Announcement • Apr 02

Cereno Scientific Reports Favorable Safety and Tolerability After 12 Months of Cs1 Treatment in Pah from the Expanded Access Program

Cereno Scientific announced initial learnings from the 12-month Expanded Access Program (EAP) with its lead drug candidate CS1 in pulmonary arterial hypertension (PAH). The data confirm a favorable safety and tolerability profile over long-term treatment, consistent with previous Phase IIa results, further strengthening the value proposition of CS1 as an oral, once-daily potentially disease-modifying therapy. The Expanded Access Program (EAP) enrolled ten patients who had completed the Phase IIa trial, enabling continued treatment with CS1 under physician supervision. Initial learnings from the completed 12-month treatment period show that CS1 was well tolerated, with no unexpected safety concerns observed. No deaths were reported, and no discontinuations were reported to be related to CS1. Six out of ten patients completed the full 12 months of continuous treatment with CS1. Of the remaining patients, two discontinued CS1 treatment following atrial fibrillation events, which was assessed as not related to CS1; one withdrew consent, and one was lost to follow-up. The EAP was conducted under a formal FDA protocol and initiated following requests from patients and physicians. It enabled the generation of additional long-term data beyond the three-month Phase IIa trial, which had demonstrated that CS1 had favorable safety and tolerability and showed encouraging efficacy signals, including improvements in right heart function, functional class and patient quality of life, with signs consistent with reverse vascular remodeling. Together, the Phase IIa trial and the EAP provide up to 15 months of treatment experience in patients, further strengthening the overall clinical understanding of CS1 in PAH. The EAP was initiated following positive results of the Phase IIa trial, which evaluated the safety, tolerability, pharmacokinetics, and exploratory efficacy of CS1 on top of standard therapy in patients with PAH. The Phase IIa trial was conducted at 10 US clinics over 3 months with a total of 25 patients of which 21 were evaluated for efficacy parameters. The trial successfully met its primary endpoint of safety and tolerability, with no drug-related serious adverse events. Encouraging efficacy signals were observed in the trial, including improvements in right heart function, functional class and patient quality of life, with early signs consistent with reverse vascular remodeling. Preparations for a larger, placebo-controlled global Phase IIb study of CS1 in PAH are ongoing, with first patient enrollment anticipated in June 2026. Further analyses from the EAP, including results from the exploratory imaging sub-study using Fluidda's technology, are planned to be communicated during Second Quarter 2026. CS1 is an orally administered histone deacetylase inhibitor (HDACi) in development as a well-tolerated, disease-modifying therapy for pulmonary arterial hypertension (PAH) with favorable safety profile. Acting through epigenetic modulation, CS1 targets key disease-driving mechanisms such as vascular remodeling, fibrosis and inflammation. CS1 has shown disease-modifying potential in early clinical evaluation and is being evaluated as an add-on (on top of standard-of-care) therapy with the potential to improve outcomes for patients with high unmet medical needs. It has been granted Orphan Drug Designation (ODD) in both the U.S. and the EU and received Fast Track designation from the U.S. FDA in August 2025, underscoring its potential to address a serious condition with high unmet medical need. CS1 has first-in-class potential and is currently in preparation for a global Phase IIb trial.

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Cereno Scientific Reports Favorable Safety and Tolerability After 12 Months of Cs1 Treatment in Pah from the Expanded Access Program

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