Cereno Scientific (Nasdaq First North: CRNO B), an innovative biotech pioneering treatments to enhance and extend life for people with rare cardiovascular and pulmonary diseases, today announced the last patient's last visit concluded the 12-month active study period of the CS1 Expanded Access Program (EAP) in pulmonary arterial hypertension (PAH). Initial learnings from the EAP expected to be available in the first quarter of 2026 and further analyses planned during second quarter of 2026, contributing to the ongoing CS1 development program and its overall value proposition.
The Expanded Access Program (EAP) for CS1 was initiated following the completion of the Phase IIa study to enable eligible patients with pulmonary arterial hypertension (PAH) to continue treatment with CS1 under physician supervision. The Phase IIa trial, conducted over 3-months, demonstrated that CS1 was well-tolerated with a favorable safety profile, and showed promising efficacy signals, including improvements in right heart function, patient quality of life, and signals consistent with reverse vascular remodeling. The EAP program enrolled 10 patients who had completed the Phase IIa study, enabling the collection of longer-term information on safety and tolerability during extended use of CS1. Insights generated from the EAP, including the exploratory imaging sub-study of vascular changes in the lung, are intended to complement the Phase IIa results and support the ongoing clinical development of CS1.
"From a clinical perspective, the Expanded Access Program has enabled longer-term treatment with CS1 beyond the Phase IIa study, which is particularly relevant in a progressive disease like PAH. While the number of patients in the program is limited, the EAP is designed to support the collection of additional information on continued use of CS1. Completion of last patient last visit represents an important operational milestone as we now move into the data handling and evaluation phase," said Rahul Agrawal, CMO and Head of R&D of Cereno Scientific.
"Reaching last patient last visit in the Expanded Access Program is an important milestone for CS1 and reflects continued progress as we advance the program toward the next stage of development. The EAP is intended to complement the Phase IIa study by contributing longer-term learnings that can help inform the ongoing development of CS1, alongside our preparations for the planned Phase IIb trial. Taken together, this progress continues to strengthen the overall value proposition of CS1 and its positioning as a differentiated, potentially disease-modifying treatment in PAH," said Sten R. Sörensen, CEO of Cereno Scientific.
The EAP is now entering standard processes including database lock, quality assurance and subsequent analysis and interpretation. Initial learnings from the program are expected to be communicated in Q1 2026, with additional analyses and interpretation planned during Q2 2026. In parallel, preparations for the global Phase IIb study of CS1 in PAH are progressing, including start-up activities in the United States and planned regulatory interactions in Europe and South America. First patient enrollment in the Phase IIb study is anticipated in the second quarter of 2026.
For further information, please contact:
Tove Bergenholt, Head of IR & Communications
Email: tove.bergenholt@cerenoscientific.com
Phone: +46 73- 236 62 46
About PAH
PAH is a rare, progressive and life-threatening disease characterized by high blood pressure in the pulmonary arteries that leads to right heart failure and premature death. Current standard treatments mainly focus on managing symptoms, leaving a significant unmet need for disease-modifying therapies that can change the course of disease and improve long-term outcomes.
About CS1
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.
About Cereno Scientific AB
Cereno Scientific is pioneering treatments to enhance and extend life. The company's innovative pipeline offers disease-modifying drug candidates to empower people suffering from rare cardiovascular and pulmonary diseases to live life to the fullest.
Lead candidate CS1 is an HDAC inhibitor that works through epigenetic modulation and represents a novel therapeutic approach by targeting the root mechanisms of the pulmonary arterial hypertension (PAH). CS1 is a well-tolerated oral therapy with a favorable safety profile that has shown encouraging efficacy signals in a Phase IIa trial in patients with PAH, including improvements in right heart function and patient quality of life, consistent with reverse vascular remodeling. An Expanded Access Program enables patients that have completed the Phase IIa trial to gain access to CS1. CS014, a new chemical entity with disease-modifying potential, showed favorable safety and tolerability profile in a Phase I trial. CS014 is a HDAC inhibitor with a multimodal mechanism of action as an epigenetic modulator having the potential to address the underlying pathophysiology of rare cardiovascular and pulmonary diseases with high unmet needs such as idiopathic pulmonary fibrosis (IPF). Cereno Scientific is also pursuing a preclinical program with CS585, an oral, highly potent and selective prostacyclin (IP) receptor agonist that has demonstrated the potential to significantly improve disease mechanisms relevant to cardiovascular diseases. While CS585 has not yet been assigned a specific indication for clinical development, preclinical data indicates that it could potentially be used in indications like thrombosis prevention without increased risk of bleeding.
The Company is headquartered in GoCo Health Innovation City, in Gothenburg, Sweden, and has a US subsidiary; Cereno Scientific Inc. based in Kendall Square, Boston, Massachusetts, US. Cereno Scientific is listed on the Nasdaq First North (CRNO B). The Company's Certified Adviser is DNB Carnegie Investment Bank AB, certifiedadviser@carnegie.se. More information can be found on www.cerenoscientific.com.
