TiGenix has receivedpositive feedback from the FDA on an improved global phase III trial protocol for its lead candidateCx601 for Crohns disease. This is expected tospeed up US approval.

TiGenix is a Belgian companydevelopingstem cell therapies. The biotech is currently pushing its lead candidateCx601to the market for the treatment ofcomplex perianal fistulas in Crohns disease patients. Cx601 recently revealedpositive resultsin a European phase III study.

Following these results, the company submitted a number of technical adjustments for itspivotal phase III study for Biologics License Application (BLA) in the US, which were now approved by the FDA and are expected to acceleratethe process to US marketing authorization.

TiGenix is wellknown for its productChondroCellect, which was the first cell therapyto reach approval on the European market for the repair of knee cartilage.After the companyrecently withdrew its market authorization for this product, due to a lack of reimbursement, the biotech is focusing on its new leadCx601.

Thisproduct, currently awaiting EMA approval, consists ofallogeneic expanded adipose-derived stem cells (eASC), which are indicated for the treatment ofperianal fistulas in Crohns disease. The therapeuticeffects of eASCs are based on immunomodulatory abilities of these stem cells, which canrestore immune balance by suppressing a variety of immune cell subsets and inducing the generation of regulatory T cells.

Areas of the colon commonly affected duringCrohns disease

The current approval from the FDA will allow TiGenix to file the BLAbased on the efficacy and safety follow-up of patients at week 24, instead of week 52.The FDA has also agreed to accept fewer patients than originally planned in the study and endorsed a broader target population that will ultimately facilitate the recruitment process.

We believe that this revised protocol will allow us to file for approval one year earlier than we had originally plannedconcludedMaria Pascual, VP Regulatory Affairs & Corporate Quality of TiGenix

The current amendments will allow TiGenix to push its therapyto the US market even faster, which might pivotal for the company in light of its financial situation. After its shares had reached a low of22 cents back in 2013, the share price is currently still under 1. Withits low 34M IPO on Nasdaq in the end of last year, its market cap is stillonly at 191M. A low sum for a late stage clinical company.

As the EMAapproval forCx601 is expected soon, which will then be commercialized by Takeda, the company may actually be underestimated. The biotech recently started a new Phase Ib/IIa trial to testCx611 as a treatment for sepsis in patients with pneumonia.

Asecond platform consisting of transplanted allogeneic cardiac stem cells (AlloCSC)is currently in Phase II for acute myocardial infarction. It seems like TiGenix is definitely clinging toits position as one of the pioneers in stem cell-based therapies.

Images via shutterstock.com / CI Photos and CC 3.0 /RicHard-59

Originally posted here:

Stem Cell Therapy receives FDA Boost to enter the US Market - Labiotech.eu (blog)