Takeda, TiGenix stem cell therapy shows sustained effect

Takeda and TiGenix have presented 52-week data on their allogeneic expanded adipose-derived stem cells in Crohn’s disease patients with treatment-refractory complex perianal fistulas. The data show the Cx601 stem cells continue to outperform placebo one year after administration.

TiGenix presented the 24-week data from the successful phase 3 trial back in 2015, sparking a surge in its stock price and setting it up to land a deal with Takeda. Last year it followed up with the release of a first look at 52-week results confirming the efficacy outcomes seen in the earlier data drop.

Takeda and TiGenix have now shared another overview of the 52-week data at the 12th Congress of the European Crohn’s and Colitis Organisation (ECCO). The abstract includes treatment-related adverse event data that were absent from TiGenix’s original release, but included in subsequent presentations.

Those 52-week data confirm the positive safety profile seen in the 24-week results. The rate of treatment-emergent adverse events was lower in the Cx601 cohort than the placebo plus standard of care arm at both time points. The same is true when only serious adverse events are analyzed.

The safety results complement the previously-released efficacy data. Among the 62% of patients who completed the 52-week follow-up, the results were comparable to those generated after 24 weeks. In the Cx601 arm, 56.3% of the modified intention-to-treat (mITT) population achieved combined remission after 52 weeks, compared to 51.5% after 24 weeks. The respective figures for the placebo cohort are 38.6% and 35.6%. The mITT population included all patients to undergo at least one post-baseline efficacy evaluation.

“These data highlight that the efficacy and safety of a single administration of Cx601 were maintained during one year of follow up,” TiGenix CMO. Marie Paule Richard said in a statement. “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.”

Relapse rates in the Cx601 group were rarer, too. Three-quarters of participants who responded to Cx601 after 24 weeks made it to 52 weeks without relapsing. The number falls to 55.9% among the placebo cohort.

TiGenix is hoping the data will prove compelling enough to secure a regulatory approval in Europe later this year. In parallel, TiGenix is setting up another phase 3 trial designed to deliver data to support approval in the U.S.. TiGenix expects the trial to start later this year.

Shares in TiGenix traded up 4% shortly after the stock exchange in Brussels opened for the day.