CARLSBAD, Calif., March 22, 2021 /PRNewswire/ -- Ionis
Pharmaceuticals, Inc. (NASDAQ: IONS) announced its partner, Roche,
has decided to discontinue dosing in the Phase III GENERATION HD1
study of tominersen in manifest Huntington's disease (HD). The
decision was based on the results of a pre-planned review of data
from the Phase III study conducted by an unblinded Independent Data
Monitoring Committee (iDMC). While there were no new or emerging
safety signals identified for tominersen, the iDMC made its
recommendation based on the investigational therapy's potential
benefit/risk profile for study participants. Participants will
continue to be followed for safety and clinical outcomes.
"We are very disappointed by this news," said Brett Monia, Ph.D., Ionis CEO. "This of course
is not the outcome we have been working towards or hoped for, as we
have grown quite close to the HD patient community over the years.
We are committed to finding treatments for this devastating disease
and want to thank the patients, their families and the entire HD
community for their partnership, trust and dedication. We look
forward to learning more once Roche has evaluated the full
data."
In addition, dosing will be paused in the open-label extension
study (GEN-EXTEND) of tominersen while data are carefully analyzed
to inform next steps on this study. Roche also stated that the
Phase I PK/PD study (GEN-PEAK) of tominersen and Roche's
observational HD Natural History Study will continue.
Roche has stated that once full data from the studies are
available and analyzed, Roche will share learnings and future plans
with the community.
About tominersen and the clinical trials
Tominersen, previously IONIS-HTTRx or RG6042, is an
investigational antisense therapy 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 molecule from Ionis.
Tominersen is being investigated in Huntington's disease in the
following clinical studies:
- GENERATION HD1: a randomized, multicenter, double-blind,
placebo-controlled Phase III 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 intrathecal injections
of tominersen, or placebo. The study has recruited 791 participants
from 18 countries around the world.
- GEN-EXTEND: an open label extension study for participants
coming from any Roche HD study. Participants receive 120 mg
tominersen every two months or every four months in the study.
- 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 a range of doses from 30 mg to 120 mg of tominersen over
two administrations.
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.
Ionis will hold a webcast on Monday,
March 22, 5 p.m. ET to discuss
this update. Visit www.ionispharma.com for more
information and to register.
About Ionis' Neurology Franchise
The Ionis neurology franchise addresses all major brain
regions and central nervous system types, currently has three Phase
3 studies, eight medicines in clinical development and five 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), commercialized by Biogen, 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, which is on
file with the SEC. Copies of this 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.
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SOURCE Ionis Pharmaceuticals, Inc.