Takeda, TiGenix’s orphan drug test meets endpoint

Takeda and TiGenix have presented new data from the Phase 3 ADMIRE-CD clinical trial of treatment refractory complex perianal fistulas in patients with Crohn’s disease at the 12th Congress of the European Crohn’s and Colitis Organisation (ECCO).

The trial indicated that investigational compound Cx601, a suspension of allogeneic adipose-derived stem cells (eASC), maintained long-term remission of treatment refractory complex perianal fistulas in patients with Crohn’s disease over 52 weeks.

Crohn’s disease is a chronic inflammatory disease of the gastrointestinal tract, which is thought to affect up to 1.6 million people in Europe.

In 2009 the European Commission granted Cx601 orphan designation for the treatment of perianal fistula. In March 2016, TiGenix announced that it submitted the Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for Cx601, and a decision by the EMA is expected in 2017. In September 2016 Cx601 received orphan drug status from the Swiss Agency for Therapeutic Products (Swissmedic).

“Perianal fistulizing Crohn’s disease is difficult to treat with currently available therapies and often leads to pain, swelling, infection and incontinence,” said Dr. Asit Parikh, head of Takeda’s Gastroenterology Therapeutic Area Unit. “Existing therapies are limited and associated with complications and a high failure rate. Cx601 may offer patients an alternative treatment option.”

“These data highlight that the efficacy and safety of a single administration of Cx601 were maintained during one year of follow up,” said Dr. Marie Paule Richard, Chief Medical Officer at TiGenix. “It is important to also note that the definition of combined remission used in the ADMIRE-CD study, which includes both clinical and radiological assessment by MRI, is more stringent than the criteria commonly used in previous large scale, randomized clinical trials evaluating perianal fistulas in Crohn’s disease, based only on clinical assessment.”

The companies announced that global pivotal Phase 3 trial for US registration with Cx601 for the treatment of complex perianal fistulas is expected to be initiated by TiGenix in 2017. In the U.S., TiGenix intends to apply for fast track designation from the U.S. Food and Drug Administration (FDA), which would facilitate and expedite the development and review process in the U.S.