CS1 is a HDAC, Histone Deacetylase inhibitor, that works through epigenetic modulation and is being developed by Cereno Scientific as a disease modifying treatment for pulmonary arterial hypertension. CS1 targets the root cause of the disease, aiming to reverse the pathological vascular remodeling of the small lung arteries. The Phase IIa trial evaluated the safety, tolerability, pharmacokinetics, and exploratory efficacy of CS1 on top of standard-of-care in patients with pulmonary arterial hypertension.
Cereno Scientific had previously received approval from the US Food and Drug Administration (FDA) for expanded access of CS1 in the Phase II trial, which allowed patients who completed the Phase II to continue CS1 therapy. Positive outcomes from the study prompted the FDA approval, with the expanded access program providing an opportunity for long-term safety and efficacy data collection (see our article about this here)
Read more on the company press release at this link