Cereno Scientific has submitted the clinical trial protocol for the planned global Phase IIb trial of its lead drug candidate CS1 to the U.S. Food and Drug Administration (FDA). CS1 is an oral histone deacetylase inhibitor (HDACi) intended as a disease-modifying therapy for pulmonary arterial hypertension (PAH), working through epigenetic modulation to target underlying mechanisms such as vascular remodeling, inflammation, and fibrosis. The proposed trial builds on encouraging Phase IIa data that showed favorable safety, tolerability, and signals of efficacy—including reverse vascular remodeling and improvement in right-heart function.
The Phase IIb study will be randomized, placebo-controlled, and conducted with a global CRO (Contract Research Organization). Cereno expects the U.S. Food and Drug Administration to complete its 30-day review and anticipates starting the trial in the first half of 2026. The move marks a key step In advancing CS1 toward potentially becoming a first-in-class, epigenetic therapy for pulmonary arterial hypertension addressing unmet needs beyond symptom management.
Read more at this link on the Cereno Scientific website
Summary by Deger Kesimoglu, volunteer for the Alliance for Pulmonary Hypertension
See also our previous article here