BioCardia®, Inc. [Nasdaq: BCDA], a developer of cellular and
cell-derived therapeutics for the treatment of cardiovascular and
pulmonary disease, today announced it has finalized external review
of interim data and evaluated interim efficacy results for the
ongoing Phase III pivotal trial of CardiAMP® Cell Therapy
for Heart Failure (CardiAMP HF, clinicaltrials.gov Identifier:
NCT02438306), for which the FDA granted Breakthrough Designation
for the treatment of heart failure of reduced ejection fraction
(HFrEF).
The external review both confirmed the quality and
appropriateness of the work by the Statistical Data Analysis Center
at the University of Wisconsin and the DSMB in their review and
recommendation. In order to enhance understanding of our heart
failure program, BioCardia has taken steps, previously reviewed by
the DSMB, to unblind itself to the interim results of the study and
was provided a copy of the closed session study report presented to
the DSMB for review.
The interim efficacy review by the DSMB did not include the
final results of the study but is an analysis of the data as part
of the adaptive statistical analysis plan. This review was based on
the three-tiered Finkelstein-Schoenfeld (FS) hierarchical analysis
of 12-month data, whose composite score comprises the primary
efficacy endpoint of the CardiAMP HF study.
- For the first-tier, all cause death, including cardiac death
equivalents such as heart transplant or left ventricular assist
device placement at 12 months there were 5.6% of CardiAMP treatment
patients versus 5.3% for the control group (p = 0.42). All
available follow-up in the patients followed up to 24 months showed
a separation between groups with 8.3% all cause death including
cardiac death equivalents in the treatment group versus 13.2% in
the control group (p = 0.94), corresponding to a 37% relative risk
reduction for Tier 1 events for the treated patients compared to
control patients on guideline directed medical therapy (GDMT).
- For the second-tier, non-fatal major adverse cardiac events
(MACCE), excluding those deemed procedure-related occurring within
the first seven days and defined as heart failure hospitalization,
stroke and myocardial infarction, 16.7% of treatment group had
MACCE compared to 15.8% of the control group (p=0.82). All
available follow-up data for patients followed up to 24 months
showed a separation between groups, with 16.7% MACCE rate in the
treatment group versus 23.6% in the control group (p=.76). This
corresponds to an 18% relative risk reduction for MACCE over
GDMT.
- For the third-tier of change in Six Minute Walk Test (6MWT)
distance from baseline to 12 months, both CardiAMP treatment
patients and control patients experienced a clinically meaningful
improvement at 12 months which was not statistically different. The
treated group median 6MWT at 12M increased 36.1 with a standard
deviation of ±70.8 meters, and the control group median 6MWT
increased at 12M by 33.4 meters ± 74.8 meters (p =0.6). Results at
24 months also showed improvements in both groups with the
treatment group median improving 1.8 ± 76.1 meters and the control
group median improving 17.4 ± 95.7 meters (p=0.33). The improvement
in 6MWT at 12M for the control group was greater than was expected
in this population.
Based on these measures at one year follow-up, the 250-patient
randomized controlled study is unlikely to meet its primary
three-tiered FS efficacy endpoint, as seen from the initial 102
randomized patient set assessed at 12-month follow-up (110 patients
had been randomized and eight of them had not completed their
12-month follow-up visit at the time of the analysis). This interim
randomized study data does not include the study’s roll-in open
label cohort of 10 patients treated who did well with 100% survival
at two-year follow-up. These limited interim results related to the
primary efficacy endpoint have been shared with the Co-National
Principal Investigators of the study.
The study’s Co-National principal Investigator, Dr. Carl Pepine,
MD, MACC, Professor of Medicine at the University of Florida at
Gainesville, said, “I am pleased that the aggregate outcomes data
show promising low rates of adverse outcomes for a cohort of over
100 subjects with HFrEF due to ischemic heart disease. Furthermore,
the study is the first to demonstrate that it is feasible to
‘personalize’ therapy using a novel bone marrow cell population
analysis, process the cells at the bedside, and safely administer
them by a trans myocardial injection system.”
Dr. Amish Raval, MD, Professor of Medicine at University of
Wisconsin at Madison and Co-National Principal Investigator, said,
“Although the trial interim efficacy results show that the trial is
unlikely to achieve its primary endpoint at one year, these results
show that, while not statistically significant, there were trends
towards patient benefits for the CardiAMP cell therapy over the
course of the study, including a reduction in all cause death,
including heart death equivalents and MACCE. I congratulate the
study investigators on the absence of treatment emergent safety
concerns.”
There were additional prespecified outcome measures detailed in
the comprehensive SDAC closed session report. We also have
significant data for the autologous cell dosing as performed in the
study procedure, enabling assessments of efficacy on a
patient-by-patient basis, which we will study. Knowledge from these
interim details may be incorporated in its ongoing development
plans for its autologous CardiAMP and allogeneic CardiALLO cell
therapy programs.
“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,” said Peter Altman, BioCardia President
and CEO. “We anticipate working with the Principal Investigators
and Executive Steering committee on these efforts. In parallel we
expect to support centers as they complete treatment of previously
enrolled patients in the next six weeks and follow these patients
in a double blinded fashion as guided by the DSMB.”
Anticipated Upcoming Milestones and Events:
- BCDA-01: CardiAMP Cell Therapy for
Heart Failure Phase III Trial
- Q4 2023: Japan PMDA Formal
Consultation
- BCDA-02: CardiAMP Cell Therapy for
Chronic Myocardial Ischemia Phase III Trial
- Q4 2023: Completion of Roll-in Cohort
and Transition to Randomized Pivotal Trial
- BCDA-03: NK1R+ MSC Allogeneic Cell
Therapy in ischemic HFrEF Phase I/II Trial
- Q3 2023: First Patient Enrolled
- Helix Biotherapeutic Delivery System
- Q4 2023: Completion of Enrollment in
Partner CellProthera’s EXCELLENT Trial
- Q4 2023: Update on Licensing /
Partnerships
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: 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 future data
analysis, future protocol submissions to FDA, anticipated
milestones and events, conclusions of results based on the interim
data report generated by the CardiAMP Heart Failure statistics
core, the likelihood of safety and patient benefit, and the
ultimate success of our clinical cell therapy programs. 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. Factors that could cause or contribute to such
differences include, but are not limited to, the Company’s
liquidity position and its ability to raise additional funds, as
well as the Company’s ability to successfully progress its clinical
trials. 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” and in its subsequently filed
Quarterly Reports on Form 10-Q. 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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