Here’s What Happened To Karyopharm Therapeutics Inc (NASDAQ:KPTI) and PTC Therapeutics, Inc. (NASDAQ: PTCT)

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Here’s What Happened To Karyopharm Therapeutics Inc (NASDAQ:KPTI) and PTC Therapeutics, Inc. (NASDAQ: PTCT)

The end of the week is upon us, and it’s been a bit of a wild ride in the biotech space. Macro forces have come into play from an industry wide sentiment perspective, and there have been plenty of micro rooted catalysts to both buck, and compound, the action that these macro forces have brought about.

So, as we draw this week to a close, here are two end of the week movers in the space, what’s driving the action in both, and what we think could be on the cards going forward.

The two companies we are looking at are Karyopharm Therapeutics Inc (NASDAQ:KPTI) and PTC Therapeutics, Inc. (NASDAQ: PTCT).

We’ll say it right off the bat – neither of these companies have had a particularly desirable start to the week. One, however, has a gotten off a little more lightly than the other.

First up, then, Karyopharm.

This company is a development stage biotech focused on bringing treatments to market that target oncology indications, and specifically, various forms of leukemia. Its lead asset is called selinexor, and it’s currently in a host of trials – one of which is the focus of this discussion. The trial in question is a phase II study called SOPRA, which Karyopharm set up to evaluate single agent selinexor in relapsed/refractory acute myeloid leukemia (AML).

The patients in the trial are elderly patients who aren’t able to undergo the more traditional standard of care therapies because – to put it bluntly – their bodies won’t be able to take it. Anyway, there was a predefined interim analysis set up, and that interim analysis just took place. As the recent release reported, the company determined in concert with the study’s independent Data Safety Monitoring Board (DSMB) that the trial will not reach statistical significance for overall survival (OS). OS was the study’s primary endpoint, so the trial is basically now classed as failed. There are some patients that have drawn benefit from the treatment, or at least seem to have done based on the available data, and these will continue to receive therapy on a compassionate use basis. Other than that, however, we can consider this trial discontinued.

Now, in another instance, this might be a real downside hit. In this instance, however, things aren’t as bad as they initially seem. The drug is still promising, as demonstrated by a raft of data that is both in-place and currently under-collection from other trials, and expectations are that selinexor will pick up approval in a similar indication. Additionally, there are ongoing combination trials in the elderly patient population, and it looks as though the drug is going to come out of them with some great data as well.

Karyopharm is down premarket heading into the close of this week, having initially taken a jump on a knee jerk market response to the release on Thursday. We think the company should stay pretty flat over the coming weeks, and then start to run into some mid year data releases rooted in its wider pipeline.

Bottom line, not great data, but far from the end of the line.

That’s more than can be said for the second company on this list – PTC.

PTC just announced results from a phase III investigation of a drug called Ataluren. The company was investigating the asset as part of this pivotal study in a cystic fibroses target indication, and this was a confirmatory trial in a subset of CF called nonsense mutation cystic fibrosis, which is widely regarded as one of the most difficult forms to treat in the disease type. The drug was generally well tolerated and the data derived from more than 1000 patients across multiple indications has shown it to be safe. However, as per the latest release, the trial failed to meet both its primary and secondary endpoints.

Unlike Karyopharm’s asset, in the CF indication at least, this is the end of the line for Ataluren. The company is discontinuing investigation in this asset, and will add the data to that ever expanding pool if failed numbers to guide future endeavors in the space.

Unsurprisingly, this one is down considerably on the news. At last count, PTC was down 20% on its pre-release market capitalization.