Clinuvel reports strong Phase III results for Scenesse

Melbourne biotech Clinuvel has reported positive results from a Phase III trial of its UV protection drug Scenesse in treating sufferers of the rare genetic disorder erythropoietic protoporphyria (EPP).

EPP is a painful and debilitating condition which sees sufferers develop skin burns, blisters and scars when exposed to normal levels of sunshine and light. The 12-month Australian and European study is the first large scale study of a preventative drug for EPP, which is classified as an ‘orphan’ disease because of its rarity and severity. There are currently an estimated 10,000 cases of EPP worldwide.

The global trial of Scenesse involved 91 patients in Australia, Italy and other countries. At the Italian Porphyria Centre at San Gallicano Dermatology Institute (IFO) in Rome, 22 patients underwent treatment with Scenesse, with such startling results that the drug was immediately approved for reimbursement through Italy’s healthcare system. It was the first time that an EU country had approved a drug still in trial phase.

Analysis of results from the Clinuvel study showed that Scenesse treatment significantly reduced the average daily pain severity scores experienced by EPP patients compared to placebo. Further results suggested that the drug allowed EPP patients to expose their skin to sunlight and spend more time outdoors, leading to dramatic improvements in quality of life. Finally, the study presented no tolerance or safety concerns.

“We have shown mathematically what we already learned from anecdotal reports from our clinics: Scenesse has great potential to help those patients with a genuine medical need for protection from UV and light,” said Dr Hank Agersborg, Clinuvel’s Chief Scientific Officer. “Perhaps more importantly, we have again seen that the drug is safe for these patients longer-term.”

Last week Clinuvel announced its first manufacturing deal for Scenesse with Minnesota-based U.S partner SurModics.

Clinuvel said that complete results from the study will be presented at the 19th Congress of the European Association for Dermatology and Venereology in Gothenburg, Sweden, in October.