CAMBRIDGE, Mass. and
CARLSBAD, Calif., Jan. 4, 2022 /PRNewswire/
-- Biogen Inc. (Nasdaq: BIIB) and Ionis
Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that Biogen
exercised its option to obtain from Ionis a worldwide, exclusive,
royalty-bearing license to develop and commercialize
BIIB115/ION306. The companies have a broad strategic collaboration
to develop novel therapies to treat neurological disorders. BIIB115
is an investigational antisense oligonucleotide (ASO) in
development for spinal muscular atrophy (SMA) that may have the
potential to help address additional unmet needs of patients as
well as to be administered at extended dosing intervals. Biogen
plans to advance BIIB115 to clinical trials to investigate safety,
tolerability, pharmacokinetics, and efficacy.
"Combining Biogen's expertise in neurology with Ionis'
leadership in antisense technology has led to SPINRAZA®
(nusinersen) being a foundation of care in SMA," said Toby Ferguson, M.D., Ph.D., Vice President and
Head of the Neuromuscular Development Unit at Biogen. "But
unmet needs still remain for people impacted by SMA. We are
excited to continue to pursue innovative treatments, such as
BIIB115, that may have the potential to make a meaningful impact
for patients in the SMA community."
SMA is characterized by loss of motor neurons in the spinal cord
and lower brain stem, resulting in severe and progressive muscular
atrophy. People with SMA do not produce enough survival motor
neuron (SMN) protein, which is critical for the maintenance of
motor neurons. BIIB115 is designed to target a root cause of
SMA by increasing the production of functional SMN protein.
"SPINRAZA has transformed the standard of care for SMA, allowing
patients to reach milestones that may have been previously
unattainable and providing hope to families. BIIB115/ION306
represents another example of our productive collaboration with
Biogen to discover and develop medicines that have potential to
significantly benefit patients suffering from neurological
diseases," said C. Frank Bennett,
Ph.D., Executive Vice President, Chief Scientific Officer and
Franchise Leader for Neurological Programs at Ionis.
As a part of the option exercise, Biogen made a one-time
$60 million payment to Ionis in the
fourth quarter of 2021. Future payments may include potential
post-licensing development, regulatory and commercial milestone
payments and royalties on annual worldwide net sales. Biogen will
be solely responsible for the costs and expenses related to the
development, manufacturing and potential future commercialization
of BIIB115 following the option exercise.
About SPINRAZA® (nusinersen) injection,
for intrathecal use 12 mg/5 mL
The SPINRAZA clinical development program encompasses 10 clinical
studies, which have included more than 300 individuals across a
broad spectrum of patient populations1, 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.2 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.2 Some individuals with SMA may never sit;
some sit but never walk; and some walk but may lose that ability
over time.3 In the absence of treatment, children
with the most severe form of SMA would usually not be expected to
reach their second birthday.2
SMA impacts approximately 1 in 10,000 live
births,4-7 is a leading cause of genetic death
among infants8 and causes a range of disability in
teenagers and adults.3
About Biogen
As 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 the 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.
We routinely post information that may be important to investors
on our website at www.biogen.com. Follow us on social media
– Twitter, LinkedIn, Facebook, YouTube.
About Ionis Pharmaceuticals, Inc.
For more than 30
years, Ionis has been the leader in RNA-targeted therapy,
pioneering new markets and changing standards of care with its
novel antisense technology. Ionis currently has three marketed
medicines and a premier late-stage pipeline highlighted by
industry-leading neurological and cardiometabolic franchises. Our
scientific innovation began and continues with the knowledge that
sick people depend on us, which fuels our vision of becoming one of
the most successful biotechnology companies.
To learn more about Ionis visit www.ionispharma.com and follow
us on Twitter @ionispharma.
Biogen Safe Harbor
This news release contains forward-looking statements, including
statements made pursuant to the safe harbor provisions of the
Private Securities Litigation Reform Act of 1995, including
statements about potential clinical effects of BIIB115; the
potential benefits, safety and efficacy of BIIB115; the clinical
development program for BIIB115; the identification and treatment
of SMA; our research and development program for the treatment of
SMA; the potential of our commercial business and pipeline
programs, including BIIB115; and risks and uncertainties associated
with drug development and commercialization. These forward-looking
statements may be accompanied by words such as "aim," "anticipate,"
"believe," "could," "estimate," "expect," "forecast," "intend,"
"may," "plan," "potential," "possible," "will," "would" and other
words and terms of similar meaning. 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. Results in early stage clinical
trials may not be indicative of full results or results from later
stage or larger scale clinical trials and do not ensure regulatory
approval. 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, uncertainty of
success in the development and potential commercialization of
BIIB115; the risk that we may not fully enroll our clinical trials
or enrollment will take longer than expected; unexpected concerns
may arise from additional data, analysis or results obtained during
our clinical trials; regulatory authorities may require additional
information or further studies, or may fail or refuse to approve or
may delay approval of our drug candidates, including BIIB115; the
occurrence of adverse safety events; the risks of unexpected
hurdles, 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; product liability claims; 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.
Ionis' Forward-looking Statements
This press release
includes forward-looking statements regarding Ionis' business and
the therapeutic and commercial potential of Ionis' technologies,
BIIB115/ION306, SPINRAZA® (nusinersen) and other
products in development. 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, including those related to the impact COVID-19 could
have on our business, and including but not limited to those
related to our commercial products and the medicines in our
pipeline, and particularly those inherent in the process of
discovering, developing and commercializing medicines that are safe
and effective for use as human therapeutics, and in the endeavor of
building a business around such medicines. 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, 2020, and the most
recent Form 10-Q quarterly filing, 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.
References:
- 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: December 2021.
- 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, Tulinius M, Kroksmark AK, Henricsson M, Darin N. 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, Milewski M, Zimowski J, Zagozdzon P,
Kostera-Pruszczyk A, Borkowska J, Sielska D, Jurek M,
Hausmanowa-Petrusewicz I. 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, Snyder PJ, Rink BD, Pearl DK, Pyatt RE, Mihal DC,
Conlan T, Schmalz B, Montgomery L, Ziegler K, Noonan C, Hashimoto
S, Garner S. 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, Nagan N, Zhu H, Akmaev VR, Zhou Z, Rohlfs EM,
Flynn K, Hendrickson BC, Scholl T, Sirko-Osadsa DA, Allitto BA.
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: December 2021.
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SOURCE Ionis Pharmaceuticals, Inc.