Preliminary Results in Clinuvel’s Phase III Porphyria Trial

Clinuvel Pharmaceuticals Limited (ASX: CUV; XETRA-DAX: UR9; ADR: CLVLY) today announced that it has obtained promising 4-months results in testing afamelanotide in a multicentre randomised double-blind placebo controlled Phase III study in EPP (CUV017). In 2008, afamelanotide was awarded orphan drug designation (ODD) in the treatment of EPP by the FDA and EMEA.

EPP patients are known to be absolutely intolerant to visible and UV light. This genetic disorder leads to an accumulation of protoporphyrin IX in the skin and an increased risk of incapacitating phototoxic reactions manifesting as pain and severe ulceration of the skin. As expected, the quality of life of EPP patients is much reduced by the significant restriction of activities that involve exposure to sunlight such as outdoor activities.

Recent worldwide specialty meetings (haematology, dermatology, gastro-enterology) have confirmed the strong need for an effective preventative treatment for EPP. No effective symptomatic treatment currently exists.

Preliminary Results An interim analysis of data from the first 4 months of treatment (March-October) in this 12 month trial was undertaken. The study employed a crossover comparison of afamelanotide (3 doses) and placebo (3 doses) in 100 patients. At dose intervals of 2 months, each patient received a single dose of either treatment.

Analysis of 2 treatment arms showed an overall reduction in the average number of phototoxic reactions. Thirty five patients with severe and/or moderate pain reported the greatest reduction in mean number of reactions (p=0.03, 95% CI). Analysis of pain severity was positively correlated with treatment, indicating that patient pain scores differed significantly between treatment groups (p=0.006, 95% CI).

Although the analysis of quality of life data is not yet complete, all 8 physicians involved in this trial reported a dramatic improvement in the patients’ ability to engage in outdoors activities. Safety reports from all academic centres are excellent to date.

This study will be completed by the end of December 2009, after which a full analysis of data will follow.

Clinuvel’s Chief Scientific Officer, Dr Hank Agersborg said: ”The impact of this life-long disease is seen and reported in our trials by the adult patients who have been unable to lead a normal pain-free existence since early childhood.”

“These first statistical analyses confirm the overwhelmingly positive anecdotal reports during the trial. The patients’ response to our novel therapy and the physicians’ assessment will play a significant role in the regulatory review process.”

Managing Director, Dr Philippe Wolgen said: ”I am excited by the positive response stated by the physicians and patients during this therapy. Part of the clinical benefit seen in EPP is attributable to our choice of a unique controlled release delivery formulation.”

“We will obtain the full EPP analyses and incorporate these data in our next regulatory filing. Clinuvel plans to start confirmatory EPP trials (CUV029) in several European countries in the spring and summer, while we prepare continuation of our US program in 2010.”