Breakthrough Australian drug to start US trials

Clinuvel’s afamelanotide to be tested for absolute light intolerance in US

A novel Australian drug to protect patients who are intolerant of light and sunlight will begin trials in the US next month. The US Food and Drug Administration (FDA) has given clearance to Clinuvel Pharmaceuticals of Melbourne to start a Phase II study (CUV030) of its drug, afamelanotide, in up to 60 patients diagnosed with the rare light intolerance erythropoietic protoporphyria (EPP).

The six-month study, to be conducted in medical centres in Alabama, California, New York, North Carolina, Texas and Utah, is the first therapeutic trial of afamelanotide in the US.

It is estimated that around 3,000 Americans are afflicted with EPP, which is incurable and affects patients for life. Sunscreens are of no use in this disease as because they do not block out visible light (in the blue spectrum), which is the cause of toxic reactions in EPP.

Clinuvel has spent more than A$70m in the past 10 years developing afamelanotide, which works to activate a barrier of pigmentation (melanin) between light and a person’s skin. The implantable drug, the size of a rice grain, is able to stimulate and increase skin pigmentation in fair- skinned patients who are less protected from UV damage.

Afamelanotide has been administered to more than 500 patients to date in clinical trials in Europe and Australia, with preliminary Phase III results showing good safety and first signs that EPP symptoms can be prevented.

‘Clinuvel’s entry to the US to conduct a therapeutic trial is a major step in the company’s existence. In afamelanotide we are developing a unique drug to serve patients whose skin is most affected by ambient light,’ said Clinuvel’s ceo, Dr Philippe Wolgen.