CS014 is a new chemical entity that acts through HDAC inhibition and has demonstrated disease-modifying potential in preclinical studies. The phase I study consists of two parts: a single ascending dose (SAD) and a multiple ascending dose (MAD). The SAD part has now been completed and confirmed an acceptable safety profile.
“We are pleased with the progress of the CS014 Phase I trial and eagerly anticipate its completion in mid-2025. The successful completion of the single ascending dose (SAD) part provides a strong initial validation of HDACi CS014s favorable safety profile,” said Rahul Agrawal, CMO & Head of R&D of Cereno Scientific.
Potential to transform IPF treatment
HDAC inhibitors have shown positive effects in preclinical studies on the reversal of pathological pulmonary vascular changes as well as antifibrotic and antithrombotic properties. These mechanisms are central to IPF and may help address important medical needs.
“We believe that our novel HDACi CS014 has the potential to become an important treatment meeting high unmet clinical needs in the rare disease IPF. There is a void in the market for safe and well-tolerated novel drugs with a profile addressing the underlying pathophysiology of the IPF disease and its progression. CS014 aims to be a safe, well-tolerated oral drug with disease-modifying capacity and, as such, targets a large market potential,” said Sten R. Sörensen, CEO of Cereno Scientific.
Study design and next steps
The phase I study is being conducted by the contract research organisation CTC in Uppsala and involves a total of 48 healthy participants. It is designed to evaluate safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD). So far, 30 participants have received single ascending doses, which have demonstrated a favorable safety profile. The dose-escalation part of the study is ongoing as planned and is expected to be completed by mid-2025, after which data analysis and topline results will be presented.
