Published on 1 Jul 2023 on Zacks via Yahoo Finance
Denali Therapeutics DNLI announced positive interim results of the ongoing phase I/II study of DNL310 (ETV:IDS) in children with MPS II, which is also known as Hunter syndrome.
The early-stage study is an open-label, single-arm study, evaluating the efficacy and safety of DNL310 in treating MPS II. DNL310 is an investigational brain-penetrant enzyme replacement therapy designed to address the neurological manifestations of MPS II.
Among the 13 patients who reached the two-year treatment mark at the time of the interim analysis, a significant mean reduction of 64% from baseline in serum neurofilament light (NfL) levels was observed.