Cereno Scientific’s lead drug candidate, CS1, is an HDAC inhibitor that acts through epigenetic modulation. CS1 is being developed as a first-in-class treatment for the rare disease pulmonary arterial hypertension (PAH).
The company has completed a phase IIa study of CS1 in PAH, demonstrating a favorable safety profile and signals of disease-modifying effects. In the first quarter, additional data from the phase IIa study were shared following completion of the clinical study report. The results showed sustained improvements in REVEAL 2.0 risk scores, functional capacity, and patient-reported quality of life – early indicators of long-term therapeutic benefit. Further data analysis also revealed early signs of improved right ventricular function, which is a key predictive factor for survival in PAH.
Focus on Expanded Access Program and phase IIb preparations
CS1 has been approved by the FDA for an Expanded Access Program (EAP) as an extension of the phase IIa study, providing continued access to CS1 for patients who completed the trial. The EAP has progressed according to plan, and in February, a sub-study was initiated using Fluidda’s imaging technology to evaluate structural changes in the pulmonary vessels during long-term treatment with CS1.
Preparations are also underway for the next clinical development phase. In April, a Type C meeting was held with the FDA, resulting in alignment on the design of an upcoming phase IIb study – a key step toward broader clinical validation of CS1.
Milestone reached with CS014
In parallel, Cereno Scientific has made clinical progress with its second HDAC inhibitor, CS014. The first part of the phase I study was completed in February without any safety concerns. The second part (multiple ascending dose, MAD) proceeded as planned.
On April 16, the company announced that the entire phase I study had been completed, and top-line results are expected in June 2025. According to Cereno Scientific, CS014 has a multimodal mechanism of action with the potential to address underlying pathophysiology in rare cardiovascular and pulmonary diseases with significant unmet medical needs. The initial focus is the rare disease idiopathic pulmonary fibrosis (IPF).
Strengthened patent protection
During the first quarter, the intellectual property protection for CS1 was further strengthened. On March 17, a new U.S. patent was granted for CS1’s second patent family. Two additional patent applications have been filed to expand the candidate’s IP protection. These efforts enhance the prospects for long-term market exclusivity for CS1 in PAH, potentially extending to 2045.
Cereno’s cash position
According to the Q1 report, the company primarily invested in the ongoing Expanded Access Program for CS1, the phase I study evaluating the safety and tolerability of CS014, toxicology studies in preparation for phase II of CS014, and the preclinical development of its third candidate, CS585.
Operating cash flow for the quarter was SEK -24.3 million, and the company’s cash balance at the end of the quarter amounted to SEK 77.0 million.
– With two HDACi programs now in clinical development, a preclinical candidate advancing in its program, a growing IP portfolio, and strong scientific and regulatory momentum, Cereno Scientific is well-positioned to continue its trajectory toward becoming a leader in epigenetically modulating therapies for rare cardiovascular and pulmonary diseases, CEO Sten R. Sörensen concludes in the report.
