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Massive Leap!

By January 8, 2016 October 30th, 2016 Tracy's Blog

Today Akashi announced big news: a major partner has committed to fund the final steps to take HT-100 over the goal line to boys with DMD. The press release features some fancy-sounding business jargon like global partnership, royalties, and commercialization rights. While we’re excited about all that, the best part is what it all adds up to for boys with DMD.

By now you probably know that a few years ago Charley’s Fund spun out a biotech company called Akashi Therapeutics to develop a cocktail of treatments for Duchenne. Frustrated that certain promising therapies were not being developed, we brought the compounds under one roof and began to “de-risk” them. We hired a drug development team and raised enough money to conduct the preclinical and early clinical testing necessary to better understand whether these therapies could really improve patients’ lives. Our plan was to more urgently and efficiently move these therapies through this risky phase where drugs so often get stuck, and build the evidence necessary to get a partner with more funding to finance the later, much more expensive phases of drug development.

And so with this in mind, over the last several years we – with the crucial help of other patient organizations – have been funding the development of Akashi’s drugs – most notably today HT-100. Certainly, being fully responsible for the funding and execution of a drug development program is a major undertaking, but to us the advantages to our mission to save boys with DMD are worth it. Driving this program ourselves, we’ve been uniquely poised to be lean and aggressive, and take critical strategic risks that prioritize benefit to our boys.

The HT-100 program has been very successful, with early clinical data indicating that the drug is safe and increases muscle strength. This success meant it was time to ramp up the clinical testing, manufacturing and other activities necessary to get a new medicine approved and available to patients.

Enter the Grunenthal Group, a European family-owned pharmaceutical company worth billions of dollars. They have focused on developing pain medications, but they are expanding their business to include rare disease treatments. In the partnership with Akashi announced today, Grunenthal plans to invest more than $100 million into the development and commercialization of HT-100. What’s more, they understand that it will take a cocktail of therapies to effectively treat Duchenne, and they plan to develop additional medicines that will work to complement HT-100’s beneficial effects.

Akashi is still 100% owned by Charley’s Fund, the Nash Avery Foundation, and the management team who co-founded the company. Yet we now have the funding, firepower and technical expertise of a large company to push HT-100 over the goal line.

Congratulations are in order to everyone who played a role in making this happen: the stellar Akashi management team, our nonprofit partners who helped fund the program, our supporters who contributed donations that enabled us to pull off such an amazing feat, and – most important –– the boys who have been poked and prodded and imaged and questioned as part of the clinical trials. All the hard work and sacrifice has brought us one huge step closer to safe and effective treatments for all.