Denali Sees Some Early Progress With Fusion Protein For Hunter Syndrome

But Share Price Knocked Back

Denali Therapeutics has reported promising early clinical data in Hunter syndrome for its fusion protein, DNL310, an investigational agent that crosses the blood-brain barrier and is expected to address disease-associated CNS effects; however, some biomarker data disappointed, and there are potential competitors also under development.

hunter syndrome brain
• Source: Alamy

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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