Cereno Scientific shares 4-month follow-up data from Expanded Access Program in line with Phase IIa trial results of CS1 in rare disease PAH
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 4-month follow-up data from the Expanded Access Program (EAP) following the Phase IIa trial of CS1 in patients with pulmonary arterial hypertension (PAH). The 4-month data from the EAP is in line with the observations made in the Phase IIa trial. Drug candidate CS1, an epigenetic modulating HDAC-inhibitor, has a strong potential to transform the lives of PAH patients as a well-tolerated oral therapy with a favorable safety and tolerability profile and disease-modifying effects.
“The safety and tolerability profile of CS1 observed in the first four months of the Extended Access Program is consistent with findings from our three-month Phase IIa trial, which is an encouraging outcome in this progressive disease. Safety remains a key limitation of currently available PAH therapies, with several recent development programs discontinued due to tolerability concerns. The combination of a favorable safety profile and early signals of efficacy underscores CS1’s potential in this rare and serious condition. As a next step, we are advancing into a global, placebo-controlled Phase IIb trial designed to more comprehensively assess the clinical impact of CS1,” said Rahul Agrawal, CMO and Head of R&D at Cereno Scientific.
“I am glad that we are able to extend the support for the PAH patients in our Phase IIa trial by enabling a continued CS1 treatment through the EAP. This was on request from patients and their physicians, which is a highly encouraging indication of CS1’s potential. While the time period on this follow-up data from the EAP is not much longer than the previous trial, I am pleased to see that the data is in line with the safety, tolerability and signals of efficacy observed in the Phase IIa trial. We will now let the EAP continue to completion and look forward to the results that will provide insights on long-term use of CS1 after up to 12 months treatment,” said Sten R. Sörensen, CEO at Cereno Scientific.
The Expanded Access Program (EAP) includes 10 eligible patients from the Phase IIa trial to continue CS1 treatment. Under an FDA-approved protocol, the EAP enables Cereno to collect long-term safety and efficacy data on CS1 use in PAH patients. A sub-study of the EAP utilizes a non-invasive imaging technology, developed by medtech company Fluidda, on certain enrolled patients to gain insight into how long-term use of CS1 influences changes in pulmonary arteries. These initiatives support ongoing treatment while providing valuable data for regulatory discussions and planning future Phase IIb or pivotal Phase III trials. The EAP will run for 12 months with results anticipated in Q1 2026.
For further information, please contact:
Tove Bergenholt, Head of IR & Communications
Email: [email protected]
Phone: +46 73- 236 62 46
This information is information that Cereno Scientific AB is obliged to make public pursuant to the EU Market Abuse Regulation. The information was submitted for publication, through the agency of the contact person set out above, at 08:25 CET on June 16, 2025.
About CS1
Drug candidate CS1 is currently in Phase II development, being advanced as a first-in-class treatment for the rare disease pulmonary arterial hypertension (PAH). CS1 is a histone deacetylase inhibitor (HDACi) that works through epigenetic modulation, uniquely targeting the underlying mechanisms driving disease progression in PAH.
Drug candidate CS1 is a well-tolerated oral therapy with a favorable safety profile and showed signals of disease-modifying effects as observed in a Phase IIa trial in patients PAH. The aim for CS1 is to offer an effective disease-modifying treatment with the ability to enhance quality of life and extend life for PAH patients. Unlike standard therapy that focus on managing symptoms, CS1 represents a novel therapeutic approach by targeting the root mechanisms of PAH. Specifically, CS1 aims to reverse the pathological vascular remodeling of the small pulmonary arteries. A successful Phase IIa trial was concluded in 2024. Insights into the long-term use of CS1 are being gathered in an expanded access program with 10 patients from the Phase IIa trial. Preparations are currently underway for a larger placebo-controlled Phase IIb trial to continue advancing CS1 toward regulatory approval and wider patient access. The Phase IIb trial is planned to be initiated in H1 2026.
About Cereno Scientific AB
Cereno Scientific is pioneering treatments to enhance and extend life. Our innovative pipeline offers disease-modifying drug candidates to empower people suffering from rare cardiovascular and pulmonary diseases to live life to the full.
Lead candidate CS1 is an HDACi that works through epigenetic modulation, being developed as an effective and disease modifying treatment with a favorable safety and tolerability profile for rare disease Pulmonary Arterial Hypertension (PAH). A Phase IIa trial evaluating CS1’s safety, tolerability, and exploratory efficacy in patients with PAH demonstrated that CS1 has a favorable safety profile, is well-tolerated and showed a positive impact on exploratory clinical efficacy parameters. An Expanded Access Program enables patients that have completed the Phase IIa trial to gain access to CS1. CS014, in Phase I development, is a new chemical entity with disease-modifying potential. 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 and Pulmonary Hypertension.
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, [email protected]. More information can be found on www.cerenoscientific.com.