Stem Cell Therapeutics has announces positive interim results from its Phase IIa study of Beta-hCG plus erythropoietin (EPO) in acute stroke. This uncontrolled, open-label safety trial was designed to determine whether NTxTM-265 could be safely administered to a population of patients with acute stroke. In addition to the trial's primary safety endpoint, a number of secondary endpoints are being studied to characterize early indicators of efficacy in patients receiving this novel stroke therapy.

To date, five patients have been enrolled. Each had a moderate to severe stroke, defined by study entry criterion of NIH Stroke Scale scores between 6 and 24. Of these, four patients have safely completed the NTxTM-265 regimen of human chorionic and EPO, initiated 24 to 48 hours after stroke. So far, no drug-related serious adverse events (SAEs) have been noted. A fifth study patient died before dosing was completed. This SAE was judged to be completely unrelated to the study drug regimen.

Accompanying the primary safety endpoint measures in this study is a battery of secondary endpoints that measure functional recovery. An earlier preclinical stroke study in rats established the proof of principle and impetus for proceeding into this clinical trial by demonstrating that administration of NTxTM-265, as compared with placebo, was associated with rapid and robust recovery of visual and tactile motor control of forelimb function, as well as reduced final infarct volume.

Interim results from the current trial show that each of the patients who completed the therapy demonstrated significant recovery from their stroke symptoms. In addition, magnetic resonance imaging readings at days one and 90 post-stroke, available from two of the patients, indicate that infarct volume had been reduced by 39 to 79 percent over this 90-day period.

NTxTM-265 is a therapeutic regimen of two drugs being developed for the treatment of stroke. Human chorionic gonadotropin is the first drug administered in the regimen, and aims to increase the number of neural stem cells (NSCs) located in the brain of a patient suffering from a recent stroke. EPO is the second drug administered in the regimen, and aims to promote the differentiation of these newly formed NSCs into new neurons. New neurons thus formed are anticipated to provide benefit to the patient through the replacement of the brain cells that were lost or damaged by the stroke, according to the company.