Clinuvel has completed three Phase III clinical trials of SCENESSE® (afamelanotide).
Erythropoietic protoporphyria (EPP)
Erythropoietic protoporphyria (EPP) is a rare genetic disease found mainly in people with fair skin. It is characterised by severe phototoxicity (or intolerance to light) of the skin resulting in intolerable pain, swelling, and scarring, usually of the exposed areas such as the face, hands and feet. The pain experienced and expressed by EPP patients when their skin is exposed to light is reported as intolerable. EPP patients are often forced to remain indoors, severely affecting their quality of life. Read more about erythropoietic protoporphyria.
In 2010 Clinuvel completed its first Phase III study of SCENESSE® (afamelanotide) in patients with erythropoietic protoporphyria (CUV017). A total of 91 patients completed the 12-month study, in which an 11-point Likert scale and physician assessments through case report forms (CRF) were used to evaluate pain as a principal symptom of phototoxicity. The duration of daily (sun)light exposure was used to assess the willingness of patients to expose themselves during all seasons. Melanin density (reflecting changes in skin pigmentation, measured by spectrophotometry) and quality of life (Short Form 36 surveys) were also evaluated.
In an analysis of the total number of days (frequency distribution) on which patients experienced pain in the specific pain severity categories (severe, moderate, mild and none), a significant reduction of frequency was observed in patients on active drug [p=0.0023]. Characteristic to EPP, the majority of phototoxic reactions occurred during spring and summer.
In analysing the average pain severity experienced by the total number of patients, the assessment of all individual daily pain scores was significantly lower in patients receiving SCENESSE® compared to those receiving placebo [p=0.0017].
An additional evaluation of the pain scores in patients willing to modify behaviour by continuous exposure to daily (sun)light showed a positive trend toward a reduction in average pain score following active drug treatment [p=0.1654]. You can view the full Phase III results from our EPP study here.
A confirmatory European Phase III study of SCENESSE® (afamelanotide) in EPP patients (CUV029) was completed in 2011. Patients from eight study sites were allocated at random to either the afamelanotide or placebo group, being treated every 60 days. Of the 74 patients initially enrolled, 68 [91.9%] completed the medication and clinical visits.
The study, which ran for nine months, demonstrated that SCENESSE® was once again well tolerated by patients, with no safety concerns identified during this period. The study primarily evaluated whether the prophylactic treatment of EPP provided a clinical benefit which was meaningful, with the impact of afamelanotide on the QoL also assessed using a specialised EPP-specific QoL questionnaire.
Patients receiving afamelanotide experienced half as many phototoxic reactions as placebo recipients [p=0.044] and had a lower total median pain score [6.0 v 17.5; p=0.035]. They were also able to spend more time in direct sunlight without pain [10 AM-3 PM, p=0.005] and reported greater improvement in their Quality of Life [Day 270, p=0.011].
You can read the full results of the Phase III EU EPP study here.
Read more about our erythropoietic protoporphyria program for SCENESSE®.
Polymorphous light eruption (PLE)
PLE is the most common recurrent photodermatosis causing sensitivity and, after sunburn (solar erythema), is the most common sun-related problem seen by physicians. PLE is a distressing seasonal skin condition with episodes typically beginning in spring and resolving by late-summer or autumn, and symptoms include non-scarring, burning red papules, vesicles or plaques which appear on sun-exposed skin 30 minutes to several hours following exposure to sunlight. Read more about polymorphous light eruption.
In 2009 Clinuvel released preliminary results from a Phase III study of SCENESSE® (afamelanotide) in patients diagnosed with polymorphous light eruption (CUV015). You can view the Phase III preliminary results from our PLE study here.
Read more about our polymorphous light eruption program for SCENESSE®.