Has anyone been tracking the current research being done on Utrophin upregulation. At what point are they in terms of clinical trials? From what I can gather, there are a few of them:
BioFocu DPI - Netherlands Utrophin Upregulation Assay
Cominbaion RX - Cambridge, MA Hi-Thruput Screening/ Nash Avery Foundation
Brown University - Justin Fallon, PhD Biglycan Utrophin
TAT-Utrophin, Dr. Ervasti.
Are any one of these pulling ahead in terms of bringing this to trials? Anybody leading the pack?
So much talk has been concentrated on exon skipping, they I don't hear much about utrophin upregulation.

Views: 67

Reply to This

Replies to This Discussion

My brain is not functioning at full throttle today, but I don't see Kate Busby's Utrophin from the UK. She is partnered with BioMarin (I believe) and their trials MAY be underway in the UK now....or they are about to start. In a recent (June) conversation with Dr. Wong, she said that realistically, the phase 2 trial in the US should be in 3-4 years...of course that is assuming all goes as planned. Then there is also PTC's Utrophin and at conference they said they should be in phase 1 in early 2011...
Kay Davies (UK) worked on a utrophin upregulation drug with a company called Summit and was sold to BioMarin. They plan to start Phase I trials next year.
http://www.bmrn.com/pipeline/utrophin-inducer.php

I do not see how Phase II can be that far in the future (3-4 years) -- did Dr. Wong say that it could be approved in 3-4 years (IF all goes well)? BioMarin developed 3 products until now: first 2 took 5 year from IND to market, the third took 3.5 years until marketed.
No, Dr. Wong and I were talking about Phase 2 coming to America. I am sure she was being overly cautious, she normally is. And I know she was considering the FDA in her comments. And...you are right, the trials for BioMarin are planned to start 1st quarter 2010...

Ofelia Marin said:
Kay Davies (UK) worked on a utrophin upregulation drug with a company called Summit and was sold to BioMarin. They plan to start Phase I trials next year.
http://www.bmrn.com/pipeline/utrophin-inducer.php

I do not see how Phase II can be that far in the future (3-4 years) -- did Dr. Wong say that it could be approved in 3-4 years (IF all goes well)? BioMarin developed 3 products until now: first 2 took 5 year from IND to market, the third took 3.5 years until marketed.
What does it mean: Phase II coming to US? BioMarin is in US and Phase I will be here as well. I never heard about a Phase I trial taking so long...3 years (assuming it starts 2010!).

Lori Ware said:
No, Dr. Wong and I were talking about Phase 2 coming to America. I am sure she was being overly cautious, she normally is. And I know she was considering the FDA in her comments. And...you are right, the trials for BioMarin are planned to start 1st quarter 2010...

Ofelia Marin said:
Kay Davies (UK) worked on a utrophin upregulation drug with a company called Summit and was sold to BioMarin. They plan to start Phase I trials next year.
http://www.bmrn.com/pipeline/utrophin-inducer.php

I do not see how Phase II can be that far in the future (3-4 years) -- did Dr. Wong say that it could be approved in 3-4 years (IF all goes well)? BioMarin developed 3 products until now: first 2 took 5 year from IND to market, the third took 3.5 years until marketed.
I thought BioMarin was going on with phase I here in usa. A short while ago Summit went BK and I watched things with them to see what impact that would have on BioMarin's progress with Utrophin. I contacted BioMarin aprox 6 mos ago to find more and they responded by saying they are going ahead but their trial was a bit slower to start. I was reassured they were not devistated by Summit's financial status, were a few mos behind their original start date and going on ahead.
Like I said, my brain is not working at full throttle, but Dr. Wong said that the phase 1 will be done in the UK....

