Biogen Plans to Initiate Phase 4 Study Evaluating Benefit of
SPINRAZA® (nusinersen) in Patients Treated with Zolgensma®
(onasemnogene abeparvovec)
Biogen Inc. (Nasdaq: BIIB) today announced it plans to initiate a
global Phase 4 clinical study, RESPOND, to examine the clinical
benefit and assess the safety of SPINRAZA® (nusinersen) in infants
and children with spinal muscular atrophy (SMA) who still have
unmet clinical needs following treatment with gene therapy
Zolgensma® (onasemnogene abeparvovec).
“As clinicians, we continue to pursue improved outcomes for
infants and children with SMA, and the need for additional benefit
in some patients treated with gene therapy has been observed. There
is compelling clinical rationale for the potential for additional
efficacy with SPINRAZA in these patients,” said Crystal Proud,
M.D., Pediatric Neuromuscular Neurologist, Children’s Hospital of
The King’s Daughters, Virginia and a member of the RESPOND study
steering committee. “We expect that the RESPOND study will generate
valuable data to help inform future treatment decisions for our
youngest SMA patients.”
People with SMA do not produce enough survival motor neuron
(SMN) protein, which is critical for the maintenance of motor
neurons that support sitting, walking and basic functions of life,
including breathing and swallowing. The RESPOND study will seek to
understand if the proven efficacy of SPINRAZA and its continuous
production of SMN protein may also benefit patients previously
treated with gene therapy.
“Available data now show that some patients in the long-term
study of Zolgensma have moved on to treatment with SPINRAZA. We
believe that, for certain patients, motor neurons may be
insufficiently treated by this gene therapy, and we plan to
initiate this study to understand the extent to which SPINRAZA may
potentially improve outcomes,” said Maha Radhakrishnan, M.D., Chief
Medical Officer at Biogen. “The impact of SPINRAZA since its launch
has been unprecedented compared to the natural history of the
disease, with 100 percent of pre-symptomatic infants in the NURTURE
study alive after nearly five years of treatment. More than 10,000
SMA patients have now been treated globally.”
In the long-term study of Zolgensma, it has been reported that,
to date, 4 out of 10 patients have been subsequently treated with
SPINRAZA.1 Based on the planned study design, RESPOND will be a
two-year, open-label study to evaluate the efficacy and safety of
SPINRAZA in SMA patients previously treated with Zolgensma to
further inform treatment decisions. Efficacy will be assessed by
change from baseline on motor function measures, additional
clinical outcomes (e.g., swallowing) and caregiver burden.
Neurofilament levels, an exploratory endpoint, will also be
evaluated as a marker of biological disease activity. The primary
study group aims to include 40 infants aged 9 months or younger (at
the time of first SPINRAZA dose) who have 2 copies of SMN2 (likely
to develop SMA Type 1) and received Zolgensma at 6 months old
or younger. A second study group will include 20 children and will
generate data in patients with a broader age range (up to 3 years
old at the time of first SPINRAZA dose). After a screening period,
participants will receive the approved 12 mg dose of SPINRAZA,
which is four loading doses followed by maintenance doses every
four months, over the two-year study period.4
The company plans to submit the study protocol to the regulatory
authorities in the coming months and aims for the first eligible
patients to be enrolled in the RESPOND study in Q1 2021. The study
is projected to enroll 60 children up to 3 years old who are
determined by the investigator to have the potential for additional
clinical improvement after receiving Zolgensma. Physicians will
evaluate children for participation using criteria that may include
one or more of the following: suboptimal motor function (e.g., a
score lower than 50 on the Children’s Hospital of Philadelphia
Infant Test of Neuromuscular Disorders [CHOP INTEND]); the need for
respiratory support; abnormal swallowing or feeding ability; or
other factors deemed relevant by the investigator.
About SPINRAZA® (nusinersen)5--7 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 significant 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. It 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 an extensive 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), the 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; our research and
development program for the identification and treatment of SMA;
the clinical development program for SPINRAZA, including the study
protocol and enrollment of the RESPOND study and the timing
thereof; 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; 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:
- Zolgensma EU Summary of Product Characteristics (SmPC).
Available at:
https://www.ema.europa.eu/en/documents/product-information/zolgensma-epar-product-information_en.pdf.
Accessed on July 13, 2020.
- Finkel R, et al. Presented at the Muscular Dystrophy
Association’s (MDA) 2020 Clinical & Scientific Conference.
- Harada Y, et al. Presented at the Muscular Dystrophy
Association’s (MDA) 2020 Clinical & Scientific Conference.
- SPINRAZA U.S. Prescribing Information. Available at:
https://www.spinraza.com/content/dam/commercial/specialty/spinraza/caregiver/en_us/pdf/spinraza-prescribing-information.pdf.
Accessed on July 13, 2020.
- 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+ 1 617 679
4945public.affairs@biogen.com |
INVESTOR CONTACT:Joe Mara+1 781 464 2442IR@biogen.com |
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