TACT(TREAT-NMD Advisory Committee for Therapeutics) to consider 2 DMD and 1 CMD related drugs for second round of review

Cristina Csimma (Virdante Pharmaceuticals Inc), as TACT chair, is leading this exciting initiative with the support of the TACT core, nominated committee members and the TREAT-NMD secretariat. TREAT-NMD is confident that the breadth and calibre of the international TACT experts will ensure the quality of the output.

The aim of TACT is to provide transparent and consistent guidance and advice to the neuromuscular community, in an educational and directional context, on the readiness of drugs and/or therapeutic targets for trials in neuromuscular diseases.

The remit of the committee is to evaluate the therapeutic potential of drugs based on preclinical data, thereby assisting TREAT-NMD in prioritising clinical trials to be run via the network. TACT advice will be helpful for preparing funding applications and investigational drug applications, and serve as an unbiased appraisal to be published for the wider neuromuscular community. We hope that this process would be endorsed by the major funders.

In addition to the expert analysis of preclinical data TACT will also address issues of drug formulation, bioavailability and toxicology as well as possible regulatory requirements and marketing considerations. In close collaboration with the TREAT-NMD clinical trials coordination centre in Freiburg TACT is also dedicated to providing information on rational and economical trial design.  The ultimate goal of TACT is to help pave the pathway for successful drug registration by providing professional and independent advice.

The TACT appraisal process of potential therapeutics for clinical trials will take a maximum of 6 months. TACT will consider more than one drug at a time and therefore will have panels of experts, selected from the TACT members below, reviewing the drugs concurrently, with the same chair and core committee for continuity.

Applications for the second round of reviews which will take place on 5th & 6th June 2010 have now been accepted.

The applications are:

  1. Laminin-111 for Duchenne Muscular Dystrophy
  2. N-Acetyl cysteine for a Congenital Muscle Disorder
  3. P-188 for Duchenne Muscular Dystrophy



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What is P-188?
It's a "poloxymer"--a sort of relatively inert substance that may be able to physically seal tears in the muscle cell membrane (like a bicycle patch). It's being developed by a company called Phrixus under the name "Carmaseal" for cardiomyopathy in Duchenne.

Actually a better analogy is the bicycle "goo" that you inject into the tire that forms its own seal...
Hi Sharon,
Is it same as the molecular Band-Aid which was discovered by University of Minnesota and was recently in news. I guess Dr. Joseph Metzger was involved into that paper.
But I think that poloxymer(by Phrixus) is in news for quite sometime. I think even PPMD and ActionDuchenne had earlier funded this also.
Are both same? Is this getting done by University of Minnesota and Phrixus together? Any idea on what stage it is in currently?
Yes, it's all the same stuff. The company has completed the preliminary work to do a clinical trial in cardiomyopathy in DMD and is trying to raise funds for the trial. PPMD has a call with them later this week--we've had some concerns about the clinical protocol, bu they are working hard to address the issues. After the Ataluren experience I guess it's really clear why we want to be satisfied that the clinical protocol is the best it can be before launching into a multi-million dollar trial. That's not meant as a criticism of PTC because I think the Ataluren study was really well-designed, it's just very hard to do these studies and we don't know as much as we should about heart problems in DMD (which makes it hard to plan a trial).

RAKTIM SINGH said:
Hi Sharon,
Is it same as the molecular Band-Aid which was discovered by University of Minnesota and was recently in news. I guess Dr. Joseph Metzger was involved into that paper.
But I think that poloxymer(by Phrixus) is in news for quite sometime. I think even PPMD and ActionDuchenne had earlier funded this also.
Are both same? Is this getting done by University of Minnesota and Phrixus together? Any idea on what stage it is in currently?
Thanks Sharon. I am sure we will get the update after PPMD call with them.

Raktim
Thank you Raktim and Sharon for helping me understand this. So much to wrap one's head around with this and all the other options out there.

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