Denali Therapeutics Inc. (NASDAQ: DNLI) has revealed upcoming presentations from the DNL310 (ETV: IDS) clinical development initiative to be issued virtually and at the 18th Annual WORLD Symposium, expected to take place on February 7, through 11, 2022, in San Diego, California. DNL310 is an experimental brain-penetrant enzyme replacement therapy for treating the central nervous system and peripheral manifestations of MPS II.
Poster presentation to outline expected Phase 2/3 clinical study of DNL310
Longer-term safety, biomarker (lysosomal and glycosaminoglycans lipids), and exploratory clinical results on Cohorts A and B from the current Phase 1/2 clinical study of DNL310 in MPS II will be presented in an oral presentation. A poster presentation will outline a planned Phase 2/3 clinical study of DNL310 in MPS II, which could lead to registration. On February 7 and February 10, PDFs of the oral and poster presentations will be uploaded on Denali’s company website under the Investor Events tab.
The first poster is titled “Interim 49-week results of a phase 1/2 study of intravenous DNL310 (brain-penetrant enzyme replacement therapy) in MPS II,” to be presented during the Contemporary Forum Platform Presentations session on Thursday, February 10, 2022, by Anna Bakardjiev at 8:00 AM. Jeff Harris will present the other poster entitled, “A blinded randomized phase 2/3 study of the efficacy and safety of intravenous DNL310 (brain-penetrant enzyme replacement therapy) in MPS II,” on Thursday, February 10, 2022, between 3:00 to 5:00 PM pacific times.
MPS II results from mutation of iduronate-2-sulfatase enzyme gene
Hunter syndrome (MPS II) is an uncommon degenerative lysosomal storage disorder resulting from mutations in the iduronate-2-sulfatase enzyme gene (IDS). As a result of the decreased or lost IDS enzyme activity, glycosaminoglycans accumulate, causing lysosomal malfunction and neurodegeneration and also gradual damage to many organs such as cartilage, bone, the heart, and the lungs. Because it does not sufficiently pass the blood-brain barrier, the existing standard of care replacement therapy partly cures peripheral indications of MPS II but does not treat neuronopathic features of the disease.
