Biogen Exercises Option with Ionis to Develop and Commercialize
Investigational Treatment BIIB067 for a Subtype of Familial
Amyotrophic Lateral Sclerosis (ALS) Based on Positive Phase 1
Data
Biogen Inc (Nasdaq: BIIB) and Ionis Pharmaceuticals, Inc.
(Nasdaq: IONS) announced today that Biogen exercised its option to
obtain from Ionis a worldwide, exclusive, royalty-bearing license
to develop and commercialize BIIB067 (IONIS-SOD1RX), an
investigational treatment for amyotrophic lateral sclerosis (ALS)
with superoxide dismutase 1 (SOD1) mutations. ALS with SOD1
mutations is a subtype of familial ALS and accounts for
approximately two percent of all ALS cases.
“Biogen and Ionis have a shared goal of bringing innovative
therapies to those living with severe neurological diseases where
there is significant unmet medical need,” said Michael Ehlers,
M.D., Ph.D., executive vice president, research and development at
Biogen. “The progress made to date in the BIIB067 clinical program
is a positive step forward. We are committed to our goal of
bringing forward a therapy for patients with ALS with SOD1
mutations, who currently have limited or no treatment options.”
The decision to exercise the option was based on a positive
interim analysis of a randomized, placebo controlled single- and
multiple-ascending dose Phase 1 study (n=70) that achieved
proof-of-biology and proof-of-concept for BIIB067. At the highest
dose tested (n=10), treatment with BIIB067 over a three month
period resulted in a statistically significant lowering of SOD1
protein levels in the cerebrospinal fluid (p=0.002) and a numerical
trend towards slowing of clinical decline as measured by the ALS
Functional Rating Scale-Revised, both compared to placebo (n=12).
The safety and tolerability profile in this analysis supports
continued development of BIIB067 in ALS. Based on these results,
Biogen plans to advance BIIB067 to a pivotal clinical study.
“The results from this study provide hope to SOD1 ALS patients
who are suffering with this devastating disease,” said C. Frank
Bennett, Ph.D., senior vice president of research and franchise
leader for neurological programs at Ionis. “BIIB067 is the first
investigational medicine targeting the known cause of this familial
form of ALS to advance towards a pivotal study. Our broad strategic
collaborations with Biogen have produced not only SPINRAZA, but six
more drugs moving forward in development and a large number of
preclinical and research programs. We believe our proprietary
antisense technology will continue to bring benefit to patients now
and in the future.”
As a part of the option exercise, Biogen made a one-time $35
million payment to Ionis. Future payments may include potential
post-licensing milestone payments of up to $55 million and
royalties in the low to mid-teen percentages on annual worldwide
net sales. Biogen will be solely responsible for the costs and
expenses related to the development, manufacturing and
commercialization of BIIB067 following the option exercise.
ALS is a rare, fatal neurodegenerative disease characterized by
motor neuron loss in the brain and spinal cord that are responsible
for controlling voluntary muscle movement. Symptoms may vary
depending on the location of the motor neuron failure and may
include muscle stiffness and spasticity or spontaneous muscle
twitching followed by atrophy. The average life expectancy for an
ALS patient is less than five years from the time of diagnosis.
Mutations within multiple genes are believed to cause ALS,
including mutations in the SOD1 gene.
Final data results from the Phase 1 study are expected to be
communicated at a future scientific forum.
About BIIB067BIIB067 is an antisense
oligonucleotide (ASO) RNase H-mediated inhibitor of SOD1 messenger
ribonucleic acid (mRNA) being developed for the treatment of ALS
with SOD1 mutations. BIIB067 binds to SOD1 mRNA, allowing its
degradation by RNase-H and reducing protein production. This is
thought to decrease the toxicity of mutant SOD1 and potentially
provide therapeutic benefit through improved survival and function
to people with ALS with SOD1 mutations. BIIB067 demonstrated
proof-of-biology and proof-of-concept in a Phase 1 interim
analysis. Biogen licensed BIIB067 from Ionis Pharmaceuticals under
a collaborative development and license agreement.
