PPMD’s research strategy has long had three distinct facets—to develop treatments Better, Faster, and Now. In the past, I have talked about how we have teamed up with TREAT-NMD to use its panel of experts on Duchenne and drug development to review projects with the goal of putting the best possible, most highly optimized drug candidates into clinical testing (view my talk last year at FasterCures). I have also talked about our efforts to prioritize data on drugs that are approved and available now for repurposing in Duchenne (RADD, Eplerenone, Tadalafil, and Tamoxifen).

What about faster?

Yesterday, PPMD sat down with large and small companies in the Duchenne space to better understand their needs to conduct clinical trials for drug development. This meeting follows on the heels of a $300,000 commitment by PPMD to the Duchenne Superhighway project (in collaboration with DART Therapeutics) to strengthen the clinical trial infrastructure in the Duchenne space so that trials can be conducted faster and more efficiently. As phase III studies for Cialis, Ataluren, and eteplirsen are either launched or in preparation, and numerous phase I/II trials are underway or set to follow, we need to be able to reduce the burden to families for participating in these trials and improve the ability of companies to identify and pair appropriate participants with the right study sites.

We extend a special thanks to Catabasis, Halo, Genzyme, GSK, Eli Lilly, Pfizer, Prosensa, Sarepta, and Shire for sharing their time and clinical operations teams to identify mutual challenges and help us brainstorm solutions that can be nurtured and supported in collaboration with the broader community. The discussion was fruitful and reflected a genuine interest on the part of the for-profit world to work together to increase the chances of approval for all drugs in development. A detailed white paper of the findings from that meeting will be circulated, first to meeting participants, and then made available to other groups so that this information can be widely shared and used by all. We are also exploring the potential for a Webinar on this topic to update the whole community.


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Comment by Sharon Hesterlee on December 2, 2013 at 3:59pm

Hi Amit--that was interim data.  He is still finishing up the work.  We also funded Dr. Dominic Wells to work with Dr. Ruegg and duplicate the work in his lab. 



Comment by amit gupta on December 2, 2013 at 3:34pm

Shron, correct me if I am wrong, but I thought Dr Ruegg presented preclinical report back in April 2013 at the MDA? What do you think is taking so long to present any other report if the preclinical study was complete prior to April?

Comment by Sharon Hesterlee on December 2, 2013 at 1:59pm

Hi Amit--I think they're ability to get a trial up and running will depend on having the funding secure and we are still waiting for the final report from the preclinical study.  We'll need to analyze those results to decide if a human trial is warranted.  So, I think 2014 is a reasonable goal for a trial, but probably not before the second quarter of the year.  It's sort of counter-intuitive, but one way of doing things faster is to make sure that you have your i's dotted and t's crossed before embarking on a trial--if you go down the wrong road with a human trial and have to start over then you will ultimately slow things down.  We are working on it! Some things still take as long as they take--we are focusing on dealing with the things that take time and don't add value like having to get multiple IRB approvals for the same protocol.   Sharon

Comment by amit gupta on December 2, 2013 at 12:52pm

Thanks Sharon. For Tamoxifen - the goal is to have tamoxifen in a clinical trial with Duchenne patients starting in early 2014.

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