​Here's a Catalayst Play In PTC Therapeutics, Inc. (NASDAQ:PTCT)

Mark Collins  |

Earlier this year, markets had all but completely written off PTC Therapeutics, Inc. (PTCT) as far as the company's chances of getting its Duchenne muscular dystrophy (DMD) drug approved in the US was concerned.

For those new to the story, the drug is called Translarna and it is designed to restore production of a functioning protein in patients with nonsense mutation inherited disorders like nmDMD. Nonsense mutations are mutations that stop the creation of a protein that would otherwise be essential to a healthy life and, in the instance of DMD, the protein that is unable to be created effectively is called dystrophin. A lack of dystrophin, or even a small degree of deficiency, can have a pretty dramatic impact on quality of life for patients with DMD, with the most common symptoms of the disease rooted in a lack of structural stability of skeletal, diaphragm and heart muscles.

Patients with this condition have very little in the way of treatment options available to them right now. The only drug that is approved for on label and direct therapy of DMD is called Eteplirsen and it was approved by the FDA back in September 2016. Some reading might remember that the approval was controversial at the time; the drug failed to show a substantial degree of efficacy and there were some safety concerns surrounding the data that underpinned the registration application. However, because of the severe lack of treatment options available in the space, the FDA was essentially forced into approving the asset.

Unfortunately for PTC, however, the agency has been a little bit harsher on Translarna than it was on Eteplirsen.

The drug is already approved in Europe and generates around $25 million quarterly from the European market. When PTC tried to replicate this approval in the US, however, and submitted a New Drug Application (NDA) to the FDA last year. In response, the FDA cervical refusal to file, essentially implying that there just wasn't enough data built into the submission to justify any sort of review.

This sent PTC tumbling and, just as mentioned in the introduction to this piece, the company was all but written off from the point of view of DMD.

Fast forward to March, however, and things got a little more interesting. The company used what are called file over protest regulations to support an appeal and, surprisingly, the regulations won out – the FDA was forced to accept the submission.

At the time, the agency set a PDUFA date of October 24, 2017, and that date still stands. Near term, however, and as announced during summer 2017, the FDA has put in place a meeting of one of its advisory committees at which regulatory insiders and industry personnel will discuss the relative merits and drawbacks of Translarna against the backdrop of severe unmet need in the DMD space.

This serves up what could be a dramatic catalyst for PTC very near term.

If the advisory panel meeting reports back as positive and recommends approval for the drug at conclusion, PTC is going to appreciate very quickly as markets load up ahead of the PDUFA date set by the FDA.

It's a risk play, of course. There's a chance (and it's a relatively strong one) that the advisory panel will vote against any approval recommendation. Further, even if the vote comes in as favorable for Translarna, the FDA is under no obligation to follow the panel's recommendation. Factor in some disappointing mid-summer trial data and the risk only serves to increase exponentially.

With that said, however, if on the off chance things do play out in PTC's favor, there is considerable reward to be had on an exposure ahead of the event.

Disclosure: The author holds no positions in any of the stocks mentioned.

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