Hi All,

 

A quick reminder for all of us since I see very little mention of Biomarin's utrophin phase 1 trial.If they get desired results phase 2 could start early next year.We all need to get behind this and do all we can to help speed this one into next level where our son's can potentially take part in their future trial.

 

Somehow we need to influence biomarin and who better than Pat Furlong can do this for us. We need Biomarin to be as good as Ptc in terms of communication and patient advocacy.Any Ideas please share and lets get behind pushing this one fast.

 

Regards

 

Neeraj

Views: 275

Reply to This

Replies to This Discussion

I emailed Biomarin some questions and got this response...really nothing....but it is hopeful. I just hope the FDA does not make them go back to phase 1 in DMD boys...but still, it is progress.

I also wonder with the PTC 124 announcment, how will that affect THEIR utrophin product????


Dear Ms. Ware,

BMN 195, a small molecule utrophin upregulator, is an investigational product for the treatment of Duchenne Muscular Dystrophy (DMD), an X-linked recessive disorder that is characterized by progressive skeletal muscle and cardiac dysfunction due to mutations in the structural gene, dystrophin. In January 2010 BioMarin initiated a Phase 1, Double-blind, Placebo-controlled Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of Single and Multiple Doses of BMN 195 administered orally to healthy volunteers. Initial top-line study results are expected in the third quarter of 2010.

At this time the Phase 1 study is only accepting healthy volunteers. BioMarin will keep your request on file as the program continues its development. Please visit the BioMarin website for updates regarding this program.

Please understand that Medical Information is not able to respond to specific questions. Thank you for your interest in our company's programs.

For additional questions of a medical nature, please contact the BioMarin Medical Information Services at (800) 983-4587 or medinfo@bmrn.com. If you received this information in error, or did not request this information to be sent to you, please contact BioMarin Medical Information Services at (800) 983-4587.

Sincerely,

David Wells, PhD, RPh
Medical Information
On Behalf of BioMarin Pharmaceutical Inc.
You've echoed my thoughts...

I, like everyone, was disappointed by the results of Alaturen this week, At the same time, as a dad whose son has a relatively obscure deletion, I have placed my hope in treatments that will help all boys, regardless of their mutation. Why has Biomarin received so little attention? I have begun to wonder if my optimism is misplaced. Does anyone know something I don't know?
I think this Biomarin product is high on everyone's wishlist - maybe it seems like it's had less attention here because it's a a phase 1 trial with healthy volunteers - ie, none of our boys are involved yet.
BioMarin's BMN-195 is important because it is a follow up on Summit Plc's SMT C-1100, which was developed under the leadership of Dr. Kay Davies, perhpas the leading researcher on the upregulation of utrophin. BioMarin purchased this line of business from Summit some years ago. I understand that in mice the upregulation of utrophin has worked well in terms of protecting the muscle fibers, but as we all know mice are not a particularly suitable model to extrapolate to humans, as they do not have any of the symptons of DMD. However, for what I read, they have tried this method in genetically modified mice which lack both utrophin and dystrophin, and the presence of the "new" utrophin through the drug has extended those particular mice's life spans considerably. Bue this is going to take some years, since BMN-195 is only in Phase 1.

Another line of research that involves the upregulation of utrophin is PTC's Project Catalyst, where thay are aiming at screening a compound that can produce that effect, among others. I wonder what will happen with Project Catalyst now.

Regards

Bernardo
Thanks Keith and Bernardo -

After last week's awful disappointment, hope can feel a little dangerous, but I agree, everything I have read about utrophin up-regulation is guardedly optimistic.
I listened in to the BIOMARIN Webinar for investors today...here's what was said about BMN-195. And before anyone asks, I'm not sure what kind of increase in utrophin expression one would want to see to translate into strength gains (will check on that), and also keep in mind that this result was in cultured cells.

Hank Fuchs, M.D., Chief Medical Officer of BIOMARIN reported that its utrophin upregulating molecule BMN-195 is currently in a phase I safety study, which was initiated in the first quarter of this year and is scheduled to be completed in the third quarter. He showed data suggesting that in cultured human muscle cells BMN-195 can increase the level of messenger RNA for utrophin by 37% and can increase the level of utrophin protein by 45%.
Sharon,

A not directly related question...is there a 'typical' amount of time that it takes for a trial to run entirely through the trial process?

Steve
Hi Steve: This is probably the long answer to a short question, but here's a link to my blog post about clinical trials...it includes a discussion of the length of the different stages: http://community.parentprojectmd.org/profiles/blogs/the-process-of-...

Sharon
They presented the same data on January 11. This is the slide presented at that time:

BMN 195 induces utrophin upreg in human muscle cells in-vitro
Increase in Utrophin
-mRNA level (by 37%)
-protein level (by 45%)

Optimal dose 2 uM

Same range as found in positive controls (heregulin and okadaic acid)

Phase I results Q3 2010 hopefully shortly followed by launch of Phase II


Sharon Hesterlee said:
I listened in to the BIOMARIN Webinar for investors today...here's what was said about BMN-195. And before anyone asks, I'm not sure what kind of increase in utrophin expression one would want to see to translate into strength gains (will check on that), and also keep in mind that this result was in cultured cells.

Hank Fuchs, M.D., Chief Medical Officer of BIOMARIN reported that its utrophin upregulating molecule BMN-195 is currently in a phase I safety study, which was initiated in the first quarter of this year and is scheduled to be completed in the third quarter. He showed data suggesting that in cultured human muscle cells BMN-195 can increase the level of messenger RNA for utrophin by 37% and can increase the level of utrophin protein by 45%.

Reply to Discussion

RSS

Need help using this community site? Visit Ning's Help Page.

Members

Events

© 2020   Created by PPMD.   Powered by

Badges  |  Report an Issue  |  Privacy Policy  |  Terms of Service