Biogen Inc. (Nasdaq: BIIB) today announced new data from the
SPINRAZA® (nusinersen) clinical development program aimed at
optimizing outcomes for people with spinal muscular atrophy (SMA)
and advancing understanding of the disease. These data are being
presented at the American Academy of Neurology (AAN) 2021 Virtual
Annual Meeting, April 17-22.
“Biogen is humbled that we were pioneers in developing the first
treatment for SMA, along with our collaboration partners Ionis
Pharmaceuticals. We continue to be driven by the pursuit of better
outcomes for patients, including exploring the potential of a
higher dose of SPINRAZA in the DEVOTE study, in addition to
initiating the RESPOND study,” said Alfred Sandrock, Jr.,
M.D., Ph.D., Head of Research and Development at Biogen. “We are
also evaluating the use of biomarkers and digital tools to
supplement traditional clinical assessments for SMA and enhance
disease monitoring. Through these collective research efforts, we
aim to provide valuable data that may help guide future treatment
approaches and decisions for people with SMA.”
Exploring Opportunities to Optimize Treatment in
SMA Building on the proven efficacy
and well-established safety of SPINRAZA in a broad range
of patients with SMA, the Phase 2/3 DEVOTE study is evaluating the
safety, tolerability and potential for even greater efficacy of
SPINRAZA when administered at a higher dose than currently
approved. The three-part study includes an open-label safety
evaluation cohort (Part A), a pivotal, double-blind, active control
randomized treatment cohort (Part B) and an open-label cohort (Part
C) transitioning from the approved 12-milligram (mg) dose of
SPINRAZA to the higher dose.
An analysis of the higher loading and maintenance dosing regimen
in Part A (n=6; 28 mg) showed no new safety concerns in study
participants who were followed for up to approximately five months
(64-158 days). There were no adverse events (AEs) reported that
were considered related to the higher dose study drug and there
were no severe or serious AEs. Four patients reported mild or
moderate AEs, including AEs considered related to the treatment
administration procedure. This emerging safety profile supports
Biogen’s continued development of a higher dose of SPINRAZA,
including ongoing enrollment of patients in the pivotal Part B of
the DEVOTE study. This part will evaluate the higher-dose regimen
(2 loading doses of 50 mg two weeks apart followed by 28 mg
maintenance doses every four months) compared to the approved 12 mg
dose of SPINRAZA: four loading doses, followed by maintenance doses
every four months.1 More information about DEVOTE is available at
ClinicalTrials.gov (NCT04089566).
Using Biomarkers and Digital Tools to Enhance Disease
MonitoringBiogen is advancing research to evaluate
biomarkers and digital tools that expand on traditional clinical
assessments and incorporate more sensitive measures to help better
predict and monitor the course of SMA.
New data in patients (n=75) from the CHERISH/SHINE studies build
upon the body of evidence suggesting neurofilament levels – an
indicator of ongoing biological disease activity – warrant further
evaluation as a biomarker for treatment response in SMA. Data show
that higher neurofilament levels at baseline were, on average,
associated with greater improvements in motor function scores among
SPINRAZA-treated individuals with later-onset SMA over a median of
approximately four years. The use of biomarkers could improve the
understanding of disease mechanisms and interventions for SMA and
other neurological diseases. Therefore, measuring neurofilament
levels have been integrated as an exploratory endpoint in the
DEVOTE and RESPOND (NCT04488133) studies.
Additionally, in consultation with SMA experts, Biogen has
developed a conceptual clinical framework to evaluate the potential
value of Konectom™, a mobile application, to enable adults living
with SMA to quantitatively and remotely self-assess motor function
in their daily lives. Currently used only in research settings,
Konectom leverages smart sensing technologies like touchscreen and
accelerometry to capture tangible data in studying neurological
diseases. In SMA, monitoring fatigue and smartphone typing skills
may be useful to assess functional impact across a broad range of
patients with varying levels of disease severity. Biogen is also
studying Konectom’s potential utility in multiple sclerosis and
other neurological diseases, with the goal of providing a more
accurate and complete picture of how neurological diseases impact a
person’s daily life.
