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| Published November 10, 2025

Cereno Scientific takes the next step towards phase IIb study in PAH

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Cereno Scientific has submitted the trial protocol for its planned global phase IIb trial of its drug candidate CS1 to the U.S. Food and Drug Administration (FDA). The submission is an important step in the company's work to develop new treatments for pulmonary arterial hypertension (PAH), a rare and serious disease that affects the blood vessels in the lungs.

The upcoming phase IIb study will evaluate the safety, tolerability and efficacy of CS1 – an oral histone deacetylase inhibitor (HDAC inhibitor) that affects gene expression through epigenetic modulation. Unlike current treatments that primarily alleviate symptoms, CS1 aims to affect the fundamental mechanisms behind the disease.

Promising clinical results from the phase IIa study

Previous clinical data from the company's phase IIa study showed signs that the treatment can improve heart function and contribute to partial remodeling of vascular changes. The upcoming study, which will be global, multicentre and placebo-controlled, is being conducted in collaboration with a leading international contract research organisation (CRO).As part of the preparations, regulatory processes in other important regions will also follow.

“"The submission of the protocol marks a major milestone and underscores the focus, commitment and outstanding collaboration across our team and global CRO," said Rahul Agrawal, CMO and Head of R&D of Cereno Scientific. "This protocol has been developed in alignment with the FDA's feedback from our recent Type C meeting, and we now look forward to the agency's review ahead of initiating the trial."

Fast Track status strengthens development

CS1 has been granted Fast Track status by the FDA, which allows the company to have closer dialogue with the agency and expedite the regulatory process. The program is intended for drugs that target serious diseases with high medical needs, and the decision underscores the FDA's recognition of CS1's potential as a future treatment option.

“This milestone moves us closer to our goal of delivering a first-in-class therapy that targets the root mechanisms of PAH through epigenetic modulation," said Sten R. Sörensen, CEO of Cereno Scientific. "With CS1, we have an opportunity to potentially change the treatment paradigm for patients facing this devastating disease and, in doing so, create meaningful long-term benefits for both patients and shareholders.

After the FDA's customary 30-day review, Cereno is expected to receive the go-ahead to start the study in the first half of 2026.

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