Cereno Scientific announces that a study protocol synopsis for the FDA approved Expanded Access Program for CS1 in PAH has been published on ClinicalTrials.gov

Cereno Scientific (Nasdaq First North: CRNO B), a company developing innovative treatments for common and rare cardiovascular disease, today announced that a study protocol synopsis for the Expanded Access Program (EAP) for its lead candidate drug, the HDAC inhibitor CS1, in the rare disease Pulmonary Arterial Hypertension (PAH), has been published. The EAP will support long-term documentation of safety and efficacy data of CS1 treatment in the Phase II study in PAH.

The study protocol synopsis for the CS1-004 Expanded Access Study, titled; “Expanded Access, Open-Label, Safety Extension Study for Patients That Have Completed Parent Study CS1-003 and Who Are Judged by the Investigator to Benefit From Continued CS1 Treatment” is now published on ClinicalTrials.gov.

The EAP study named CS-004, is an extension of the CS1-003 Study. The primary objective of the CS1-004 study is to evaluate long-term safety and tolerability of continued treatment with CS1. The exploratory objectives are to evaluate clinical benefit by using CardioMEMS, echocardiography, cardiac magnetic resonance imaging, right heart catheterization, quality of life assessments, biomarkers and actigraphy to measure changes in clinical response with continued treatment with CS1 in patients with Pulmonary Arterial Hypertension (PAH).

Safety and exploratory efficacy endpoints will be measured, using the same methods as in the parent study, at the start of the study and subsequently at 4, 8 and 12 months every year. As such, the EAP study will allow Cereno to gather further documentation of CS1 use in patients suffering from PAH, which will help in discussions with regulatory authorities and to design our Phase IIb/III pivotal study with CS1.             

Up to 30 patients could participate in Study CS1-004, if they completed the parent study, tolerated CS1 treatment, and if, in the investigator’s judgement, the benefits of continued treatment with CS1 outweigh the risk. Some patients will directly roll-over into Study CS1-004 with no disruption in CS1 treatment, while other patients that have already completed Study CS1-003, will need to be restarted on CS1. Patients will, initially, receive the same dose they received in the parent study CS1-003. All patients will later have the option to be titrated to the most efficacious and safe dose determined from the ongoing CS1-003 study, when completed.

On a yearly basis, the principal investigator will determine if a patient continues the Expanded Access study for a subsequent year based on safety, tolerability, and clinical benefit of CS1. Continuation of the Expanded Access study will also be evaluated on a yearly basis by Cereno Scientific.

As previously reported, investigators have shown substantial interest in the EAP with two-thirds of the patients, having completed the study or are currently on therapy, deemed by investigators to be eligible for continued access to CS1. Currently site-specific and Ethics Committee (IRB) approvals are underway and once these are in place the first patients can be dosed in the EAP.

"The EAP is a long-term follow-up study of CS1 treatment in patients with PAH. We are happy not only that we can continue to, under an FDA approved protocol, provide patients, judged to benefit from further CS1 treatment, with continued access to CS1 for as long as deemed favorable by their treating physician. In addition, we value that this extended study design allows us to gather additional data on the long-term safety and efficacy of CS1 treatment. Data that can be used to guide discussions with regulators and, in the future, support commercial positioning of CS1,” said Sten R. Sörensen, CEO, Cereno Scientific.

The information on ClinicalTrials.gov contains a detailed description of study design, inclusion and exclusion criteria as well as primary and secondary objectives.

Read the protocol synopsis here.
ClinicalTrial.gov identifier: NCT06321705

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 common and rare cardiovascular disease. The lead drug candidate, CS1, is a HDAC (histone deacetylase) inhibitor that acts as an epigenetic modulator with pressure-reducing, reverse-remodeling, anti-inflammatory, anti-fibrotic and anti-thrombotic properties. A Phase II study is ongoing to evaluate CS1’s safety, tolerability, and efficacy in patients with the rare disease pulmonary arterial hypertension (PAH). A collaboration agreement with global healthcare company Abbott allows Cereno to use their cutting-edge technology CardioMEMS HF System in the study. Two initiatives performed during the ongoing Phase II study have shown positive findings suggesting the potential clinical benefit of CS1 in PAH patients. These initial findings are, however, not a guarantee of the final study results that are expected in Q3 2024. Since January 2024, we are delighted that the FDA´s Expanded Access Program will enable patients with PAH, a serious life-threatening disease condition, to gain access to CS1 where no comparable alternative therapy options are available. Cereno also has two promising preclinical drug candidates in development through research collaborations with the University of Michigan. Investigational drug CS014 is a HDAC inhibitor in development as a treatment for arterial and venous thrombosis prevention. The innovative drug candidate represents a groundbreaking approach to antithrombotic treatment potentially without the associated increased risk of bleeding in humans. CS014 is a new chemical entity with a multi-fold mechanism of action as an epigenetic modulator – regulating platelet activity, fibrinolysis, and clot stability for the prevention of thrombosis without increased risk of bleeding as documented in preclinical studies. Drug candidate CS585 is a prostacyclin receptor agonist that has been documented in several preclinical studies to target the IP receptor for prevention of thrombosis without increased risk of bleeding, which also has been recognized in the medical community. CS585 was in-licensed from the University of Michigan in 2023. The company is headquartered in Gothenburg, Sweden, and has a US subsidiary Cereno Scientific Inc. based in Kendall Square in Boston, Massachusetts, US. Cereno is listed on the Nasdaq First North (CRNO B). More information on www.cerenoscientific.com.