- New data from Phase 3 ENDEAR study
demonstrated earlier initiation of treatment with SPINRAZA may
improve motor function outcomes in infants with Spinal Muscular
Atrophy (SMA)
- Phase 2 EMBRACE interim analysis showed
greater motor milestone achievement in infants and children treated
with SPINRAZA, compared to those untreated, in patient populations
not studied in the pivotal trials
- Interim analysis of EMBRACE also
supported the dosing regimen of four loading doses in the first two
months, followed by the administration of SPINRAZA every four
months thereafter for infantile- and later-onset SMA
Biogen (NASDAQ: BIIB) presented new data demonstrating that
earlier initiation of treatment with SPINRAZA® (nusinersen) may
improve motor function outcomes in infants and children with spinal
muscular atrophy (SMA). Results continued to reinforce the
favorable efficacy and safety profile of SPINRAZA. The data were
shared at the 22nd International Annual Congress of the World
Muscle Society in Saint Malo, France (October 3-7, 2017).
A new analysis from the Phase 3 ENDEAR study showed infants with
SMA who initiated treatment earlier in the disease (shorter disease
duration) demonstrated greater benefit and improvement in motor
function outcomes.
As measured by the Hammersmith Infant Neurological Examination
(HINE), significant differences in motor milestone responders were
observed between infants treated with SPINRAZA compared to
untreated infants with disease duration less than or equal to 12
weeks (75% vs. 0%; P<.0001) and those with disease duration
greater than 12 weeks (32% vs. 0%; P=.0026). There was also a
significant benefit in event-free survival in infants treated with
SPINRAZA with disease duration less than or equal to 12 weeks
(P=.0004).
“These studies contribute to a growing body of evidence that
SPINRAZA can make a meaningful difference in the lives of people
with SMA regardless of their age or stage of the disease,” said
Alfred Sandrock, M.D., Ph.D., executive vice president and chief
medical officer at Biogen. “Across studies, we continue to see
evidence that earlier initiation of treatment with SPINRAZA can
lead to improved clinical and functional outcomes.”
Interim analyses were also presented from the Phase 2 EMBRACE
study which was designed to assess the efficacy and safety of
SPINRAZA in individuals with infantile- and later-onset SMA who
were ineligible for the two earlier pivotal studies.
The EMBRACE interim analysis showed a larger proportion of
infants and children treated with SPINRAZA were HINE motor
milestone responders compared to those who were untreated. Results
from the interim analysis also supported the dosing regimen of four
loading doses in the first two months, followed by the
administration of SPINRAZA every four months thereafter, for
individuals with infantile- and later-onset SMA.
In the ENDEAR and EMBRACE studies SPINRAZA demonstrated a
favorable benefit-risk profile. Safety data involving the
intrathecal administration of SPINRAZA showed the incidence and
nature of the most common lumbar puncture-related adverse events in
the clinical studies were similar in children with later-onset SMA
with or without scoliosis.
For more information about SPINRAZA and prescribing information
in the United States, please visit www.SPINRAZA.com. Prescribing
information in the European Union is available at
http://www.ema.europa.eu/ema/.
About ENDEAR and EMBRACEENDEAR is a randomized,
double-blind, sham-procedure controlled 13-month study in patients
with infantile-onset SMA. The end of study efficacy analysis
included all patients (n=121) who had their final study visit after
the interim analysis (n=78) and had the opportunity to attend the
six-month study visit assessment. The Hammersmith Infant
Neurological Examination (HINE) is a reliable and clinically
validated tool to assess motor milestone achievement in infants
with SMA.
EMBRACE is a Phase 2, multicenter, randomized, double-blind,
sham-procedure controlled 14-month study of SPINRAZA in infants and
children not eligible to participate in ENDEAR (symptom onset less
than or equal to six months, less than or equal to seven months of
age at screening; 2 SMN2 copies) or CHERISH (symptom onset age
greater than six months, age 2-12 years at screening).
SPINRAZA Program StatusSPINRAZA is the first approved
medicine for the treatment of SMA and is currently approved in the
United States, the European Union, Brazil, Japan and Canada. Biogen
has submitted regulatory filings in additional countries and plans
to initiate additional filings in other countries.
Globally, in 2016, in response to the urgent need for treatment
for the most severely affected individuals living with SMA, Biogen
sponsored one of the largest, pre-approval Expanded Access Programs
(EAP) in rare disease, free of charge.
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 SMA1-5Spinal 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 “aim,” “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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