CARLSBAD, Calif., Aug. 11, 2017 /PRNewswire/ -- Ionis
Pharmaceuticals, Inc. (NASDAQ: IONS) today reported that the
Company has retained all rights to inotersen and
IONIS-FB-LRx. As part of a reprioritization of its
pipeline and strategic review of its Rare Diseases business, GSK
declined its options on both drugs. Ionis plans to file for
marketing authorization for inotersen this year to support a
commercial launch of inotersen in 2018. Inotersen is a drug
designed to treat patients with TTR amyloidosis (ATTR). The first
indication Ionis is pursuing for inotersen is to treat patients
with polyneuropathy due to hereditary TTR amyloidosis
(hATTR-PN).
"We are pleased to move forward these two important drugs
ourselves. We are prepared to independently advance inotersen and
remain on track to file for marketing approval of inotersen in the
U.S. and EU this year," said B. Lynne
Parshall, chief operating officer of Ionis Pharmaceuticals.
"We want to thank our collaboration team at GSK for their support
and commitment to patients with TTR amyloidosis, and their efforts
to work closely with us to ensure a smooth transition so that this
important medicine can be available to patients as planned."
Ionis completed the Phase 3 NEURO-TTR study of inotersen in
which the drug demonstrated significant benefit on both primary
clinical endpoints of neurological disease progression and quality
of life in patients with hATTR-PN.
"Our goals for inotersen are to maximize its commercial success
and optimize our commercial participation. To achieve these goals,
we are actively considering forming a commercial subsidiary to
commercialize or co-commercialize inotersen in North America, as well as other options. Our
recent experience building a commercial subsidiary has prepared us
for this opportunity. We have substantial interest from potential
partners and are in discussions with several parties. We believe
that, together with the right commercial partner, we can maximize
the commercial success of the drug worldwide," said Sarah Boyce, chief business officer of Ionis
Pharmaceuticals.
"We are also accelerating the expansion of our TTR program for
patients with cardiomyopathy due to TTR amyloidosis and the
development of our LICA follow-on drug. Our experience in the
completed Phase 3 NEURO-TTR study provides important information to
aid in design of a study in patients with cardiomyopathy due to TTR
amyloidosis. We have already identified a more potent and
convenient LICA follow-on and we expect development of the LICA
drug to also proceed rapidly," said Stanley
T. Crooke, chairman and chief executive officer of Ionis
Pharmaceuticals. "We are deeply committed to the TTR amyloidosis
patient community. Patients with TTR amyloidosis, their families
and healthcare providers are desperately seeking improved
therapeutic options for this devastating, progressive, fatal
disease."
IONIS-FB-LRx is a ligand conjugated antisense (LICA)
drug in development for the treatment of complement-mediated
diseases. In a Phase 1 study completed earlier this year,
IONIS-FB-LRx achieved dose-dependent reductions in
plasma factor B (FB) and demonstrated a safety and tolerability
profile that supports further clinical development. Ionis plans to
initiate the first Phase 2 study with IONIS-FB-LRx in
patients with dry age-related macular degeneration (AMD) later this
year, and studies in other indications in 2018.
"IONIS-FB-LRx represents a unique opportunity to
develop a treatment for underserved rare and broad patient
populations affected by a variety of complement-mediated diseases,"
said Brett P. Monia, senior vice
president of drug discovery and franchise leader for oncology and
rare diseases at Ionis Pharmaceuticals. "IONIS-FB-LRx
takes advantage of our LICA technology, which can potentially
provide greater patient convenience by allowing for significantly
lower doses and less frequent administration."
GSK, consistent with its focus on treatments for infectious
diseases, continues to advance two drugs targeting hepatitis B
virus (HBV) under its collaboration with Ionis:
IONIS-HBVRx and IONIS-HBV-LRx. GSK is
currently conducting Phase 2 studies for both drugs.
Conference Call
At 8:30 a.m.
Eastern Time today, August 11,
2017, Ionis will conduct a live webcast conference call to
discuss this announcement. 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 patients with TTR amyloidosis (ATTR). Inotersen is
administered once weekly as a single 300 mg subcutaneous injection.
Ionis designed inotersen 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 ATTR. Inotersen
has demonstrated sustained and robust TTR reductions in clinical
studies in different populations of patients with ATTR.
Ionis has successfully completed the Phase 3 NEURO-TTR study
with inotersen in patients with polyneuropathy due to hereditary
TTR amyloidosis (hATTR-PN). Ionis plans to file for marketing
approval of inotersen in the U.S. and EU in the second half of
2017. The Company also plans to present results from the NEURO-TTR
study at an upcoming medical meeting and to submit results from the
study for publication in a peer-reviewed medical journal.
The U.S. Food and Drug Administration has granted Orphan Drug
Designation and Fast Track Status to inotersen for the treatment of
patients with familial amyloid polyneuropathy. The European
Medicines Agency has granted Orphan Drug Designation to inotersen
for the treatment of patients with TTR amyloidosis.
ABOUT TTR AMYLOIDOSIS
Transthyretin amyloidosis (ATTR)
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.
One key manifestation of ATTR results when TTR amyloid fibrils
deposit in peripheral nerves, which causes nerve damage throughout
the patient's body resulting in the progressive loss of motor
functions, such as walking. When this occurs, patients are
diagnosed with polyneuropathy due to hereditary TTR amyloidosis
(hATTR-PN). Another major manifestation of ATTR occurs when
TTR amyloid fibrils build up in heart tissue, which results in
cardiomyopathy due to TTR amyloidosis (ATTR-CM). In all
manifestations of ATTR, TTR accumulates in major organs,
progressively impacts organ function and eventually leads to death.
Therapeutic options for the treatment of ATTR are very limited and
there are currently no drugs approved for the treatment of ATTR in
the United States.
ABOUT IONIS-FB-LRx
IONIS-FB-LRx
is a generation 2.0+ ligand conjugated antisense (LICA) drug
designed to reduce the production of complement factor B (FB).
Complement factor B is produced predominately in the liver and
circulates at high levels throughout the vascular system where it
plays a pivotal role in an innate immunogenic cascade. Genetic
association studies have shown that overactivation of this cascade
has been associated with the development of several
complement-mediated diseases, including dry age-related macular
degeneration (AMD).
Ionis has completed a Phase 1 study evaluating
IONIS-FB-LRx in healthy volunteers. In the Phase 1
study, IONIS-FB-LRx achieved dose-dependent reductions
in FB and demonstrated a safety and tolerability profile that
supports further clinical development. Ionis plans to initiate the
first Phase 2 study with IONIS-FB-LRx in patients with
dry AMD later this year, and studies in other indications in
2018.
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 the U.S., Europe,
Japan and Canada for the treatment of spinal muscular
atrophy (SMA). Biogen is responsible for commercializing SPINRAZA.
Drugs that have successfully completed Phase 3 studies include
inotersen (IONIS-TTRRx), 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. Both inotersen and volanesorsen are
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' alliance with
GSK and the therapeutic and commercial potential of inotersen,
IONIS-FB-LRx, IONIS-HBVRx and
IONIS-HBV-LRx. 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.