CARLSBAD, Calif., July 10, 2017 /PRNewswire/ -- Ionis
Pharmaceuticals, Inc. (NASDAQ: IONS) presented new top-line data
from the Phase 3 NEURO-TTR study of inotersen in patients with
familial amyloid polyneuropathy (FAP) at the 2017 Peripheral Nerve
Society Meeting. Results from the study demonstrated benefit across
both primary endpoints of the study: modified Neuropathy Impairment
Score +7 (mNIS+7) and Norfolk Quality of Life
Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN) at 15 months of
treatment. Inotersen-treated patients benefited significantly in
quality of life compared to placebo, with a difference in magnitude
of 11.68 points in the Norfolk QoL-DN score at 15 months of
treatment (mean change from baseline of 0.99 vs. 12.67, p=0.0006).
Statistically significant benefit was also observed for both mNIS+7
and Norfolk QoL-DN at eight months of treatment with inotersen. The
results continue to support the favorable benefit-risk profile for
inotersen in patients with FAP. A detailed review of efficacy and
safety from the study is ongoing.
"The magnitude of benefit on quality of life achieved with
inotersen treatment has never been observed before in patients with
FAP," said Teresa Coelho, MD,
neurologist and neurophysiologist at Santo António Hospital,
Porto, Portugal. "As their disease
progresses, FAP patients lose the ability to do even simple
activities like feeding themselves, which can limit their
independence. They also suffer from debilitating symptoms, like
severe extremity pain and diarrhea. The quality of life score
assesses almost every aspect of this devastating disease on a
patient's life. The nearly 12-point difference observed with
inotersen treatment from placebo represents a clinically meaningful
benefit and provides hope that inotersen could allow FAP patients
to maintain greater independence by alleviating debilitating
symptoms and preserving their ability to perform daily
activities."
"The clinically meaningful benefit in quality of life bring a
new energizing level of hope to the amyloidosis community around
the world, a community whose critical needs have not been
sufficiently addressed," said Isabelle
Lousada, president and chief executive officer of the
Amyloidosis Research Consortium (ARC). "Going from Phase 1 to
Phase 3, inotersen's development program hallmarks the importance
of rapidly advancing valuable, life-changing therapies that can
have transformational benefits on patients' lives. We hope for a
rapid approval process for inotersen to create access and
availability of this important medicine, which has the potential to
help patients maintain their independence and provide a brighter
future for patients who suffer from this desperate and devastating
disease."
The Phase 3 NEURO-TTR study met both its primary endpoints,
Norfolk QoL-DN and mNIS+7, with high statistical significance.
Statistical significance was observed regardless of TTR mutation
(V30M vs. Non-V30M) or disease severity (Stage 1 vs. Stage 2). The
Norfolk QoL-DN measures patients' perception of symptoms associated
with specific nerve fiber damage. The questionnaire includes
questions assessing physical function, disease symptoms, activities
of daily living, physical sensation and autonomic neuropathy. The
mNIS+7 is a composite score that includes measures of muscle
strength, quantitative sensation testing and nerve conduction.
- Treatment with inotersen resulted in a statistically
significant benefit in Norfolk QoL-DN score compared to placebo of
6.14 points at 8 months of treatment (mean change from baseline of
0.81 vs. 6.95, p=0.032) and 11.68 points at 15 months of treatment
(mean change from baseline of 0.99 vs. 12.67, p=0.0006).
- Treatment with inotersen resulted in a statistically
significant benefit in mNIS+7 compared to placebo at eight months
of treatment (p=0.0005) and at 15 months of treatment
(p=0.00000004).
- As previously identified, the key safety findings in the
NEURO-TTR study were thrombocytopenia and renal dysfunction.
Enhanced platelet and renal monitoring have effectively managed
these safety events since implementation in the NEURO-TTR
study.
"The new top-line data presented today reaffirm inotersen's
effect in patients with FAP. Importantly, we observed
statistically significant disease benefit with inotersen treatment
at only eight months of treatment, and this benefit was further
amplified at the end of study analysis. We are very encouraged by
the significant benefit demonstrated in quality of life, and, these
results further support a strong relationship between patients'
perception of benefit in quality of life and clinical measurements
of neurological disease progression," said Brett Monia, senior vice president of drug
discovery and franchise leader for oncology and rare diseases at
Ionis Pharmaceuticals. "We are continuing to review the full data
package from the NEURO-TTR study and prepare the regulatory
marketing applications for submission this year. We look forward to
sharing detailed results in future publications and medical
meetings."
