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I was able to listen today's CC. They will post the audio within approximately 24 hours at www.avibio.com/events.php.
The Q&A session was very interesting.
A few key points:
- DMD is a national priority for Dept. of Defense along with breast and prostate cancer hence the funding;
- $ 2.5 MM will be used on preclinical GLP tox work, from what I got this is NOT a new request from FDA; it is the original request for completing toxicology 3-months exposure in animals (after the 3 months, they need to analyze the data, write the report and send to the FDA for a 30-day review); 2 of the GLP tox studies already started in non-human primates and mice; they plan to send the clinical trial protocol to the FDA at the same time;
- the 1st Phase of the US trial will be Safety with dose escalation design probably (the guy did not seem very convinced which shows that they did not finalize their plans);
- when asked directly about timeline they did not give a clear answer about the start of the US trials;
- when asked about milestones for 2009 they only mentioned the UK trial, when they hit 2-4 mg/kg dose level (during Q4 2009) they will give a full update; they still hope that the therapeutic range will be close to 4 mg/kg, for a 30 kg boy, that means 1500 mg/12 weeks/patient; they plan have an amendment to increase this dose to obtain higher % dystrophin (he said something like, if 4 mg/kg could give us half of the IM's 40% we would still like to increase the dose w/i safety limits);
- about the PPMO skipping 50, IND data package expected to be available in the 2nd half of 2010
Ofelia,
This is what I don't understand. They are currently doing safety dose escalation studies in the UK. Why would AVI spend tons of money just to repeat the same exact study in the US? It makes no sense at all. I'm not too psyched about moving to Columbus to participate in a safety study. That absolutely won't happen.
Also - Dr. Hoffman told me that 200 mg/kg is what worked in the dogs. I wonder why they think that 4 mg/kg will work in the boys?
Ofelia,
This is what I don't understand. They are currently doing safety dose escalation studies in the UK. Why would AVI spend tons of money just to repeat the same exact study in the US? It makes no sense at all.
I'm not too psyched about moving to Columbus to participate in a safety study. That absolutely won't happen.
Also - Dr. Hoffman told me that 200 mg/kg is what worked in the dogs. I wonder why they think that 4 mg/kg will work in the boys?
One thing that I really cannot understand is why they spend $2.5 MM on preclinical data when, by Q4 2009, they will have more than 3 months of clinical data at the highest dose level from the UK trial. How does spending the money on preclinical in mice accelerate developing this treatment when AVI-4658 is already tested in boys since Jan 2009? I would kind of understand if the completion of these GLP tox studies would be much earlier than obtaining the data from the UK trial, but that doesn't seem to be the case anymore. What is the reasoning for not looking at human data when it's available and waiting for animal data instead, not to mention spending $2.5 MM for this animal data?
Cheryl - if we lived in the same area, we could sit together and have a really, really good outburst.
Columbus told me that this was indeed an efficacy study, not a safety study, and I trust the people I talked to, so that means AVI isn't being forthright with the clinicians who are conducting the trial.
I get that the FDA wants a round of safety studies, but then wouldn't they just say, OK - wait for the results of the UK systemic trial? Why repeat them? It's not like anyone's got that deep of pockets these days.
I, too, am very frustrated.
Cheryl - if we lived in the same area, we could sit together and have a really, really good outburst.
Columbus told me that this was indeed an efficacy study, not a safety study, and I trust the people I talked to, so that means AVI isn't being forthright with the clinicians who are conducting the trial.
I get that the FDA wants a round of safety studies, but then wouldn't they just say, OK - wait for the results of the UK systemic trial? Why repeat them? It's not like anyone's got that deep of pockets these days.
I, too, am very frustrated.
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