Lyon, France – Austin, Texas, USA – Gentilly, France – September 1st, 2026 – [TIME] CET – THX Pharma (Theranexus), an innovative biopharmaceutical company developing treatments for rare neurological diseases, together with its partners, the Beyond Batten Disease Foundation (BBDF) and Biocodex, today announced the submission of regulatory applications to initiate the Phase 3 clinical trial evaluating Batten-1 (study Batten-1-02), a proprietary oral liquid formulation of miglustat developed for the treatment of juvenile Batten disease (CLN3).
The regulatory applications have been submitted to the U.S. Food and Drug Administration (FDA), the UK Medicines and Healthcare products Regulatory Agency (MHRA), and, for the European Union, through the Clinical Trials Information System (CTIS), covering the competent authorities of the participating European countries (France, Germany, the Netherlands, Denmark, Sweden and Italy). Subject to regulatory approvals, patient enrollment is expected to begin by year end.