CORRECTION FROM SOURCE: Theranexus and BBDF Win FDA Approval on Efficacy Endpoints for the Phase III Trial to Evaluate Batten-1 in CLN3 Batten Disease - Seite 2

Patient enrollment should begin by the end of 2023. The trial will run in parallel in several centers internationally, including the United States and Europe. Provided positive results are registered, the FDA confirms that a sole Phase III trial as defined above would secure approval of the Batten-1 candidate in CLN3 Batten disease. The company's goal is to use the results of this trial to win product approval in both the United States and Europe.

As a reminder, the Phase III trial follows on from Phase I/II in which initial results showed good safety and tolerability of miglustat in a population of CLN3 patients over 17 years of age, and a pharmacokinetic profile in line with expectations (see press release of 5 January 2023²).

In conclusion, Craig Benson, Chair of the Beyond Batten Disease Foundation , said: "We are absolutely delighted to have had such a constructive and productive meeting with the FDA enabling us to initiate the Phase III clinical trial for Batten-1 in the near future. Patients and their families are eagerly awaiting the launch of enrollment for this trial in a disease for which there is currently no available treatment. This trial is a source of tremendous hope for them."

About Batten-1
Batten-1 is a novel and exclusive proprietary drug containing the active ingredient miglustat. The mechanism of action of this substance blocks the accumulation of glycosphingolipids and neuroinflammation. For patients over 17 years of age in the Phase I/II trial, the product is administered in solid form. In the Phase III trial, it will be administered in a liquid form better suited to pediatric patients.

Phase I/II trial design: this is an open-label trial involving 6 patients over 17 years of age with CLN3 Batten disease, treated with miglustat up to 600 mg/day for a 2-year period. The primary endpoint is patient safety and tolerability, assessed using reports of adverse effects, biological tests and ECG, as well as the pharmacokinetics of miglustat. The secondary endpoints include efficacy monitoring: Unified Batten Disease Rating Scale, visual acuity, and brain MRI and optical coherence tomography scans. Administration of Batten-1 in escalating doses with a maximum of 600 mg/day was well tolerated, with no severe side effects observed causing treatment discontinuation. The most commonly reported adverse events are reversible gastrointestinal effects of often light to moderate severity, thus demonstrating the good tolerability profile of Batten-1 in this population. Batten-1 will continue to be assessed in these patients treated over a 24-month period. Further information about the trial is available on https://clinicaltrials.gov/ct2/show/NCT05174039 .