Clinical Trial Results

Clinuvel has completed five Phase II clinical trials of SCENESSE® (afamelanotide) in four clinical indications – erythropoietic protoporphyria (EPP), polymorphous light eruption (PLE), solar urticaria (SU) and photodynamic therapy (PDT). These studies are summarised below:

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 2007 Clinuvel completed a Pilot Phase II study (CUV010) of SCENESSE® in Europe assessing the drug’s ability to reduce the number and severity of phototoxic reactions in EPP. In all five patients, the time to provoke pain (photoprovocation) was markedly prolonged (range: 384% - 1266%). Read the company’s Phase II EPP results announcement.

Results from this study have been published in the New England Journal of Medicine and Photochemistry Photobiology.

Clinuvel’s erythropoietic protoporphyria program in Europe has progressed into a Phase III and subsequent confirmatory trial program. You can view the Phase III results here. Read more about our erythropoietic protoporphyria program for SCENESSE®.

In 2011 Clinuvel completed a Phase II study (CUV030) of SCENESSE® in adult US EPP patients. This six-month, randomised, multicentre, double-blind, placebo-controlled US study (CUV030) was primarily designed to confirm the efficacy and safety of subcutaneous bioresorbable afamelanotide implants (SCENESSE®) in reducing the severity of phototoxic skin reactions in patients with the rare light intolerance disorder erythropoietic protoporphyria (EPP), allowing them to lead ‘more normal’ lives. Results of the study showed that SCENESSE® was well tolerated, allowed EPP patients to expose their skin to sunlight during the middle of the day and improved their Quality of Life (QoL). Read the company's Phase II US EPP results announcement.

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 2006 Clinuvel completed two Phase II studies of SCENESSE® in patients diagnosed with PLE. Clinuvel’s polymorphous light eruption program progressed into Phase III in 2007. Read more about our polymorphous light eruption program for SCENESSE®.

Solar urticaria (SU)

Solar urticaria is a photodermatosis, with diverse clinical presentations and causes. SU is a rare subset of physical urticaria, where symptoms are induced by direct exposure of the skin to sunlight. As little as 5 minutes of sun exposure can cause flares and whealing on exposed skin sites, accompanied by severe itching. These symptoms can vary in manifestation, and anaphylaxis is a clinical risk. The wavelengths of radiation causing the severe skin eruption (i.e. the action spectrum) are in the ultraviolet or visible light range. SU may have a very sudden and dramatic onset, and may rapidly disappear once exposure ceases. A delayed form of SU has also been reported. Read more about solar urticaria.

In 2009 Clinuvel completed a Phase II study of SCENESSE® in 5 patients diagnosed with SU. Read the company’s Phase II solar urticaria results announcement.

Results from this study have been published in the British Medical Journal.

Clinuvel’s solar urticaria program was deferred in February 2010.

Photodynamic therapy (PDT)

In PDT, a photosensitising drug (Photofrin^TM) is administered intravenously to enhance and accelerate tumour treatment by LASER illumination. The photosensitiser in the tumour absorbs the light and produces an active form of oxygen that destroys nearby cancer cells.

Photosensitising agents such as porfimer sodium (Photofrin^TM) make skin and eyes ultra sensitive to light for up to 90 days following treatment. Patients suffer intense pain and second degree burns associated with this phototoxicity and are forced to avoid sunlight/artificial light for up to 90 days following treatment.

The main advantages of PDT over other cancer therapies include the significant degree of selectivity of drug accumulation in the tumour tissue, and the ability to retreat a recurrent tumour. PDT has proven valuable as a treatment option in cancers such as esophageal cancer, gastric, endobronchial, papillary bladder and gliomas. Phototoxicity of the skin is the dominant and clinically significant side effect of PDT and precludes wider use of the therapy in these patients.

In 2009 Clinuvel completed a Phase II study of SCENESSE® in patients undergoing PDT. Read the company’s Phase II PDT results announcement.

Clinuvel’s PDT program was deferred in February 2010.

Clinuvel has completed four Phase I, pharmacokinetic and pharmacodynamic trials of SCENESSE® (afamelanotide). These studies, conducted in 178 patients in Australia and the United States have been used to determine the early safety profile of afamelanotide in man as well as the optimal dose for the SCENESSE® implant as an effective photoprotective and repigmentary therapeutic.