SPINRAZA Demonstrated Motor Function
Improvements in Infants on Permanent Ventilation; No Increase in
Risk of Adverse Events in Children with Scoliosis
Biogen Highlights SPINRAZA Data at the Cure SMA
2017 Annual SMA Conference
Biogen (NASDAQ: BIIB) will present robust efficacy and safety
data from Phase 2 and 3 SPINRAZA® (nusinersen) studies at the Cure
SMA 2017 Annual SMA Conference in Orlando, Fl, June 29 – July 2,
2017. The breadth of data presented reinforces the significant and
clinically meaningful efficacy of SPINRAZA on the achievement of
motor milestones and measures of motor function across a broad
range of individuals with spinal muscular atrophy (SMA), as well as
on survival endpoints in infantile-onset SMA.
“Data presented at the Cure SMA 2017 Annual SMA Conference
further demonstrate the significant impact of SPINRAZA and the
benefits of early treatment initiation. We are encouraged to see
unprecedented motor function gains in infants on permanent
ventilation and a continued favorable benefit-risk profile across a
broad population including no increase in risk of adverse events in
children who have developed scoliosis.” said Wildon Farwell, M.D.,
M.P.H., senior medical director, Clinical Development, Biogen. “As
part of our mission to make a meaningful difference in the lives of
those affected by SMA, we continue to collect and evaluate data to
provide a deeper understanding of the impact of SPINRAZA across SMA
populations and share those results with the SMA community.”
New SPINRAZA Data Show Robust Efficacy and Safety Across
Broad Range of Individuals with SMAIn an analysis of the Phase
3 ENDEAR end of study results, a greater proportion of infants with
SMA on permanent ventilation treated with SPINRAZA demonstrated
clinical benefits compared to untreated infants.
End of study data from both the Phase 3 ENDEAR and CHERISH
studies further demonstrate that earlier SPINRAZA treatment in
individuals with SMA may lead to improved outcomes. In individuals
with shorter disease durations (i.e., generally younger at symptom
onset), infants in ENDEAR demonstrated a lower risk of death or
permanent ventilation and children in CHERISH demonstrated greater
motor function improvement from baseline to 15 months compared to
untreated individuals.
In addition, further results from the interim analysis of the
Phase 2 NURTURE study highlight the clinically meaningful efficacy
of SPINRAZA on event-free survival, measures of motor function and
achievement of motor milestones when administered to infants with
genetically-diagnosed SMA before symptom onset.
“New SPINRAZA data continue to reinforce the positive results
seen in clinical studies and in my own practice,” said Thomas
Crawford, M.D., co-director, Muscular Dystrophy Association Clinic
at Johns Hopkins Medicine. “The SPINRAZA clinical development
program demonstrates the impact of early treatment. The additional
NURTURE data extends this finding by showing substantial
improvements in motor milestones, generally consistent with normal
development among infants with SMA who have yet to manifest
symptoms before they were treated with SPINRAZA.”
SPINRAZA demonstrated a favorable benefit-risk profile, with
commonly reported adverse events consistent with those expected in
the general SMA population or related to a lumbar puncture
procedure. Safety data involving the intrathecal administration of
SPINRAZA showed the incidence and nature of the most common lumbar
puncture-related adverse events were similar in children with
later-onset SMA with or without scoliosis in the clinical
studies.
Biogen to Participate in 15 Presentations at the
MeetingSelect SPINRAZA data highlights are included below:
- Infants and children with SMA treated
with nusinersen in clinical trials: Experience of risk for
respiratory or other events with repeat anesthesia/sedation for
intrathecal administration. June 29, 2017: 4:30-6:30 p.m. ET
- Infants and children with SMA treated
with nusinersen in clinical trials: An integrated safety analysis.
June 30, 2017: 12:45 p.m.-2:45 p.m. ET
- Nusinersen demonstrates efficacy in
infants with and without permanent ventilation: Final results from
the ENDEAR study. July 1, 2017: 11:00 a.m. ET
- Efficacy and safety of nusinersen in
genetically diagnosed infants with presymptomatic spinal muscular
atrophy (SMA): Results from the second interim analysis of the
ongoing, phase 2 NURTURE study. July 1, 2017: 11:20 a.m. ET
- Efficacy and safety of nusinersen in
children with later-onset spinal muscular atrophy (SMA): End of
study results from the phase 3 CHERISH. July 1, 2017: 11:40 a.m.
ET
For more information about SPINRAZA and U.S. prescribing
information, visit www.SPINRAZA.com.
SPINRAZA Program StatusSPINRAZA was first approved by the
U.S. Food and Drug Administration (FDA) for the treatment of SMA in
pediatric and adult patients on December 23, 2016 within three
months of regulatory filing. The European Commission (EC) granted a
marketing authorization for SPINRAZA for the treatment of 5q SMA on
June 1, 2017, making SPINRAZA the first approved treatment in the
European Union for SMA.
Biogen has also submitted regulatory filings in Japan, Canada,
Australia, Switzerland, and Brazil and plans to initiate additional
filings in other countries in 2017.
Biogen licensed the global rights to develop, manufacture and
commercialize SPINRAZA from Ionis Pharmaceuticals (NASDAQ: IONS), a
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 SMA 1-5Spinal muscular atrophy (SMA) is
characterized by loss of motor neurons in the spinal cord and lower
brain stem, resulting in severe and progressive muscular atrophy
and weakness. Ultimately, individuals with the most severe type of
SMA can become paralyzed and have difficulty performing the basic
functions of life, like breathing and swallowing.
Due to a loss of, or defect in, the SMN1 gene, people with SMA
do not produce enough SMN protein, which is critical for the
maintenance of motor neurons. The severity of SMA correlates with
the amount of SMN protein. People with Type 1 SMA, the form that
requires the most intensive and supportive care, produce very
little SMN protein and do not achieve the ability to sit without
support or live beyond two years without respiratory support.
