Denali Therapeutics Inc.’s brain-penetrating enzyme replacement therapy DNL310 (ETV:IDS) has shown beneficial clinical effects in children with Hunter syndrome (MPS II), in interim data from a Phase I/II study, although the performance of a new potential biomarker for the condition apparently disappointed some investors.
The South San Francisco, CA-based firm said it was excited about the results emerging from the study, which continue to validate the company’s blood-brain barrier technology, and was accelerating efforts to start a pivotal Phase II/III study in the first half of next year
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