Denali Therapeutics Inc. (NASDAQ: DNLI) watched its shares go south Monday. The biopharmaceutical company developing a broad portfolio of product candidates engineered to cross the blood-brain barrier (BBB) for neurodegenerative diseases and lysosomal storage disorders, today announced interim results from a Phase 1b study of its eIF2B agonist, DNL343, in participants with amyotrophic lateral sclerosis (ALS).
Once-daily oral dosing with DNL343 for 28 days was generally well tolerated and demonstrated extensive BBB penetration as well as robust inhibition of biomarkers associated with the integrated stress response (ISR) in blood samples from study participants. By inhibiting the ISR pathway, DNL343 is intended to prevent or slow disease progression associated with stress granule formation and TDP-43 aggregation, a hallmark pathology present in nearly all individuals with ALS. The Phase 1b results will be presented at the 33rd International Symposium on ALS/MND, which is being held virtually this week.
Denali also announced initiation of the design phase of a Phase 2/3 study for entry into the HEALEY ALS Platform Trial led by the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital (MGH) in collaboration with the Northeast ALS Consortium.
“These initial Phase 1b results with DNL343 in ALS are consistent with our previously reported Phase 1 healthy volunteer data and are an important milestone for the program,” said Carole Ho, M.D., Chief Medical Officer at Denali.
DNLI shares declined 7.5 cents to $32.55.
