What’s Next for Sarepta Therapeutics Inc (SRPT)?

Page 1 of 2

What’s next for Sarepta Therapeutics Inc (NASDAQ:SRPT) ? After announcing encouraging results last October from a phase 2b study of eteplirsen targeting treatment of Duchenne muscular dystrophy, or DMD, shares nearly tripled in value overnight. While the initial exuberance faded to some extent, the stock is still up around 87%. Will Sarepta march back up to those October highs and beyond? Here are three key next steps that will make the difference.

1. To file or not to file?
Sarepta has two important meetings with the Food and Drug Administration over the next few weeks. One is the end-of-phase 2 clinical meeting while the other is the end-of-phase 2 Chemistry, Manufacturing, and Controls — or CMC — meeting. Both are standard meetings for drug companies that conclude phase 2 testing. However, for Sarepta these meetings with the FDA are especially important.

Sarepta Therapeutics IncMuch speculation has centered on Sarepta filing for accelerated approval of eteplirsen. The FDA can decide to grant accelerated approval for drugs that fill an unmet medical need by allowing the drug company to use surrogate endpoints rather than clinical outcomes. Obtaining accelerated approval allows the company to begin marketing the drug earlier than normal. Sarepta’s management has stated that the decision whether to file for accelerated approval will depend on how the first quarter meetings with the FDA go.

Gaining accelerated approval certainly isn’t always easy. Onyx Pharmaceuticals, Inc. (NASDAQ:ONXX) found that out in December 2011 when the FDA initially rejected accelerated review of Kyprolis. The agency’s concern at that time was that Onyx had not conducted sufficient clinical trials for the faster approval. Kyprolis did ultimately gain FDA approval in 2012.

It isn’t easy, but it can be done. The FDA last week approved accelerated approval for Celgene Corporation (NASDAQ:CELG)‘s Pomalyst in treating multiple myeloma. However, the phase 2 study for the drug included 221 patients — much larger than the 12 patients in Sarepta’s study.

The challenge for Sarepta will be in proving to the FDA that the data establishes that eteplirsen is both effective and safe. As for effectiveness, Sarepta will argue that dystrophin production is a solid surrogate endpoint and that all 20 patients across several studies who took at least 10 mg/kg/week of eteplirsen experienced increased dystrophin production.

Regarding safety, the company will try to convince the FDA by noting that no patients experienced significant adverse events related to taking the drug even at high dosages. I expect that Sarepta will also emphasize that the morpholino chemistry upon which eteplirsen is based has been used safely in 400 patients with other diseases.

2. Further clinical testing
Regardless of whether accelerated approval is pursued and given, more clinical testing is on the horizon for eteplirsen. If the FDA grants accelerated approval, confirmatory studies will be required in larger patient groups. If it doesn’t, Sarepta will move forward with phase 3 studies with more patients.

Page 1 of 2
blog comments powered by Disqus
Insider Monkey Headlines
Insider Monkey Small Cap Strategy
Insider Monkey Small Cap Strategy

Insider Monkey beat the market by 52 percentage points in 24 months Click to see monthly returns in table format!

Lists

The 10 Most Technologically Advanced Countries in the World

The 10 Safest Countries in the World to Live in Peace

The 10 Top Selling Smartphones in the World 2014

The 12 Biggest Shopping Centers in the World

The 10 Friendliest Countries in the World

Qihoo 360 Technology Co Ltd (QIHU), Actavis plc (ACT), Yahoo! Inc. (YHOO) Among 20 Long Ideas from Sohn Canada

The 10 Laziest Countries in the World

The 10 Most Polluted Countries in the World

The 10 Most Dangerous Cities in America 2014

The Top 10 Gold Producing Countries in the World

The 10 Tallest Buildings in the World

The 10 Richest Stand Up Comedians in the World

The 10 Fattest Countries in the World

The 5 Best Summer Jobs for Teens

The 10 Most Religious Countries in the World Keeping the Faith

The 10 Most Educated Countries in the World

The 10 Most Popular Cell Phones in the World

The 10 Drunkest Countries in the World

The 10 Most Expensive Private Schools in the World

The 10 Smallest Countries in the World

Walking Dead Season 5 Spoilers You’ll Wish You Didn’t Know

The 10 Poorest Countries in the World

The 10 Greenest Countries in the World

The Top 10 Countries with the Highest Population in the World

The 10 Most Visited Countries in the World

The Top 10 Star Wars Characters in the Iconic Series

The 10 Most Expensive Android Phones in the World

5 Reasons Why The Illuminati Is Real and a Threat to Society

The 6 Scariest Halloween Costumes Ever Screamed At

The 4 Biggest Hedge Fund Managers in the World Today

The 15 Most Densely Populated Countries in the World

The 10 Biggest Tea Drinking Countries in the World

Top 6 Ways to Improve Your Checkout Process and Close Sales

The 5 Most Profitable Online Businesses You Can Start Today

The 20 Most Profitable Hospitals in the US

The 5 Most Profitable Home Businesses to Start

The 7 Teams that Will Win the Stanley Cup in 2015

The Top 10 Most Expensive Digital Cameras to Snap Stunning Shots With

The 10 Highest Quality Fast Food Restaurants In America Today

The 8 Best Halloween Decorating Ideas to Spook Up Your House

10 Marvel Women that Should Get a Movie Right Now

The 20 Best Remixes of Popular Songs that Will Make You Forget the Originals

7 Most Expensive Cities in the World

5 Least Expensive Cities in the World

10 Celebrities Who Believe In Scientology

10 High Margin Food Products to Build a Business Around

The 10 Most Expensive Clothing Stores in the United States to Get Decked Out At

The 5 Biggest Kickstarter Scams That Swindled Backers’ Donations

The 10 Most Expensive Boarding Schools In the World

50 Crazy Facts About Japan You Won’t Believe

Subscribe

Enter your email:

Delivered by FeedBurner

X

Thanks! An email with instructions is sent to !

Your email already exists in our database. Click here to go to your subscriptions

Insider Monkey returned 47.6% in its first year! Wondering How?

Download a complete edition of our newsletter for free!