CARLSBAD, Calif., May 15, 2017 /PRNewswire/ -- Ionis
Pharmaceuticals, Inc. (NASDAQ: IONS) announced today that the Phase
3 NEURO-TTR study of inotersen (IONIS-TTRRx) in patients
with familial amyloid polyneuropathy (FAP) met both primary
endpoints. Over the 15-month period of the study, inotersen-treated
patients achieved statistically significant benefit compared to
placebo in the modified Neuropathy Impairment Score +7 (mNIS+7) and
the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy
(Norfolk QoL-DN) (p<0.0001 and p=0.0006, respectively).
Statistically significant differences were also observed for both
endpoints at eight months.
"Familial amyloid polyneuropathy is a devastating genetic
disease that is painful and rapidly progressive leading to early
death. The positive results from the NEURO-TTR study today are very
encouraging for this underserved patient population," said
Morie Gertz, MD, M.A.C.P., Division
of Hematology, Roland Seidler Jr.
Professor Department of Medicine, College of Medicine, Mayo
Distinguished Clinician. "I have been treating patients with this
disabling disease for many years, and I am excited about the
promise that inotersen holds to restore their lives. I believe
inotersen has the potential to transform the current standard of
care for patients with TTR amyloidosis."
Treatment-emergent adverse events considered related to
treatment were seen more commonly with inotersen than placebo. Two
key safety findings were observed during the study that required
changes to the monitoring schedule. Three serious adverse events of
thrombocytopenia were observed in inotersen-treated patients; two
patients recovered and one patient died due to intracranial
hemorrhage. One additional inotersen-treated patient discontinued
treatment due to non-serious thrombocytopenia. Four
inotersen-treated patients discontinued treatment due to a renal
observation; two patients met a predefined renal stopping rule and
two experienced serious renal adverse events, one of whom
experienced chronic renal insufficiency. One placebo-treated
patient also met a predefined renal stopping rule. Enhanced
monitoring was implemented during the study to support early
detection and management of the thrombocytopenia and renal issues.
All five serious adverse events occurred before enhanced monitoring
was fully implemented. A detailed review of safety data from the
study is ongoing.
"Data showing symptomatic patients experience a quality of life
benefit is truly exciting and provides hope for the amyloidosis
community. We are excited by this very important milestone and
welcome the opportunity of inotersen to potentially address the
unmet and critical needs of amyloidosis patients and transform
lives," said Isabelle Lousada,
president and chief executive officer of the Amyloidosis Research
Consortium (ARC). "The ARC is committed to building collaborative
relationships to advance the best research and speed new therapies
to market for this disease that has truly devastated generations of
affected families. With no FDA-approved drugs, treatment options
are extremely limited and desperately needed."
"We are grateful to the patients who participated in the
NEURO-TTR study, along with their families, the investigators and
the broader TTR amyloidosis community, for their dedication,
commitment and support. We share the collective sense of urgency to
bring a new treatment to patients and their families facing this
devastating disease," said Brett P.
Monia, senior vice president of drug discovery and franchise
leader for oncology and rare diseases at Ionis Pharmaceuticals. "We
are excited about the positive topline results from the Phase 3
NEURO-TTR study. We observed a benefit in disease progression in
patients treated with inotersen, regardless of disease stage (Stage
1 and Stage 2) or TTR mutation (V30M and non-V30M). We believe
these preliminary results suggest a favorable benefit-risk profile
for inotersen in patients with FAP."
Long-term safety and efficacy data with inotersen are currently
being collected in an open-label extension of the Phase 3 NEURO-TTR
study. More than 80% of patients completed the NEURO-TTR study, of
these more than 95% participated in the open-label extension
study.
Review of the full data package from the NEURO-TTR study by
Ionis and GSK is ongoing and detailed results from the study will
be presented at an upcoming medical meeting and submitted for
publication in a peer-reviewed medical journal.
The preparation of regulatory marketing applications for
inotersen is underway. GSK has the option to license inotersen
following review of additional data and prior to the submission of
regulatory applications.
WEBCAST INFORMATION
Interested parties may listen to the call by dialing
877-443-5662 or access the webcast at www.ionispharma.com. A
webcast replay will be available for a limited time at the same
address.
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.
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. for the treatment of spinal muscular atrophy (SMA) in
pediatric and adult patients. Biogen is responsible for
commercialization of SPINRAZA. Drugs currently in Phase 3
development 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, a drug Ionis is
developing with GSK to treat patients with TTR amyloidosis. 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 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. Akcea Therapeutics™ is a trademark of Ionis Pharmaceuticals,
Inc. SPINRAZA® is a registered trademark of Biogen.
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SOURCE Ionis Pharmaceuticals, Inc.