CARLSBAD, Calif., Jan. 18, 2022 /PRNewswire/ -- Ionis
Pharmaceuticals, Inc. (Nasdaq: IONS) announced that its partner,
Roche, is designing a new Phase 2 trial to evaluate tominersen in
Huntington's disease (HD). After halting dosing in the Phase 3
GENERATION HD1 study, exploratory post-hoc analyses suggest
tominersen may benefit younger adult patients with lower disease
burden. These results require confirmation in a randomized,
placebo-controlled study.
"These findings are promising and warrant a new study designed
to test tominersen in this specific patient group. We are pleased
that Roche has determined that there is a path to advance the
tominersen development program," said C. Frank Bennett, Ph.D., Ionis' executive vice
president, chief scientific officer and franchise leader for
neurological programs. "This is an encouraging development for the
HD community. We and Roche are grateful to the HD community's
continued partnership, which has led to these important
insights and a new scientific hypothesis."
Roche is in the early stages of designing the Phase 2 clinical
trial to explore different doses of tominersen in a younger adult
patient population with less disease burden. Roche will share
further details about the design of the new Phase 2 trial at future
scientific meetings. Beginning with the scientific community on
Jan. 20, 2022, Roche will participate
in a series of webinars to discuss the findings from their post-hoc
analysis of the GENERATION HD1 study data and next steps for the
tominersen program.
About tominersen and the clinical trials
Tominersen, previously IONIS-HTTRx or RG6042, is an
investigational antisense medicine designed to reduce the
production of all forms of the huntingtin protein (HTT), including
its mutated variant, mHTT. In December
2017, Roche licensed the investigational medicine from
Ionis. The tominersen clinical development program included
the following studies:
- GENERATION HD1: a randomized, multicenter, double-blind,
placebo-controlled Phase 3 clinical study evaluating the efficacy
and safety of treatment with tominersen in people with manifest HD
over 25 months. Study participants were randomized to either 120 mg
every two months or 120 mg every four months with intrathecal
injections of tominersen, or placebo. The study recruited 791
participants from 18 countries around the world. In March 2021, Roche announced that dosing would be
stopped in the tominersen Phase 3 GENERATION HD1 study following a
recommendation from the independent data monitoring committee
(iDMC) based on an overall benefit/risk assessment. The study is
ongoing without dosing to allow participants to be followed for
safety and clinical outcomes. The study will complete at the
last-patient last-visit, as planned in March/April 2022.
- GEN-EXTEND: an open label extension study for participants
coming from any Roche HD study. Participants received 120 mg
tominersen every two months or every four months in the study. The
study is ongoing without dosing to allow participants to be
followed for safety and clinical outcomes. The study will
complete in
March/April 2022.
- GEN-PEAK: a Phase I study aiming to better understand the
pharmacokinetics of tominersen and how tominersen affects mHTT
levels and other markers in the spinal fluid and blood, which
studies different doses of tominersen over two
administrations. The study will now be completed as all patients
have completed Part 1 of the study and Part 2 will not be
conducted.
Study participants should contact their local clinical trial
site for questions or support about their
situation.
About Huntington's disease
Huntington's disease is a rare genetic, progressive condition
that causes the nerve cells in the brain to break down, causing
problems with a person's ability to think, move and function,
leading to increasing disability and loss of independence. It has a
devastating impact on people living with the disease, and the
hereditary nature of HD means it profoundly affects entire families
for generations. Survival ranges from approximately 10-20 years
following motor onset of the disease. There is no known cure for HD
and no approved therapies that treat the underlying cause.
About Ionis' Neurology Franchise
The Ionis neurology franchise addresses all major brain
regions and central nervous system types, currently has three
medicines in Phase 3 studies and 12 medicines in clinical
development of which three are wholly owned. Ionis is leading
the way in treating the root causes of many neurological diseases
and developing antisense medicines for common diseases, like
Alzheimer's and Parkinson's, rare diseases like ALS, Huntington's
disease, and Alexander disease. Ionis' marketed neurological
disease medicines include SPINRAZA®, the global
foundation of care for spinal muscular atrophy (SMA), and
TEGSEDI®, the first and only self-administered,
subcutaneous treatment for the polyneuropathy of hereditary ATTR
amyloidosis in adults.
About Ionis Pharmaceuticals
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.
Ionis' Forward-looking Statement
This press release
includes forward-looking statements regarding Ionis' business,
Ionis' technologies, tominersen, 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. Spinraza is commercialized by Biogen.
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SOURCE Ionis Pharmaceuticals, Inc.