Cereno Scientific announced today that its innovative drug candidate CS014 has successfully cleared the first hurdle in human testing, completing the single ascending dose (SAD) portion of its Phase I trial without safety concerns. The drug, developed for idiopathic pulmonary fibrosis (IPF), represents a novel approach as a histone deacetylase (HDAC) inhibitor with disease-modifying potential.
The initial phase of the trial, conducted by CTC in Uppsala, evaluated the safety, tolerability, and pharmacokinetics of CS014 in 30 healthy volunteers. The results demonstrated an acceptable safety profile, supporting further clinical development of the compound.
Innovative Mechanism of Action
CS014 functions as an epigenetic modulator through HDAC inhibition, with preclinical studies revealing strong vascular remodeling effects. The drug has shown promising results in animal models, including the reversal of pathological remodeling of pulmonary vessels and anti-fibrotic effects in pulmonary arterial hypertension (PAH) models.
"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," stated Sten R. Sörensen, CEO of Cereno Scientific. "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."
Trial Design and Future Outlook
The Phase I trial follows an open-label, first-in-man design, structured in two parts:
- Single ascending dose (SAD) - now completed
- Multiple ascending dose (MAD) - currently ongoing
Dr. Rahul Agrawal, CMO & Head of R&D at Cereno Scientific, expressed optimism about the progress: "The successful completion of the single ascending dose part provides a strong initial validation of HDACi CS014's favorable safety profile."
The complete Phase I program will involve approximately 48 subjects total, with final results expected by mid-2025. The trial aims to comprehensively evaluate the drug's safety, tolerability, pharmacokinetics, and pharmacodynamics through oral administration.
Addressing Unmet Medical Needs
CS014's development addresses significant gaps in current IPF treatment options. Preclinical data suggests the drug can:
- Reverse fibrosis in IPF models
- Prevent pathological remodeling of pulmonary vessels
- Regulate platelet activity and clot stability without increasing bleeding risk
The drug's oral administration route and potential disease-modifying capabilities position it as a promising candidate in the IPF treatment landscape, where current therapeutic options remain limited.
