On the heels of a phase 3 win that failed to impress investors, Ironwood Pharmaceuticals is back with more data in efforts to prove that its long-acting GLP-2 analog can do more than beat placebo.
The new subgroup analyses come from a pivotal trial, dubbed STARS, assessing apraglutide in patients with short bowel syndrome (SBS) with intestinal failure. The data are set to be presented tomorrow at the American College of Gastroenterology 2024 Annual Scientific Meeting & Postgraduate Course in Philadelphia.
The new findings indicate that apraglutide treatment has a consistent effect across varying baseline demographics, including gender, age, weight, region, race, ethnicity and certain SBS characteristics such as length of remnant bowel.
Since the market opened, Ironwood’s stock has risen slightly, up 6% from Friday’s market close of $3.96 to $4.23 at 12 p.m. ET today.
In February, Ironwood shared top-line STARS data measuring the weekly volume of parenteral support (PS), a necessary intravenous treatment for people with rare and severe organ failure conditions.
After 24 weeks, the once-weekly subcutaneous treatment was tied to a 25.5% decrease in weekly PS volume, compared to a 12.5% reduction in the placebo arm, hitting the study’s main goal. Despite the trial win, investors weren’t convinced the treatment could best Takeda’s Gattex and Zealand Pharma’s glepaglutide, the latter of which is currently under FDA review.
At the time of the initial data drop, Ironwood shares fell 30% to $10.50, down from a market close of $15.12 the day before.
The phase 3 trial also met its first two secondary endpoints, tying apraglutide to an increase in the number of patients who had at least one day a week off PS relative to baseline and an improvement in weekly PS volume in the 50% of patients with a stoma that allows body waste to enter a drainage bag. However, the study missed its two secondary endpoints in colon-in-continuity (CIC) patients.
People with CIC make up about half of the 18,000 SBS patients worldwide, with the stoma subpopulation accounting for the other half. While Ironwood has heralded apraglutide’s potential to address both groups, apraglutide was no better than placebo at increasing the number of CIC patients who had at least one day a week off PS or who reached enteral autonomy at Week 48.
The new subgroup analyses examined the primary endpoint by demographics, which included patients with stoma and CIC, and found a consistent treatment effect for apraglutide across subgroups, according to Ironwood.
The additional data come as Ironwood pushes ahead with plans to file for FDA approval of apraglutide in adults with SBS who are dependent on PS.
“As we work to make apraglutide available as potentially the first once-weekly treatment option for patients with SBS who are dependent on PS, we are focused on continuing to educate the SBS community on the potential of this compound across profiles of adult patients given the known patient heterogeneity,” Michael Shetzline, M.D., Ph.D., chief medical officer, senior vice president and head of R&D at Ironwood, said in an Oct. 28 release.
The Boston-based company acquired apraglutide last year through a $1 billion takeover of VectaBio. Ironwood believes the candidate could eventually serve as a best-in-class drug, touting the drug’s potency, selectivity and weekly dosing.