Biogen Inc. (Nasdaq: BIIB) will present new data from
clinical studies aimed at assessing remaining unmet needs for
people living with spinal muscular atrophy (SMA) and evaluating the
potential impact of SPINRAZA® (nusinersen) in different patient
populations at the SMA Research & Clinical Care Meeting hosted
by Cure SMA this week in Anaheim, Calif. Biogen is a presenting
sponsor of Cure SMA’s 2022 Annual SMA Conference, the world’s
largest meeting dedicated to SMA research and care.
“The data we are presenting at this year’s Cure SMA conference –
including the latest updates from the RESPOND and DEVOTE studies –
reinforce Biogen’s commitment to evaluating the potential of
SPINRAZA to further improve clinical outcomes for individuals with
SMA,” said Maha Radhakrishnan, M.D., Chief Medical Officer at
Biogen. “There are key unmet needs within the SMA community and we
are committed to addressing these through our ongoing research that
includes active enrollment in three global clinical studies.”
SMA Research Updates Growing enrollment in the
RESPOND study indicate there are residual unmet clinical needs in
infants and toddlers with SMA following treatment with Zolgensma®
(onasemnogene abeparvovec). The Phase 4 study is evaluating the
clinical benefit and safety of SPINRAZA in infants and toddlers
with SMA who have unmet needs following treatment with the gene
therapy.
Since initial findings from nine patients were shared in March
2022, baseline and safety data from 16 patients enrolled in RESPOND
(as of November 2021) are being presented. All enrolled study
participants reported suboptimal clinical status across a variety
of measures at baseline, with 13 of 16 showing this in multiple
areas, including motor and respiratory functions and
swallowing/feeding ability. After beginning SPINRAZA treatment,
initial safety findings (median duration of 132.5 days) show three
participants experienced a serious adverse event (AE) during the
study period; none of these events were considered related to
SPINRAZA treatment. The RESPOND study (NCT04488133) is currently
enrolling participants at 20 sites worldwide; more information
about the eligibility criteria is available at clinicaltrials.gov.
Biogen will also share final data from Part A of the ongoing,
three-part DEVOTE study evaluating the safety and tolerability of
investigational, higher doses of nusinersen.* Results from Part A,
an open-label safety evaluation period in children and teens with
later-onset SMA, suggest that a higher dosing regimen (28 mg) of
nusinersen leads to higher levels of the drug in the cerebrospinal
fluid and is generally well-tolerated, with most AEs reported
considered to be mild in severity. The most common AEs reported
were headache and procedural pain. Two serious AEs (fall, femur
fracture) were reported in one participant during the study period.
No AEs were considered related to nusinersen and some were related
to treatment administration. The totality of Part A data supports
further development of a higher dose of nusinersen.
Currently, Part B and Part C of DEVOTE evaluating an
investigational, higher dose of nusinersen are enrolling at 52
sites worldwide. Information on the DEVOTE trial (NCT04089566) is
available at clinicaltrials.gov.
Featured SPINRAZA Data Presentations
Include:
- Results From the End of Part A of
the Ongoing 3-Part DEVOTE Study to Explore Higher Doses of
Nusinersen in SMA – Friday, June 17, 2022 at 9:40 a.m. PT
- Baseline Characteristics and
Initial Safety Results in RESPOND: A Phase 4 Study of Nusinersen in
Children With SMA Who Received Onasemnogene Abeparvovec
– Friday, June 17, 2022 at 10 a.m. PT
*Nusinersen is currently commercialized under the brand name
SPINRAZA® and the U.S. Food and Drug Administration-approved dose
is 12 mg. As a foundation of care in SMA, more than 13,000
individuals have been treated with SPINRAZA worldwide.1
About
SPINRAZA® (nusinersen)The SPINRAZA
clinical development program encompasses 10 clinical studies, which
have included more than 300 individuals across a broad spectrum of
patient populations,2 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 Spinal Muscular Atrophy (SMA)SMA 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.3 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.3 Some individuals with SMA may never sit; some sit
but never walk; and some walk but may lose that ability over
time.4 In the absence of treatment, children with the most
severe form of SMA would not be expected to reach their second
birthday.3
SMA impacts approximately 1 in 10,000 live births,5-8 is a
leading cause of genetic death among infants9 and causes a range of
disability in teenagers and adults.4
About BiogenAs pioneers in neuroscience, Biogen
discovers, develops, and delivers worldwide innovative therapies
for people living with serious neurological 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, Sir Kenneth Murray, and Nobel Prize
winners Walter Gilbert and Phillip Sharp. Today, Biogen has a
leading portfolio of medicines to treat multiple sclerosis, has
introduced the first approved treatment for spinal muscular
atrophy, and developed the first and only approved treatment to
address a defining pathology of Alzheimer’s disease. Biogen is also
commercializing biosimilars and focusing on advancing one of the
industry’s most diversified pipelines in neuroscience that will
transform the standard of care for patients in several areas of
high unmet need.
In 2020, Biogen launched a bold 20-year, $250 million initiative
to address the deeply interrelated issues of climate, health, and
equity. Healthy Climate, Healthy Lives™ aims to eliminate fossil
fuels across the company’s operations, build collaborations with
renowned institutions to advance the science to improve human
health outcomes, and support underserved communities.
We routinely post information that may be important to investors
on our website at www.biogen.com. Follow us on social
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Biogen Safe HarborThis 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 nusinersen; the results of certain real-world data; our
research and development program for the identification and
treatment of SMA; clinical development programs, clinical trials
and data readouts and presentations; the potential benefits and
results from 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, including from the
DEVOTE, RESPOND and ASCEND studies; 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:
- Based on commercial patients, early access patients, and
clinical trial participants through March 31, 2022.
- Core Data Sheet, Version 13, October 2021. 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 2022.
- 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.
- Arkblad E, et al. A population-based study of genotypic and
phenotypic variability in children with spinal muscular atrophy.
Acta Paediatr. 2009 May;98(5):865-72. doi:
10.1111/j.1651-2227.2008.01201.x. Epub 2009 Jan 20.
- Jedrzejowska M, et al. Incidence of spinal muscular atrophy in
Poland--more frequent than predicted? Neuroepidemiology.
2010;34(3):152-7. doi: 10.1159/000275492. Epub 2010 Jan 15.
- Prior TW, et al. Newborn and carrier screening for spinal
muscular atrophy. Am J Med Genet A. 2010 Jul;152A(7):1608-16. doi:
10.1002/ajmg.a.33474.
- Sugarman EA, et al. Pan-ethnic carrier screening and prenatal
diagnosis for spinal muscular atrophy: clinical laboratory analysis
of >72,400 specimens. Eur J Hum Genet. 2012 Jan;20(1):27-32.
doi: 10.1038/ejhg.2011.134. Epub 2011 Aug 3.
- Cure SMA. About SMA. Available
at https://www.curesma.org/about-sma/. Accessed: June
2022.
MEDIA CONTACT:Ashleigh Koss+ 1 908 205
2572public.affairs@biogen.com |
INVESTOR CONTACT:Mike Hencke+1 781 464 2442IR@biogen.com |
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