The Global Revolution in Biotechnology and the Impact of Regenerative Medicine

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The Global Revolution in Biotechnology and the Impact of Regenerative Medicine

"This is the golden age of science."  That's what Mike Milken, chairman of the Milken Institute, told CNBC's Kelly Evans on Monday during an interview with Milken and National Institute of Health director Francis Collins at the 5th Annual Partnering for Cures convention at the Grand Hyatt in Manhattan. Francis Collins referenced healthcare as being in an evolutionary period and noted that steps need to be taken for the United States to maintain a world leadership position. "When you look at the rest of the world, they have read America's playbook and have seen how success happened and they are trying to become what we used to be," commented Collins. 

In our view, a cornerstone in a robust U.S. (and global) biotechnology ecosystem is regenerative medicine and many of the next generation treatments that cell-based therapeutics has to offer. The rapidly growing field as an adjunctive or front line treatment embodies a new wave of therapies to meet countless areas of great unmet medical need where legacy, small molecule-based drugs are showing limited impact, or facing extreme challenges. This impact is being recognized globally - a good example is Japan, which has embraced the potential of regenerative medicine and is actively seeking to establish itself as a global leader in the area. It has earmarked regenerative medicine specifically, announcing $3.2 billion in funding for programs to advance the area, using pluripotent stem cells and other approaches. 

In the annual "Meeting on the Mesa," a three-day convention of world leaders and investors in regenerative medicine held in La Hoya, California last month, Dr. Gil Van Bokkelen, Chief Executive Officer and Chairman of Athersys, Inc. (NASDAQ: ATHX) touched not only on the initiatives of Japan, and global potential of the field of regenerative medicine, but also the unique potential of Athersys'MultiStem(R) technology.   


Interested investors can watch the entire video interview on YouTube here:

Dr. Van Bokkelen noted that MultiStem is being developed as an "off the shelf" medicine, or in technical terms an "allogeneic" stem cell therapy that can be manufactured on a large scale, stored for years in frozen form, and administered without tissue matching or immune suppressive drugs. Athersys is developing the technology for several indication areas, including some that are recognized by analysts and pundits as having breakthrough potential. Current clinical programs include, ischemic stroke, acute myocardial infarction, preventing Graph versus Host Disease (GvHD) and treating Inflammatory Bowel Disease (IBD) in patients where other medicines have proven ineffective. This latter program is part of an ongoing international Phase II trial in partnership with Pfizer, Inc. (NYSE: PFE) for treatment-refractory inflammatory bowel disease, with results expected in early 2014.   

New treatments for a condition like IBD, focusing on patients where other therapies have proven ineffective, could validate the potential of regenerative medicines to reverse the course of an advanced disease, at a time when treatment expenses skyrocket and quality of life deteriorates dramatically. In fact, a hallmark feature of regenerative medicine is its apparent utility in addressing areas of substantial unmet medical need, where the cost burden is high.  Success from ongoing or planned MultiStem trials, especially for an area like ischemic stroke, could provide a powerful catalyst for Athersys shares.Top-line results from the IBD trial are expected in the first quarter of 2014, with results from the stroke clinical trial expected several months later. 

As noted by Dr. Van Bokkelen's presentation, MultiStem has great potential as a novel therapeutic approach for conditions where current standards of care are essentially non-existent or ineffective. One of the reasons for this is the multimodal activity of MultiStem, which addresses key shortcomings of traditional drugs or technologies that typically focus on a single mechanism of action.  He notes that cells are different because they are capable of promoting healing in several ways, such as by expressing factors that reduce inflammatory damage, protect and preserve tissue that is at risk following an injury, promote formation of new blood vessels in regions of inadequate blood supply, and by stimulating recovery in other ways.   

Recent deal activity also suggests growing acceptance of regenerative medicine technologies, including the Cytori Therapeutics, Inc. (NASDAQ: CYTX) partnership to commercialize technology in Australia and Asia, and the recent Mesoblast, Inc. (ASX: MSB) acquisition of the rights to the Osiris Therapeutics, Inc. (NASDAQ: OSIR) Prochymal franchise. With new regulatory initiatives in Japan and other countries poised to speed development options for companies focused on clinical development of new medicines, it seems that regenerative medicine is now beyond its flash point.  The tremendous potential is now being recognized in multiple areas around the world, including in some corners of Wall Street.

CNBC video:


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