Cereno Scientific’s innovative study design of the Phase II study of CS1 in PAH published in renowned journal Pulmonary Circulation
Cereno Scientific (Nasdaq First North: CRNO B), a company developing innovative treatments for common and rare cardiovascular disease, today announced that a research article on the innovative study design of the ongoing Phase II study of drug candidate CS1 in pulmonary arterial hypertension (PAH) has been published in the renowned medical journal Pulmonary Circulation.
The research article describes how the design of clinical trials is important to fully understand the mechanisms and benefits of new treatments. Numerous potential pharmacological treatments for PAH have shown promise in preclinical models but have failed to show clinical benefit in humans or have been associated with poor safety and tolerability. A novel feature of the CS1 Phase II study is that the patients are monitored for pulmonary hemodynamics and right heart ventricle function on a daily basis with the implanted CardioMEMS HF technology. Furthermore, in the study, which has safety and tolerability as primary objective, a wide array of functional, hemodynamic, and structural measures and biomarkers as well as patient-reported outcomes are being collected to provide an insight into not only the treatment effects but the disease progression itself. The research article concludes that CS1 represents a potential novel disease-modifying treatment for PAH.
“We are very proud of how our innovative study design in the Phase II study is at the forefront of drug development in the debilitating rare disease PAH. The study was designed in collaboration with the key thought leaders in our Clinical Steering Committee, who collectively represent extensive experience in trial design and PAH, as well as with our collaborative study partner Abbott who provides the cutting-edge measurement technology CardioMEMS HF System in the study. Combined, we have ensured that our drug candidate CS1 has the possibility to document safety and tolerability and indicate clinical utility and patient benefit in this Phase II study, ” said Sten R. Sörensen, CEO at Cereno Scientific.
Access article: Benza RL, Adamson PB, Bhatt DL, Frick F, Olsson G, Bergh N, Dahlöf B. CS1, a controlled-release formulation of valproic acid, for the treatment of patients with pulmonary arterial hypertension: Rationale and design of a Phase 2 clinical trial. Pulm Circ. 2024; 14:e12323. https://doi.org/10.1002/pul2.12323
Pulmonary Circulation, an official journal of the Pulmonary Vascular Research Institute (PVRI), is a peer-reviewed medical research journal that publishes original research articles, review articles, case reports, guidelines and consensus articles exclusively in the fields of pulmonary circulation and pulmonary vascular disease. The journal focuses on increasing survival rates for pulmonary hypertension and other pulmonary vascular diseases worldwide, and developing new therapeutic approaches for the diseases.
The Clinical Steering Committee for the Phase II study of CS1 includes R. Benza, System Director of Pulmonary Hypertension at Mount Sinai Icahn School of Medicine, New York City; and Principal Investigator of the Phase II study of CS1; D. Bhatt, Director of Mount Sinai Heart and the Dr. Valentin Fuster Professor of Cardiovascular Medicine at the Icahn School of Medicine at Mount Sinai Health System; G. Olsson, MD & PhD in Medical Sciences, Karolinska Institute; P. Adamson, Divisional Vice President and Chief Medical Officer of the Heart Failure Division, Abbott, and collaborative partner in the Phase II study of CS1; B. Dahlöf, Chief Medical Officer, Cereno; and Fredrik Frick, Head of Clinical Operations, Cereno.
For further information, please contact:
Tove Bergenholt, Director IR & Communications
Email: tove.bergenholt@cerenoscientific.com
Phone: +46 732-366 246
Sten R. Sörensen, CEO
Email: sten.sorensen@cerenoscientific.com
Phone: +46 73-374 03 74
About the Phase II study of CS1
The Phase II study of CS1 in the rare disease pulmonary arterial hypertension (PAH) is actively recruiting patients at 10 specialist clinics in the US, with one additional new clinic currently in late-stage start-up process. In 2023, the company reported positive findings from the ongoing study suggesting a potential positive effect of drug candidate CS1 in patients with the severe rare disease PAH. First, a patient case study performed on the first patient who had 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 she had next to normal functional physical capacity with CS1. In addition, Cereno reported in October 2023 that a Data Quality Control Review (DQCR) was concluded with positive findings. The data quality of the CardioMEMS HF System (Abbott Inc.) 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. The initial findings are, however, not a guarantee of the final study result. The review included data obtained by the CardioMEMS HF System from the first 16 patients enrolled in the study and the reported findings can be read in full in a previous announcement. A request for expanded access to CS1 (also called “compassionate use”) was submitted to the FDA on January 3, 2024, upon inquiry from investigators in the study. The study is designed to randomize 30 PAH patients and the top-line result of the Phase II study is estimated to be reported in Q2 2024.
About Cereno Scientific AB
Cereno Scientific develops innovative treatments for common and rare cardiovascular disease. The lead drug candidate, CS1, is a 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 study 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 study. Two initiatives performed during the ongoing Phase II study 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 study results that are expected in Q2 2024. After requests by investigators in the Phase II study, a “compassionate use” application for CS1 is currently being pursued. Cereno also has two promising preclinical drug candidates in development through research collaborations with the University of Michigan. Investigational drug CS014 is a HDAC inhibitor in development as a treatment for arterial and venous thrombosis prevention. The innovative drug candidate represents a groundbreaking approach to antithrombotic treatment potentially without the associated increased risk of bleeding in humans. 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 increased risk of bleeding as documented in preclinical studies. Drug candidate CS585 is a prostacyclin receptor agonist that has been documented in several preclinical studies to target the IP receptor for prevention of thrombosis without increased risk of bleeding, which also has been recognized in the medical community. CS585 was in-licensed from the University of Michigan in 2023. The company is headquartered in Gothenburg, Sweden, and has a US subsidiary Cereno Scientific Inc. based in Kendall Square in Boston, Massachusetts, US. Cereno is listed on the Nasdaq First North (CRNO B). More information on www.cerenoscientific.com.