ANNUAL FINANCIAL REPORT 2010 2010 - TiGenix

Fig. 6.8: Overview of estimated patient population suffering from complex perianal fistulaCellerix is developing Cx601 for the treatment of complexperianal fistula in Crohn’s disease patients. Cx601 utilizesexpanded adult stem cells which are derived from adiposetissue, which have shown to have anti-inflammatory propertiesand to be an effective mechanism for the treatment of fistulae.In Phase I/II clinical trials, Cx601 showed efficacy at 24 weeksof 56% in treated fistula tracts while confirming safetyof the treatment. The effective mechanism of action ofCx601 represents a new approach to cell-based treatmentswith adult stem cells and has potential application in otherinflammatory and autoimmune therapeutic indications.Complex perianal fistulae in Crohn patients tend to occur inindividuals between the ages of 20 and 40, though 10-15% ofpatients are diagnosed before adulthood 44 . Persons who sufferfrom the condition are often unable to carry out ordinary dailyactivities and the recurrent nature of the condition significantlydecreases patients’ quality of life. They generally experiencesevere discomfort, pain and embarrassment and, in many cases,have significant psychological problems, requiring additionaltreatment and often causing substantial burdens for nationalhealth care systems that cover the associated treatment costs.Currently, treatments of choice are antibiotics and azathioprineor 6-mercaptopurine as first-line therapy, and the biologicdrug Remicade® (Infliximab) which is reserved as a second-linetreatment in case of failure. Both treatment alternatives offerpoor outcomes and in many instances notable side effects suchas the reactivation of tuberculosis and increased risk of infectionwith Aspergillus, Listeria and Cryptococcus.44 Source: Panés, Gomollón, Taxonera et al. “Crohn’s Disease. A Review ofCurrent Treatment with a Focus on Biologics Drugs”. 2007; 67 (17): 2511-2537.135 •