Denali Therapeutics Announces Broad Pipeline Progress Including Positive Results From Its LRRK2 Program for Parkinson’s Disease - Seite 3

DNL343 (EIF2B Activator) for ALS and other neurodegenerative diseases

Denali submitted a CTA in early December 2019 for DNL343, a small molecule brain-penetrant activator of EIF2B. Mutations in EIF2B are genetically linked to neurodegenerative diseases.  Activation of EIF2B with DNL343 is expected to restore protein synthesis and reduce formation of stress granules, which are commonly associated with disease pathology in ALS and Alzheimer’s disease. The CTA cleared in January 2020, and Denali plans to initiate a Phase 1 clinical trial for DNL343 in healthy volunteers.

Further data from the clinical trials with DNL201 and DNL151, and data from preclinical studies with DNL310 and DNL343 will be shared at the J.P. Morgan 38^th Annual Healthcare Conference on January 14, 2020 at 8.30am PT in San Francisco. The presentation will also be accessible by webcast through Denali’s website at https://www.denalitherapeutics.com/investors/events.

About the DNL151 Phase 1b clinical trial 

This study (NCT04056689) is a 28-day, multicenter, randomized, placebo controlled, double-blind Phase 1b clinical trial in patients with mild-to-moderate Parkinson’s disease. Its purpose is to evaluate safety, tolerability, pharmacokinetics, pharmacodynamics, including target and pathway engagement biomarkers as well as certain exploratory clinical endpoints, after multiple oral doses of DNL151. To date, 25 patients have been enrolled in the study, and the protocol has been amended to expand the study with up to 10 additional patients. Final data readout from this trial is expected to be presented in mid-2020. Further details are available at ClinicalTrials.gov

About Denali

Denali is a biopharmaceutical company developing a broad portfolio of product candidates engineered to cross the 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.

Cautionary Note Regarding Forward Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements expressed or implied in this press release include, but are not limited to, Denali’s plans and expectations to select either  DNL151 or DNL201 in mid-2020 to advance into Phase 2/3 clinical trials in Parkinson’s disease patients; Denali’s expectations regarding what the results of the DNL151 Phase 1b clinical trial will inform; Denali’s expectations regarding patient enrollment in, and the timing, objectives and results of, the DNL151 Phase 1b clinical trial; Denali’s plans to conduct further clinical testing in connection with its LRRK2 program; Denali’s expectations regarding what the results of the DNL747 Phase 1b clinical trials will inform; Denali’s expectations regarding anticipated regulatory and development timelines and plans to initiate clinical trials for DNL310 and DNL343, including timing and objectives; Denali’s expectations that DNL310 will generate proof-of-concept in humans of its Transport Vehicle BBB delivery platform; Denali’s expectations with respect to anticipated benefits from the activation of EIF2B with DNL343; and statements made by Denali’s CMO and CEO.