ANNUAL FINANCIAL REPORT 2010 2010 - TiGenix

6.14.2 Competitive strengthsTiGenix believes that Cellerix’ competitive strengths are asfollows:• Allogeneic treatments. eASCs are isolated from healthydonors, expanded and stored in a cell bank ready to bethawed when the treatment is required and thereforeconstituting an “off-the-shelf “product (in comparison to anautologous product, where the patient needs to undergoa liposuction himself in order to obtain his own eASCs asthe source of the pharmaceutical treatment). Allogeneictreatments offer several advantages, including timing andpatient comfort (liposuction on the patient is avoided),optimization and scale up of production and easier logistics.• Well characterised stem cell platform. Cellerix’ stemcell platform based on expanded adipose-derived stemcells (eASCs) has been extensively characterized in line withEMA requirements and benefits from exhaustive preclinicaland CMC packages that have been discussed with EMAon various occasions at scientific advice level. Analyticalprocedures and methods are fully validated according toICH Quality Guidelines and EMA validated release assays(potency, purity, identity) are in place. Cellerix has thecombined experience of more than 600 cell productprocedures performed/documented to date.protocol, an expedited regulatory review period by the EMA,as well as up to 10 years marketing exclusivity from the dateof the product’s launch.• R&D engine which has resulted in several promisingpre-clinical candidates. The effective mechanism ofaction of eASCs represents a new approach to cell-basedtreatments of immune-mediated inflammatory diseases.The potential application in other therapeutic indicationsis being explored in several preclinical stage programs:Cx621 for the intralymphatic treatment of autoimmunediseases anticipated to enter in the clinic in 2011, Cx602 forthe endoscopic treatment of IBD and Cx603 for the intraarticulartreatment of osteoarthritis.• In-house industrial cell manufacturing capability.Cellerix has accredited facilities which meet the GMPstandards of the European Medicines Agency (EMA)and is also the first Spanish company approved by theSpanish Medicines and Medical Devices Agency (AEMPS)for the manufacture of ATMPs both for clinical trials andcommercialization purposes. The manufacturing processis fully validated according to specifications established foreach cell bank, which have registered stability data for morethan 2.5 years.• Two products in clinical development. Cellerix’ leadproduct, Cx601, successfully completed Phase II clinicaltrials in 2010 and has received positive scientific advicefor Phase III from EMA in March 2011. Complex perianalfistula, for which Cx601 is being developed, represents adebilitating condition underserved by available treatmentoptions and for which there are, to the best knowledgeof TiGenix, relatively few competing programmes indevelopment. The condition is characterised by a welldefined patient population, potentially enabling Cellerixto penetrate rapidly the target market in a highly focusedmanner. Cx611 has recently commenced Phase I/IIclinical trials and is targeting Rheumatoid Arthritis (RA), atherapeutic indication underpinned by high levels of unmetmedical need despite current therapeutics.• Orphan Drug designation for lead clinicalprogramme. Cx601 has been granted Orphan Drugdesignation by the EMA in 2009. This designation confersseveral significant benefits including: a streamlineddevelopment process, potential financial R&D incentivesfrom the EU, detailed development feedback from theregulatory authorities and support for the clinical trial133 •