Theranexus and BBDF Announce Positive Results for Batten-1 Phase 1/2 Study

Theranexus company in collaboration with the Beyond Batten Disease Foundation (BBDF) revealed the final results of the phase 1/2 study of the drug Batten-1, treating the juvenile form of Batten disease (CLN3). The data, after 18 months of treatment, show a significant stabilization of motor symptoms in the six young adult participants.

In these patients, the mean score on the modified UBDRS scale, a measure of motor symptoms, showed very limited progression compared to a control study, suggesting significant effectiveness of the treatment. Theranexus CEO Mathieu Charvériat highlighted the encouragement that these results represent for patients and their families, and discussed the upcoming launch of a phase 3 trial, already approved by the FDA and EMA.