Biogen Inc. (Nasdaq: BIIB) announced new SPINRAZA® (nusinersen)
data aimed at answering critical questions for the spinal muscular
atrophy (SMA) community. The data were presented at the SMA
Research & Clinical Care Meeting hosted by Cure SMA this week
in Orlando, Fla.
“Cure SMA’s annual conference is a unique opportunity to connect
with and learn from the health care providers, patients and
caregivers in attendance and share research intended to address the
unmet needs of the SMA community,” said Maha Radhakrishnan, M.D.,
Chief Medical Officer at Biogen. “We are pleased to present our new
data, including early results from the RESPOND study evaluating the
clinical benefit and safety of SPINRAZA treatment after gene
therapy.”
Interim Clinical Outcomes from RESPOND
RESPOND is an ongoing two-year, phase 4 open-label study to
evaluate clinical outcomes and safety following treatment with
SPINRAZA in infants and toddlers with SMA who have unmet clinical
needs after treatment with Zolgensma® (onasemnogene abeparvovec).
Interim efficacy results at six months from 29* study participants
treated with SPINRAZA show:
- Improvements in motor function in most participants as measured
by increased mean total Hammersmith Infant Neurological Examination
Section 2 (HINE-2) score from baseline
- Participants with two SMN2 copies (n=24) improved by a mean of
over 5 points on HINE-2
- All participants with three SMN2 copies (n=3) improved; a mean
change from baseline was not calculated due to the small number of
participants
- Most participants (25/27) with
investigator-reported suboptimal motor function at baseline
improved
After a median of 230.5 days in the study, serious adverse
events (AEs) were reported in 13/38 (34%) participants. No serious
AEs were considered related to SPINRAZA or led to study withdrawal.
No new emerging safety concerns have been identified in enrolled
participants who received SPINRAZA after Zolgensma. Additional
interim clinical outcomes from the RESPOND study are being
presented at the conference.
“We are learning that gene therapy may not be treating all motor
neurons leaving the potential for disease progression,” said
Crystal Proud, M.D., Pediatric Neurologist at Children’s Hospital
of the King’s Daughters. “The RESPOND study has begun to
characterize remaining unmet need in some SMA patients treated with
Zolgensma whose outcomes have not met clinical expectations. These
interim results provide the community with the first clinical study
data evaluating SPINRAZA treatment following Zolgensma and suggest
there may be potential for additional benefit with SPINRAZA
treatment.”
New Analysis Evaluating Real-World Impact of
SPINRAZA
A systematic literature review and meta-analysis evaluating
real-world impact of SPINRAZA for infantile-onset SMA was presented
and highlights the importance of generating real-world evidence to
achieve a comprehensive understanding of the treatment benefits of
SPINRAZA. Improvements in motor function and motor milestones
observed in real-world studies were greater than or comparable to
those observed in clinical trials, and patients continued to
improve with longer duration of SPINRAZA treatment.
Progress on Novel Device Aimed at Enhancing Treatment
Experience
Together with Alcyone Therapeutics, Biogen is working to develop
the first implantable device designed to enable routine
subcutaneous access for delivery of antisense oligonucleotide
therapies. This week, Alcyone announced that the U.S. Food and Drug
Administration has approved an Investigational Device Exemption to
initiate a pivotal trial of the ThecaFlex DRx™ System (ThecaFlex).
This summer, Alcyone plans to begin initial enrollment of the
PIERRE study (clinicaltrials.gov), which will evaluate the safety
and performance of ThecaFlex for the delivery of SPINRAZA in SMA
patients.
About
SPINRAZA® (nusinersen)SPINRAZA is
approved in more than 60 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
root cause of SMA 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 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 global biotechnology company that has pioneered multiple
breakthrough innovations including a broad portfolio of medicines
to treat multiple sclerosis, the first approved treatment for
spinal muscular atrophy, and two co-developed treatments to address
a defining pathology of Alzheimer’s disease. Biogen is advancing a
pipeline of potential novel therapies across neurology,
neuropsychiatry, specialized immunology and rare diseases and
remains acutely focused on its purpose of serving humanity through
science while advancing a healthier, more sustainable and equitable
world.
We routinely post information that may be important to investors
on our website at www.biogen.com. Follow us on social media -
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Biogen Safe HarborThis 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 the potential benefits, safety and
efficacy of nusinersen; the potential benefits of our
collaborations, including with Alcyone; the potential benefits of
an implantable device designed to enable the administration of ASO
therapies; the results of certain real-world data; our research and
development program for the identification and treatment of SMA;
clinical development programs, clinical trials and data readouts
and presentations; the potential benefits and results from
treatment of SMA; 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. 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 relating to
the occurrence of adverse safety events and/or unexpected concerns
that may arise from additional data or analysis; the risk that we
may not fully enroll our clinical trials, or enrollment will take
longer than expected; failure to obtain regulatory approvals in
other jurisdictions; risks of unexpected 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; regulatory authorities may require
additional information or further studies; product liability
claims; third party collaboration risks; and the direct and
indirect impacts of the ongoing COVID-19 pandemic on our 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 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.
Note: * Two participants in
the RESPOND study were not assessed at Day 183 and, therefore, not
included in the mean calculation.
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: June 2023.
- Core Data sheet, Version 13, October 2021. SPINRAZA. Biogen
Inc, Cambridge, MA.
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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