Biogen Inc. (Nasdaq: BIIB) announced interim 6-month biomarker data
from the initial 29 participants in the open-label RESPOND study.*
The Phase 4 study evaluates clinical outcomes and safety following
treatment with SPINRAZA over a 2-year period in infants and
toddlers with spinal muscular atrophy (SMA) who have unmet clinical
needs after treatment with Zolgensma® (onasemnogene abeparvovec).
The new data show that plasma neurofilament light chain (NfL)
levels, an objective biomarker of axonal injury and
neurodegeneration, were reduced in nearly all study participants
treated with SPINRAZA. These data will be presented at the 2024
Muscular Dystrophy Association (MDA) Clinical & Scientific
Conference (March 3-6, 2024).
“Our evolving understanding of gene therapy indicates there may
be an opportunity for better outcomes,” said Crystal Proud, M.D.,
Pediatric Neurologist at Children’s Hospital of the King’s
Daughters. “Improvements in motor function together with decreases
in neurofilament levels seen after treatment with SPINRAZA in
RESPOND show that we may be able to further maximize benefits for
patients.”
Today NfL data are being presented from study participants
treated with SPINRAZA for 6 months showing:
Among participants with 2 SMN2 copies:
- All participants had elevated baseline NfL levels relative to
healthy children of similar age
- In infants (n=11) who were 9 months or younger at first
SPINRAZA dose (mean baseline NfL: 148.3 pg/mL), NfL levels
decreased by a mean of 70% from baseline.
- In children (n=11) over 9 months of age at first SPINRAZA dose
(mean baseline NfL: 121.8 pg/mL), NfL levels decreased by a mean of
78% from baseline.
Among participants with 3 SMN2 copies:
- Baseline NfL levels were elevated in 2 of 3 children (mean:
60.6 pg/mL).
- NfL reductions were observed in those with elevated levels at
baseline and remained stable in the participant without an elevated
level at baseline.
“Biogen is at the forefront of pioneering research aimed at
advancing biomarkers to accelerate development of drugs for people
living with devastating neurodegenerative diseases like SMA and
ALS,” said Priya Singhal, M.D., M.P.H., Head of Development and
interim Chief Medical Officer at Biogen. “Prior to receiving
SPINRAZA at the start of RESPOND, we saw that participants had
elevated neurofilament levels, as compared to healthy children
suggesting ongoing neuronal injury. The RESPOND findings underscore
the value of neurofilament as an objective marker for assessing
remaining unmet needs in SMA patients who have previously received
gene therapy.”
As reported at the SMA Research & Clinical Care Meeting in
June 2023 from the same 29 participants, improvements in motor
function were observed in most participants as measured by
increased mean total Hammersmith Infant Neurological Examination
Section 2 (HINE-2) score from baseline.1 No new emerging safety
concerns have been identified in enrolled RESPOND participants who
received SPINRAZA after Zolgensma. After a median of 230.5 days in
the study, serious adverse events (AEs) were reported in 13/38
(34%) participants. Any AEs were reported in 31/38 (81.6%)
participants. No serious AEs were considered related to SPINRAZA or
led to study withdrawal, although some were related to
administration.
These data and additional data from the RESPOND study will be
presented at subsequent conferences this year including the 4th
International Congress on Spinal Muscular Atrophy hosted by SMA
Europe.
About SPINRAZA® (nusinersen)SPINRAZA 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.2
SPINRAZA is an antisense oligonucleotide (ASO) that targets the
root cause of SMA by continuously increasing the amount of
full-length survival motor neuron (SMN) protein produced in the
body.3 It is administered directly into the central nervous system,
where motor neurons reside, to deliver treatment where the disease
starts.3
SPINRAZA has demonstrated sustained efficacy across ages and SMA
types with a well-established safety profile based on data in
patients treated up to 8 years,3 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 SHINE and NURTURE open-label extension studies
are 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 patient’s 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 Harbor This news release
contains forward-looking statements, 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:
- Proud C. Interim results from the ongoing RESPOND study
evaluating nusinersen in children with spinal muscular atrophy
previously treated with onasemnogene abeparvovec. June 2023. SMA
Research & Clinical Care Meeting. Orlando, Fla.
- 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: February 2024.
- Core Data sheet, Version 13, October 2021. SPINRAZA. Biogen
Inc, Cambridge, MA.
* Clinical outcomes and NfL were analyzed in the 29 participants
who had the opportunity for at least six months of treatment at the
time of the interim analysis. Analysis of mean change in NfL
includes participants with baseline and Day 183 assessments; a mean
change from baseline was not reported in the 3 SMN2 copies group,
due to the small number of participants. Safety data are reported
in all participants (n=38) who received at least one dose of
SPINRAZA in the trial.
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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