Cellerix

Product platform based on expanded adipose tissue-derived mesenchymal cells (eASCs) from allogeneic sources. Three programs based on different local administration routes are currently ongoing:

Medicinal product profile
Cx601 is a 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.

• 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.
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:
First allogeneic therapy based on eASCs in clinical phase
Program based on 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 is ongoing in 24 patients and 6 centers. The primary objectives of this study are as follows:

• 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.

Cellerix expects the final report of this study in the last quarter of 2010.

Cx602: Endoscopic administration of allogeneic eASCs for the treatment of IBD (intestinal bowel disease). The preclinical program for this product is currently ongoing in cooperation with Fundación La Paz, and it is hoped that its results may translate into clinical development in the short/mid-term. Cellerix is open to discuss opportunities of co-development of this product with potentially interested companies.

Cx603: Intra-articular administration of allogeneic eASCs for the treatment of osteoarthritis. The objective of Cellerix is to have a proof of concept in animals in the short term.