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Denali's Enzyme Replacement Therapy Shows Encouraging Biomarker Effects In Early-Stage Hunter Syndrome Study

  • Denali Therapeutics Inc (NASDAQ: DNLI) has announced additional interim results from its ongoing Phase 1/2 study evaluating ETV:IDS (DNL310) as a potential brain-penetrant enzyme replacement therapy for Hunter syndrome (MPS II).
  • MPS II is a rare neurodegenerative lysosomal storage disorder caused by a mutation in the gene that encodes for the enzyme iduronate-2-sulfatase (IDS), thus accumulating GAGs, which causes lysosomal dysfunction and neurodegeneration as well as progressive damage to multiple organs.
  • Denali will present the data later today during a late-breaker session at WORLD Symposium.
  • The study included five patients whom all received three months of weekly intravenous doses of DNL310 after switching from idursulfase enzyme replacement therapy.
  • Normal levels of heparan sulfate, a glycosaminoglycan (GAG), in cerebrospinal fluid (CSF) that were seen after four weeks of dosing in 4/5 patients were sustained after three months of dosing (mean 85% reduction was observed across the cohort)
  • For the fifth patient, heparan sulfate levels were significantly reduced and approached normal levels (from 25% to 73% reduction from one to three months, respectively).
  • Reductions in downstream exploratory CSF biomarkers, GM3 and BMP, 39% and 15%, respectively, were observed after eight weeks of dosing with DNL310, consistent with an improvement in lysosomal function.
  • Reductions in urine heparan sulfate and dermatan sulfate following a switch from idursulfase, 76% and 82%, respectively, were observed after eight weeks of dosing.
  • DNL310 was generally well tolerated with no dose reductions, and all five patients continue in the study. The most frequent adverse events were mild or moderate infusion-related reactions in three of five patients. 
  • Doses of DNL310 from 3 mg/kg to 30 mg/kg are generally well tolerated and provide flexibility for dose selection in clinical studies.
  • Price Action: DNLI stock is up 4.96% at $73 in the premarket trading on the last check Friday.

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Posted-In: Hunter Syndrome Phase 1 TrialBiotech News FDA General

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