Products / Pipeline

Thursday, 15 December 2011 09:06

Encouraging interim safety results of SCENESSE® in Phase II study in organ transplant recipients (OTRs)

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In organ transplant patients who experience a high rate of skin cancers and co-morbidity, no significant safety concerns were seen during first 12 months of consecutive use of SCENESSE®.

Melbourne, Australia and Baar, Switzerland, December 15, 2011

Executive summary

  • Blinded interim analysis of safety data from 12 months of treatment in OTR study
  • No major safety concerns were identified in this population
  • Treatment was well tolerated with the most frequent adverse events being abdominal pain, fatigue, headaches, nausea and vomiting, mostly mild in severity.
  • Final efficacy and safety results due by end of 2012.

Clinuvel Pharmaceuticals Limited (ASX: CUV; XETRA-DAX: UR9; ADR: CLVLY) today announced results from an interim safety analysis of data from its Phase II study evaluating the use of SCENESSE® (afamelanotide 16mg in controlled-release formulation) in organ transplant recipients (OTRs; study CUV011). The analysis was conducted on blinded safety data from patients who had completed at least 12 months of therapy. Results did not raise any significant safety concerns in this patient population.

In the CUV011 study SCENESSE® is being evaluated to determine if the drug can protect OTR patients from UV-induced damage, specifically actinic keratoses (AKs, premalignant skin cancers) and squamous cell carcinomas (SCCs) of the skin. Some patients have already completed the 24 month study with the final patient due to complete treatment by July 2012.

SCENESSE® is the first drug to be developed as an overall photoprotective agent in organ transplant patients.

Organ transplant recipients (OTRs) and skin cancer in Caucasian patients (fair-skinned)
Due to the essential long term use of immunosuppressant drugs to prevent organ rejection, OTR patients are at an extreme risk of skin cancer and other co-morbidities compared to the general population. It has been found that OTR patients are up to 250 times more likely to develop skin cancer than those who have not had an organ transplant. The most common skin tumours seen in patients post-transplantation are SCC, basal cell carcinoma (BCC) and melanoma. SCCs and BCCs are the direct result of chronic exposure of skin to UV light (280-400nm).

The highest incidence of skin cancers is seen in countries with high UV exposure. Skin cancers develop primarily on sun exposed areas and in transplant recipients with a history of high sun exposure. Transplant recipients with fair skin are at much higher risk for the development of skin carcinoma than are those with dark skin, suggesting that melanin offers protection to skin cells from UV light. The development of a prophylactic treatment or photoprotective therapy to diminish the rate of these severe skin disorders in OTRs is of high interest to the patient and medical community.

Study rationale and design
CUV011 is a randomised double-blind placebo-controlled study of 24 months duration in 85 patients, involving 10 specialist skin cancer centres across Australia and Europe. The first centre started in early 2008 and the last centre is expected to complete treatment by July 2012.

This study and its objectives were designed to analyse the prophylactic effect of SCENESSE® on the development of AKs and SCCs in Caucasian patients who are highly prone to develop these skin lesions after organ transplantation.

Interim safety results
Data from all 85 patients enrolled were evaluated in this analysis. with no significant safety concerns identified. A low rate of skin conditions, including all forms of skin cancer, was reported across the patient group and no melanomas were reported. Treatment has not been shown to interfere with immune suppressive medications. The most frequent adverse events seen were abdominal pain, fatigue, headaches, nausea and vomiting, mostly of mild severity, across the patient groups. Many of these adverse events have been reported as mild drug-related adverse events in other studies of SCENESSE®.

Clinuvel’s Chief Scientific Officer, Dr Hank Agersborg, said: “These first results are meaningful in many ways. Treatment in this study appears safe, an important parameter for a new drug in patients who are already susceptible to a range of disorders due to their lifelong immune suppression. Additionally, although we know that Caucasian patients are prone to experience multiple sequential skin cancers on sun exposed skin, there are only a few reports of skin tumours in the first 12 months. These results suggest that treatment does not interfere with systemic immunosuppressive drugs or with the donor organ graft. From a safety point of view, it was a novel and important approach to investigate the safety in this group of patients.”

Clinuvel’s Senior Vice-President of Scientific Affairs, Dr Dennis Wright, concluded: “Safety analyses are an essential part of evaluating a new drug prior to it being licensed on the market. These results contribute to our understanding of SCENESSE® when used longer term in patients. This is the first time that Clinuvel has evaluated its drug in a pool of patients who are known to have a high risk of systemic diseases and skin cancers. Further efficacy analyses will follow to establish the treatment effect on actinic keratoses..”

Next steps
These interim safety data from study CUV011 will be added to Clinuvel’s regulatory application for marketing authorisation for SCENESSE® for its lead indication of erythropoietic protoporphyria (EPP), which is expected to be filed with the European Medicines Agency in the coming weeks.

Final efficacy and safety data from the 24 months treatment in study CUV011 for organ transplant patients is expected to be available by the end of 2012.

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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 to 45 million. 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. Based in Melbourne, Australia, Clinuvel has operations in Europe and the US. For further information please visit

Investor relations contacts:

Australia: Clinuvel Pharmaceuticals Limited, T: +61 3 9660 4900
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Media contacts:

Lachlan Hay
Clinuvel AG
Baar, Switzerland
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Nick Miles
Cabinet Privé de Conseils s.a.
Geneva, Switzerland
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Ted Agne
The Communications Strategy Group Inc.
Boston, Massachusetts, USA
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