Cereno Scientific (CRNO) Study Result summary

Study design and objectives

• Phase IIa CS1-003 trial in pulmonary arterial hypertension (PAH) evaluated safety, tolerability, pharmacokinetics, and exploratory efficacy 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 treatment period; doses were pooled due to small group sizes and PK/PD data.

• The primary endpoint was safety and tolerability; secondary/exploratory endpoints included REVEAL Risk Score, functional class, and hemodynamic parameters.

• CardioMEMS technology enabled continuous, objective hemodynamic monitoring, including mean pulmonary arterial pressure (mPAP).

• 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, or mortality.

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

• CS1 was well tolerated across all dose groups, with no serious treatment-related adverse events.

• TEAEs occurred in 76% of patients, but only 8% led 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 reduced mortality risk.

• 33% improved functional class; 86% improved or remained stable, reflecting better daily activity.

• 67% had sustained reduction in mean pulmonary artery pressure, associated with lower mortality.

• 24–25% of patients showed remarkable reductions in pulmonary vascular resistance (PVR), with robust increases in right ventricular stroke volume, mostly in the low-dose group.

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