Theranexus and BBDF Announce Positive Results at 18 Months for Batten-1 in Phase 1/2 Study

Theranexus and the Beyond Batten Disease Foundation (BBDF) confirmed positive results after 18 months of Batten-1 treatment in a Phase 1/2 study in Batten Disease (CLN3). Data shows a reduction in neurofilament light chains (NfL), a biomarker of neuronal degeneration, indicating the therapeutic potential of Batten-1.

The results were consistent with those obtained after 12 months, with an average reduction of 33% in NfL in patients' serum. This biomarker, which reflects neuronal death, has continued to decline, reinforcing the interest in Batten-1 for the juvenile form of Batten disease.

Professor Gary Clark, principal investigator of the study, highlighted the absence of notable progression of motor symptoms in patients, confirming the importance of Batten-1 for the management of this disease.

Theranexus and BBDF have implemented a compassionate access program allowing patients to continue treatment, given its favorable benefit-risk ratio. The results will be presented at the “Translational Research Conference for the Management of NCLs” in November 2024.