To DMD Patients and Their Families:
On January 8th, 2016 we announced a partnership that will enhance our capability to quickly and efficiently develop new treatments for Duchenne. We are thrilled to welcome our new partner, The Grünenthal Group, to the fight against DMD.
We are beyond pleased that our new partners agree that Duchenne is a disease that can be treated, and that HT-100 will be a key part of the ultimate therapeutic cocktail. This deal is excellent for the Duchenne community not only because the full funding needed to develop and commercialize HT-100 is now secured.
Beyond our lead drug, we are confident that the partnership will have a profound impact on DMD. One of the primary reasons Grünenthal selected Duchenne is the need for a multi-product approach, and therefore the ability to build a franchise of DMD products. Grünenthal intends to review Akashi’s other assets with an eye toward licensing those as well for their territories if the review is favorable and we reach agreement on terms.
Grünenthal’s leadership has been highly impressed by the dedication of the patient community to Akashi’s lead program. A main reason we are excited about working with them is their enthusiasm for closely collaborating with the patient community. This milestone is a huge step toward fulfilling our company mission of developing a cocktail of medications to turn Duchenne from an aggressive killer to a chronic, manageable condition. Thank you for being a part of it, and congratulations!
FAQs about the Partnership with The Grünenthal Group
If the Grünenthal Group is such a great pharma partner, how come I’ve never heard of them?
• Grünenthal is a family-owned, multi-billion dollar German pharmaceutical company. The firm spends upwards of $250 million/year on research to discover and develop new medicines. The company has a large presence in Europe and Latin America, but a smaller one in the U.S. They recently made a strategic decision to move into rare diseases, and Duchenne is the first rare disease they have chosen to impact.
Is HT-100 in danger of being deprioritized?
• No. Akashi is still owned by patients and maintains its unwavering commitment to DMD. We carefully selected our partner for their commitment to DMD, and the agreement clearly defines Akashi’s role in governance and decision-making.
Why is this business announcement good news for kids with Duchenne?
• Our arrangement with Grünenthal ensures that all the funds needed to develop and commercialize HT-100 are fully committed, up to $100 million. In addition to cash, the company has significant technical resources that will ensure continued aggressive development of HT-100. What’s more, Grünenthal’s business model is to develop multiple treatments for the same disease. They intend to review and potentially license Akashi’s other assets, and are also considering complementary treatments being developed by other companies.
What’s the status of the clinical trial?
• In the clinic, we are full steam ahead with the HT-100 trial. We’re expanding the phase 2a study to include additional patients and more sites. At the same time, we are actively planning a phase 3 study to begin early next year. Over the next few months, we plan to fill out our team so we can get DT-200 into the clinic this year as well.