CLINUVEL Progresses DNA Repair Program

CLINUVEL PHARMACEUTICALS LTD today announced that it has received the necessary regulatory and ethics committee approvals to commence a new study in patients with the rare DNA repair disorder Xeroderma Pigmentosum (XP).

The Phase II study (CUV156) will evaluate the safety of SCENESSE® (afamelanotide 16mg)¹ in XP-C patients, as well as the drug’s ability to assist reparative processes following ultraviolet (UV) provoked DNA damage of the skin. Following the lifting of pandemic restrictions in hospitals, the first XP-C patients are expected to be screened by the end of October.

“We have used the time to add more parameters to this trial, and patients and clinicians are very excited to start this innovative study, evaluating afamelanotide as a treatment for Xeroderma Pigmentosum,” CLINUVEL’s Head of Clinical Operations, Dr Pilar Bilbao said. “XP is a severely life-limiting disorder without approved treatments. The regulatory authorities and ethics committees have been understandably cautious to subject these patients to interventional studies but have given their clearance to commence this program.

“Following the first XP patient treated safely in 2020, SCENESSE® will be evaluated in XP patients as part of our DNA repair program. It has been a long time in the making to treat this group of patients.”

UV radiation damages the nucleus of skin cells, and causes defects of the DNA helix known as “photoproducts”. If left unrepaired, these chemical DNA changes may replicate as mutations, leading to irreversible damage (photoaging) and progression to skin cancers, including melanoma.

Due to eight distinct genetic defects severely impairing their DNA repair mechanisms, XP patients are at the most extreme risk of skin cancers from an early age and have a life expectancy of around 30 years. Most patients are lifelong shielded from all UV light exposure.

The CUV156 study aims to evaluate the effect of SCENESSE® administered every two weeks to six patients with the XP-C complementation. CLINUVEL will assess whether SCENESSE® increases the quantum of UV light needed to cause DNA damage of skin cells, as well as the extent of skin repair before and after treatment. The CUV156 study will be conducted in a single European expert clinic.

CUV156 is the first in a set of studies planned as part of the DNA repair program. The CUV150 study was initially planned to be conducted in early 2021, but the delays incurred by the hospital due to COVID-19 offered the opportunity to improve study design and add more analytical parameters, following consultation with XP clinical experts.

¹SCENESSE® (afamelanotide 16mg) is approved in the European Union and Australia 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 “pain- free” light exposure in adult EPP patients with a history of phototoxicity. Information on the product can be found on CLINUVEL’s website at