Biogen Inc. (Nasdaq: BIIB) and Denali Therapeutics Inc. (Nasdaq:
DNLI) today announced that dosing has commenced in the global Phase
3 LIGHTHOUSE study to evaluate the efficacy and safety profile of
BIIB122 (DNL151), as compared to placebo in approximately 400
participants with Parkinson’s disease and a confirmed pathogenic
mutation in the leucine-rich repeat kinase 2 (LRRK2) gene.
The primary endpoint of the LIGHTHOUSE study is time to
confirmed worsening, as assessed using the Movement Disorder
Society-Sponsored Revision of the Unified Parkinson’s Disease
Rating Scale (MDS-UPDRS) over the treatment period, up to 180
weeks. Participants will be randomized to receive oral BIIB122 or
placebo once daily.
“Mutations in the LRRK2 gene comprise the most
frequent mutations found in Parkinson’s disease, indicating that
LRRK2 inhibition may be a promising therapeutic approach to the
disease,” said Samantha Budd Haeberlein, Ph.D., Head of
Neurodegeneration Development at Biogen. “The LIGHTHOUSE study will
specifically recruit individuals with a pathogenic mutation in
LRRK2, enabling us to test the genetic hypothesis and implicated
lysosomal pathway. The LIGHTHOUSE study is the largest study ever
undertaken in individuals with Parkinson’s disease caused by a
LRKK2 mutation.”
BIIB122 is an investigational small molecule inhibitor of LRRK2
that was discovered and initially developed by Denali. Denali and
Biogen are co-developing and co-commercializing BIIB122 for the
potential treatment of Parkinson’s disease.
“In collaboration with Biogen, we are excited to be pursuing the
potential of LRRK2 inhibition as an effective treatment for
Parkinson’s disease,” said Carole Ho, M.D., Chief Medical Officer
of Denali. “The initiation of the Phase 3 LIGHTHOUSE study marks an
important milestone in the BIIB122 development program. Together
with the recent initiation of the Phase 2b LUMA study in
early-stage Parkinson’s disease, we hope to have the opportunity to
bring a novel therapeutic option to people living with Parkinson’s
disease.”
More information about LIGHTHOUSE (NCT05418673) is available at
ClinicalTrials.gov.
About LRRK2 and BIIB122Following discovery of
the LRRK2 mutation as a pathogenic genetic factor for Parkinson’s
disease, further research has uncovered that it has the potential
to be a novel therapeutic target for Parkinson’s disease. Mutations
in leucine-rich repeat kinase 2 (LRRK2) account for 4-5% of
familial and 1-2% of sporadic Parkinson’s disease.1,2
BIIB122 is a selective, central nervous system-penetrant small
molecule inhibitor of LRRK2 that is hypothesized to improve
lysosomal dysfunction. BIIB122 is an investigational drug that is
not approved by any regulatory authority, and its safety and
efficacy have not been established.
About BiogenAs pioneers in neuroscience, Biogen
discovers, develops, and delivers worldwide innovative therapies
for people living with serious neurological diseases as well as
related therapeutic adjacencies. One of the world’s first global
biotechnology companies, Biogen was founded in 1978 by Charles
Weissmann, Heinz Schaller, Sir Kenneth Murray, and Nobel Prize
winners Walter Gilbert and Phillip Sharp. Today, Biogen has a
leading portfolio of medicines to treat multiple sclerosis, has
introduced the first approved treatment for spinal muscular
atrophy, and developed the first and only approved treatment to
address a defining pathology of Alzheimer’s disease. Biogen is also
commercializing biosimilars and focusing on advancing one of the
industry’s most diversified pipelines in neuroscience that will
transform the standard of care for patients in several areas of
high unmet need.
In 2020, Biogen launched a bold 20-year, $250 million initiative
to address the deeply interrelated issues of climate, health, and
equity. Healthy Climate, Healthy Lives™ aims to eliminate fossil
fuels across the company’s operations, build collaborations with
renowned institutions to advance the science to improve human
health outcomes, and support underserved communities.
We routinely post information that may be important to investors
on our website at www.biogen.com. Follow us on social
media - Twitter, LinkedIn, Facebook, YouTube.
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.
