Pilot study evaluating afamelanotide as adjunct therapy in systemic photodynamic therapy (PDT)
Clinuvel Pharmaceuticals Limited (ASX: CUV; XETRA-DAX: UR9; ADR: CLVLY) today announced that it had obtained positive results from an experimental randomised placebo-controlled Phase II trial (CUV025) evaluating the photoprotective effect of afamelanotide in 16 patients.
Objectives and results
The objective of this exploratory trial was to determine the effect of a single 16mg dose of afamelanotide on the quality of life and phototoxicity in patients undergoing PDT. The trial was conducted by 4 French academic departments of gastro-enterology. Patients were followed up during 90 days.
In total 16 Caucasian patients were included, 9 patients were administered afamelanotide and 7 patients placebo treatment as a subcutaneous implant at the same time as the photosensitising agent PhotofrinTM.
Post-operative analysis at 7 and 12 days revealed a positive trend to tolerate ambient light at standardised exposure by 7 out of 9 patients receiving afamelanotide. In patients on active drug, a significant improvement in quality of life assessment was demonstrated at 60 days of treatment (p=0.02). Clinical observations from all physicians and reports from patients supported and encouraged further use of afamelanotide in PDT cancer trials. No significant drug-related adverse events were reported.
In oncology, treatment of cholangiocarcinoma (bile duct cancer) and esophageal cancer remains clinically challenging. Recently, with systemic photodynamic therapy (PDT) remarkable progress has been made in the palliative treatment of advanced stage cholangiocarcinoma, where a median time survival of 498 days has been reported in PDT as opposed to 98 days with conventional therapy.1
In PDT, a photosensitising drug (PhotofrinTM) is administered intravenously to enhance and accelerate tumour treatment by LASER illumination. This photosensitisation restricts patients to an indoor life for up to 90 days after treatment, or they risk phototoxic reactions and second degree burns. Phototoxicity of the skin is the dominant and clinically significant side effect of PDT and precludes wider use of the therapy in these patients.1,2
Clinuvel’s Chief Scientific Officer, Dr Hank Agersborg said:
“These results are meaningful as they statistically confirmed the benefits of the adjunctive use of afamelanotide in a small group of oncology and terminally ill patients. In the next few weeks we will decide on a further Phase III trial in PDT. The particular choice for afamelanotide as an adjuvant photoprotective drug in gastro-intestinal cancer stems from the common biochemical pathways seen in both PDT and erythropoietic protoporphyria (EPP), a disease in which we are using afamelanotide in parallel advanced Phase III trials.”
“Today’s results are of particular relevance because we have learned from regulatory agencies that data from PDT studies may be used as supporting evidence when we file for EPP registration. Therefore, the confirmation of safety and the improvement in quality of life in these light intolerant patients provides a substantial step toward the registration of afamelanotide for EPP,”3,4 Dr Agersborg said.
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See the attached PDF for all appendices.
Appendix I: References
- Ortner M-A., (2009). “Photodynamic therapy for cholangiocarcinoma: overview and new developments.” Curr Opin Gastroenterol. 25 (5): 472-476.
- Jong C.T., et al., (2008). ”The quality of life of 790 patients with photodermatoses.” Brit J Derm. 159 (1): 192-197.
- Millward L.M., et al., (2001). “Self-rated psychosocial consequences and quality of life in acute porphyrias.“ J Inherit Metab Dis. 24 (7): 733-747.
- Lecluse A.L.Y., et al., (2007). “Erythropoietic protoporphyria without skin symptoms-you do not always see what they feel.” EU J Pediat. 167 (6): 703-706.
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Clinuvel is an Australian biopharmaceutical company focussed on developing its photoprotective drug, SCENESSE® (afamelanotide) for a range of UV-related skin disorders resulting from exposure of the skin to harmful UV radiation. Pharmaceutical research and development involves long lead times and significant risks. Therefore, while all reasonable efforts have been made by Clinuvel to ensure that there is a reasonable basis for all statements made in this document that relate to prospective events or developments (forward-looking statements), investors should note the following:
- actual results may and often will differ materially from these forward-looking statements;
- no assurances can be given by Clinuvel that any stated objectives, outcomes or timeframes in respect of its development programme for SCENESSE® can or will be achieved;
- no assurances can be given by Clinuvel that, even if its development programme for SCENESSE® is successful, it will obtain regulatory approval for its pharmaceutical products or that such products, if approved for use, will be successful in the market place