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Denali Therapeutics Announces Completion of Enrollment for

SOUTH SAN FRANCISCO, Calif., May 1, 2024 (GLOBE NEWSWIRE) — Denali Therapeutics Inc. (Nasdaq: DNLI), a biopharmaceutical company developing a broad portfolio of product candidates designed to cross the blood-brain barrier (BBB) ​​for the treatment of neurodegenerative and lysosomal storage diseases, today announced that it has acquired Sean M. Healey & AMG Center in collaboration with the Northeast ALS Consortium (NEALS) has completed enrollment for Phase 2/3 Regimen G HEALEY ALS platform trialevaluating Denali’s eIF2B agonist DNL343.

“We are excited to move to the next phase of Regimen G and evaluate the effects of DNL343,” said Merit Cudkowicz, MD, MSc, principal investigator and sponsor of the HEALEY ALS Platform Trial, director of the Sean M. Healey & AMG Center for ALS, Chair of the Department of Neurology at MGH, and Julieanne Dorn Professor of Neurology at Harvard Medical School. “We are grateful for the continued support from the ALS community and look forward to sharing the results.”

Regimen G is co-led by Suma Babu, MBBS, MPH, and Sabrina Paganoni, MD, PhD, physician-investigators at the Healey & AMG Center for ALS at MGH.

“The closure of enrollment for Regimen G marks a critical step forward for the HEALEY ALS platform study,” said Drs. Paganoni and Babu. “We express our gratitude to Denali, academic collaborators and benefactors of the trial as we move into the next phase.”

“We are thrilled to have achieved this important clinical milestone in the DNL343 development program and thank the HEALEY ALS Platform Trial investigators and their sites for making this possible, and the participants and families for their participation,” said Carole Ho, MD, Chief Medical Officer of Denali. “We look forward to continued collaboration with the ALS community to advance the science and ultimately provide effective treatment options for people with ALS.”

About DNL343

DNL343 is a novel investigational ALS therapy that targets eIF2B, a central regulator of the integrated stress response (ISR). The ISR appears to be overactive in ALS, leading to the formation of stress granules containing TDP-43. Accumulation of TDP-43 is harmful and leads to neuronal degeneration. In the laboratory, inhibition of ISR by DNL343 resolves TDP-43-containing stress granules and reduces ISR biomarkers. The safety, pharmacokinetics and pharmacodynamics of DNL343 have been characterized in both healthy participants and people with ALS in a Phase 1 (N=47) and a Phase 1b (N=29) study, respectively, with dosing for up to 28 days. . The results of both studies showed that once-daily oral dosing with DNL343 was generally well tolerated and demonstrated extensive cerebrospinal fluid (CSF) penetration. Furthermore, robust inhibition of biomarkers associated with the ISR pathway was observed in blood samples from study participants. DNL343 is an investigational drug and has not been approved for commercial use by any regulatory agency.

About the HEALEY ALS Platform trial

The HEALEY ALS Platform Trial is a large-scale collaboration made possible by contributions from patients and families, clinical trial sites, industry partners and research collaborators to evaluate multiple investigational therapies simultaneously with the goal of accelerating the development of potential new treatments for ALS. The platform trial is led by the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital (MGH) in collaboration with the Northeast ALS Consortium (NEALS). Therapeutic candidates participating in the platform trial will be chosen by a group of expert ALS scientists and members of the Healey & AMG Center.

About Denali Therapeutica

Denali Therapeutics is a biopharmaceutical company developing a broad portfolio of product candidates designed to cross the blood-brain barrier (BBB) ​​for the treatment of neurodegenerative and lysosomal storage diseases. Denali pursues novel 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 located in South San Francisco. For more information please visit www.denalitherapeutics.com.

Cautionary Statement 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, statements regarding Denali’s plans, timelines and expectations regarding DNL343, including the ongoing regimen G of the Phase 2/3 study and the timing and availability of data; the therapeutic potential of DNL343; and statements from the Healey ALS Platform Trial Principal Investigator and Denali’s Chief Medical Officer. Actual results are subject to risks and uncertainties and may differ materially from those indicated in these forward-looking statements as a result of these risks and uncertainties, including but not limited to risks related to: Denali’s dependence on the successful development of its BBB platform technology and TV -compatible product candidates; Denali’s ability to initiate and enroll patients in its current and future clinical trials; Denali’s ability to conduct or complete clinical trials within expected timelines; Denali’s reliance on third parties for the production and supply of its product candidates for clinical trials; the possibility that clinical trial results may differ from preclinical, early clinical, preliminary or anticipated results; the risk of significant side effects, toxicities or other undesirable side effects; the risk that results from early clinical biomarker studies will not translate into clinical benefit in late clinical studies; the risk that product candidates may not receive regulatory approval necessary for commercialization; developments relating to Denali’s competitors and its industry, including competing product candidates and therapies; Denali’s ability to acquire, maintain or protect intellectual property rights; and other risks and uncertainties. In light of these risks, uncertainties and assumptions, the forward-looking statements contained in this press release are inherently uncertain and may not occur, and actual results may differ materially and adversely from those anticipated or implied in the forward-looking statements. Accordingly, you should not rely on forward-looking statements as predictions of future events. Information regarding additional risks and uncertainties can be found in Denali’s annual and quarterly reports filed with the Securities and Exchange Commission (SEC) on February 28, 2024, and in Denali’s future reports filed with the SEC will be submitted. Denali undertakes no obligation to update or revise any forward-looking statements, to conform these statements to actual results or to make any changes to Denali’s expectations, except as required by law.

Investor and media contact:
Laura Hansen, PhD
Vice President, Investor Relations
(650) 452-2747
[email protected]