Leading European cell therapy company TiGenix NV has just announced the successful full enrollment of patients for a Phase III clinical trial of Cx601, the company’s pipeline treatment for complex perianal fistulas in patients with Crohn’s disease. Crohn’s disease is a type of inflammatory bowel disease (IBD) that causes serious abdominal discomfort, alterations in bowel movement, malabsorption, and even bleeding. It affects approximately 3.2 people out of every 1,000 in Europe and North America, and still does not have a known cure.
TiGenix employed its expertise in developing breakthrough treatments using allogeneic expanded adipose-derived stem cells in inflammatory and autoimmune diseases in evaluating Cx601’s safety and efficacy in over 278 patients, who received the experimental drug across 51 centers in Europe and Israel. The study’s main goal is to achieve remission of fistulous disease in patients with Crohn’s disease, which is complete healing of the perianal tracts. The first collection of treatment responses will be at 24 weeks, followed by a post-treatment evaluation at 52 weeks.
Assessments of healing will be done clinically, and confirmed via MRI. Any visible abscesses should be smaller than 2 cm². This final phase, if successful, should allow the company to file for final marketing authorization in Europe and give the drug a boost in filing for approval in other territories, the U.S. included.
“I’m delighted to announce that TiGenix has completed the patient recruitment for this important study ahead of schedule,” said Eduardo Bravo, CEO of TiGenix. “Clinical results are expected in the third quarter of 2015. With positive results, we expect to submit a request for marketing authorisation with the EMA early in 2016, so that a decision by the European Commission would allow for marketing in Europe in 2017.”
Meanwhile, the company has begun working on US Food and Drug Administration approval after receiving optimistic feedback during a meeting with the agency’s Center for Biologics Evaluation and Research. TiGenix will be transferring proprietary technologies to a contract manufacturing organization (CMO), and will be applying for a special protocol assessment (SPA) with the FDA, which is a prerequisite to an investigational new drug (IND) application to conduct a similar Phase 3 study in the country.
The application for a SPA will be arranged according to the company’s advisory board of gastroenterology and IBD experts in the US. If this Phase III trial is successfully completed and prepared with promising findings from the Europe study, TiGenix will have the green light to apply for a biologics license application (BLA).