CERENO SCIENTIFIC AB Cereno Scientific announced that one new additional patient have been enrolled in the study, one additional patient has been randomized on CS1 drug and one additional patient has completed the study. This update serves as the latest progress update for the Phase II study of drug candidate CS1 in rare disease pulmonary arterial hypertension (PAH). The study is set to be completed by third quarter 2024.
The Phase II study of CS1 in the rare disease PAH is actively running at 10 specialist clinics in the US, with the latest clinic activated in late December. One additional new clinic is currently in late-stage start-up process. Since the last phase II study progress update communicated on February 21, 2024, one new additional patient have now been enrolled in the study, one additional patient has now been randomized to CS1 drug in the study and one additional patient has now completed the study.
Enrolled patients are scheduled to receive CardioMEMS HF System implantation within two weeks from enrollment and are, following successful implantation of CardioMEMS and up to 6 weeks baseline evaluation, randomized to CS1 drug therapy on their next scheduled visit. The next step for the recently enrolled patient is to undergo CardioMEMS implantation. 25 patients have already received CardioMEMS implantation, of which 24 patients have already been randomized to CS1 drug therapy and one is still in baseline period prior to randomization.
To date 20 patients have completed the study. EAP approval follows positive findings from the PAH study with CS1 drug therapy reported since 2023: Cereno Scientific has during the last few months reported positive findings from the ongoing study suggesting a potential positive effect of drug candidate CS1 in patients with the severe rare disease PAH. Study completion and top-line results are expected during third quarter of 2024.
Remarkable Patient Case with CS1 reported June 2023: First, a patient case study performed on the first patient having completed the study at a specific clinic showed remarkable efficacy data. In 12 weeks of treatment with CS1, the patient showed a 30% reduction in pulmonary 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.
Data Quality Control Review reported October 13th 2023: In addition, Cereno reported in October 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. Efficacy findings showed a clinically meaningful reduction of pulmonary pressure in several patients, included in the data quality control, of a similar or greater magnitude as in the Patient Case.
EAP for CS1 Approved Jan 30th 2024: Further, the Expanded Access Program (EAP) (?Compassionate Use?) approved by the FDA on 31st January 2024 is progressing according to plan, propelled by investigator and patient interest. Investigators indicate that close to two-thirds of the patients, having completed the study or are currently on therapy, have been judged to be interested in continued access to CS1 following study completion. Currently site-specific and IRB approvals are being progressed.
The 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. The EAP will thus allow Cereno to gather further documentation of CS1 use in patients suffering from PAH, which will help in discussions with regulatory authorities and to design Phase IIb/III pivotal study with CS1.
