PPMD Conference 2008

Could someone who attended the conference summarize the info presented during research sessions? Any news about clinical trials, existing ones (Prosensa, MDEX) and upcoming (Summit's Utrophin upregulation, Byglican)?

Thanks,
Ofelia

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Permalink Reply by Pat Moeschen on July 21, 2008 at 3:04pm
A summary would be so hard, since there was so much info! Things are moving ahead, with of course, safety at the top of this list. We had 2 hours of pharma presentations on Saturday, with positive news. I believe some presentations will be available on the site later in the month.
Permalink Reply by Ofelia Marin on July 21, 2008 at 3:34pm
Any news about clinical trials other than what we already know: Prosensa's proof of principle completed, systemic delivery started May '08; MDEX proof of principle started Dec '07; Summit's utrophin upregulation scheduled to start '09.

What about MDEX/AVI systemic delivery for exon 51 (any news about their proof of principle?), Byglican, Losartan etc.???
Permalink Reply by Lori Ware on July 21, 2008 at 5:46pm
Ofelia Marin said:
Any news about clinical trials other than what we already know: Prosensa's proof of principle completed, systemic delivery started May '08; MDEX proof of principle started Dec '07; Summit's utrophin upregulation scheduled to start '09.

What about MDEX/AVI systemic delivery for exon 51 (any news about their proof of principle?), Byglican, Losartan etc.???
I think we will all have specific things that we remember specifically; for me it is byglican will be in trial around the 2010-2011 timeframe with Summet's Utrophin within 18 mths. There is also Debio-025 from Cincinatti in conjunction with a Swiss lab that will be in the 2010 time frame as well. it was agreed by all that the answer is closer, but it will still be a cocktail...albeit a good one!
Permalink Reply by Ofelia Marin on July 21, 2008 at 6:12pm
Lori Ware said:
Ofelia Marin said:
Any news about clinical trials other than what we already know: Prosensa's proof of principle completed, systemic delivery started May '08; MDEX proof of principle started Dec '07; Summit's utrophin upregulation scheduled to start '09.

What about MDEX/AVI systemic delivery for exon 51 (any news about their proof of principle?), Byglican, Losartan etc.???
I think we will all have specific things that we remember specifically; for me it is byglican will be in trial around the 2010-2011 timeframe with Summet's Utrophin within 18 mths. There is also Debio-025 from Cincinatti in conjunction with a Swiss lab that will be in the 2010 time frame as well. it was agreed by all that the answer is closer, but it will still be a cocktail...albeit a good one!

I was under the impression that Summit's trial will start next year. 18 months is not what they told me 2 months ago. Also, last year Justin Fallon said that Byglican will be in trials in 18 months, that is why I expected that trial to start next year also. Did he say 2010-2011 now?
Permalink Reply by Ofelia Marin on July 22, 2008 at 4:46pm
It looks like Summit is planning a 2009 clinical trail.

BioMarin and Summit plc Sign Worldwide Licensing Agreement for Duchenne Muscular Dystrophy Program

http://www.pharmalive.com/News/index.cfm?articleid=557720&categ...

NOVATO, CA, and OXFORD, UK, July 22, 2008 – BioMarin Pharmaceutical Inc. (Nasdaq and SWX: BMRN) and Summit Corporation plc (AIM: SUMM) announced today that they have entered into an exclusive worldwide licensing agreement for Summit’s novel preclinical candidate SMT C1100 and all follow-on molecules, which are being developed to treat the fatal genetic disorder Duchenne muscular dystrophy (DMD).
Permalink Reply by MicahsDaddy on July 22, 2008 at 5:20pm
the good thing about utrophin upregulation, since we already have this in our system, is that the initial trial in humans can be done with healthy subjects.
Permalink Reply by Jacobs Mommom on July 22, 2008 at 8:54pm
So where do we sign up:)

MicahsDaddy said:
the good thing about utrophin upregulation, since we already have this in our system, is that the initial trial in humans can be done with healthy subjects.
Permalink Reply by Ofelia Marin on July 22, 2008 at 9:08pm
More details about Summit's C1100 and the deal with BioMarin can be found in the presentation here: http://www.investorcalendar.com/IC/CEPage.asp?ID=132320

-2-3 interesting slides about C1100
-clinical trial to start early 2009
-6 minute walk outcome measure to be used
-launch potential in 2011 IF everything goes well (BioMarin has good track record with orphan diseases, 3 other drugs for orphan diseases launched in 6 years)
-3 independent lab confirmations in Italy, US and UK of initial benefit of drug work

StaffPermalink Reply by Ryan Fischer on July 23, 2008 at 12:47pm
Hey everyone-

We hope to have slides up in the coming weeks, I will also be working on gettting podcasts of some of the sessions as well.

I'll be in touch!

Ryan
Permalink Reply by R. Gardiner on July 25, 2008 at 10:14am
Podcasts would be great. Please try and get podcasts out here.

Ryan Fischer said:
Hey everyone-

We hope to have slides up in the coming weeks, I will also be working on gettting podcasts of some of the sessions as well.

I'll be in touch!

Ryan
Permalink Reply by Kristi Koop on September 11, 2009 at 1:40pm
Did I dream reading somewhere that the slides are up from the conference? I can't seem to find them on here anywhere.

Thanks,
Kristi

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