TiGenix, a leading biopharmaceutical company developing therapeutics based on adult stem cells, recently announced the results of the Phase III ADMIRE-CD clinical trial, testing the company’s lead compound Cx601 on Crohn’s disease patients with complex perianal fistulas.
Crohn’s disease is a type of inflammatory bowel disease (IBD) and is characterized by inflammation of the digestive tract, with inflammation being triggered in any location from mouth to anus. Crohn’s disease patients can develop perianal fistulas (about 25% of all CD patients), which are associated with local pain but are also a cause of morbidity (including by sphincter and perineal tissue destruction) and for which no currently effective treatment is available.
The Phase III ADMIRE-CD trial investigated Cx601’s efficacy and safety when administered as a single injection in Crohn’s disease patients. The treatment (Cx601) consists of injecting previously allogeneic expanded adipose-derived stem cells, eASC, (i.e. stem cells derived from adipose tissue of a donor, and not derived from patients themselves) directly into the lesions.
The randomized, double blind, placebo-controlled Phase III ADMIRE-CD trial enrolled 289 Crohn’s disease patients who failed to respond to previous treatments (including those treated with anti-TNF therapies). Patients were recruited within European countries and Israel and were randomly assigned to receive either a single injection of Cx601 or a placebo control.
The study’s primary endpoint was defined as closure of all treated external openings draining at baseline within 24 weeks of treatment with lack of collections over 2 cm (confirmed by magnetic resonance imaging, MRI). Patients randomly assigned to receive Cx601 showed a significant improvement, with 49.5% of patients exhibiting combined remission at week 24 compared to 34.3% in the placebo arm. Importantly, adverse effects were comparable between Cx601 and placebo arms.
Dr Marie Paule Richard, Chief Medical Officer of TiGenix commented the Phase III results, “We are extremely excited about the results of Cx601 in this severely debilitating and difficult to treat condition. Achieving more than 50% combined remission in patients who have not responded adequately to previous treatments, including anti-TNFs, is a remarkable accomplishment. We are committed to submit these data to the EMA and to bring this innovative new treatment to patients whose life is impacted by the challenges of this serious condition”.
Dr Julian Panes, Head of the Gastroenterology Department, Head of the Inflammatory Bowel Diseases Unit, and Associate Professor of Medicine at the Hospital Clinic of Barcelona, President-Elect of ECCO, and Chairman of TiGenix ADMIRE-CD Scientific Advisory Board noted, “The results of this large robust controlled study are clinically relevant and open a completely new avenue for the treatment of perianal fistulising Crohn’s disease, one of the most severe manifestation of this process. The therapy affords a 44% increased chance for patients of closing their fistula with a single injection, which is a major breakthrough.”
Eduardo Bravo, CEO of TiGenix added, “This is a landmark achievement for TiGenix. These positive results, together with the recent endorsement by the FDA of our Phase III trial design for the US, let us move full steam ahead making Cx601 available to the more than 100.000 patients who every year suffer from this serious condition.”
The complete results confirming both the safety and tolerability profile of Cx601 with increased efficacy will be presented at the 11th Congress of ECCO (European Crohn’s and Colitis Organization), to be held in Amsterdam from March 16-19, 2016.