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 that the US Food & Drug Administration (FDA) provided endorsement of the plans for the Phase IIb trial of CS1 as noted by the official meeting minutes from the Type C-meeting held on April 21. The next clinical development step of CS1 is a larger, placebo-controlled Phase IIb trial aiming to further evaluate the encouraging efficacy signs including reverse vascular remodeling and improvement of right heart function as observed in the Phase IIa trial.
"This is a very exciting milestone for our pipeline. Receiving an FDA endorsement for the CS1 program in PAH at this stage in development is paving the way for further interactions regarding upcoming Phase IIb and pivotal Phase III trials. The plans for the Phase IIb trial are in alignment with the expectations of the FDA, meaning that we have high confidence that the Phase IIb trial design will include the relevant criteria contributing to the development program's marketing approval process. We are now continuing steps for preparations ahead of the Phase IIb trial in PAH, with a target of initiating the trial in H1 2026," said Rahul Agrawal, Chief Medical Officer (CMO) and Head of R&D at Cereno Scientific.
"The endorsement from the FDA for CS1 validates that the development plans have sound rationale to be able to support documentation for future marketing approval and adds credibility to our scientific and clinical capabilities. These are important factors in the dialogues we have with potential partners, but also other stakeholders we interact with such as collaborators, investors and the scientific community. We are keen to progress towards the initiation of the Phase IIb trial to further evaluate CS1 as a PAH treatment with favorable safety and tolerability and disease-modifying capacity. Our ultimate ambition is to be able to develop a treatment that could enhance and extend life of patients suffering from this devastating and fatal disease," said Sörensen, CEO at Cereno Scientific.
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 with the rare disease pulmonary arterial hypertension (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.
For further information, please contact:
Tove Bergenholt, Head of IR & Communications
Email:tove.bergenholt@cerenoscientific.com
Phone: +46 73- 236 62 46
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, certifiedadviser@carnegie.se. More information can be found on www.cerenoscientific.com.
