AVI BioPharma Receives Approval of Nonproprietary Name Eteplirsen for Lead Duchenne Muscular Dystrophy Therapeutic Candidate, AVI-4658


AVI BioPharma (NASDAQ: AVII), a developer of RNA-based therapeutics, today announced that the United States Adopted Names (USAN) Council has approved the nonproprietary name eteplirsen for AVI-4658, the Company's exon-skipping therapy for the treatment of Duchenne muscular dystrophy (DMD), a genetic muscle wasting disease caused by the absence of functional dystrophin. In addition, the World Health Organization (WHO) has approved eteplirsen as the International Nonproprietary Name (INN) for AVI-4658.


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My monday was starting off badly, until reading this.  Thanks Raktim for posting this article.  Guess I've a couple of years to learn how to say eteplirsen before they get around to exons 44 & 45 though. 

Is there anything new in this presentation? They already presented all data from the UK trial. 


GSK however will present new data 48 week follow-up...that should be really interesting to see!


GSK2402968 Clinical Development Programme Update

The forty-eight week follow-up data from the phase I/IIa extension study (PRO051-02) will be presented at an oral session on April 13, 2011 during the 63rd annual meeting of the American Academy of Neurology.


arnold said:

Hi Cheryl,

It's really frustrating to see how AVI is moving. Sad part is that though they are working with MDEX team members but somehow that work has not moved. I just hope and PRAY TO GOD that they move faster.



avi is not sitting on a lot of cash, although they just had a stock offering to raise more. Some of this money will go to the duchenne trials.   I think they are working as fast as they can considering how small there are. The new CEO is focused on duchenne like no other one in the past. 





Thanks Arnold for the clarification. I do hope and PRAY TO GOD for their SUCCESS in duchenne.

Tomorrow AVI has a webcast and I expect to hear more from the CEO about the Duchenne trial




He is pressing forward as fast as he possible  can





I agree with Arnold, Dr. Mendell said that the put off from September to now was because of the CEO and as much as we hated the lag and the way everything came to a halt for sometime that this new CEO is really helping push things forward unlike the one in the past and that even with the lag once it gets moving he feels that because of this CEO that things will move together quite quickly because of the new CEO's passion for DMD.  He did say as well, (I asked about funding and whether the trial was funded fully/whether AVI Needed more money, etc etc) and he said that the AVI study for Duchenne was funded fully (even when mentioning stocks being sold, etc) and that the only thing left to work with would be raising funds to make sure that 12 families can live comfortably there for a year and that if we wanted to do some fundraising it would be ideal to send it to Dr. Mendell's team directly to help with this....guess we will see...We are up in the air as to whether we are "officially" in or not, but I think we might start doing a fundraiser to help with this....just wish I had the backing of a nonprofit as if I do the fundraising and send it off there's no tax writeoffs =(

I think the big problem is funding the pivotal study following this small 12 boy dosing study. AVI doesn't have the manufacturing facility for producing as much drug as needed in the large Phase 3 trial and afterwards if the drug is approved. They are working on it as we speak, I'm assuming the $30 mil raised will be mostly used for this.

 Thanks Cori and Arnold for the updates. This is looking good. PRAY TO GOD for successful and fast trial.


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