AnaCardio and Helsinn receive US USPTO patent issuance for AC01 heart failure treatment

AnaCardio AB (Sweden), a clinical-stage biopharmaceutical company developing contractile agents for heart failure, and its partner Helsinn Healthcare SA (Switzerland) announced the issuance of US Patent No. 12,569,476 by the United States Patent and Trademark Office (USPTO). The AnaCardio AC01 patent covers the use of AC01, a first-in-class calcium sensitizing inotrope and oral ghrelin mimetic small molecule, for the treatment of heart failure with reduced ejection fraction (HFrEF). This dual mechanism targets cardiac contractile dysfunction through enhanced myofilament calcium sensitivity while simultaneously engaging the ghrelin receptor pathway, which has been linked to cardioprotective and metabolic effects in failing hearts.

AnaCardio US patent extends AC01 exclusivity into the 2040s

The biopharmaceutical patent provides protection in the United States until June 2, 2042, with potential patent term extensions beyond that date. The patent is co-owned by AnaCardio AB and Helsinn Healthcare SA. The Helsinn Healthcare AnaCardio partnership, which originated with Helsinn's out-licensing of the compound to AnaCardio, now benefits from what the companies describe as a layered intellectual property position supporting AC01 well into the 2040s. This biopharmaceutical patent 2040s timeline positions the asset with a lengthy exclusivity window in the world's largest pharmaceutical market, a consideration that will factor into any future licensing, partnership, or commercialization decisions.

AC01 contractile agent: mechanism and clinical development

AC01 functions through two pharmacological pathways. As a calcium sensitizer, the AC01 contractile agent increases the responsiveness of cardiac myofilaments to intracellular calcium, improving the force of heart contractions without raising intracellular calcium concentrations. This distinction is relevant because traditional inotropes that increase calcium influx carry risks of arrhythmia and elevated myocardial oxygen demand. As an oral ghrelin mimetic, AC01 activates the growth hormone secretagogue receptor (GHS-R1a), which is expressed in cardiovascular tissue. Ghrelin receptor activation has been associated with anti-apoptotic signaling, improved cardiac remodeling, and metabolic regulation in preclinical heart failure models. The combination of these two mechanisms in a single oral small molecule differentiates AC01 from existing agents in the HFrEF treatment landscape.

AnaCardio completed a Phase Ib/IIa trial (NCT information via AnaCardio pipeline) enrolling 58 patients with HFrEF across sites in Italy and the Netherlands. The company disclosed positive topline results, reporting that AC01 demonstrated a favorable safety and tolerability profile alongside exploratory efficacy signals described as rapid and sustained improvements in cardiac function and hemodynamics over 28 days. A second study enrolling 62 patients across the United Kingdom, Italy, and Sweden (trial record) has also been conducted. As of late 2025, AnaCardio stated plans to advance AC01 into Phase IIb development. Specific clinical results for individual endpoints and detailed adverse event data have not yet appeared in peer-reviewed publications.

Context and competitive landscape

The AC01 heart failure treatment enters a field with several mechanistically relevant comparators. Levosimendan, marketed by Orion Pharma (Finland), is the most established calcium sensitizer in clinical use. Approved in multiple countries for acute decompensated heart failure, levosimendan enhances myofilament calcium sensitivity and produces vasodilation through potassium channel activation. It is administered intravenously, and ongoing trials are evaluating repetitive infusion regimens in advanced chronic heart failure (NCT record). AC01's oral formulation and dual mechanism represent a differentiation strategy relative to levosimendan's IV-only route and single-pathway activity.

Omecamtiv mecarbil, developed by Cytokinetics (US) in partnership with Amgen (US), operates through a distinct contractility mechanism as a cardiac myosin activator. Rather than sensitizing myofilaments to calcium, it directly targets cardiac myosin ATPase to increase the duration and magnitude of systolic contraction. Omecamtiv mecarbil completed the Phase III GALACTIC-HF trial (NCT01920711) and has been the subject of regulatory review discussions, though it has not received US FDA approval to date. Its mechanism does not overlap with ghrelin signaling, distinguishing it from AC01's pharmacological profile.

In the broader HFrEF treatment landscape, teplizumab-mzwv (Tzield, Provention Bio/Sanofi) serves as a reference point for antigen-specific immune modulation in a different disease context, but the functional benchmarks most relevant to AC01 are the established guideline-directed medical therapies. Sacubitril/valsartan (Entresto, Novartis, Switzerland), an angiotensin receptor-neprilysin inhibitor, and dapagliflozin (Farxiga, AstraZeneca, UK/Sweden), an SGLT2 inhibitor, are both approved by the US FDA for HFrEF and represent the current standard of care. These agents address neurohormonal dysregulation and volume overload rather than contractile dysfunction directly. AC01's strategy of targeting the contractile apparatus through calcium sensitization and ghrelin receptor engagement positions it as a potential complement or alternative for patients with persistent contractile impairment despite optimized background therapy, though this hypothesis remains untested in comparative trials.

The issuance of the AnaCardio AC01 patent secures a defined exclusivity position as the company prepares to enter Phase IIb development. Whether the dual-mechanism approach translates into clinical differentiation against these established and emerging competitors will depend on data from later-stage trials that have not yet been initiated.

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