Please ensure Javascript is enabled for purposes of website accessibility

Why PTC Therapeutics Dropped 25.3% in March

By Brian Feroldi - Apr 7, 2017 at 7:45AM

You’re reading a free article with opinions that may differ from The Motley Fool’s Premium Investing Services. Become a Motley Fool member today to get instant access to our top analyst recommendations, in-depth research, investing resources, and more. Learn More

A handful of negative developments overwhelmed the positive news items released during the month, causing the biotech's stock to crater.

What happened

Shares of PTC Therapeutics (PTCT 0.27%), a commercial-stage biotech focused on rare diseases, plunged 25.3% in March, according to data from S&P Global Market Intelligence

A businessman lights money on fire

Image source: Getty Images.

So what

Here's a look at the key headlines from March that triggered the stock's movement:

  • PTC Therapeutics announced that its phase 3 ACT CF trial failed to meet its primary or secondary endpoints. This study was testing its drug Translarna as a potential treatment for nonsense mutation cystic fibrosis. The clinical setback caused management to abandon all clinical development of Translarna in treating cystic fibrosis and the company also withdrew its regulatory application in Europe.
  • Management announced that the Food and Drug Administration has "acknowledged the filing over protest" related to PTC's application seeking approval for Translarna as a treatment for Duchenne muscular dystrophy, or DMD. A PDUFA date of October 24, 2017, has been set.
  • The company agreed to spend $140 million to acquire Marathon Pharmaceuticals' DMD drug Emflaza. While Emflaza was approved by the FDA last month, it has drawn a lot of criticism for its astronomical list price of $89,000 per year. The reason is that Emflaza is simply a generic steroid that costs around $1,200 a year outside of the United States. With lawmakers crying foul on pricing, it is possible that PTC could have vastly overpaid for this deal.
  • Its fourth-quarter revenue nearly doubled from the year-ago period thanks to growing Translarna sales in Europe. The rising sales caused the company's net loss to get cut in half, too.

Regardless of the upbeat financial results and regulatory news, the disappointing clinical update and controversial acquisition caused shares to take a beating in March.

Now what

The ACT CF trial data was a big blow to the bull thesis for owning PTC's stock. The company had estimated that Translarna could have been used to treat more than 9,000 cystic fibrosis patients had the drug made it to market. That's a big market potential than DMD, so it was disappointing to see this label expansion opportunity disappear. 

The Emflaza deal also appears to be a bit of head-scratcher. While PTC has a relationship in place with the DMD community, there is a very real risk that the company just lit a bunch of money on fire if political pressure causes the drugmaker to adjust its pricing.

On the bright side, Translarna appears to be selling well in Europe, and the FDA's decision to finally accept the company's application does offer the real potential to bring the drug to the U.S. Given that the agency gave the thumbs-up to Sarepta Therapeutics' DMD drug Exondys 51 last year, the company appears to have a decent argument in favor of approval. 

Still, PTC remains a highly controversial company and its future is far from certain. Given those realities, I, for one, plan to keep far away. 

Invest Smarter with The Motley Fool

Join Over 1 Million Premium Members Receiving…

  • New Stock Picks Each Month
  • Detailed Analysis of Companies
  • Model Portfolios
  • Live Streaming During Market Hours
  • And Much More
Get Started Now

Stocks Mentioned

PTC Therapeutics, Inc. Stock Quote
PTC Therapeutics, Inc.
$40.22 (0.27%) $0.11
Sarepta Therapeutics, Inc. Stock Quote
Sarepta Therapeutics, Inc.
$75.22 (4.73%) $3.40

*Average returns of all recommendations since inception. Cost basis and return based on previous market day close.

Related Articles

Motley Fool Returns

Motley Fool Stock Advisor

Market-beating stocks from our award-winning analyst team.

Stock Advisor Returns
S&P 500 Returns

Calculated by average return of all stock recommendations since inception of the Stock Advisor service in February of 2002. Returns as of 06/27/2022.

Discounted offers are only available to new members. Stock Advisor list price is $199 per year.

Premium Investing Services

Invest better with The Motley Fool. Get stock recommendations, portfolio guidance, and more from The Motley Fool's premium services.