Cell therapy specialist TiGenix has announced that their product for the treatment of complex perianal fistulas, Cx601, has met the primary endpoint of their European phase three clinical trial. These results are a huge relief for the Leuven-based company, as the positive outcome secures the company's future.
Cx601 was developed for the treatment of complex perianal fistulas in patients with Crohn's disease. These fistulas are small connections between the perianal space and the outside skin. Not only can these be very debilitating for patients, they are also extremely difficult to treat, both medically and surgically.
Stem cells from fat
Cx601 is based on the expanded adipose stem cell (eASC) technology platform, TiGenix’ flagship technology. This technology allows scientists to derive stem cells from adipose tissue. When compared to other types of stem cells, the fat tissue derived stem cells have multiple advantages. For example, fat tissue is readily available from patients and easy to isolate, as opposed to stem cells derived from bone marrow. Additionally, adipose tissue contains about 1000 times more stem cells than bone marrow. As the adipose derived stem cells are extracted from the patient's own fat tissue, there is no ethical debate surrounding the use of these cells, as is the case for embryonic stem cells.
Contrary to what one might expect when treating perianal fistulas, the stem cells are injected for their anti-inflammatory and immunomodulatory properties instead of their differentiation potential as stem cells. . Cx601, which is a suspension of eASC's, is injected locally and tempers inflammation and initiates wound healing.
Promising clinical results
A phase three clinical trial shows that more than 50% of the patients display remission after a single intralesional injection by week 24. This is a remarkable number, since patients selected for the study showed no or little response to other therapies, such as TNF-inhibitors.
These positive results pave the way for FDA-approved phase three clinical trials in the US and commercialization of Cx601 on the European market. The reaction of the stock market on the news was overwhelming, as the value of the company's shares rose a whopping 23.5% in a single day after the results were made public.
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