Submission of applications for authorization for the Batten-1 clinical trial

THX Pharma, in collaboration with the Beyond Batten Disease Foundation (BBDF) and Biocodex, announced that it has submitted applications for approval of the Phase 3 clinical trial of Batten-1 in juvenile Batten disease (CLN3). Applications have been submitted to the FDA, MHRA, and the EU Centralized System. Patient recruitment could begin by the end of the year.

The phase 3 study, Batten-1-02, aims to demonstrate the efficacy of Batten-1 in juvenile patients with still-evaluable visual acuity. Biocodex is fully funding this study, which will take place in specialized centers in Europe and the United States.

The leaders of the three organizations emphasize the importance of this advance for families and the hope it represents for children suffering from this rare and degenerative disease.