With ASCO now in the rear view mirror, it’s back to good old traditional biotech catalysts. The meeting brought with it plenty to discuss, much of which we’ve already covered in previous releases this week. As we head into the latter half of the week, then, and as much of the volume we are seeing in the sector remains driven by ASCO news, here’s a look at some of the biggest midweek movers in the biotechnology sector, what’s moving each and where we’re looking next for the companies in question.
The two companies that we’ve got in our cross hairs for the session today are PTC Therapeutics, Inc. (NASDAQ:PTCT) and Alkermes plc (NASDAQ:ALKS).
So let’s kick things off with PTC.
This one’s rooted in the development of a drug called ataluren, or commercially known as Translarna, that the company is trying to get approved in a target indication of the treatment of nonsense Duchenne muscular dystrophy (nmDMD). This condition is basically an alternative form of DMD that’s caused by a what’s called a nonsense mutation. Nonsense mutations are implicated in a variety of genetic disorders and create a premature stop signal in the translation of the genetic code contained in mRNA, which prevents the production of full-length, functional proteins.
The idea is that, through the use of ataluren, the company can bypass the premature stop signals in the protein production process (essentially hurdling it) and – as a result – produce functional proteins. Functional proteins should overcome the underlying defect that causes the condition and therein lies the MOA.
And we already know it works.
The drug is already approved within the European Union (EU) Member States, Iceland, Liechtenstein, Norway, Israel and South Korea under the above-noted trade name, Translarna for the treatment of nmDMD in ambulatory patients aged five years and older. These already approved regions make it the first approved drug that goes after the underlying cause of DMD.
Those familiar with this indication will already be aware that it’s dramatically underserved from a treatment perspective right now. There are a couple of off-label therapies, only one real targeted treatment and no completely effective assets available in the US. Sure Eteplirsen is an option, but there’s a strong argument for a lack of clinical benefit and the market is crying out for fresh alternatives.
PTC and its shareholders are hoping that ataluren can meet this need.
The latest news, then, is that the FDA has set an advisory panel meeting for the drug ahead of PDUFA. The meeting is slated for September 28, 2017, and will see the Peripheral and Central Nervous Systems Drugs Advisory Committee get together to discuss the New Drug Application (NDA) and vote as to whether its constituent members recommend approval based on the data available.
We think this one’s got a great chance of picking up a regulatory green light in the US. The FDA doesn’t have to go with the recommendation of the panel, of course, but given that the drug is already approved in a number of regions, and that the data is strong (as well as this being a major unmet need in the US healthcare landscape, we think the meeting will vote in favor and – in turn – the agency will give the drug a thumbs up.
PDUFA for this one is October 24, 2017, or around four weeks’ post-panel meeting.
PTC is up a little over 6% on the news of the panel meeting and the PDUFA.
Next up is Alkermes plc (NASDAQ:ALKS).
This one’s a good old biotech approval move.
Alkermes noted on Tuesday that the FDA had approved a drug called Aristada as a treatment for schizophrenia. The drug is already approved in this indication and marketed by Alkermes, but the approval is for a one-month injection and a six-month injection. The nature of schizophrenia, combined with the MOA of this drug (it converts into aripiprazole once it’s been injected into the body) necessitates flexibility in administration (as it affects different people in different ways, for different lengths of time).
As such, when Alkermes set out to get this one to market, it did so wit ha view to providing a range of different dosing frequencies in an attempt to capture as large a portion of the market as possible.
With the latest approval, there are now three dosing frequencies available – the above noted one-month injection and six-month injection as well as the just announced two-month injection. This covers a large portion of the patient population and – in doing so – makes the drug a real contender in the space.
On the announcement, Alkermes gained around 2% – a standard proportionate move on an approval like this, for a company of this size.