Preclinical research of AT-300

Akashi’s Novel Modulator of Stretch-Activated Calcium Channels

Questions and Answers

What stage is this research?

This compound is in preclinical research, meaning it has not yet advanced to clinical trials.

What is the goal or purpose of this research?

The objectives of this preclinical research program were to identify the optimal dosing level and regimen for use in a future clinical study and provide essential proof of concept data of efficacy in Duchenne. The research compound, AT-300, is a novel modulator of stretch-activated calcium channels. It is intended to help restore normal levels of calcium in Duchenne skeletal and cardiac muscle. Abnormally high levels of calcium in Duchenne muscle contribute to loss of function and eventually to muscle cell death. AT-300 would be relevant for all boys and young men with Duchenne, regardless of mutation. The results of these studies were extremely successful, showing significant protection from muscle strength loss after exercise.

Akashi Therapeutics is conducting this research and has received Orphan Drug status for AT-300 for Duchenne.

Who is funding this research?

The drug has been developed for Duchenne by Akashi Therapeutics, Inc. At this point in the development of AT-300, Akashi is in discussions with several funding partners to further advance this novel treatment.

What steps need to be completed before moving into a clinical trial?

We need to explore long term efficacy of this drug and conduct a series of preclinical safety studies to establish preliminary safety before filing for an IND and conducting a study in humans, most likely in healthy volunteers.

What is your best estimate of when AT-300 could enter a clinical trial?

The initial clinical study, a safety study that would most likely be conducted in healthy volunteers,and could begin as early as 2019.

Where can I learn more about this research?

You can learn more at www.akashirx.com

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