Sorry!! The article you are trying to read is not available now.
Thank you very much;
you're only a step away from
downloading your reports.

Sarepta Therapeutics' Shares Skyrocket on Success of Muscular Dystrophy Drug

By

The company hopes positive study results for the drug eteplirsen will help it win US approval of its treatment for the genetic disorder Duchenne muscular dystrophy.

PrintPRINT
MINYANVILLE ORIGINAL Shares of Sarepta Therapeutics (NASDAQ:SRPT) shot up more than 170% after the drugmaker said its experimental treatment for a type of muscular dystrophy helped boys' ability to walk in a study.

In a study of boys with the genetic disorder Duchenne muscular dystrophy, those taking Sarepta's drug eteplirsen were able to walk much farther
after 48 weeks. In a six-minute walking test, those on Sarepta's medicine were able to walk on average almost 90 meters farther than those taking a placebo.

The results were "unprecedented" for treatment of this disease, and the drug so far is shown to be safe, CEO Chris Garabedian told investors on a conference call Wednesday morning.

Shares of Sarepta soared 170% to $40.46 in morning trading Wednesday. The stock is up more than 800% for the year.

Garabedian said Sarepta hopes to win an expedited path to US approval. The drug is in the second of three phases of study usually needed for Food and Drug Administration clearance.

"These results provide tremendous promise for patients," Garabedian said.

The drug also is attracting attention of bigger drug companies who may be interested in partnering to help develop and market eteplirsen, according to Garabedian.

The walking improvement was demonstrated in a small group of patients -- just 12 boys, ages seven to 13. But the results were impressive for such a hard-to-treat disease. Duchenne muscular dystrophy takes place in about one in every 3,600 male infants, according to government figures. It is caused by a defective gene for the protein dystrophin.

"These data represent a significant milestone and a defining moment of progress and hope for patients with (Duchenne muscular dystrophy) and their families, as well as those of us in the scientific community who have been pursuing potential treatments for this devastating and deadly disease for decades," the study's lead researcher Jerry Mendell says in a statement.

Mendell, director of the the Centers for Gene Therapy and Muscular Dystrophy at Nationwide Children's Hospital, says the study results showed that eteplirsen both helped production of dystrophin and slowed progression of the Duchenne disease. The drug targets a specific genetic mutation but implications for all Duchenne "patients with related genetic mutations are clearly evident," he says.

Twitter: @brettchase

Follow the markets all day every day with a FREE 14 day trial to Buzz & Banter. Over 30 professional traders share their ideas in real-time. Learn more.
< Previous
  • 1
Next >
No positions in stocks mentioned.
The information on this website solely reflects the analysis of or opinion about the performance of securities and financial markets by the writers whose articles appear on the site. The views expressed by the writers are not necessarily the views of Minyanville Media, Inc. or members of its management. Nothing contained on the website is intended to constitute a recommendation or advice addressed to an individual investor or category of investors to purchase, sell or hold any security, or to take any action with respect to the prospective movement of the securities markets or to solicit the purchase or sale of any security. Any investment decisions must be made by the reader either individually or in consultation with his or her investment professional. Minyanville writers and staff may trade or hold positions in securities that are discussed in articles appearing on the website. Writers of articles are required to disclose whether they have a position in any stock or fund discussed in an article, but are not permitted to disclose the size or direction of the position. Nothing on this website is intended to solicit business of any kind for a writer's business or fund. Minyanville management and staff as well as contributing writers will not respond to emails or other communications requesting investment advice.

Copyright 2011 Minyanville Media, Inc. All Rights Reserved.
PrintPRINT
 
Featured Videos

WHAT'S POPULAR IN THE VILLE