As for a phase on taking 3 years, well PTC 124 was about that long...maybe 2, but I think 3. I know the boys that were in the phase one has to wait YEARS to get the drug back (yes, they were taken off of it!) to get back on it. I KNOW at least 2 and I think longer (Jill Castle would know for sure!)...

cheryl cliff said:
I thought BioMarin was going on with phase I here in usa. A short while ago Summit went BK and I watched things with them to see what impact that would have on BioMarin's progress with Utrophin. I contacted BioMarin aprox 6 mos ago to find more and they responded by saying they are going ahead but their trial was a bit slower to start. I was reassured they were not devistated by Summit's financial status, were a few mos behind their original start date and going on ahead.
Phase I will be in the US. BioMarin is working on this product alone, no connection with the UK company anymore.

I do not know how long it was from Phase I to Phase II of PTC124 BUT we need to take into account that 124 was PTC's first product (they did not have any experience marketing any drug before!) and the 6-minute walk test was not even validated at that point. So one can say that PTC was a pioneer in the field. BioMarin has experience bringing drugs for orphan diseases to market in short time. I would definitely expect less than 3 years for Phase I. In fact BioMarin said (during a CC last year) that they try to replicate the experience from their last drug approved in 3.5 years. Of course, the results need to look good etc.

Lori Ware said:
Like I said, my brain is not working at full throttle, but Dr. Wong said that the phase 1 will be done in the UK....

As for a phase on taking 3 years, well PTC 124 was about that long...maybe 2, but I think 3. I know the boys that were in the phase one has to wait YEARS to get the drug back (yes, they were taken off of it!) to get back on it. I KNOW at least 2 and I think longer (Jill Castle would know for sure!)...

cheryl cliff said:
I thought BioMarin was going on with phase I here in usa. A short while ago Summit went BK and I watched things with them to see what impact that would have on BioMarin's progress with Utrophin. I contacted BioMarin aprox 6 mos ago to find more and they responded by saying they are going ahead but their trial was a bit slower to start. I was reassured they were not devistated by Summit's financial status, were a few mos behind their original start date and going on ahead.
Thank you everyone for your replies. I now have a better idea of where the research stands for Utrophin upregulation. Ofelia, you made a good point about PTC being sort of a pioneer in the business and that may be why the trials took longer. I'm hoping that BioMarin's experience with orphan drugs will get this to market in a shorter time span, if everything goes well, especially since it doesn't look like my grandsons will be good candidates for exon skipping in the near future if at all. (They have a duplication of exons 8-11.)
You are very right Ofelia, PTC was a pioneer and that very well maybe why it won't take so long! Lets certainly hope so!!!!

Ofelia Marin said:
Phase I will be in the US. BioMarin is working on this product alone, no connection with the UK company anymore.

I do not know how long it was from Phase I to Phase II of PTC124 BUT we need to take into account that 124 was PTC's first product (they did not have any experience marketing any drug before!) and the 6-minute walk test was not even validated at that point. So one can say that PTC was a pioneer in the field. BioMarin has experience bringing drugs for orphan diseases to market in short time. I would definitely expect less than 3 years for Phase I. In fact BioMarin said (during a CC last year) that they try to replicate the experience from their last drug approved in 3.5 years. Of course, the results need to look good etc.

Lori Ware said:
Like I said, my brain is not working at full throttle, but Dr. Wong said that the phase 1 will be done in the UK....

As for a phase on taking 3 years, well PTC 124 was about that long...maybe 2, but I think 3. I know the boys that were in the phase one has to wait YEARS to get the drug back (yes, they were taken off of it!) to get back on it. I KNOW at least 2 and I think longer (Jill Castle would know for sure!)...

cheryl cliff said:
I thought BioMarin was going on with phase I here in usa. A short while ago Summit went BK and I watched things with them to see what impact that would have on BioMarin's progress with Utrophin. I contacted BioMarin aprox 6 mos ago to find more and they responded by saying they are going ahead but their trial was a bit slower to start. I was reassured they were not devistated by Summit's financial status, were a few mos behind their original start date and going on ahead.
We need to ask Pat F. how are things going on with Biomarin and with Project catalyst.

Reply to Discussion

RSS

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

Members

Events

© 2019   Created by PPMD.   Powered by

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