Athersys' proprietary cell therapy product, MultiStem, is currently being evaluated in a Phase II clinical study for ischemic stroke in the United States and Europe, and Athersys has begun preparations for clinical development in Japan, including engagement with the Japanese Health Authority.
"By entering into an agreement with Chugai, Athersys will ensure it’s able to market MultiStem – once it has passed Phase II US clinical trials – in Japan for 7 years while gathering data on product efficacy,” said Jane Andrews, senior consultant at Frost & Sullivan in Texas.
Human stem cells
Multistem is a therapy product derived from human stem cells that can promote tissue repair and healing. Once the cells are obtained they are expanded, tested for function and subsequently frozen. The frozen cells can be shipped to a physician’s offices where they can be stored for patient treatment, says Andrews.
As part of the collaboration, Chugai will be responsible for the development and commercialization of MultiStem for ischemic stroke in Japan, and Athersys will have responsibility for product supply. Under the agreement, Athersys will receive an up-front cash payment of $10m from Chugai and will receive additional payments as the program is further advanced.
“In 2013, the Japanese parliament enacted new legislation for conditional approval of new cell therapy products. Cell therapy technologies that have been shown to be safe, by passing phase 2 of clinical trials, can be sold in Japan for up to 7 years while the later stage trials (III-IV) are conducted,” explained Andrews.
“During this time, the company must collect and file clinical data to support the product. If these data prove efficacy, the company can file for the equivalent of a “Biologics License Application” (BLA).” The product can remain on the market if approved.
“Because once the product passes Phase II clinical trials, Athersys and Chugai can market the product in Japan while Phases III and IV are being conducted,” Andrews continues. “Advantages to marketing the product in Japan after completion of Phase II is that it generates revenue while the later stage clinical trials are being conducted. This also increases patient participation in the trials.”
More than two million people experience a stroke per year in the US, Europe and Japan, combined. Ischemic stroke is caused by an obstruction in the blood vessels that supply blood to the brain, and accounts for 87% of all strokes. It represents a priority disease area in Japan, given the high healthcare burden of the condition and the expected increase in incidence associated with the country’s aging population.
Current therapeutic options for ischemic stroke victims are limited. The only therapy, a clot dissolving agent, must be administered within several hours of stroke, meaning only a small percentage of stroke victims are treated.
“The stem cell therapy industry represents a new emerging market. Because of this, many of the new stem cell therapies will be somewhat controversial until there is definitive evidence of efficacy, as indicated by the completion of clinical trial Phases III and IV. Once MultiStem has completed these trials it will be increasingly accepted as a treatment to minimize the effects of ischemic stroke,” Andrews commented.