CARLSBAD, Calif., Aug. 30, 2017 /PRNewswire/ -- Ionis
Pharmaceuticals, Inc. (NASDAQ: IONS) announced today that it has
earned a $40 million milestone
payment from Biogen associated with the pricing approval of
SPINRAZA® (nusinersen) in Japan. SPINRAZA was approved for individuals
with infantile-onset spinal muscular atrophy (SMA) by the
Pharmaceuticals and Medical Devices Agency in Japan in June
2017. To date, Ionis has earned more than $435 million from Biogen related to SPINRAZA.
"We are grateful to the Japanese SMA community, including the
patients who participated in the clinical studies, along with their
families and the investigators, for their dedication, perseverance
and support. Each of them played a pivotal role in bringing
SPINRAZA to the Japanese market and we are pleased that SPINRAZA is
now available to Japanese infantile-onset individuals with SMA,"
said Stanley T. Crooke, chairman and
chief executive officer of Ionis Pharmaceuticals. "SPINRAZA is now
approved in the U.S., EU, Japan,
Canada and Brazil, which brings us closer to achieving
our shared goal with Biogen that all patients with SMA will have
access to this life-changing medicine."
"We expect to see continued strong patient demand for SPINRAZA
as the launch continues in the U.S. and with the commercial
rollouts in Europe, Japan and around the globe," said B. Lynne Parshall, chief operating officer at
Ionis Pharmaceuticals. "Biogen is working to expand access to
SPINRAZA, with marketing authorization applications currently under
review in Switzerland,
Israel, South Korea and Australia, and additional filings planned. We
believe the speed and breadth at which SPINRAZA has achieved
marketing approval across geographies so far is a recognition of
the immense value SPINRAZA may bring to patients."
Biogen licensed the global rights to develop, manufacture and
commercialize SPINRAZA from Ionis Pharmaceuticals in August 2016 and is now responsible for all
development, regulatory and commercialization activities and costs
for SPINRAZA. SPINRAZA was first approved by the U.S. Food and Drug
Administration (FDA) on December 23,
2016, within three months of regulatory filing.
ABOUT SMA
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 type 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 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. It was discovered and
co-developed by Ionis Pharmaceuticals, a leader in antisense
therapeutics, and Biogen. 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, where motor neurons degenerate in patients with
SMA due to insufficient levels of SMN protein.
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. The EAP has successfully led
to the initiation and ongoing treatment of almost 600 eligible
individuals with infantile-onset SMA (most likely to develop Type
1) across 24 countries.
For complete SPINRAZA U.S. prescribing information please visit
www.SPINRAZA.com.
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 three
dozen drugs in development. SPINRAZA® (nusinersen) has
been approved in the U.S., Europe,
Japan, Canada and Brazil for the treatment of spinal muscular
atrophy (SMA). Biogen is responsible for commercializing SPINRAZA.
Drugs that have successfully completed Phase 3 studies include
inotersen (IONIS-TTRRx), an antisense drug Ionis is
developing to treat patients with TTR amyloidosis, and
volanesorsen, an antisense drug discovered by Ionis and
co-developed by Ionis and Akcea Therapeutics to treat patients with
either familial chylomicronemia syndrome or familial partial
lipodystrophy. Akcea, an affiliate of Ionis, is a biopharmaceutical
company focused on developing and commercializing drugs to treat
patients with serious cardiometabolic diseases caused by lipid
disorders. If approved, volanesorsen will be commercialized through
Ionis' affiliate, Akcea. Both inotersen and volanesorsen are
progressing toward regulatory filings for marketing authorization.
Ionis' patents provide strong and extensive protection for its
drugs and technology. Additional information about Ionis is
available at www.ionispharma.com.
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 SPINRAZA. 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, 2016, 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.
In this press release, unless the context requires otherwise,
"Ionis," "Company," "we," "our," and "us" refers to Ionis
Pharmaceuticals and its subsidiaries.
Ionis Pharmaceuticals™ is a trademark of Ionis Pharmaceuticals,
Inc. Akcea Therapeutics™ is a trademark of Ionis Pharmaceuticals,
Inc. SPINRAZA® is a registered trademark of Biogen.
View original content with
multimedia:http://www.prnewswire.com/news-releases/ionis-earns-40-million-spinraza-regulatory-milestone-payment-from-biogen-300511251.html
SOURCE Ionis Pharmaceuticals, Inc.