New York, August 15, 2012 – Amorcyte, a company of NeoStem, Inc. (NYSE MKT:NBS) (“NeoStem” or the “Company”), a rapidly emerging market leader in the fast growing cell therapy market, today announced that it received on August 9, 2012 approval to continue its PreSERVE AMI Phase 2 clinical trial following its first interim data and safety review by the Data Safety Monitoring Board (DSMB). The PreSERVE trial is a Phase 2, randomized, placebo controlled, double-blind study expected to include 160 patients at more than 40 clinical sites. The trial’s product candidate, AMR-001, is designed to prevent major adverse cardiac events following acute myocardial infarction (AMI). Patient enrollment for the PreSERVE trial began in January 2012 and the Company anticipates completing enrollment in 2013 with six months initial data readout near the end of 2013.
“We are pleased that, similar to our Phase 1 trial, the first external review of our Phase 2 trial data confirms that there are no safety signals that would preclude the trial from continuing as planned,” said Andrew L. Pecora, M.D. FACP CPE, Chief Medical Officer of NeoStem. “The PreSERVE AMI study to date indicates that multiple National Study sites are capable of acquiring the necessary volume of bone marrow to create the AMR-001 product five to seven days after an AMI in a safe and practical manner, and once created the product can be delivered and administered without a safety signal.”
NeoStem management believes that cell therapy is a disruptive technology in the $50 billion worldwide regenerative medicine market. Many key opinion leaders in the scientific, medical and investment communities consider AMR-001 to be best in class. Peak annual worldwide sales of AMR-001 for this indication could exceed $1 billion based upon a conservative market penetration of its qualified target patient population. AMR-001 is protected by two issued and multiple pending U.S. patents with corresponding patent coverage in selected markets around the world. The Amorcyte AMR-001 product development program also extends to congestive heart failure (CHF). The Company is preparing to launch its CHF Phase 1 clinical trials in early 2013. The worldwide CHF patient population is estimated to be four times larger than that of AMI.
About NeoStem, Inc.
NeoStem, Inc. (“we,” “NeoStem” or the “Company”) continues to develop and build on its core capabilities in cell therapy to capitalize on the paradigm shift that we see occurring in medicine. In particular, we anticipate that cell therapy will have a large role in the fight against chronic disease and in lessening the economic burden that these diseases pose to modern society. Our January 2011 acquisition of Progenitor Cell Therapy, LLC (“PCT”) provides NeoStem with a foundation in both manufacturing and regulatory affairs expertise. We believe this expertise, coupled with our existing research capabilities and collaborations, will allow us to achieve our mission of becoming a premier cell therapy company. Our PCT subsidiary’s manufacturing base is one of the few current Good Manufacturing Practices (“cGMP”) facilities available for contracting in the burgeoning cell therapy industry. Amorcyte, LLC (“Amorcyte”), which we acquired in October 2011, is developing a cell therapy for the treatment of cardiovascular disease. Amorcyte’s lead compound, AMR-001, represents NeoStem’s most clinically advanced therapeutic and Amorcyte is enrolling patients for a Phase 2 trial to investigate AMR-001’s efficacy in preserving heart function after a heart attack. We also expect to begin a Phase 1 clinical trial in 2013 to investigate AMR-001’s utility in arresting the progression of congestive heart failure and the associated comorbidities of that disease. Athelos Corporation (“Athelos”), which is approximately 80%-owned by our subsidiary, PCT, is engaged in collaboration with Becton-Dickinson that is exploring the earlier stage clinical development of a T-cell therapy for autoimmune conditions. In addition, our pre-clinical assets include our VSELTM Technology platform as well as our MSC (mesenchymal stem cells) product candidate for regenerative medicine.
Forward-Looking Statements for NeoStem, Inc.
This press release 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 press release, and involve certain risks and uncertainties. Forward-looking statements include statements herein with respect to the successful execution of the Company’s business strategy, including with respect to the Company’s or its partners’ successful development of AMR-001 and other cell therapeutics, the size of the market for such products, its competitive position in such markets, the Company’s ability to successfully penetrate such markets and the market for its CDMO business, and the efficacy of protection from its patent portfolio, as well as the future of the cell therapeutics industry in general, including the rate at which such industry may grow. Forward looking statements also include statements with respect to satisfying all conditions to closing the disposition of Erye, including receipt of all necessary regulatory approvals in the PRC. The Company’s actual results could differ materially from those anticipated in these forward- looking statements as a result of various factors, including but not limited to (i) the Company’s ability to manage its business despite operating losses and cash outflows, (ii) its ability to obtain sufficient capital or strategic business arrangement to fund its operations, including the clinical trials for AMR-001, (iii) successful results of the Company’s clinical trials of AMR-001 and other cellular therapeutic products that may be pursued, (iv) demand for and market acceptance of AMR-001 or other cell therapies if clinical trials are successful and the Company is permitted to market such products, (v) establishment of a large global market for cellular-based products, (vi) the impact of competitive products and pricing, (vii) the impact of future scientific and medical developments, (viii) the Company’s ability to obtain appropriate governmental licenses and approvals and, in general, future actions of regulatory bodies, including the FDA and foreign counterparts, (ix) reimbursement and rebate policies of government agencies and private payers, (x) the Company’s ability to protect its intellectual property; (xi) the company’s ability to successfully divest its interest in Erye, .and (xii) matters described under the “Risk Factors” in the Company’s Annual Report on Form 10-K filed with the Securities and Exchange Commission on March 20, 2012 and in the Company’s other periodic filings with the Securities and Exchange Commission, all of which are available on its website. The Company does not undertake to update its forward-looking statements. The Company’s further development is highly dependent on future medical and research developments and market acceptance, which is outside its control.