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 second and final part of the Phase I trial of drug candidate CS014 in healthy volunteers has been concluded with the last patient's last visit. The Phase I trial intends to primarily evaluate the safety and tolerability profile of CS014 in humans and is divided into two parts - a single ascending dose part (SAD) and a multiple ascending dose part (MAD). Data management, database lock, and analysis will now commence after the last patient's last visit, and the trial's top-line results are expected to be announced in June 2025.
The new chemical entity 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). Drug candidate CS014 has shown strong vascular remodeling effects in preclinical studies, which indicates a disease-modifying potential. The Phase I trial of CS014 conducted by Cereno's CRO partner CTC in Uppsala evaluated safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of single ascending oral doses and multiple ascending oral doses of CS014 for seven days in 48 healthy volunteers. Completed in February 2025, part one of the trial indicated that CS014 has an acceptable safety and tolerability profile supporting its potential for further clinical development. Top-line results of the complete Phase I trial will be available in June 2025.
"We are pleased with the progress of the CS014 Phase I trial, which has been well executed according to planned timelines. The CRO, CTC in Uppsala, has been an excellent partner in this trial. The safety and tolerability data observed from part one of the trial suggest that CS014 may have a favorable safety profile. We now eagerly look forward to the top-line results, and further clinical development exploring CS014's potential," said Rahul Agrawal, CMO & Head of R&D of Cereno Scientific.
"We are excited to have reached the 'last patient last visit'- milestone for the Phase I trial. We believe that CS014 can bring much needed improvement for patients as a well-tolerated oral treatment with a favorable safety profile and disease-modifying capacity. Based on the indicative results from part one of the Phase I trial combined with promising effects seen in preclinical models, CS014 has a large market potential with relevance as a treatment for rare cardiovascular and pulmonary diseases with high unmet medical needs," said Sten R. Sörensen, CEO of Cereno Scientific.
The Phase I trial is an open-label, first-in-man trial designed to evaluate safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of single ascending oral doses of CS014 and multiple ascending oral doses of CS014 for seven days in healthy volunteers. The Phase I trial of CS014 involves 48 subjects. Top-line results of the study is anticipated in June 2025.
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 CS104
HDAC inhibitor CS014 is a new chemical entity with a multi-modal mechanism of action as an epigenetic modulator. A Phase I trial is ongoing evaluating the safety, tolerability, pharmacokinetics, pharmacokinetics (PK), and pharmacodynamics (PD) of single and multiple ascending oral doses of CS014 in healthy volunteers with expected top-line results in June 2025. CS014 has the potential to address the underlying pathophysiology of rare cardiovascular and pulmonary diseases with high unmet needs such as idiopathic pulmonary fibrosis (IPF).
Preclinical studies of HDAC inhibitors show that these drugs can reverse fibrosis in models of IPF. Studies also show that these drugs prevent the pathological remodeling of pulmonary vessels that ultimately leads to pulmonary hypertension in many IPF patients. Preclinical studies of CS014 have demonstrated an effect on reversal of fibrosis and a dose-dependent beneficial effect on pathological vascular remodeling in an established model of PAH. CS014 has demonstrated, in preclinical studies, the ability to regulate platelet activity, local fibrinolysis, and clot stability, contributing to prevent thrombosis without increasing the risk of bleeding.
Cereno's HDAC inhibitor portfolio, utilizing on the principle of epigenetic modulation, comprises Cereno's lead drug candidate CS1 in Phase II and CS014.
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 Certified Adviser is Carnegie Investment Bank AB, certifiedadviser@carnegie.se. More information can be found on www.cerenoscientific.com.
