- Roche gains exclusive worldwide rights to develop,
manufacture and commercialize the investigational medicines
discovered by Ionis
- Ionis to receive a $60 million
upfront payment
CARLSBAD, Calif., Sept. 27,
2023 /PRNewswire/ -- Ionis Pharmaceuticals,
Inc. (Nasdaq: IONS) today announced that it has entered an
agreement with Roche for two undisclosed early-stage programs for
RNA-targeting investigational medicines for the treatment of
Alzheimer's disease (AD) and Huntington's disease (HD). Roche gains
exclusive worldwide rights and will be responsible for clinical
development, manufacturing, and commercialization of the medicines
if they receive regulatory approval. The companies will leverage
Ionis' expertise discovering medicines that target the root cause
of central nervous system diseases and Roche's global experience
developing and commercializing therapies for nervous system
disorders.
"We are excited to expand our partnership with Roche, a global
leader in developing and delivering innovative treatments to
patients," said Brett P. Monia,
Ph.D., Ionis' chief executive officer. "With this new
collaboration, we are joining forces to accelerate the discovery
and development of novel therapies for people living with
Alzheimer's disease and Huntington's disease globally.
Collaborating on these two programs enables Ionis to advance our
wholly owned programs, including those in neurology, aligned with
our strategic priorities."
"Our lasting partnership with Ionis, a leader in RNA-targeted
therapeutics, is a great example of two collaborators mutually
benefiting from their relationship by complementing and
learning from each other," said James
Sabry, Ph.D., global head of Roche Pharma partnering. "By
expanding our alliance, we bring together the companies' combined
knowledge of the science in Alzheimer's disease and Huntington's
disease with Roche's proven capabilities in the development and
commercialization of innovative treatments in
neuroscience."
Under terms of the agreement, Ionis will have responsibility to
advance the two programs through pre-clinical studies; Roche will
have sole responsibility for clinical development and, if approved,
commercialization worldwide. Ionis will receive a $60 million upfront payment from Roche and is
eligible to receive development, regulatory and commercial
milestone payments, and tiered royalties.
Ionis and Roche have been strong partners for the past 10 years,
starting with their collaboration in 2013 for tominersen, an
Ionis-discovered investigational medicine for HD. Tominersen,
designed to reduce the production of all forms of the huntingtin
protein, is currently being evaluated by Roche in a Phase 2 proof
of concept study (GENERATION HD2) assessing safety, biomarker and
efficacy trends in people with prodromal or early manifest HD. In
addition to tominersen, both companies are working together on a
pre-clinical stage mutant HTT selective antisense oligonucleotide
for HD. The companies entered another collaboration in 2018 to
develop IONIS-FB-LRx targeting Factor B for the
treatment of IgA nephropathy (IgAN) and geographic atrophy (GA).
IONIS-FB-LRx is currently in a Phase 2 study for GA, led
by Ionis, and a Phase 3 study for IgAN led by Roche.
About Alzheimer's
Disease
AD is the most common cause of dementia and, according to the
World Health Organization, more than 55 million people have
dementia worldwide1. During the course of the disease,
protein plaques appear in the brain, leading to the death of brain
cells. No single factor has been identified as the cause of
Alzheimer's disease.
About Huntington's
Disease
HD is a rare, genetic, neurodegenerative disease characterized
by a triad of cognitive, behavioral, and motor symptoms leading to
functional decline, progressive loss of independence, and impacting
families across generations. It is caused by the expansion of the
cytosine-adenine-guanine (CAG) trinucleotide sequence in the HTT
gene. The resulting mutant HTT protein is toxic and gradually
destroys neurons. Symptoms usually appear between the ages of 30
and 50 and worsen over a 10 to 25-year period. There is no
effective treatment or cure for the disease and currently available
medicines only mask the patient's symptoms but do not slow down the
underlying loss of neurons.
About Ionis Pharmaceuticals,
Inc.
For more than 30 years, Ionis has been a leader in RNA-targeted
therapy, pioneering new markets and changing standards of care.
Ionis currently has four marketed medicines and a promising
late-stage pipeline highlighted by cardiovascular and neurological
franchises. Our scientific innovation began and continues with the
knowledge that sick people depend on us, which fuels our vision to
become the leader in genetic medicine, utilizing a multi-platform
approach to discover, develop and deliver life-transforming
therapies.
To learn more about Ionis visit www.ionispharma.com and
follow us on Twitter @ionispharma.
Ionis' Forward-looking
Statements
This press release includes forward-looking statements regarding
Ionis' business and the therapeutic and commercial potential of
tominersen, Ionis' newly initiated programs for Alzheimer's disease
and Huntington's disease, IONIS-FB-LRx, Ionis'
technologies 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 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 Dec. 31, 2022, and the most
recent Form 10-Q quarterly filing, which are on file with the
Securities and Exchange Commission. 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.
1 World Health Organization. Dementia.
https://www.who.int/news-room/fact-sheets/detail/dementia
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SOURCE Ionis Pharmaceuticals, Inc.