A protoporphyrin IX molecule
Over recent months I have written several times of the need for Clinuvel to prove clinical relevance in our trials with the use of SCENESSE® (afamelanotide) in erythropoietic protoporphyria (EPP). In orphan populations the need to demonstrate how a novel drug assists in their daily activities and improve their lives is at the forefront of the regulators’ minds. And so it should be, after all the objective of the pharmaceutical industry is to develop drugs which address either disease or symptoms adequately and safely. The results the company released yesterday from our Phase II US study of the drug in EPP (CUV030) have given us important data towards demonstrating clinically relevant improvement of patients’ lives. (more…)
Development of novel drugs is truly like no other business: one attempts to address questions that may have never been previously posed – let alone answered – in the pursuit of improving the lives and quality of life of patients. As I eluded to in my recent 
Stem cells are immature, unspecialised cells which have the ability to develop into other adult cells with a specific function, i.e. skin cells, red blood cells, nerve cells (neurons), etc.
Scarcely a day goes by that the Clinuvel team is not contacted regarding the latest conference: everything from monitoring to manufacturing to marketing is analysed, discussed and disputed across the globe in conference form. It’s impossible – and unwise – for small companies to try to attend all these forums, but it is equally impossible to work in the space without looking outside the office door: there is much to be learned from what others have done or not done to succeed in an industry fraught with failure.
Drug development is time consuming, expensive and fraught with risk. Setting aside commercial considerations, the scientific rational for the use of a drug and its introduction into the clinic requires a deep level of scientific and medical understanding in an environment which is continuously changing. The less common the drug class or clinical indication, the less likely one is to find individual experts who have the capacity to properly evaluate whether a drug is safe and effective for use in a clinical setting. Here one needs to learn while progressing, learning on the job due to lack of precedent.
