Biogen Announces First Patient Treated With Higher Dose of
SPINRAZA® (nusinersen) in Phase 2/3 DEVOTE Study
Biogen (Nasdaq: BIIB) today announced that the first patient has
been treated in the global clinical study, DEVOTE. The study is
designed to evaluate the safety, tolerability and potential for
even greater efficacy of SPINRAZA (nusinersen) when administered at
a higher dose than currently approved for the treatment of spinal
muscular atrophy (SMA). The Phase 2/3 randomized, controlled,
dose-escalating study will be conducted at approximately 50 sites
around the world and aims to enroll individuals of all ages with
SMA.
“Today, people of all ages with SMA are achieving better
outcomes related to survival, mobility and independence, yet there
is still no cure and unmet needs remain for many,” said Kenneth
Hobby, President of Cure SMA, a patient advocacy organization
dedicated to the treatment and cure of SMA. “Our community is
energized by the advances we’ve made and believes that ongoing
research like the DEVOTE study is instrumental to help deepen our
understanding and potentially further transform and improve what it
means to live with SMA.”
The three-part study will include an open-label safety
evaluation cohort and a pivotal, double-blind, active control
randomized treatment cohort followed by an open-label treatment
cohort. After the safety evaluation cohort completes, the pivotal
cohort will begin and include a treatment group receiving two
loading doses of 50 milligrams (mg) 14 days apart, followed by a
maintenance dose of 28 mg every four months. A second treatment
group will receive 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 cohort will be an open-label evaluation to assess the safety
and tolerability of transitioning patients from the currently
approved dose of SPINRAZA to the higher dose being tested in the
study.
“As the first treatment for SMA, SPINRAZA fundamentally shifted
the disease course compared to natural history, allowing many
individuals to reach milestones that may have been previously
unattainable,” said Alfred Sandrock Jr., M.D., Ph.D., Executive
Vice President, Research and Development at Biogen. “With the
success we’ve seen to date with the 12 mg dose, we are exploring a
higher dose of SPINRAZA for the potential to deliver even better
clinical outcomes.”
More information on the study (NCT04089566) is available at
clinicaltrials.gov.
About SPINRAZA® (nusinersen)1-3 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 December 31, 2019, 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
www.biogen.com and follow us on social media – Twitter,
LinkedIn, Facebook, YouTube.
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 DEVOTE 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 DEVOTE study or it will take longer than expected;
uncertainty of success in the development and potential
commercialization of higher dose SPINRAZA; unexpected concerns that
may arise from additional data, analysis or results obtained during
the DEVOTE study; regulatory authorities may require additional
information or further studies, or may fail or refuse to approve or
may delay approval of higher dose SPINRAZA; 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 impact related to the effect of
COVID-19 or other public health epidemics on our sales and
operations, including employees. 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:
- As of December 31, 2019, more than 10,000 patients have been
treated with SPINRAZA, including commercial patients, early access
patients and clinical trial participants.
- 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
617 679 4945public.affairs@biogen.com |
INVESTOR CONTACT:Joe Mara+1 781
464 2442IR@biogen.com |
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