New SPINRAZA® (nusinersen) Data Reinforce Sustained Efficacy and
Longer-Term Safety Across Broad Range of Spinal Muscular Atrophy
(SMA) Patients
Biogen Inc. (Nasdaq: BIIB) today announced additional data from the
SPINRAZA (nusinersen) clinical development program that further
demonstrate the sustained efficacy and longer-term safety of
SPINRAZA in a broad range of patients with spinal muscular atrophy
(SMA). These new data were selected for presentation at the 72nd
American Academy of Neurology (AAN) annual meeting and will be
available online via the 2020 AAN Science Highlights virtual
platform.
“As the first approved treatment for SMA, SPINRAZA offers a
significant data set that allows us to uniquely assess the safety
and durability of repeated doses over time in individuals across
age groups and varying disease severity,” said Alfred Sandrock,
Jr., M.D., Ph.D., Executive Vice President, Research and
Development at Biogen. “New data show that continuous treatment
with SPINRAZA for up to six and a half years improved or stabilized
motor function and disease activity in a broad spectrum of patients
with SMA. These results are in stark contrast to the expected
natural history of the disease. Further, in a progressive disease
like SMA, stabilization is an important measure of treatment
success, allowing patients to retain motor function that may
otherwise be lost.”
New Data Reinforce Sustained Efficacy and Longer-Term
Safety of SPINRAZA Across Age Groups and SMA TypesThe
SHINE open-label extension study (NCT02594124) has enrolled 292
patients (infants through teenagers) from five previous SPINRAZA
clinical studies, including ENDEAR. New findings from the SHINE
study show treatment with SPINRAZA resulted in motor function
improvement or disease stabilization in toddlers, children and
young adults who were treated continuously, some for up to six and
a half years.
Key highlights include:
- Patients with infantile-onset SMA included in the ENDEAR-SHINE
study (n=105) and who had earlier initiation of SPINRAZA treatment
experienced the greatest benefit, and those with later initiation
showed evidence of motor function stabilization or
improvement.
- A separate analysis evaluated a cohort of seven young adults
(Type 2 or 3) who began treatment with SPINRAZA as teenagers (aged
13 to nearly 16 years old) and have since been treated for up to
six and a half years (range of 5.3 to 6.8 years). Most of these
patients demonstrated generally stable or improved motor function
throughout the follow-up period as assessed by the Hammersmith
Functional Motor Scale Expanded (HFMSE), Revised Upper Limb Module
and Upper Limb Module (RULM/ULM) and Six-Minute Walk Test
(6MWT).- Results also measured the impact on participants’
caregivers via the Assessment of Caregiver Experience with
Neuromuscular Disease (ACEND), with the majority reporting stable
or decreased impact over the same period. ACEND is an outcomes
instrument specifically designed to assess the caregiver impact
experienced by caregivers raising children affected by
neuromuscular disease, including physical, emotional and financial
domains.1
- The durability of SPINRAZA was also demonstrated in individuals
with later-onset SMA (n=126), as HFMSE and RULM scores were
stable.
- In all SHINE presentations, the safety profile of SPINRAZA was
consistent with previously reported findings.
AAN data presentations highlighted in this release include:
- Nusinersen in Infantile-onset Spinal Muscular Atrophy: Results
From Longer-term Treatment From the Open-label SHINE Extension
Study
- Longer-term Experience With Nusinersen in Young Adults With
Spinal Muscular Atrophy: Results From the CS2/CS12 and SHINE
Studies
- Longer-term Treatment With Nusinersen: Results in Later-onset
Spinal Muscular Atrophy From the SHINE Study
- Safety Profile of Nusinersen in Presymptomatic and
Infantile-Onset Spinal Muscular Atrophy (SMA): Interim Results From
the NURTURE and ENDEAR-SHINE Studies
About SPINRAZA® (nusinersen)2-4 SPINRAZA is the
first therapy approved to treat infants, children and adults with
spinal muscular atrophy (SMA) and is approved in more than 50
countries. As of March 31, 2020, more than 10,000 individuals have
been treated with SPINRAZA. It is the only SMA treatment to combine
unsurpassed real-world experience with a robust level of clinical
evidence across a broad spectrum of patient populations.
SMA is a rare, genetic, neuromuscular disease that is
characterized by a loss of motor neurons in the spinal cord and
lower brain stem that can result in severe, progressive muscle
atrophy and weakness. Approximately one in 10,000 live births have
a diagnosis of SMA, and people of all ages are impacted by the
disease. It is a leading genetic cause of infant mortality.
SPINRAZA, a foundation of care in SMA, is an antisense
oligonucleotide (ASO), developed using Ionis Pharmaceuticals’
proprietary technology that is designed to target a root cause of
SMA by increasing the amount of full-length survival motor neuron
(SMN) protein, which is critical to maintaining motor neurons. It
is administered by intrathecal injection into the fluid surrounding
the spinal cord where motor neurons reside to deliver the treatment
where the disease starts.
SPINRAZA currently maintains a robust clinical data set in SMA
based on data from approximately 300 patients across a broad range
of SMA populations demonstrating a favorable benefit:risk profile.
SPINRAZA was evaluated in two randomized, double-blind,
sham-controlled studies of infantile and later-onset SMA (ENDEAR
and CHERISH, respectively) and supported by open-label studies that
include pre-symptomatic infants (NURTURE), individuals with
later-onset SMA (CS2/CS12) and an extension study of individuals
who previously participated in the clinical development program
(SHINE). The most common adverse events observed were respiratory
infection, fever, constipation, headache, vomiting and back pain.
Hypersensitivity, meningitis and hydrocephalus have been observed
in the post-marketing setting. Renal toxicity and coagulation
abnormalities, including acute severe low platelet counts, have
been observed after administration of some ASOs. Laboratory tests
can monitor for these signs.
Biogen licensed the global rights to develop, manufacture and
commercialize SPINRAZA from Ionis Pharmaceuticals, Inc. (Nasdaq:
IONS), a leader in antisense therapeutics. Biogen and Ionis
conducted an innovative clinical development program that moved
SPINRAZA from its first dose in humans in 2011 to its first
regulatory approval in five years.
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. To learn more, please visit
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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; clinical development
programs, clinical trials, and data readouts and presentations; and
the potential benefits and results from early treatment of SMA.
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 the occurrence of
adverse safety events; risks of unexpected costs, delays or 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; failure to obtain regulatory
approvals in other jurisdictions; 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:
- Matsumoto H, Clayton-Krasinski DA, Klinge SA, et al.
Development and initial validation of the assessment of caregiver
experience with neuromuscular disease. J Pediatr Orthop.
2011;31(3):284-292.
- Based on Commercial Patients, Early Access Patients, and
Clinical Trial Participants as of March 31, 2020.
- Finkel R, Chiriboga C, Vajsar J, et al. Treatment of
infantile-onset spinal muscular atrophy with nusinersen: a phase 2,
open-label, dose-escalation study. Lancet.
2016;388(10063):3017-3026.
- Darras B, Markowitz J, Monani U, De Vivo D. Chapter 8 - Spinal
Muscular Atrophies. In: Vivo BTD, ed. Neuromuscular Disorders of
Infancy, Childhood, and Adolescence (Second Edition). San Diego:
Academic Press; 2015:117-145.
MEDIA CONTACT:David Caouette+ 1
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INVESTOR CONTACT:Joe Mara+1 781
464 2442IR@biogen.com |
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