Biogen Announces New Updates Across its SMA Research Program at
2022 MDA Conference
Biogen Inc. (Nasdaq: BIIB) today announced new data and
updates from its SPINRAZA® (nusinersen) and spinal muscular atrophy
(SMA) research program aimed at improving clinical outcomes for
people impacted by the disease, including the ASCEND, RESPOND and
NURTURE studies. These updates will be presented at the 2022
Muscular Dystrophy Association (MDA) Clinical & Scientific
Conference (March 13-16, 2022).
First Patient Treated in Phase 3b ASCEND Study
The ASCEND study is currently enrolling with the first patient
treated in Q1 2022. At the conference, Biogen will present the
design of the open-label, global Phase 3b study to evaluate the
potential benefit of investigational higher dose nusinersen* in
later-onset, non-ambulatory SMA patients previously treated with
Evrysdi® (risdiplam).
“As the SMA community gains more experience with available
therapies, we are gleaning new insights about how these treatments
can enhance the lives of individuals impacted by SMA. However,
unmet needs still remain for the community,” said Basil Darras,
M.D., professor of Neurology at Harvard Medical School and director
of the Neuromuscular Center and Spinal Muscular Atrophy Program at
Boston Children's Hospital. “The ASCEND study seeks to generate
data that have the potential to inform decisions regarding
treatment for our patients with later-onset SMA.”
The primary endpoint in ASCEND is the total change from baseline
in the Revised Upper Limb Module (RULM) score. The study will
integrate smartphone-based digital assessments as an exploratory
endpoint using Konectom™ NMD, a mobile application developed by
Biogen Digital Health that will allow teen and adult participants
to quantitatively and remotely self-assess motor function in their
daily lives. Biogen Digital Health, a global unit of the company
dedicated to pioneering personalized and digital medicine in
neuroscience, will present more details about using digital outcome
assessments within the core clinical development program to
evaluate daily activities impacted by neuromuscular diseases.
ASCEND aims to enroll approximately 135 children, teens and adults
previously treated with Evrysdi (a nusinersen-naïve group
and a nusinersen-experienced group). All participants will
receive higher dose nusinersen in the study.
Information on the ASCEND study (NCT05067790) is available at
clinicaltrials.gov.
Ongoing Research Aims to Inform SMA Treatment
Decisions Biogen will also present baseline
characteristics from the RESPOND study investigating the efficacy
and safety of SPINRAZA in infants and toddlers who still have unmet
clinical needs following treatment with
Zolgensma® (onasemnogene abeparvovec). All study participants
(enrolled as of August 2021, n=9) who previously received the gene
therapy showed suboptimal clinical status in two or more domains at
baseline, the most common being motor and respiratory function.
Initial safety findings indicate none of the adverse events (AEs)
or serious AEs (parainfluenza virus infection and viral upper
respiratory tract infection) reported were considered related to
SPINRAZA treatment.
Additionally, the latest results from NURTURE, a study in
infants treated in the presymptomatic stage of SMA, demonstrate
that early and sustained treatment with SPINRAZA for up to 5.7
years (median 4.9 years) helped participants to maintain and make
progressive gains in motor function. After 11 months of additional
follow-up since the 2020 interim analysis, all children who were
able to walk alone maintained this ability and one child gained the
ability to walk alone, increasing the total percentage to 92
percent (23/25). Most children achieved motor milestones within
age-appropriate timelines1 and no major motor milestones were lost.
The safety of SPINRAZA over this extended follow-up period was
consistent with previously reported findings.
“SPINRAZA has demonstrated significant benefit in individuals
with SMA, from presymptomatic infants to adults with later-onset
SMA. Biogen is working to address remaining unmet needs and answer
critical questions for the SMA community through our new and
ongoing research, including studies like ASCEND and RESPOND, as
well as digital solutions to advance clinical care and patient
empowerment,” said Maha Radhakrishnan, M.D., Chief Medical Officer
at Biogen. “Additionally, the latest results from the landmark
NURTURE study continue to show that most infants who began
treatment with SPINRAZA before the clinical onset of symptoms
achieved motor milestones in timeframes consistent with normal
development.”
Featured SPINRAZA Data Presentations
Include:
- Nusinersen Effect in Infants in the Presymptomatic Stage of
SMA: 4.9-Year Interim of the NURTURE Study (oral presentation)
- Rationale and Design of ASCEND: A Phase 3b Study Evaluating
Higher Dose Nusinersen in Risdiplam-Treated Participants With
Spinal Muscular Atrophy
- Baseline Characteristics and Initial Safety Results in RESPOND:
A Phase 4 Study of Nusinersen in Children With SMA Who Received
Onasemnogene Abeparvovec
- Exploring the Content Validity of KonectomTM NMD Digital
Outcome Assessments: Perspectives of Adults Living With SMA and
ALS
*Nusinersen is currently commercialized under the brand name
SPINRAZA® and the U.S. Food and Drug Administration-approved dose
is 12 mg.
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 11,000 live births,5-8 is a
leading cause of genetic death among infants9 and causes a
range of disability in teenagers and adults.4About
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 is providing the first and only approved treatment to
address a defining pathology of Alzheimer’s disease. Biogen is also
commercializing biosimilars and focusing on advancing the
industry’s most diversified pipeline 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.
The company routinely posts information that may be important to
investors on its website at www.biogen.com. To learn more,
please visit www.biogen.com and follow Biogen on social
media
– Twitter, LinkedIn, Facebook, YouTube.
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 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, including from the
ASCEND study; the risk that we may not fully enroll our clinical
trials, including the ASCEND study, 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:
- WHO Multicentre Growth Reference Study Group. WHO Motor
Development Study: Windows of achievement for six gross motor
development milestones. Acta Paediatr Suppl. 2006 Apr;450:86-95.
doi: 10.1111/j.1651-2227.2006.tb02379.x.
- 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: March 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: March
2022.
MEDIA CONTACT:Ashleigh Koss+ 1 908 205
2572public.affairs@biogen.com |
INVESTOR CONTACT:Mike Hencke+1 781 464 2442IR@biogen.com |
Biogen (TG:IDP)
Historical Stock Chart
From Jun 2024 to Jul 2024
Biogen (TG:IDP)
Historical Stock Chart
From Jul 2023 to Jul 2024