Biogen (Nasdaq: BIIB) and Eisai, Co., Ltd. (Tokyo, Japan) today
announced that, after consulting with the U.S. Food and Drug
Administration (FDA), Biogen plans to pursue regulatory approval
for aducanumab, an investigational treatment for early Alzheimer’s
disease (AD). The Phase 3 EMERGE Study met its primary endpoint
showing a significant reduction in clinical decline, and Biogen
believes that results from a subset of patients in the Phase 3
ENGAGE Study who received sufficient exposure to high dose
aducanumab support the findings from EMERGE. Patients who received
aducanumab experienced significant benefits on measures of
cognition and function such as memory, orientation, and language.
Patients also experienced benefits on activities of daily living
including conducting personal finances, performing household chores
such as cleaning, shopping, and doing laundry, and independently
traveling out of the home. If approved, aducanumab would become the
first therapy to reduce the clinical decline of Alzheimer’s disease
and would also be the first therapy to demonstrate that removing
amyloid beta resulted in better clinical outcomes.
The decision to file is based on a new analysis, conducted by
Biogen in consultation with the FDA, of a larger dataset from the
Phase 3 clinical studies that were discontinued in March 2019
following a futility analysis. This new analysis of a larger
dataset that includes additional data that became available after
the pre-specified futility analysis shows that aducanumab is
pharmacologically and clinically active as determined by
dose-dependent effects in reducing brain amyloid and in reducing
clinical decline as assessed by the pre-specified primary endpoint
Clinical Dementia Rating-Sum of Boxes (CDR-SB). In both studies,
the safety and tolerability profile of aducanumab was consistent
with prior studies of aducanumab.
“With such a devastating disease that affects tens of millions
worldwide, today’s announcement is truly heartening in the fight
against Alzheimer’s. This is the result of groundbreaking research
and is a testament to Biogen’s steadfast determination to follow
the science and do the right thing for patients,” said Michel
Vounatsos, Chief Executive Officer at Biogen. “We are hopeful about
the prospect of offering patients the first therapy to reduce the
clinical decline of Alzheimer’s disease and the potential
implication of these results for similar approaches targeting
amyloid beta.”
Based on discussions with the FDA, the Company plans to file a
Biologics License Application (BLA) in early 2020 and will continue
dialogue with regulatory authorities in international markets
including Europe and Japan. The BLA submission will include data
from the Phase 1/1b studies as well as the complete set of data
from the Phase 3 studies.
The Company aims to offer access to aducanumab to eligible
patients previously enrolled in the Phase 3 studies, the long-term
extension study for the Phase 1b PRIME study, and the EVOLVE safety
study. Biogen will work towards this goal with regulatory
authorities and principal investigators with a sense of
urgency.
Study ResultsEMERGE (1,638 patients) and ENGAGE
(1,647 patients) were Phase 3 multicenter, randomized,
double-blind, placebo-controlled, parallel-group studies designed
to evaluate the efficacy and safety of two dosing regimens of
aducanumab. These studies were discontinued on March 21, 2019,
following the results of a pre-specified futility analysis which
relied on an earlier and smaller dataset. The futility analysis was
based on data available as of December 26, 2018, from 1,748
patients who had the opportunity to complete the 18-month study
period and predicted that both studies were unlikely to meet their
primary endpoint upon completion. Futility analyses are common in
large clinical studies and use statistical modeling to attempt to
predict the outcome of the studies based on a number of
pre-specified assumptions and criteria.
Following the discontinuation of EMERGE and ENGAGE, additional
data from these studies became available resulting in a larger
dataset, which included a total of 3,285 patients, 2,066 of whom
had the opportunity to complete the full 18 months of treatment. A
new extensive analysis of this larger dataset showed a different
outcome than the outcome predicted by the futility analysis.
Specifically, the new analysis of this larger dataset showed EMERGE
to be statistically significant on the pre-specified primary
endpoint (P=0.01). Biogen believes that data from a subset of
ENGAGE support the findings from EMERGE, though ENGAGE did not meet
its primary endpoint. Biogen consulted with external advisors and
the FDA on these different results and their implications.