People with Type 2 and Type 3 SMA produce greater amounts of SMN
protein and have less severe, but still life-altering forms of
SMA.
About SPINRAZA® (nusinersen)SPINRAZA is
being developed globally for the treatment of SMA.
SPINRAZA is an antisense oligonucleotide (ASO), using Ionis
Pharmaceuticals’ proprietary antisense technology, that is designed
to treat SMA caused by mutations or deletions in the SMN1 gene
located in chromosome 5q that leads to SMN protein deficiency.
SPINRAZA alters the splicing of SMN2 pre-mRNA in order to increase
production of full-length SMN protein.6 ASOs are short synthetic
strings of nucleotides designed to selectively bind to target RNA
and regulate gene expression. Through use of this technology,
SPINRAZA has the potential to increase the amount of full-length
SMN protein in individuals with SMA.
SPINRAZA must be administered via intrathecal injection, which
delivers therapies directly to the cerebrospinal fluid (CSF) around
the spinal cord,7 where motor neurons degenerate in individuals
with SMA due to insufficient levels of survival motor neuron (SMN)
protein.8
SPINRAZA demonstrated a favorable benefit-risk profile. The most
common adverse reactions reported for SPINRAZA were upper
respiratory infection, lower respiratory infection and
constipation. Serious adverse reactions of atelectasis were more
frequent in SPINRAZA-treated patients. Coagulation abnormalities
and thrombocytopenia, including acute severe thrombocytopenia, have
been observed after administration of some antisense
oligonucleotides. Individuals may be at increased risk of bleeding
complications. Renal toxicity has been observed after
administration of some antisense oligonucleotides. SPINRAZA is
present in and excreted by the kidney.
About BiogenThrough cutting-edge science and medicine,
Biogen discovers, develops and delivers innovative therapies
worldwide for people living with serious neurological and
neurodegenerative diseases. Founded in 1978, Biogen is a pioneer in
biotechnology and today the Company has the leading portfolio of
medicines to treat multiple sclerosis, has introduced the first and
only approved treatment for spinal muscular atrophy, and is at the
forefront of neurology research for conditions including
Alzheimer’s disease, Parkinson’s disease and amyotrophic lateral
sclerosis. Biogen also manufactures and commercializes biosimilars
of advanced biologics. For more information, please visit
www.biogen.com. Follow us on social media – Twitter, LinkedIn,
Facebook, YouTube.
Biogen Safe HarborThis press release contains
forward-looking statements, including statements made pursuant to
the safe harbor provisions of the Private Securities Litigation
Reform Act of 1995 relating to the potential benefits, safety and
efficacy of SPINRAZA, the results of certain real-world data, the
status of current regulatory filings, plans for additional
regulatory filings in other jurisdictions, planning and timing for
commercial launch, and availability of patient access and
reimbursement pathways, which may vary on a country-by-country
basis. These forward-looking statements may be accompanied by words
such as “anticipate,” “believe,” “could,” “estimate,” “except,”
“forecast,” “intend,” “may,” “plan,” “potential,” “possible,”
“will” and other words and terms of similar meaning. You should not
place undue reliance on these statements or the scientific data
presented. Drug development and commercialization involve a high
degree of risk. 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 commercialization of SPINRAZA, which may
be impacted by, among other things, the level of preparedness of
healthcare providers to treat patients, difficulties in obtaining
or changes in the availability of reimbursement for SPINRAZA, the
effectiveness of sales and marketing efforts, problems with the
manufacturing process for SPINRAZA, the occurrence of adverse
safety events, unexpected concerns that may arise from additional
data or analysis; regulatory authorities may require additional
information or further studies, or may fail to approve or may delay
approval of Biogen’s drug candidates or expansion of product
labeling; or Biogen may encounter other unexpected hurdles which
may be impacted by, among other things, the occurrence of adverse
safety events, failure to obtain regulatory approvals in certain
jurisdictions, failure to obtain regulatory approvals in other
jurisdictions, failure to protect intellectual property and other
proprietary rights; product liability claims; or third party
collaboration risks. 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 Biogen’s most recent annual or quarterly report and
in other reports Biogen has 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
press 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.
1. 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.
2. Lefebvre S, Burglen L, Reboullet S, et al. Identification and
characterization of a spinal muscular atrophy-determining gene.
Cell.1995;80(1):155-165.
3. Mailman MD, Heinz JW, Papp AC, et al. Molecular analysis of
spinal muscular atrophy and modification of the phenotype by SMN2.
Genet Med. 2002;4(1):20-26.
4. Monani UR, Lorson CL, Parsons DW, et al. A single nucleotide
difference that alters splicing patterns distinguishes the SMA gene
SMN1 from the copy gene SMN2. Hum Mol Genet.
1999;8(7):1177-1183.
5. Peeters K, Chamova T, Jordanova A. Clinical and genetic
diversity of SMN1-negative proximal spinal muscular atrophies.
Brain.2014;137(Pt 11):2879-2896.
6. Hua Y, Sahashi K, Hung G, Rigo F, Passini MA, Bennett CF,
Krainer AR. Antisense correction of SMN2 splicing in the CNS
rescues necrosis in a type III SMA mouse model. Genes Dev. 2010 Aug
1; 24(15):16344-44.
7. Evers MM, Toonen LJ, van Roon-Mom WM. Antisense
oligonucleotides in therapy for neurodegenerative
disorders. Adv Drug Deliv Rev. 2015;87:90-103.
8. Lunn MR, Wang CH. Spinal muscular
atrophy. Lancet. 2008;371(9630):2120-2133.
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