Business
Denali Therapeutics Announces Presentations on Enzyme Transport Vehicle (ETV) Development Programs for MPS Diseases at the Upcoming WORLDSymposium™
SOUTH SAN FRANCISCO, Calif., Feb. 15, 2023 (GLOBE NEWSWIRE) -- Denali Therapeutics Inc. (Nasdaq: DNLI), a biopharmaceutical company developing a broad
About this update from Denali Therapeutics Inc.
[{"type":"text","content":"SOUTH SAN FRANCISCO, Calif., Feb. 15, 2023 (GLOBE NEWSWIRE) -- Denali Therapeutics Inc. (Nasdaq: DNLI), a biopharmaceutical company developing a broad portfolio of product candidates engineered to cross the blood-brain barrier for the treatment of neurodegenerative diseases and lysosomal storage diseases, today announced upcoming presentations from Enzyme Transport Vehicle (ETV) development programs, DNL310 (ETV:IDS) and DNL126 (ETV:SGSH), to be given at the 19th Annual WORLDSymposium™, which will be held February 22-26, 2023, in Orlando, Florida. DNL310 is an investigational, ETV-enabled, brain-penetrant enzyme replacement therapy designed to address the behavioral, cognitive, and physical manifestations of MPS II (Hunter syndrome). Denali is evaluating DNL310 in the global Phase 2/3 COMPASS study, which is enrolling participants with MPS II in North America, South America, and Europe, and in an open-label, single-arm Phase 1/2 study. DNL126 is Denali’s second most advanced ETV-enabled program in development for the potential treatment of MPS IIIA (Sanfilippo syndrome type A) for which Denali plans to submit an Investigational New Drug (IND) application in the first half of 2023. Presentations on DNL310 will include new interim data on biomarkers, safety, exploratory clinical outcomes and an analysis of toileting ability from the ongoing Phase 1/2 study as well as the Phase 2/3 COMPASS study design. In addition, another oral presentation will highlight data from preclinical studies of DNL126 in an MPS IIIA mouse model. The presentation schedule at WORLDSymposium™ is as follows: Title: Interim analysis of key clinical outcomes from a phase 1/2 study of weekly intravenous DNL310 (brain-penetrant enzyme replacement therapy) in MPS IISession: Clinical Applications Platform PresentationsDate: Friday, February 24, 2023Time: 9:00 AM Eastern TimeTitle: Interim analysis of key clinical outcomes from a phase 1/2 study of weekly intravenous DNL310 (brain-penetrant enzyme replacement therapy) in MPS II (Poster #248)Session: Clinical Applications – Poster Session V Date: Friday, February 24, 2023Time: 3:00 PM - 4:00 PM Eastern Time Title: COMPASS, A double-blinded randomized phase 2/3 study of the efficacy and safety of intravenous DNL310 (brain-penetrant enzyme replacement therapy) in MPS II (Poster #13)Session: Contemporary Forum – Poster...