Clinuvel: positive interim Phase III results for unique skin protection drug
Photoprotective drug afamelanotide moves a step closer to market
Melbourne-based Clinuvel Pharmaceuticals Limited achieved positive interim results of its photoprotective drug, afamelanotide, in a Phase III trial conducted in Switzerland. The trial investigated the medical benefits of afamelanotide in the orphan disease erythropoietic protoporphyria (EPP), a severe genetic disorder causing absolute ultraviolet radiation (UV) and light intolerance in the skin. This is the company’s first Phase III test result.
People with EPP, a rare disease, suffer phototoxicity, painful symptoms from sunlight and UV exposure. Phototoxicity usually starts in early childhood with patients experiencing severe burning pain and blistering of the skin, typically on the face and hands. Severe symptoms may last for several days and may be accompanied by swelling and redness on sun exposed areas. EPP is exacerbated in the spring and summer months.
The drug afamelanotide stimulates the body’s own natural melanin production. Melanin is known for its photoprotection as it absorbs ultraviolet radiation, shielding the skin from the effects of UV. This offers EPP patients the possibility of a better quality of life where they don’t have to avoid the sun or live in the shadows.
The interim results are based on the trial’s first 14 Swiss patients, with a 12 month study period analysed. A further 87 patients will complete the trial in Europe and Australia. The European and Australian Phase III EPP trial is expected to be completed in the second half of 2009.
The results showed that the maximum severity of phototoxic reactions for patients on the drug was significantly reduced compared to patients on the placebo (p<0.001). Further, the total severity of phototoxic reactions was reduced during spring and summer by afamelanotide, compared with placebo (p=0.028). Potentially, the drug offers EPP patients the possibility of an existence where exposure to light and UV become tolerable, and where patients are not confined to life indoors. The patients were tested at higher altitude where ultraviolet radiation is more intense. The result showed a statistically significant reduction in the severity of phototoxic reactions in EPP patients and increased melanin density in the skin.
At the end of the study all 14 Swiss patients requested continuation of the drug for photoprotection for the next 12 months. This compassionate use request was granted by SwissMedic, the Swiss regulatory agency.
An independent Data and Safety Monitoring Board has reviewed the data on the safety and efficacy of afamelanotide in this clinical setting and considered it appropriate, and of benefit to patients, to continue the study to its conclusion. Clinuvel hopes to file for the drug’s approval in Europe in the next 18 months.
Clinuvel has identified five UV and light related disorders where clinical use of afamelanotide serves the needs of patients who suffer severe and chronic symptoms. One of the most severe categories is organ transplant recipients who endure high incidence of skin cancers, due to their life-long reliance on immune suppressing drugs in combination with UV daily exposure.
Clinuvel anticipates commencing trials in the US in early 2009, pending the US Food and Drug Administration’s (FDA) approval of its first Investigational New Drug (IND) application, lodged in December 2008. A successful outcome will be important progress in ultimately marketing afamelanotide in the US as well as the rest of the world.
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