Biogen Advances Spinal Muscular Atrophy (SMA) Clinical Research
with New Study Evaluating a Higher Dose of SPINRAZA® (nusinersen)
and Additional Data in a Broad Range of Patients
Biogen Inc. (Nasdaq: BIIB) today announced updates to the SPINRAZA
(nusinersen) clinical development program including the initiation
of a new global clinical trial, DEVOTE. The DEVOTE study will
evaluate if a higher dose of SPINRAZA can provide even greater
efficacy in the treatment of spinal muscular atrophy (SMA) across a
broad patient population. In addition, new data further
demonstrating the safety and efficacy of treatment with SPINRAZA in
individuals with later-onset SMA will be featured in a podium
presentation at the 13th Congress of the European Paediatric
Neurology Society (EPNS) in Athens (September 17-21).
A foundation of care in SMA, SPINRAZA is the only treatment
approved for infants, children and adults with evidence across a
broad range of patients and real-world experience treating more
than 8,400 patients of all ages in over 40 countries.1 These
clinical program updates will add to the largest and longest
clinical data set available to date in SMA.
New study, DEVOTE, to evaluate if SPINRAZA can offer
even greater efficacy in treating SMA Building on
the demonstrated long-term safety profile and proven efficacy of
SPINRAZA in a broad range of patients, the DEVOTE trial will
examine the potential for even greater efficacy, as well as the
safety and tolerability of SPINRAZA, when administered at a higher
dose. The trial is a Phase 2/3 randomized, controlled
dose-escalating study that will be conducted at 50 sites around the
world with a projected enrollment of 126 individuals with SMA of
all ages, including adults.
“SPINRAZA has fundamentally changed the natural history of SMA,”
said Alfred Sandrock, Jr., M.D., Ph.D., executive vice president
and chief medical officer at Biogen. “Antisense oligonucleotides
directly intervene at the origin of disease providing a
transformative therapeutic option. SPINRAZA’s highly targeted
approach and well-characterized safety profile allows us to
continue exploring ways to potentially address the remaining
medical needs in the SMA community.”
The three-part trial will include an open-label safety
evaluation and a pivotal, double-blind, active control randomized
treatment period followed by an open-label treatment period. After
the safety evaluation, the trial will compare two loading doses of
50 milligrams (mg) 15 days apart followed by a maintenance
dose of 28 mg every four months with the current U.S.
Food and Drug Administration-approved administration of SPINRAZA,
which is four loading doses with 12 mg maintenance doses every four
months. The third part of the trial will be an open-label
evaluation to determine how to safely and efficiently transition
patients from the currently approved dose of SPINRAZA to the higher
dose being tested in the study.
More information on the trial (NCT04089566) is available at
clinicaltrials.gov.
Data to be presented at EPNS demonstrate improvements or
stabilization in motor function following longer-term
treatmentAn integrated analysis from SHINE (NCT02594124),
an open-label extension study for patients with SMA who
participated in prior SPINRAZA studies, found that children with
later-onset SMA (Type 2 or Type 3) experienced improvements or
stabilization in one or more measures of motor function for up to
nearly six years, in contrast to the expected decline observed in
natural history cohorts. SHINE is following 24 patients aged 2-15
at treatment initiation (SMA Type 2; n=10 and Type 3; n=14) who
transitioned from the CS2/CS12 studies, which previously showed
that individuals with later-onset SMA who were treated with
SPINRAZA demonstrated improvements in motor function and disease
stabilization over approximately three years, that were not
observed in natural history cohorts.2 Patients in the study
were between 7 and 21 years old at the last study visit.
Motor function measures in this analysis of the SHINE study
included the Hammersmith Functional Motor Scale–Expanded (HFMSE),
Upper Limb Module (ULM), and Six-Minute Walk Test (6MWT). No
participants discontinued treatment due to adverse events, and no
new safety concerns were identified during the nearly six-year
follow-up period.
“These findings are important in understanding the need for
long-term treatment in individuals with SMA,” said Basil Darras,
M.D., lead study author, director of the Neuromuscular Center and
Spinal Muscular Atrophy Program at Boston Children’s Hospital, and
professor of neurology at Harvard Medical School. “These data
reinforce the long-term safety and durability of SPINRAZA to
improve or stabilize motor function in individuals with later-onset
SMA.”
About SPINRAZA® (nusinersen)3-4 SPINRAZA is the
first therapy approved to treat infants, children and adults with
spinal muscular atrophy (SMA) and is available in more than 40
countries. As of June 30, 2019, more than 8,400 individuals have
been treated with SPINRAZA for up to nearly six years, based on
patients across the post-marketing setting, Expanded Access Program
(EAP) and clinical trial participants. SPINRAZA is the only SMA
treatment to combine unsurpassed real-world experience and the
highest 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, resulting 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) 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 the largest clinical data set in
SMA based on data from over 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 (ENDEAR and CHERISH) in infantile and
later-onset SMA patients and supported by open-label studies in
pre-symptomatic infants (NURTURE) and individuals who were treated
into adulthood with later-onset SMA (CS2/CS12). The most common
adverse events observed were respiratory infection, fever,
constipation, headache, vomiting and back pain. 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, and today 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, neuromuscular disorders, movement disorders,
Alzheimer’s disease and dementia, 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; our research and
development program for the treatment of SMA; the clinical
development program for SPINRAZA, including the enrollment of the
DEVOTE study; the potential benefits and results from early
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; and third party collaboration risks. 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
1As of June 30, 2019, more than 8,400 patients on therapy across
the post-marketing setting, the expanded access program and
clinical trials.2Darras BT, Chiriboga CA, Iannaccone ST, et al.
Nusinersen in later-onset spinal muscular atrophy: Long-term
results from the phase 1/2 studies. Neurology. 2019 May
21;92(21):e2492-e2506. 3Finkel 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):3017-3026.4Darras 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.
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