THX Pharma, Biocodex, and the Beyond Batten Disease Foundation Prepare for Phase 3 of Batten-1 in 2026
On the occasion of International Batten Disease Awareness Day, THX Pharma (Theranexus), Biocodex, and the Beyond Batten Disease Foundation announce a joint partnership as platinum sponsors of the BDSRA Annual Family Conference 2026. The three partners will present the progress of the Batten-1 program and the imminent start of its pivotal Phase 3 study.
Collective Commitment to the CLN3 Community
The three partners reaffirmed on Tuesday their joint commitment to patients, families, caregivers, and clinicians dealing with Batten disease CLN3, a rare hereditary neurodegenerative disease characterized by progressive vision loss during the first decade of life, followed by the onset of epileptic seizures, motor decline, and cognitive deterioration. To date, no disease-modifying treatment has been approved for this juvenile form. THX Pharma, Biocodex, and the Beyond Batten Disease Foundation marked this day by jointly becoming platinum sponsors of the BDSRA Annual Family Conference 2026, scheduled from July 10 to 12, 2026, at the Westin Chicago Lombard in Illinois. This event is described as the largest annual gathering of families, clinicians, researchers, and industrial partners committed to Batten disease in North America.
Batten-1 Prepares for Its Pivotal Phase 3 Study
Representatives from the three organizations will jointly present an update on Batten-1, a drug candidate based on miglustat developed to slow the progression of Batten disease CLN3. Phase 1/2 data (study Batten-1-01, conducted in six young adults) demonstrated a favorable tolerance and pharmacokinetic profile, accompanied by encouraging biomarker and clinical signals, including a measured slowdown in the progression of motor symptoms and a decrease in neurofilament light chain (NfL) levels. Real-world data generated in 2025 in collaboration with the Beyond Batten Disease Foundation suggested a clinical benefit of miglustat on the visual acuity of CLN3 patients. Based on this set of data, the partners are preparing to start the international pivotal Phase 3 clinical study of Batten-1 in 2026, fully funded by Biocodex under a global licensing agreement announced in February 2026.
Statements from the Partners
Mathieu Charvériat, CEO of THX Pharma, stated: 'The progress made on Batten-1—from positive results in Phase 1/2 to recent real-world data confirming a clinical benefit on visual function, to the imminent launch of our pivotal Phase 3 study—has only been possible thanks to the trust and mobilization of the CLN3 community and our partners BBDF and Biocodex.' Nicolas Coudurier, CEO of Biocodex, emphasized that 'meaningful progress in rare pediatric neurology is built with patient communities, not for them.' Craig Benson, Founder and Chairman of the Board of the Beyond Batten Disease Foundation, described the partnership as 'a strong signal for our community,' marking a milestone eighteen years after the foundation's creation.
