Preliminary Results in Clinuvel’s Phase III Porphyria Trial
December 21 2009 - 6:10AM
Business Wire
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.”
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