Denali Therapeutics Announces New Data and Expansion of Its Blood-Brain Barrier (BBB)-Crossing Enzyme Replacement Therapy Programs at WORLDSymposium™
SOUTH SAN FRANCISCO, Calif., Feb. 07, 2024 (GLOBE NEWSWIRE) -- Denali Therapeutics Inc. (NASDAQ: DNLI), a biopharmaceutical company developing a broad portfolio of product candidates engineered to cross the blood-brain barrier (BBB) for the treatment of neurodegenerative diseases and lysosomal storage diseases, today announced new data presentations highlighting the broad potential of its BBB-crossing enzyme replacement therapies in development for the treatment of mucopolysaccharidoses (MPS). New clinical data on tividenofusp alfa (DNL310) in MPS II (Hunter syndrome) and mouse model data on DNL126 (ETV:SGSH) in MPS IIIA (Sanfilippo syndrome type A) are being presented this week at the 20th Annual WORLDSymposium™ in San Diego, California.
“It is exciting to see new clinical outcomes data, now up to two years of treatment with tividenofusp alfa,” said Joseph Muenzer, M.D., Ph.D., Bryson Distinguished Professor in Pediatric Genetics, University of North Carolina at Chapel Hill, who presented the Phase 1/2 data on tividenofusp alfa in MPS II. “The robust and sustained biomarker reductions, including NfL, and the effects on measures of behavior, cognition, hearing, liver volumes and growth indicate positive treatment effects in brain and somatic tissues. Denali’s BBB-crossing enzyme replacement approach has the potential to prevent cognitive and behavioral manifestations in MPS IIIA. Together with Denali and the MPS community, I am passionately advocating for the fastest path to approval to enable new treatment options for people living with MPS diseases.”
In addition, new data on somatic outcomes from the same study of tividenofusp alfa will be presented for the first time by Barbara Burton, M.D., Professor of Pediatrics, Genetics, Genomics and Metabolism at Feinberg School of Medicine in Chicago. The Phase 1/2 results demonstrate normalization of enlarged liver and spleen volumes and maintenance of normal growth compared to healthy boys in almost all participants. New two-year peripheral biomarker data demonstrate high magnitude and sustained reduction of urine heparan sulfate and dermatan sulfate, including in participants who switched from standard-of-care enzyme replacement therapy to tividenofusp alfa, suggesting enhanced peripheral activity. Tividenofusp alfa treatment continued to be generally well tolerated.
New MPS IIIA mouse model data on DNL126 will also be presented at the conference showing improvements in lysosomal and microglial morphology, neurodegeneration, and cognitive function. Treatment with DNL126 resulted in lowered heparan sulfate accumulation in the brain and in cerebrospinal fluid and improved cognitive function in adult MPS IIIA mice. A correlation between the levels of heparan sulfate and cognitive behavioral performance was observed. Denali also announced that dosing has begun in the Phase 1/2 study of DNL126 for the potential treatment of MPS IIIA.
“Our goal is to bring effective new medicines to MPS families as soon as possible,” said Carole Ho, M.D., Chief Medical Officer of Denali. “We are excited to present additional Phase 1/2 data demonstrating the potential for tividenofusp alfa to treat both brain and physical symptoms of MPS II disease, and we look forward to continued collaboration with the MPS community to complete enrollment in our global Phase 2/3 COMPASS study this year. We are also pleased to share that dosing has begun in the Phase 1/2 study of DNL126 for the potential treatment of MPS IIIA. We are driven by the urgent need of patients and families and will work with the community of scientists, physicians, advocates, and the FDA to find the fastest path to approval.”
About MPS II (Hunter syndrome)
About tividenofusp alfa (DNL310)
About the Phase 2/3 COMPASS study
About MPS IIIA (Sanfilippo syndrome Type A)
About DNL126 (ETV:SGSH)
About Denali’s Transport Vehicle Platform
About Denali Therapeutics
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