Denali Therapeutics Announces Initiation of Phase 1b Study of EIF2B Activator DNL343 in ALS

— Investigational EIF2B activator approach has potential to be a first-in-class treatment for ALS —

SOUTH SAN FRANCISCO, CA, USA I September 09, 2021 I Denali Therapeutics Inc. (NASDAQ: DNLI), a biopharmaceutical company developing a broad portfolio of product candidates engineered to cross the blood-brain barrier (BBB) for neurodegenerative diseases, today announced that dosing recently commenced in a Phase 1b study of DNL343, its brain-penetrant small molecule activator of EIF2B, in participants diagnosed with amyotrophic lateral sclerosis (ALS).

ALS, often called Lou Gehrig's disease, refers to a group of progressive neurodegenerative diseases that affect nerve cells in the brain and spinal cord, leading to loss of voluntary muscle control and movement. At least 20,000 people in the United States are currently affected and approximately 5,000 individuals are newly diagnosed with ALS each year.

“Initiation of this Phase 1b study marks a significant milestone in the development of DNL343 for the potential treatment of people living with ALS,” said Carole Ho, M.D., Denali’s Chief Medical Officer. “Our preclinical and Phase 1 healthy volunteer data demonstrate activity of DNL343 on biomarkers believed to be highly relevant to ALS, and we look forward to sharing this data at an upcoming scientific conference. This Phase 1b study will help further characterize the safety and activity of DNL343 in participants with ALS for whom treatment options are a critical unmet medical need.”

Denali plans to present the DNL343 Phase 1 healthy volunteer data at the 2021 Annual Northeast Amyotrophic Lateral Sclerosis (NEALS) Meeting on October 6, 2021.

About the Phase 1b study of DNL343 in ALS
The Phase 1b clinical trial (study number NCT05006352) is a multicenter, randomized, placebo-controlled, double-blind, 28-day study followed by an 18-month open-label extension, designed to evaluate the safety, pharmacokinetics, and pharmacodynamics of DNL343 in participants with ALS. Further information on the Phase 1b study can be accessed on the ClinicalTrials.gov website or by clicking here.

About DNL343 and targeting EIF2B as a potential treatment for ALS
Modulation of EIF2B activity with DNL343 is a novel and targeted investigational approach with first-in-class potential for the treatment of ALS. EIF2B is an intracellular protein complex that regulates protein synthesis and is required for neuronal health and function. When neurons experience stress, as occurs in ALS, EIF2B activity is suppressed. This leads to impaired protein synthesis and results in the formation of "stress granules," which are thought to be a precursor of TDP-43 aggregation, a hallmark pathology in ALS. DNL343 is designed to activate EIF2B and thereby restore protein synthesis, disperse TDP-43 aggregates, and improve neuronal survival.

About Denali Therapeutics
Denali Therapeutics is a biopharmaceutical company developing a broad portfolio of product candidates engineered to cross the blood-brain barrier (BBB) for neurodegenerative diseases. Denali pursues new treatments by rigorously assessing genetically validated targets, engineering delivery across the BBB and guiding development through biomarkers that demonstrate target and pathway engagement. Denali is based in South San Francisco. For additional information, please visit www.denalitherapeutics.com.

SOURCE: Denali Therapeutics

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