CAMBRIDGE, Mass.--(BUSINESS WIRE)--
Ironwood
Pharmaceuticals, Inc. (NASDAQ: IRWD), a commercial biotechnology
company, today announced the initiation of STRONG-SCD, a Phase II
clinical trial evaluating once-daily IW-1701, an orally administered
soluble guanylate cyclase (sGC) stimulator, in patients with sickle cell
disease.
Sickle cell disease is a debilitating genetic disorder that causes red
blood cells to become sickle-shaped, reducing normal red blood cell
number, blood flow and oxygen delivery to organs. Many of the symptoms
of sickle cell disease are believed to be caused by a deficiency of
nitric oxide (NO), an important regulator of blood flow and inflammatory
processes. IW-1701 has been shown to improve NO signaling in
non-clinical studies, and is being investigated for its potential to
treat multiple aspects of sickle cell disease pathophysiology, including
red blood cell sickling, decreased blood flow and vascular inflammation.
"There is a considerable unmet need in sickle cell disease, and patients
with this condition would benefit from innovative treatments that not
only prevent painful crises, organ damage and other serious
complications, but also address underlying causes of these symptoms,"
said Christopher Wright, MD, PhD, senior vice president of global
development and chief development officer at Ironwood. "By stimulating
the sGC pathway, we believe IW-1701 has the potential to improve NO
signaling and address the causes of both acute and chronic sickle cell
disease symptoms."
STRONG-SCD is a multicenter, randomized, double-blind,
placebo-controlled, dose-ranging Phase II clinical trial designed to
assess the safety and tolerability of oral, once-daily IW-1701 in
patients with sickle cell disease. Additional exploratory objectives
include the evaluation of the pharmacokinetic profile of IW-1701 as well
as its effects on sickle cell disease symptoms, health-related quality
of life and biomarkers of pharmacodynamic activity. The trial is
expected to enroll approximately 88 patients between 16 and 70 years of
age. Patients will remain on their existing treatment regimens
throughout the trial. After a two-week single-blind placebo run-in
period, patients will be randomized to receive placebo or one of three
dose levels of IW-1701 administered for approximately 12 weeks.
Ironwood recently presented IW-1701 data at the 2017 American Society of
Hematology (ASH) Annual Meeting. In an oral presentation entitled "The
Clinical-Stage sGC Stimulator IW-1701 Prevents Increase of Plasma
Biomarkers of Intravascular Inflammation and Suppresses
Leukocyte-Endothelial Interactions in TNFα-Treated Mice," non-clinical
data were presented demonstrating that, in these models, pre-treatment
with IW-1701 inhibited the intravascular inflammation typically caused
by TNFα, and that the effect of IW-1701 on inflammation was enhanced
when co-administered with hydroxyurea, the current standard of care for
sickle cell disease. In addition, Ironwood presented data from a
randomized, placebo-controlled Phase Ib multiple ascending dose study of
IW-1701 tablets in healthy subjects. Data from both of these studies
support the continued clinical evaluation of IW-1701 in sickle cell
disease.
About Sickle Cell Disease
Sickle cell disease is an inherited red blood cell disorder that causes
red blood cells to deform into a sickle shape, impacting blood flow to
organs and tissues.These sickled red blood cells are more
susceptible to hemolysis (rupturing). Upon red blood cell rupturing,
nitric oxide (NO) is depleted due to arginase release and hemoglobin
scavenging. NO is an important regulator of blood flow,and
the resulting deficiency of NO is believed to contribute to disease
mechanisms and symptoms of sickle cell disease.
A life-long disease, sickle cell disease affects an estimated 100,000
Americans and millions of people throughout the world. Patients with
sickle cell disease can experience various complications, including
attacks of severe pain called pain crises, chronic pain, acute chest
syndrome, pulmonary hypertension, ankle ulcers, renal complications and
an increased risk of serious infections. Severe symptoms may include
strokes and pulmonary complications, which can be fatal.
About IW-1701
IW-1701, an investigational soluble guanylate cyclase (sGC) stimulator
discovered and wholly-owned by Ironwood, is being studied in patients
with sickle cell disease and in patients with achalasia. IW-1701 has
been shown in non-clinical studies to modulate the nitric oxide/soluble
guanylate cyclase/cyclic guanosine monophosphate (NO/sGC/cGMP) signaling
pathway, which is believed to be implicated in achalasia and sickle cell
disease. Currently in Phase II development for sickle cell disease and
for achalasia, IW-1701 has the potential to address the underlying
causes of these diseases by improving NO signaling and thereby
increasing the second messenger cGMP.
