Denali Therapeutics Announces Fast Track Designation Granted by the U.S. FDA to ETV:IDS (DNL310) for the Treatment of Patie...
March 11 2021 - 7:30AM
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 the U.S. Food
and Drug Administration (FDA) has granted Fast Track designation
to ETV:IDS (DNL310) for the treatment of patients with
Hunter syndrome (MPS II). DNL310, Denali’s lead
investigational brain-penetrant enzyme replacement therapy, is
under evaluation in a Phase 1/2 study in patients with Hunter
syndrome as a potential treatment for both central nervous system
(CNS) and peripheral manifestations of the disease.
“Hunter syndrome is a devastating disease for
which current approved treatments fail to effectively cross the
blood-brain barrier and therefore do not address CNS symptoms,”
said Carole Ho, M.D., Denali’s Chief Medical Officer. “Using
Denali’s Transport Vehicle technology, we have designed DNL310 to
treat both body and brain in Hunter syndrome after intravenous
administration. Receiving Fast Track designation is an important
milestone in the DNL310 development program, and we look forward to
our continued collaboration with the FDA to bring safe and
effective treatments to patients with Hunter syndrome.”
Fast Track is an FDA process designed to
facilitate the development and expedite the review of drugs to
treat serious conditions and fill an unmet medical need. Denali
expects that Fast Track designation may allow for early and
frequent communication with the FDA regarding the development of
DNL310 for the treatment of Hunter syndrome. This designation also
enables rolling review, and potentially priority review, of the
marketing application.
About the DNL310 Development Program for
the Potential Treatment of Hunter Syndrome (MPS II)Hunter
syndrome (MPS II) is a rare neurodegenerative lysosomal storage
disorder caused by a mutation in the gene that encodes for the
enzyme iduronate-2-sulfatase (IDS). The resultant reduction or loss
of IDS enzyme activity leads to accumulation of glycosaminoglycans
(GAGs), which causes lysosomal dysfunction and neurodegeneration as
well as progressive damage to multiple organs including bone,
cartilage, heart and lung. Current standard of care enzyme
replacement treatment does not address neuronopathic manifestations
of the disease as it does not sufficiently cross the BBB. DNL310 is
a fusion protein composed of IDS fused to Denali’s proprietary
Enzyme Transport Vehicle (ETV), which is engineered to cross the
BBB via receptor-mediated transcytosis into the brain. Denali
previously announced human biomarker proof-of-concept for its TV
technology from Cohort A (n=5) of an ongoing Phase 1/2 study of
DNL310 in patients with Hunter syndrome (ClinicalTrials.gov
Identifier: NCT04251026). The study is currently enrolling Cohort
B, and a Cohort C is planned to further explore clinical endpoints.
DNL310 is an investigational drug and is not approved by any health
authority.
About Denali’s
TV Platform The BBB is essential in maintaining
the brain’s microenvironment and protecting it from harmful
substances and pathogens circulating in the bloodstream.
Historically, the BBB has posed significant challenges to drug
development for CNS diseases by preventing most drugs from reaching
the brain in therapeutically relevant concentrations. Denali’s
TV platform is a proprietary technology designed to effectively
deliver large therapeutic molecules such as antibodies, enzymes,
proteins, and oligonucleotides across the BBB after intravenous
administration. The TV technology is based on engineered Fc
fragments that bind to specific natural transport receptors, such
as transferrin receptor, which are expressed at the BBB and deliver
TV and its therapeutic cargo to the brain through receptor-mediated
transcytosis. In animal models, antibodies and enzymes engineered
with the TV technology demonstrate more than 10- to 30-fold greater
brain exposure than similar antibodies and enzymes without this
technology. Improved exposure and broad distribution in the brain
may increase therapeutic efficacy by enabling widespread
achievement of therapeutically relevant concentrations of product
candidates. ETV:IDS (DNL310) is Denali’s lead TV-enabled
program in Phase 1/2 development for the potential treatment of
Hunter syndrome (MPS II).
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.
Cautionary Note Regarding
Forward-Looking StatementsThis 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 and
business plans; plans, timelines and expectations related to
DNL310, timing and frequency of interactions with the FDA about,
and its review of applications for, the DNL310 development program
for Hunter syndrome, Denali’s TV technology platform, and the
ongoing Phase 1/2 study, and planned future studies, of DNL310; the
therapeutic potential of DNL310 and Denali’s TV platform; 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 agreements with its
partners; 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 dependence on successful development of its
blood-brain barrier platform technology and TV-enabled product
candidates; Denali’s and its partners' ability to conduct or
complete clinical trials on expected timelines; the potential for
clinical trial results of DNL310 to differ from preclinical,
preliminary or expected results, whether DNL310 will cause any
serious adverse events, whether DNL310 will impact downstream
biomarkers of neurodegeneration, and that DNL310 may not receive
regulatory approval as a treatment of Hunter syndrome necessary to
be commercialized; 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. 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 and 10-Q filed with the Securities and Exchange
Commission (SEC) on February 26, 2021, 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 Relations
Contact: Laura Hansen,
Ph.D. Vice President, Investor Relations (650)
452-2747 hansen@dnli.comMedia
Contacts: Lizzie Hyland (646)
495-2706 lhyland@gpg.com or Morgan
Warners (202) 295-0124 mwarners@gpg.com
Denali Therapeutics (NASDAQ:DNLI)
Historical Stock Chart
From Sep 2024 to Oct 2024
Denali Therapeutics (NASDAQ:DNLI)
Historical Stock Chart
From Oct 2023 to Oct 2024