Cereno Scientific announced that the Phase II trial with CS1 in the rare disease Pulmonary Arterial Hypertension, is closing for recruitment and proceeding to topline. Based on a recommendation from the Study Clinical Steering Committee, Cereno Scientific has decided to close patient recruitment to study CS1-003 by July 1st, 2024. The Study Clinical Steering Committee concluded that there is sufficient data for evaluating the next steps in development.

Topline results will be shared in third quarter, 2024. CS1's unique efficacy profile fits well with the pathogenetic mechanisms of PAH and is believed to be able to address today's major unmet need for better treatment alternatives. The aim of CS1's development is to offer improved quality of life and prolonged life with CS1 for patients with PAH.

Cereno Scientific has over the last year reported encouraging findings from the ongoing Phase II study suggesting a potential positive effect of drug candidate CS1 in patients with the severe rare disease PAH. A patient case study performed on the first patient having completed the study at one of the study centers showed remarkable efficacy data. After 12 weeks of treatment with CS1, the patient showed a 30% reduction in pulmonary arterial pressure and a 20% increase in cardiac output.

The patient's overall functional status was changed from NYHA/WHO functional class II to I at the end of the treatment period, meaning that the patient had next to normal functional physical capacity with CS1 added to stable conventional therapy. In addition to the data related to the effects of CS1 in the PAH patient, the case study indicated that using the CardioMEMS permits safe daily remote monitoring of pulmonary arterial (PA) pressure over time in patients with PAH, permitting assessment of medication effectiveness on an individual patient level. Data Quality Control Review reported October 13th, 2023 that a Data Quality Control Review (DQCR), of data obtained by the CardioMEMS HF System from the first 16 patients, was concluded with positive findings.

The data quality of the CardioMEMS measurements was found satisfactory with adherence to study protocol and with timely data transfers from the patient's home to the clinic. EAP for CS1 Approved Jan 30th, 2024, CS1 is approved by the FDA for Expanded Access, an extension of the ongoing Phase II trial evaluating CS1 in PAH. The Expanded Access Program (EAP) will provide Cereno with the opportunity to, under a formal FDA-approved protocol, collect safety and efficacy data from long-term exposure to CS1 in patients with PAH.

This initiative not only supports the treatment of PAH patients but also enables Cereno to gather additional CS1 usage documentation for regulatory discussions and Phase IIb/III pivotal study design planning. Currently site-specific contracts and IRB approvals are being progressed.