Cereno Scientific’s Phase II trial with CS1 in Pulmonary Arterial Hypertension is closing for recruitment and proceeding to topline
Cereno Scientific (Nasdaq First North: CRNO B), a pioneering biotech developing innovative treatments for rare and common cardiovascular disease, today announced that the Phase II trial with CS1 in the rare disease Pulmonary Arterial Hypertension (PAH), 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 Q3, 2024.
“We are very happy that we have reached this point in the development of CS1, and I am excited that we now are entering the next phase towards sharing topline results in Q3”, said Sten R. Sörensen, CEO, Cereno Scientific.
About CS1
The drug candidate CS1 is an HDAC inhibitor that works through epigenetic modulation, being developed as a treatment for the rare disease PAH. CS1 has the potential to be an effective, safe and disease-modifying drug. 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.
Remarkable Patient Case with CS1 reported June 2023
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
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
Since January 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.
The final results of the study may differ from the above findings, and they should not in any way be seen as a guarantee regarding the outcome and conclusions of the upcoming final Phase II study results.
For further information, please contact:
Henrik Westdahl, Director IR & Communications
Email: henrik.westdahl@cerenoscientific.com
Phone: +46 70-817 59 96
Sten R. Sörensen, CEO
Email: sten.sorensen@cerenoscientific.com
Phone: +46 73-374 03 74
This information is information that Cereno Scientific AB is obliged to make public pursuant to the EU Market Abuse Regulation. The information was submitted for publication, through the agency of the contact person set out above, at 08:30 (CET) on June 28, 2024.
About Cereno Scientific AB
Cereno Scientific develops innovative treatments for rare and common cardiovascular disease. The lead drug candidate, CS1, is an HDAC (histone deacetylase) inhibitor that acts as an epigenetic modulator with pressure-reducing, reverse-remodeling, anti-inflammatory, anti-fibrotic and anti-thrombotic properties. A Phase II trial is ongoing to evaluate CS1’s safety, tolerability, and efficacy in patients with the rare disease pulmonary arterial hypertension (PAH). A collaboration agreement with global healthcare company Abbott allows Cereno to use their cutting-edge technology CardioMEMS HF System in the trial. Two initiatives performed during the ongoing Phase II trial have shown positive findings suggesting the potential clinical benefit of CS1 in PAH patients. These initial findings are, however, not a guarantee of the final trial results that are expected in Q3 2024. Since January 2024, we are delighted that the FDA´s Expanded Access Program will enable patients with PAH, a serious life-threatening disease condition, to gain access to CS1 where no comparable alternative therapy options are available. Cereno’s pipeline comprises two additional programs in development through research collaborations with the University of Michigan. Investigational drug CS014 is an HDAC inhibitor in Phase I development as a treatment for arterial and venous thrombosis prevention. The innovative drug candidate represents a groundbreaking approach to antithrombotic treatment. CS014 is a new chemical entity with a multi-fold mechanism of action as an epigenetic modulator – regulating platelet activity, fibrinolysis, and clot stability for the prevention of thrombosis without an increased risk of bleeding as documented in preclinical trials. In June 2024, Cereno received approval from the EMA to conduct a first-in-human Phase I trial of CS014. Preclinical candidate CS585 is an oral, highly potent and selective prostacyclin (IP) receptor agonist that has demonstrated the potential to significantly improve disease mechanisms relevant to cardiovascular disease. While CS585 has not yet been assigned a specific indication for clinical development, preclinical data indicates that it could potentially be used in indications like Pulmonary Hypertension and thrombosis prevention without increased risk of bleeding. CS585 was in-licensed from the University of Michigan in 2023. The Company is headquartered in GoCo Health Innovation City, in Gothenburg, Sweden, and has a US subsidiary; Cereno Scientific Inc. Based in Kendall Square, Boston, Massachusetts, US. Cereno is listed on the Nasdaq First North (CRNO B). The Certified Advisor is Carnegie Investment Bank AB, CA@carnegie.se. More information is on www.cerenoscientific.com.