Submission of Marketing Authorization
Application to the European Medicines Agency Planned in the Coming
Weeks
Biogen (NASDAQ: BIIB) and Ionis (NASDAQ:IONS) today announced
that Biogen has completed the rolling submission of a New Drug
Application (NDA) to the U.S. Food and Drug Administration (FDA)
for the approval of nusinersen, an investigational treatment for
spinal muscular atrophy (SMA). Biogen has also applied for Priority
Review which, if granted, would shorten the review period of
nusinersen following the Agency’s acceptance of the NDA.
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“Since announcing the positive results of the ENDEAR interim
analysis in infantile-onset SMA last month, we have heard from many
families expressing their excitement about nusinersen. Their
stories continue to inspire us and they are in the forefront of our
minds as we work to support the FDA’s review of nusinersen,” noted
Alfred Sandrock, M.D., Ph.D., executive vice president and chief
medical officer at Biogen. “We appreciate the FDA’s collaboration
with us during the application process, and we look forward to
continuing this productive dialogue, with the goal of rapidly
bringing the first treatment for SMA to as many patients as
possible.”
In addition to the NDA filing with FDA, Biogen plans to submit a
Marketing Authorization Application (MAA) for nusinersen to the
European Medicines Agency (EMA) in the coming weeks. The EMA’s
Committee for Medicinal Products for Human Use (CHMP) recently
granted Accelerated Assessment to nusinersen, which can reduce the
standard review time. Biogen will initiate regulatory filings in
other countries in the coming months.
“Our ability to advance the nusinersen program as quickly as we
have is largely due to the tremendous contributions of the entire
SMA community, from the patients and families who participated in
the clinical trials to the doctors, nurses and advocates who work
tirelessly on their behalf,” said B. Lynne Parshall, chief
operating officer at Ionis Pharmaceuticals. “We are deeply
appreciative of their unwavering commitment to finding a
treatment for SMA and today’s milestone is truly a
collective achievement.”
The regulatory submissions are comprised of results from the
pre-specified interim analysis of ENDEAR, the controlled Phase 3
study evaluating nusinersen in infantile-onset (most likely to
develop Type 1) SMA, as well as all other clinical and preclinical
data currently available, which includes open-label data in other
patients types. The ENDEAR interim analysis demonstrated that
infants receiving nusinersen experienced a statistically
significant improvement in the achievement of motor milestones
compared to those who did not receive treatment. Biogen anticipates
hearing from regulatory authorities regarding the acceptance and
validation of these submissions within the next couple of
months.
The Nusinersen Clinical Trial Program
The nusinersen clinical trial program is comprised of two
controlled studies, ENDEAR and CHERISH. ENDEAR was designed as a
thirteen-month study investigating nusinersen in 122 patients with
infantile-onset SMA; the onset of signs and symptoms of SMA less
than or equal to 6 months of age and age less than or equal to 7
months at screening. Based on the results of the pre-specified
interim analysis, the ENDEAR study will be stopped; patients who
elect to are currently being transitioned to the SHINE open-label
study where they will all receive nusinersen. Results from the
ENDEAR interim analysis will be presented at future medical
congresses.
CHERISH is a fifteen-month study investigating nusinersen in 126
non-ambulatory patients with later-onset SMA, consistent with Type
2; onset of signs and symptoms greater than 6 months and age 2 to
12 years at screening. CHERISH was fully enrolled in May 2016 and
remains ongoing.
Additionally, the SHINE open-label extension study, for patients
who previously participated in ENDEAR and CHERISH, is open and is
intended to evaluate the long-term safety and tolerability of
nusinersen.
Two additional Phase 2 studies, EMBRACE and NURTURE, were
designed to collect additional data on nusinersen. The EMBRACE
study is designed to collect additional data on a small subset of
patients with infantile or later-onset SMA who do not meet the age
and other criteria of ENDEAR or CHERISH. Due to the evidence
demonstrated in the infantile-onset SMA (most likely to develop
Type 1) population, the sham arm of the EMBRACE study is being
stopped and patients are being given the option to receive
nusinersen through an open-label extension study. NURTURE is an
open-label, ongoing study in pre-symptomatic infants who are less
than or equal to 6 weeks of age at time of first dose to determine
if treatment before symptoms begin would prevent or delay onset of
SMA symptoms.
About SMA1-5
Spinal 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 survival motor neuron (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 most severe life-threatening form, produce very little SMN
protein and do not achieve the ability to sit without support or
live beyond 2 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.
Currently, there is no approved treatment for SMA.
To support awareness and education in SMA, Biogen is launching
Together in SMA in the United States, a program created to provide
informational materials and resources to the SMA community. Learn
more at TogetherinSMA.com.
About Nusinersen
Nusinersen is an investigational, potentially disease-modifying
therapy6 for the treatment of SMA. Nusinersen is an antisense
oligonucleotide (ASO) that is designed to alter the splicing of
pre-mRNA from the SMN2 gene in order to increase production of
fully functional SMN protein. SMN2 is a gene that is nearly
identical to SMN1.7
ASOs are short synthetic strings of nucleotides designed to
selectively bind to target RNA and regulate gene expression.
