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

10 Cities with High Demand for Nurses

6 of the Worst Greeting Card Messages Ever Crafted

6 Ways to Make Money in ArcheAge and Build Your Empire

10 Foods To Eat To Lower Cholesterol Levels

The 10 Most Hated Television Characters of All Time

The 30 Worst Halloween Costume Ideas Ever Brought to Horrible Life

10 Vocational Skills in Demand Today with Jobs Waiting to be Filled

10 Best Places to Visit in Central and South America

The 10 Greatest Empires in History Which Nearly Conquered the World

The 6 Cheapest Boarding Schools In America 2015

5 Clear Reasons LoL is Better than DotA, Continues to Rule MOBAs

The Only 9 Teams with a Chance to Win the Super Bowl

The 15 Most Common Phobias in America that Induce Fits of Panic

Top 6 Least Expensive Tourist Destinations in 2014

Jim Goetz, Peter Fenton, Jim Breyer: Top 6 Venture Investors for 2014

Top 15 Billionaires in 2014

5 Pitfalls To Avoid When Buying a Franchise

Top 20 Medical Schools in the US – 2014 Rankings

4 Business Strategies that Turned Jamie Oliver into the World’s Richest Chef

6 Qualities That Make You A Good Team Player

10 High Paying Seasonal Jobs in America this Holiday Season

The 10 Busiest Shipping Lanes in the World

5 Most Valuable Brands in China

The 10 States with Highest Substance Abuse Rates Crippling Their Populace

The Top 10 Things to Do Before You Die That Will Echo for Eternity

The 10 Best Selling Items on Etsy

Top 10 Things to Do in Tokyo, the Greatest City in the World

10 Mistakes on Social Media that Can Harm You and Will Probably Get You Canned

The 10 Best Cities to Find Jobs in 2014

The 10 Most Controversial Songs Of All Time to Hit (and get Banned from) the Airwaves

The 20 Biggest IPOs in US History

The 10 Best Places to Visit in Mexico that Are Beautiful and Safe

7 Bad Habits that Age You Beyond Your Years

The 40 Best Fortune Cookie Sayings That Will Leave You Bemused, Befuddled, or Beguiled

10 Foods to Eat Before a Workout to Make Every Drop of Sweat Count

The 5 Best Documentaries On Netflix You Must See

The Most Heartwarming and Inspirational Story Of This Halloween Season, It Will Make You Cry and Jump For Joy

10 Best Party Songs of All Time to Bring the House Down With

5 New World Order Conspiracy Theories that Will Strangle the World

The 10 Highest Rated Movies of 2014

The 10 Largest Container Shipping Companies in the World

The 10 Largest Armies in the World: Who Should We Be Afraid Of?

Best Warren Buffett Quotes on Money You Need to Hear

The 10 Highest Suicide Rates by Profession

The 20 Most Underrated Movies of All Time

The 10 Fastest Growing Companies in America

The 10 Biggest Outlet Malls in USA

The 5 Most Popular Rap Songs of All Time

The 10 Countries that Eat the Most Meat

The10 Most Expensive Countries to Fly To

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!