Biogen Inc. (Nasdaq: BIIB) today announced that the journal
Neuromuscular Disorders has published data from NURTURE, the first
study investigating a treatment targeting the underlying cause of
spinal muscular atrophy (SMA) in infants treated
pre-symptomatically. Data from the NURTURE study demonstrated that
infants who initiated treatment with SPINRAZA prior to the onset of
clinical symptoms attained unparalleled results compared to the
natural history of the disease. As of March 2019 all participants
were alive, without the need for permanent ventilation and
experienced continuous improvements with the majority achieving
motor milestones in timelines consistent with normal development.
The results also demonstrated durability of effect with children
making progress throughout the study.
“This pioneering study has far surpassed what we thought was
possible and redefined our expectations of how early treatment
helps individuals with SMA achieve optimal outcomes,” said Darryl
De Vivo, M.D., Sidney Carter Professor of Neurology and Pediatrics,
Columbia University Irving Medical Center in New York. “The
extraordinary results achieved in this study underline the critical
importance of newborn screening and early treatment of the
clinically normal infants with SMA before the onset of any clinical
symptoms.”
NURTURE is an ongoing, Phase 2, open-label study of 25 infants
with the genetic diagnosis of SMA (most likely to develop SMA Type
1 or 2) who received their first dose of SPINRAZA in the
pre-symptomatic stage and before six weeks old. The study,
conducted at 15 sites in seven countries, has results up to 45.4
months. When compared with the natural history of the disease, the
results are dramatic in their impact on changing the course of
SMA.
As of March 2019 all infants in the study were 25 months or
older, past the typical age of symptom onset for SMA Type 1 and
Type 2, and were alive without the need for permanent ventilation.
In comparison to the natural history of SMA, many of these infants
would likely have passed away or require permanent ventilation on
average by 13.5 months. In both the children with two and three
copies of SMN2, treatment with SPINRAZA demonstrated rapid onset of
improvement and durability of effect with their mean Children’s
Hospital of Pennsylvania Infant Test of Neuromuscular Disorders
(CHOP-INTEND) score of motor function reaching the maximum mean
score of 64 for all participants with three copies of SMN2 (n=10)
and a mean of 62.1 for those with two copies of SMN2
(N=15).
Additional highlights include:
- The majority of study participants achieved motor milestones in
timeframes consistent with the World Health Organization (WHO)
standards, with 100 percent sitting independently and 88 percent
walking independently.
- Hammersmith Infant Neurologic Examination, Section 2 (HINE-2)
development of motor function scores increased for all participants
with the mean score for both those with two or three SMN2 copies
approaching the maximum score of 26 points at the last
assessment.
- SPINRAZA demonstrated longer term efficacy up to nearly four
years, with participants continuing to make progress.
- SPINRAZA was well-tolerated with no new safety concerns
identified after up to nearly four years of treatment.
These published results from the NURTURE study were previously
presented at the 2019 Cure SMA Annual SMA Conference and the 5th
Congress of the European Academy of Neurology.
About SPINRAZA® (nusinersen)1-3 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), developed using Ionis’ 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 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; 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 risks relating to the
occurrence of adverse safety events and/or unexpected concerns that
may arise from additional data or analysis; 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
1Darras 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.
2Finkel 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):3017-3026.3Darras BT, Markowitz JA, Monani UR, De
Vivo DC. Chapter 8 - Spinal Muscular Atrophies. In: Darras BT,
Jones Jr. HR, Ryan MM, De Vivo DC, ed. Neuromuscular Disorders of
Infancy, Childhood, and Adolescence (Second Edition). San Diego:
Academic Press; 2015:117-145.
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