– First prospective trial of a glucagon-like
peptide (GLP-2) analog in SR GI aGVHD –
– Results up to Day 91 show that apraglutide in
SR GI aGVHD was well-tolerated with an acceptable safety profile,
the study’s primary objective –
– Exploratory secondary efficacy data showed
lower GI tract responses maintained through Day 91 in SR GI aGVHD
patients treated with apraglutide and standard of care –
Ironwood Pharmaceuticals, Inc. (Nasdaq: IRWD), a
GI-focused healthcare company, today announced positive, primary
results up to Day 91 for its Phase II exploratory STARGAZE trial
evaluating apraglutide in patients with steroid-refractory
gastrointestinal acute Graft-versus-Host Disease (SR GI aGVHD). The
primary objective of the trial was to evaluate the safety and
tolerability of once-weekly apraglutide, a next-generation,
long-acting glucagon-like peptide-2 (GLP-2) analog, in SR GI aGVHD
patients treated with standard of care, including systemic
corticosteroids and ruxolitinib. This was a first-of-its-kind study
combining a GLP-2 analog with immunosuppressive therapy in patients
with SR GI aGVHD.
The safety and tolerability observed in this trial was
consistent with the standard of care and known safety of GLP-2
analogs, as well as observed events in the GVHD population. The
emerging safety profile of apraglutide in the SR GI aGVHD
population was monitored by an independent safety review committee
that endorsed the continuation of the study without
modification.
In addition to evaluating safety, secondary endpoints evaluated
efficacy using lower GI and all-organ responses, indicative of
organ-level improvement from baseline per the Mount Sinai Acute
GVHD International Consortium (MAGIC) grading system. The majority
of patients responded to treatment by Day 28 and Day 56. All
lower GI responders at Day 28 maintained their response through
Days 56 and 91, considering available organ staging
assessments.
“The findings presented here are an important step
forward for patients with SR GI aGVHD given limited duration of
response with currently approved agents,” said Robert Zeiser, M.D.,
Professor of Hematology and Oncology at the University of Freiburg
and Principal Investigator for the STARGAZE study. “Given its
potential GI effect on tissue regeneration and function,
apraglutide may have a role in the advanced management of acute GI
GVHD through a non-immunosuppressive approach, and we look forward
to longer-term findings in this patient population.”
aGVHD is a rare, life-threatening condition that occurs when
immune cells of the donor (the graft) attack a recipient’s (host)
healthy cells after an allogeneic hematopoietic stem cell
transplant (HSCT). The condition typically affects the skin, GI
tract, and liver. GI aGVHD, is a leading cause of morbidity and
mortality following HSCT with approximately 70% of aGVHD cases
having GI involvement. While treatment options exist for aGVHD, a
significant number of patients do not respond to 1st line therapy,
such as corticosteroids. Despite receiving 2nd line therapy, many
patients also do not maintain response over time, with significant
non-relapse mortality (NRM) after six months.
“These results from the first-of-its-kind STARGAZE trial in a
population of patients with SR GI aGVHD add to the body of evidence
about the safety and tolerability profile of apraglutide,” said
Michael Shetzline, M.D., Ph.D., Senior Vice President, Chief
Medical Officer and Head of Research and Development at Ironwood
Pharmaceuticals. “Other studies have shown that GLP-2 promotes
intestinal healing and regeneration, and we look forward to further
understanding the data from this trial given the high unmet medical
need of these patients who suffer from high morbidity and
mortality.”
Additional data from the STARGAZE trial are expected to be
presented at an upcoming medical congress. The STARGAZE study will
continue through its two-year endpoint, where apraglutide will be
re-evaluated for safety and efficacy.
About STARGAZE
STARGAZE (Study of Apraglutide in Graft-versus-Host Disease) is
a randomized, single-blind, Phase II exploratory trial designed to
evaluate weekly dosing of apraglutide in combination with standard
of care, including systemic corticosteroids and ruxolitinib in
patients with steroid-refractory GI aGVHD for up to 90 days with a
follow-up period lasting up to two years after the first dose.
STARGAZE is the first prospective trial of a glucagon-like peptide
(GLP-2) analog focusing on patients with steroid-refractory GI
aGVHD. The trial enrolled 31 patients and at this primary analysis,
evaluated them for safety and tolerability, pharmacokinetics, and
efficacy, including response rate, duration of response, and
survival-related outcomes. This study’s population consisted of
more than 87% Grade III and IV patients, the most severe cohort as
measured by the Mount Sinai Acute Graft Versus Host Disease
International Consortium (MAGIC) scale.
