November 18, 2014
By Riley McDermid, BioSpace.com Breaking News Editor
Cell therapy company NeoStem was taking a beating in midday trading Tuesday, after its shares fell 26 percent on news that a Phase 2 trial of experimental heart drug NBS10 had failed to meet its primary endpoint of rebuilding damaged heart muscle.
NeoStem presented the results from a small study of cardiac stem-cell therapy NBS10 at the American Heart Association annual meeting on Monday.
Its Phase 2 PreSERVE AMI (or acute myocardial infarction) Clinical Trial tracked 161 patient being treated and having received six month follow-up for imaging and 12 month median length follow up for mortality, adverse events, serious adverse events (SAEs) and major adverse cardiac events (MACE).
Unfortunately, NBS10, formerly known as AMR-001, was no different than a placebo at reviving blood flow through the heart, and it was only slight better than a placebo at preventing MACE in patients studied. The drug is an autologous stem-cell therapy that is taken from a patient’s own bone marrow.
In theory, the stem cells received are then supposed to rebuild damaged heart muscle and tissue and get blood circulating again. But when patients treated with an infusion of the drug underwent non-invasive imaging to assess blood flow through the heart six months after the treatment, there was no statistical difference between those treated with NBS10 and those treated with a placebo.
Perhaps more concerning was the data that showed 17 percent of patients treated with NBS10 still subsequently suffered a MACE event, only a slight dip from the 19 percent of patients given a placebo.
NeoStem attempted to put a sanguine face on the news, but Wall Street investors weren’t buying it Tuesday, pushing shares of the company down more than a quarter to rest at $5.01 as of midday trading.
“For cardiologists, our key goal is to keep patients from progressing to worsening heart muscle function and death after a major heart attack,” said Dr. Arshed Quyyumi, professor of Medicine at Emory University and lead principal investigator of the PreSERVE AMI study. “It is encouraging to see clinically meaningful results this early in the study, and I look forward to future data readouts.”
NeoStem said it will continue to test the drug, saying that some of the data gathered was helpful and instructive and may show possible ways it could work in different therapies.
“Similar to what was seen in Phase 1, we have observed a relationship between the dose of CD34 cells administered and a positive effect on cardiac function. In our current trial, in addition to improved cardiac function, a CD34 dose-related reduction in serious adverse events was observed, highlighting the potential for clinical benefit,” said Andrew Pecora, director and chief visionary officer of NeoStem.
