Clinuvel Commences US Phase III Trial of SCENESSE® in Rare Light and UV Disorder

Melbourne, Australia and Baar, Switzerland, May 22 2012 -- Clinuvel Pharmaceuticals Limited (ASX: CUV; XETRA-DAX: UR9; ADR: CLVLY) today announced that it has commenced its confirmatory Phase III US study of the novel drug SCENESSE® (afamelanotide) in patients diagnosed with the rare light intolerance disorder erythropoietic protoporphyria (EPP). The six-month, randomised, multicentre, double-blind, placebo-controlled study (CUV039) will recruit up to 100 adult EPP patients in seven specialist centres (Alabama, California, Michigan, New York, North Carolina, Texas and Utah).

The US Food and Drug Administration (FDA) allowed the trial to proceed earlier this month. It is expected that the treatment of all patients will be completed before the end of 2012.

“This Phase III trial protocol has been designed in close consultation with the FDA,” Clinuvel’s Chief Scientific Officer, Dr Hank Agersborg said. “We anticipate that the results will confirm the safety and efficacy profiles seen in previous trials and enable us to file a New Drug Application (NDA) for the drug in the US.

“Clinuvel is working with all study sites to facilitate recruitment of patients during early summer. This period of the year is a particular burden to EPP patients who are prone to incur severe skin reactions when exposed to sunlight,” Dr Agersborg said.

Erythropoietic protoporphyria (EPP)

EPP is a rare genetic disease found mainly in fair-skinned people. It is characterised by severe phototoxicity (intolerance to light) of the skin resulting in intolerable pain, swelling and scarring, usually of exposed areas such as the face, hands and feet. Symptoms can vary from mild to extreme lasting pain requiring hospitalisation. Patients often lead an indoor and sheltered life, avoiding light and UV exposure to prevent symptoms. Presently there is no known effective treatment for EPP, which affects approximately 10,000 people globally.

Results were recently announced from pivotal Phase II US and Phase III EU trials (CUV029 and CUV030). These showed SCENESSE® could reduce the severity of EPP symptoms and enable patients to lead more normal lives. A marked improvement in quality of life was also reported. Thus far, no serious safety concerns have been identified from the use of afamelanotide in more than 650 patients, including more than 250 EPP patients, involved in various clinical trials. In February, Clinuvel submitted a marketing authorisation application for SCENESSE® for EPP with the European Medicines Agency. SCENESSE® has been granted Orphan Drug Status in the US and Europe.