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ARCA Biopharma, Inc. (NASDAQ: ABIO) Brings Precision Medicine Closer to Reality

  • Drug candidate for top cause of death in the United States
  • Genetically-targeted therapy increases chances of recovery
  • Interim efficacy analysis soon to be conducted by the Data Safety Monitoring Board

Atrial fibrillation affects up to six million people in the U.S. The condition, if untreated, can lead to blood clots, stroke, heart failure and other heart-related complications, but many medications currently in use to treat it come with worrying side effects, perhaps because of their one-size-fits-all approach. In an effort to mitigate the unwanted, unintended action of these agents, ARCA Biopharma, Inc. (NASDAQ: ABIO) is applying a precision medicine approach, developing genetically-targeted therapies for cardiovascular diseases, as it works to bring its lead candidate for atrial fibrillation, Gencaro™, to market. The company believes that by tailoring medical treatment to the individual genetic characteristics of patients, more effective therapies will be enabled, patient outcomes will be improved and health care costs will go down.

Atrial fibrillation (AFib) is the most common kind of heart arrhythmia, a condition where the heart beats too fast, too slow, or in an irregular fashion. In a heart suffering from AFib, the regular pumping action of its upper chambers, the atria, is impaired. As a result, blood flow to the lower chambers, the ventricles, is impeded. AFib increases a person’s risk for stroke by four to five times compared with people who do not have it, and strokes caused by complications from AFib tend to be more severe than strokes with other underlying causes. AFib causes 15%–20% of ischemic strokes, which occur when blood flow to the brain is blocked by a clot or by fatty deposits called plaque in the blood vessel lining.

According to the Centers for Disease Control and Prevention (, “more than 750,000 hospitalizations occur each year because of AFib… (and) the condition contributes to an estimated 130,000 deaths each year. (In addition), the death rate from AFib as the primary or a contributing cause of death has been rising for more than two decades.” AFib costs the United States about $6 billion each year, and medical costs for people who have AFib are about $8,705 higher per year than for people who do not have AFib.

Current treatments for AFib include drugs for heart rate control, drugs for heart rhythm control, and blood thinners. These medication, however, give rise to a plethora of side effects, including breast enlargement, diarrhea, low blood pressure, and heart failure, as well as psychological problems. One class of drugs that controls heart rate is known as beta-blockers; drugs of this type work by reducing heart rate, an example of which is ARCA’s lead candidate, Gencaro.

Gencaro (bucindolol hydrochloride), is a pharmacologically unique beta-blocker and mild vasodilator, now under evaluation in a clinical trial for the treatment and prevention of recurrent AFib in heart failure patients with reduced left ventricular ejection fraction, or HFREF. The company believes that Gencaro’s mechanism of action (MOA) differs from other beta-blockers because of its sympatholytic (norepinephrine lowering) and inverse agonism (inactivation of constitutively active receptors) properties. Genetic variations in receptors in the cardiovascular system of the general population vary, and these variations are thought to be a factor in how well patients respond to treatment. With Gencaro, ARCA has developed a formulation which interacts positively with a genotype, beta-1 389 arginine homozygous, found in approximately 50 percent of the U.S. population. Consequently, a Gencaro regimen promises a reduced range of side effects for a larger population of patients.

In June 2017, ARCA announced database lock for the GENETIC-AF interim efficacy analysis to be conducted by the trial Data Safety Monitoring Board (DSMB). Locking or closing a database ensures that no unauthorized or unintentional changes are made after the final data entry, check-up, and analysis. GENETIC-AF is a phase 2B/3 double-blind, clinical superiority trial comparing the safety and efficacy of Gencaro to an approved drug, TOPROL-XL, for the treatment and prevention of recurrent AFib in heart failure patients with reduced left ventricular ejection fraction (HFrEF). The company expects to announce the DSMB’s recommendation based on this interim analysis in August 2017. The Gencaro development program was previously granted Fast Track designation by the U.S. Food and Drug Administration (FDA).

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