Biogen Inc. (Nasdaq: BIIB) today announced positive, topline data
from the pivotal cohort (Part B) of the Phase 2/3 DEVOTE study
evaluating the safety and efficacy of a higher dose regimen of
nusinersen in treatment-naïve, symptomatic infants with spinal
muscular atrophy (SMA). The investigational higher dose regimen of
nusinersen comprises a more rapid loading regimen, two 50 mg doses
14 days apart, and a higher maintenance regimen, 28 mg, every 4
months, compared to the approved nusinersen regimen (SPINRAZA). The
study met its primary endpoint at six months, achieving a
statistically significant improvement in motor function in infants
who received the higher dose regimen as compared to a prespecified
matched sham (untreated) control group from the ENDEAR study.
“While there has been remarkable progress in the treatment of
SMA, there remains significant unmet need. Building on the
well-characterized profile of SPINRAZA established over the past 10
years, we continue to explore the potential for maximizing efficacy
outcomes while maintaining our commitment to safety,” said
Stephanie Fradette, Pharm.D., Head of the Neuromuscular Development
Unit at Biogen. “The encouraging topline results from DEVOTE
show that the higher dose regimen can slow neurodegeneration
faster, as shown by greater reductions in neurofilament at day 64
relative to the approved dose. Over time, the higher dose regimen
led to meaningful clinical benefit in infants with symptomatic SMA.
We look forward to sharing the detailed results with the SMA
community and health authorities.”
DEVOTE is a three-part study that enrolled 145 patients across
ages and SMA types. The pivotal Part B cohort was comprised of
treatment-naïve children with infantile-onset SMA (n=75) who were
randomized 2:1 to receive the investigational higher dose regimen
of nusinersen or the approved 12 mg regimen (comprising four
loading doses and maintenance doses every four months). The primary
endpoint of Part B measured the change from baseline on the
Children's Hospital of Philadelphia-Infant Test of Neuromuscular
Disorders (CHOP-INTEND) at six months comparing the higher dose
regimen of nusinersen to a matched, untreated sham control group
from the Phase 3 ENDEAR study. ENDEAR is one of the two pivotal
studies that formed the basis of regulatory approval for SPINRAZA®
12 mg.
The higher dose cohort showed statistically significant
improvement over the matched sham comparator on the primary
endpoint of change in CHOP-INTEND from baseline to six months
(least squares mean difference: 26.19; p<0.0001). Results
favored the higher dose regimen relative to sham across secondary
endpoints and trended in favor of the higher dose regimen over the
currently approved 12mg regimen on key biomarker and efficacy
measures. The higher dose regimen was generally well tolerated,
with reported adverse events generally consistent with SMA and the
known safety profile of nusinersen. The percentage of serious
adverse events was lower in the higher dose regimen (60%; 30) as
compared to the 12 mg group (72%; 18). Detailed results from DEVOTE
will be presented at upcoming medical conferences.
More information about the DEVOTE study (NCT04089566) is
available at clinicaltrials.gov. Nusinersen is currently
commercialized under the brand name SPINRAZA and the U.S. Food
and Drug Administration-approved dose is 12 mg.
About the DEVOTE StudyDEVOTE was a Phase 2/3
randomized, controlled, dose-escalating study designed to evaluate
the safety, tolerability, pharmacokinetics and potential for even
greater efficacy of nusinersen when administered at a higher dose
(50 mg/28 mg) than currently approved regimen (12 mg) for the
treatment of spinal muscular atrophy (SMA). The study enrolled 145
patients across ages and SMA types at approximately 42 sites around
the world. DEVOTE includes an open-label safety evaluation cohort
(Part A), a double-blind, active control randomized treatment
cohort (Part B), followed by an open-label treatment cohort (Part
C) to assess the safety and tolerability of transitioning patients
from the currently approved dose of SPINRAZA to the higher dose
being tested in the study. Part B is comprised of an
infantile-onset cohort which is considered pivotal and a
later-onset cohort.
