Novel HDACi CS014
CS014 is a new chemical entity, designed as a HDAC inhibitor with a multi-modal mechanism of action. By acting as an epigenetic modulator, CS014 could target the underlying pathophysiology of several rare cardiovascular and pulmonary diseases with significant unmet medical needs. The drug has successfully concluded a Phase I trial.
CS014 has the potential to reverse the fibrosis developing in IPF as shown in preclinical models
Mechanism of action and disease-modifying potential
CS014 employs a novel mechanism of action through epigenetic modulation, making it highly relevant for a variety of conditions, including idiopathic pulmonary fibrosis (IPF) and pulmonary arterial hypertension (PAH). In preclinical studies, CS014 has demonstrated the ability to reverse fibrosis and exhibit a dose-dependent beneficial effect on pulmonary pathological vascular remodeling, with a reduction in plexiform lesions, suggesting strong disease-modifying potential.
A therapy that directly targets thrombosis, which no currently approved or investigational treatment does, could be particularly valuable in diseases such as idiopathic pulmonary fibrosis (IPF) and pulmonary arterial hypertension (PAH), where vascular injury, abnormal clotting, and impaired blood flow are key drivers of disease progression.
In IPF, microvascular thrombosis exacerbates tissue remodeling and fibrosis. In PAH, thrombosis in the small pulmonary arteries contributes to elevated pulmonary pressure and right heart failure. By addressing the thrombotic component of these diseases, CS014 may slow disease progression, improve oxygenation, and enhance overall cardiopulmonary function.
Importantly, this mechanism of action may also have therapeutic relevance across a broader spectrum of cardiovascular and pulmonary diseases where thrombosis and vascular dysfunction play a central role.
Potential for treating rare cardiovascular and pulmonary diseases
Given its multi-modal mechanism of action, CS014 has the potential to address a broad range of cardiovascular and pulmonary diseases that currently lack effective disease-modifying therapies. The drug's ability to target fibrosis, vascular remodeling, and thrombosis positions it as a strong candidate for treating rare and life-threatening cardiovascular and pulmonary diseases.
Phase I trial: Safety and tolerability
An open-label Phase I trial was successfully concluded in April 2025. The Phase I trial evaluated safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of single and multiple ascending oral doses of CS014 in healthy volunteers. The trial was conducted in two parts: part one explored safety, tolerability and PK of single ascending oral doses (SAD) of CS014; part two explored safety, tolerability, PK, and PD following multiple ascending doses (MAD) of CS014, dosed for seven days. In total, 48 subjects were included in the trial, 30 in the SAD and 18 in the MAD part. The study was conducted in Sweden.
The first part (SAD) of the trial has successfully been completed with results showing that CS014 exhibited an acceptable safety profile supporting its potential for further clinical development.
Current status of CS014 development*
The top-line results of the Phase I trial are in preparation. After the last patient’s last visit in April 2025, a structured set of activities has taken place to close out the trial, analyze the data, and prepare for regulatory and strategic next steps. The trial’s top-line results are expected to be announced in mid-July 2025.
Simultaneously, preparations and studies are underway to enable the transition to a Phase II trial of CS014.
*as of June 2025.