Denali therapeutics announces phase 1/2 study single dose healthy volunteer data with tak-594/dnl593 (ptv:pgrn) and progression to enrolling participants with ftd-grn

South san francisco, calif., nov. 01, 2022 (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 neurodegenerative diseases and lysosomal storage disorders, today announced interim results from part a of a phase 1/2 study evaluating tak-594/dnl593 (ptv:pgrn) in healthy subjects. progranulin (pgrn) levels measured in cerebrospinal fluid (csf) increased in a dose-dependent manner compared to baseline and placebo, consistent with brain delivery of dnl593 and exceeding levels believed to be necessary to rescue deficits associated with progranulin deficiency, based on preclinical models.1 single doses of dnl593 were generally well tolerated, based on blinded safety analysis. these data support dosing in participants with frontotemporal dementia and a mutation in the granulin gene (ftd-grn) in part b of the study. dnl593 is an investigational, brain-penetrant progranulin replacement therapy being co-developed by denali and takeda. these data were presented at the ftd prevention initiative meeting in paris, france. a copy of the presentation is available on denali's website on the investor & media relations section under the events page.
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