Additional data strengthens Cereno’s CS1 in PAH
Following the promising phase IIa results for its drug candidate CS1 in pulmonary arterial hypertension (PAH) in the fall of 2024, Cereno Scientific now delivers additional data that further reinforce the candidate’s potential. The new data shows further signs of disease-modifying effects in a disease with limited treatment options.
Cereno Scientific is advancing the drug candidates CS1, CS014, and CS585 in three parallel development programs targeting rare cardiovascular and pulmonary diseases.
CS1 is being developed as a treatment for PAH, a progressive and life-threatening disease in which the pulmonary arteries and the right ventricle gradually deteriorate due to pathological vascular changes. The need for new treatment options is urgent, and CS1 – an epigenetically modulating HDAC inhibitor – has previously demonstrated promising phase IIa topline results regarding safety, tolerability, and efficacy parameters. Now, with the full phase IIa data at hand, an even clearer picture of CS1’s potential emerges; the candidate not only has the potential to slow disease progression but also to profoundly improve patients’ lives.
New data highlights CS1’s disease-modifying effects
The 12-week study included 25 PAH patients, of whom 21 were evaluated for efficacy parameters. The topline results, announced in the fall of 2024, showed that CS1 reduced risk scores according to REVEAL 2.0 – an important measure for predicting clinical worsening and mortality – in 43 percent of patients, while 71 percent either improved or remained stable. At the same time, the NYHA functional class, which assesses patients’ physical capacity, improved in 33 percent, and a remarkable 86 percent reported either improvement or stability.
An additional analysis conducted after the topline results were reported continues to highlight the candidate’s potential. A significant improvement in right ventricular function, measured as Global Longitudinal Strain (RVGLS), was observed. RVGLS is a sensitive measure of right heart function and treatment response, as well as a predictive factor for right ventricular remodelling in early disease stages and future mortality. In connection with this, a stabilisation or improvement in tricuspid regurgitation – a condition where leakage in the tricuspid valve increases pressure in the right ventricle – was also noted. Additionally, a marked improvement in pulmonary vascular resistance was observed in a subgroup of patients at an early disease stage.
Gradual improvement and quality of life
Furthermore, the analysis shows that CS1’s effects grow over time. Both REVEAL 2.0 risk scores and NYHA functional class improved gradually throughout the study, suggesting that the effects may become even more pronounced with longer treatment durations. Patients themselves also reported a significant improvement in quality of life, as measured by validated tools such as PAH-SYMPACT and the Minnesota Living with Heart Failure Questionnaire.
The road ahead – a phase IIb study
With the phase IIa study completed, Cereno Scientific is now setting its sights on the next phase. A larger, placebo-controlled phase IIb study is planned to further confirm and expand on these findings. Based on this, the company has initiated a Type C meeting with the FDA to define the next development steps for CS1. Additionally, preparations are underway to publish these scientific findings.
Rahul Agrawal, CMO and Head of R&D at Cereno Scientific, commented on the results:
“We are pleased to have completed the Clinical Study Report, successfully concluding the Phase IIa trial and enhancing our understanding of CS1’s reverse remodeling effects. PAH is a progressive and fatal disease with pathological changes to the pulmonary vasculature and the right-heart. Despite the trial’s short duration, CS1 showed improvement of right-ventricular function signaling a disease-modifying effect by halting disease progression or through the reversal of pathological vascular remodeling.”
CEO Sten R. Sörensen commented on the progress with a positive outlook on the future:
“All signs are pointing toward our epigenetic modulating HDACi CS1 having a real potential to be a disease-modifying game-changer in the treatment of PAH, a fatal rare disease with high unmet need. We are excited for the next part of the journey to further evaluate CS1’s transformative potential as a treatment for PAH […] The Cereno Team is also well underway working toward the next key clinical development step for CS1. Activities include discussing regulatory strategies, exploring and engaging various service providers supporting us operationally, and working with the scientific community to further establish our company at key scientific meetings. We are further continuing preparations for an important interaction with the FDA in a Type C meeting this spring.”
On March 4, 2025, the company will hold a webcast to present the full phase IIa results in detail.
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