FDA Requires Phase III Trial for Apraglutide
Ironwood Pharmaceuticals, Inc. announced that, based on a recent discussion with the FDA, a confirmatory Phase III trial is needed to seek approval of apraglutide for patients with short bowel syndrome (SBS) with intestinal failure (IF) who are dependent on parenteral support
While continuing to advance apraglutide, Ironwood has engaged Goldman Sachs & Co. LLC to explore strategic alternatives for the company to maximize value for stockholders.
Apraglutide is a once weekly, long-acting synthetic GLP-2 analog with the potential to treat a range of rare gastrointestinal diseases where GLP-2 can play a central role in addressing disease pathophysiology, and it is the only GLP-2 analog to achieve a statistically significant reduction in weekly parenteral support volume with once-weekly dosing in patients with SBS.
In preparation for the new drug application (NDA) submission, pharmacokinetic analysis indicated that the exposure and dose delivered in the STARS Phase III trial were lower than planned due to dose preparation and administration. Based on the strength of the STARS Phase III results, Ironwood believed that there was a regulatory path forward. However, following recent dialogue with the FDA it became clear that a confirmatory Phase III trial is needed to seek approval. Ironwood plans to work with the FDA on the design of a confirmatory Phase III trial and the regulatory path forward.
Apraglutide generated strong safety and efficacy data in the STARS Phase III trial, the largest SBS-IF trial to date, and, following an analysis of long-term extension trial data, 27 apraglutide-dosed patients dependent on parenteral support achieved enteral autonomy, which is the ultimate goal for these patients. Consistent with FDA discussions, Ironwood plans to continue the long-term extension trial and believes the data from the STARS trial will continue to be an integral part of an NDA submission package.
“We are disappointed in this outcome, as we firmly believe apraglutide has the potential to provide tremendous value to patients with SBS-IF who suffer from increased mortality, and reduced quality of life, and will now have to wait for the results of a confirmatory Phase III trial,” said Tom McCourt, chief executive officer of Ironwood. “We are focused on the best path forward to get apraglutide to market, which we believe still has the potential to be a blockbuster drug. Concurrently, we will be exploring strategic alternatives for the company in an effort to maximize stockholder value. We are grateful to the patients, families, and clinicians who have and continue to support our clinical development programs.”
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