Chidamide

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 drug candidate CS014 has completed the first part of two in its Phase I trial in healthy volunteers without any safety concerns. CS014 is a novel histone deacetylase (HDAC) inhibitor with disease-modifying potential being developed for the rare disease idiopathic pulmonary fibrosis (IPF). The Phase I trial intends to evaluate the safety profile of CS014 in humans and is divided into two parts – a single ascending dose part (SAD) and a multiple ascending dose part (MAD). Part two of the trial (MAD) is currently ongoing according to plan and the Phase I trial is expected to be completed in mid-2025. HDACi CS014 has the potential to transform the treatment landscape of IPF. Drug candidate CS014 is a new chemical entity that acts through HDACi with strong vascular remodeling effects and disease-modifying potential as seen in preclinical studies. The potential of the HDACis has been demonstrated through preclinical studies including reversal of pathological remodeling of pulmonary vessels and anti-fibrotic effects in an animal model of PAH, as well as in vivo anti-thrombotic effects across a broad range of vasculature. These properties align with the key disease mechanisms of idiopathic pulmonary fibrosis (IPF) and addresses significant unmet medical needs. The HDACi CS014 has a safety and efficacy profile that is consistent with the significant medical needs remaining despite current IPF treatments. Part one of the Phase I trial of CS014 conducted by our CRO partner CTC in Uppsala explored the safety, tolerability and pharmacokinetics (PK) of single ascending oral doses (SAD) of CS014 in 30 patients. This first part of the trial has successfully been completed with results showing that CS014 exhibited an acceptable safety profile supporting its potential for further clinical development. “We are pleased with the progress of the CS014 Phase I trial and eagerly anticipate its completion in mid-2025. The successful completion of the single ascending dose (SAD) part provides a strong initial validation of HDACi CS014s favorable safety profile,” said Rahul Agrawal, CMO & Head of R&D of Cereno Scientific. “We believe that our novel HDACi CS014 has the potential to become an important treatment meeting high unmet clinical needs in the rare disease IPF. There is a void in the market for safe and well-tolerated novel drugs with a profile addressing the underlying pathophysiology of the IPF disease and its progression. CS014 aims to be a safe, well-tolerated oral drug with disease-modifying capacity and, as such, targets a large market potential,” 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 and multiple ascending oral doses of CS014 in healthy volunteers. The Phase I trial of CS014 will involve approximately 48 subjects and is expected to be completed mid-2025 whereafter top-line results can be shared after data analysis has been conducted. 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, under development for idiopathic pulmonary fibrosis (IPF). An ongoing Phase I trial evaluating the safety, tolerability, pharmacokinetics, pharmacokinetics (PK), and pharmacodynamics (PD) of single and multiple ascending oral doses of CS014 in healthy volunteers is expected to conclude in mid-2025. 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. Together, these findings indicate that CS014 has the potential to address the underlying pathophysiology behind the development of IPF. CS014 has demonstrated, in preclinical studies, the ability to regulate platelet activity, local fibrinolysis, and clot stability, helping to prevent thrombosis without increasing the risk of bleeding. This supports CS014's potential to address key unmet needs in IPF patients. Cereno’s HDAC inhibitor portfolio, capitalizing 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 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. Read PDF Share