TiGenix announces positive 52-week Phase III results of Cx601 in complex perianal fistulas in Crohn's disease patients

TiGenix NV , an advanced biopharmaceutical company focused on developing and commercialising novel therapeutics from its proprietary platforms of allogeneic expanded stem cells, has announced that a single injection of Cx601 was statistically superior to placebo in achieving combined remission at week 52 in the Phase III ADMIRE-CD trial in Crohn's disease patients with complex perianal fistulas with inadequate response to previous therapies, including anti-TNFs. The one-year data also confirm the favorable safety and tolerability profile of Cx601 already reported at week 24.

ADMIRE-CD is a randomized, double-blind, placebo-controlled Phase III study designed to confirm the efficacy and safety of a single injection of Cx601 in the treatment of complex perianal fistulas in Crohn's disease patients. In total, 212 patients were randomized in 7 European countries and Israel. Patients included in this study had an inadequate response to at least one previous therapy, including anti-TNFs. Continuation of medical standard of care was allowed during the duration of the trial in both groups. The study primary endpoint was combined remission at week 24, defined as closure of all treated external openings draining at baseline despite gentle finger compression, and absence of collections >2cm confirmed by MRI^[1]. This same endpoint of combined remission has been analyzed after 52 weeks as a secondary variable.

In the ITT^[2] population (n=212), Cx601 achieved statistical superiority (p=0.012) with 54.2% combined remission at week 52 compared to 37.1% in the placebo arm. In the mITT^[3] population (n=204), the combined remission at week 52 was 56.3% and 38.6% for Cx601 and placebo respectively (p=0.010). Efficacy results were robust and consistent across all statistical analyses.

The week 52 data also shows a higher rate of sustained closure in those patients treated with Cx601 and in combined remission at week 24 (75.0%) compared to patients in the placebo group (55.9%).

Treatment-emergent adverse events (non-serious and serious) and discontinuations due to adverse events were comparable between Cx601 and placebo groups.

"We are delighted with the remarkable consistency of these results with respect to those reported at 24 weeks. In particular, the 75% persistence in the combined remission from week 24 to week 52 confirms Cx601 as a promising therapeutic solution for a hard to treat population that is refractory to existing treatments including anti-TNFs," said Dr Marie Paule Richard, Chief Medical Officer of TiGenix. "The improvement brought by Cx601 compared to the best standard of care available could make a real difference to the life of patients suffering from this chronic disease".

"The one year results of this large robust controlled study open up a new paradigm for the treatment of perianal fistulising Crohn's disease, one of the most severe manifestations of this process. With a single injection of Cx601 more than 50% of patients are in remission at one year with a favourable safety profile. Remarkably, most patients that were already in remission at six months continue to benefit from the treatment six months later, a major breakthrough in this field", said Dr Julián Panés, Head of the Gastroenterology Department, Head of the Inflammatory Bowel Diseases Unit, and Associate Professor of Medicine at the Hospital Clínic of Barcelona, President-Elect of ECCO, and Chairman of TiGenix ADMIRE-CD Scientific Advisory Board.