Denali Therapeutics Reveals New Interim Data From Early-Stage Hunter Syndrome Study - Denali Therapeutics (NASDAQ:DNLI)

Denali Therapeutics Inc DNLI announced new interim results from the ongoing open-label, single-arm Phase 1/2 study of DNL310 in children with MPS II (Hunter syndrome), including data from additional participants and up to 104 weeks of treatment.
Over 49 weeks of DNL310 treatment in the Phase 1/2 study, positive changes across measures of exploratory clinical outcomes, including VABS-II (adaptive behavior) and BSID-III (cognitive capabilities) scores and global impression scales, were observed.
Interim Phase 1/2 data also suggest that DNL310 improves hearing.
The data demonstrate that DNL310 enables rapid and sustained normalization of heparan sulfate in cerebrospinal fluid (CSF) with mean reductions from baseline of 91% and 90% at weeks 24 and 49, respectively.
Normalization of CSF heparan sulfate was observed even in participants with high levels of preexisting anti-iduronate-2-sulfatase antibodies.
A sustained reduction of lysosomal lipid biomarkers in CSF was also observed, consistent with improved lysosomal function.
At week 24, the mean decline in gangliosides GM2, GM3, and glucosylsphingosine lipids were 64%, 54%, and 57%, respectively, sustained at week 49 (63%, 49%, and 48%, respectively).
After switching from idursulfase to DNL310, a mean decline from baseline of 85% and 89% was observed for heparan sulfate and dermatan sulfate biomarkers in the urine, respectively, at week 49, suggesting DNL310 has added peripheral activity over approved enzyme replacement therapy.
Price Action: DNLI shares closed higher by 1.70% at $29.23 on Wednesday.

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