Denali Reports Results of DNL310 in P-I/II Study in Patients with the Lysosomal Storage Disease Hunter Syndrome (MPS II)

 Denali Reports Results of DNL310 in P-I/II Study in Patients with the Lysosomal Storage Disease Hunter Syndrome (MPS II)

Shots:

  • Denali reports data from the P-I/II study evaluating DNL310 (qw, IV) for both CNS and peripheral MPS II. All patients received doses of DNL310 after switching from idursulfase enzyme replacement therapy on Day 1
  • Results of Cohorts A & B showed the levels of heparan sulfate in CSF normalized in all patients analyzed with rapid response observed in patients @7wks., and is consistent with crossing of the BBB by DNL310 and activity in tissues of the CNS
  • Exploratory biomarker data demonstrated reductions in CSF lysosomal lipid biomarkers & high variability in exploratory biomarker Nf-L was observed pre- & post-treatment. Safety profile with up to 43wks. of dosing was consistent with SOC

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