TiGenix completes patient recruitment for the European Phase III trial of Cx601 ahead of schedule

TiGenix NV (Euronext Brussels: TIG), an advanced biopharmaceutical company focused on developing and commercialising novel therapeutics from its proprietary platform of allogeneic expanded adipose-derived stem cells in inflammatory and autoimmune diseases, announced today that it has completed the patient recruitment for its Phase III trial of Cx601 in Europe for the treatment of complex perianal fistulas in Crohn’s patients.

The trial is a randomised, double-blind, placebo-controlled Phase III study designed to confirm the efficacy and safety of Cx601 in the treatment of complex perianal fistulas in Crohn’s disease patients. The trial has recruited more than 278 patients across 51 centres in 7 European countries and Israel. The study’s primary endpoint is remission of fistulous disease, defined as 100% healing of the tracts. The  first complete analysis of results will be at 24 weeks, with a follow-up analysis to be performed at 52 weeks post-treatment. Evaluation of healing includes both clinical assessment and MRI confirmation (lack of abscesses larger than 2 cm²). This pivotal study is intended to allow filing for marketing authorisation in Europe, and to serve as a key supportive study in filing for approval in other territories, including the United States.

“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.”

Following the positive feedback received at a meeting with the Center for Biologics Evaluation and Research within the Food and Drug Administration (FDA), TiGenix is moving ahead with the development of Cx601 for the United States market. The Company will start the process of technology transfer to a US-based contract manufacturing organization (CMO) later this year. Also by the end of this year, TiGenix will file a special protocol assessment (SPA) with the FDA in order to be able to file an investigational new drug (IND) application for a Phase III trial in the United States. That SPA file will be prepared by the company in cooperation with its US advisory board of North American clinical experts in gastroenterology and inflammatory bowel disease.  The Phase III trial in the US, if successful, together with positive data from the European Phase III trial, would enable the Company to file a biologics license application (BLA) with the FDA.

Crohn’s disease is a chronic inflammatory disease of the intestine, of which complex fistulas are a complication that appear in around 10% of patients. There are approximately 100,000 new cases every year in Europe and the US. A fistula is an abnormal tract connecting two surfaces, and  a perianal fistula is a tract between the perianal space and the epithelial surface next to the anus. A complex perianal fistula is a serious condition that affects the anal sphincter, can have multiple tracts, and is associated with a perianal abscess. Individuals who suffer from the condition are often unable to carry out ordinary daily activities and experience a significant reduction in their quality of life due to the recurring nature of the condition. They generally endure severe discomfort, pain and embarrassment and, in many cases, have significant psychological problems, requiring additional treatment which creates substantial additional burdens for healthcare systems. In the longer term, these patients suffer an increased risk of developing anal epithelial carcinoma. Current treatment options, which include antibiotics, immunosuppressants and biologics are mainly symptomatic and do not offer a long term solution. The risk of recurrence is high. Surgical intervention may be required in severe cases with an increased risk of anal incontinence.