First up on today’s list of biotechnology movers is Aclaris Therapeutics, Inc. (NASDAQ:ACRS). The company just announced that it has picked up an FDA approval for its lead development asset and is running up on the back of the news.
The drug in question here is called ESKATA (hydrogen peroxide) topical solution, 40%, and it’s designed to target the treatment of raised seborrheic keratoses. These are a type of a non-cancerous (benign) skin tumor that originates from cells in the outer layer of the skin and that – for the most part – are entirely harmless. With that said, they can be unsightly and, in some instances, can be itchy and unpleasant.
As such, many people who get these type of keratosis prefer to treat them and, as things stand, treatment is pretty much limited to surgical removal. With ESKATA, Aclaris was trying to bring ales invasive option to the market for these patients and – as per the latest approval – it’s been able to do exactly that.
What’s the market size for something like this?
Right now, the exact size of the market that awaits ESKATA is uncertain. A huge amount of people would qualify for treatment (estimates are that more than 90% of people over 60 in the US develop these sorts of keratoses) but not all of these will seek treatment.
Even if only a small portion of those with the condition does seek treatment, however, it’s not going to take much to shift the market size into the millions in the US alone. Couple this with the fact that the company plans to price treatment at around $125 (at the high end) and there’s some real impact potential when considered against a backdrop of Aclaris’ current market capitalization of $742 million.
Markets are yet to respond to the news and – heading into the session on Monday – Aclaris is trading a couple of percentage points down on its preannouncement market capitalization.
Next up, Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY).
This one is another regulatory driven development but this time, instead of a regulatory-related decision, the movement is rooted in a clinical trial and – specifically – a clinical hold that was previously placed on said trial.
Alnylam is trying to bring a drug called fitusiran to market as a treatment for patients with hemophilia and – as per some early to mid-stage data – it looked as though the drug had a pretty good chance of mounting a successful development effort.
Earlier this year, however, the agency in the US put a clinical hold on the program, with the hold applying to a phase III (but rooted in an incident in a phase II).
So what happened?
According to reports, a patient in the phase II trial that preceded that phase III pivotal had died as a result of a fatal thrombotic event, which later turned out to be a blood clot in the brain. There was some concern at the time that the blood clot arose as a result of treatment (these patients’ blood doesn’t clot properly and fitusiran is designed to promote clotting) and based on these concerns, the FDA put a hold on both the phase II and the phase III studies.
Alnylam took a real hit on the news and not just because of the implications for the drug’s future – the company had signed a $100 million deal with healthcare behemoth Sanofi SA (ADR) (NYSE:SNY) just a few months before the hold was announced that saw the two team up on the phase III study, after having initially teamed up on a $700 million collaboration for the early to mid stage development of fitusiran back in 2014.
As such, concerns were that the clinical hold could derail this collaboration – a collaboration on which a large portion of the positive sentiment surrounding Alnylam’s potential future in the hemophilia space rests.
The latest news, then, is that the FDA has lifted the hold and that patients can once again be dosed as part of the study. Alnylam has had to put some additional risk mitigation measures in place to help avoid future issues and the company has said it will resume dosing around year-end.