- Denali Therapeutics Inc (NASDAQ: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.
Novartis Announced Investigational Oral Therapy Iptacopan SSubstantially Reduced Both Intra- and Extravascular Hemolysis When Given as Monotherapy in a Phase Ii Study of Anti-C5 Naïve Paroxysmal Nocturnal Hemoglobinuria Patients
Novartis (NYSE: NVS) today announced new Phase II data for iptacopan (LNP023), an investigational oral treatment for paroxysmal nocturnal hemoglobinuria (PNH), presented at the 26th Annual Congress of the European Hematology Association (EHA).
