DT-200

A Selective Androgen Receptor Modulator to Improve Muscle Strength & Function in Duchenne

Questions and Answers

What stage is this research?

The DT-200 development program has completed three Phase 1 trials in 78 healthy adult volunteers. These studies showed that the SARM is safe and well tolerated for up to 14 days of treatment. Testing in Duchenne has not yet begun.

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

DT-200 is wholly owned by Akashi Therapeutics. Akashi is currently seeking funding for the next development step, a 4-week Proof of Concept (POC) clinical trial.

What is the goal or purpose of this research?

DT-200 is a selective androgen receptor modulator (SARM). Selective androgen receptor modulators (SARMs) have been developed to mimic the muscle building effects of androgens (testosterone), without their undesirable side effects. Akashi hopes that the more precise action of this drug will confer better long term safety and tolerability in both adult and pediatric muscle diseases compared to androgens. DT-200 is effective in multiple animal models, including mice that lack dystrophin. Importantly, compared to other SARMs in clinical development, DT-200 shows significantly greater preference for skeletal muscle. Accordingly, Akashi plans to develop DT-200 with the objective of improving muscle strength and function in pediatric neuromuscular diseases such as Duchenne and Spinal Muscle Atrophy (SMA). SARMs may also have utility in Charcot Marie Tooth disease (CMT) and Facioscapulohumeral Muscular Dystrophy (FSHD), stress urinary incontinence (SUI) and hip fracture.

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

Three Phase 1 studies with DT-200 were successfully completed in Belgium and Germany, having confirmed the product is safe and well tolerated at the once daily oral dose of 0.5 mg. Akashi met with the UK Regulatory Agency (MHRA) for scientific advice and input into the design of a Proof of Concept clinical trial and has received ethics approval for a 4-week POC trial in the UK in healthy adult volunteers. The main objective of the POC trial is first to evaluate DT-200’s ability to increase mass, strength and function of healthy muscle.

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

If the POC trial in healthy adult subjects is positive, Akashi anticipates initiation of clinical trials in Duchenne muscular dystrophy could begin within 12 months from the conclusion of the POC trial.

Where would a clinical trial take place?

Phase 2 trials would be initiated in both the US and Europe.

Who would be eligible to participate in a clinical trial?

It is too early to know what the inclusion criteria would be for a future clinical trial. This drug could be beneficial for all Duchenne boys regardless of specific mutation.

Where can I learn more about this research?

You can learn more about DT-200 (and other Akashi initiatives) by consulting the Akashi website at: http://akashirx.com

www.ClinicalTrials.gov will post all DT-200 clinical trials once they are actively recruiting subjects.

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