About Acute Graft-Versus-Host Disease (aGVHD)
Acute Graft-Versus-Host Disease (aGVHD) is a severe condition
which occurs when immune cells from the donor attack a recipient’s
healthy cells after an allogeneic hematopoietic stem cell
transplant (HSCT), the standard last-line treatment for many forms
of blood cancer. aGVHD typically emerges within the first 100 days
post-transplant and predominantly affects the skin,
gastrointestinal (GI) tract and liver. GI GvHD is one of the
leading causes of morbidity and mortality following HSCT. The
predominant manifestations of GI GVHD are abdominal pain and
diarrhea. The diarrhea is secretory, occurs independently of oral
intake and can be profound and incessant, with up to several liters
of output and more than 10 episodes per day, often requiring
parenteral support to feed and hydrate patients. More than 26,000
allogeneic HSCTs take place in the U.S., Europe and Japan annually
and aGVHD occurs in an estimated 30-50% of patients. While
treatment options exist for aGVHD, a significant number of patients
do not respond to 1st line therapy, such as corticosteroids.
Despite receiving 2nd line therapy, many patients also do not
maintain response over time, with significant non-relapse mortality
(NRM) after six months.
About Apraglutide
Apraglutide is an investigational, next-generation, long-acting
synthetic GLP-2 analog being developed for a range of rare
gastrointestinal diseases where GLP-2 can play a central role in
addressing disease pathophysiology, including short bowel syndrome
with intestinal failure (SBS-IF) and Acute Graft-Versus-Host
Disease (aGVHD).
About Ironwood Pharmaceuticals
Ironwood Pharmaceuticals (Nasdaq: IRWD), an S&P SmallCap
600® company, is a leading global gastrointestinal (GI) healthcare
company on a mission to advance the treatment of GI diseases and
redefine the standard of care for GI patients. We are pioneers in
the development of LINZESS® (linaclotide), which is the U.S.
branded prescription market leader for adults with irritable bowel
syndrome with constipation (IBS-C) or chronic idiopathic
constipation (CIC) and is also indicated for the treatment of
functional constipation in pediatric patients ages 6-17 years old.
Ironwood is also advancing apraglutide, a next-generation,
long-acting synthetic GLP-2 analog being developed for rare
gastrointestinal diseases, including short bowel syndrome with
intestinal failure (SBS-IF), as well as several earlier stage
assets. Building upon our history of GI innovation, we keep
patients at the heart of our R&D and commercialization efforts
to reduce the burden of GI diseases and address significant unmet
needs. Founded in 1998, Ironwood Pharmaceuticals is headquartered
in Boston, Massachusetts, with a site in Basel, Switzerland.
We routinely post information that may be important to investors
on our website at www.ironwoodpharma.com. In addition, follow us on
X and on LinkedIn.
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 the
assessment of the data from the Phase II exploratory STARGAZE trial
of apraglutide in patients with steroid-refractory GI aGVHD and the
continuation of the study through its two-year endpoint; the
safety, tolerability and efficacy of apraglutide in this trial; the
potential effect of apraglutide on intestinal healing, function and
regeneration; and the therapeutic potential of apraglutide for the
treatment of aGVHD. These forward-looking statements speak only as
of the date of this press release, and Ironwood undertakes no
obligation to update these forward-looking statements. 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 effectiveness of
development and commercialization efforts by us and our partners;
preclinical and clinical development, manufacturing and formulation
development of apraglutide; the risk that clinical programs and
studies may not progress or develop as anticipated, including that
studies are delayed or discontinued for any reason, such as safety,
tolerability, enrollment, manufacturing, economic or other reasons;
the risk that findings from completed nonclinical and clinical
studies may not be replicated in later studies; the risk of
competition or that new products may emerge that provide different
or better alternatives for treatment of the conditions that our
products are approved to treat; the risk that healthcare reform and
other governmental and private payor initiatives may have an
adverse effect upon or prevent our products’ or product candidates’
commercial success; the efficacy, safety and tolerability of our
product candidates; the risk that the commercial and therapeutic
opportunities for our product candidates are not as we expect; the
risk that we are unable to successfully partner with other
companies to develop and commercialize products or product
candidates; decisions by regulatory and judicial authorities; the
risk we may never get additional patent protection for our product
candidates, that patents for our products may not provide adequate
protection from competition, or that we are not able to
successfully protect such patents; the risk that the development of
apraglutide is not successful or that any of our product candidates
does not receive regulatory approval or is not successfully
commercialized; outcomes in legal proceedings to protect or enforce
the patents relating to our products and product candidates,
including abbreviated new drug application litigation; challenges
from and rights of competitors or potential competitors; and the
risks listed under the heading “Risk Factors” and elsewhere in our
Annual Report on Form 10-K for the year ended December 31, 2023,
and in our subsequent Securities and Exchange Commission
filings.
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version on businesswire.com: https://www.businesswire.com/news/home/20240328865627/en/
Media:
Beth Calitri, 978-417-2031 bcalitri@ironwoodpharma.com
Investors:
Greg Martini, 617-374-5230 gmartini@ironwoodpharma.com
Matt Roache, 617-621-8395 mroache@ironwoodpharma.com
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