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Denali Therapeutics Announces New Interim Data from Phase 1/2 Study of DNL310 (ETV:IDS) in MPS II (Hunter Syndrome) at SSIEM 2023

  • Additional biomarker data up to two years of treatment continue to demonstrate rapid and sustained normalization of CSF heparan sulfate to normal healthy levels and improvement in lysosomal function biomarkers
  • Additional safety data up to two years of treatment continue to demonstrate that DNL310 is generally well tolerated
  • Previously reported data showed robust and statistically significant reduction of NfL, a marker of neuronal damage, and positive clinical outcome changes in adaptive behavior, cognition, and auditory function
  • Recruitment in the global Phase 2/3 COMPASS study is ongoing

Excerpt from the Press Release:

SOUTH SAN FRANCISCO, Calif., Aug. 30, 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 data from the ongoing open-label, single-arm Phase 1/2 study of DNL310 (ETV:IDS) in children with MPS II (Hunter syndrome). DNL310 is an investigational enzyme replacement therapy designed to cross the BBB and address the behavioral, cognitive, and physical manifestations of MPS II. The interim data from the Phase 1/2 study of DNL310 were highlighted in an oral presentation at the Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium 2023 in Jerusalem, Israel. A PDF of the Phase 1/2 presentation is available on Denali’s website on the Events page of the Investor section.

“It is encouraging to see normalization of heparan sulfate in CSF as well as reductions in lysosomal lipid biomarkers and neurofilament light over two years, even in patients with high pre-existing anti-drug antibodies,” said Barbara Burton, MD, Attending Physician, Genetics, Genomics and Metabolism at the Ann & Robert H. Lurie Children’s Hospital of Chicago, who presented the Phase 1/2 data at SSIEM. “The treatment effect seen on these biomarkers of neurocognitive decline, along with one-year data demonstrating positive changes in adaptive behavior, cognition, and auditory function, continue to support the potential of DNL310 to offer meaningful benefit for people living with MPS II. I look forward to learning more as recruitment continues in the global Phase 2/3 COMPASS study.”

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