We don't hear any news about SMT C 100 (Utrophin booster) which suppose to start in the 2H of 2013!!!!!

Any updates,please?!

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Is this trial fully enrolled?

Is anyone funding the trial design for phase 2, so we don't waste time if this phase is successful?

Kieth, I'm not certain re this phase, but as it's only 12 boys and UK based, I suspect it is or is close to being enrolled.

I understand the phase 2 is designed but will be further informed by the phase 1b in terms of dosing.  They also have a biomarker programme running alongside, so hopefully will have a measure over and above the 6MWT. They have most but not all the funding needed for the phase 2 , including contributions from a number of the DMD charities.  It's a lot less expensive to run than some of the Exon skipping trials, both because it's much cheaper to produce but also as they can treat all boys, rather than a sub set, as such recruitment is a much easier process. They will do trials in the US, Europe and Australia (I think).  I take the below from a note produced on the company:

"The Phase 2 study will likely involve about 40 DMD boys over age seven with at least 350 6MWT at baseline. Patients will be randomized 2:1 versus placebo for 48 weeks. The six-minute walk test will be the primary efficacy endpoint. Muscle biopsy taken at baseline and the end of the planned 48-week treatment period will be used to evaluate biomarkers including utrophin expression. The study will be powered to show a 30 meter treatment benefit and if it hits this hurdle with statistical significance, it could be used for approval"  IF it works, it could be approved before the end of 2015.

Hi Steven,

I am impressed with your knowledge of Summit and it`s approach. You are correct the drug is not in injection form which has to be a plus especially for the boys concerned.

As far as I am aware this trial P1b is fully funded Phase 2 on the other hand is a different matter.

Summit are in discussions with a couple of charities though but no decisions one way or the other have been made.

I believe that enough of the drug is being produced both for P2 and a P3 if it is required.

Australia as yet to be confirmed though if a P3 is required it will definitely be included.

I believe the biomarker programme is what will set the results apart from previous trials by other companies.

I am hopeful given the drugs orphan and accelerated (?) status that it could be approved before the end of 2015 IF it is successful though that is purely personal conjecture.

In the recent RNS "enrolled" means passed all the tests, has agreed to join the trial and been accepted by both the hospital and the company. "Dosed" means the patient has had the first dose.

The fantastic thing about this drug is IF it is successful that is applicable to 100% of boys and that this drug is the first to move toward P2...the recent agreement with Kay Davies and the University of Oxford for the pipeline of utrophin uploading drugs have in Kay`s opinion even more potential.

I hope that this first generation is successful and that the following generations build on that success.



Paul or Steven...can you tell me where your information regarding a possible approval could come as early as 2015?

Chris, can't answer for Paul (who I don't know as yet) but think I should qualify that, as "in my humble opinion", although a couple of stock broker notes do mention it as a real possibility - IF - during phase 2 it shows to be effective!  Might be wishful thinking, but you need a little of that if you are to hope a cure will be found!

Company timeline below (taken from Wedbush presentation): 

Safety and dose finding study in H2 2013, results H1 2014
– Phase 2 patient study expected to start in 2014, results projected H2 2015
– Novel biomarker program underway, includes collaboration with Children’s National Medical
Center in Washington DC
– Scale-up and manufacture of clinical drug product on-going: completion Q1 2014
– Long-term regulatory safety studies to be conducted: completion H2 2014

I think caution should be taken with giving potential dates for approval although hopefully this will fall under the ema for approval which does seem more lenient. Also the recent FDA bomb shell questioning whether producing Dystrophin is the way forward for potential treatments won't or at least shouldn't matter with this drug. What gives me hope with this drug is that it is switching on an already functional gene apposed to trying to repair a fault in a gene, and scientists have been switching parts of the body on and off for years. fingers crossed this is one of the most promising treatments out there and I believe summit are already in pre clinical for a second generation utrophin drug.

Dear all,

I have some news that may interest you from Summit. The preliminary results of their P1b trial are discussed below. Released this morning in the U.K.




  • Safe and well tolerated at all doses tested in the study
  • Reduction in CK levels, an enzyme associated with muscle damage
  • Next patient clinical trial expected to start in Q4 2014

As I understand it the reduction in CK levels was hoped for but not expected and very encouraging as an indication that it works and does so quickly as it was only a 10 day trial. The absorption levels however were variable, mainly due to many of the boys being on low or no fat diets (C1100 is oral so it effects the rate of absorption).

Worth noting again that if  C1100 works, it will do so for all boys, regardless of deletion. 

Oh my god!!!!!great news!!!!

Thanks Steven for posting that.

This is the link to the full RNS.


The brrmedia link is also well worth looking at/listening to as it fleshes out the RNS and the points Steven posted.



This is excellent news. Is it confirmed that the P2 trial would also include sites in US? I thought only P3 of SMT C1100 trials were going to be in US. Can someone please confirm?

And, as Utrophin does not localize nNOS in the cell, does anyone know what the supplement would be that will help with that. Of course this is assuming everything goes well with Utrophin upregulation and it proves to be disease modifying.


As far as I am aware there will be P2 trials in the US. If/when there are P3 trials Australia (not the U.S.) will then be added to the list of countries taking part in the trials.

I am afraid your other question is too technical for me. I suggest you try contacting the company on this point.




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