Belgian biotech TiGenix has racked up another positive result for its Takeda-partnered stem cell therapy for fistulas caused by Crohn's disease.
Results of the phase III ADMIRE-CD trial show that a single administration of Cx601 - a suspension of allogeneic adipose-derived stem cells - was significantly more likely to heal perianal fistulas in Crohn's patients, and stop them coming back for at least a year, compared to placebo.
Crohn's disease is a chronic inflammatory disease of the gastrointestinal tract affecting some 1.6 million people in Europe, and in around 25% of patients can cause fistulae - abnormal channels connecting the bowel to another organ or the outside surface of the body.
Perianal fistulas are the most common type, linking the bowel to the skin near the anus, but are still relatively rare, and Cx601 has been granted orphan status by the European Commission and Swissmedic for this indication. The therapy was submitted for EU approval last March and a decision is expected in the first half of this year.
In ADMIRE-CD, 56% of patients treated with Cx601 and usual supportive care went into remission, compared to 39% of patients who received placebo plus standard care. After 52 weeks the clinical remission rate was 59% and 42%, respectively, with three quarters of patients receiving TiGenix' therapy experiencing no relapses versus just over half of the control group.
The data has just been presented at the Congress of the European Crohn's and Colitis Organisation (ECCO) and raise hopes that Cx601 will be approved, and justify Takeda's decision to license the drug last year.
Shortly after TiGenix reported 24-week results from ADMIRE-CD, the Japanese drugmaker stumped up €25m upfront for rights to Cx601 for Crohn's disease-related complex perianal fistulae outside the US, with another €355m in milestone payments in the offing. The first milestone of €15m is due on EU approval, and TiGenix has said the Takeda deal will give it the financial resources it needs to start developing Cx601 for the US market.
"Perianal fistulising Crohn's disease is difficult to treat with currently available therapies and often leads to pain, swelling, infection and incontinence,” said Dr Asit Parikh, who heads Takeda's gastroenterology therapy unit.
"Existing therapies are limited and associated with complications and a high failure rate [so] Cx601 may offer patients an alternative treatment option," he added.
Cx601 assumed greater importance for TiGenix after the Belgian biotech decided last year to withdraw its EU license for the autologous chondrocyte implant ChondroCelect, used to treat cartilage damage in the knee.