Excited to hear about Phase1!!! I understand that they will release more information at the end of the month but does anyone have an idea how long until they get a Phase 2 going?

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It is supposed to start earlier of 2013

One can hope that PPMD money might convince them to

a) run trial in the US

b) start phase II sooner

I was told earlier that donations to PPMD can be earmarked for specific programs. Don't know about the rest of you, but my year-end donations will be earmarked for this program.


I would love to do the same. I didn't see it when I looked under the donate tab. I also would like to email a link to some others who may be interested in donating. If you come across it can you forward it to me. Thank you!

I haven't figured out exactly how to make this work yet. It was part of this discussion (link below), about half-way down page 3 of comments is a comment from Pat Furlong.


I had been donating to Ballou Skies for last few years, am shifting some $ back to PPMD. I'll let you know what I find out.



Does any one know, at what stage did they last time terminate and decided to reformulate?

I am just wondering if we are sure that this new formualtion will be effective? thanks.

I think you are referring to Biomarin's work on utrophin drug, which is a different pharma company. They terminated their work in phase 2 study, not phase 1. I don't recall the details as to why they terminated it.

Summit then took up the the project and reformulated the drug with the goal of correcting whatever problem was there before under Biomarin. We won't know until phase 2 if they've succeeded.



Previously, the California-based biotechnology company BioMarin Pharmaceutical had tested a different formulation of the drug, which they called BMN 195, in healthy human volunteers and found that the compound was safe and well-tolerated. However, inconsistent levels of the drug were generated, with only some individuals achieving expected therapeutic levels in the blood.

Summit subsequently resumed development of the utrophin booster SMT C1100 and is using a different formulation, with the goal of increasing blood levels of the drug.

I believe Summit reached this goal in their Phase 1

Noooo!it was terminated in phase 2
I mean phase 1

Moein is right, the trial that failed at Biomarin was technically a Phase 1 trial.



thats great, it means we are moving forward. And lets hope and pray that it works.



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