Cereno Scientific, a biotechnology company pioneering treatments for rare cardiovascular and pulmonary diseases, has announced positive topline results from its Phase 1 clinical trial of CS014, a novel histone deacetylase (HDAC) inhibitor being developed for idiopathic pulmonary fibrosis (IPF).

The Phase 1 trial was designed to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of CS014 in healthy volunteers. The study enrolled 48 participants who received single ascending doses (SAD) or multiple ascending doses (MAD) of the oral drug. CS014 demonstrated a favourable safety profile with no serious adverse events reported, and all treatment-related adverse events were mild, transient, and resolved without complications. Notably, all volunteers completed the study without early withdrawal.

CS014 is intended to target pulmonary vascular remodeling and fibrosis, key pathological processes in IPF and other rare pulmonary and cardiovascular diseases. Blood concentration levels achieved in the trial met or exceeded those predicted from preclinical data to have therapeutic effects. Supporting non-clinical studies using a Sugen/Hypoxia rat model showed CS014’s potential to reduce plexiform lesions associated with vascular remodeling.

Rahul Agrawal, chief medical officer and head of research and development at Cereno Scientific, commented: “CS014 was well tolerated at exposure levels expected to impact disease pathways in pulmonary fibrosis and vascular remodeling. These results, combined with promising preclinical data, reinforce our confidence in advancing to Phase 2 clinical trials.”

Cereno Scientific plans to initiate the Phase 2 trial in the first half of 2026, focusing on further evaluating CS014’s safety and efficacy in patients with IPF. The company views CS014 as a cornerstone of its HDAC inhibitor portfolio, which aims to deliver disease-modifying therapies across a range of rare diseases with significant unmet medical needs.

CEO Sten Sörensen added: “We are encouraged by these Phase 1 results and remain committed to pioneering innovative treatments that improve patient outcomes in rare cardiovascular and pulmonary conditions.”

Full Phase 1 data will be submitted for publication in a peer-reviewed journal, marking a key milestone in the development pathway of CS014 and Cereno Scientific’s broader pipeline.