Denali Therapeutics announces interim results from Phase 1/2 study of DNL310

Denali Therapeutic announced new interim results from the ongoing open-label, single-arm Phase 1/2 study of DNL310 in children with MPS II. Among the 13 participants who reached two years of treatment at the time of the interim analysis, a mean reduction of 64% from baseline in serum neurofilament light was observed. The U.S. Food and Drug Administration, FDA, recently recommended to Denali the assessment of NfL as an exploratory endpoint to assess its potential as a possible biomarker to assess diagnostic, prognostic, or therapeutic response in subjects with neuronopathic MPS II. DNL310 is an investigational brain-penetrant enzyme replacement therapy designed to address the behavioral, cognitive, and physical manifestations of MPS II. The global Phase 2/3 COMPASS study is ongoing. “The robust reduction and normalization of CSF heparan sulfate, and now the downstream reduction in NfL after treatment, are consistent with positive changes in clinical outcomes measures we have observed from interim analyses of the ongoing Phase 1/2 study,” said Carole Ho, M.D., Chief Medical Officer at Denali. “As we advance DNL310 as a potential treatment option for individuals living with MPS II, we look forward to ongoing engagement with the FDA and the MPS community.”