STOCKHOLM, Sweden, June 25, 2026
AnaCardio announced that results from its Phase 1b/2a GOAL-HF1 clinical trial evaluating AC01, a first-in-class oral calcium-sensitising contractile agent for heart failure with reduced ejection fraction (HFrEF), have been published in The Lancet. The peer-reviewed publication highlights a favorable safety and tolerability profile, dose-proportional pharmacokinetics, and evidence of target engagement, while exploratory analyses demonstrated rapid and sustained numerical improvements in cardiac output, stroke volume, cardiac structure, and heart function in patients receiving optimized guideline-directed medical therapy. The publication represents an important scientific milestone for AnaCardio and supports the continued clinical advancement of AC01 as a potential new oral therapy for heart failure, an area where safe contractility-enhancing medicines remain limited.
GOAL-HF1 Study Demonstrates Encouraging Safety and Cardiac Function Improvements
The GOAL-HF1 study enrolled 58 patients with NYHA Class II–III HFrEF across 14 clinical sites in the Netherlands, United Kingdom, Sweden, and Italy. The randomized, double-blind, placebo-controlled Phase 1b/2a trial evaluated the safety, tolerability, pharmacokinetics, pharmacodynamics, and exploratory efficacy of AC01. According to the published findings, no AC01-related serious adverse events, deaths, or treatment discontinuations were reported. Importantly, investigators observed no evidence of tachycardia, new arrhythmias, myocardial ischemia, symptomatic hypotension, or conduction abnormalities, addressing long-standing safety concerns associated with traditional inotropic therapies. Exploratory efficacy analyses showed rapid improvements in cardiac output and stroke volume beginning on Day 1, with sustained benefits through Day 28. Patients receiving the 3 mg dose experienced a mean improvement of 4.8 percentage points in left ventricular ejection fraction, compared with 1.6 percentage points in the placebo group, suggesting clinically meaningful improvements in cardiac performance.
Publication Supports Advancement of AC01 into Phase 2b Development
AnaCardio stated that publication in The Lancet provides strong independent scientific validation for the company’s innovative approach to treating heart failure. The company believes AC01’s unique calcium-sensitising mechanism differentiates it from conventional inotropic therapies by enhancing cardiac contractility without introducing significant cardiovascular safety concerns. Company leadership emphasized that the findings reinforce confidence in AC01’s potential to become the first approved oral therapy specifically designed to improve cardiac contractility in HFrEF patients. Clinical experts involved in the study noted that improvements observed in cardiac output, ventricular function, and heart structure, combined with the favorable safety profile, establish a solid foundation for larger late-stage studies. AnaCardio has also received positive scientific advice from both the U.S. FDA and the European Medicines Agency (EMA) regarding its development strategy and is preparing to rapidly advance AC01 into Phase 2b clinical development.
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AC01 Represents a Novel Therapeutic Strategy for Heart Failure
Heart failure remains one of the leading causes of hospitalization and mortality worldwide despite advances in standard medical treatment. AnaCardio believes AC01 has the potential to address a significant unmet medical need by providing a first-in-class oral calcium-sensitising contractile therapy capable of improving heart function while maintaining a favorable safety profile. Developed from research originating at Karolinska Institutet, AC01 is a ghrelin mimetic small molecule designed to improve myocardial contractility through a differentiated biological mechanism. With more than USD 40 million raised to support development, the Stockholm-based company is focused on advancing AC01 into Phase 2b clinical trials with the goal of delivering a novel long-term treatment option capable of improving survival, reducing hospitalizations, and enhancing quality of life for patients living with heart failure with reduced ejection fraction.
Source: AnaCardio press release
