Cereno Scientific (CRNO) Study Result summary

Study design and objectives

• Phase IIa trial of CS1 (CS1-003) in pulmonary arterial hypertension (PAH) focused on safety, tolerability, pharmacokinetics, and exploratory efficacy endpoints at 10 US sites, with 25 patients randomized and 21 completing treatment.

• Patients were stable on standard of care and randomized to three CS1 dose levels (480 mg, 960 mg, 1920 mg) for a 12-week period; data were pooled due to small group sizes and similar PK/PD profiles.

• CardioMEMS technology enabled continuous, non-invasive hemodynamic monitoring throughout the study.

• The study was not powered for statistical significance in efficacy but aimed to explore clinical signals and inform future pivotal trials.

• Recruitment was stopped early after sufficient data was collected for next development steps.

Key safety and tolerability findings

• The primary endpoint of safety and tolerability was met, with no CS1-related serious adverse events, hospitalizations, mortality, or significant lab changes.

• No clinically significant changes in liver function, platelets, or bleeding events were reported.

• CS1 was well tolerated across all dose groups, with TEAEs in 76% of patients but only 8% leading to discontinuation.

• Most remarkable responders were in the low-dose group.

Efficacy and clinical impact

• 43% of patients improved REVEAL Risk Score; 71% improved or remained stable, indicating a potential reduction in 12-month mortality.

• 33% improved functional class; 86% improved or remained stable, despite PAH being a progressive disease.

• 67% had sustained reduction in mean pulmonary arterial pressure (mPAP), with reductions of 2.5–5 mmHg linked to lower mortality.

• 24–25% of patients showed >30% reduction in pulmonary vascular resistance (PVR), mostly in the low-dose group, with associated increases in right ventricular stroke volume (>10 mL).

• Efficacy data and preclinical results support CS1’s potential to reverse pathological vascular remodeling in PAH.