has entered into a strategic licensing agreement with Biocodex, an independent international pharmaceutical company, to advance two drug candidates targeting three rare genetic disorders: Batten disease, Gaucher disease, and Niemann-Pick type C disease. The collaboration is focused on accelerating development in areas where treatment options remain limited or unavailable and where patients, many of them children, face serious and progressive health challenges.
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Under the terms of the agreement, Biocodex has secured two separate licenses from THX Pharma. The first is an exclusive global license for the development and commercialization of Batten-1, a therapy in development for juvenile Batten disease, also known as CLN3. The second is an exclusive license covering the United States and Canada for TX01, a novel oral formulation of an already approved molecule being developed for Gaucher disease and Niemann-Pick type C disease. These conditions are rare inherited disorders that can cause severe neurological, visceral, and hematological complications, often leading to a significant reduction in quality of life and life expectancy.
The partnership reflects a shared commitment to addressing major unmet medical needs in rare diseases. Both companies aim to combine scientific expertise and close collaboration with healthcare professionals and patient communities to move these programs forward. THX Pharma will remain responsible for leading the clinical development of both candidates, while Biocodex will provide financial backing and scientific support. In addition, Biocodex will oversee compassionate use programs, market access strategies, and commercialization efforts in the regions included under the licensing terms.
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The financial structure of the agreement includes potential total payments of up to 173 million euros to THX Pharma. This amount comprises an upfront payment of 12 million euros and up to 161 million euros tied to development and commercial milestones. THX Pharma is also eligible to receive double digit tiered royalties based on net sales of the products.
Batten-1 is currently preparing to enter Phase 3 clinical development, with the trial expected to begin in 2026. Juvenile Batten disease is an ultra rare neurodegenerative disorder that typically begins in childhood and leads to progressive vision loss, cognitive decline, motor impairment, and premature death in early adulthood. At present, there are no approved treatments for this condition. Batten-1 is being developed with the goal of becoming the first approved therapy for CLN3, with a potential global launch targeted for 2030. The program was originally initiated by the Beyond Batten Disease Foundation, which played a key role in its early development.
TX01 is designed to offer a more patient friendly formulation of an established active substance. By adapting the oral delivery method, the program seeks to improve ease of use and better align treatment with the specific needs of patients living with Gaucher disease type 1 and Niemann-Pick type C disease. Both conditions are lysosomal storage disorders that require long term management and can lead to serious complications if not effectively treated.
Biocodex, headquartered in France and operating in more than 100 countries, has built a longstanding presence in the field of rare diseases. In addition to its focus on orphan conditions, the company is active in microbiota research, women’s health, and a range of established therapeutic areas. Through this agreement with THX Pharma, Biocodex is expanding its rare disease portfolio while reinforcing its commitment to supporting patients who face limited treatment options and complex medical needs.
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