ABOUT INOTERSEN
Inotersen (IONIS-TTRRx) is
a generation 2.0+ antisense drug Ionis is developing for the
treatment of TTR amyloidosis. Inotersen is administered once
weekly as a single 300 mg subcutaneous injection. The drug is
designed to inhibit the production of all forms of TTR protein,
including both the hereditary and wild-type forms, offering a
unique approach to treat all types of TTR amyloidosis. Inotersen
has demonstrated sustained and robust TTR reductions in clinical
studies in different populations of patients with TTR-related
amyloidosis.
The U.S. Food and Drug Administration has granted Orphan Drug
Designation and Fast Track Status to inotersen for the treatment of
patients with FAP. The European Medicines Agency has granted Orphan
Drug Designation to inotersen for the treatment of patients with
TTR amyloidosis.
GSK has the option to license inotersen prior to the submission
of regulatory applications.
ABOUT TTR AMYLOIDOSIS – FAP
FAP, now referred to as
hereditary transthyretin amyloidosis with polyneuropathy
(hATTR-PN), is a progressive, debilitating and fatal genetic
disease in which patients experience TTR build up in major organs,
including peripheral nerves, heart, intestinal tract, kidney and
bladder.
Patients with hATTR-PN primarily experience nerve damage
throughout their body resulting in the progressive loss of motor
functions, such as walking. As TTR accumulates in major organs, it
progressively impacts organ function and eventually leads to death.
Therapeutic options for the treatment of hATTR-PN are very limited
and there are currently no drugs approved for the treatment of
hATTR-PN in the United States.
There are an estimated 10,000 hATTR-PN patients worldwide.
ABOUT THE NEURO-TTR PHASE 3 STUDY
Inotersen was
evaluated in a Phase 3 randomized (2:1), double-blind,
placebo-controlled, international study in 172 patients with
hATTR-PN. The study was designed to support an application for
marketing approval of inotersen in patients with hATTR-PN. The
15-month study measured the effects of inotersen on neurological
dysfunction and on quality-of-life by measuring the change from
baseline in the modified Neuropathy Impairment Score +7 (mNIS+7)
and in the Norfolk Quality of Life Questionnaire-Diabetic
Neuropathy (Norfolk QOL-DN) total score. For further study
information, please visit www.clinicaltrials.gov and search for the
identifier number NCT01737398.
ABOUT IONIS PHARMACEUTICALS, INC.
Ionis is the leading
company in RNA-targeted drug discovery and development focused on
developing drugs for patients who have the highest unmet medical
needs, such as those patients with severe and rare diseases. Using
its proprietary antisense technology, Ionis has created a large
pipeline of first-in-class or best-in-class drugs, with over three
dozen drugs in development. SPINRAZA® (nusinersen) is a
drug that has been approved in the U.S. and Europe for the treatment of spinal muscular
atrophy (SMA) in pediatric and adult patients. Biogen is
responsible for commercializing SPINRAZA. Drugs that have
successfully completed Phase 3 studies include volanesorsen, a drug
Ionis is developing and plans to commercialize through its
subsidiary, Akcea Therapeutics, to treat patients with either
familial chylomicronemia syndrome or familial partial
lipodystrophy; and inotersen (IONIS-TTRRx), a drug Ionis
is developing with GSK to treat patients with TTR amyloidosis. Both
drugs are progressing toward regulatory filings in the second half
of 2017. Ionis' patents provide strong and extensive protection for
its drugs and technology. Additional information about Ionis is
available at www.ionispharma.com.
IONIS' FORWARD-LOOKING STATEMENT
This press release
includes forward-looking statements regarding Ionis' alliance with
GSK and the therapeutic and commercial potential of 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, 2016, and its most recent quarterly
report on Form 10-Q, 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. Akcea Therapeutics™ is a trademark of Ionis Pharmaceuticals,
Inc. SPINRAZA® is a registered trademark of Biogen.
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SOURCE Ionis Pharmaceuticals, Inc.