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 mLThe 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 BiogenAs 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 StatementsThis 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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MEDIA CONTACTS:BiogenAshleigh
Koss+ 1 908 205
2572public.affairs@biogen.comIonisRoslyn
Patterson+ 1 760 603 4679rpatteron@ionisph.com |
INVESTOR CONTACTS:BiogenMike
Hencke+1 781 464 2442IR@biogen.com
IonisJennifer Capuzelo+1 760 603
2331jcapuzelo@ionisph.com |
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