Preclinical research of ARM210

Using ARM210 to Improve Muscle Strength and Function in Duchenne Muscular Dystrophy

Questions and Answers

What stage is this research?

This research in Duchenne has completed the Phase 1 clinical stage, meaning an initial study in Duchenne is planned for mid-2020.

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 treatment of Duchenne.

What is the goal or purpose of this research?

ARMGO® has identified a new class of small 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 occurring through the RyR and improved daily activity, strength, muscle force and muscle damage. These studies help establish the rationale for conducting a clinical trial with this compound in Duchenne. This molecule can help both the heart and skeletal muscle.

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

All of the necessary toxicology and Phase 1 work necessary to move into a clinical trial in patients have been completed. A Phase 1b in adult patients with mutations in RYR1 (RYR1- related myopathy) will begin shortly.

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

The expectation is that an initial clinical study in Duchenne patients is planned to start in mid-2020

Where would a clinical trial take place?

Discussions are still ongoing for possible clinical locations.

Who would be eligible to participate in a clinical trial?

The initial clinical trial will be in non-ambulatory boys ages 12 and up to determine if heart and lung functions will be improved.

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