Posted on March 19th, 2015
by David Mazzo
Dear NeoStem Shareholders,
As you might imagine, the first two months of my tenure as the Chief Executive Officer of NeoStem have been hectic, stimulating and enlightening. I can assure you that I am as excited as ever about our prospects to bring paradigm-changing new therapies to bear on diseases with high unmet medical need. I remain confident that our objective to significantly enhance shareholder value will become a reality in the not too distant future. Allow me to share some of the reasons why I remain so bullish on our company.
NBS20, our most advanced clinical program, focused on developing an autologous cell therapy to treat Stage III recurrent and Stage IV metastatic melanoma, is poised to randomize the first patient in Phase 3 in the near future. In addition to the compelling Phase 2 data on which the Phase 3 trial is based, this program enjoys Orphan Drug and Fast Track designations from the FDA and the protocol has been granted a Special Protocol Assessment (SPA) allowing for a single successful Phase 3 trial for registration. To add to our enthusiasm for this program, we note that the platform technology on which NBS20 is based is likely applicable to many other solid tumor types. Given that we acquired this asset less than one year ago and, in the ensuing months, much of the external focus on our company has been on our ischemic repair Phase 2 program, we believe that the market has not yet integrated the full potential of this mature immunotherapy asset into the value of our company. We base our opinion on a comparison of the market capitalization of NeoStem with other companies with much higher market capitalizations also working in the immuno-oncology therapeutic area (e.g., Lion Biotechnologies and Northwest Biotherapeutics).
Following NBS20 in terms of clinical advancement is NBS10, our ischemic repair program in Phase 2 clinical development (the PreSERVE trial). NBS10 is the lead program for our autologous cell therapy platform based on CD34 cells which we believe have the ability to stimulate the formation of new blood vessels. We reported what we perceive as promising interim data last fall and, as many of you know, added to the data coming from the trial with new and supplemental information presented at the American College of Cardiology’s Annual Scientific Sessions and Expo on Sunday, March 15. Based on the results that have been reported to date, we see the PreSERVE study as a real success as a Phase 2 study. Not only has it given us important information regarding tolerance, administration, dose, endpoints and design for future clinical development, but it has provided what we see as compelling signals supporting a conclusion of positive treatment effects. While we will continue to follow patients and complete the study as required by the protocol, we believe that we have learned sufficient information from the trial to formulate next steps for development and attract relevant partners into more serious discussions.
Finally, for NBS03D, our T regulatory cell immunomodulation program, we recently submitted our Phase 2 Trutina study protocol to the FDA and are now able to proceed with the study as soon as we so choose. This study is designed to evaluate NBS03D in adolescents with type 1 diabetes mellitus (T1DM). We look forward to starting this Phase 2 trial later this year or in early 2016. In the meantime, we are exploring means, including partnering, by which we can accelerate the initiation of the trial. In keeping with the potential broad applicability of our other platforms, we expect this immunomodulation platform to be applicable to other autoimmune disorders which cumulatively affect over 50 million people in the United States alone.
As you can see, there is much about which to be excited at NeoStem. We feel that the combination of our rich yet focused therapeutics pipeline and an externally recognized in-house center of excellence for cell therapy process development and manufacturing has created an organization with unique capabilities for accelerated and efficient product development. We further believe that this profile gives us a significant competitive advantage which should result, ultimately, in an enhanced ability to create value for shareholders. We look forward to making this objective a reality. In the meantime, allow me to express the gratitude of our entire company to our loyal shareholders for their continued support. Our company is in the midst of an evolution and we hope you will join us as we work to make NeoStem a market leader in personalized medicines.
To get more details on our programs and information on the company, please review the most recent investor presentation on the neostem.com homepage along with our most recently filed form 10K. Also, consider viewing the webcast presentation I will make at the BioCentury 22nd Annual Future Leaders in the Biotech Industry conference this Friday, March 20 at 1:30 PM ET (http://www.neostem.com/investors/investor-events/).
With best regards,
David J. Mazzo, Ph.D.
Chief Executive Officer
Forward Looking Statements
This blog contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements reflect management's current expectations, as of the date of this blog, and involve certain risks and uncertainties. Forward-looking statements include statements herein with respect to the successful execution of the Company's business strategy, the Company's ability to develop and grow its business, the successful development of cellular therapies with respect to the Company's research and development and clinical evaluation efforts in connection with the Company's Immuno-oncology Program, Ischemic Repair Program, Immune Modulation Program and other cell therapies, the future of the regenerative medicine industry and the role of stem cells and cellular therapy in that industry, and the performance and planned expansion of the Company's wholly-owned subsidiary and its center of excellence for cell therapy process development, engineering and manufacturing, PCT. The Company's further development is highly dependent on future medical and research developments and market acceptance, which is outside of its control. The Company's actual results could differ materially from those anticipated in these forward-looking statements as a result of various factors. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of the Company in general, see the factors described under the heading, “Item 1A. Risk Factors" in the Company's Annual Report on Form 10-K filed with the SEC on March 2, 2015 and those described in the Company's other periodic filings with the SEC. The Company undertakes no obligation to update or revise any forward-looking statements.
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