Orphan Drug Update: Phase 2 Study Shows Promise in Congenital and Childhood Onset Myotonic Dystrophy Type 1

MedicalResearch.com Interview with:

 Michael Snape, Ph.D., chief executive officer and chief scientific officer of AMO Pharma

Dr. Michael Snape

Michael Snape, Ph.D.
Chief executive officer
Chief scientific officer
AMO Pharma 

MedicalResearch.com: What is the background for this study? What are the main findings? 

Response: This is a further analysis of the Phase II Proof of Concept study that was described in March.  We have extended the findings from that study by performing further analyses of the data obtained and also commencing an analysis of the relationship between the levels of AMO-02 involved and the clinical response seen.

The main finding is a confirmation that our previously reported conclusions are supported.  The concordant trend analysis revealed a clear dose-response relationship that favored the 1000 mg over 400 mg dose for four of the 10 response variables differed in favor of 1000 mg over 400 mg dose on key clinician and caregiver measures, autism scores and grip strength. 

MedicalResearch.com: What should readers take away from your report?

Response: These data confirm and extend our previous report of a broad pattern of benefit for AMO-02 in this initial Proof of Concept study in this very rare disorder.

MedicalResearch.com: What recommendations do you have for future research as a result of this work?

Response: These data support an approach to analysis suitable for clinical research in rare, ultra-Orphan conditions where patient numbers are by definition small and it is important to maximize the use of information provided by each patient.  A concordant trends approach can help give a more comprehensive picture of potential benefit than, for example, relying solely on a traditional primary outcome measure approach

Nothing to disclose


7th International Conference on Neurology and Neuromuscular Diseases abstract: 

The utility of concordant trend analyses in a Phase 2 study in congenital and childhood onset myotonic dystrophy type 1: a case example

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Oct 28, 2018 @ 2:46 pm

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