ARCA Biopharma Announces FDA Agreement for a Single Phase 3 Clinical Trial to Support Approval for the First Genetically-Targ...
February 20 2019 - 7:30AM
ARCA biopharma, Inc. (Nasdaq: ABIO), a biopharmaceutical company
applying a precision medicine approach to developing
genetically-targeted therapies for cardiovascular diseases, today
announced that it has reached agreement with the U.S. Food and Drug
Administration (FDA) regarding a Special Protocol Assessment (SPA)
on the design of a pivotal Phase 3 clinical trial, PRECISION-AF, to
assess the safety and efficacy of GencaroTM (bucindolol
hydrochloride) as a genetically-targeted treatment for atrial
fibrillation (AF) in patients with a specific type of heart failure
(HF). The target population for the planned Phase 3 clinical trial,
partially identified by precision therapeutic phenotyping,
currently has no FDA approved drug therapies. This SPA provides
agreement that the Phase 3 protocol design, clinical
endpoints, trial population and statistical analyses adequately
address objectives that, if met, would support a regulatory
submission seeking approval of Gencaro for the prevention of AF
recurrence in a genotype-defined HF population.
If PRECISION-AF is successful and Gencaro gains
regulatory approval, it has the potential to be unique in several
aspects, including:
- The first genetically-targeted
cardiovascular therapy;
- The only drug therapy indicated in HF patients with mid-range
ejection fraction (HFmrEF); and,
- The only drug therapy for AF approved against an active
comparator.
The SPA process is designed to facilitate review
and approval of drugs by allowing FDA to evaluate the proposed
design and size of specific clinical trials that are intended to
form the primary demonstration of a drug product’s efficacy and
safety. FDA ultimately assesses whether the protocol design and
planned analysis of the trial are acceptable to support regulatory
approval of the product candidate for the indication studied. An
SPA agreement can potentially reduce the regulatory risk of
bringing a drug to market.
"Consistent with our mission to develop
precision therapies on a pharmacogenetic platform, this SPA
agreement with the FDA provides a clearly defined regulatory
pathway for the Phase 3 development of Gencaro in a
genotype-specific heart failure population that currently has no
FDA approved drug therapy," said Michael R. Bristow, MD, PhD, Chief
Executive Officer of ARCA biopharma. "If the previous foundational
therapeutic observations in the GENETIC-AF and BEST trials are
confirmed in PRECISION-AF, we believe Gencaro could potentially
provide a new standard of treatment for AF prevention for the
estimated 2.5 million HFmrEF patients in the major markets in U.S.,
Europe and Japan."
In accordance with the Company’s SPA agreement
with FDA, PRECISION-AF is designed as a single, adequate and
well-controlled Phase 3 clinical trial that may be sufficient to
support an New Drug Application (NDA) submission for an AF
indication if the objectives of the trial are achieved consistent
with the requirements of the SPA. The trial is designed as a
double-blind, active-controlled, multicenter, international study
comparing Gencaro with Toprol-XL (metoprolol succinate) for the
prevention of AF recurrence or all-cause mortality (ACM) in HFmrEF
patients. HFmrEF is defined as HF with a left ventricular
ejection fraction (LVEF) ≥ 40% and < 50%, which constituted
approximately half of the enrolled population in the Phase 2
GENETIC-AF trial. PRECISION-AF is designed to enroll
approximately 400 patients who have: HFmrEF, a recent AF event, and
the genotype which responds most favorably to Gencaro. The primary
endpoint of the trial will be time to first event of atrial
fibrillation/atrial flutter (AF/AFL) or ACM during the 26-week
Follow-up Period. In the recently completed GENETIC-AF trial,
Gencaro showed a 58% treatment benefit compared to Toprol-XL in
reducing AF recurrence in the HFmrEF population targeted for Phase
3 (hazard ratio = 0.42; 95% CI: 0.21, 0.86; p = 0.017). With 400
patients (200 per arm) the trial will have 90% power at a p-value
of 0.01 to detect a 45% treatment benefit for Gencaro compared to
Toprol-XL. Subject to securing additional financing, ARCA
anticipates initiating PRECISION-AF in the fourth quarter of
2019.