Through use of this technology, nusinersen has the potential to
increase the amount of functional SMN protein in infants and
children with SMA.
Both the U.S. and EU regulatory agencies have granted special
status to nusinersen in an effort to expedite the review process,
including Fast Track Designation in the U.S. Additionally,
nusinersen has received an Orphan Drug designation in both U.S. and
EU.
We acknowledge support from the following organizations for
nusinersen: Muscular Dystrophy Association, SMA Foundation, Cure
SMA and intellectual property licensed from Cold Spring Harbor
Laboratory and the University of Massachusetts Medical School.
About Biogen
Through cutting-edge science and medicine, Biogen discovers,
develops and delivers worldwide innovative therapies for people
living with serious neurological, autoimmune and rare diseases.
Founded in 1978, Biogen is one of the world’s oldest independent
biotechnology companies and patients worldwide benefit from its
leading multiple sclerosis and innovative hemophilia therapies. For
more information, please visit www.biogen.com. Follow us
on Twitter.
About Ionis Pharmaceuticals Inc.
Ionis is the leading company in RNA-targeted drug discovery and
development focused on developing drugs for patients who have the
highest unmet medical needs, such as those patients with severe and
rare diseases. Using its proprietary antisense technology, Ionis
has created a large pipeline of first-in-class or best-in-class
drugs, with over a dozen drugs in mid- to late-stage development.
Drugs currently in Phase 3 development include volanesorsen, a drug
Ionis is developing and plans to commercialize through its wholly
owned subsidiary, Akcea Therapeutics, to treat patients with either
familial chylomicronemia syndrome or familial partial
lipodystrophy; IONIS-TTRRx, a drug Ionis is developing with GSK to
treat patients with all forms of TTR amyloidosis; and nusinersen, a
drug Ionis is developing with Biogen to treat infants and children
with spinal muscular atrophy. Ionis' patents provide strong and
extensive protection for its drugs and technology. Additional
information about Ionis is available at www.ionispharma.com.
Biogen Safe Harbor
This press release contains forward-looking statements,
including statements relating to the submission of marketing
authorization applications for nusinersen to regulatory authorities
and the timing thereof and the anticipated regulatory filing review
process. These statements may be identified by words such as
“believe,” “except,” “may,” “plan,” “potential,” “will” and similar
expressions, and are based on our current beliefs and expectations.
Drug development and commercialization involve a high degree of
risk, and only a small number of research and development programs
result in commercialization of a product. Factors which could cause
actual results to differ materially from our current expectations
include the risk that unexpected concerns 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 our drug candidates or grant marketing approval that is
more restricted than anticipated, or we may encounter other
unexpected hurdles. For more detailed information on the risks and
uncertainties associated with our drug development and
commercialization activities, please review the Risk Factors
section of our most recent annual report or quarterly report filed
with the Securities and Exchange Commission. Any forward-looking
statements speak only as of the date of this press release and we
assume no obligation to update any forward-looking statement.
Ionis Forward-Looking Statement
This press release includes forward-looking statements regarding
Ionis' strategic relationship with Biogen and the development,
activity, therapeutic potential, safety and commercialization of
nusinersen. Any statement describing Ionis' goals, expectations,
financial or other projections, intentions or beliefs is a
forward-looking statement and should be considered an at-risk
statement. Such statements are subject to certain risks and
uncertainties, particularly those inherent in the process of
discovering, developing and commercializing drugs that are safe and
effective for use as human therapeutics, and in the endeavor of
building a business around such drugs. Ionis' forward-looking
statements also involve assumptions that, if they never materialize
or prove correct, could cause its results to differ materially from
those expressed or implied by such forward-looking statements.
Although Ionis' forward-looking statements reflect the good faith
judgment of its management, these statements are based only on
facts and factors currently known by Ionis. As a result, you are
cautioned not to rely on these forward-looking statements. These
and other risks concerning Ionis' programs are described in
additional detail in Ionis' annual report on Form 10-K for the year
ended December 31, 2015, and its most recent quarterly report on
Form 10-Q, which are on file with the SEC. Copies of these and
other documents are available from the Company.
BIOGEN and the BIOGEN logo are registered trademarks of
BIOGEN.
Ionis Pharmaceuticals™ is a trademark of Ionis Pharmaceuticals,
Inc. Akcea Therapeutics™ is a trademark of Ionis Pharmaceuticals,
Inc.
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. Rigo F,
Hua Y, Krainer AR, Bennett CF. Antisense-based therapy for the
treatment of spinal muscular atrophy. J Cell Biol.
2012;199(1):21-25 7. 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
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version on businesswire.com: http://www.businesswire.com/news/home/20160926005499/en/
Media:BiogenLigia Del Bianco, +1
781-464-3260public.affairs@biogen.comorIonis
PharmaceuticalsWade Walke, +1
760-603-2741CorpComm@ionisph.comorInvestors:BiogenBen
Strain, +781-464-2442IR@biogen.comorIonis
PharmaceuticalsWade Walke, +1
760-603-2741CorpComm@ionisph.com
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