BioCardia's CardiAMP Heart Failure Cell Therapy Faces Uncertain Future


A DSMB futility analysis found that the trial was unlikely to meet its primary composite endpoint and trial enrollment has been paused.

BioCardia is pausing enrolment in its phase 3 trial (NCT02438306) assessing CardiAMP cell therapy in patients with heart failure with reduced ejection fraction while additional blinded data are collected, in accordance with a data safety monitoring board (DSMB) recommendation.1

The recommendation concluded that the trial was unlikely to meet its primary composite endpoint based on a 3-tiered Finkelstein-Schoenfeld (FS) hierarchical analysis including outcomes of all-cause death, non-fatal major adverse cardiac and cerebrovascular events (MACCE)events, and change for 6-minute walk test (6MWD) from baseline to month 12.

“Based on an analysis of the trial data, the primary FS composite endpoint assessment and a supplemental analysis presented on 7/19/23, unrelated to any emergent safety events, the DSMB recommends pausing new patient enrollment and any potential crossover patient procedures pending an outcomes analysis of patients currently completing the 1-year follow-up as well as the patients completing their imminently scheduled treatment,” the recommendation stated.1 “The DSMB recommends notifying currently enrolled patients completing their treatment sequence that the trial will be paused following their scheduled treatment to assess intermediate study results. The DSMB recommends the blind not be broken at this time to protect the integrity of the outcomes yet to be collected and to ensure that the study may be restarted without compromise after completion of the 1-year data analysis.”

The DSMB conducted a futility analysis on the unblinded data and concluded that the trial was unlikely to meet its primary endpoint, although the trial had not met the prespecified criteria for termination, and also cited the trial’s slow rate of enrollment. The data reviewed excluded patients that had not yet reached 12-month follow-up. After reviewing additional data, including a prespecified statistical analysis, other measures of health outcomes, and information on the increased enrollment rate, the DSMBrecommended the pause.

READ MORE: BioCardia Follows up CardiAMP With CardiALLO Clearance for Heart Failure

The interim review was based on data from 132 procedures in 111 patients randomized to treatment or control and was based onlyoff the FS composite endpoint. So far, the blinded data show benefits in survival compared to similar trials and improvements in health outcomes from baseline on 6MWD, New York Heart Association scores, quality of life, and heart function as measured by the left ventricular ejection fraction.

“We look forward to enabling the DSMB to have the requested analysis at twelve-month follow-up. We are pleased that there appear to be no treatment emergent safety issues and that the health outcomes measured in the trial show improvement for patients as a group,” Peter Altman, PhD, CEO, BioCardia, said in a statement.1 “We will utilize this pause to accelerate our CardiAMP Chronic Myocardial Ischemia trial and our CardiALLO Heart Failure trial with our world class clinical partners.”

The CardiAMP autologous cell therapy is intended to be a comprehensive therapeutic treatment that includes a proprietary molecular diagnostic to determine eligibility of patients’ bone marrow cells, a point of care processing platform that prepares the cells at the patient’s bedside, and the company’s proprietary Helix biotherapeutic delivery system to administer the therapy.

The most recent data on CardiAMP for heart failure were from 10 patients in a roll-in cohort of the phase 3 trial and were presented at the Heart Failure Society of America meeting in Washington, DC, in October 2022. The cohort had a 100% survival rate at 2 years after treatment, there were no serious adverse events related to treatment, and significant improvements in 6MWD were observed at 6 (6% improvement) and 24 months (8% improvement) compared to baseline.2

“While this smaller cohort is not a head-to-head comparison, current state of the art therapies for these patients which have been successful in slowing disease, are assessed by us to have 79.9% survival after 2 years,” Altman said in a statement at that time.2

1. BioCardia announces DSMB recommendation to pause new enrollment in phase III pivotal CardiAMP cell therapy heart failure trial while additional blinded data is collected. News release. BioCardia. July 24, 2023.
2. BioCardia presents positive CardiAMP cell therapy heart failure trial two-year data at HFSA Annual Meeting. News release. BioCardia. October 3, 2022. Accessed July 24, 2023.
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