TiGenix has received positive feedback from the FDA on an improved global phase III trial protocol for its lead candidate Cx601 for Crohn’s disease. This is expected to speed up US approval.

TiGenix is a Belgian company developing stem cell therapies. The biotech is currently pushing its lead candidate Cx601 to the market for the treatment of complex perianal fistulas in Crohn’s disease patients. Cx601 recently revealed positive results in a European phase III study.

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

TiGenix is well known for its product ChondroCellect, which was the first cell therapy to reach approval on the European market for the repair of knee cartilage. After the company recently withdrew its market authorization for this product, due to a lack of reimbursement, the biotech is focusing on its new lead Cx601.

This product, currently awaiting EMA approval, consists of allogeneic expanded adipose-derived stem cells (eASC), which are indicated for the treatment of perianal fistulas in Crohn’s disease. The therapeutic effects of eASCs are based on immunomodulatory abilities of these stem cells, which can restore immune balance by suppressing a variety of immune cell subsets and inducing the generation of regulatory T cells.

Areas of the colon commonly affected during Crohn’s disease

The current approval from the FDA will allow TiGenix to file the BLA based 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 planned” concluded Maria Pascual, VP Regulatory Affairs & Corporate Quality of TiGenix

The current amendments will allow TiGenix to push its therapy to the US market even faster, which might pivotal for the company in light of its financial situation. After its shares had reached a low of 22 cents back in 2013, the share price is currently still under €1. With its low €34M IPO on Nasdaq in the end of last year, its market cap is still only at  €191M. A low sum for a late stage clinical company.

As the EMA approval for Cx601 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 test Cx611 as a treatment for sepsis in patients with pneumonia.

A second 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 to its position as one of the pioneers in stem cell-based therapies.

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

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