CUV Clinuvel Pharmaceuticals Limited

Clinuvel Pharmaceuticals Limited (ASX: CUV; XETRA-DAX: UR9; ADR: CLVLY), a global biopharmaceutical company with unique expertise on the interaction of light and human skin, today announced positive results of its first US Phase II study of the novel photoprotective drug afamelanotide, known as SCENESSE®.

The six-month, randomised, multicentre, double-blind, placebo-controlled US study (CUV030) was primarily designed to confirm the efficacy and safety of subcutaneous bioresorbable afamelanotide implants (SCENESSE®) 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.

Commenting on the results, Dr Robert J Desnick, Dean for Genetic and Genomic Medicine and Professor and Chairman Emeritus of the Department of Genetics and Genomic Sciences at Mount Sinai School of Medicine, New York and a lead investigator on the CUV030 study, said, “This clinical study indicates the effectiveness of the drug in preventing the severe pain resulting from sun exposure. This is important for patients as it is the first treatment for EPP that will improve their now limited quality of life."

Clinuvel’s CEO, Dr Philippe Wolgen, said, “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. 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.”

Severe Genetic Disease with no Current Therapy

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.

Results of Phase II US study (CUV030): protecting skin, improving Quality of Life

Seventy-seven adult patients diagnosed with EPP participated in the trial during six months of spring and summer in six centres across the US in Alabama, California, New York, North Carolina Texas and Utah. Sixty-eight patients completed the trial, the drop-out rate of 12% being consistent with previous studies and confirming 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.

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). Overall the study demonstrated a strong clinical benefit to patients, despite their deeply learned behaviour to avoid reactions caused by sun exposure.

Based on analysis of time spent outside, afamelanotide was shown to increase patients’ ability to expose their skin to direct sunlight. Patients who received afamelanotide spent significantly more time in direct sunlight between the most intense hours of 10 AM and 3 PM (p=0.036) and between 10 AM and 8 PM (p=0.025). Patients on drug reported a three-fold increase in the median amount of time in direct sunlight compared to placebo. Consequently many patients on drug reported no pain or only mild pain compared to their previous life of experiencing severe phototoxic reactions.

The results also show that SCENESSE® improved EPP patients’ QoL. Data from an EPP-specific quality of life assessment tool demonstrated a dramatic improvement from baseline for patients on the drug compared to the placebo group at 60 days (p=0.001), 120 days (p=0.003) and 180 days (p

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