Clinuvel announces positive results of first Phase II US study of SCENESSE® in orphan disease erythropoietic protoporphyria (EPP

Clinuvel announces positive results of first Phase II US study of SCENESSE® in orphan disease erythropoietic protoporphyria (EPP)
New drug improves quality of life, allows patients exposure to sunlight
Baar, Switzerland and Melbourne, Australia, November 3 2011
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<0.001).
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.
An independent Data Safety Monitoring Board (DSMB) reviewed and confirmed the analyses and deemed the drug safe for further use on the basis of the adverse event profile seen in this and other studies.
Results from a confirmatory Phase III study (CUV029) in Europe are expected in the near future. Pending these European results, together with data from this US study (CUV030), Clinuvel plans to file SCENESSE® as a prophylaxis of the phototoxic symptoms of EPP before the end of 2011 for review by the European Medicines Agency.
About Erythropoietic Protoporphyria (EPP)
EPP is caused by an enzyme (ferrochelatase) deficiency which leads to a build-up of protoporphyrin IX (PPIX) in the body, mostly in the skin and liver. PPIX is phototoxic in skin; when exposed to light PPIX reacts with varying intensity which can cause excruciating pain. EPP patients describe the symptoms as an unimaginable pain for which no painkillers can offer relieve. EPP patients are often forced to lead an indoors existence, severely affecting their quality of life.
SCENESSE® (afamelanotide) is a first-in-class dermatological drug being developed solely by Clinuvel. SCENESSE® activates melanin in the skin, which shields against UV radiation (UVR) and sunlight. SCENESSE® is delivered as a subcutaneous, dissolving implant approximately the size of a rice grain. Increased pigmentation of the skin appears after two days and lasts up to two months.
The active ingredient in SCENESSE® is afamelanotide, a chemical analogue of α-MSH, a linear peptide with 13 amino acids. Two amino acids in α-MSH have been changed to produce afamelanotide which has more potent biological effects and a much longer half life than naturally occurring α-MSH. This allows SCENESSE® to increase the skin's melanin content, even when delivered at a remote location on the body and without exposing the skin to the damaging effects of UVR. Afamelanotide was granted orphan drug status in 2008 in Europe and in the US for the treatment of EPP.
Since 2006, Clinuvel has conducted global clinical trials with SCENESSE® in over 250 EPP patients. To date, Phase II and III clinical trials have shown that the drug can reduce the incidence and severity of phototoxic reactions in EPP, and importantly, can assist EPP patients in leading a more normal life. Pending results from these current clinical studies, Clinuvel intends to file SCENESSE® for marketing authorisation in Europe in late 2011. SCENESSE® is a registered trademark of Clinuvel Pharmaceuticals Ltd.
About Clinuvel Pharmaceuticals Limited
Clinuvel Pharmaceuticals Ltd (ASX: CUV; XETRA-DAX: UR9; ADR: CLVLY) is a global biopharmaceutical company focused on developing drugs for the treatment of a range of severe skin disorders. With its unique expertise in understanding the interaction of light and human skin, the company has identified three groups of patients with a clinical need for photoprotection and another group with a need for repigmentation. These patient groups range in size from 10,000 with a need for photoprotection to 45 million who require repigmentation. Clinuvel's lead compound, SCENESSE® (afamelanotide), a first-in-class drug targeting erythropoietic protoporphyria (EPP), is in Phase II and III trials in the US and Europe, and is expected to be filed before the end of 2011 for review by the European Medicines Agency.  Presently, there is no known effective treatment for EPP and SCENESSE® has been granted orphan drug status. Based in Melbourne, Australia, Clinuvel has operations in Europe and in the US. For more information please visit
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Media note: Dr Robert J Desnick is available for interview.
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Clinuvel is an Australian biopharmaceutical company focussed on developing its photoprotective drug, SCENESSE® (afamelanotide) for a range of UV-related skin disorders resulting from exposure of the skin to harmful UV radiation. Pharmaceutical research and development involves long lead times and significant risks. Therefore, while all reasonable efforts have been made by Clinuvel to ensure that there is a reasonable basis for all statements made in this document that relate to prospective events or developments (forward-looking statements), investors should note the following:
·          actual results may and often will differ materially from these forward-looking statements;
·          no assurances can be given by Clinuvel that any stated objectives, outcomes or timeframes in respect of its development programme for SCENESSE® can or will be achieved;
·          no assurances can be given by Clinuvel that, even if its development programme for SCENESSE® is successful, it will obtain regulatory approval for its pharmaceutical products or that such products, if approved for use, will be successful in the market place