BioCardia, Inc. [Nasdaq: BCDA], a developer of cellular and
cell-derived therapeutics for the treatment of cardiovascular and
pulmonary disease, today announces that the independent Data Safety
Monitoring Board (DSMB) has completed its prespecified data review
for the ongoing Phase III pivotal CardiAMP® Cell Therapy
Heart Failure Trial (clinicaltrials.gov Identifier: NCT02438306),
for which FDA granted Breakthrough Designation. Pursuant to the
DSMB recommendation, BioCardia is pausing the trial pending the
one-year follow-up outcomes analysis for patients that have been
treated and those that have been enrolled but not yet treated.
The DSMB recommendation stated:
“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 one-year follow-up as well as the
patients completing their imminently scheduled treatment.
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 one-year data analysis.”
The DSMB interim review was based on available data for 132
procedures involving 111 randomized patients, including treated
patients and the control group. The pre-specified statistical
analysis plan provided that the DSMB would utilize a composite
endpoint that considered survival, major adverse cardiac and
cerebrovascular events (MACCE), and six-minute walk distance at 12
months. This is the Finkelstein Schoenfeld (FS) composite endpoint
for the study. The pre-specified interim analysis did not impute
data for patients who had not performed the six-minute walk, either
because they were not yet at the one-year follow-up date or because
of orthopedic or other health issues as will be done in the final
statistical analysis per plan. The pre-specified interim
statistical analysis did not include other endpoints that are part
of the trial, including assessment of patient status using the New
York Heart Association classification, quality of life measured
using the Minnesota Living with Heart Failure questionnaire, and
heart function assessment such as left ventricular ejection
fraction.
The blinded data report available to the Company groups together
all patients, including treated and control patients. That blinded
data shows that the aggregate survival rate at follow-up is greater
than that observed in recent similar large pivotal trials in
patients with heart failure with reduced ejection fraction.
The blinded data also shows improved health outcomes in aggregate
for patients followed-up through all key visit dates through
12-months such as measured by the six-minute walk test, patient
classification per the New York Heart Association system, quality
of life measured using the Minnesota Living with Heart Failure
questionnaire, and heart function as measured by the left
ventricular ejection fraction. The blinded data does not disclose
the difference in outcomes between the treated group and the
control group of patients.
The DSMB panel includes two cardiologists with experience in
heart failure and interventional cardiology and a biostatistician
experienced in evaluating trial data for chronic diseases,
including heart failure. The mission of the DSMB is to ensure
patient safety and to promote/monitor the quality and integrity of
the data. Their primary responsibility is to ensure patient safety
and to halt or pause the clinical investigation if the risk of the
therapy appears to outweigh its potential benefit. Such
risk-benefit analysis typically includes an analysis of futility to
reach the primary endpoint of the clinical trial as
designed.
The DSMB reviewed an initial analysis of the unblinded data and
concluded that the trial was unlikely to meet its primary FS
composite endpoint, even though the prespecified criteria for
termination of the trial had not been met. This initial analysis
excluded patients who had not yet made it to 12-month follow-up.
The DSMB also cited the slow rate of enrollment in the trial.
Additional data was subsequently provided to the DSMB including, we
believe: (1) statistical analysis prepared strictly in accordance
with the prespecified data review plan, which had not initially
been provided to the DSMB, (2) analysis of health outcomes as
measured by endpoints other than the six-minute walk, and (3)
information on the increased enrollment rates across many clinical
centers. After consideration of the additional data, the DSMB
recommended that the Company pause enrollment of new patients
pending the 12-month outcomes analysis for all patients, and that
the Company continue to treat patients already enrolled in the
trial. As noted, the DSMB also recommended that the data blind be
preserved so that the trial may be continued following analysis of
the twelve-month follow-up data for all enrolled patients.
“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,” said BioCardia CEO Peter Altman, Ph.D. “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.”
About the CardiAMP Cell Therapy Program
CardiAMP Cell Therapy uses a patient’s own (autologous)
bone marrow cells delivered to the heart in a minimally invasive,
catheter-based procedure to potentially stimulate the body’s
natural healing response. The CardiAMP Cell Therapy Heart Failure
Trial is the first multicenter clinical trial of a stem cell
therapy to prospectively screen for stem cell therapeutic potency
in order to improve patient outcomes. CardiAMP Cell Therapy
incorporates three proprietary elements not previously utilized in
an investigational cardiac cell therapy trial, which the company
believes improves the probability of success of the Phase III
Trial: a pre-procedural cell analysis for patient selection, a high
target dosage of cells, and a proprietary delivery system that has
been shown to be safer than other intramyocardial delivery systems
and exponentially more successful in cell retention. CAUTION -
Limited by United States law to investigational use.
About BioCardia®
BioCardia, Inc., headquartered in Sunnyvale, California, is
developing cellular and cell-derived therapeutics for the treatment
of cardiovascular and pulmonary disease. CardiAMP™ autologous and
NK1R+ allogeneic cell therapies are the Company’s biotherapeutic
platforms that enable four product candidates in development. The
CardiAMP Cell Therapy Heart Failure Trial investigational product
has been granted Breakthrough Designation by the FDA, has CMS
reimbursement, and is supported financially by the Maryland Stem
Cell Research Fund. The CardiAMP Chronic Myocardial Ischemia Trial
also has CMS Reimbursement. BioCardia also partners
with other biotherapeutic companies to provide its delivery systems
and development support to their programs studying therapies for
the treatment of heart failure, chronic myocardial ischemia and
acute myocardial infarction. For more information
visit: www.biocardia.com.
Forward Looking Statements:
This press release contains forward-looking statements that are
subject to many risks and uncertainties. Forward-looking statements
include, among other things, statements relating to pausing the
clinical trial, study enrollment expectations, future data
analysis, future DSMB reviews, the likelihood of safety and patient
benefit, the ultimate success of our clinical cell therapy
programs, and the expansion or acceleration of CardiALLO heart
failure or CardiAMP Chronic Myocardial Ischemia trials. These
forward-looking statements are made as of the date of this press
release.
We may use terms such as “believes,” “estimates,” “anticipates,”
“expects,” “plans,” “intends,” “may,” “could,” “might,” “will,”
“should,” “approximately” or other words that convey the
uncertainty of future events or outcomes to identify these
forward-looking statements. Although we believe that we have a
reasonable basis for each forward-looking statement contained
herein, we caution you that forward-looking statements are not
guarantees of future performance and that our actual results may
differ materially from the forward-looking statements contained in
this press release. As a result of these factors, we cannot assure
you that the forward-looking statements in this press release will
prove to be accurate. Additional factors that could materially
affect actual results can be found in BioCardia’s Form 10-K filed
with the Securities and Exchange Commission on March 29, 2023,
under the caption titled “Risk Factors.” BioCardia expressly
disclaims any intent or obligation to update these forward-looking
statements, except as required by law.
Media Contact:Miranda Peto, Marketing /
Investor RelationsEmail: mpeto@BioCardia.comPhone:
650-226-0120
Investor Contact:David McClung, Chief Financial
OfficerEmail: investors@BioCardia.comPhone: 650-226-0120
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