Drug candidate CS014

Mechanism of action and disease-modifying potential

CS014 is an HDAC inhibitor, from the same drug platform as CS1, working through epigenetic modulation to target the underlying drivers of cardiopulmonary disease, including fibrosis, vascular remodeling, thrombosis and inflammation. This shared mechanism positions both assets as potentially disease-modifying rather than symptom-managing, addressing pathways that current approved therapies do not.

In preclinical studies, CS014 has demonstrated the ability to reverse fibrosis and reduce pulmonary vascular remodeling, suggesting strong disease-modifying potential. Uniquely, CS014 also targets thrombosis, a driver of disease progression in several cardiopulmonary conditions, without the bleeding risk typically associated with antithrombotic treatments.

A recent publication in the Journal of Thrombosis and Haemostasis provided the first peer-reviewed look at CS014's design and preclinical data. The paper confirmed that CS014 can effectively reduce harmful blood clotting without increasing the risk of bleeding, a combination that sets it apart from existing antithrombotic treatments. These findings add independent scientific validation to the rationale behind CS014's development and support its potential as a treatment candidate across a range of cardiovascular and pulmonary diseases.

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 serious 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 trial was conducted by CTC in Uppsala, Sweden.

Summary of the topline results from the Phase I trial:

• CS014 demonstrated favorable safety and tolerability in healthy volunteers.
• All 48 healthy volunteers completed the study; no early withdrawals or deaths were reported.
• No serious adverse events (SAEs) occurred.
• All treatment-related adverse events (AEs) reported were mild, transient, and fully recovered.
• CS014 achieved levels in the blood stream at and above those projected, based on non-clinical data, to be required for achieving maximal effects on reversal of pulmonary vascular remodeling and fibrosis.

These findings, combined with non-clinical data demonstrating a favorable impact on plexiform lesions in the Sugen/Hypoxia rat model, offer insights that support dose selection and support advancement into Phase II development.

The positive Phase I results, combined with strong non-clinical data, supports advancement into Phase II. Cereno Scientific is advancing preparations for a Phase IIb study in pulmonary hypertension associated with interstitial lung disease (PH-ILD) planned to start in Q1 2027.

Full results from the Phase I trial will be submitted for publication in a peer-reviewed scientific journal.

Updated April 2026.