Cereno Scientific announces initiation of Multiple Ascending Dose (MAD) part of Phase I trial of the novel HDAC inhibitor CS014
Cereno Scientific (Nasdaq First North: CRNO B), a pioneering biotech developing innovative treatments for diseases with high unmet medical needs, today announced that the Company has initiated the Multiple Ascending Dose (MAD) part of the first-in-man Phase I trial of novel HDACi CS014, after the Safety Monitoring Committee ensuring that the safety profile in the Single Ascending Dose (SAD) part is within acceptable limits. The CS014 development program is targeting rare disease Idiopathic Pulmonary Fibrosis (IPF).
The MAD part (Part II) of the Phase I trial will explore safety, tolerability, pharmacokinetics (PK), and efficacy parameters following multiple ascending doses of CS014, dosed for seven days. The MAD was preceded by the SAD part (Part I) that is exploring safety, tolerability and pharmacokinetics (PK) of single ascending oral doses of CS014.
“We are delighted that the SAD part of the Phase I trial of CS014 is now almost completed, as the trial now proceeds into the second phase, exploring multiple ascending doses of CS014 in healthy volunteers”, said Dr. Rahul Agrawal, Head of R&D and CMO, Cereno Scientific.
“We are happy that the Phase I trial is proceeding according to plan, reaching the milestones we have set up. We have a good momentum to finalize the trial on time and we look forward to sharing more results following the conclusion of the trial in the middle of 2025”, said Sten R. Sörensen, CEO, Cereno Scientific.
The Phase I trial CS014-001, titled “A First-in-human, Open-label Trial to Investigate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of CS014 in Healthy Volunteers After Single and Multiple Administration”, is conducted in partnership with the contract research organization (CRO) Clinical Trial Consultants AB (CTC), in Uppsala, Sweden.
The trial is a Phase I, open-label, first-in-man trial designed to evaluate safety, tolerability, pharmacokinetics, and efficacy parameters 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 conclude by mid-2025.
About CS014
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).
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 and the investigational drug candidate CS014.
A first-in-man, Phase I trial of CS014 was initiated in June 2024.
For further information, please contact:
Henrik Westdahl, Director IR & Communications
Email: henrik.westdahl@cerenoscientific.com
Phone: +46 70-817 59 96
Sten R. Sörensen, CEO
Email: sten.sorensen@cerenoscientific.com
Phone: +46 73-374 03 74
About Cereno Scientific AB
Cereno Scientific develops innovative treatments for diseases with high unmet medical needs. Lead drug candidate, CS1, is an HDAC inhibitor that acts as an epigenetic modulator, with pressure-reducing, reverse-remodeling, anti-fibrotic, anti-inflammatory and anti-thrombotic properties. CS1 is being developed as a disease-modifying treatment for the serious and life-threatening 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. CS1 study data, together with preclinical information, is consistent with reversing pathological remodeling. A collaboration agreement with global healthcare company Abbott allowed Cereno to use their cutting-edge technology CardioMEMS HF System in the trial. An Expanded Access Program enables patients that have completed the Phase IIa trial to gain access to CS1 when no comparable alternative therapy options are available. CS1 holds orphan drug designations in both the US and EU. HDAC inhibitor CS014, in Phase I development, is a new chemical entity being developed as a disease-modifying treatment for rare disease 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. 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. Preclinical candidate CS585 is an oral, highly potent and selective prostacyclin (IP) receptor agonist that has demonstrated the potential to significantly improve disease mechanisms relevant to cardiovascular disease. 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. A target indication for CS585 is currently being evaluated; rare diseases with high unmet medical needs are being considered. CS014 and CS585 are developed through research collaborations with the University of Michigan. CS585 was in-licensed from the University of Michigan in 2023. 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 is on www.cerenoscientific.com.