AnaCardio's AC01 Demonstrates Progress in Heart Failure Clinical Trials

Key Insights

• AnaCardio completed the AC01-FE study in the US, finding AC01 safe and well-tolerated in healthy volunteers under fed and fasted conditions.
• The phase 1b portion of the GOAL-HF1 study, evaluating AC01 in HFrEF patients, has been successfully completed, showing promising initial results.
• The GOAL-HF1 study is a randomized, double-blinded, placebo-controlled trial assessing AC01's safety, tolerability, pharmacokinetics, and pharmacodynamic effects.

AnaCardio, a portfolio company of Karolinska Development, has announced advancements in the clinical development of its drug candidate AC01 for heart failure. The AC01-FE study, conducted in the US, and the initial phase 1b of the GOAL-HF1 study have both yielded positive results, marking significant steps forward in the evaluation of this potential new treatment.

AC01-FE Study Results

The AC01-FE study, which enrolled 24 healthy volunteers, assessed the impact of food on the pharmacokinetics of AC01. The results indicated that AC01 was safe and well-tolerated under both fed and fasted conditions. This is an important finding for the drug's future development, as it suggests that AC01 can be administered without specific dietary restrictions.

GOAL-HF1 Phase 1b Completion

The GOAL-HF1 study is a phase 1b/2a clinical trial designed to evaluate AC01 in patients with heart failure and reduced ejection fraction (HFrEF). The study employs a randomized, double-blinded, placebo-controlled design. The primary objectives include assessing the safety profile, tolerability, and pharmacokinetics of AC01, as well as its effects on various pharmacodynamic parameters. The phase 1b portion of the study has been successfully completed, with 32 patients treated across four ascending dose cohorts of AC01 or placebo over a 7-day period.

Mechanism of Action and Future Plans

AC01 is a small molecule drug candidate that mimics the action of ghrelin, a peptide hormone. Ghrelin has demonstrated positive effects on the heart's contractility and increases the volume of blood ejected from the heart. "We are happy to learn about the milestones achieved in our portfolio company AnaCardio’s clinical development program of the drug candidate AC01, a new potential treatment of heart failure with unique properties compared to currently available medications," said Viktor Drvota, CEO of Karolinska Development.

The second part of the GOAL-HF1 study, phase 2a, is anticipated to commence in Q1 2025. This phase will further evaluate the efficacy and safety of AC01 in a larger patient population.