About Biogen At Biogen, our mission is clear:
we are pioneers in neuroscience. Biogen discovers, develops and
delivers worldwide innovative therapies for people living with
serious neurological and neurodegenerative diseases. One of the
world’s first global biotechnology companies, Biogen was founded in
1978 by Charles Weissmann, Heinz Schaller, Kenneth Murray and Nobel
Prize winners Walter Gilbert and Phillip Sharp, and today has the
leading portfolio of medicines to treat multiple sclerosis, has
introduced the first and only approved treatment for spinal
muscular atrophy and is focused on advancing neuroscience research
programs in Alzheimer’s disease and dementia, multiple sclerosis
and neuroimmunology, movement disorders, neuromuscular disorders,
pain, ophthalmology, neuropsychiatry and acute neurology. Biogen
also manufactures and commercializes biosimilars of advanced
biologics.
We routinely post information that may be important to investors
on our website at www.biogen.com. To learn more, please
visit www.biogen.com and follow us on social media
– Twitter, LinkedIn, Facebook, YouTube.
About Ionis Pharmaceuticals, Inc.As the leader
in RNA-targeted drug discovery and development, Ionis has created
an efficient, broadly applicable, proprietary antisense technology
platform with the potential to treat diseases where no other
therapeutic approaches have proven effective. Our drug discovery
platform has served as a springboard for actionable promise and
realized hope for patients with unmet needs – such as children and
adults with spinal muscular atrophy (SMA). We created SPINRAZA®
(nusinersen)* and are proud to have brought new hope to the SMA
community by developing the first and only approved treatment for
this disease.
Our sights are set on all the patients we have yet to reach with
a pipeline of more than 40 drugs with the potential to treat
patients with cardiovascular disease, rare diseases, neurological
diseases, infectious diseases and cancer. We created TEGSEDI™
(inotersen) the world's first RNA-targeted therapeutic approved for
the treatment of polyneuropathy of hereditary transthyretin (TTR)
amyloidosis (ATTR) in adult patients that our affiliate Akcea
Therapeutics is commercializing. Together with Akcea, we are also
bringing new medicines to patients with cardiometabolic lipid
disorders.
To learn more about Ionis follow us on twitter @ionispharma or
visit http://ir.ionispharma.com/.
*SPINRAZA® is marketed by Biogen.
Biogen Safe Harbor StatementThis press 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 results
from the Phase 1 study of BIIB067; the potential clinical effects
of BIIB067; the potential benefits, safety and efficacy of BIIB067;
the identification and treatment of ALS; the potential of Biogen’s
commercial business and pipeline programs, including BIIB067; the
anticipated benefits and potential of Biogen’s collaboration
arrangements with Ionis; 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” 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
BIIB067; the risk that Biogen may not fully enroll its clinical
trials or enrollment will take longer than expected; unexpected
concerns may arise from additional data, analysis or results
obtained during Biogen’s clinical trials; regulatory authorities
may require additional information or further studies, or may fail
or refuse to approve or may delay approval of Biogen’s drug
candidates, including BIIB067; the occurrence of adverse safety
events; the risks of other unexpected hurdles, costs or delays;
failure to protect and enforce Biogen’s data, intellectual property
and other proprietary rights and uncertainties relating to
intellectual property claims and challenges; product liability
claims; and third party collaboration risks. The foregoing sets
forth many, but not all, of the factors that could cause actual
results to differ from Biogen’s 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
Securities and Exchange Commission. These statements are based on
Biogen’s current beliefs and expectations and speak only as of the
date of this press release. Biogen does 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 StatementThis press
release includes forward-looking statements regarding the
therapeutic and commercial potential of Ionis' technologies and
products in development, including BIIB067 (IONIS-SOD1Rx),
SPINRAZA® and TEGSEDI™ (inotersen). 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, 2017, and most recent Form 10-Q quarterly
filing, which are on file with the SEC. Copies of this and other
documents are available from the Company.
Ionis Pharmaceuticals™ is a trademark of Ionis Pharmaceuticals,
Inc. Akcea Therapeutics™ is a trademark of Akcea Therapeutics, Inc.
TEGSEDI™ is a trademark of Akcea Therapeutics, Inc. SPINRAZA® is a
registered trademark of Biogen.
BIOGEN MEDIA CONTACT: David
Caouette +1 617 679 4945public.affairs@biogen.com |
BIOGEN INVESTOR CONTACT: Mike
Hencke+1 781 464 4471IR@biogen.com |
IONIS MEDIA CONTACT: Roslyn
Patterson +1 760 603 2681rpatterson@ionisph.com |
IONIS INVESTOR CONTACT: D.
Wade Walke+1 760 603 2741wwalke@ionisph.com |