VERICEL CORP.’s (Ann Arbor MI) stem-cell therapy helped reduce deaths and hospitalizations in advanced heart failure patients, according to data from a mid-stage study, potentially providing a treatment that could delay the need for heart transplants.
However, the treatment (ixmyelocel-T) failed to improve secondary goals of the trial, such as heart-pumping efficiency and six-minute walking distance, and Vericel shares fell 48% for the week to $3.14.
“It’s a little baffling that you’re keeping patients alive, but not having any effect on those secondary endpoints,” said Needham & Co. analyst Chad Messer. “People were looking for downside and they had one.”
The data compared Vericel’s bone marrow-derived ixmyelocel-T stem cells with placebo in 109 well-treated patients with advanced heart failure who had exhausted medical and device therapies. After one year, the Vericel treatment led to a 37% reduction versus placebo in a composite of adverse events, including death, heart-related hospitalizations and unplanned clinic visits related to heart failure.
Vericel last month reported that the study succeeded. Detailed results were unveiled last week at an American College of Cardiology meeting in Chicago and in the Lancet medical journal (pdf). The result was primarily driven by the difference in deaths of 13.7% in the placebo group, or 7 deaths, compared with 3.4%, or 2 deaths, for the ixmyelocel-T group. In addition, 38% of stem-cell patients required hospitalization, versus 47% for placebo.
“This is strong evidence in a well-designed trial that we can decrease events,” said Dr. Timothy Henry, the study’s lead investigator and chief of cardiology at Cedars Sinai Heart Institute in Los Angeles. “For patients, this is a really hopeful thing.”
Henry, who presented the data at the ACC meeting, stressed the need for larger trials to prove the benefit of ixmyelocel-T. For the treatment, bone marrow is taken from the patient and enhanced over two weeks to increase two types of cells associated with healing. They are then injected directly into the patient’s heart.
Monday, April 11, 2016 / Vol. 24 / No. 14