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Sarepta Therapeutics: Business Report

March 7, 2013

Sarepta Therapeutics is busy this week with two meetings :

  1. Monday, March 4, Sarepta Therapeutics gives a live webcast presentation at the Cowen Health Care Conference
  2. Thursday, March 7, Sarepta Therapeutics’ President and CEO, Chris Garabedian, reports on the company’s fourth quarter and Full-Year (FY) 2012 financial results.

What is on the mind of most investors and Duchenne Muscular Dystrophy (DMD) advocates, are the questions related to orphan drug Eteplirsen :

  1. Will the company file for FDA Accelerated Approval ?
  2. How favorable will the FDA be ?
  3. Will the company seek the new FDA Breakthrough Therapy Designation ?

The following are highlights from the Sarepta Therapeutics’ March 7 webcast from SeekingAlpha’s transcript :

–  2012 is a great year for exon-skipping drugs for DMD :

  1. Eteplirsen, a exon 51 skipping agent drug, makes extensive progress
  2. 3 additional DMD drugs targeting exons 45, 53, & 50 are in preclinical development.

– There is $187 million in cash on the company’s balance sheet at the end of 2012.  This allows the company to “maintain Eteplirsen on a critical path toward approval & to invest in larger scale production capability necessary to supply the drug for future …”

– In December 2012, additional clinical information is presented from the Phase 2B (Study 202) extension study of Eteplirsen –  data from the 62-week 6-minute walk test

– The company plans on presenting further clinical data for Phase 2B (Study 202), including 74-week 6-minute walk test data in the 1st half of 2013

– A meeting with the FDA for Eteplirsen is scheduled for later in March 2013. The purpose of this meeting is to review results from Study 201 & Study 202, results from earlier clinical & non-clinical data, and to discuss what is the feasibility of filing for an accelerated FDA regulatory pathway for Eteplirsen

– There is no trial discontinuation or patient hospitalization, and all patients continue to receive treatment

– Acknowledgement of the key role the DMD community plays in representing “the unmet medical need in the patient population”

– Will communicate the next steps for the Eteplirsen program as soon as the company has “sufficient information and are confident in our plan”

– Will have an additional conversation with the FDA in the 2nd Quarter of 2013, about the manufacturing or CNC section of an NDA

– The plan is to start the initiation of a confirmatory study in the latter part of 2013, with dosing to start in the 1st Quarter 2014

– Announcement in November 2012 of a new collaboration to develop an exon-skipping drug to target exon 53 for DMD

– Selection of lead sequences in all 3 of our follow-on DMD drugs (will be identified as SRP 4045, SRP 4050, SRP 4053) with preclinical studies to begin later in 2013

– Filing for Eteplirsen for the new FDA Breakthrough Therapy designation : “ … this is something that we have prepared ourselves for. If we decided to pursue a breakthrough designation filing, I’ve communicated previously that we would make that determination in the 1st half of 2013 … stay tuned .. will have our strategy determined in the 1st half of this year & stay tuned …”.

Thus, on the question of whether or not Sarepta Therapeutics will seek FDA’s Accelerated Approval or the FDA’s Breakthrough Therapy designation for Eteplirsen is still not clear. The decision will hinge on the meeting with the FDA at the end of March 2013. It is clear though that both Sarepta Therapeutics and DMD patient advocates would like to see Eteplirsen follow an accelerated regulatory pathway.

Recently, GlaxoSmithKline ‘s DMD drug, drisapersen, has raised safety concerns. There were reports about the drug resulting in kidney toxicity. Mr. Garabedian expressed confidence in the safety profile of Eteplirsen.

Referenes

SeekingAlpha.com article “Sarepta Therapeutics’ CEO Discusses Q4 2012 Results – Earnings Call Transcript”

Please Note : “Dialog Ballons” by User:Felix.leg (Polski: praca własna English: own work) [Public domain] | Wikimedia Commons.

Copyright © 2012-2013, Orphan Druganaut Blog. All rights reserved.

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