Enhancing the Body’s Healing Response
We are pursuing the development of AMR-001, a chemotactic hematopoietic stem cell product comprised of autologous bone marrow derived CD34+/CXCR4+ cells selected to treat damaged heart muscle following AMI. AMR-001 is being evaluated to determine safety and the effect of intracoronary infusion of AMR-001 on myocardial perfusion (amount of blood in the heart) at six months post randomization in subjects post acute ST elevation myocardial infarction (“STEMI”), a particular type of AMI. AMR-001 is also being evaluated to determine its effect on preservation of left ventricular function and risk of major adverse cardiac events following AMI, and impact on patient reported outcomes, among other endpoints. Multiple peer reviewed publications from a range of laboratories and investigators indicate that AMR-001 should increase microvascular blood flow in the myocardium (heart muscle) via angiogenesis (development and formation of new blood vessels), thereby reversing post-heart attack induced restriction of blood supply and rescuing tissue from eventual cell death. This would result in prevention of myocardial infarct expansion and thus block ventricular remodeling – the driver of long-term risk in AMI patients.
At the time of a heart attack, doctors rush to open up the coronary artery, usually using a stent. Within the first 5 to 11 days following the heart attack is believed to be the optimal time to administer AMR-001 via that same artery. With angiogenesis initiated in the peri-infarct zone (that is, the living tissue on the periphery of the dead tissue), the myocardium surrounding the site of the heart attack is preserved.
How Does It Work?
In December 2010, Amorcyte (which we acquired in October 2011) reported results of a Phase 1 study of AMR-001 treating 31 patients with damaged heart muscle following AMI. In January 2012, we commenced a Phase 2 study of AMR-001 for the same indication as a multicenter, randomized, double-blind, placebo-controlled clinical trial evaluating the safety and efficacy of infusing AMR-001 into an infarct-related artery. Patient enrollment was completed in December 2013 with data read out expected in third quarter of 2014. Based on experiences in drug development, the literature and our Phase 1 results, we believe AMR-001 shows promise to be efficacious and safe for treating patients who suffer a ST Elevation Myocardial Infarction (“STEMI”), particularly when using a pure, potent and adequately dosed CD34 cell population.
We believe that the AMR-001 platform may be applicable to other conditions resulting from underlying ischemia. In that regard, we expect to file an IND for the use of AMR-001 in arresting the progression of congestive heart failure and treating the associated comorbidities of that disease. We are also exploring the possibility of filing an IND for the treatment of traumatic brain injury.
NeoStem’s AMR-001 is protected by one of the most comprehensive IP estates in the industry, including composition of matter.