Denali Therapeutics Announces Completion of Enrollment for Regimen G Evaluating eIF2B Agonist DNL343 in the Phase 2/3 HEALEY ALS Platform Trial
May 01 2024 - 8:00AM
Denali Therapeutics Inc. (Nasdaq: DNLI), a biopharmaceutical
company developing a broad portfolio of product candidates
engineered to cross the blood-brain barrier (BBB) for the treatment
of neurodegenerative and lysosomal storage diseases, today
announced that the Sean M. Healey & AMG Center in collaboration
with the Northeast ALS Consortium (NEALS) completed enrollment for
Regimen G of the Phase 2/3 HEALEY ALS Platform Trial, which
evaluates 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 the Julieanne Dorn
Professor of Neurology at Harvard Medical School. “We are grateful
for the continued support of 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 conclusion of enrollment for Regimen G marks a critical
step forward for the HEALEY ALS platform Trial," said Drs. Paganoni
and Babu. "We extend our thanks to Denali, academic collaborators,
and benefactors of the trial as we move to the next stage."
“We are thrilled with the achievement of 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 to deliver
effective treatment options for people living 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. Buildup of TDP-43
is harmful and leads to neuronal degeneration. In the lab,
inhibition of the ISR by DNL343 dissolves TDP-43 containing stress
granules and decreases 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. Results from both studies demonstrated
that once-daily oral dosing with DNL343 was generally well
tolerated and exhibited extensive cerebrospinal fluid (CSF)
penetration. In addition, robust inhibition of biomarkers
associated with the ISR pathway was observed in blood samples from
study participants. DNL343 is an investigational therapeutic and
has not been approved by any regulatory authority for any
commercial use.
About the HEALEY ALS Platform Trial
The HEALEY ALS Platform Trial is a large-scale collaborative
effort 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 that enter the platform trial are chosen by
a group of expert ALS scientists and members of
the Healey & AMG Center.
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 the treatment of
neurodegenerative and lysosomal storage 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, statements
regarding Denali's plans, timelines, and expectations related to
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 made by the Healey ALS Platform Trial's
principal investigator and Denali's Chief Medical Officer. Actual
results are subject to risks and uncertainties and may differ
materially from those indicated by these forward-looking statements
as a result of these risks and uncertainties, including but not
limited to, risks related to: Denali’s dependence on successful
development of its BBB platform technology and TV-enabled 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 on expected timelines; Denali’s reliance
on third parties for the manufacture and supply of its product
candidates for clinical trials; the potential for clinical trial
results to differ from preclinical, early clinical, preliminary or
expected results; the risk of significant adverse events,
toxicities, or other undesirable side effects; the risk that
results from early clinical biomarker studies will not translate to
clinical benefit in late clinical studies; the risk that product
candidates may not receive regulatory approval necessary to be
commercialized; developments relating to Denali’s competitors and
its industry, including competing product candidates and therapies;
Denali’s ability to obtain, maintain, or protect intellectual
property rights; and other risks and uncertainties. In light of
these risks, uncertainties, and assumptions, the forward-looking
statements in this press release are inherently uncertain and may
not occur, and actual results could differ materially and adversely
from those anticipated or implied in the forward-looking
statements. Accordingly, you should not rely upon forward-looking
statements as predictions of future events. Information regarding
additional risks and uncertainties may be found in Denali’s Annual
and Quarterly Reports filed on Forms 10-K with the Securities and
Exchange Commission (SEC) on February 28, 2024, and Denali’s future
reports to be filed with the SEC. Denali does not undertake any
obligation to update or revise any forward-looking statements, to
conform these statements to actual results or to make changes in
Denali’s expectations, except as required by law.
Investor and Media Contact:Laura Hansen, PhD
Vice President, Investor Relations (650) 452-2747
hansen@dnli.com
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