“This large dataset represents the first time a Phase 3 study
has demonstrated that clearance of aggregated amyloid beta can
reduce the clinical decline of Alzheimer’s disease, providing new
hope for the medical community, the patients, and their families,”
said Dr. Anton Porsteinsson, William B. and Sheila Konar Professor
of Psychiatry, Neurology and Neuroscience, director of the
University of Rochester Alzheimer's Disease Care, Research and
Education Program (AD-CARE), and principal investigator. “There is
tremendous unmet medical need, and the Alzheimer’s disease
community has been waiting for this moment. I commend Biogen, the
FDA, the medical community, and the patients and their study
partners for their persistence in working to make today’s
announcement a reality.”
In EMERGE, which met its pre-specified primary endpoint in the
new analysis, patients treated with high dose aducanumab showed a
significant reduction of clinical decline from baseline in CDR-SB
scores at 78 weeks (23% versus placebo, P=0.01). In EMERGE,
patients treated with high dose aducanumab also showed a consistent
reduction of clinical decline as measured by the pre-specified
secondary endpoints: the Mini-Mental State Examination (MMSE; 15%
versus placebo, P=0.06), the AD Assessment Scale-Cognitive Subscale
13 Items (ADAS-Cog 13; 27% versus placebo, P=0.01), and the AD
Cooperative Study-Activities of Daily Living Inventory Mild
Cognitive Impairment Version (ADCS-ADL-MCI; 40% versus placebo,
P=0.001). Imaging of amyloid plaque deposition in EMERGE
demonstrated that amyloid plaque burden was reduced with low and
high dose aducanumab compared to placebo at 26 and 78 weeks
(P<0.001). Additional biomarker data of tau levels in the
cerebrospinal fluid supported these clinical findings. Biogen
believes that data from patients in ENGAGE who achieved sufficient
exposure to high dose aducanumab supported the findings of
EMERGE.
In both studies, the most commonly reported adverse events were
amyloid-related imaging abnormalities-edema (ARIA-E) and headache.
The majority of patients with ARIA-E did not experience symptoms
during the ARIA-E episode, and ARIA-E episodes generally resolved
within 4 to 16 weeks, typically without long-term clinical
sequelae. Biogen plans to present further detail on the new
analysis of the larger dataset from EMERGE and ENGAGE at the
Clinical Trials on Alzheimer's Disease (CTAD) meeting in December
2019.
After reviewing the data in consultation with the FDA, Biogen
believes that the difference between the results of the new
analysis of the larger dataset and the outcome predicted by the
futility analysis was largely due to patients’ greater exposure to
high dose aducanumab. Multiple factors contributed to the greater
exposure to aducanumab in the new analysis of the larger dataset,
including data on a greater number of patients, a longer average
duration of exposure to high dose, the timing of protocol
amendments that allowed a greater proportion of patients to receive
high dose, and the timing and pre-specified criteria of the
futility analysis.
Biogen Conference Call and WebcastOn October
22, 2019, at 8:00 a.m. ET, Biogen will host its third quarter 2019
earnings conference call, which will include a discussion of the
new analysis of the larger dataset from the Phase 3 studies of
aducanumab. This conference call will be broadcast via the internet
and will be accessible through the Investors section of Biogen’s
website, www.biogen.com. Following the live webcast, an archived
version of the call will be available on the website. Supplemental
information in the form of a slide presentation is also accessible
at the same location on the internet and will be subsequently
available on the website for at least one month.
About AducanumabAducanumab (BIIB037) is an
investigational human monoclonal antibody studied for the treatment
of early Alzheimer’s disease. Biogen licensed aducanumab from
Neurimmune under a collaborative development and license agreement.
Since October 2017 Biogen and Eisai have collaborated on the
development and commercialization of aducanumab globally.
EMERGE and ENGAGE were Phase 3 multicenter, randomized,
double-blind, placebo-controlled, parallel-group studies designed
to evaluate the efficacy and safety of aducanumab. The primary
objective of the studies was to evaluate the efficacy of monthly
doses of aducanumab as compared with placebo in reducing cognitive
and functional impairment as measured by changes in the CDR-SB
score. Secondary objectives were to assess the effect of monthly
doses of aducanumab as compared to placebo on clinical decline as
measured by MMSE, ADAS-Cog 13, and ADCS-ADL-MCI.
