Denali Says Hunter Syndrome Candidate Lowers Disease-Associated Biomarker
Denali Therapeutics Inc (NASDAQ: DNLI) announced new interim results from the ongoing open-label, single-arm Phase 1/2 study of DNL310 IDS in children with MPS II (Hunter syndrome).
Among the 13 participants who reached two years of treatment at the time of the interim analysis, a mean reduction of 64% (p<0.001) from baseline in serum neurofilament light (NfL) was observed.
NfL is a key biomarker of neurodegeneration.
Also Read: Also Read: Biogen and Denali Realign Parkinson's Disease Research Priorities Amidst R&D Restructure.
The 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 aneuronopathic 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.
Additional interim data from the Phase 1/2 study of DNL310 will be highlighted in an oral presentation at the Society for the Study of Inborn Errors of Metabolism Annual Symposium 2023 in Jerusalem, Israel, August 29 – September 1, 2023.
Price Action: DNLI shares are up 2.74% at $31.89 on the last check Tuesday.
Don't miss real-time alerts on your stocks - join Benzinga Pro for free! Try the tool that will help you invest smarter, faster, and better.
This article Denali Says Hunter Syndrome Candidate Lowers Disease-Associated Biomarker originally appeared on Benzinga.com
.
© 2023 Benzinga.com. Benzinga does not provide investment advice. All rights reserved.