ARCA Biopharma, located in Westminster, Colorado, announced mixed but positive outcomes from a Phase II clinical trial of its beta-blocker Gencaro for atrial fibrillation (AF) patients with heart failure (HF).
ARCA Biopharma, located in Westminster, Colorado, announced mixed but positive outcomes from a Phase II clinical trial of its beta-blocker Gencaro for atrial fibrillation (AF) patients with heart failure (HF). After an End-of-Phase II meeting with the U.S. Food and Drug Administration (FDA) the company has a recommended go-ahead for a Phase III clinical trial under a Special Protocol Assessment (SPA) process. Shares rocketed from $0.82 on Friday to $1.19 today, so far.
The company stated, “Based on review of the Phase II GENETIC-AF trial results, as well as its alignment with a previous Phase III pharmacogenetic substudy data, the FDA stated that data from a single pivotal Phase III clinical trial may be sufficient to support approval of Gencaro for the treatment of AF in patients with HF. The company, in consultation with the FDA, has established key elements of the Phase III clinical trial needed to support a New Drug Application (NDA), details of which will be confirmed via the FDA Special Protocol Assessment (SPA) process.”
On May 27, the company presented data from the GENETIC-AF trial at the European Society of Cardiology Heart Failure 2018 World Congress. The study population was made up of patients at high risk for recurrent atrial fibrillation. In that trial, Gencaro, guided with a pharmacogenetic diagnostic panel, did not reduce AF/AFL/ACM recurrence compared to TOPROL-XL. But, in U.S. patients, which made up 48 percent of the entire cohort, there was a trend for potential benefit in favor of Gencaro, about a 30 percent risk reduction over TOPROL-XL.
Another trend for a potential benefit in favor of Gencaro was observed in a subset of patients from the U.S., Canada and Europe who had their heart rhythm continuously monitored with a Medtronic implanted device.
“With additional analysis of the trial data, and taking into consideration recent studies in animal models of AF, it is likely that AF-HFrEF phenotypic differences are responsible for the heterogeneity in treatment response observed,” said William Abraham, professor of Medicine, Physiology and Cell Biology, and director, Division of Cardiovascular Medicine at Ohio State University, in a statement. “These Phase IIb data support and provide guidance for potential additional development of Gencaro as a treatment for atrial fibrillation in patients with heart failure, an indication for which there are currently no FDA approved therapeutics.”
The Phase IIb trial was a double-blind, superiority clinical trial comparing Gencaro as a genetically-targeted treatment for AF in patients with HF and reduced left ventricular ejection fraction (HFrEF). It looked at 267 patients from the U.S., Canada and Europe.
The market was likely surprised by the go-ahead from the FDA because the Phase II data didn’t show a treatment benefit compared to TOPROL-XL. However, the FDA found trends worth investigating and recommended submitting an SPA application. The company expects to submit the application in the third quarter. ARCA indicates it will launch the Phase III clinical trial when it pulls together additional financing.
The FDA had previously granted the drug Fast Track Designation. The Phase III trial will probably be a single randomized, active-controlled clinical trial with the primary endpoint based on AF Burden, which is defined as the amount of time per day a patient experienced AF, as measured by an implanted monitoring device.