About BiogenAt Biogen, our mission is clear: we
are pioneers in neuroscience. Biogen discovers, develops, and
delivers worldwide innovative therapies for people living with
serious neurological and neurodegenerative 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, Kenneth Murray, and Nobel Prize winners
Walter Gilbert and Phillip Sharp. Today Biogen has the leading
portfolio of medicines to treat multiple sclerosis, has introduced
the first approved treatment for spinal muscular atrophy,
commercializes biosimilars of advanced biologics, and is focused on
advancing research programs in multiple sclerosis and
neuroimmunology, neuromuscular disorders, movement disorders,
Alzheimer’s disease and dementia, ophthalmology, immunology,
neurocognitive disorders, acute neurology, and pain.
We routinely post information that may be important to investors
on our website at www.biogen.com. To learn more, please
visit www.biogen.com and follow us on social media
– Twitter, LinkedIn, Facebook, YouTube.
About Eisai Co., Ltd.Eisai Co., Ltd. is a
leading global research and development-based pharmaceutical
company headquartered in Japan. Eisai’s corporate philosophy is to
give first thought to patients and their families, and to increase
the benefits that health care provides to them. Under this
philosophy, the company endeavors to become a human health care
(hhc) company. With approximately 10,000 employees working across
our global network of R&D facilities, manufacturing sites and
marketing subsidiaries, we strive to realize our hhc philosophy by
delivering innovative products to address unmet medical needs, with
a particular focus in our strategic areas of Neurology and
Oncology.
Leveraging the experience gained from the development and
marketing of Aricept®, a treatment for Alzheimer’s disease and
dementia with Lewy bodies, Eisai has been working to establish a
social environment that involves patients in each community in
cooperation with various stakeholders including the government,
healthcare professionals and care workers, and is estimated to have
held over ten thousand dementia awareness events worldwide. As a
pioneer in the field of dementia treatment, Eisai is striving to
not only develop next generation treatments but also to develop
diagnosis methods and provide solutions.
For more information about Eisai Co., Ltd., please visit
www.eisai.com.
Biogen Safe Harbor This news release
contains forward-looking statements, including statements made
pursuant to the safe harbor provisions of the Private Securities
Litigation Reform Act of 1995, about additional results from the
Phase 3 clinical studies of aducanumab; the potential clinical
effects of aducanumab; the potential benefits, safety, and efficacy
of aducanumab; potential regulatory discussions, submissions, and
approvals and the timing thereof; clinical development programs,
clinical trials, data readouts, and presentations related to
aducanumab; the enrollment of any future clinical studies of
aducanumab; the treatment of AD; the potential of Biogen’s
commercial business and pipeline programs, including aducanumab;
the anticipated benefits and potential of Biogen’s collaboration
arrangements with Eisai; and risks and uncertainties associated
with drug development and commercialization. These statements may
be identified by words such as “aim,” “anticipate,” “believe,”
“could,” “estimate,” “expect,” “forecast,” “goal,” “intend,” “may,”
“plan,” “possible,” “potential,” “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 actual timing and
content of submissions to and decisions made by the regulatory
authorities regarding aducanumab; regulatory submissions may take
longer or be more difficult to complete than expected; regulatory
authorities may require additional information or further studies,
or may fail or refuse to approve or may delay approval of Biogen’s
drug candidates, including aducanumab; actual timing and enrollment
of future studies of aducanumab; the occurrence of adverse safety
events and/or unexpected concerns that may arise from additional
data or analysis; risks of unexpected costs or delays; the risks of
other unexpected hurdles; uncertainty of success in the development
and potential commercialization of aducanumab; failure to protect
and enforce Biogen’s data, intellectual property, and other
proprietary rights and uncertainties relating to intellectual
property claims and challenges; risks relating to the potential
launch of aducanumab, including preparedness of healthcare
providers to treat patients, the ability to obtain and maintain
adequate reimbursement for aducanumab, and other unexpected
difficulties or hurdles; product liability claims; and third party
collaboration risks. 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 risk factors
identified in Biogen’s most recent annual or quarterly report and
in other reports it 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 news
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.
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Contacts |
MEDIA CONTACT:Biogen Inc.David Caouette+ 617 679
4945public.affairs@biogen.com INVESTOR CONTACT:Biogen Inc.Joe
Mara+781 464 2442IR@biogen.com |
MEDIA CONTACT:Eisai Co., Ltd.Public Relations DepartmentTEL:
+81-(0)3-3817-5120Eisai Inc.Public Relations DepartmentTEL:
+1-551-262-2686 INVESTOR CONTACT:Eisai Co., Ltd.Investor Relations
DepartmentTEL: +81-(0)3-3817-5327 |
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