About SPINRAZASPINRAZA is approved in more than
71 countries to treat infants, children and adults with spinal
muscular atrophy (SMA). As a foundation of care in SMA, more than
14,000 individuals have been treated with SPINRAZA
worldwide.1
SPINRAZA is an antisense oligonucleotide (ASO) that targets the
underlying cause of motor neuron loss by continuously increasing
the amount of full-length survival motor neuron (SMN) protein
produced in the body.2 It is administered directly into the
central nervous system, where motor neurons reside, to deliver
treatment where the disease starts.2
SPINRAZA has shown sustained efficacy across ages and SMA types
with a well-established safety profile based on data in patients
treated up to 10 years,3,4 combined with unsurpassed
real-world experience. The nusinersen clinical development program
encompasses more than 10 clinical studies, which have included more
than 460 individuals across a broad spectrum of patient
populations, including two randomized controlled studies
(ENDEAR and CHERISH). The NURTURE open-label extension study is
evaluating the long-term impact of SPINRAZA. The most common
adverse events observed in clinical studies were respiratory
infection, fever, constipation, headache, vomiting and back pain.
Laboratory tests can monitor for renal toxicity and coagulation
abnormalities, including acute severe low platelet counts, which
have been observed after administration of some ASOs.
Biogen licensed the global rights to develop, manufacture and
commercialize SPINRAZA from Ionis Pharmaceuticals, Inc. (Nasdaq:
IONS). Please click here for Important Safety Information and full
Prescribing Information for SPINRAZA in the U.S., or visit your
respective country’s product website.
About BiogenFounded in 1978, Biogen is a
leading biotechnology company that pioneers innovative science to
deliver new medicines to transform patients’ lives and to create
value for shareholders and our communities. We apply deep
understanding of human biology and leverage different modalities to
advance first-in-class treatments or therapies that deliver
superior outcomes. Our approach is to take bold risks, balanced
with return on investment to deliver long-term growth.
We routinely post information that may be important to investors
on our website at www.biogen.com. Follow us on social
media - Facebook, LinkedIn, X, YouTube.
Biogen Safe HarborThis news release contains
forward-looking statements, including related to the potential
clinical effects of SPINRAZA; the potential benefits, safety and
efficacy of SPINRAZA; the clinical development program for
SPINRAZA; the identification and treatment of SMA; our research and
development program for the treatment of SMA; the potential of our
commercial business and pipeline programs, including SPINRAZA; 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,” “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 our forward-looking statements.
These statements involve risks and uncertainties that could
cause actual results to differ materially from those reflected in
such statements, including without limitation, uncertainty of
success in the development and potential commercialization of
SPINRAZA; the risk that we may not fully enroll our clinical trials
or enrollment will take longer than expected; unexpected concerns
may arise from additional data, analysis or results obtained during
our clinical trials; regulatory authorities may require additional
information or further studies, or may fail or refuse to approve or
may delay approval of our drug candidates, including SPINRAZA; the
occurrence of adverse safety events; the risks of unexpected
hurdles, costs or delays; failure to protect and enforce our data,
intellectual property and other proprietary rights and
uncertainties relating to intellectual property claims and
challenges; product liability claims; 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 our
expectations in any forward-looking statement. Investors should
consider this cautionary statement, as well as the risk factors
identified in our most recent annual or quarterly report and in
other reports we have filed with the U.S. Securities and Exchange
Commission. These statements speak only as of the date of this news
release.
We do not undertake any obligation to publicly update any
forward-looking statements.
References:
- Based on commercial patients, early
access patients, and clinical trial participants through December
31, 2022.
- SPINRAZA U.S. Prescribing
Information. Available
at:https://www.spinraza.com/content/dam/commercial/specialty/spinraza/caregiver/en_us/pdf/spinraza-prescribing-information.pdf.
Accessed: July 2024.
- Core Data sheet, Version 13,
October 2021. SPINRAZA. Biogen Inc, Cambridge, MA.
- Finkle et al. Cure SMA 2024. “Final
Safety and Efficacy Data From the SHINE Study in Participants With
Infantile-Onset and Later-Onset SMA “
MEDIA CONTACT:BiogenJack Cox+1
781-464-3260public.affairs@biogen.com |
INVESTOR CONTACT:BiogenChuck
Triano+1 781-464-2442IR@biogen.com |
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