Firms will receive development funding, and upon approval, 20,000 doses will be sold for a total of $200 million.
The DoD commissioned Osiris Therapeutics and Genzyme in a deal worth $224.7 million to develop and stockpile Prochymal, an adult stem cell therapy, for the repair of gastrointestinal injury resulting from radiation exposure.
In July 2007, Osiris and Genzyme inked an agreement to jointly develop Osiris’ Prochymal. The DoD decision marks the first award under this partnership.
Under the terms of the contract, the DoD will fund development of Prochymal for acute radiation syndrome (ARS) in two stages. The firms will initially receive $4.2 million in 2008. Upon FDA approval in this indication, the contract provides for the purchase of up to 20,000 doses at $10,000 per dose in four 5,000 dose increments.
Prochymal is a highly purified formulation of mesenchymal stem cells (MSCs), isolated from the bone marrow of healthy adult donors. Studies have shown that the stem cells in Prochymal are able to facilitate tissue repair through a number of mechanisms, according to Osiris. Specifically, MSCs are able to down-regulate severe inflammation, which is responsible for much of the tissue destruction that occurs as a result of radiation exposure. MSCs also work at the cellular level to rebuild damaged tissue through the coordinated release of tissue-specific growth factors.
Prochymal is designed to offer certain advantages. Osiris says that since it is being developed as a postexposure treatment, Prochymal eliminates the need for immediate administration in a predefined treatment window. The company also points out that since Prochymal has advanced to Phase III trials in other indications, it has a proven stability profile that permits long-term storage. Additionally, Osiris confirms that it has capacity at multiple manufacturing sites to mass produce large quantities.
“Prochymal represents a breakthrough in countermeasure development for ARS,” according to Thomas J. MacVittie, Ph.D., a member of Osiris’ nuclear and radiological countermeasure advisory board. “Prochymal has demonstrated therapeutic utility in humans repairing many of the major organ systems affected by radiation injury. Where most approaches only target a single component of ARS, Prochymal has the potential to address the entire syndrome including both acute and delayed effects in multiple organ systems.”