TiGenix on track for pivotal cell therapy results in Crohn's

Cell therapy specialist TiGenix is mere months away from reporting phase III trial results for its fistulising Crohn's disease candidate Cx601 that could support an EU marketing application early next year.

The ADMIRE trial of Cx601 is looking at the use of TiGenix' allogeneic expanded adipose-derived stem cells (eASCs) – given as a local injection – to treat complex perianal fistulas that develop in a proportion of Crohn's disease patients.

These fistulas – small channels that develop between the end of the bowel and the skin near the anus – can be very painful and cause bleeding and discharge. They affect around 12 per cent of Crohn's patients, with around 80 per cent of these considered to be complex, with multiple interlocking channels.

At the moment perianal fistulas generally require surgery to aid healing, which carries a risk of infection and bowel incontinence – with a one-in-five chance that the fistula will recur.

Cx601 was granted orphan designation for the treatment of anal fistulas in 2009, recognising the debilitating nature of the disease and the lack of treatment options, and some analysts have suggested the therapy could eventually grow into a $900 million product, assuming an EU launch in 2017 and a US launch by 2020.

The Belgian-Spanish company, which is part-owned by Grifols, refocused its activities on its eASC platform last year, selling off a manufacturing plant to PharmaCell and licensing its only commercial product – ChondroCelect for cartilage repair – to Swedish Orphan Biovitrum (Sobi).

The move was welcomed by analysts at Edison, who said "the commercial potential of the eASC platform is much greater."

Positive results in the ADMIRE trial of Cx601 in fistulising Crohn's could be enough to support an EU filing, according to the analysts, and would prompt a second, US study that could set it on a path to approval on the other side of the Atlantic.

A phase III trial design has already been submitted to the US FDA and TiGenix recently signed an outsourcing contract with Lonza for the manufacture of Cx601 in the US.

Meanwhile, the company is moving ahead with the development of a second eASC candidate, Cx611 for early rheumatoid arthritis and severe sepsis, that will be delivered intravenously rather than by local injection. Patients are currently being recruited into a phase I sepsis trial.

The global stem cell therapy market is poised to grow at a compound annual growth (CAGR) rate of 39.5 per cent between 2015 and 2020, to reach a level of $330 million, according to a recently-published market research report.

Allogeneic stem cell therapy, in which stem cells are harvested from a genetically-similar but not identical donor, is expected to command the largest share of the market in 2015. It is also expected to grow at a higher CAGR than autologous (self-harvested) cell therapies from 2015 to 2020.