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



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.





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Thank you Jon Tinsley.    Having your input in this discussion is extremely wonderful and a refreshing break from the norm Typically we frightened parents discuss, with limited information, the positive or negative aspects of something in the pipeline.  God forbid SMT C100 fails to work for our children.  But even if that does happen, please know how much we appreciate the effort you, Summit and all the others are putting in to kill duchenne. 

As long as there are formulas in the cooker we can retain hope. 



I just wanted to respond to an earlier comment about the value of Summit.  We are actually only a small UK biotechnology company with limited financial resources and the business has a current market valuation of approximately £7.5 million or $12 million.  In addition, as we are a publically traded company, we do have someone dedicated to looking after enquiries from existing or potential investors and they can be contacted by email at investors@summitplc.com.

Keith Van Houten said:

Buying a stock on an exchange from another owner has little effect on a company's ability to finance its operations.  If this was a new stock offering - yes - but not buying it on the stock exchange.  The money you pay goes to the owner of the shares you're buying - not to the company.


If you had a very large sum of money, you could buy enough shares to get a seat on the board of directors and try to influence company direction.  Or, buy a controlling interest.  The company's market cap is about USD$1.6 billion, so you're talking hundreds of millions...


Bob Getler said:


For discussional sake, let's say someone has a rich Uncle or a family member has a large sum of money in their 401K's.   I see Summit PLC is a publicly traded company (http://www.summitplc.com/investors1.aspx).  


If a person is not inclined to donate cash but would rather trade a stock in the direction of finding a cure/treatment for Duchenne, how would the purchase of Summit PLC stock directly or indirectly help the chance of seeing SMT C1100 through trials to general availability?


 Thanks Jon for the clarifications. I still have 2 questions

(1) Whether SMTC1100 will help Becker boys also.

(2) You had explained that in older boys, utrophin 'tap' gets turned off and this get replaced by dystrophin ( which our sons are lacking). SMTC1100 will try to interfere will this turning off process and try to turn this 'utrophin tap' on.Now whether SMTC1100 will help older boys also(where utrophin tap is already turned off). I mean,in younger boys where 'utrophin tap' is in active state, it may be easier to just get this utrophin tap remain on.But will it help older boys also.Or we will come to know of this in human clinical trials.

Same Summit PLC referenced earlier?  http://www.summitplc.com/shareprice-information.aspx


168 million+ shares outstanding at 5.8p / share = 975M pounds? = $1.5 B USD   Maybe 5.8p doesn't mean 5.8 pounds?  Not sure.  Never been involved with shares on foreign markets.


As Cheryl said, the conversation here is refreshing, Jon.  Your explanation of utrophin in the previous post is great...

5.8p/share is 5.8 pence per share?  which is 0.05 pounds/share = 8.4 mil pounds.
Yep, that must be it.  That makes it close to what Jon said for market cap.  Now I've learned about utrophin and british currency in this thread....

I will throw in my thanks to all who engaged in this discussion but particularly Jon T.


While I did not hear what wanted to hear - that Summit's "next study" is actually a Phase 1 safety study, while I expected a Phase 2 study - I too learned several important things along the way.


Probably the most important lesson is that at least for 2011, there is not likely to be utrophin-based treatment from Summit that will be in a Phase 2 study in the US. Best bet for utrophin-based treatment to reach Phase 2 in this time frame appears to be Project Catalyst.


any updates with regard funding for the trial?

Can Steroids really regulate Utrophin?

Did they test Utrophin in boys on steroids?

Does anyone know when Utrophin upregulation trial is expected to start?

Ilack the information about this trial


In May of this year you commented about a potential "clinical candidate" for utrophin from Project Catalyst by the end of this year (exact comment is below)

However, PTC states that the start of a Phase 1 trial is at least 15 months away as of today. Can you please explain what "clinical candidate" means to a very frustrated parent who sees nearly no movement whatsoever on utrophin, despite it's promise to the entire DMD population?


Pat Furlong said:

Dear Friends,   I wanted to respond to the questions about SMT C1100.    We have a proposal from Jon Tinsley currently under review.   As you know, Biomarin terminated the study some time ago.   Since that time, considerable work has been done on the compound and it appears that the issue is one of formulation.   We have gotten several opinions from PharmD on re-formulation of the compound as well.   We are anxious to help move SMT C1100 forward.  Jon Tinsley has sent us a draft Q/A about SMT C1100 which will be posted in the next day or so.   Like you, we are anxious to see compounds that upregulate utrophin move into trial - in the US and Europe - and will do whatever we can to help.    

In addition, Project Catalyst has a compound that upregulates Utrophin moving forward and we expect to announce a clinical candidate before the end of this year.   With luck, the SMT C1100 compound and the catalyst compound may be synergistic.  

There was also a question about earmarking funds. We are happy to earmark funds for projects/programs we are considering funding.  Please let me know if you have questions.  Sincerely, Pat

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