About Ironwood's sGC Program
As a pioneering expert in cyclic GMP (cGMP), Ironwood is building on its
success with linaclotide, which stimulates guanylate cyclase-C in the
intestine, to develop a pipeline of soluble guanylate cyclase (sGC)
stimulators. sGC plays an important role in regulating diverse
physiological processes; dysregulation of sGC may play a role in
multiple serious diseases. Ironwood's sGC stimulators are believed to
harness the nitric oxide (NO)/sGC/cGMP pathway by working
synergistically with NO to improve blood flow and metabolism and
decrease inflammation and fibrosis.
Ironwood is advancing IW-1973, its lead sGC stimulator, for the
potential treatment of diabetic nephropathy and of heart failure with
preserved ejection fraction (HFpEF). Ironwood's second clinical sGC
stimulator, IW-1701, is being developed for the potential treatment of
achalasia and of sickle cell disease. In addition, Ironwood has a
pipeline of other sGC stimulators in pre-clinical development.
About Ironwood Pharmaceuticals
Ironwood Pharmaceuticals (NASDAQ: IRWD) is a commercial biotechnology
company focused on creating medicines that make a difference for
patients, building value for our fellow shareholders, and empowering our
passionate team. We are commercializing two innovative primary care
products: linaclotide, the U.S. branded prescription market leader for
adults with irritable bowel syndrome with constipation (IBS-C) or
chronic idiopathic constipation (CIC), and lesinurad, which is approved
to be taken with a xanthine oxidase inhibitor (XOI), or as a fixed-dose
combination with allopurinol, for the treatment of hyperuricemia
associated with gout. We are also advancing a pipeline of innovative
product candidates in areas of significant unmet need, including
uncontrolled gastroesophageal reflux disease, diabetic nephropathy,
heart failure with preserved ejection fraction, achalasia and sickle
cell disease. Ironwood was founded in 1998 and is headquartered
in Cambridge, Mass. For more information, please visit www.ironwoodpharma.com
or www.twitter.com/ironwoodpharma;
information that may be important to investors will be routinely posted
in both these locations.
This press release contains forward-looking statements. Investors are
cautioned not to place undue reliance on these forward-looking
statements, including statements about Ironwood's sGC program and the
clinical program for IW-1701, including the design, size, scope and
potential results of the Phase II clinical trial; the mechanism of
action of IW-1701; the size of the potential patient population; the
data to be generated from the Phase II clinical trial; the timing that
these data are expected to be available; the cause of the disease and
the symptoms suffered by the potential patient population; and IW-1701
as a potential treatment for sickle cell disease. Each
forward‐looking statement is subject to risks and uncertainties that
could cause actual results to differ materially from those expressed or
implied in such statement. Applicable risks and uncertainties include
those related to the risk that we are unable to enroll as many patients
in the clinical study or complete the Phase II clinical trial on the
same timeline as we currently anticipate; the risk that the data from
the clinical trial may not be available when we currently anticipate
them or do not demonstrate the results we expect, including with respect
to efficacy, safety and tolerability; the risk that the Phase II
clinical trial needs to be discontinued for any reason, including
safety, enrollment, manufacturing or economic reasons; the patient
population is not as large as we presently estimate; the effectiveness
of development and commercialization efforts by us and our partners;
preclinical and clinical development, manufacturing and formulation
development; the risk that findings from our completed nonclinical and
clinical studies may not be replicated in later studies; decisions by
regulatory authorities; the risk that we may never get sufficient patent
protection for IW-1701 or that we are not able to successfully protect
such patents; the outcomes in legal proceedings to protect or enforce
the patents relating to IW-1701; developments in the intellectual
property landscape; challenges from and rights of competitors or
potential competitors; the risk that our planned investments do not have
the anticipated effect on our business or the IW-1701 program; and those
risks listed under the heading "Risk Factors" and elsewhere in
Ironwood's Quarterly Report on Form 10-Q for the quarter ended September
30, 2017, and in our subsequent SEC filings. These forward-looking
statements (except as otherwise noted) speak only as of the date of this
press release, and Ironwood undertakes no obligation to update these
forward-looking statements.
View source version on businesswire.com: http://www.businesswire.com/news/home/20171221005133/en/
Investor and Media Relations
Ironwood Pharmaceuticals
Meredith
Kaya, 617-374-5082
Senior Director, Investor Relations and
Corporate Communications
mkaya@ironwoodpharma.com
Source: Ironwood Pharmaceuticals, Inc.
News Provided by Acquire Media