About Special Protocol Assessment
(SPA)
An SPA is an agreement with the FDA that the
proposed trial protocol design, clinical endpoints and statistical
analyses are acceptable to support regulatory approval. For
further information regarding the SPA process, please visit the FDA
website, www.fda.gov. A SPA agreement is not a guarantee of
approval, and there are no assurances that the design of, or data
collected from, the planned Gencaro clinical trial (PRECISION-AF)
will be adequate to obtain the requisite regulatory approvals for
the marketing of Gencaro.
About Atrial Fibrillation
(AF)
AF, the most common sustained cardiac
arrhythmia, is a serious disorder in which the normally regular and
coordinated contraction pattern of the heart’s two small upper
chambers, or the atria, becomes irregular, rapid and
uncoordinated. AF can cause distressing symptoms that
significantly impact quality of life and can also bring potentially
serious medical consequences, including increasing the risk of
stroke and serious cardiovascular complications. AF is
considered an epidemic cardiovascular disease and a major public
health burden. In 2015, there were approximately 5.2 million
patients who had been diagnosed with AF in the United States.
It is estimated that AF costs the U.S. economy about $6.0 billion
annually.
About ARCA biopharma
ARCA biopharma is dedicated to developing
genetically-targeted therapies for cardiovascular diseases through
a precision medicine approach to drug development. ARCA’s lead
product candidate, GencaroTM (bucindolol hydrochloride), is an
investigational, pharmacologically unique beta-blocker and mild
vasodilator being developed for the potential treatment of atrial
fibrillation in heart failure patients with mid-range ejection
fraction. ARCA has identified common genetic variations that it
believes predict individual patient response to Gencaro, giving it
the potential to be the first genetically-targeted AF prevention
treatment. The Gencaro development program has been granted Fast
Track designation by FDA. ARCA is also developing AB171, a
thiol-substituted isosorbide mononitrate, as a potential
genetically-targeted treatment for heart failure and peripheral
arterial disease (PAD). For more information, please visit
www.arcabio.com.
Safe Harbor Statement
This press release contains "forward-looking
statements" for purposes of the safe harbor provided by the Private
Securities Litigation Reform Act of 1995. These statements include,
but are not limited to, potential future development plans for
Gencaro, ARCA’s ability to complete any Phase 3 clinical trial, the
likelihood for PRECISION-AF results to satisfy the requirements of
the SPA, ARCA’s ability to raise sufficient capital to fund the
PRECSION-AF trial and its other operations, the expected features
and characteristics of Gencaro, including the potential for genetic
variations to predict individual patient response to Gencaro,
Gencaro’s potential to treat AF and/or HFmrEF, future treatment
options for patients with AF and/or HFmrEF, and the potential for
Gencaro to be the first genetically-targeted AF prevention
treatment. Such statements are based on management's current
expectations and involve risks and uncertainties. Actual
results and performance could differ materially from those
projected in the forward-looking statements as a result of many
factors, including, without limitation, the risks and uncertainties
associated with: ARCA’s financial resources and whether they will
be sufficient to meet its business objectives and operational
requirements; ARCA may not be able to raise sufficient capital on
acceptable terms, or at all, to continue development of Gencaro or
to otherwise continue operations in the future; an FDA SPA
agreement does not guarantee approval of Gencaro or any other
particular outcome from regulatory review; results of earlier
clinical trials may not be confirmed in future trials; the
protection and market exclusivity provided by ARCA’s intellectual
property; risks related to the drug discovery and the regulatory
approval process; and, the impact of competitive products and
technological changes. These and other factors are identified
and described in more detail in ARCA’s filings with the Securities
and Exchange Commission, including without limitation ARCA’s annual
report on Form 10-K for the year ended December 31, 2017, and
subsequent filings. ARCA disclaims any intent or obligation to
update these forward-looking statements.
Investor & Media
Contact:Derek Cole720.940.2163derek.cole@arcabio.com
A photo accompanying this announcement is available at
http://www.globenewswire.com/NewsRoom/AttachmentNg/af496e97-20da-420a-bf93-e51b3a3ed740
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