Boston— Ironwood Pharmaceuticals (Nasdaq: IRWD) announced Monday that the U.S. Food and Drug Administration (FDA) is requiring a confirmatory Phase 3 trial for its experimental drug apraglutide, delaying the company’s plans to seek regulatory approval for treating short bowel syndrome with intestinal failure (SBS-IF).
The FDA’s guidance comes after a recent meeting in which the agency concluded that results from Ironwood’s previous STARS Phase 3 study were insufficient for approval. Although the trial showed statistically significant reductions in weekly parenteral support with once-weekly dosing, pharmacokinetic analysis revealed that the dose delivered was lower than originally intended due to preparation and administration issues.
Apraglutide is a synthetic GLP-2 analog designed to treat rare gastrointestinal diseases, and is the only GLP-2 analog to demonstrate efficacy with weekly dosing in SBS-IF patients. The STARS trial, the largest to date in this indication, also showed that 27 patients achieved full enteral autonomy during a long-term extension phase.
Despite this, Ironwood acknowledged that FDA approval would now require additional clinical evidence. The company plans to work with the FDA on the design of a confirmatory Phase 3 trial.
“We are disappointed in this outcome,” said Ironwood CEO Tom McCourt. “We firmly believe apraglutide has the potential to provide tremendous value to patients with SBS-IF. Our priority remains getting the therapy to market, and we still believe it could become a blockbuster drug.”
In parallel, Ironwood has retained Goldman Sachs & Co. LLC to evaluate strategic alternatives, which may include a potential sale or merger, to enhance shareholder value.
Ironwood said it will continue enrolling patients in its ongoing long-term extension study and include the existing STARS data as part of a future new drug application (NDA) submission.
