Denali Therapeutics Announces Initiation of Phase 1b Study of EIF2B Activator DNL343 in ALS
09 September 2021 - 11:00PM
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 ALSThe
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 ALSModulation 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 TherapeuticsDenali
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.
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 progress, business plans, business strategy, product
candidates, planned preclinical studies and clinical trials and
expected milestones; EIF2B activators as therapy for amyotrophic
lateral sclerosis; plans, timelines and expectations related to
DNL343, including its use as a potential treatment of people living
with amyotrophic lateral sclerosis; and statements made by 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: any
and all risks to Denali’s business and operations caused directly
or indirectly by the evolving COVID-19 pandemic; risk of the
occurrence of any event, change or other circumstance that could
give rise to the termination of Denali’s collaboration agreements;
Denali’s early stages of clinical drug development; Denali’s and
its partners’ ability to complete the development and, if approved,
commercialization of its product candidates; Denali’s and its
partners’ ability to enroll patients in its ongoing and future
clinical trials; Denali’s reliance on third parties for the
manufacture and supply of its product candidates for clinical
trials; Denali’s and it's partners' ability to conduct or complete
clinical trials on expected timelines; the risk that preclinical
profiles of Denali’s product candidates may not translate in
clinical trials; the potential for clinical trials or clinical
trials of any other product candidates to differ from preclinical,
preliminary or expected results; the uncertainty that product
candidates will receive regulatory approval necessary to be
commercialized; Denali’s ability to continue to create a pipeline
of product candidates or develop commercially successful products;
Denali’s ability to obtain, maintain, or protect intellectual
property rights related to its product candidates; implementation
of Denali’s strategic plans for its business, product candidates
and blood-brain barrier platform technology; and other risks,
including those described in Denali’s most recent Quarterly Report
on Form 10-Q filed with the Securities and Exchange Commission
(SEC) on August 4, 2021, Denali’s Annual Report on Form 10-K filed
with the SEC on February 26, 2021, and Denali’s future reports to
be filed with the SEC. The forward-looking statements in this press
release are based on information available to Denali as of the date
hereof. Denali disclaims any obligation to update any
forward-looking statements, except as required by law.
Investor Relations Contact: |
Media Contacts: |
Laura Hansen, Ph.D.Vice
President, Investor Relations(650) 452-2747hansen@dnli.com |
Lizzie Hyland(646)
495-2706lizzie.hyland@fgh.comorMorgan Warners(202)
295-0124morgan.warners@fgh.com |
Denali Therapeutics (NASDAQ:DNLI)
Historical Stock Chart
From Jun 2024 to Jul 2024
Denali Therapeutics (NASDAQ:DNLI)
Historical Stock Chart
From Jul 2023 to Jul 2024