Biogen Inc. (Nasdaq: BIIB) today announced that the first
patient has been treated in the global clinical study, RESPOND. The
Phase 4 study will examine the clinical benefit and assess the
safety of SPINRAZA® (nusinersen) in infants and children with
spinal muscular atrophy (SMA) who still have unmet clinical needs
following treatment with gene therapy Zolgensma® (onasemnogene
abeparvovec). RESPOND will be conducted at approximately 20 sites
worldwide and aims to enroll up to 60 children with SMA.
“SMA treatments have changed what is possible for
children born with the disease but they have also raised new
questions,” said Dr. Nicole Gusset, President of SMA Europe and
mother of a child with SMA. “We appreciate that the RESPOND study
will collect data to help provide answers so individuals living
with SMA can make informed treatment decisions.”
Children with SMA do not produce enough survival
motor neuron (SMN) protein, which is critical for the maintenance
of motor neurons that support sitting, walking and basic functions
of life like breathing and swallowing. The RESPOND study will seek
to understand if the proven efficacy of SPINRAZA and its mechanism
of action, which leads to continuous production of SMN protein, may
also benefit patients who have been insufficiently treated with
gene therapy.
“In clinical practice, there is a sense of urgency
to address motor neuron loss in SMA from the earliest sign or even
prior to symptoms, to prevent additional disease progression,” said
Julie Parsons, M.D., professor of Clinical Pediatrics and Neurology
and Haberfeld Family Endowed Chair in Pediatric Neuromuscular
Disorders at Children’s Hospital Colorado and the University of
Colorado School of Medicine, and primary investigator of the
RESPOND study. “In some patients treated with gene therapy, we have
recognized that further motor neuron protection may be needed. Our
hope is that results from RESPOND will demonstrate if SPINRAZA can
optimize treatment for some of our youngest patients.”
RESPOND is a two-year, open-label study to evaluate
the efficacy and safety of SPINRAZA in SMA patients previously
treated with Zolgensma to further optimize treatment decisions. The
primary endpoint is the total score on the Hammersmith Infant
Neurological Examination Section 2. Secondary endpoints include
safety, change from baseline on additional motor function measures,
other clinical outcomes (e.g., swallowing) and caregiver burden.
Neurofilament levels, an exploratory endpoint, will also be
evaluated as a marker of biological disease activity.
The study will enroll 60 children up to 3 years old
who are determined by the investigator to have the potential for
additional clinical improvement after receiving Zolgensma. It has
been reported that, to date, 40 percent of children in the
long-term study of Zolgensma have been subsequently treated with
SPINRAZA.1 Physicians will use criteria that may include one or
more of the following: suboptimal motor function (e.g., a score
lower than 50 on the Children’s Hospital of Philadelphia Infant
Test of Neuromuscular Disorders [CHOP INTEND]); the need for
respiratory support; abnormal swallowing or feeding ability; or
other factors deemed relevant by the investigator.
The primary study group will include 40 infants
aged 9 months or younger (at the time of first SPINRAZA dose) who
have 2 copies of SMN2 (likely to develop SMA Type 1)
and received Zolgensma at 6 months old or younger. A second study
group will include 20 children within a broader age range (up to 3
years old at the time of first SPINRAZA dose). After a screening
period, participants will receive the approved 12 mg dose of
SPINRAZA: four loading doses, followed by maintenance doses every
four months,5 over the two-year study period.
More information on the study (NCT04488133) is
available at clinicaltrials.gov.
About
SPINRAZA® (nusinersen)
SPINRAZA is approved to treat infants, children and adults with
spinal muscular atrophy (SMA) and is available in more than 50
countries. As a foundation of care in SMA, more than 11,000
individuals have been treated with SPINRAZA worldwide.6
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.5 It is administered directly into the central nervous system,
where motor neurons reside, to deliver treatment where the disease
starts.5
SPINRAZA has demonstrated sustained efficacy across
ages and SMA types with a well-established safety profile based on
data in patients treated up to 7 years, combined with unsurpassed
real-world experience.7 The SPINRAZA clinical development program
encompasses 10 clinical studies, which have included more than 300
individuals across a broad spectrum of patient populations,7
including two randomized controlled studies (ENDEAR and CHERISH).
The ongoing 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), the leader in antisense therapeutics. 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 Spinal Muscular Atrophy
(SMA)SMA is a rare, genetic, neuromuscular disease that
affects individuals of all ages. It is characterized by a loss of
motor neurons in the spinal cord and lower brain stem, resulting in
progressive muscle atrophy and weakness.8 SMA is caused by a
deficiency in the production of survival motor neuron (SMN) protein
due to a damaged or missing SMN1 gene, with a spectrum of disease
severity.8 Some individuals with SMA may never sit; some sit but
never walk; and some walk but may lose that ability over time.9 In
the absence of treatment, children with the most severe form of SMA
would not be expected to reach their second birthday.8
SMA impacts approximately one in 11,000 live
births,10 is a leading cause of genetic death among infants10 and
causes a range of disability in teenagers and adults.9
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, Alzheimer’s disease and dementia, neuromuscular
disorders, movement disorders, 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. Follow us on
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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 the potential
benefits, safety and efficacy of SPINRAZA; the results of certain
real-world data; the identification and treatment of SMA; our
research and development program for the treatment of SMA; the
potential benefits and results from early treatment of SMA; the
enrollment of the RESPOND study; risks and uncertainties associated
with drug development and commercialization; and the potential of
our commercial business, including SPINRAZA. 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
that we may not fully enroll the RESPOND study or it will take
longer than expected; unexpected concerns that may arise from
additional data, analysis or results obtained during the RESPOND
study; the occurrence of adverse safety events; risks of unexpected
costs or delays; the risks of other unexpected hurdles; 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 are based on
our current beliefs and expectations and speak only as of the date
of this news release. We do not undertake any obligation to
publicly update any forward-looking statements, whether as a result
of new information, future developments or otherwise.
References:
- Zolgensma EU Summary of Product Characteristics (SmPC).
Available
at: https://www.ema.europa.eu/en/documents/product-information/zolgensma-epar-product-information_en.pdf.
Accessed: December 2020.
- Finkel R, et al. Presented at the Muscular Dystrophy
Association (MDA) 2020 Clinical & Scientific Conference.
- Harada Y, et al. Presented at the Muscular Dystrophy
Association (MDA) 2020 Clinical & Scientific Conference.
- Finkel R, et al. Presented at the World Muscle Society’s (WMS)
2020 Virtual Congress.
- 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: December 2020.
- Based on commercial patients, early access patients, and
clinical trial participants through September 30, 2020.
- Core Data Sheet, Version 9, January 2019. SPINRAZA. Biogen Inc,
Cambridge, MA.
- National Institute of Neurological Disorders and Stroke, NIH.
Spinal Muscular Atrophy Fact Sheet. Available at
https://www.ninds.nih.gov/Disorders/Patient-Caregiver-Education/Fact-Sheets/Spinal-Muscular-Atrophy-Fact-Sheet.
Accessed: December 2020.
- Wadman RI, Wijngaarde CA, Stam M, et al. Muscle strength and
motor function throughout life in a cross-sectional cohort of 180
patients with spinal muscular atrophy types 1c–4. Eur J Neurol.
2018;25(3):512-518.
- Cure SMA. About SMA. Available at
https://www.curesma.org/about-sma/. Accessed: December 2020.
MEDIA CONTACT:David Caouette+ 1 617 679
4945public.affairs@biogen.com |
INVESTOR CONTACT:Joe Mara+1 781 464 2442IR@biogen.com |
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