SMA Data Presentations Featured at AAN:
- Escalating Dose and Randomized, Controlled Study of Nusinersen
in Participants With Spinal Muscular Atrophy (SMA); Study Design
and Part A Data for the Phase 2/3 DEVOTE (232SM203) Study to
Explore High-Dose Nusinersen – P6.080
- Baseline Plasma Phosphorylated Neurofilament Heavy Chain
(pNF-H) Level Is Associated With Future Motor Function in
Nusinersen-treated Individuals With Later-onset Spinal Muscular
Atrophy (SMA) – S13.005 – Monday, April 19, 2:40 p.m. ET
- Konectom™ Smartphone-Based Digital Outcome Assessments for
Adults Living With Spinal Muscular Atrophy (SMA): A Conceptual
Framework – P8.010
About
SPINRAZA® (nusinersen)SPINRAZA
is approved to treat infants, children and adults with spinal
muscular atrophy (SMA) and is available in more than 50 countries.
As a foundation of care in SMA, more than 11,000 individuals have
been treated with SPINRAZA worldwide.2
SPINRAZA is an antisense oligonucleotide (ASO) that targets the
root cause of SMA by continuously increasing the amount of
full-length survival motor neuron (SMN) protein produced in the
body.1 It is administered directly into the central nervous
system, where motor neurons reside, to deliver treatment where the
disease starts.1
SPINRAZA has demonstrated sustained efficacy across ages and SMA
types with a well-established safety profile based on data in
patients treated up to 7 years, combined with unsurpassed
real-world experience.3 The SPINRAZA clinical development
program encompasses 10 clinical studies, which have included more
than 300 individuals across a broad spectrum of patient
populations,3 including two randomized controlled studies
(ENDEAR and CHERISH). The ongoing SHINE and NURTURE open-label
extension studies are evaluating the long-term impact of SPINRAZA.
The most common adverse events observed in clinical studies were
respiratory infection, fever, constipation, headache, vomiting and
back pain. Laboratory tests can monitor for renal toxicity and
coagulation abnormalities, including acute severe low platelet
counts, which have been observed after administration of some
ASOs.
Biogen licensed the global rights to develop, manufacture and
commercialize SPINRAZA from Ionis Pharmaceuticals, Inc. (Nasdaq:
IONS), the leader in antisense therapeutics. Please click here
for Important Safety Information and full
Prescribing Information for SPINRAZA in the U.S., or visit
your respective country’s product website.
About SMASMA is a rare, genetic, neuromuscular
disease that affects individuals of all ages. It is characterized
by a loss of motor neurons in the spinal cord and lower brain stem,
resulting in progressive muscle atrophy and weakness.4 SMA is
caused by a deficiency in the production of survival motor neuron
(SMN) protein due to a damaged or missing SMN1 gene, with a
spectrum of disease severity.4 Some individuals with SMA may never
sit; some sit but never walk; and some walk but may lose that
ability over time.5 In the absence of treatment, children with the
most severe form of SMA would not be expected to reach their second
birthday.4
SMA impacts approximately one in 11,000 live births in the
U.S.,6 is a leading cause of genetic death among infants6 and
causes a range of disability in teenagers and adults.5
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, neuropsychiatry,
immunology, acute neurology and neuropathic pain.
We routinely post information that may be important to investors
on our website at www.biogen.com. Follow us on social media –
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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;
results from the DEVOTE study; 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
and/or higher dose SPINRAZA; the enrollment of the DEVOTE study and
the RESPOND study; the potential of our commercial business,
including SPINRAZA; 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 that we may not
fully enroll the DEVOTE study and/or the RESPOND 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 and/or the
RESPOND 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; product liability claims; 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:
- 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: April 2021
- Based on commercial patients, early
access patients, and clinical trial participants through December
31, 2021.
- Core Data sheet, Version 9, January
2019. SPINRAZA. Biogen Inc, Cambridge, MA.
- National Institute of Neurological
Disorders and Stroke, NIH. Spinal Muscular Atrophy Fact Sheet.
Available at
https://www.ninds.nih.gov/Disorders/Patient-Caregiver-Education/Fact-Sheets/Spinal-Muscular-Atrophy-Fact-Sheet.
Accessed: April 2021.
- Wadman RI, Wijngaarde CA, Stam M,
et al. Muscle strength and motor function throughout life in a
cross-sectional cohort of 180 patients with spinal muscular atrophy
types 1c–4. Eur J Neurol. 2018;25(3):512-518.
- Cure SMA. About SMA. Available at
https://www.curesma.org/about-sma/. Accessed: April 2021
MEDIA CONTACT:David Caouette+ 1 617 679
4945public.affairs@biogen.com |
INVESTOR CONTACT:Mike Hencke +1 781 464
2442 IR@biogen.com |
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