Last week at the BioTech showcase Tigenix (NYSE Euronext: TIG) presented as part of a panel of companies talking about auto-immune disease and many possible indications. One that caught our attention was Cx601.
Cx601 is an "EU-medicinal product" based on expanded mesenchymal stem cells obtained from adipose tissue (eASCs). This product is a member of the new generation of cell therapies.
- According to Tigenix: Cx601 is the first medicinal product based on allogeneic (collected from donors) eASCs to be approved for clinical trials worldwide.
- Cx601 is able to activate the physiological repair mechanisms of the body thanks to its capacity to interact with the body.
- Prior clinical trials with eASCs suggest that Cx601 has a great therapeutic potential based on its anti-inflammatory, healing, and immunoregulatory properties.
Cx601 is a suspension for injection that contains donor-derived stem cells isolated and expanded from adipose tissue obtained by liposuction.
Cx601 is being evaluated in a Phase II clinical trial for the treatment of complex perianal fistula in patients with Crohn’s disease. Cellerix estimates that it will be able to start in 2011 the last phase of clinical development before starting the approval process.
A perianal fistula is an abnormal connection between the perianal space and outside skin surface. A fistula is considered to be complex when its treatment involves a high risk of causing loss of anal continence, the fistulous tract crosses more than 30% of external sphincter (high trans-sphincteric, suprasphinctetic, and extrasphincteric), several tracts are found, is recurrent, or patient has incontinence, local irritation, or Crohn’s disease.
Current treatment for complex fistula in patients with Crohn’s disease is poorly effective and costly. Patients with complex fistula but no inflammatory disease require surgery, with its attendant risk of fecal incontinence. We would note that Ventrus (NASDAQ: VTUS) is another company outside of Cell Therapy that is working in this area.
Cx601 acts by controlled reduction of inflammation in the fistula, promoting adequate homeostasis through the release of anti-inflammatory factors which in turn promote natural fistula closure.
Cx601 has been designated as an orphan drug by the European Medicines Evaluation Agency.
Clinical Development: An allogeneic therapy based on eASCs in clinical phase
The program is based on the local administration of allogeneic eASCs for the treatment of complex perianal fistula in patients with Crohn’s disease. Safety of this treatment has been documented by its use for treating patients in a compassionate use program. Designation as an orphan drug for Europe was achieved in a record time.
A Phase I/II, open label study of this product for the indication of complex perianal fistula derived from Crohn’s disease has been done (24 patients across 6 centers). The primary objectives of this study were to :
- To show that administration of allogeneic cells from healthy donors is safe.
- To assess the efficacy of Cx601 in terms of complete closure of complex perianal fistula 12 and 24 weeks after initial drug administration.
Tigenix is now hoping to move this program forward to a pivotal trial. Given the fact that Tigenix has developed ChondroCelect®, the company’s lead product for cartilage regeneration in the knee we believe this company is worth watching.
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