The 24-week results of a Phase 3 trial showing the effectiveness of the investigational therapy Cx601 in the treatment of complex perianal fistulas in patients with Crohn’s disease were recently published in The Lancet, according to Takeda Pharmaceutical and TiGenix.
The study is titled “Expanded allogeneic adipose-derived mesenchymal stem cells (Cx601) for complex perianal fistulas in Crohn’s disease: a phase 3 randomised, double-blind controlled trial.”
Patients with Crohn’s disease often exhibit complex perianal fistulas for which there are limited treatment options. The investigational agent Cx601 is a suspension of allogenic adipose-derived stem cells (eASC) that are being developed to treat these fistulas in patients who failed conventional therapies, including immunosuppressants or anti-TNF agents.
Following positive Phase 2 results, TiGenix initiated a randomized, double-blind, placebo-controlled Phase 3 trial in Europe and Israel, called ADMIRE-CD (NCT01541579), which showed that a single injection of Cx601 significantly increased the combined remission of the complex perianal fistulas, as assessed by the closure of all treated external openings that were draining at baseline, despite gentle finger compression. MRI confirmed the absence of collections larger than 2 cm.
Cx601 is a suspension of allogeneic expanded adipose-derived stem cells (eASCs) delivered locally through intra-lesional injection that is being developed specifically to treat complex perianal fistulas in Crohn’s patients.
Trial results were reported in August 2015, showing that Cx601 achieved statistical superiority on the primary endpoint, with 49.5 percent of patients exhibiting combined remission, compared to 34.3 percent in the placebo group.
This translated into a 44 percent increased probability of the Cx601-treated patients achieving combined remission compared to the placebo arm. Importantly, both serious and non-serious treatment-related adverse events were similar in both arms of the study.
“We are very proud of the results of this study,” Professor Dr. Julián Panés, ADMIRE-CD global study coordinator and head of the Inflammatory Bowel Diseases Unit at the Hospital Clínic of Barcelona, said in a press release. “The Lancet is one of the most highly regarded and well known medical journals in the world; we are very delighted to have the Cx601 data selected by this prestigious publication.”
Recently, ADMIRE-CD has completed a follow-up analysis at 52 weeks after treatment, showing that the efficacy and safety of the treatment were maintained for at least one year.
Based on the 24-week results, TiGenix submitted a Marketing Authorization Application to the European Medicines Agency (EMA) earlier this year, and is preparing to develop the investigational agent in the United States. This follows an agreement with the U.S. Food and Drug Administration (FDA) through a Protocol Assessment Procedure (SPA), which means the study asks the proper questions and makes the appropriate measurements for the drug’s approval.
A Phase 3 trial for Cx601 in the U.S. is expected to start in 2017.