TiGenix submits its pivotal US trial design for Cx601 to the FDA for Special Protocol Assessment

Leuven (BELGIUM) - 22 December, 2014 -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, or eASC's, in inflammatory and autoimmune diseases, announced today that it has submitted to the Food and Drug Administration (FDA) the required documentation for a Special Protocol Assessment (SPA) of its pivotal Phase III trial design for Cx601 in the treatment of complex perianal fistulas in patients with Crohn's disease in the United States. Agreement with the FDA on the SPA will ensure that the trial design is aligned with the FDA's requirements for the future approval of Cx601.

The planned US study design is similar to the ongoing Phase III trial in Europe, whose results are expected in the third quarter of 2015. The US trial design protocol incorporates guidance both from the FDA and from the Company's US Scientific Advisory Board of six leading North American clinical experts in gastroenterology and inflammatory bowel disease.

The randomised, double-blind, placebo-controlled Phase III trial in the US is designed to confirm the efficacy and safety of Cx601 in the treatment of complex perianal fistulas in Crohn's disease patients. It will enrol approximately 180 patients and its primary endpoint will be the 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 at 52 weeks post-treatment.

As discussed with the FDA, TiGenix plans to use data from both the Phase III trial in Europe and from this Phase III trial in the US as the basis for its eventual submission of a Biologics License Application (BLA). On completion of the SPA review process, and of the technology transfer of its cell manufacturing process to a contract manufacturing organisation (CMO) in the US, TiGenix will submit its investigational new drug application (IND) for this Phase III study to the FDA.

"This is another important milestone, achieved according to plan, to prepare Cx601 for approval in the United States, the largest healthcare market in the world", said María Pascual, VP Regulatory Affairs and Corporate Quality at TiGenix. "The product's novel mechanism of action in treating perianal fistulas, and its advanced stage of clinical development in Europe, enables Cx601 to move directly into Phase III in the US, significantly shortening timelines, and the financial burden, of bringing the product to Crohn's disease patients in the US."