Other Countries in Asia
Neuralstem is also involved in partnerships with Asian companies, with deals initially focused on research. While Neuralstem last year established a wholly owned subsidiary in China, it has yet to come to terms with commercial or regulatory collaborators.
By year-end the firm plans to start a clinical trial in China focused on transplantation of cells into the brain to treat stroke. The trial would take place at Beijing’s BaYi Brain Hospital, which has been working with Neuralstem to prepare a clinical protocol for treatment of motor deficits due to ischemic stroke.
In Japan the company came to terms with the wholly owned subsidiary of Sumitomo, Summit Pharmaceuticals, to market development and licensing rights for NSI-189, Neuralstem’s lead small molecule neurogenic compound. It is currently in an FDA-approved Phase I trial for major depression. The company has said it intends to take NSI-189 through Phase II trials before seeking a partner for worldwide rights.
Neuralstem has also entered into collaborations in Taiwan, including one with China Medical University & Hospital, to advance development of its human spinal cord neural stem cell therapies for amyotrophic lateral sclerosis. It is also working with the hospital to commence a clinical trial focused on treating stroke.
Additionally, in India, the company is planning a clinical trial for later this year to assess the ability of its cell therapy to treat spinal cord injury. The Indian market is so large that companies like Neuralstem need to find a technological partner first to ensure access to the best neurosurgeons it can find, according to Richard Garr, CEO. “We’re happy to do the proof of principle human studies ourselves before we look for a commercial and regulatory partner.”
Neuralstem notes that it has done most of its proof of principle collaborations with American universities such as the University of California, San Diego and the University of Michigan. “We haven’t gone overseas because we can’t do it here in the U.S.,” Garr remarked. “We went overseas because we believe those are their own independent markets.” Garr points out that commercialization efforts for each country are independent of each other.
“The other reason” to move into Asian countries, he added, “is because if you don’t, someone else will. And then you end up in a fight trying to protect it.”
Neuralstem broke into Asia in 2008, when Korean conglomerate CJ CheilJedang (CJ) bought $2.5 million worth of Neuralstem stock. CJ has an exclusive option to Neuralstem’s spinal cord cell products for five Asian nations: South Korea, Vietnam, Indonesia, Malaysia, and Singapore.
CJ would have the responsibility to take the products through regulatory approvals and commercialization in their markets. Garr expects CJ to decide what its first clinical trial will be and where later this year.
Another U.S. stem cell company that has teamed up with a Korean company is Advanced Cell Technology (ACT). In 2008, ACT joined with CHA Biotech to form a joint venture aimed at developing ACT’s hESC-based hemangioblast (HG) platform for the treatment of blood and cardiovascular diseases.
On July 21, the companies announced that their venture, Stem Cell & Regenerative Medicine International (SCRMI), exclusively licensed the rights to the HG program to ACT for the U.S. and Canada and to CHA Biotech for Korea and Japan.
“The partnership itself had research scientists working on trying to get things ready for starting human trials. And the way the deal works, ACT has hired substantially all the scientists that were working in the joint venture”—10 SCRMI employees in all, Gary Rabin, ACT’s interim chairman and CEO, told GEN.
Rabin said the first IND will be filed for using the HG platform to generate renewable sources of transfusable blood platelets. The platelets could unlock a significant opportunity for ACT, namely in the military wound-care market.
Last year, Robert Lanza, M.D., ACT’s CSO, and Kwang-Soo Kim, Ph.D., of Harvard University’s McLean Hospital and the CHA Stem Cell Institute, won a $1.9 million NIH director’s opportunity award to explore the potential of induced pluripotent stem cells (iPSCs) as a source of universal red blood cells and platelets for transfusion.
Another Asian market ACT is keeping an eye on is China. In March the company reported that China’s State Intellectual Property Office allowed its patent application to provide broad intellectual property protection for the manufacturing and pharmaceutical preparations of retinal pigment epithelial (RPE) cells from hESCs for degenerative retinal disease.
ACT has initiated a Phase I study with the RPE therapy in the U.S. Additionally, ACT has launched human trials for Stargardt macular dystrophy and advanced dry age-related macular degeneration. Data from those trials is expected to be published this fall, after the first three patients are tested in both trials.