Clinuvel’s Scenesse sees positive trial results

Clinuvel Pharmaceuticals (ASX:CUV) today announced positive results from its first phase II clinical study of its novel photoprotective drug, afamelanotide, now known as Scenesse.

The six-month, randomised, multicentre, double-blind, placebo-controlled U.S. study was primarily designed to confirm the efficacy and safety of subcutaneous bioresorbable afamelanotide implants in reducing the severity of phototoxic skin reactions in patients with the rare light intolerance disorder erythropoietic protoporphyria (EPP), allowing them to lead ‘more normal’ lives.

Results of the study showed that Scenesse was well tolerated, allowed EPP patients to expose their skin to sunlight during the middle of the day and improved their Quality of Life (QoL).

Read more about Clinuvel and afamelanotide/Scenesse.

Seventy-seven adult patients diagnosed with EPP participated in the trial during six months of spring and summer in six centres across the U.S.. Sixty-eight patients completed the trial, the drop-out rate of 12 per cent being consistent with previous studies and suggesting the good tolerability of the drug.

Patients in two parallel study groups were administered either the afamelanotide implant or a placebo at the start of study, after 60 days and 120 days, and were subsequently followed up to 180 days.

The patients were asked to record the number and severity of reactions experienced as well as the duration of time they spent outside, exposing their skin to sunlight.

Patients were also asked to complete regular Quality of Life (QoL) surveys. These measurements were used to determine whether the drug reduced the impact of EPP on patients’ lives compared to baseline and compared to placebo. Safety and tolerability of afamelanotide was evaluated by measuring treatment-emergent adverse effects (AEs). The results showed the drug to be safe and well-tolerated.

Importantly, no drug related serious safety concerns have been identified to date with Scenesse in all global clinical trials. To date over 600 patients have been treated with Scenesse across a number of indications.

“We are pleased with the outcome of this trial as it adds to the mounting body of evidence that afamelanotide is effective as a prophylactic treatment for EPP in the clinic and allows a better quality of life to those who suffer from a terrible disorder which is not well understood by the general public,” said Clinuvel’s CEO, Dr Philippe Wolgen.

“Scenesse acts as a protective umbrella for the ultra light sensitive skin of these patients, and enables them to participate in a life they have never known before. Proposing a new pharmaceutical therapy which is safe and clinically relevant to EPP patients is Clinuvel’s most important objective, since we will be expected to demonstrate a positive risk-benefit ratio as part of our European regulatory submission later this year.”

Erythropoietic protoporphyria (EPP) is a rare life-long 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.

Reactions can vary from mild to extreme with hospitalisation and powerful pain killers and morphine required in severe cases. Children and adults living with EPP must avoid sunlight and even reflected light, often staying indoors or wearing protective clothing.

Conventional sunscreens have no effect. Approximately 10,000 people globally are affected by EPP, an estimated 4,000 in the US. Presently, there is no known effective treatment for EPP.

Scenesse has been granted orphan drug status both in the US and Europe. Scenesse activates melanin in the skin, which shields against UV radiation (UVR) and sunlight, while the drug is delivered as a subcutaneous, dissolving implant approximately the size of a rice grain. Increased pigmentation of the skin may appear after two days and the therapeutic effect lasts up to two months.

Clinuvel (ASX:CUV) shares are steady at $1.50 in morning trading.