Akashi Therapeutics Provides Update on Three Duchenne Compounds

This weekend, Akashi Therapeutics announced they are working toward having three novel, complementary compounds in the clinic in 2017 with potential to treat all Duchenne patients independent of their specific genetic mutation.

This is promising news for a company that, as you know, halted the Duchenne work as an investigation took place into the death of a patient in their HT-100 trial. As the communication from Akashi CEO, Marc Blaustein explains below, a thorough examination of this tragedy uncovered that there was an unexpected drug interaction between HT-100 and another drug which resulted in very high HT-100 exposure levels, beyond the safe range. Precautions have been taken to ensure drug interactions do not happen again and the FDA has given the company the go-ahead to continue with this and two other compounds.


Akashi will move forward with compounds aimed at three well-defined targets: fibrosis, muscle building/maintenance, and cell necrosis (death).


PPMD has stayed in regular contact with the company during this time period, and looks forward to hosting a webinar with Marc and team in the coming weeks. Stay tuned for more details.


To ensure you are kept up to data about Akashi’s various trials, make sure you register or update your registration in DuchenneConnect today.

Click here to read the full release.

Read a letter to the Duchenne community from Akashi CEO, Marc Blaustein:

Dear Foundation Supporters,


You may have seen our press release (attached) from yesterday morning announcing our plans to have three compounds in the clinic in 2017. The release provides an overview of our 2017 plans, but for you we wanted to provide a few more details on our approach and strategy.


First, as always, Akashi is committed to developing a cocktail of therapies to turn Duchenne from an aggressive killer to a chronic, manageable condition. Experts universally agree that a combination of therapies will be needed to significantly delay disease progression, much like the standard of care for HIV and many cancers. We are working on the premise that no single “magic bullet” is on the near-term horizon. Helpfully, much is known about the pathology of Duchenne so there are a variety of targets to attack. We are aiming at three well-defined targets: fibrosis, muscle building/maintenance, and cell necrosis (death).


Fibrosis: As you know, HT-100 has been shown to prevent and even reduce fibrosis in animal models. HT-100 is also a potent anti-inflammatory, and promotes healthy muscle regeneration and utrophin upregulation.  Boys and young men with DMD who have been treated with HT-100 show an increase in strength, an extremely encouraging finding that we are committed to exploring further. Dosing of HT-100 has been suspended for the past year. During that time, we have conducted a thorough investigation into the death of a patient in our trial. Our exhaustive studies led us to conclude that there was an unexpected drug-drug interaction between HT-100 and another drug which resulted in very high HT-100 exposure levels, beyond the safe range.  By taking steps including using low doses and prohibiting concomitant administration of a drug that may interact with HT-100, we believe the risk of such high exposures will be greatly reduced.  HT-100 is a highly potent compound, and can be given at low doses with expectation of benefit. We are now in advanced discussions with the FDA with the goal of first resuming dosing and then planning for a new trial with more patients.


Muscle Building: Our SARM drug (selective androgen receptor modulator) has the potential to build muscle and increase lean muscle mass, thereby increasing strength and function. We have done all of the prep work necessary to conduct a short study in healthy adult volunteers in the UK. This “proof of concept” study will tell us if DT-200 increases lean muscle mass and function in humans.


Cell necrosis: Lack of dystrophin leads to a cascade of negative effects, one of which is abnormal influx of calcium into muscle cells. High levels of intracellular calcium lead to cell necrosis, or death.  Our third molecule, AT-300, has the potential to restore a more normal calcium balance. AT-300 is derived from the venom of a Chilean spider, and was discovered at the State University of New York at Buffalo. We have financed multiple preclinical studies on this compound, and based on favorable results we will proceed with IND enabling studies.


This comprehensive approach to treating Duchenne is an expensive proposition. We are currently talking with several investors and strategic partners who can help ensure the finances are in place to execute on this ambitious agenda.  This coming week I will be at the JP Morgan conference to further advance these discussions.

Thank you very much for enabling us to get this far. We are pretty awestruck by the incredibly committed families and foundations that have had the vision and the fundraising prowess to advance our multi-drug pipeline to this relatively mature point. As always, don’t hesitate to call or e-mail me with any questions or observations. Here’s to a productive 2017!


Warmest regards,

Marc, on behalf of the Akashi team


Marc B. Blaustein


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