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Permalink Reply by cheryl cliff on January 21, 2009 at 9:38am

Thanks Eileen! This is fantastic news and it was JUST announced - minutes ago!!! Thank God for PPMD parents!
cheryl

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Permalink Reply by Joanne Haswell on January 21, 2009 at 12:21pm

Thank you Eileen---I pray that this keeps rolling along quickly for our boys.
Joanne

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StaffPermalink Reply by Pat Furlong on January 21, 2009 at 12:38pm

Eileen,

I understand completely about the urgency/need/anxiety around the loss of ambulation and while the discussion today suggested that muscle quality presents a significant factor with regard to uptake. But please remember that, when you are thinking about independence, it is actually the upper body strength that is essential. At this time, there are no validated measures for upper body strength. We will be getting together with our international partners to discuss this urgent need and do whatever it takes to move forward. As we think about outcome measures - stabilization of disease (no further progression) would be remarkable for instance. OR turning over in bed which improves the life of both the individual as well as his caregivers. The important point here is that the goal is to EndDuchenne, stop the progression for every boy.
Warm regards,
Pat

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Permalink Reply by Ofelia Marin on January 21, 2009 at 12:40pm

I was able to listen to the Q&A session in the conference call. Very interesting and informative.
http://www.avibio.com/pr/pr406.php

About the dystrophin expression the answer was “unequivocal, wide spread, robust response in terms of dystrophin positive fibers” and “results highly competitive with Prosensa”. Very important, no immune response was detected! They cannot provide any data at this time, the results will be published.

Important, about PPMO, only the work funded by Charley’s Fund for skipping exon 50 using PPMOs was mentioned. One such trial is possible to start in 2010 in the US. Other than that, skipping exon 51 in US could start in 2009 using PMO (not PPMO!) after the clinical hold from FDA is lifted. Unfortunately, all the info coming from Eric Hoffman’s group about starting trials using PPMOs for other exons like 51 etc. in 2009 doesn’t seem to hold. The only trial planned using PPMOs is for skipping exon 50, work funded by Charley's Fund.

The earliest they could fill for market approval (if everything works fine) would be 2012.

Of course, they would like to find a partner with deep pockets, which would allow for conducting parallel trials for several exons.

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Permalink Reply by Bains on January 21, 2009 at 8:06pm

This is really good. I hope they consider expanding th scope of trials to all possible (shippable) exons. We need exon 11 skipping and it is a anxious race against time!!!!

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Permalink Reply by Mindy on January 22, 2009 at 12:46pm

The conference call mentioned that the IND status in the US for this drug is on clinical hold by the FDA. Does anyone know why?

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Permalink Reply by Ofelia Marin on January 22, 2009 at 1:36pm

My understanding from the CC was that the hold is until the mouse toxicology work is completed. Now I am not sure how they were able to start the trial in UK w/o complete toxicology data (?). Are the FDA requirements a lot more strict than the EMEA's?

Of course, the hope is that the first results from the UK systemic trial will be convincing and FDA will lift the hold. I think he mentioned that the results for the first cohort of patients in that trial could be available Q2-Q3 2009.


Mindy said:

The conference call mentioned that the IND status in the US for this drug is on clinical hold by the FDA. Does anyone know why?

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Permalink Reply by Mindy on January 22, 2009 at 7:41pm

Ofelia - that's what I'm wondering. Toxicology studies would have to be in place before the EMEA would allow the study to begin. What is the FDA requiring that the EMEA did not? Why am I not British?

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