Denali Therapeutics Presented Results from P-I/II Clinical Trial for DNL310 in Children to Treat MPS II (Hunter Syndrome) at SSIEM 2023
Shots:
- The P-I/II clinical trial evaluates the safety & efficacy of DBL310 in children with MPS II (Hunter Syndrome)
- The results showed normal & sustained levels of CSF heparan sulfate (incl. patients with high pre-existing anti-drug Abs) & lysosomal lipids (improved lysosomal function) along with a reduction in serum NfL (statistical significance after 61wks. & 64% reduction after 2 years) and improvements in mean cognitive BSID-III, VABS-II & ABR at 49wks.
- Denali's Enzyme Transport Vehicle (ETV) and IDS are combined to form DNL310, designed to penetrate the blood-brain barrier and enter the brain by receptor-mediated transcytosis. Denali expects to continue the evaluation of DNL310 in the P-II/III (COMPASS) clinical trial
Ref: GlobeNewswire | Image: Denali
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