BioCardia’s attempts to revive its paused phase 3 heart failure trial have hit a major setback, as results fail to inspire optimism regarding the primary endpoint. Following a recommendation from the independent data safety monitoring board to halt enrollment and conduct a thorough analysis, the biotech company has now revealed that the study is unlikely to achieve its primary objective, causing further strain on its stock value.
Concerns about the phase 3 trial’s progress emerged in late July when BioCardia informed investors about the enrollment pause, pending an evaluation of patients who had recently completed their one-year follow-up. The recent results stemming from the 102 randomized patients who finished their one-year review do not bode well.
The primary endpoint comprises three components, and BioCardia’s cell therapy CardiAMP showed no significant advantage over the control group in any of them. Specifically, the rate of all-cause death and cardiac death equivalents after one year was nearly identical in both groups, with 5.6% for the CardiAMP cohort and 5.3% for the control group.
Similarly, CardiAMP failed to demonstrate superiority in the other two elements of the composite endpoint. The rate of nonfatal major adverse cardiac events was comparable between the two groups, at 16.7% for the treatment group and 15.8% for the control group. Additionally, there was no significant difference in the change in the six-minute walk test distance.
CardiAMP involves delivering a patient’s own bone marrow cells to the heart using a catheter-based procedure, with the goal of triggering the body’s natural healing response. The therapy’s inability to significantly enhance outcomes on any aspect of the composite endpoint in the phase 3 trial led BioCardia to conclude that the study is unlikely to achieve its goals. The trial sites are currently wrapping up the treatment of patients who were previously enrolled, and BioCardia will continue to monitor these participants.
Despite the discouraging primary endpoint results, BioCardia’s analysis of all available follow-up data up to 24 months suggests some potential efficacy. The CardiAMP cohort exhibited a lower rate of all-cause death and cardiac death equivalents (8.3%) compared to the control arm (13.2%). Additionally, there was a numerical improvement in nonfatal major adverse cardiac events (16.7% for CardiAMP versus 23.6% for the control group).
“We are actively engaged in identifying patients who responded the most to the therapy and are considering other learnings with respect to trial design to inform this program and our other two ongoing clinical programs. We anticipate working with the principal investigators and executive steering committee on these efforts.”
– Peter Altman, Ph.D., BioCardia’s president and CEO
Investor sentiment toward BioCardia remains subdued, with the latest update causing the company’s stock to plummet approximately 20% to 62 cents during premarket trading. This adds to a series of losses that have seen the share price decline by more than 60% in the past six months.