Cereno Scientific gets FDA clearance for phase IIb PAH trial

Cereno Scientific AB (Nasdaq First North: CRNO B) received U.S. Food and Drug Administration clearance to initiate a Phase IIb trial of its drug candidate CS1 for treating pulmonary arterial hypertension, the Swedish biotech company announced.

The FDA clearance enables Cereno to target first patient enrollment in the second quarter of 2026, with top-line data expected around the fourth quarter of 2028, subject to enrollment timelines. The approval follows regulatory interactions and builds on safety and tolerability data from a Phase IIa study.

The global, multicenter trial will enroll approximately 126 patients with PAH who are stable on background therapy. The study design includes 36 weeks of initial treatment where participants receive once-daily CS1 capsules at one of two dose levels or matching placebo, followed by a re-randomization period. The total study duration spans 60 weeks, including screening and follow-up.

The trial will evaluate CS1's effect on pulmonary vascular resistance at Week 36 via right-heart catheterization and changes in 6-minute walk distance. Additional assessments include heart function measures, biomarker changes, clinical worsening, patient-reported outcomes, and pharmacokinetics.

The dose-finding trial will be conducted across 10-12 countries in the United States, Europe and South America at approximately 65 investigative sites. CS1 has received Orphan Drug Designation and Fast Track designation in the U.S.

"The planned Phase IIb trial is designed to determine the optimal dose for a Phase III trial and to assess CS1's potential to meaningfully reduce pulmonary vascular resistance and improve functional capacity when added to today's standard therapies," said Rahul Agrawal, chief medical officer and head of R&D at Cereno Scientific.

CS1 is an orally administered histone deacetylase inhibitor being developed as a treatment for PAH, a rare and progressive disease characterized by high blood pressure in pulmonary arteries. The information was disclosed in a company press release.

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