On February 18, 2020 ARCA biopharma, Inc. (Nasdaq: ABIO), a biopharmaceutical company applying a precision medicine approach to developing genetically-targeted therapies for cardiovascular diseases, reported financial results for the year ended December 31, 2019 and provided a corporate update (Press release, Arca biopharma, FEB 18, 2020, View Source [SID1234554421]).

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Dr. Michael Bristow, ARCA’s President and Chief Executive Officer, commented, "During 2019, we continued to advance development of Gencaro as a potential genetically-targeted treatment for heart failure patients with atrial fibrillation. In published or submitted for presentation/publication material, we have identified important new effectiveness evidence from the GENETIC-AF trial that have enhanced our understanding of the clinical profile of Gencaro. These data have allowed us to broaden the design of the single, pivotal Phase 3 clinical trial that was developed after consultation with the U.S. Food and Drug Administration via a Special Protocol Assessment agreement. There are currently no FDA approved drug therapies indicated for the treatment of atrial fibrillation in heart failure patients with left ventricular ejection fraction values greater than 40%. Based on our clinical data to date, we believe Gencaro has the potential to help address this substantial unmet medical need. We look forward to further evaluating the pharmacogenetic benefits of Gencaro for patients with our targeted genetic profile in PRECISION-AF, the Phase 3 clinical trial, which, subject to obtaining additional financing, we plan to initiate in the fourth quarter of 2020."

Pipeline Update – Second Half Review

GencaroTM (bucindolol hydrochloride) – a pharmacologically unique beta-blocker and mild vasodilator being developed as a potential genetically-targeted treatment for atrial fibrillation (AF) in patients with heart failure (HF).

The U.S. Food and Drug Administration (FDA) issued a Special Protocol Assessment (SPA) agreement for a single Phase 3 clinical trial (PRECISION-AF) to examine Gencaro as a genetically-targeted therapy for the prevention of AF recurrence in certain heart failure patients.
PRECISION-AF is designed as a double-blind, active-controlled, multicenter, international, adaptive study comparing Gencaro with TOPROL-XL (metoprolol succinate) for the prevention of AF recurrence or all-cause mortality in approximately 400 heart failure patients who have left ventricular injection fraction (LVEF) values ≥ 40% and ≤ 55% and the genotype which ARCA believes responds best to Gencaro (ADRB1 Arg389Arg).
Clinical data from the GENETIC-AF Phase 2B clinical trial was published in the Journal of the American College of Cardiology: Heart Failure in May 2019 and was presented at the Heart Failure Society of America (HFSA) Annual Scientific Meeting in September 2019. These data indicate that the response to Gencaro may be greater in HF patients with less severe left ventricular dysfunction, a patient population with no FDA approved drug therapeutic options for AF prevention or heart failure.
Subject to securing additional financing, ARCA anticipates initiating PRECISION-AF in the fourth quarter of 2020.
AB171 – a thiol-substituted isosorbide mononitrate being developed as a potential genetically-targeted treatment for HF and peripheral arterial disease (PAD).

Subject to securing additional financing, the Company anticipates conducting non-clinical studies to support a potential IND submission and initiation of clinical development in 2021.
2019 Summary Financial Results

Cash and cash equivalents were $8.4 million as of December 31, 2019, compared to $6.6 million as of December 31, 2018. ARCA believes that its current cash and cash equivalents will be sufficient to fund its operations, at its current cost structure, after giving effect to potential cost reductions, through the end of the third quarter of 2020.

Research and development (R&D) expenses for the year ended December 31, 2019 were $1.8 million compared to $4.2 million for 2018. The $2.4 million decrease was primarily due to decreased clinical expenses following the completion of the GENETIC-AF clinical trial. If the PRECISION-AF clinical trial is initiated in the second half of 2020, R&D expense in 2020 is expected to be higher than 2019.

General and administrative (G&A) expenses were $4.0 million for 2019 compared to $3.9 million for 2018. The Company expects G&A expenses in 2020 to be consistent with those in 2019 as it maintains administrative activities to support its ongoing operations.

Total operating expenses for 2019 were $5.8 million compared to $8.1 million for 2018. The decrease in total operating expenses was primarily attributable to the decrease in R&D expense due to the completion of the GENETIC-AF clinical trial.

Net loss was $5.5 million, or $4.15 per basic and diluted share, for 2019 compared to $7.9 million, or $10.31 per basic and diluted share, for 2018.

The Company will need to raise additional capital, and/or complete a partnership or other possible strategic transaction, to fund future operations and develop Gencaro or any other product candidates.