Clinuvel Pharmaceuticals has obtained positive 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 in the treatment of EPP by the FDA and EMEA.
An interim analysis of data from the first four months of treatment (March-October) in the 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 two months, each patient received a single dose of either treatment.
Analysis of two treatment arms showed an overall reduction in the average number of phototoxic reactions. Over 35 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 eight physicians involved in this trial reported a dramatic improvement in the patients’ ability to engage in outdoors activities.
This study will be completed by the end of December 2009, after which a full analysis of data will follow, said the company.
Hank Agersborg, chief scientific officer of Clinuvel, said: “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.”
Philippe Wolgen, managing director of Clinuvel, 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.”