Thursday, August 31, 2023
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."
"We are excited to share sustained effects on key disease biomarkers in MPS II with DNL310 treatment, now out to two years," said Carole Ho, MD, Chief Medical Officer of Denali. "Importantly, the robust reduction in serum levels of NfL suggests improvement in neuronal health, which is not addressed by current enzyme replacement therapies. We are encouraged to see positive changes across multiple clinical outcomes measures in the ongoing Phase 1/2 study. We look forward to continued partnership with the MPS community as we enroll the global Phase 2/3 COMPASS study in MPS II and plan for expanding the enzyme transport vehicle, including initiating clinical studies in other lysosomal storage diseases."
About Denali Therapeutics
Denali Therapeutics is a biopharmaceutical company developing a broad portfolio of product candidates engineered to cross the blood-brain barrier for neurodegenerative diseases and lysosomal storage diseases. Denali pursues new treatments by rigorously assessing genetically validated targets, engineering delivery across the blood-brain barrier, and guiding development through biomarkers that demonstrate target and pathway engagement. Denali is based in South San Francisco. For additional information, please visit www.denalitherapeutics.com.
Investor and Media Contact:
Laura Hansen, Ph.D.
Vice President, Investor Relations
(650) 452-2747
[email protected]
