DNLI - Denali up 4% on encouraging data on DNL310 in rare lysosomal storage disorder

Denali Therapeutics (DNLI) announces successful biomarker proof-of-concept for its Transport Vehicle ((TV)) technology in a Phase 1/2 clinical trial evaluating ETV-IDS (DNL310) for the potential treatment of mucopolysaccharidosis type II ((MPSII)), a rare inherited lysosomal storage disorder in which large sugar molecules called glycosaminoglycans ((GAG)) build up in the body's tissues due to the absence or dysfunction of an essential enzyme.Four weeks after treatment with DNL310, significant reductions in GAG levels in cerebrospinal fluid ((CSF)) were observed. Specifically, CSF levels of heparan sulfate, a GAG that accumulates in MPS disorders with central nervous system involvement, dropped 76% from baseline. 80% (n=4/5) of patients achieved normal levels of heparan sulfate.Dose escalation will continue as will the evaluation of the effects of DNL310 on additional downstream biomarkers of neurodegeneration.The company says its TV platform is designed to enable large therapeutic molecules (e.g., antibodies, enzymes, proteins, oligonucleotides) to cross the blood-brain barrier after intravenous

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Denali up 4% on encouraging data on DNL310 in rare lysosomal storage disorder