There were some interesting biotech trial results this month. The first is Clinuvel, whose treatment 'Scenesse' was approved last night in the US. Scenesse treats EPP, which has been described as an allergy to the sun. The stock rallied about 60%, which was something of a surprise to us, as approval seemed quite likely.
Scenesse has been sold in the EU for years now, and there’s plenty of data from patients, doctors and regulators that it works. If I knew the market had such a dim view of the drug’s prospects this would have been a larger position!
Scenesse costs about EUR14,000 per implant, with 3-4 required per year. Perhaps the bearishness was because the UK regulators did not approve the drug. But that was a cost-benefit analysis. Allowing long-suffering patients to move more freely in the sun was not deemed worthy of the cost.
The US regulator is purely concerned with safety and whether the thing actually works.
EPP, while devastating, only affects a small group of patients. This approval now opens the firm up to US trials of their product in a far more common condition, Vitiligo.
Vitiligo is the condition Michael Jackson had. The darker your skin, the more dramatic the contrast.
On balance, today’s world is far more accepting and body-positive place than the one of even two decades ago. Even so, many still choose to bleach their skin to minimize the effects.
Scenesse has had past success in treating Vitiligo, albeit with a cultural wrinkle: the treatment darkened all skin, which didn’t suit the preferences of some patients in Singapore. This is complex and sensitive.
Results from trials of Scenesse in Vitiligo
Given that perhaps 1% of the world has vitiligo to some degree (it runs in my family), this is an enormous opportunity.
Clinuvel will soon have profits from treating EPP in the United States to pursue this otherwise untreatable condition.
We have written about Alzheimer’s before, and Actinogen joined a long string of companies with failed Alzheimer’s trials. That was not why we were interested in the company though.
Actinogen’s drug Xanamem was designed to reduce cortisol, the stress hormone, in the brain. This is interesting.
Cortisol is implicated in a wide range of diseases (not least Alzheimer’s, Parkinsons, and epilepsy) and has proven to impair judgement in healthy adults. The literature is comprehensive on this point: cortisol is bad. Things that reduce cortisol are good.
Specifically, reducing cortisol was shown to improve cognition in healthy people, but the drug was found to be unsuitable. Xanamem was designed as a safer, more targeted version, but there was no evidence it had the desired effect in humans. The Alzheimer's failure was ominous.
So we were pleasantly surprised to see Actinogen report that a small group of healthy adults on Xanamem recorded statistically significant improvements in cognition.
There is almost unbearable drama in these trials. Xanamem represents decades of work from multiple research groups, and until this data was released, looked like a complete dead end.
We now have in-human proof that the drug passes the blood-brain barrier, targets the right enzyme, reduces cortisol, and that all this causes a noticeable positive effect in cognition in healthy adults.
Quite a striking result, and even more so as it it's entirely in line with the original hypothesis.
Alzheimer’s is a large market, but there is a larger one: anti-aging.
Actinogen now has evidence that Xanamem, under their chosen trial conditions, did not improve cognition in Alzheimer’s.
But they do have evidence that in the healthy elderly:
- Xanamem crosses the human blood-brain barrier
- It reduces cortisol
- This improves cognition
Ageing may not be a druggable condition, but healthy elderly with high cortisol levels may well be. These trial numbers were small, so plenty of work needs to be done.
We earnestly hope that Actinogen follows the data here, and plans their next trials accordingly.