PPMD supported the development of the HT-100 program from early on, including a $500,000 investment via our GIFTED program for the clinical trial. We are excited for the expansion of the clinical trial and thrilled for the relationship with Grunenthal. This incredible partnership enables Akashi to continue to be managed and led by parents and we are all hopeful HT-100 will be part of the cocktail of combined therapies that will achieve our highest priority--to stop progression, to end Duchenne.
Learn more by reading Akashi's press release:
Akashi Therapeutics Establishes International Partnership with Grünenthal Group on HT-100 for DMD
- Companies to collaborate on advancement of a novel small-molecule therapeutic for Duchenne muscular dystrophy -
CAMBRIDGE, Mass. and Aachen, Germany, January 8, 2016 – Akashi Therapeutics, Inc. a clinical stage biopharmaceutical company developing treatments for Duchenne muscular dystrophy (DMD), today announces a $100 million partnership with Grünenthal Group for a global drug development program for the investigational therapeutic HT-100, an orally available small molecule drug candidate in Phase 1b/2a for the reduction of fibrosis and inflammation, and promotion of healthy muscle fiber regeneration in DMD. HT-100 has been granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA).
“This agreement represents a critical turning point for the company and offers powerful new hope for DMD patients and their families,” said Marc Blaustein, CEO of Akashi Therapeutics, a company that to date has been funded entirely by DMD patient foundations. “We are thrilled to find a partner who shares our commitment to this patient community, and look forward to accessing Grünenthal’s world-class scientific, clinical, regulatory and commercial capabilities to accelerate development and broad global availability of HT-100.”
Under the terms of the agreement, Grünenthal will make upfront and milestone payments to Akashi. In addition, the company will assume all post-Phase 2 global development costs through commercialization of an approved product. Grünenthal gains commercialization rights in Europe and Latin America, while Akashi retains rights for the U.S. and other markets. Akashi will receive royalties on net sales. In total, Grünenthal plans to commit more than $100 million to the partnership and will receive royalties on U.S. net sales in exchange for funding development of Akashi’s U.S. commercial infrastructure.
“We are very excited about this unique collaboration with Akashi. At Grünenthal, we are highly committed to innovation and have been focusing on bringing innovative therapies to patients with high medical need. We are very motivated to use all our strength for the development of HT-100 together with our partners from Akashi and the patient groups supporting them,” said Dr. Klaus-Dieter Langner, Chief Scientific Officer of Grünenthal.
The agreement also lays the groundwork to expand the collaboration to include Akashi’s other pipeline compounds for DMD and to explore additional indications for HT-100, which may also have activity in other fibrotic diseases such as scleroderma and idiopathic pulmonary fibrosis.
HT-100 (delayed-release halofuginone) is an orally available, small molecule drug candidate designed to reduce fibrosis and inflammation and promote healthy muscle fiber regeneration in DMD patients. HT-100 has been granted orphan designation for DMD in both the U.S. and E.U., and fast track designation in the U.S. A phase 1b/2a clinical program is currently underway at five hospitals in the U.S. For more information, please contact Akashi Therapeutics (www.akashirx.com).
HT-100 development is generously supported by patient advocacy organizations. A list of these organizations can be found at http://akashirx.com/financial-supporters/.
About Duchenne Muscular Dystrophy (DMD)
Duchenne muscular dystrophy (DMD) is an X-linked recessive, inheritable disease that affects approximately 1 in 3,600 boys. DMD results in muscle degeneration and premature death. Symptoms usually become visible in early childhood: progressive proximal muscle weakness of the legs and pelvis associated with loss of muscle mass is observed first, and this weakness spreads to other parts of the body. As the disease progresses, muscle tissue is replaced by fat and fibrotic tissue (fibrosis). Untreated, most patients are wheelchair dependent by age 10. Due to progressive deterioration of muscle, patients lose ambulation, then arm function, and ultimately experience respiratory and/or cardiac failure. While life expectancy varies, patients typically survive until late in the second or the third decade.
About Akashi Therapeutics
Akashi Therapeutics is a clinical stage biopharmaceutical company whose mission is to develop treatments for Duchenne muscular dystrophy. Akashi was founded by leading patient organizations Charley’s Fund and Nash Avery Foundation in collaboration with biotechnology industry veterans to impact a central problem in rare diseases: rapid therapy development. Akashi is developing a pipeline of therapies with the goal of transforming Duchenne from a 100% fatal, aggressive muscle-wasting disease to a chronic, manageable condition. For more information, please visit www.akashirx.com.
The Grünenthal Group is an independent, family-owned, international research-based pharmaceutical group headquartered in Aachen, Germany. Grünenthal is an entrepreneurial specialist delivering true benefits to patients. By sustainably investing in research and development above the industrial average, Grünenthal is committing to innovation in order to treat unmet medical needs and bring value-adding products to markets. Grünenthal GmbH is a fully integrated research & development company with a long track record of bringing innovative pain treatments and state-of-the-art technologies to patients.
Altogether, the Grünenthal Group is present in 32 countries with affiliates in Europe, Australia, Latin America and the US. Grünenthal products are sold in more than 155 countries and approx. 5,300 employees are working for the Grünenthal Group worldwide. In 2014, Grünenthal achieved revenues of € 1.154 bn. More information: www.grunenthal.com.