ARM210 - Using ARM210 to Improve Muscle Strength & Function in Duchenne

 

Frequently Asked Questions (FAQs) about this research:

 

What stage is this research?

  • This research in Duchenne Muscular Dystrophy is in Phase 1 clinical stage, meaning a first time in human clinical safety study in adult healthy volunteers is underway.

 

Where is this research being done and who is funding this research?

  • This research is being done by ARMGO® Pharma, Inc.  The Muscular Dystrophy Association awarded the company $1 million to develop the compound, ARM210, for Duchenne.

 

What is the goal or purpose of this research?

  • ARMGO® has identified a new class of small Glossary Link molecule therapeutics (called Rycals®) that restore normal balance of calcium within muscle cells by  correcting the activity of a type of channel called the “ryanodine receptor calcium channel complex” (RyR).  In mice that lack dystrophin Rycal® ARM210 corrected a calcium leak occuring through the RyR and improved daily activity, strength and muscle force.  These studies help establish the rationale for conducting a clinical trial with this compound in Duchenne.

 

What is the current state of this research and what steps need to be completed before moving into a clinical trial?

  • Formal non-clinical toxicology studies required by the FDA have been completed. ARM210 was found to be safe and well tolerated. A first time in human Phase 1 clinical safety study in adult healthy volunteers began at the end of 2015 and is expected to complete this year.

 

What is your best estimate for the length of time it will take to move this research into clinical trials in Duchenne patients?

  • Following successful completion of a Phase 1 clinical safety study in adult healthy volunteers and discussions with clinical experts and regulators, an initial clinical study in Duchenne patients is planned to start in early 2018.  

 

Where would a clinical trial take place?

  • It is too early to know where a clinical trial for this research would be located.  Many complex factors go into determining the right location(s) for a clinical trial.

 

Who would be eligible to participate in a clinical trial?

  • Again, it is too early to know what the inclusion criteria would be for a future clinical trial.

 

Where can I learn more about this research?

  • You can learn more about ARM210 at ARMGO's website (www.armgo.com).
  • www.ClinicalTrials.gov will post all clinical trials once they are actively recruiting patients.                                                                           

 

(Updated 4/17/2017)