Business
Denali Therapeutics Reports Fourth Quarter and Full Year 2022 Financial Results and Business Highlights
SOUTH SAN FRANCISCO, Calif., Feb. 27, 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. 27, 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 (BBB) for the treatment of neurodegenerative diseases and lysosomal storage diseases, today reported financial results for the fourth quarter and year ended December 31, 2022, and provided business highlights. “2022 was an important year for Denali, marked by transition to late-stage clinical development in our therapeutic programs for Parkinson's, MPS II, and ALS, as well as expansion of our Transport Vehicle (TV)-enabled portfolio with first-in-human studies in programs for FTD-GRN and Alzheimer’s,” said Ryan Watts, Ph.D., Denali's Chief Executive Officer. “We remain focused on progressing our broad and diversified therapeutic portfolio, further validating and expanding our TV platform, and building commercial capabilities with the ultimate goal of delivering effective medicines to people living with neurodegenerative and lysosomal storage diseases worldwide.” Recent Program Updates and Expected Milestones: TV-ENABLED PROGRAMS DNL310 (ETV:IDS): MPS II (Hunter syndrome)DNL310 is an investigational, Enzyme Transport Vehicle (ETV)-enabled, brain-penetrant iduronate-2-sulfatase (IDS) replacement therapy designed to address the behavioral, cognitive and physical manifestations of MPS II (Hunter syndrome). In February 2023, at the WORLDSymposiumTM, Denali reported additional interim data from the open-label, single-arm Phase 1/2 study of DNL310. 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. The data also suggested that DNL310 improved hearing, as assessed by auditory brainstem response testing. Additional biomarker data out to 49 weeks continued to demonstrate that DNL310 enabled rapid and sustained normalization of CSF heparan sulfate to normal healthy levels and improvement in lysosomal function biomarkers. The DNL310 safety profile, with up to two years of treatment, remained consistent with standard of care.The Phase 2/3 COMPASS study continues to enroll up to 54 participants with MPS II with and without ...