French biopharma duo THX Pharma and Biocodex strike rare diseases licensing deal worth up to €173M
French biopharma THX Pharma has signed a strategic licensing deal with Biocodex, an independent French pharmaceutical group, to advance two drug candidates in three rare genetic disorders with high unmet need.
THX Pharma (formerly known as Theranexus) may receive up to €173 million under the agreement. The package includes €12 million upfront and up to €161 million in development and commercial milestones. In addition, Biocodex will pay double-digit tiered royalties on net sales.
For THX Pharma, the deal strengthens its rare disease portfolio and secures a partner with international reach. Biocodex is active in more than 100 countries and has built a long-standing presence in rare neurological disorders.
Biocodex secures global rights to Batten-1
Under the agreement, Biocodex gains an exclusive worldwide license to develop and commercialize Batten-1, a candidate targeting juvenile Batten disease (CLN3). The ultra-rare pediatric disorder leads to progressive vision loss, cognitive and motor decline, and premature death. No treatment is currently approved. Batten-1 is a proprietary formulation based on miglustat. This active substance is designed to block the accumulation of glycosphingolipids and reduce neuroinflammation, with the aim of preventing brain cell death. Other experimental approaches, including gene therapy, RNA-based strategies and small-molecule programs, are under investigation, but Batten-1 is currently among the most advanced clinical candidates.
Batten-1 is preparing to enter Phase 3 testing in 2026. An international launch is targeted for 2030. The program was originally initiated by the Beyond Batten Disease Foundation. “Batten-1 aims to become the first available treatment for this condition,” the companies stated in the press release.
North American focus for TX01
The agreement also grants Biocodex exclusive rights in the United States and Canada for TX01. It targets Gaucher disease type 1 and Niemann-Pick type C disease, two rare lysosomal storage disorders. Both conditions cause progressive and severe symptoms that can affect the liver, spleen, blood or nervous system. Although treatments are available for some patients (for instance, enzyme replacement or substrate reduction therapies for Gaucher disease type 1, Miplyffa for Niemann-Pick type C disease), no cure exists. TX01 is a novel oral formulation of an approved active ingredient, the exact compound of which THX Pharma has chosen not to disclose when we contacted them. According to the press release, the redesigned therapy is intended “to improve treatment administration and better address the specific needs of these patients”.
THX Pharma will lead clinical development for both programs. Biocodex will provide financial and scientific support. The company will also handle compassionate use, market access and commercialization in the licensed territories.
Addressing high unmet medical need
Juvenile Batten disease, Gaucher disease type 1 and Niemann-Pick type C disease are all genetic disorders that often affect children. They can lead to severe and progressive visceral, hematological or neurological impairment. Therapeutic options remain limited or absent, and the collaboration between THX Pharma and Biocodex may help advance two promising drug candidates.
The partners said they share the ambition to advance new solutions where needs remain significant, relying on science, partnership and dialogue with healthcare professionals and patient organizations. By combining THX Pharma’s development expertise with Biocodex’s regulatory and commercial capabilities, the companies aim to accelerate access to new therapies for patients who currently have few or no options. “Together, we will bring forward innovative therapeutic solutions in three rare diseases affecting thousands of people worldwide”, says Mathieu Charvériat, President and Chief Executive Officer of THX Pharma.
