Cereno Scientific

The study is intended to support the translation of the well-documented reverse vascular remodeling effects of CS1 in preclinical models to clinical practice. The lack of non-invasive methods available to demonstrate this effect in patients present a challenge. The innovative imaging technology Functional Respiratory Imaging (FRI), developed by Fluidda, has been explored as a potential non-invasive tool to solve this challenge by providing detailed, patient-specific insights into pulmonary vascular changes. The study, also referred to as the "Fluidda study", is designed to include three CT scans in certain patients enrolled in the EAP during a 12-month period. The study is expected to provide a visualization of how long-term treatment of CS1 on top of standard therapy may impact disease characteristic structural changes in small pulmonary arteries, demonstrated by improvements in blood vessel volume in these arteries on the CT images.

"By using the Fluidda technology, we will be able to obtain detailed images of the patient's lungs and in so doing, detect changes in the pulmonary vasculature in patients treated with CS1. We believe that by monitoring the treatment response of CS1 with this technology, we will be able to capture signs of CS1's reverse remodeling effect in PAH patients," said Rahul Agrawal, CMO & Head of R&D of Cereno Scientific.

"Our hope is to offer a pioneering approach to PAH therapy uniquely using HDAC inhibition as a mode of action in cardiovascular and pulmonary disease. CS1, part of our HDACi portfolio, targets the pathophysiology of disease progression in PAH with the potential to prevent or reverse vascular remodeling," said Sten R. Sörensen, CEO of Cereno Scientific. "Following promising preclinical data and Phase IIa results, we aim to further understand CS1's disease-modifying potential with the Fluidda study."

A Phase IIa trial of drug candidate CS1 in PAH successfully met the primary endpoint and was concluded in the fall 2024. The trial showed that CS1 is safe and well-tolerated and revealed compelling data supporting a disease-modifying capacity on top of standard therapy. The EAP comprising 10 patients was approved as an extension of the Phase IIa trial in PAH, allowing patients who have completed the Phase IIa trial to continue CS1 treatment. The EAP, an FDA-approved protocol, enables Cereno to obtain valuable long-term safety and efficacy data of CS1 in PAH patients supporting required regulatory interactions and planning future Phase IIb or pivotal Phase III trials.

For further information, please contact:

Tove Bergenholt, Head of IR & Communications

Email:  tove.bergenholt@cerenoscientific.com

Phone: +46 73- 236 62 46

Sten R. Sörensen, CEO

Email: sten.sorensen@cerenoscientific.com

Phone: +46 73-374 03 74

About CS1

Drug candidate CS1 is an HDAC inhibitor (HDACi) that works through epigenetic modulation, being developed as a safe, effective and disease-modifying treatment for the rare disease pulmonary arterial hypertension (PAH). CS1 targets the root mechanism of the disease, aiming to reverse the pathological vascular remodeling of the small lung arteries.

In a Phase IIa study, CS1 successfully met the primary endpoint of safety and tolerability. CS1 further showed compelling positive impact on exploratory parameters of clinical relevance over the 12-week treatment period. The Phase IIa data, together with preclinical data from the HDACi program, supports reverse remodeling effects in small lung arteries. This provides a basis of assumption that CS1 may act with disease-modifying capacity in PAH. In preclinical cardiovascular disease models, HDAC inhibitors have also shown anti-fibrotic, anti-inflammatory, pulmonary pressure-reducing, and anti-thrombotic effects. CS1's unique efficacy profile aligns well with the underlying mechanisms of disease that drives the progression of PAH, positioning it to address the critical unmet need for more effective treatment options. The goal of CS1's development is to enhance and extend life for patients with PAH. CS1 is part of Cereno's HDACi portfolio, untapping the potential of epigenetic modulation in rare cardiovascular and pulmonary diseases. CS1 has been granted orphan drug status in both the US and EU.

CS1 has been approved by the FDA for an Expanded Access Program (EAP) as an extension of the Phase IIa trial in PAH, which enables Cereno to obtain valuable long-term safety and efficacy data of CS1 in PAH patients. 10 patients are currently enrolled in the program, which allows patients who have completed the Phase IIa trial to continue CS1 treatment if deemed suitable by physicians in the study. Through a collaboration with innovative medical company Fluidda, their non-invasive imaging technology called Functional Respiratory Imaging (FRI) will be used on certain patients enrolled in the EAP to visualize how long-term use of CS1 influences changes in pulmonary arteries. The data obtained by the EAP supports required regulatory interactions and planning future Phase IIb or pivotal Phase III trials.

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 a safe, effective and disease modifying treatment 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 was safe, 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. HDACi CS014, in Phase I development, is a new chemical entity with disease-modifying potential. CS014 employs a multi-modal mechanism of action as an epigenetic modulator, targeting key unmet needs in patients with rare disease 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 Certified Adviser is Carnegie Investment Bank AB, certifiedadviser@carnegie.se. More information can be found on www.cerenoscientific.com.