Medicine

Experimental Ghrelin Drug Shows Promise for Treating Heart Failure Patients

How the science connects

Heart failureEjection fractionGhrelin

AI Insight

This phase 1b/2a randomized, double-blind, placebo-controlled trial evaluated AC01, an oral ghrelin receptor agonist, in patients with heart failure with reduced ejection fraction (HFrEF). Over a 28-day treatment period, AC01 demonstrated an acceptable safety profile and was well tolerated, with no major adverse events identified. The study provides preliminary evidence supporting the potential therapeutic use of ghrelin receptor agonists in HFrEF management.


Heart failure with reduced ejection fraction affects millions globally and requires new therapeutic approaches beyond current standard treatments. This early-phase trial suggests that targeting the ghrelin receptor pathway through oral medication could represent a novel treatment avenue, warranting larger clinical trials to establish efficacy and long-term safety.


Understand the Science

Heart failure 29 articles Explore Concept → Ejection fraction Concept coming soon Ghrelin Concept coming soon

In patients with HFrEF, AC01 over 28 days appeared safe and well tolerated, and no major harms were identified in this early-phase study. These findings support further investigations of AC01 in larger studies.

Source: [Articles] Safety, pharmacokinetics, and exploratory efficacy of the oral ghrelin receptor agonist AC01 in heart failure with reduced ejection fraction (GOAL-HF1): a randomised, double-blind, placebo-controlled, phase 1b/2a study