Trial confirms efficacy of Clinuvel drug in EPP

Clinuvel‎ (ASX:CUV) has announced positive results from a phase III trial of its Scenesse drug in patients with the rare and painful skin condition ererythropoietic protoporphyria (EPP).

The trial in 87 adult EPP sufferers shows that treatment with Scenesse improves patients’ ability to expose their skin to light.

EPP is a rare genetic disease characterised by a severe intolerance of light. Symptoms include swelling, scarring and mild to severe lasting pain, forcing patients to remain indoors to avoid light exposure.

But the trial results show that patients treated with Scenesse (afamelanotide) can spend more time outside and experience less pain, according to trial lead investigator Dr Robert J Desnick.

“Professionally this is satisfying, as we may now, finally, be able to tell EPP patients that we can manage or prevent their painful symptoms and give them a freedom never before experienced,” he said.

Desnick is Dean for Genetic and Genomic Medicine and Professor and Chairman Emeritus of the the Department of Genetics and Genomic Sciences at The Icahn School of Medicine at Mount Sinai ‎in New York.

The primary endpoint of the trial was the extent to which patients could expose their skin to direct sunlight between 10 am and 6 pm. Patients receiving Scenesse in the double-blind, placebo-controlled trial demonstrated a strong trend towards greater direct sunlight exposure.

Patients also reported improved quality of life using the validated EPP-Quality of Life questionnaire. Scenesse also demonstrated a good safety profile, with headaches and nausea the most common adverse events.

Scenesse has been granted orphan drug status in EPP in the US and the EU, and Clinuvel is awaiting approval for a marketing authorisation application for the product in the EU.

Clinuvel (ASX:CUV) shares were trading 4.79% higher at $1.53 as of around 1.30 pm on Monday.