Conference Call 48-Week Results From the Phase IIb exon 51 skip DMD Study

Sarepta Therapeutics Announces Conference Call and Webcast on Wednesday, October 3, 2012, to Discuss 48-Week Results From the Phase IIb DMD Study

Oct 02, 2012 (Marketwire via COMTEX) --Sarepta Therapeutics, Inc. (NASDAQ: SRPT), a developer of innovative RNA-based therapeutics, announced today it will hold a conference call at 8:00 a.m. EDT (5:00 a.m. PDT) on Wednesday, October 3, 2012 to discuss 48-week results from its Phase IIb study evaluating eteplirsen for the treatment of Duchenne muscular dystrophy (DMD). 

The conference call may be accessed by dialing 866.356.3093 begin_of_the_skype_highlighting FREE 866.356.3093 end_of_the_skype_highlighting for domestic callers and 617.597.5381 begin_of_the_skype_highlighting FREE 617.597.5381 end_of_the_skype_highlighting for international callers. The passcode for the call is 93880948. Please specify to the operator that you would like to join the "Sarepta Therapeutics 48-Week Results Call." The conference call will be webcast live under the events section of Sarepta's website at and will be archived there following the call for 90 days. Please connect to Sarepta's website several minutes prior to the start of the broadcast to ensure adequate time for any software download that may be necessary.

About Sarepta Therapeutics

Sarepta Therapeutics is focused on developing first-in-class RNA-based therapeutics to improve and save the lives of people affected by serious and life-threatening rare and infectious diseases. The Company's diverse pipeline includes its lead program eteplirsen, for Duchenne muscular dystrophy, as well as potential treatments for some of the world's most lethal infectious diseases. Sarepta aims to build a leading, independent biotech company dedicated to translating its RNA-based science into transformational therapeutics for patients who face significant unmet medical needs. For more information, please visit us at

Sarepta Investor and Media Contact:         Erin Cox         425.354.5140 begin_of_the_skype_highlighting FREE 425.354.5140 end_of_the_skype_highlighting         Email Contact 

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Hi Phil,


As different mutations cause the disruption of the genetic code, different exons have to be skipped for different patients.

Most patients (13%) would benefit from exon 51 skipping. This is currently tested in phase III clinical trials to see whether exon skipping is indeed effective (slows down disease progression) and safe.


In the case of my son, he would need skipping of exon 34. This would bypass the small mutation, but would not disrupt the genetic code.


At the moment the focus of the clinical development is on those exons that apply to larger groups of patients (exon 51 skipping, exon 45 skipping, exon 53 skipping and exon 44 skipping).

The companies who are developing exon skipping want to develop compounds for as many exons as possible.

But this depends on whether exon 51 skipping really works, as we all hope and believe, and whether the rules for development of therapies for this disease become more flexible. If we can organize and try to accelerate this, will be excellent for all the DMD community, so there could be treatment per each specific mutation.


Best regards




Thanks for your answers, David & Gonzalo.  This is the transcript from the 48 week call

 A disturbing article:

"One caution is that the study included only 12 participants; larger studies will be needed before eteplirsen can be considered for approval as a drug."  I put a comment at the end.  IMO the DMD community as a whole needs to be pushing for the accelerated approval of eteplirsen.   The MDA saying it can't happen is irresponsible.

Summer Street Research had a conference call this am with an ex FDA official>  Some second hand comments from that call regarding eteplirsen:

  • clinically meaningful and the safety profile is clean - makes it very likely the FDA can find a way to approve
  • Docs summary of thougts - we see data the product is working on the route cause of the disease - seeing improved performance that is
  • Former FDA reviewer downplays concerns about 30 mg dose response. "Clinical outcome trumps surrogate marker."
  • Former FDA officials say mfr is important detail. can't be approved w/o satsifactory means of manufacturing
  •  Former FDA official calls competing$GSK/Prosensa drug "far inferior" to eteplirsen
  • Former FDA official is POSITIVE on $SRPT receiving accelerated approval.

WOW! These are encouraging comments.I personally didn't hear the call, but think these arre reasonably accurate.

Just for the record, I am a small investor in Sarepta.  I don't personally know any families with duchenne.  At one time in distant past I helped design some devices for impaired children.  I usually stay quiet in here, but if I think I can help, I will jump in.




Very sloppy, I agree

We must be united in our support of accelerated approval. My son can't be helped (directly) by this approval, but I believe it nonetheless.

As micro kid points out in his comment, how many other drugs are tested on nearly 1% of affected US population during trials? That is A HUGE NUMBER, compared to other drugs that have been approved. The absolute number of 12 or 10 is meaningless. This is the only number FDA should consider when asking - has this been tested broadly enough?


Careful about expectations for what the drug can do.  There's no indication that exon skipping restores fibrotic tissue.  The drug turns Duchenne in to Becker, and exon skipping will work better on some exons than others.  It's not a cure for MD, but certainly a very big step in the right direction.

David said:

What I cannot stop thinking about ... not the stock PPS, the conditions for FDA approval, the financial partnerships that may develop ...

What I cannot stop thinking about are a handful of young men who are PUTTING AWAY THEIR CHAIRS.

2012 is Year 0


David said:

They are NOT the same chemistry, no...

But Prosensa uses PPMO chemistry while Sarepta uses PMO. The exact differences are beyond my understanding, but there are significant differences.

Prosensa uses 2'-O-methyl phosphorothioate RNA oligos (2'-O-me PS) while Sarepta uses phosphorodiamidate Morpholino oligos (PMO). PPMO are also used by Sarepta; these are peptide PMOs, Morpholino oligos linked to cell-penetrating peptides to improve their entry into cells. The 2'-O-me PS oligos degrade slowly in cells, releasing monomers with toxic terminal phosphorothioate groups. Morpholino oligos do not degrade in cells and survivable doses have been reported up to 3g/kg in mouse (at that dose, the mice were 0.3% oligo by mass -- the paper was from Qilong Lu's group).

It is my understanding that it is now FACT (not expectation) that at least 1 young man has a) stopped using a chair to get to school and b) stopped using the accessible bus since the stairs no longer prove a challenge.

Of course, we don't know that such gains will be sustainable over time (yet). But that does not diminish the enormity of this event.

So what is the next step? Do they have to run another trial with more children or do they have

enough to ask the FDA for approval?

It's so ironic that when I was looking for answers, genetics testing was the last exam to get the diagnose, and even the FDA has not approved this test. 

For this kind of diseases, we need velocity to get more effective treatments approvals, and an integral solution for all kind of mutations, if the technique seems to work and turns a DMD into a BMD, it's a great goal! 

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