Update: SCENESSE® in DNA Repair

CLINUVEL PHARMACEUTICALS LTD provided today a clinical update on the use of its drug SCENESSE®(afamelanotide 16mg) in the first xeroderma pigmentosum (XP-C) patient receiving treatment as part of the Company’s DNA Repair Program.1

Ongoing safety assessment
The first male XP-C patient started SCENESSE® treatment in September under a Special Access Program and has been closely monitored by the expert clinical centre responsible for medical care. Regular clinical observations have been made over the 42-day treatment period to assess the patient’s health, including the response to overall afamelanotide treatment.

The patient tolerated the melanocortin drug well and no drug related adverse events have been reported. Specific attention has been given to the consequences of ultraviolet (UV) exposure, pigmentation, and overall status of the patient’s skin. XP patients are known to exhibit poikiloderma (degeneration and disintegration of the skin) and are prone to frequent bleeding from chronic wounds. At the end of the 42 days, the integrity of the skin of the XP-C patient has shown to be unaffected by afamelanotide.

Commentary
“We are delighted with the consistent safety reports from the XP-C patient receiving systemic treatment with afamelanotide,” CLINUVEL’s Clinical Operations Manager, Dr Pilar Bilbao said.“This positive observation and clinical feedback from the treating physician form the basis for progressing the planned XP study, CUV150.”

Xeroderma Pigmentosum (XP) & DNA repair.
XP patients exhibit extreme deficiency in repair of UV-provoked damage to the DNA helix within the nucleus of skin cells. If left unrepaired, damaged DNA replicates and significantly increases the risk of skin cancers, including melanoma in these patients. XP-C is one of eight XP variants (XP-A to G, and V), reflecting different genes involved in the DNA repair process nucleotide excision repair (NER).

Due to the inability to initiate or complete the NER process, XP patients are at approximately 10,000-fold risk of developing non-melanoma and melanoma skin cancers. Most XP patients will experience the first skin cancer before adolescence, while the leading cause of death remains progressive non-melanoma skin cancers and melanoma in the third decade. Due to the extreme rate of these malignancies, surgical intervention is frequently required, resulting in loss of extremities, facial anatomy such as ears, and eyesight.

Scientific evidence supports the use of afamelanotide, the active ingredient in SCENESSE®, to protect skin from UV and light (systemic photoprotection), and repair UV-induced DNA damage. Further details of CLINUVEL’s DNA Repair Program will be provided in a Strategic Update, to be released this month.

1 SCENESSE® (afamelanotide 16mg) is approved in the European Union as an orphan medicinal product for the prevention of phototoxicity in adult patients with erythropoietic protoporphyria (EPP). SCENESSE® is approved in the USA to increase “painfree” light exposure in adult EPP patients with a history of phototoxicity. Information on the product can be found on CLINUVEL’s website at www.clinuvel.com.
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