Biotech Movers: Argenx (NASDAQ:ARGX) and Blueprint Medicines Corporation (NASDAQ:BPMC).


In yesterday’s biotechnology movers coverage, we too a look at some of the biggest movers in the sector, all of which were moving on the back of the American Society of Hematology (ASH) Annual Meeting and, specifically, data reported at the event.

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We’re now midway through the week (the ASH event took place over the weekend and into Monday) and ASH is still dominating headlines in the healthcare sector.

With this in mind, here’s a look at another group of major movers in biotech with a discussion of what’s moving each and where things are likely to go next for the companies in question.

The two companies that we are focusing on for the session today are Argenx (NASDAQ:ARGX) and Blueprint Medicines Corporation (NASDAQ:BPMC).

So, first, up, Argenx.

As is to be expected, this one’s all about data. The company put out data at ASH from a trial investigating one of its lead development assets, a drug called ARGX-113 (efgartigimod). The trial is a phase II trial set up to investigate the safety and efficacy of the drug in a target indication of myasthenia gravis (MG). For those unfamiliar with this condition, it’s a chronic autoimmune neuromuscular disease that causes weakness in the skeletal muscles, which are responsible for breathing and moving parts of the body, including the arms and legs. It’s incredibly unpleasant and the only real treatments for this condition right now are corticosteroids which, as most reading will already be aware, are a pretty nasty therapy to have to rely on and especially, as is the case here, when the disease being treated is chronic


Because a chronic condition requires chronic treatment and corticosteroids are one of the worst drug types as far as side effects and tolerability issues are concerned. So patients in this target population need fresh treatment options and, with ARGX-113, Argenx is trying to offer them just that.

So what did the data tell us?

Well, as per the results, it looks as though the drug can work pretty well. From an MOA perspective, it blocks what’s called IgG recycling (including disease-causing autoantibodies) and increases something called IgG clearance, which results in faster remission and drastically reduced length and seriousness of acute autoimmune crisis in these patients (MG is an autoimmune condition).

Anyway, as per the data, the drug showed a clinically meaningful and statistically significant benefit over placebo with 75% of patients showing an improvement versus 25% of patients on placebo.

That’s strong data and – to boot – the company also reported what amounts to a relatively clean safety profile, putting it ahead of the pack of current SOC assets.

So what’s next?

Now it’s all about getting this one into a pivotal and getting the data required to push for approval in the US. Given the strength of the data just seen, the drug shouldn’t have any problem getting through a successful phase III. Of course, nothing is guaranteed in this space, and therein lies the risk of picking up a position ahead of the trial kicking off.

Moving on, Blueprint Medicines.

Blueprint just put out data (at ASH) from an ongoing phase I trial of a drug called avapritinib (BLU-285), which the company is trying to show can be safe and effective in a target indication of advanced systemic mastocytosis. This is a condition in which mast cells are abnormally increased in multiple organs including (and, to a degree most importantly as far as severity is concerned) the bone marrow. Mast cells are immune cells that produce a variety of mediators, such as histamine, that are important in the body’s allergic responses.

When there are too many of them in the body, it can lead to a whole host of symptoms, including anemia, pain, pruritus and – in the most severe cases – can be deadly.

There’s essentially no treatment outside of those that target symptoms available right now and, if Blueprint can get this asset to market, it would have a good shot at becoming first line.

So what did the data show?

Again, this one looks pretty good. As per the company’s presentation, the data showed an ORR of 72% and a disease control rate (DCR) of 100%. Next up, then, is a mid-stage study in the same indication. We’re looking for initiation at some point between now and the end of the second quarter next year.

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