• A pre-phase III specialty pharma company focused on niche indications with high margin potential
Cellerix is a specialty pharma company focused on the application of stem cell therapeutics for niche indications. The Company has developed a broadly applicable platform which harnesses the potential of stem cells derived from adipose tissue. The platform has already generated two product candidates that are currently undergoing clinical evaluation for well-characterised indications of significant unmet clinical need. Cx401was granted Orphan Drug status by the EMEA in July 2005 for its use in complex perianal fistulae (in Crohn and non-Crohn patients) and has recently completed Phase II evaluation. Cx501 was granted Orphan Drug status by the EMEA in April 2006 and will shortly start Phase II/ III trials for epidermolysis bullosa (“EB”), a rare hereditary disease affecting proper adhesion between various skin layers. Although indications targeted by the Company may be classified as being niche, lead candidates address substantial markets.
• The Opportunity
Following technological proof-of-concept of its adipose stem cell isolation, production and treatment technology, Cellerix is now looking to out-license Cx401 and Cx501 for Japan, with other geographical rights also available. Company plans to retain all European rights to its product portfolio and to undertake sales and marketing itself in Europe.
• Research Background
Cellerix is pioneering the use of adipose and other tissue derived stem cells for the treatment of indications of unmet clinical need. Some of the advantages of using adipose as the source material are the higher abundance of stem cells (between x500 to x1000) and the relatively easy procedure – a simple and fast liposuction (100 ml), instead of a painful iliac puncture.
The Company’s research has been guided by its proprietary understanding of the isolation, amplification and reintroduction of stem cells. Underpinning each product candidate programme is the Company’s high quality, GMP compliant cell processing and production facility. Following the success of its lead products, the Company is now applying this platform to develop treatments for indications of related aetiology. In doing this, the Company benefits from owning the first laboratory in Spain approved by the EMEA for the production of cellular medicines.
Figure 1:Initial Focus on Autologous Therapies
• Product Pipeline
Figure 2: Cellerix’s current portfolio of product candidates.
A Phase I trial indicated that Cx401 is safe and efficacious when used to treat fistula: a 50 patient open randomized controlled Phase II trial has also been completed and data is already available under confidentiality
This product demonstrated a 75% response rate and no adverse effects over a substantial two-year period in an initial Phase I trial. The efficacy is well above that of available treatment options, especially in fistulizing Crohn's disease. Further, the Company recently completed a phase II 50 patient, open randomized controlled trial, the results of which will be made available under confidentiality. The Company is in the process of finalising the design of a Phase III trial in Europe which is anticipated to start Q4 2006. In addition, the Company has already gone through a pre-IND meeting with FDA in the USA.
• Cx401 and Cx501: first stem cell therapy products with Orphan Drug status in Europe.
Cellerix’s first two product candidates each target specialty indications for which there is currently no or limited therapy. Cellerix has progressed its two lead programmes past the point of clinical proof-of-concept in its initial chosen indications of Crohn’s and non-Crohn’s related perianal fistula and epidermolysis bullosa. The Company’s products and the underlying technologies are protected by a series of 23 patents and patent applications world-wide.
EMEA has awarded Orphan Drug status to Cx401 in July 2005 and to Cx501 in April 2006. Obtaining Orphan Drug status gives the Company advantages that include commercial exclusivity (for ten years following market launch), financial incentives, protocol assistance by EMEA as well as shorter evaluation periods by the regulatory agencies.
Cx401 is based on a suspension of adipose-derived stem cells and is currently being evaluated for efficacy in Crohn’s and non-Crohn’s disease related complex perianal fistula, a distressing and painful indication whereby a patient suffers a connection from the rectum or other anorectal area to the skin surface. Currently, this indication is treated either by surgery or Infliximab. Both of these treatments are expensive and have a poor response rate. In the EU in 2005, it has been estimated that approximately 63,100 individuals suffered from complex perianal fistulas representing a total market of approximately €1.5 billion. In the US the corresponding figure is estimated to be 48,300 individuals.
Figure 3: Cx401: Addressing a clear unmet clinical need
Figure 4: Cx401’s Competitive Advantage
Cx501 is based on a combination of autologous keratinocytes plus fibroblast allogeneic stem cells for epidermolysis bullosa, a hereditary disorder that causes lesions through blistering and shearing of the skin from even the mildest of friction, often from simple day to day activities. The most affected areas are hands and feet, and in many cases these lesions give rise to a fusion of the fingers or toes. Currently, wound treatment in EB is only supportive i.e. by use of bandages. General therapies have not been successful. Cellerix estimates that the market in the EU and USA for a successful therapy for EB to be €230 million per annum.
An initial trial indicated proof-of-concept for Cx501: Phase II/III evaluation anticipated to start in Q4 2006
Cx501 demonstrated clinical proof-of-concept in an investigator-lead trial with a Phase II/III trial anticipated to begin in Q4 2006. Due to the nature of the indication, this will be an open label trial. This product candidate received Orphan Drug designation from EMEA in April 2006.
Figure 5: Epidermolysis – A rare genetic disease with orphan drug status
Figure 6: Cx501: Cellerix’s chimeric skin solution
Figure 7: Cx501’s Competitive Advantage
• Further pre-clinical pipeline
The Company is centering its preclinical efforts in developing an allogeneic version of Cx401 to broaden the applicability of the product candidate as well as new applications for its adipose-derived stem cells. These new applications are centered on autoimmune and/or inflammatory diseases, particularly rheumatoid arthritis. Animal models have been developed and proof of concept has been demonstrated for treating this indication in rats.
Calle Marconi, 1, Parque Tecnológico de Madrid,
Tres Cantos, 28760 Madrid
Tel: +91 804 92 64 • Fax: +91 804 92 63
(Key Contact: Claudia Jimenez)
Euro Japan Marketing Ltd – Tokyo Office
AT Building 6F, 1-18-9 Nihonbashi-Ningyocho, Chuo-ku,
Tokyo 103-0013, Japan
Tel: +81 3 3664 5062 • Fax: +81 3 3664 5063
(Key Contact: Christopher Jackson)