AVI BioPharma Announces Treatment of First Patient in Systemic Clinical Trial of AVI-4658 for Treatment of Duchenne Muscular Dystrophy

This is good news, even though the timeline is not that good -- 2 years for this Phase(?):

Estimated Enrollment: 16
Study Start Date: January 2009
Estimated Study Completion Date: December 2011
Estimated Primary Completion Date: March 2011 (Final data collection date for primary outcome measure)

Let's hope that this will not delay the entry of the PPMO in trials.



PORTLAND, OR — February 19, 2009 — AVI BioPharma, Inc. (NASDAQ: AVII), a developer of RNA-based drugs, today announced treatment of the first patient in a clinical trial evaluating the systemic delivery of AVI-4658 for the treatment of Duchenne muscular dystrophy (DMD).

“We are very pleased to begin the systemic evaluation of our exon skipping drug — AVI-4658 — for the treatment of DMD,” said Stephen Shrewsbury, M.D., Chief Medical Officer and Senior Vice President, Clinical and Regulatory Affairs of AVI BioPharma. “We believe that this trial will build significantly on the data generated by the successful recent trial evaluating intramuscular administration of the same drug in DMD boys.” ...

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Is this the one for skipping exon 50?
No, this is bare morpholino (PMO) skipping exon 51.
This is exciting! =]
"The Company was granted an orphan drug designation for AVI-4658 by the U.S. Food and Drug Administration in November 2007 and by the European Medicines Agency in December 2008."

The phase 2 trials are taking place in the UK...even though the FDA granted the orphan drug designations a full year earlier. The FDA has become nothing but a road block to all new trials. North America is falling behind other parts of the world.

At some point we have to face the facts . We will all have to find treatments in other countries if we want to give our boys a chance.

What happened to this agency?
It looks like the lead researchers on this are all physically located in the UK, and I suspect this part also has something to do with the trial being located there...

"...awarded funding support of $1.3 million from the UK Medical Research Council to offset some of the clinical costs of the trial."
We are so new to this. My son was diagnosed in Oct. of 2008,. He is 6 years old and his deletion is exon 51. Does this mean he is eligible for this trial?
If he is eligible, who do I contact for him being a possible participant?
For an exon 51 deletion, I believe you need exon skipping for exon 50. My son has a deletion of 50 and needs 51 skipped, which is the trial they're speaking of. I do believe they are also working on skipping exon 50 in a separate trial, but it's a little futher behind in the process.

Julie Hathaway said:
We are so new to this. My son was diagnosed in Oct. of 2008,. He is 6 years old and his deletion is exon 51. Does this mean he is eligible for this trial?
Keith, I think that the FDA is making clinical trials very difficult. They have become so restrictive, that lead research will have to come from other countries.

I do not think that we would have started Phase-1 intermuscular trials here if it was up to the FDA. Thank God there are other regulatory agencies that still understand (within normal safety measures) what the objective is.
To help treat and/or cure.

Researchers will follow the path of least resistence to clinical trials and it will not be where the FDA rules. I guess you can question safety issues to the point were you never start. I hope I am wrong.
Frank - sounds like you're up on this.

Did AVI apply for a trial in this country and were denied or the decision delayed by the FDA? Are there other potential MD therapies that you feel have been held up at the FDA level?
Keith - I Just feel that there seems to be a trend.
- Two exon skipping trials in the UK, Netherlands
- Utrophin trials expected to start soon in the UK - after fast track approvals.
- Lots going on in Italy with stem cells

Why grant orphan drug status if you don't plan on helping fast track a clinical trial?
I do not know if AVI was denied clinical trials. They did apply for the orphan drug status, so you would assume they did this to fast track a trial. What happened?

Apparently, it was faster to get orphan dug approval a year later in the UK in order to start clinical trials.
I was informed that additional safety data was still required by the FDA. You can always question anything to death. Eventuallly you give up and move on. I think this is what AVI had to do to advance this drug.

Isn't AVI an American company? I think they would have prefered to have thier trials conducted in the US.

Thank God for other agencies.

I have to agree with Kelly. The FDA is moving very, very slowly. There are 2 systemic delivery exon skipping trials in Europe, this should be a good enough reason for them to lift the clinical hold they have on AVI's PMOs if safety was a concern.

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