What can we do NOW to get next trials for SMT C1100 (utrophin upgregulator) in the US in the future?

http://www.ox.ac.uk/media/news_stories/2011/110505_1.html

 

Considering that BioMarin has scrapped their similar program, and that Dr. Davies is at Oxford, I'm very concerned that the next phase of trials for SMT C1100 will happen in Europe or elsewhere outside the U.S.

 

In my opinion when this happens the FDA uses it as an excuse to take "wait and see" approach and for those of us with boys in their teens, that is of great concern.

 

My son in particular will likely not benefit from exon-skipping or other mutation-specific treatments. Utrophin-based treatments appear to be the most directly applicable to him.

 

What can I, as a parent, do NOW to help ensure that he can participate in the first human trials? We just don't want to wait and see any more.

 

-David

 

 

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Is the utrophin upregulation compound identified in Project Catalyst something new that would require FDA approval, or is it something like an already approved drug for another condition or supplement?

Jon,

 

I realize you may hear this frequently but I think it is always worth stating again...

 

I want to convey my deepest, deepest appreciation for Summit's work and persistence in trying to develop a treatment for Duchenne.  

 

My son Kyle, age 6, is a great, happy little boy and I pray he will remain happy and survive this situation.  While I was never hugely religious, since the diagnosis in Jun 2010  I kiss Kyle every night as he sleep and then literally get on my knees at the foot of his bed and pray for all the boys and god's help for the scientists to gain the wisdom to identify a cure.  I have added Summit by name to my prayers.

 

Jon, there is a family in Elizabethtown, Kentucky and families all over the world praying for your success. 

 

Thanks for all you do.

 

Bob Getler

 

www.CureKyle.com

Ofelia,

I realize I'm probably jumping the gun on discussing regulatory issues here. I'm trying - grasping really - to identify something I can do NOW to grease the wheels to bring SMT C1100 trials (whatever phase) to the US as soon as possible.

 

I suppose I just don't want to accept the snails pace of drug development that seems to be the norm. At the very least I've learned I can earmark my PPMD donations for specific projects which is something I will surely do.

 

Hi Jon, it is hoped that clinical trials will also take place in Europe?

 

Jon Tinsley said:

Hi David, I can comment specifically on Summit’s proposed drug, SMT C1100. Because our drug is a classic small molecule chemical, there should be no unexpected hurdles with regulatory authorities.  The development of small molecule drugs is a well-trodden path for the FDA/EMA that has produced many marketed drugs over many decades.

Summit understands all the steps required to satisfy the regulatory authorities the product is safe and well tolerated.

Hope this helps, Jon

Yes, everything moves much slower than any of us want to accept. There might be many reasons for that some more credible than others.

David said:

Ofelia,

I realize I'm probably jumping the gun on discussing regulatory issues here. I'm trying - grasping really - to identify something I can do NOW to grease the wheels to bring SMT C1100 trials (whatever phase) to the US as soon as possible.

 

I suppose I just don't want to accept the snails pace of drug development that seems to be the norm. At the very least I've learned I can earmark my PPMD donations for specific projects which is something I will surely do.

 

May I suggest you have a look at Summit's DMD pages which includes frequently asked questions regarding SMT C1100.

Jon,

 

In the content you suggested, appears the following quote, with a particular emphasis added by me:

 

Summit believes that the formulation of SMT C1100 used in this trial was a major factor in the variability in exposure levels observed and our plan is to commence new clinical studies of SMT C1100 as soon as possible using alternative formulations of the compound, all of which would be suitable for use in infants

 

Does this imply that Summit believes this treatment is unsuitable for older boys?

 

Hi David, apologies for using ambiguous language. What we were trying to convey is that actually the proposed formulation, a liquid, would be appropriate for all ages including young children. We have updated our web information to make sure this is clear.

Hi Jon thanks for being so active on this site at a time when we are truly tuned into Summit.Your prompt replies and clarification is much appreciated,

 

thanks Clare

Jon,

I'm confused by content on the FAQ page at http://www.summitplc.com/FAQs%20for%20DMD.aspx. I admit that I'm no expert on the drug approval process, but

 

In point #6, it states that previous BioMarin studies are still valid. This would include the fact that the drug was well tolerated with no side effects. In other words, Phase 1 is complete.

 

6. Are the good results previously reported by Summit still valid?
Yes.  The SMT C1100 data recently published in conjunction with Dame Prof Kay Davies in the peer reviewed scientific journal PLoS ONE is the most comprehensive set of data made publically available for any potential DMD treatment.

 

But, then in point #8, it states that 12 months AFTER funding, Summit start over in Phase 1. This adds multiple years before a possible Phase 2. Why would Summit not take advantage of work already performed by BioMarin?

 

8. When could new clinical trials begin?
Once the necessary financial funding is secured, the clinic ready formulation could be evaluated in a healthy volunteer clinical trial within twelve months. If the Phase I trial in healthy volunteers proves successful, SMT C1100 could be evaluated in DMD patients within a further 12 months.

 

I echo Clare's appreciation for all your hard work. But you can imagine that the last thing we parents want to hear is that a promising treatment will be redoing work already completed.

 

-David

Hi David, further clarification of your observations.

Point #6 refers solely to Summit’s results in that the previous work is still valid i.e. all the efficacy work done in the mdx mouse and the preclinical work leading up to phase I trials.

If you look at Point #5 this alludes to the fact that the formulation used in the BioMarin Phase I wasn’t successful in generating consistent levels of SMT C1100 in the blood and therefore another Phase I trial has to be run using a different formulation. A new Phase I trial has to be run in order to show that this new formulation is safe.

Point #8, says that within 12 months of funding the Phase I trial could be evaluated i.e. the Phase I trial completed and the data reviewed and judged whether successful enough to move in patients. Within a further 12 months (months 13 to 24) SMT C1100 could be evaluated in DMD patients i.e. the Phase IIa trial in DMD boys completed.

I certainly can appreciate frustration in the process however I can assure you that all the proposed work is necessary because previous Phase I work was incomplete.

Jon,

 

We appreciate you communicating with us.   

 

Can you give me some concept of scale in terms of the amount of funding needed by a company such as Summit to conduct a Phase 1 trial?   What size funding gap are you trying to fill?

 

Also, my understanding is Phase I involves healthy volunteers.   Whent the time is right, would it be helpful for parents and family members to volunteer for Phase I or would we be considered poor test subjects due to our interest in a positive outcome?

 

Kindest regards,

 

Bob Getler

Elizabethtown, KY

Dad of Kyle, age 6

www.CureKyle.com

 

p.s.  shop www.ParentProjectMD.org/Amazon to raise money for PPMD!


Jon Tinsley said:

Hi David, further clarification of your observations.

Point #6 refers solely to Summit’s results in that the previous work is still valid i.e. all the efficacy work done in the mdx mouse and the preclinical work leading up to phase I trials.

If you look at Point #5 this alludes to the fact that the formulation used in the BioMarin Phase I wasn’t successful in generating consistent levels of SMT C1100 in the blood and therefore another Phase I trial has to be run using a different formulation. A new Phase I trial has to be run in order to show that this new formulation is safe.

Point #8, says that within 12 months of funding the Phase I trial could be evaluated i.e. the Phase I trial completed and the data reviewed and judged whether successful enough to move in patients. Within a further 12 months (months 13 to 24) SMT C1100 could be evaluated in DMD patients i.e. the Phase IIa trial in DMD boys completed.

I certainly can appreciate frustration in the process however I can assure you that all the proposed work is necessary because previous Phase I work was incomplete.

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