Biogen (Nasdaq: BIIB) today announced new results from the NURTURE
study, adding data to the longest study of spinal muscular atrophy
(SMA) in pre-symptomatic infants (n=25). These data reported, after
up to 45.1 months of analysis, continue to demonstrate efficacy and
safety in patients treated pre-symptomatically with SPINRAZA in
comparison to the natural history of this disease. These new data
also showed that patients treated with SPINRAZA had continuous
improvement with the overwhelming majority of patients achieving
motor milestones in a normal timeframe.
These data are being presented at the Cure SMA Annual SMA
Conference in Anaheim, CA (June 28-July 1, 2019) and the 5th
Congress of the European Academy of Neurology (EAN) in Oslo, Norway
(June 29-July 2, 2019).
View the multimedia release here:
https://www.multivu.com/players/English/8560251-biogen-nurture-study-spinraza-spinal-muscular-atrophy-treatment-data/
“These study results demonstrate the durable impact of
pre-symptomatic, proactive treatment on transforming the natural
course of this disease. We are seeing an extensive number of
patients continually meeting child motor development milestones and
making unprecedented gains in a previously hopeless and often fatal
condition,” said Darryl De Vivo, M.D., Sidney Carter Professor of
Neurology and Pediatrics, Columbia University Irving Medical Center
in New York, New York. “SPINRAZA is setting patients on a path
toward survival, greater mobility and independence, which is
helping improve outcomes for patients of all ages.”
Results from NURTURE, an ongoing, Phase 2, open-label study of
25 pre-symptomatic patients with SMA (most likely to develop SMA
Type 1 or 2) who received their first dose of SPINRAZA before six
weeks old, demonstrated unparalleled results in comparison to the
natural history of SMA. As of March 2019:
- 100 percent were alive without a need for permanent
ventilation.
- The median age of the study participants was nearly three years
old. The majority of untreated patients with SMA Type 1 never reach
their second birthday without permanent ventilation.
- 100 percent of the infants were sitting independently, in
comparison to the natural history of this disease where no patients
with SMA Type 1 would be able to do so and patients with SMA Type 2
would need assistance.
- 88 percent of the infants were walking independently with many
of them doing so in the normal timeframe for a toddler. In the
natural history of SMA, patients with SMA Type 1 or Type 2 are
never able to walk independently.
- Patients were approaching the maximum mean score of 64 on the
CHOP INTEND measure of motor function– 63.4 for patients with 3 SMN
2 copies (n=10) and 62.1 for those with 2 SMN 2 copies (n=15),
demonstrating the powerful impact of early SPINRAZA treatment.
- SPINRAZA demonstrated longer term efficacy up to nearly 4
years, with participants continuing to make progress and showing no
signs of loss of motor function.
- SPINRAZA was well-tolerated with no new safety concerns
identified after up to nearly 4 years of treatment.
“A few years ago, SMA patients had no treatment options and
faced significant care challenges,” said Kenneth Hobby, President
of Cure SMA, a patient advocacy organization dedicated to the
treatment and cure of SMA. “However, the future of SMA has changed
and especially with early treatment patients now have a very strong
chance to reach age appropriate developmental
milestones. These new data demonstrate the dramatic impact
where children are now walking independently at four years of age,
when the usual lifespan would be under two if untreated. This
study provides additional evidence on the maintenance of these
improvements. It’s critical that research in SMA continues to
support the generation of real-world evidence in patients of all
ages so that we better understand the long-term implications of SMA
and treatment across all types.”
Additional presentations at the two meetings highlight results
from the ongoing open-label SHINE extension study of children with
infantile and later-onset SMA as well the CS2/CS12 analysis of
older patients. Biogen also continues to explore the scientific
value of phosphorylated neurofilament heavy chain (pNF-H) and will
present new data on the ongoing evaluation of its potential as a
biomarker in SMA.
About SPINRAZA® (nusinersen)1-4 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 March 31, 2019, more than 7,500 individuals have
been treated with SPINRAZA for up to 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. Serious infections
of hydrocephalus and meningitis 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 and is
focused on advancing neuroscience research programs in MS and
neuroimmunology, Alzheimer’s disease and dementia, movement
disorders, neuromuscular disorders, acute neurology, neurocognitive
disorders, pain and ophthalmology. Biogen also commercializes
biosimilars of advanced biologics.
We routinely post information that may be important to investors
on our website at www.biogen.com. To learn more, please
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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;
and the identification and 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. Drug development and
commercialization involve a high degree of risk, and only a small
number of research and development programs result in
commercialization of a product. Results in early stage clinical
trials may not be indicative of full results or results from later
stage or larger scale clinical trials and do not ensure regulatory
approval. 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 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; 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
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.
Reference:
- Finkel, Richard S., et al. “Nusinersen versus Sham Control in
Infantile-Onset Spinal Muscular Atrophy.” New England Journal of
Medicine, vol. 377, no. 18, 2017, pp. 1723–1732.,
doi:10.1056/nejmoa1702752.
- Lefebvre S, Burglen L, Reboullet S, et al. Identification and
characterization of a spinal muscular atrophy-determining gene.
Cell.1995;80(1):155-165.
- Mercuri, Eugenio, et al. “Nusinersen versus Sham Control in
Later-Onset Spinal Muscular Atrophy.” New England Journal of
Medicine, vol. 378, no. 7, 2018, pp. 625–635.,
doi:10.1056/nejmoa1710504.
- Basil T. Darras, et al. “Nusinersen in later-onset
spinal muscular atrophy.” Neurology, May
2019, 92 (21) e2492-e2506; DOI:10.1212/WNL.0000000000007527
MEDIA CONTACT: David Caouette+ 617 679
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