Ironwood Pharmaceuticals Announces Positive Results from its Phase II Exploratory STARGAZE Trial of Apraglutide in Patients with Steroid-Refractory Gastrointestinal Acute Graft-versus-Host Disease (SR GI aGVHD)

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 1^st line therapy, such as corticosteroids. Despite receiving 2^nd line therapy, many patients also do not maintain response over time, with significant non-relapse mortality (NRM) after six months.