THX Pharma Files Phase 3 Trial Applications for Batten-1 in the United States, United Kingdom, and EU
THX Pharma (Theranexus) and its partners Beyond Batten Disease Foundation and Biocodex have achieved a regulatory milestone in their Batten-1 program, a drug candidate targeting the juvenile form of Batten disease (CLN3).
Phase 3 trial authorization requests have been submitted to regulatory authorities in the United States, the United Kingdom, and the European Union.
Regulatory Filing Across Three Geographic Regions
On September 1, 2026, THX Pharma, the Beyond Batten Disease Foundation (BBDF), and Biocodex announced the submission of regulatory authorization requests for the phase 3 clinical trial of Batten-1 (Batten-1-02 study), a proprietary oral liquid formulation of miglustat, in the juvenile form of Batten disease (CLN3).
These applications were submitted to the Food and Drug Administration (FDA) in the United States, to the Medicines and Healthcare products Regulatory Agency (MHRA) in the United Kingdom, and, for the European Union, via the centralized clinical trials information system (CTIS), which covers the competent authorities of participating countries (France, Germany, the Netherlands, Denmark, Sweden, and Italy).
Subject to obtaining these authorizations, patient recruitment is expected to begin by year-end.
An International Pivotal Study Conducted at Ten Reference Centers
Batten-1-02 is a phase 3 clinical trial, open-label, single-arm, and multicenter, designed to demonstrate the efficacy of miglustat in its Batten-1 formulation in pediatric patients with the juvenile form of Batten disease. The primary endpoint is an assessment of visual acuity, a standardized, validated criterion recommended by the FDA.
Approximately 27 patients aged 4 to 17 years, presenting at baseline with evaluable visual acuity in at least one eye, will be treated for 52 weeks. Visual acuity under Batten-1 will be compared to that documented in natural history studies conducted by the expert center of the National Institutes of Health (NIH) in the United States and by the University Hospital of Hamburg (UKE) in Germany.
According to THX Pharma, the expected therapeutic effect is stabilization of visual acuity after one year of treatment in patients receiving miglustat, compared to untreated patients. The study, conducted at ten reference centers in the United States and Europe, is fully funded by Biocodex, in accordance with the global license agreement for Batten-1 announced in February 2026.
