Our Lead Drug Candidate
Enabling optimism for the future
Obefazimod is an investigational, orally administered, once-daily therapy, which we believe has the potential to be a differentiated treatment option for inflammatory bowel diseases. It is the first and only molecule that enhances the expression of microRNA-124 (miR-124), a natural regulator of the inflammatory response.
Under dysregulated inflammatory conditions in preclinical studies, enhanced expression of miR-124 resulted in stabilized levels of multiple cytokines and chemokines, bringing them back to homeostatic levels. Obefazimod appears to stabilize the inflammatory response only in the presence of dysregulation, with no effect in its absence, potentially indicating preservation of immune competence. If approved, this therapy has the potential to establish a new therapeutic class currently being referred to as miR-124 enhancers.
Research and Development
Preclinical studies of obefazimod showed that under dysregulated inflammatory conditions, enhanced expression of miR-124 resulted in stabilized levels of multiple cytokines and chemokines, bringing them back to homeostatic levels.
In July 2025, we announced positive top-line results from the 8-week induction trials of obefazimod in UC, marking a key milestone in our clinical development program; 44-week maintenance data remain on track for Q2 2026.
Patient enrollment for ENHANCE-CD in Crohn’s disease was initiated in Q3 2024; 12-week induction data readout is anticipated in 2H 2026. It has not yet been approved for commercial use in any jurisdiction.
Based on the mechanistic concept of obefazimod, we have launched a research effort to generate other potential drug candidates.