CARLSBAD, Calif., Oct. 15, 2017 /PRNewswire/ -- Ionis
Pharmaceuticals, Inc. (NASDAQ: IONS), the leader in antisense
therapeutics, today announced that new data from the Phase 3
NEURO-TTR study with inotersen in patients with hereditary TTR
amyloidosis (hATTR) with polyneuropathy were presented at the
142nd annual meeting of the American Neurological
Association (ANA) in San Diego,
California. In the study, inotersen-treated patients
achieved a mean 19.73-point benefit in the mNIS+7 co-primary
endpoint after 15 months of treatment, compared to placebo-treated
patients (p = 0.00000004), further demonstrating the clinically
meaningful benefit of inotersen treatment. A statistically
significant benefit in mNIS+7 was also observed at eight months.
Key safety findings of thrombocytopenia and renal events identified
during the study were shown to be monitorable and manageable with
routine blood and urine testing. Multiple other innovative drugs
from Ionis' portfolio of drugs for patients with serious
neurological and neurodegenerative diseases will be highlighted as
part of the Antisense Oligonucleotide Treatment of Genetic
Neurological Diseases plenary session on Tuesday, October 17.
"The pivotal inotersen data presented today represent new hope
for patients suffering with hATTR. A 20-point benefit in mNIS+7 is
unprecedented and could mean the difference between the ability to
walk and being confined to a wheelchair for patients suffering from
this debilitating and fatal disease," said Annabel Wang, associate professor of neurology
at the University of California,
Irvine, School of Medicine. "Based on benefit observed in
neurological and quality of life endpoints, inotersen treatment has
shown the potential to effectively change the relentless
progression of this disease and offers a convenient, at-home
administration method, providing patients further liberation from
the burden of hATTR."
In addition to the inotersen program update, Ionis antisense
programs will be featured in eleven platform and poster
presentations throughout the meeting, including:
- Highlights from the discovery and development program for
SPINRAZA® (nusinersen), the first and only drug approved
for the treatment of spinal muscular atrophy.
- Development overviews for IONIS-HTTRx for the
treatment of patients with Huntington's disease and
IONIS-MAPTRx for the treatment of patients with
tauopathies, including Alzheimer's disease and frontotemporal
dementia.
- Presentations featuring Ionis' antisense programs designed to
treat amyotrophic lateral sclerosis, Parkinson's disease and
Alexander disease.
"Ionis' leadership in neurodegeneration is well highlighted at
ANA. The programs featured at this medical meeting presented by us
and our collaborators validate the breadth and potential impact of
our neurological disease franchise. We are rapidly expanding and
advancing our neurological disease pipeline, adding more innovative
drugs, each with the potential to transform the treatment of severe
neurological and neurodegenerative disease," said C. Frank Bennett, PhD, senior vice president of
research and leader of the neurological disease franchise at Ionis
Pharmaceuticals. "We are pleased with the impact SPINRAZA has
already made on the SMA community. We are now applying what we
learned from SPINRAZA to our other neurological disease drugs in
development."
Ionis antisense programs will be featured in the following
platform and poster presentations at the 2017 ANA congress:
- 'Safety and Efficacy of Inotersen in Patients with Hereditary
Transthyretin Amyloidosis with Polyneuropathy (NEURO-TTR)' oral
presentation on Tuesday, October 17
by Annabel Wang, MD, University of California, Irvine
- 'Antisense Oligonucleotide Therapy in ALS, Huntington's Disease
and Beyond' oral presentation on Tuesday,
October 17 by Don Cleveland, PhD, Chair, Department of
Cellular and Molecular Medicine, University of
California, San Diego
- 'ASO Therapy for SMA: Harnessing the Power of a Backup Gene'
oral presentation on Tuesday, October
17 by Adrian R. Krainer, PhD,
Cold Spring Harbor Laboratory and ANA's 2017 F.E. Bennett Memorial
Lecture Award Recipient
- 'Getting the Message: Antisense Oligonucleotide Therapy for
Duchenne Muscular Dystrophy and Spinal Muscular Atrophy' oral
presentation on Tuesday, October 17
by Richard Finkel, MD, FANA, Nemours
Children's Hospital
- 'Antisense Oligonucleotide Therapy for Huntington's Disease: A
Clinical Trials Perspective' oral presentation on Tuesday, October 17 by Sarah J. Tabrizi, MBChB, FRCP, PhD, FMedSci, UCL
Huntington's Disease
- 'Rationale for and Development of IONIS-MAPTRx, the
First Tau-lowering Antisense Oligonucleotide, in Patients
with Mild AD' poster presentation on Monday,
October 16 by Laurence Mignon, Ionis Pharmaceuticals
- 'ASO Lowering of SOD1 Markedly Extends Survival and Reverses
Muscle Denervation in SOD1 ALS Rodent Models' oral presentation on
Monday, October 16 by Timothy Miller, MD, PhD, Washington University in St. Louis
- 'Modeling C9ORF72 Disease: A Crucial Step for Therapeutic
Development in ALS and Frontotemporal Dementia' oral presentation
on Sunday, October 15 by Clotilde
Lagier-Tourenne, MD, PhD, Massachusetts
General Hospital
- 'LRRK2 Antisense Oligonucleotides Ameliorate -Synuclein
Inclusion Formation and Provide Neuroprotection in a Parkinson's
Disease Mouse Model' poster presentation on Sunday, October 15 by Hien Tran Zhao, PhD, Ionis Pharmaceuticals
- 'Snca Targeted Antisense Oligonucleotides Modulate Progression
of Pathological Deposition in Alpha Synuclein Rodent Transmission
Models of Parkinson's Disease' poster presentation on Sunday, October 15 by Tracy Cole, PhD, Ionis Pharmaceuticals
- 'Antisense Suppression of GFAP as a Therapeutic Strategy for
Alexander Disease' poster presentation on Sunday, October 15 by Berit Powers, PhD, Ionis Pharmaceuticals
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) has
been approved in global markets for the treatment of spinal
muscular atrophy (SMA). Biogen is responsible for commercializing
SPINRAZA. Drugs that have successfully completed Phase 3 studies
include inotersen, an antisense drug Ionis is developing to treat
patients with TTR amyloidosis, and volanesorsen, an antisense drug
discovered by Ionis and co-developed by Ionis and Akcea
Therapeutics to treat patients with either familial chylomicronemia
syndrome or familial partial lipodystrophy. Akcea, an affiliate of
Ionis, is a biopharmaceutical company focused on developing and
commercializing drugs to treat patients with serious
cardiometabolic diseases caused by lipid disorders. If approved,
volanesorsen will be commercialized through Ionis' affiliate,
Akcea. Volanesorsen filings for marketing approval have been
submitted in the U.S., EU and Canada. Inotersen is progressing toward
regulatory filings for marketing authorization. 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' business and
the therapeutic and commercial potential of SPINRAZA, inotersen,
IONIS-HTTRx, IONIS-MAPTRx 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, 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.