Cereno’s CS1 Shows Promising Results in Reducing PAH Risks in Phase 2 Trial

Trial Success:
Cereno Scientific's CS1, an HDAC inhibitor, has shown significant promise in reducing risks associated with Pulmonary Arterial Hypertension (PAH) in a Phase 2 trial. The drug improved the risk score of 43% of patients.

Mechanism of Action:
CS1 is a controlled-release formulation of valproic acid, which exhibits a multi-targeted mode of action including pulmonary pressure reduction, reversal of vascular remodeling, anti-inflammatory, anti-fibrotic, and anti-thrombotic effects.

Clinical Trial Design:
The Phase 2 trial is a prospective, randomized, parallel group, open-label treatment, blinded endpoint multicenter study evaluating the safety, tolerability, and exploratory efficacy of CS1 in PAH patients.

Positive Initial Findings:
A patient case study showed a 30% reduction in pulmonary pressure and a 20% increase in cardiac output after 12 weeks of treatment with CS1. The patient’s functional status improved from NYHA/WHO functional class II to I.

Expanded Access Program:
Since January 30, 2024, CS1 has been approved by the FDA for Expanded Access, allowing patients with PAH to access the drug under a formal FDA-approved protocol.

Data Quality Control Review:
A review of data from the first 16 patients using the CardioMEMS HF System showed satisfactory data quality and clinically meaningful reductions in pulmonary pressure.

Future Plans:
The trial's results will inform the design of future Phase 3 studies with CS1, aiming to establish it as a disease-modifying treatment for PAH.