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Cereno Scientific's FDA Clearance for CS1

Cereno Scientific, a pioneering biotech company focusing on innovative treatments for rare cardiovascular and pulmonary diseases, recently announced a significant milestone in the development of its lead drug candidate, CS1. On December 8, 2025, the U.S. Food and Drug Administration (FDA) granted the company clearance to initiate a Phase IIb trial aimed at treating pulmonary arterial hypertension (PAH). This approval marks a critical step forward in the quest to enhance and extend the lives of individuals affected by this serious condition.

Understanding Pulmonary Arterial Hypertension (PAH)

PAH is a rare and progressive disease that poses considerable risks to patients, significantly increasing blood pressure in the pulmonary arteries. This condition can lead to right heart failure and, if left untreated, results in premature death. Current therapies primarily manage symptoms without modifying the disease’s progression, highlighting the urgent need for more effective treatments.

Cereno Scientific's CS1 is an orally administered histone deacetylase inhibitor (HDACi) with promising potential as a disease-modifying therapy for PAH. The FDA's decision to approve the Phase IIb trial follows promising results from earlier Phase IIa studies, which indicated favorable safety, tolerability, and encouraging signals related to disease modification. CS1 has also received Orphan Drug Designation and Fast Track designation, signifying its potential to address a pressing medical need.

The Trial Design

The Phase IIb trial, officially titled "A Phase 2b, Double-Blind, Randomized, Placebo-Controlled, Dose-Finding Study, to Compare the Efficacy and Safety/Tolerability of CS1 Versus Placebo When Added to Standard of Care for the Treatment of Pulmonary Arterial Hypertension (PAH)," will enroll about 126 stable patients who are currently receiving standard treatment for PAH.

Spanning across 10–12 countries, the trial will be designed to assess the optimal dose of CS1 for future Phase III trials. Participants will be randomized to receive either CS1 at one of two dose levels or a placebo for a treatment period of 36 weeks. Following this initial phase, participants will be reassigned to either continue their current treatment or switch to a different regimen, allowing everyone in the study to eventually receive active medication. This approach not only enables the evaluation of CS1's disease-modifying potential but also maintains a controlled environment for comprehensive data collection.

Anticipated Outcomes and Timeline

The trial's effectiveness will be measured by various evaluations, including the change in pulmonary vascular resistance, functional capacity proxies such as the six-minute walk distance, as well as additional heart function assessments, biomarker analyses, and patient-reported outcomes.

Cereno Scientific aims to begin enrolling participants by the second quarter of 2026, with top-line data expected around the fourth quarter of 2028, dependent on enrollment and study timelines. The results from this trial will represent one of the largest and most comprehensive clinical efforts undertaken by the company, paving the way for potential groundbreaking developments in PAH treatment.

Cereno Scientific’s Vision

"We aim to address the underlying mechanisms driving PAH rather than just manage symptoms," stated Rahul Agrawal, CMO and Head of R&D at Cereno Scientific. The company is positioning itself as a leader in epigenetic modulation for cardiovascular disorders and is optimistic about the prospects of CS1 as an effective treatment option.

CEO Sten R. Sörensen highlighted that the FDA clearance not only serves as a significant catalyst for the company's growth but also underscores CS1's potential in enhancing the commercial landscape for innovative therapies in the realm of cardiovascular diseases. With ongoing support through the Fast Track designation, Cereno Scientific is excited to embark on this journey towards improving the lives of patients suffering from PAH.

Conclusion

Cereno Scientific's clearance to commence the Phase IIb trial for CS1 represents a beacon of hope for patients battling pulmonary arterial hypertension. As the company gears up for enrollment, the healthcare community eagerly anticipates the development of a therapy that could fundamentally alter the treatment landscape for PAH and provide patients with hope for a better quality of life.