Denali Therapeutics Announces New Interim Data from DNL310 Phase 1/2 Study for MPS II and DNL126 Preclinical Data for MPS IIIA at WORLDSymposium™

Over 49 weeks of DNL310 (ETV:IDS) treatment in the Phase 1/2 study, positive changes across measures of exploratory clinical outcomes including VABS-II (adaptive behavior) and BSID-III (cognitive capabilities) scores and global impression scales were observed

Interim Phase 1/2 data also suggest that DNL310 improves hearing, as assessed by auditory brainstem response testing

Additional biomarker data out to 49 weeks continue to demonstrate that DNL310 enables rapid and sustained normalization of CSF heparan sulfate to normal healthy levels and improvement in lysosomal function biomarkers

DNL310 safety profile, now with up to two years of treatment, remains consistent with standard of care

Preclinical data on DNL126 (ETV:SGSH) support planned submission of investigational new drug (IND) application

Excerpt from the Press Release:

SOUTH SAN FRANCISCO, Calif., Feb. 22, 2023 (GLOBE NEWSWIRE) — Denali Therapeutics Inc. (NASDAQ: DNLI), a biopharmaceutical company developing a broad portfolio of product candidates engineered to cross the blood-brain barrier for the treatment of neurodegenerative diseases and lysosomal storage diseases, today announced new interim results from the ongoing open-label, single-arm Phase 1/2 study of DNL310 (ETV:IDS) in children with MPS II (Hunter syndrome), including data from additional participants and up to 104 weeks of treatment. DNL310 is an investigational brain-penetrant enzyme replacement therapy designed to address the behavioral, cognitive, and physical manifestations of MPS II. The interim Phase 1/2 data are being presented at the 19th Annual WORLDSymposium™ in Orlando, Florida, February 22-26, 2023.

“We continue to see sustained reductions in CSF heparan sulfate to normal levels in most individuals not previously seen with any other therapy. Furthermore, over 49 weeks, DNL310 treatment is associated with positive changes across measures of adaptive behavior and cognition, global impression, and improvements in measures of hearing function,” said Joseph Muenzer, M.D., Ph.D., Bryson Distinguished Professor in Pediatric Genetics, University of North Carolina at Chapel Hill. “As the study progresses and enrollment continues for the global Phase 2/3 COMPASS study, I am excited to learn more about the potential of DNL310 to offer meaningful benefit for the entire MPS II patient population.”

“We are excited to share new interim analyses from the Phase 1/2 study, including data that suggests hearing improves within a year with DNL310 treatment in participants previously treated with idursulfase,” said Carole Ho, M.D., Chief Medical Officer at Denali.

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