GSK Leaves Prosensa Hanging

GlaxoSmithKline hands back rights to DMD program to Prosensa, which could affect rival Sarepta Therapeutics.

Jan 13, 2014 at 4:49PM

We've seen this story before:

  • Drug fails.
  • Large pharma partner hands it back to biotech developer.
  • Biotech claims it's now in "a favorable strategic position" or some such wording.
  • Stock slumps.

Today's latest victim: Prosensa (NASDAQ:RNA). The biotech licensed its Duchenne muscular dystrophy drug drisapersen to GlaxoSmithKline (NYSE:GSK), but the development hit a road block when the phase 3 trial failed to show a statistically significant difference in walking ability between patients taking drisapersen and those getting a placebo.

The whole kit and caboodle
Part of the 2009 licensing agreement gave GlaxoSmithKline the options to license three more compounds to treat Duchenne muscular dystrophy. The disease can be caused by different mutations, and each needs to be treated with a different drug, albeit using the same underlying exon-skipping technology.

Not only did Prosensa get drisapersen back, but it'll also retain the rights to the drugs further back in development. Beyond drisapersen, Prosensa has five additional drugs in development, including three that are already in the clinic.

The early-stage compounds could be the silver lining in GlaxoSmithKline's ending of their partnership. Of course, if the underlying technology is the problem, they might not work, either. With only one drug through phase 3, it's hard to say one way or the other, but it seems GlaxoSmithKline isn't willing to stick around to find out.

For that matter, we don't really even know if drisapersen is dead yet. The best time to test a Duchenne muscular dystrophy drug is right before patients start losing their ability to walk. But the timing of the disease progression is variable, making it hard to control for. It's possible the phase 3 trial failed not because drisapersen doesn't work, but because more late-progressing patients were assigned to the placebo group.

Moving forward
In its press release, Prosensa didn't say what it plans to do with drisapersen. The company is presenting at the J.P. Morgan Healthcare Conference on Thursday, but I'm not sure investors should count on getting much of an update. According to a letter sent to patient group representatives, the biotech is still analyzing the data to determine the best path forward.

The decision will affect Prosensa's biggest rival Sarepta Therapeutics (NASDAQ:SRPT), which is in the process of designing a phase 3 trial for its Duchenne muscular dystrophy drug eteplirsen. Not only is there still potential for competition down the line, but if Prosensa continues development of drisapersen, Sarepta and Prosensa could be competing for patients to enroll in their clinical trials at same time, which could slow down development of both drugs.

Our top pick in 2012
There's a huge difference between a good stock, and a stock that can make you rich. The Motley Fool's chief investment officer has selected his No. 1 stock for 2014, and it's one of those stocks that could make you rich. You can find out which stock it is in the special free report: "The Motley Fool's Top Stock for 2014." Just click here to access the report and find out the name of this under-the-radar company.

Fool contributor Brian Orelli has no position in any stocks mentioned. The Motley Fool has no position in any of the stocks mentioned. Try any of our Foolish newsletter services free for 30 days. We Fools don't all hold the same opinions, but we all believe that considering a diverse range of insights makes us better investors. The Motley Fool has a disclosure policy.

Money to your ears - A great FREE investing resource for you

The best way to get your regular dose of market and money insights is our suite of free podcasts ... what we like to think of as “binge-worthy finance.”

Feb 1, 2016 at 5:03PM

Whether we're in the midst of earnings season or riding out the market's lulls, you want to know the best strategies for your money.

And you'll want to go beyond the hype of screaming TV personalities, fear-mongering ads, and "analysis" from people who might have your email address ... but no track record of success.

In short, you want a voice of reason you can count on.

A 2015 Business Insider article titled, "11 websites to bookmark if you want to get rich," rated The Motley Fool as the #1 place online to get smarter about investing.

And one of the easiest, most enjoyable, most valuable ways to get your regular dose of market and money insights is our suite of free podcasts ... what we like to think of as "binge-worthy finance."

Whether you make it part of your daily commute or you save up and listen to a handful of episodes for your 50-mile bike rides or long soaks in a bubble bath (or both!), the podcasts make sense of your money.

And unlike so many who want to make the subjects of personal finance and investing complicated and scary, our podcasts are clear, insightful, and (yes, it's true) fun.

Our free suite of podcasts

Motley Fool Money features a team of our analysts discussing the week's top business and investing stories, interviews, and an inside look at the stocks on our radar. The show is also heard weekly on dozens of radio stations across the country.

The hosts of Motley Fool Answers challenge the conventional wisdom on life's biggest financial issues to reveal what you really need to know to make smart money moves.

David Gardner, co-founder of The Motley Fool, is among the most respected and trusted sources on investing. And he's the host of Rule Breaker Investing, in which he shares his insights into today's most innovative and disruptive companies ... and how to profit from them.

Market Foolery is our daily look at stocks in the news, as well as the top business and investing stories.

And Industry Focus offers a deeper dive into a specific industry and the stories making headlines. Healthcare, technology, energy, consumer goods, and other industries take turns in the spotlight.

They're all informative, entertaining, and eminently listenable. Rule Breaker Investing and Answers are timeless, so it's worth going back to and listening from the very start; the other three are focused more on today's events, so listen to the most recent first.

All are available for free at www.fool.com/podcasts.

If you're looking for a friendly voice ... with great advice on how to make the most of your money ... from a business with a lengthy track record of success ... in clear, compelling language ... I encourage you to give a listen to our free podcasts.

Head to www.fool.com/podcasts, give them a spin, and you can subscribe there (at iTunes, Stitcher, or our other partners) if you want to receive them regularly.

It's money to your ears.

 


Compare Brokers