Clinuvel Pharmaceuticals (ASX: CUV) has started a physician-led Phase II study of SCENESSE® drug for the treatment of the rare Hailey-Hailey Disease in Italy.
This study will enrol ten HHD patients to be treated with twelve doses of SCENESSE® during one year, with a three month clinical follow up period.
Recruitment of all patients is expected to complete by March 2014.
In 2012, the first proof of concept open-label pilot study of afamelanotidem - the active ingredient in SCENESSE® - was carried out in two female patients (age 53 and 61).
At the time of the start of the study, the patients both had existing skin lesions and ulcerations which had been present since their adolescent years.
Following afamelanotide treatment, the lesions began to visibly decrease in size and totally disappeared by day 60.
No adverse reactions were reported. Both patients experienced moderate skin tanning, as expected on the basis of the secondary pharmacology of SCENESSE®. Disease recurrence was only seen in the patients eight months after completion of the treatment.
Hailey-Hailey Disease
Hailey-Hailey Disease (HHD, also known as familial benign chronic pemphigus) is a rare, lifelong, inherited disorder where epidermal skin cells (keratinocytes) cannot properly adhere.
This causes periodic eruption of plaque-like lesions, blisters and ulcerations on areas where skin folds (flexural), often on the neck, armpits or groin. Most patients have permanent lesions. HHD usually appears in the third or fourth decade of life.
Current ineffective treatments include topical corticosteroids, antibiotics, and mTor inhibitors which attempt to manage minor outbreaks.
There is no professional consensus on a first-line therapy as no remission has been achieved in patients. Several literature reports indicate that HHD patients being predisposed to skin cancer.
The worldwide prevalence of HHD is 1:50,000.
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