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Denali Therapeutics Announces First Human Biomarker Proof of Concept for Its Transport Vehicle (TV) Technology Achieved in Phase 1/2 Study of ETV:IDS (DNL310) in Hunter Syndrome (MPS II)
After four weekly intravenous doses of DNL310, a 76% mean reduction in CSF GAG levels (heparan sulfate) from baseline was observed, with normal healthy levels
About this update from Denali Therapeutics Inc.
[{"type":"text","content":"After four weekly intravenous doses of DNL310, a 76% mean reduction in CSF GAG levels (heparan sulfate) from baseline was observed, with normal healthy levels being achieved in four of five patientsBased on Cohort A safety data review, an independent data monitoring committee recommended continuing the study without modifications, enabling progression to Cohort B, including enrollment of younger patients, and continuation of dose escalation in Cohort AData provide first biomarker proof of concept in humans for Denali’s proprietary TV technology designed to deliver biotherapeutics to the brainManagement will host a webinar at 8:30 a.m. ET today SOUTH SAN FRANCISCO, Calif., Nov. 10, 2020 (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 neurodegenerative diseases, today announced biomarker proof of concept was achieved for its Transport Vehicle (TV) technology in a Phase 1/2 study of ETV:IDS (DNL310) for the potential treatment of Hunter syndrome (MPS II). Denali’s TV platform is a proprietary technology designed to effectively deliver large therapeutic molecules such as antibodies, enzymes, proteins, and oligonucleotides across the BBB after intravenous administration.\n Hunter syndrome is a rare neurodegenerative lysosomal storage disorder caused by a mutation in the gene that encodes for the enzyme iduronate 2-sulfatase (IDS). The resultant reduction or loss of IDS enzyme activity leads to accumulation of glycosaminoglycans (GAGs), which causes lysosomal dysfunction and neurodegeneration as well as progressive damage to multiple organs including bone, cartilage, heart and lung. Current standard of care enzyme replacement treatment (ERT) does not address neuronopathic manifestations of the disease as it does not sufficiently cross the BBB. “The robust reduction in cerebrospinal fluid (CSF) GAG levels observed after only four weeks of treatment with DNL310 in the Phase 1/2 study exceeded our initial goal in both magnitude and timing of heparan sulfate reduction after treatment,” said Carole Ho, M.D., Denali’s Chief Medical Officer. “These results are encouraging because, in preclinical models of Hunter syndrome treated with intravenous DNL310, a 50% reduction in CSF GAGs is associated wit...