Our lead product candidate, ontunisertib (AGMB-129), is a selective and potent oral, gastrointestinal-restricted small molecule inhibitor of ALK5, or TGFβR1, in development for the treatment of Fibrostenosing Crohn’s Disease, or FSCD. FSCD is a severe complication of Crohn’s Disease, or CD, that is associated with significant morbidity. There are approximately 1.4 million patients under treatment for CD in the seven major markets of the United States, France, Germany, Italy, Spain, the United Kingdom and Japan, and approximately 620,000, or 46%, of these patients have FSCD. The emergence of burdensome symptomatic strictures is considered to be an inevitable consequence of long-term inflammation for the large proportion of patients with CD who progress to FSCD and eventually require surgery. There are no approved pharmacologic therapies for FSCD.

We believe ontunisertib has the potential to change the paradigm for treating FSCD patients and provide the first pharmacologic treatment for strictures. Ontunisertib is designed to act locally in the gastrointestinal tract, enabling high exposure in the target tissue. Then, following absorption, ontunisertib is rapidly inactivated in the liver to avoid potential toxicities associated with systemic TGFβ signaling inhibition.

Ontunisertib successfully completed a Phase 1 study, which included single ascending dose (SAD), multiple ascending dose (MAD) and food-effect (FE) stages, as well as an additional multiple-dose stage for the assessment of local drug exposure in terminal ileal mucosa, where the fibrostenosing strictures in FSCD patients are most often located. A total of 82 healthy subjects were randomized to receive either single or multiple daily oral doses of ontunisertib, or matching placebo.

Ontunisertib was well-tolerated at all doses tested. The incidence and intensity of adverse events were similar across all dose cohorts including the placebo cohort. No drug-related safety signal or dose-limiting toxicities were identified. In addition, the trial showed high local exposure to ontunisertib in the ileum but no clinically relevant systemic exposure, demonstrating that the GI restricted mechanism operated efficiently in humans.

In November 2025, we announced positive topline results of the STENOVA Phase 2a trial in FSCD. STENOVA is a global randomized, double-blind, placebo-controlled study in 103 FSCD symptomatic patients with at least one ileal stricture. Part A of the STENOVA study achieved its primary endpoint of assessing the safety and tolerability of ontunisertib 100mg QD and 200mg BID in FSCD patients. Pharmacokinetic results confirmed the GI-restricted profile of ontunisertib, with high local and low systemic exposure of ontunisertib in FSCD patients. We also observed positive signals on several exploratory clinical endpoints.
The 48-week open-label treatment extension of the STENOVA trial with ontunisertib is currently ongoing and we expect to report the results in the second half of 2026.

Based on the positive results observed in the STENOVA study to date, we are preparing to initiate a Phase 2b trial of ontunisertib in patients with symptomatic FSCD in the second half of 2026.

Ontunisertib (AGMB-129) has received U.S. FDA Fast Track Designation.