– Olinciguat Phase II trial in sickle cell disease continues to
enroll patients –
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Jun. 7, 2018--
Ironwood
Pharmaceuticals, Inc. (NASDAQ: IRWD), a commercial biotech company,
today announced that the U.S. Food and Drug Administration (FDA) has
granted Orphan Drug Designation to olinciguat (IW-1701) for the
treatment of patients with sickle cell disease. Olinciguat is an orally
administered soluble guanylate cyclase (sGC) stimulator.
“There is an urgent need for new, innovative treatments for patients
with sickle cell disease, a debilitating and potentially fatal inherited
blood disorder that causes painful crises, organ damage and other
serious complications,” said Christopher Wright, M.D., Ph.D., senior
vice president of global development and chief development officer. “The
orphan drug designation adds momentum to our clinical program
investigating olinciguat, which has the potential to improve multiple
aspects of sickle cell disease pathophysiology. The designation is also
an important milestone in Ironwood’s evolution as we advance our
pipeline of sGC stimulators focused on the treatment of serious and
orphan diseases.”
The FDA’s Office of Orphan Drug Products grants orphan status to drugs
intended to treat rare disorders that affect fewer than 200,000 people
in the U.S. The designation provides certain benefits to the drug
developer, including seven years of market exclusivity upon FDA
approval, prescription drug user fee waivers and tax credits for
qualified clinical trials.1,2
Ironwood is currently enrolling patients in STRONG-SCD, a multicenter,
randomized, double-blind, placebo-controlled, dose-ranging Phase II
trial evaluating olinciguat for the potential treatment of sickle cell
disease. Ironwood expects to enroll approximately 80 patients into the
Phase II trial, which is designed to evaluate the safety, tolerability,
pharmacokinetics and pharmacodynamics of olinciguat in patients with
sickle cell disease. Further details about the trial can be found at clinicaltrials.gov
using the identifier number NCT03285178.
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 Olinciguat
Olinciguat (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.
Olinciguat 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, olinciguat 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 praliciguat (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, olinciguat (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
persistent 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.
Forward-Looking Statements
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 olinciguat, including the design, size, scope and
potential results of the Phase II clinical trial; the mechanism of
action of olinciguat; the size of the potential patient population; the
data to be generated from the Phase II clinical trial; the cause of the
disease and the symptoms suffered by the potential patient population;
and olinciguat 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 olinciguat or that we are not able to successfully
protect such patents; the outcomes in legal proceedings to protect or
enforce the patents relating to olinciguat; 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
olinciguat 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 March 31, 2018, 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.
1 “Designating an Orphan Product: Drugs and Biological
Products.” US Food and Drug Administration. https://www.fda.gov/ForIndustry/DevelopingProductsforRareDiseasesConditions/HowtoapplyforOrphanProductDesignation/default.htm.
Published February 16, 2018. Accessed June 6, 2018.
2
“FDA at Rare Disease Day / February 28, 2011.” US Food and Drug
Administration. https://www.fda.gov/ForIndustry/DevelopingProductsforRareDiseasesConditions/ucm239698.htm.
Published November 3, 2017. Accessed June 6, 2018.
View source version on businesswire.com: https://www.businesswire.com/news/home/20180607006163/en/
Source: Ironwood Pharmaceuticals, Inc.
Ironwood Pharmaceuticals, Inc.
Meredith Kaya, 617-374-5082
Vice
President, Investor Relations and Corporate Communications
mkaya@ironwoodpharma.com