Biomarins Utrophin Upregulation phase 1 trial slated for first quarter of 2010

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-- BMN-195 - Utrophin upregulator for Duchenne Muscular Dystrophy:
BioMarin
is in the final stages of preparing the regulatory filing and expects
to
initiate a Phase I trial by the first quarter of 2010. BMN-195 is an
orally available small molecule which may upregulate utrophin, a
potential substitution for the missing dystrophin protein in DMD
patients.
http://www.reuters.com/article/pressRelease/idUS228413+28-Oct-2009+...

[Oct 29, 2009 1:17:24 PM]

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Permalink Reply by cheryl cliff on October 29, 2009 at 9:44am

Another FANTASTIC bit of DMD news for us in America. My understanding, such as it is, is this trial is to be held on healthy volunteers. Phase II, I think will be for DMD boys.
Keeping fingers crossed on this one since it may help all boys-my son included, Jacob too Donna!!

:)

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Permalink Reply by Lori Ware on October 29, 2009 at 10:25am

Cheryl, if that is right...phase one only on heathly volunteers (and I remember that being said from Conference 2 years ago), then they also said that that would make the phase 1 trial go faster. Which, of course, is GREAT! I will sign up if they need me too! I mean, I have PLENTY of fat that can be converted to muscle after all!!!! I will be more than happy to donate on behalf of our boys!!! Don't mean to make light...this is FABULOUS news...

cheryl cliff said:

Another FANTASTIC bit of DMD news for us in America. My understanding, such as it is, is this trial is to be held on healthy volunteers. Phase II, I think will be for DMD boys.
Keeping fingers crossed on this one since it may help all boys-my son included, Jacob too Donna!!

:)

▶ Reply

Permalink Reply by Bains on October 29, 2009 at 12:43pm

I hope they consider opening the trails for DMD boys as well. I am not sure why this extra step.

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Permalink Reply by Lori Ware on October 29, 2009 at 12:56pm

I don't think this is an extra step. THis is the phase 1 for safety. It will be quicker and easier to try this on healthy individuals vs. DMD boys. If they prove safety, they can go to the phase 2 and at that time include our boys.

Bains said:

I hope they consider opening the trails for DMD boys as well. I am not sure why this extra step.

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Permalink Reply by cheryl cliff on October 29, 2009 at 1:42pm

Hi Bains,

My understanding, again such as it is, is that doing clinical trial phase I's on healthy individuals is normal protocol. What is unusual is starting a clinical trial (phase I) using actual patients...especially children, as we have seen with Exon Skipping trials held in the UK. This phase I by BioMarin won't be considered an extra step, it is the first step toward approval for Utrophin upregulation with DMD boys.

Pray to god everyday this stuff works!!

Bains said:

I hope they consider opening the trails for DMD boys as well. I am not sure why this extra step.

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Permalink Reply by Bains on October 29, 2009 at 1:45pm

Thanks Lori,Cheryl. I was not aware of that. I hope and pray that this stuff works.

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Permalink Reply by Lori Ware on October 29, 2009 at 2:21pm

i know that phase 1 for PTC124 was on DMD boys....

Bains said:

Thanks Lori,Cheryl. I was not aware of that. I hope and pray that this stuff works.

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Permalink Reply by Jessica Rownd on October 29, 2009 at 2:24pm

God I hope this works. Wyatt can only benefit from this, exon skipping wouldn't help because of his extra large deletion 18-41.

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Permalink Reply by Allyson on October 29, 2009 at 2:48pm

Interesting for those with splice site mutations as well.

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Permalink Reply by cheryl cliff on October 29, 2009 at 2:50pm

Oh yea, PTC did do phase I on DMD boys. Keep fingers, toes, eyes, everything crossed that this stuff works Lori!!

Lori Ware said:

i know that phase 1 for PTC124 was on DMD boys....

Bains said:
Thanks Lori,Cheryl. I was not aware of that. I hope and pray that this stuff works.

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Permalink Reply by Jacobs Mommom on October 29, 2009 at 4:08pm

I'm with your Lori! Where do we sign up!

Lori Ware said:

Cheryl, if that is right...phase one only on heathly volunteers (and I remember that being said from Conference 2 years ago), then they also said that that would make the phase 1 trial go faster. Which, of course, is GREAT! I will sign up if they need me too! I mean, I have PLENTY of fat that can be converted to muscle after all!!!! I will be more than happy to donate on behalf of our boys!!! Don't mean to make light...this is FABULOUS news...

cheryl cliff said:
Another FANTASTIC bit of DMD news for us in America. My understanding, such as it is, is this trial is to be held on healthy volunteers. Phase II, I think will be for DMD boys.
Keeping fingers crossed on this one since it may help all boys-my son included, Jacob too Donna!!

:)