CLEVELAND—This month saw Athersys Inc. gain some traction in the Japanese market with regard to stem cells and regenerative medicine, with the announcement that the company had been granted patents from the Japan Patent Office for several inventions involving its proprietary cell therapy technology.
One of those patents covers non-embryonic, multipotent stem cells, such as MultiStem, and applies to all therapeutic applications, while another covers the use of such stem cell therapies for the treatment of immune dysfunctions, such as for graft-versus-host disease (GvHD) and for inflammatory diseases and autoimmune disorders—for example, inflammatory bowel disease (IBD). A third patent covers the treatment of brain injuries, such as stroke and traumatic brain injury, with multipotent stem cells.
“These patents will provide the company important protection for its cell therapy products in Japan and their use in a number of significant disease areas, including stroke, IBD, GvHD and others,” explained William Lehmann, president and chief operating officer at Athersys. “Additionally, we are encouraged by regulatory events in Japan, which have created an accelerated development path for stem cell therapies. Recognizing these developments, Japan's sizable pharmaceutical market, and the expected growth in demand for more effective treatments of age- and lifestyle-related diseases and conditions, we consider Japan a high-priority market for the development of MultiStem cell therapy product candidates.”
The Japanese parliament, known as the Diet, enacted the new legislation in mid-November 2013, revising the existing Pharmaceutical Affairs Law to define new medical products that contain stem cells as regenerative medicine products. This allows the Japanese government to grant conditional approval for stem cell and regenerative medicine products if clinical trials suggest they are safe, even if their efficacy has not yet been established.
As MarketWatch noted some days after the Athersys announcement, the new legislation is intended to promote innovation and “could provide a clear channel to rapid commercialization that will benefit industry-leading companies like Cellular Dynamics International Inc., Athersys, Inc. and Cytori Therapeutics, Inc.”
Australian regenerative medicine company Mesoblast, shortly after the legislation passed the Diet, noted: “As a result of the new legislation, a pathway now exists in Japan for Mesoblast to commercialize its mesenchymal lineage stem and precursor cell products within a significantly shorter timeframe. Mesoblast intends to establish strategic partnerships for its suite of products to facilitate earlier Japanese regulatory approvals and commercial launches.”
Mesoblast's first strategic partnership in Japan is with JCR Pharmaceuticals Co. Ltd. for its culture-expanded mesenchymal stem cell (MSC) product to treat steroid-refractory GvHD. JCR reportedly intended to file for Japanese marketing approval of its MSC product JR-031 before the end of the first quarter of this year, which would make it potentially “the first allogeneic or ‘off-the-shelf’ cell-based product approved in Japan.”
The legislation creates a new, faster pathway for cell therapy product approval, noted Athersys in its patent announcement this month, and it “offers the potential to enable more rapid entry into the Japanese market.”
“Japan is showing tremendous international leadership in an area that it recognizes has the potential to effectively address areas of serious unmet medical need. The new framework is intended to benefit patients through the rapid development and approval of innovative and cost-effective stem cell therapies,” said Gil Van Bokkelen, chairman and CEO of Athersys. "We are actively evaluating the potential for accelerated development of MultiStem cells in a number of therapeutic areas in Japan, and will seek to establish strategic collaborations to assist in the rapid development of products that will help Japanese patients.”
Cellular Dynamics International Inc.
Cytori Therapeutics Inc.
JCR Pharmaceuticals Co. Ltd.