Biogen Inc. (Nasdaq: BIIB) today announced new research supporting
the continued development of an investigational higher dose of
SPINRAZA® (nusinersen) and additional data reinforcing the strength
of SPINRAZA’s clinical profile in improving the lives of
individuals with spinal muscular atrophy (SMA) over the long term.
These data are being presented at the virtual Cure SMA Research
& Clinical Care Meeting taking place June 9-11, 2021.
“Intervention with SPINRAZA can meaningfully impact the
trajectory of SMA, and we remain relentless in our aim of improving
outcomes for people with SMA. We continue to better understand and
explore SPINRAZA’s potential with our new and ongoing global
clinical studies,” said Alfred Sandrock, Jr., M.D., Ph.D., Head of
Research and Development at Biogen. “The data we are presenting at
Cure SMA 2021 demonstrate the long-term benefits with SPINRAZA as
individuals age. Additionally, a new analysis provides further
support for the potential for a higher dose of SPINRAZA to offer
even greater improvements in motor function for SMA patients.”
New Findings Support Continued Development of an
Investigational Higher Dose of SPINRAZAAn analysis of data
from the Phase 2 CS3A and Phase 3 ENDEAR studies in children with
infantile-onset SMA used pharmacokinetic (PK)/pharmacodynamic (PD)
modelling to predict the potential efficacy of an investigational
higher dose regimen of SPINRAZA. This analysis suggests that a
higher dose of SPINRAZA may lead to a clinically meaningful
increase in the CHOP INTEND (Children’s Hospital of Philadelphia
Infant Test of Neuromuscular Disorders) score beyond that already
observed with the 12 mg approved dose. These findings further
reinforce the scientific rationale for the evaluation of a higher
dose of SPINRAZA in the ongoing DEVOTE study.
DEVOTE is a Phase 2/3 study evaluating the safety, tolerability
and potential for even greater efficacy of SPINRAZA when
administered at a higher dose than currently approved. It is a
three-part study that includes an open-label safety evaluation
cohort (Part A), a pivotal, double-blind, randomized,
active-controlled treatment cohort (Part B) and an open-label
cohort of patients transitioning from the approved 12 mg dosing
regimen of SPINRAZA to the higher dose regimen (Part C).
As previously reported,1 safety data from Part A (n=6; 28 mg)
support continued development of a higher dose of SPINRAZA and an
updated analysis presented at the meeting include data collected up
to 10 months. Enrollment in the pivotal Part B of DEVOTE is ongoing
and will evaluate a higher-dose regimen (two loading doses of 50 mg
14 days apart followed by 28 mg maintenance doses every four
months) compared to the currently approved dosing regimen for
SPINRAZA.2 More information about DEVOTE is available at
ClinicalTrials.gov (NCT04089566). Data Reinforce Long-term
Efficacy of SPINRAZA in a Broad Range of People with SMAAn
analysis of data from the NURTURE study (n=25) shows 92 percent of
patients who initiated SPINRAZA treatment as pre-symptomatic
infants maintained the ability to swallow after a median
of 3.8 years. This is in contrast with the natural history of SMA
where impaired swallowing is expected for people with 2 or
3 SMN2 copies3 and can lead to an increased risk of
aspiration pneumonia, choking and failure to thrive. In this
analysis, NURTURE study participants were consistently rated by
their caregiver as, on average, never to rarely experiencing
difficulty for the majority of measures related to general feeding,
drinking liquids and eating solid foods. Additionally, all
participants with 3 SMN2 copies and 73 percent (11 of 15)
of participants with 2 SMN2 copies were reported by their
caregiver as being fed exclusively by mouth.
In addition, post-hoc data from the open-label CS2-CS12 and
SHINE extension studies indicate children and teens with
later-onset SMA (n=14) showed improvement in walking distance over
five years of SPINRAZA treatment and stabilization in fatigue.
In all analyses presented at the meeting, the safety profile of
SPINRAZA was consistent with previously reported findings.
Featured SPINRAZA Data Presentations
Include:
- Scientific Rationale for a Higher Dose of Nusinersen
- Part A Results From the Ongoing DEVOTE Study to Explore a
Higher Dose of Nusinersen in SMA
- Preserved Swallowing Function in Infants Who Initiated
Nusinersen Treatment in the Presymptomatic Stage of SMA: Results
From the NURTURE Study
- Nusinersen in Later-onset Spinal Muscular Atrophy: Walking
Distance and Fatigue in CS2/12 and SHINE Participants
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.4
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.5 It is administered directly into the central nervous
system, where motor neurons reside, to deliver treatment where the
disease starts.5
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.6 The SPINRAZA clinical development
program encompasses 10 clinical studies, which have included more
than 300 individuals across a broad spectrum of patient
populations,6 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.7 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.7 Some individuals with SMA may never
sit; some sit but never walk; and some walk but may lose that
ability over time.8 In the absence of treatment, children with the
most severe form of SMA would not be expected to reach their second
birthday.7
SMA impacts approximately one in 11,000 live births in the
U.S.,9 is a leading cause of genetic death among infants7 and
causes a range of disability in teenagers and adults.8
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 –
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
and clinical studies of SPINRAZA; 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 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 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 clinical
studies; 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:
- Day JW et al. (2021, April).
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. Poster presented at the American
Academy of Neurology (AAN) 2021 Virtual Annual Meeting.
- 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: June 2021
- McGrattan KE, Graham RJ, DiDonato
CJ, Darras BT. Dysphagia Phenotypes in Spinal Muscular Atrophy: The
Past, Present, and Promise for the Future. Am J Speech Lang Pathol.
2021 Apr 6:1-15. doi: 10.1044/2021_AJSLP-20-00217. Epub ahead of
print.
- Based on commercial patients, early
access patients, and clinical trial participants through March 31,
2021.
- 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: June 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: June 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: June 2021
MEDIA CONTACT:Allison Parks+1 781 464
3260public.affairs@biogen.com |
INVESTOR CONTACT:Mike Hencke +1 781 464
2442 IR@biogen.com |
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