Outlook brightening for Clinuvel

If everything goes to plan Australian company Clinuvel Pharmaceuticals could have its first marketed product on the market by the second half of this year, after completed a filing for its lead drug in Europe last week. The chances for approval are looking good; the drug is being developed in a rare genetic condition where there is no existing approved treatment and so far it has demonstrated good efficacy with no associated safety concerns.

If Scenesse (afamelanotide), which has been filed for erythropoietic protoporphyria (EPP), is approved in Europe it could signal a turnaround for Clinuvel, whose stock has see-sawed wildly over the last year. As sign of things to come shares in the group have risen by 25% in the last three months thanks to positive data for Scenesse and news of the filing, leaving them at A$1.94 (Event- Clinuvel needs positive Scenesse data to spark recovery, August 19, 2011).

Shady condition

EPP is a rare form of severe phototoxicity where the build up of protoporphyrin IX in the skin reacts with sunlight, causing sufferers burning and blistering of the skin as well intense pain. Caused by an enzyme deficiency, EPP can also lead to general tissue and nerve damage that can result in abdominal pain, reflux and in extreme cases temporary psychosis.

For sufferers it often means a life lived predominantly indoors, avoiding sunlight and in severe cases people only being able to venture out at night. Sunscreens offer no protection as the photosensitivity includes light from the visual spectrum as well as UV light. Patients often describe the symptoms as excruciating pain, which conventional painkillers cannot relieve.

Scenesse is a synthetic version of a hormone called alpha-melanocyte stimulating hormone (alpha-MSH), which increases the production of protective melanin in the skin when it is exposed to sunlight.

Positive results

Recently in phase III European trials the drug demonstrated significant quality of life benefits in 68 patients in a randomised, placebo controlled trial. The trial also met its primary endpoint in reducing pain associated with phototoxicity. The drug also showed a 50% reduction in phototoxic reactions, and patients were also able to experience significantly more direct exposure to sunlight without pain; many could spend up to seven times longer in direct sunlight without pain.

With these results and the orphan drug designation in Europe many are now hopeful of a positive outcome as the drug goes through an accelerated approval process.

Despite not being yet being approved Clinuvel is banking sales for Scenesse in a handful of European countries, where trial patients have remained on the drug on a compassionate use basis. These revenues will be helpful for the company that has sunk $50m in to the product and currently only has $12.3m on its balance sheet.

The great beyond

But beyond EPP both the clinical data and Scenesse’s progress in Europe should start to get investors thinking a little bit harder about the other, much larger and more lucrative indication the drug is being developed in non-segmental vitiligo. Vitiligo, a lightening of the skin in patches is thought to effect 10 million people in Europe and the US.

An update on the study in October showed that patients treated with Scenesse and narrow band ultraviolet light achieved accelerated and deeper pigmentation of vitiligous patches. The trial is due to complete at the end of this year and could publish its results in the first or second quarter of 2013. 

So with Scenesse going through the regulators in Europe for EPP, reporting positive results in US phase II trials for the same indication and also showing initial promise in vitiligo trials, the group is making a good fist of starting to live up to some of the expectations in the market.

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