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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Hi Moein,

None to date I`m afraid. The P1b trial is expected to start sometime in the next two months over a period of 7 weeks.

The only link I can provide is the below. SMT C 1100 is number 10 of 10 and is not recruiting as of yet.

http://www.ucl.ac.uk/ich/research-ich/dubowitz/clinical_trials

As you probably know the trials are taking place in the U.K. and will involve up to 12 boys.

Best regards,

Paul

Moein,

Summit released an RNS this morning.

I hope it proves interesting reading.

SUMMIT RECEIVES REGULATORY APPROVAL TO START PHASE 1B CLINICAL TRIAL OF SMT C1100 IN DMD PATIENTS

Oxford, UK, 1 November 2013 - Summit (AIM: SUMM), a drug discovery and development company advancing therapies for Duchenne Muscular Dystrophy ('DMD') and C. difficile infection, announces that its Phase 1b Clinical Trial Application for SMT C1100 has received approval from the UK Medicines and Healthcare products Regulatory Agency ('MHRA') and the Ethics Review Committee. SMT C1100 is a small molecule utrophin modulator that has the potential to treat all patients with DMD, regardless of the underlying genetic fault.

"Securing regulatory approval for the first in patient Phase 1b clinical trial has achieved another important milestone in the development of utrophin modulator, SMT C1100, for DMD," commented Glyn Edwards, Chief Executive Officer. "We believe that utrophin modulation is a novel disease-modifying approach for all boys with DMD and this Phase 1b trial forms an integral part of our wider clinical plans towards establishing SMT C1100 as a viable treatment for this devastating condition."

The Phase 1b trial will be a dose-escalating, open-label study and will be conducted in a total of 12 paediatric patients with DMD, aged between 5 and 11 years. It will evaluate the safety and tolerability of SMT C1100, and will measure blood concentration levels of the drug as Summit aims to confirm the dose to be used in a subsequent patient proof of concept efficacy trial. The Phase 1b trial will be conducted at up to four NHS hospitals located in the UK and patient recruitment is expected to start shortly.

The Chief Investigator for the trial, Professor Francesco Muntoni, Paediatric Neurologist at Great Ormond Street Hospital and Director of the Dubowitz Neuromuscular Centreadded, "Utrophin is a promising approach for the treatment of all DMD patients, regardless of their genetic mutation. It also has the potential to be complementary to other therapeutics approaches in clinical development and the start of the first patient trial of SMT C1100 is an important moment for the whole DMD community."

Further details about the clinical trial will be made available via www.clinicaltrials.gov and www.clinicaltrialsregister.eu.

Best Regards,

Paul

Thank you Paul for updates very much

Moein,

A new presentation available on the summit homepage

http://www.summitplc.com/userfiles/file/20131109_Action%20Duchenne-...

I am going to try and find a recording of the presentation and if I do will post the link here.

Regards,

Paul


Hi Paul,

Thank you very much for updates.


paul roberts said:

Moein,

A new presentation available on the summit homepage

http://www.summitplc.com/userfiles/file/20131109_Action%20Duchenne-...

I am going to try and find a recording of the presentation and if I do will post the link here.

Regards,

Paul

Moein,

You`re welcome.

I contacted Action Duchenne and have been told they hope to have the presentations on their website in a months time after getting permission to make them available from the people involved.

Once up I will post a link.

Regards,

Paul

p.s. in the meantime if you have any questions for Summit please e-mail them at dmd@summitplc.com Hopefully they will be able to answer any you ask.

This is probably the most direct and fastest way to get information regarding SMTC 1100.

Moein,

You might find this interesting though of course things are a way off.

http://www.brrmedia.co.uk/event/preview/02qehvfdan/118623?popup=true

Best regards,

Paul

Moein,

Here is the latest news from Summit. :^)

"SMT C1100 is a promising treatment that has the potential to improve the life of all patients with DMD, irrespective of the underlying dystrophin fault," commented Dr David Roblin, Summit's Chief Medical Officer.  "There is an urgent need to develop effective medicines for DMD patients and this study forms an important step in our clinical plans towards establishing SMT C1100 and utrophin modulation as a viable treatment that could slow or even stop the progression of this devastating condition."

About the Phase 1b Clinical Trial
The Phase 1b trial is a dose-escalating, open-label study that is being conducted in paediatric patients with DMD.  The primary endpoint of the trial is the evaluation of the safety and tolerability of SMT C1100. The study will also measure blood plasma concentration levels of SMT C1100 to determine the dose to be used in a subsequent Phase 2 proof of concept efficacy trial.  

The trial is enrolling 12 patients aged between 5 and 11 years, divided equally into three dose cohorts.  Each cohort will receive daily oral doses of SMT C1100 for a total of ten days with a review taking place after each cohort has completed dosing.  The trial is being conducted at up to four NHS hospitals in the UK with the Chief Investigator being Professor Francesco Muntoni at Great Ormond Street Hospital, London.

Regards,

Paul

First Paragraph of press release:

 

SUMMIT ANNOUNCES FIRST PATIENT DOSED IN A PHASE 1B CLINICAL TRIAL OF SMT C1100 FOR TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY

Oxford, UK, 9 December 2013 - Summit (AIM: SUMM), a drug discovery and development company advancing therapies for Duchenne Muscular Dystrophy ('DMD') and C. difficile infection, announces that the first DMD patient has been enrolled and dosed in a Phase 1b clinical trial of the oral, small molecule utrophin modulator SMT C1100.  

 

Andrew,

Do think they will release an RNS for each subsequent patient dosed? I haven`t yet but I will ask what they mean by dosed...do you think this means completed or just the first injection?

Regards,

Paul

HI, thought I would reply to the above. The phase 1b trial - as I understand it, is primarily a safety/ tolerability and dose trial. It's already been proven safe in humans and to have shown Utrophin uptake in the blood, but this is the first test in DMD boys and is a short trial to help inform the phase 2 (starting in 6 months or so). Not sure if they will announce if and when each cohort is dosed.  The drug is an oral drug (not an injection) and is relatively easy and cheap to produce.  The hope is that it will, when in full phase 2 trial, demonstrate not only utrophin uptake but the effect of this in helping slow or stop the disease completely. Dame Kay Davis of Oxford has devoted her working life to this area and says she 'won't retire till a cure is found'. If it works (and it's an IF for now) it will not be gene specific (so will be available to ALL boys regardless of mutation). It also seems to work on all muscles, including the heart and lungs, which is key.  

Phase 2 will be multi-centre (including in the States).  Wishing you all and your families the best and hope, one way or another, a cure is within sight!

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