Biogen Safe HarborThis press release contains
forward-looking statements, made pursuant to the safe harbor
provisions of the Private Securities Litigation Reform Act of 1995,
including statements relating to the potential benefits and results
that may be achieved through Biogen’s collaboration with Denali;
the potential benefits, safety and efficacy of BIIB122 (DNL151) and
other LRRK2 inhibitor molecules; the clinical development program
for BIIB122 (DNL151) and other LRRK2 inhibitor molecules; the
potential benefits of Denali’s TV technology platform and TV
programs including its ATV: anti-amyloid beta program; the
treatment of Parkinson’s disease; the potential of Biogen’s
commercial business and pipeline programs; Biogen’s strategy and
plans; the potential treatment of neurological and
neurodegenerative diseases; and risks and uncertainties associated
with drug development and commercialization. These forward-looking
statements may be accompanied by words such as “aim,” “anticipate,”
“believe,” “could,” “estimate,” “expect,” “forecast,” “goal,”
“intend,” “may,” “plan,” “potential,” “possible,” “will,” “would”
and other words and terms of similar meaning. Drug development and
commercialization involve a high degree of risk, and only a small
number of research and development programs result in
commercialization of a product. Results in early stage clinical
trials may not be indicative of full results or results from later
stage or larger scale clinical trials and do not ensure regulatory
approval. You should not place undue reliance on these statements
or the scientific data presented.
These statements involve risks and uncertainties that could
cause actual results to differ materially from those reflected in
such statements, including, without limitation: risks that the
proposed transaction will be completed in a timely manner or at
all; the possibility that certain closing conditions to the
proposed transaction will not be satisfied; uncertainty as to
whether the anticipated benefits of the proposed collaboration can
be achieved; risks of unexpected hurdles, costs or delays;
uncertainty of success in the development and potential
commercialization of BIIB122 (DNL151) and other undisclosed
neurological targets, which may be impacted by, among other things,
unexpected concerns that may arise from additional data or
analysis, the occurrence of adverse safety events, failure to
obtain regulatory approvals in certain jurisdictions, failure to
protect and enforce Biogen’s data, intellectual property and other
proprietary rights and uncertainties relating to intellectual
property claims and challenges; product liability claims; third
party collaboration risks; and the direct and indirect impacts of
the ongoing COVID-19 pandemic on Biogen’s business, results of
operations and financial condition. The foregoing sets forth many,
but not all, of the factors that could cause actual results to
differ from Biogen’s expectations in any forward-looking statement.
Investors should consider this cautionary statement, as well as the
risks factors identified in Biogen’s most recent annual or
quarterly report and in other reports Biogen has filed with
the U.S. Securities and Exchange Commission. These statements
are based on Biogen’s current beliefs and expectations and speak
only as of the date of this press release. Biogen does not
undertake any obligation to publicly update any forward-looking
statements, whether as a result of new information, future
developments or otherwise.
Denali Safe HarborThis 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
BIIB122 (DNL151), including with respect to the ongoing Phase 2b
LUMA study and the Phase 3 LIGHTHOUSE study; the potential of
BIIB122 (DNL151) to be a treatment for Parkinson’s disease; the
potential for LRRK2 inhibition to impact the underlying biology or
slow the progression of Parkinson’s disease in certain patients;
the potential benefits and likelihood of success of, activity
under, and expectations related to Denali’s collaboration with
Biogen; and statements made by Denali’s Chief Medical Officer and
Biogen’s Head of Neurodegeneration Development. 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
Biogen; Denali’s transition to a late stage clinical drug
development company; Denali’s and Biogen’s ability to advance and
complete the development and, if approved, commercialization of
BIIB122 (DNL151); Denali’s and Biogen’s ability to initiate, enroll
patients in, conduct, and complete the ongoing and planned clinical
trials, including the Phase 2b LUMA and Phase 3 LIGHTHOUSE studies
of BIIB122 (DNL151), on expected timelines; 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 its
current programs and product candidates; the risk that preclinical
profiles of Denali’s product candidates may not translate in
clinical trials; the potential for the ongoing and planned clinical
trials of BIIB122 (DNL151) to differ from preclinical, early
clinical, preliminary or expected results; the risk of significant
adverse events, toxicities or other undesirable side effects; the
uncertainty that product candidates will receive regulatory
approval 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 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 most
recent Annual and Quarterly Reports filed on Forms 10-K and 10-Q
filed with the Securities and Exchange Commission (SEC) on February
28, 2022 and August 8, 2022, respectively, 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 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.
References:
- Healy DG, Falchi M, O’Sullivan SS,
et al. Phenotype, genotype, and worldwide genetic penetrance of
LRRK2-associated Parkinson’s disease: a case-control study. Lancet
Neurol. 2008;7(7):583-90.
- Hernandez DG, Reed X, Singleton AB.
Genetics in Parkinson disease: Mendelian versus non-Mendelian
inheritance. J Neurochem. 2016;139 Suppl 1:59-74. Epub
2016/04/18.
MEDIA CONTACTS:BiogenDan Haro+ 1 617 914
6936public.affairs@biogen.comDenali
TherapeuticsAngela Salerno-Robin+ 1 212 445 8219
Asalerno-robin@dna-comms.com |
INVESTOR CONTACTS:BiogenMike Hencke+1 781 464
2442IR@biogen.com Denali
TherapeuticsLaura Hansen, Ph.D.+ 1 650 452
2747hansen@dnli.com |
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