NEW YORK, Nov. 21, 2014 (GLOBE NEWSWIRE) -- NeoStem, Inc. (Nasdaq:NBS), a biopharmaceutical company developing novel cell based therapeutics, announced on November 17th initial positive data from its 161 patient Phase 2 PreSERVE AMI (or acute myocardial infarction) clinical trial.
NeoStem is sharing additional information to clarify certain facts regarding the Phase 2 PreSERVE trial as "we are extremely disappointed, in the face of exciting results, that shareholder value is being diminished by pervasive inaccurate or incomplete statements about the study," said Dr. Robin Smith, Chairman and CEO of NeoStem.
By way of background, the goal of a phase 2 trial is to continue to build evidence of the safety and feasibility of the product candidate and to gather evidence of bioactivity and potential efficacy. Ultimately, the hope for a phase 2 study is that it yields information that can guide the design of the next phase study and NeoStem believes this has been achieved.
The original phase 2 primary endpoints were an imaging endpoint that was designed to provide evidence of changes in blood flow and a safety endpoint that was limited to the bone marrow harvest and infusion period.
Prior to data lock, NeoStem elected to revise its primary safety endpoint to include a comprehensive assessment of safety including all adverse events, serious adverse events and MACE. This decision was made after consultation with the Food and Drug Administration ("FDA") and based on:
1) Published FDA guidance regarding the preferred endpoints for approval of cell therapies for acute myocardial (e.g. mortality, hospitalizations);
2) Emerging scientific literature showing a dose-dependent benefit of cell therapies in terms of reduced major adverse cardiac events (MACE).
Accordingly, by cover letter dated July 14, 2014 NeoStem filed with the FDA an amendment of its protocol entitled "A Prospective Randomized Double Blinded Placebo Controlled Phase II Trial of Intra-coronary Infusion of AMR-001, a Bone Marrow Derived Autologous CD34+ selected Cell Product, in Patients with Acute Myocardial Infarction" (v.9.0).
The letter references (i) the telephone conversation on July 1, 2014 between Company personnel and the FDA regarding the protocol and statistical analysis plan ("SAP"); (ii) the email communication between Company personnel and the FDA on July 1, 2014 to seek FDA concurrence on the changes to the protocol and the SAP, and (iii) the telephone conversation between Company personnel and the FDA on July 8, 2014. The letter reads verbatim and in pertinent part:
The key changes to the protocol and SAP include:
- The order of endpoints was changed, with MACE moved from a secondary endpoint to the primary safety endpoint. It replaces the assessment that was previously focused solely on risks of the harvest and infusion to this outcome that we believe provides a broader and more useful view of overall safety. In the revised protocol and SAP, this measure is listed both as primary safety and as a secondary efficacy endpoint. The data regarding the safety of bone marrow harvest and infusion will be included in this analysis.
- Previously, we proposed to conduct the primary analyses based on data through each patient's Month 6 visit, which is the time when the primary imaging outcome is assessed. However, doing so meant that we would potentially report on clinical effects using only a fraction of the available data. Therefore, we now propose to analyze the clinical effects of this therapy in all patients through the time of the last patient's Month 6 visit. This will markedly increase the observation period and therefore provide more power to draw inferences and conclusions about the clinical impact of this therapy, important in planning phase 3. With the submission we will include the Clinical Endpoint Committee Charter.
- While the safety analyses continue to focus on the ITT population, we changed the efficacy population of highest interest to the Per Protocol population, defined as those patients who received full dose of the investigational product and underwent SPECT imaging both at baseline and Month 6. With the goal of the study to evaluate the bioactivity of the therapy to affect myocardial perfusion, we felt that this population would be most critical to making this determination. Analyses of the ITT and mITT populations will also be performed.
- The MACE endpoint previously included cardiovascular death, heart failure hospitalizations and reinfarctions. Based on the literature suggesting potential impact on revascularization rates, we changed the MACE endpoint to include all coronary revascularizations.
Again, the Company's decision to expand the primary endpoints was done prior to the Contract Research Organization (CRO), which holds all data related to the study, locking the database and performing the initial analysis.
About NeoStem's Ischemic Repair Program
NeoStem is developing therapies to address ischemia through its Ischemic Repair Program (the Program), using CD34 cell technology. Ischemia occurs when the supply of oxygenated blood in the body is restricted. The Company's therapeutics seek to reverse this restriction through the development and formation of new blood vessels. The Program's lead product candidate is NBS10, a chemotactic hematopoietic stem cell product comprised of autologous bone marrow derived CD34/CXCR4 cells selected to treat damaged heart muscle following AMI (or heart attack). NBS10 is thought to work by increasing microvascular blood flow in the heart muscle via the development and formation of new blood vessels, thereby reversing the restriction of blood supply caused by a heart attack and rescuing at-risk cardiac tissue from eventual cell death.
About the PreSERVE AMI Clinical Trial
PreSERVE AMI is a randomized, double-blind, placebo-controlled clinical trial of intracoronary infusion of autologous CD34 cells in patients with left ventricular dysfunction post-ST elevation myocardial infarction (STEMI). The trial included 161 subjects at 60 sites in the United States, randomized 1:1 between treatment and placebo arms. Eligible patients presented with acute STEMI, had successful stenting of the infarct-related artery and had left ventricular dysfunction 4 days after AMI. Primary endpoints include occurrence of SAEs and MACE (defined as cardiovascular death, re-infarction, heart failure hospitalization, and coronary revascularization) through 3 year follow-up, occurrence of SAEs through 3 year follow-up, and 6-month change in myocardial perfusion (RTSS) measured quantitatively by gated SPECT myocardial perfusion imaging. Other endpoints include cardiovascular magnetic imaging resonance (CMR) to measure left ventricular ejection fraction (LVEF), left ventricular end-systolic volume (LVESV), left ventricular end-systolic diameter (LVEDV), regional myocardial strain, infarct/peri-infarct regional wall motion abnormalities and infarct size (baseline and six months) and quality of life measures (KCCQ and SAQ). While all 6 month data has been collected, it is subject to ongoing analysis, and results reported at this time, although promising, are preliminary. There can be no assurance that further analysis may not reveal negative, or less promising, results.
About NeoStem, Inc.
NeoStem is a biopharmaceutical company pursuing the preservation and enhancement of human health globally through the development of cell based therapeutics that prevent, treat or cure disease by repairing and replacing damaged or aged tissue, cells and organs and restoring their normal function. The business includes the development of novel proprietary cell therapy products as well as a revenue-generating contract development and manufacturing service business. This combination has created an organization with unique capabilities for cost effective in-house product development and immediate revenue and cash flow generation. www.neostem.com
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, 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 Targeted Immunotherapy Program, Ischemic Repair Program (including NBS10 for AMI), 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 contract development and manufacturing business. The Company's actual results could differ materially from those anticipated in these forward-looking statements as a result of various factors. Factors that could cause future results to materially differ from the recent results or those projected in forward-looking statements include the "Risk Factors" described in the Company's Annual Report on Form 10-K filed with the Securities and Exchange Commission ("SEC") on March 13, 2014, the Company's Current Report on Form 8-K filed with the SEC on May 8, 2014 and in the Company's other periodic filings with the SEC. The Company's further development is highly dependent on future medical and research developments and market acceptance, which is outside of its control.
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