Cereno Scientific

The Phase II study of CS1 in the rare disease PAH is actively running at 10 specialist clinics in the US, with the latest clinic activated in late December. One additional new clinic is currently in late-stage start-up process. Since the last phase II study progress update communicated on February 21, 2024, one new additional patient have now been enrolled in the study, one additional patient has now been randomized to CS1 drug in the study and one additional patient has now completed the study. Enrolled patients are scheduled to receive CardioMEMS HF System implantation within two weeks from enrollment and are, following successful implantation of CardioMEMS and up to 6 weeks baseline evaluation, randomized to CS1 drug therapy on their next scheduled visit. The next step for the recently enrolled patient is to undergo CardioMEMS implantation. 25 patients have already received CardioMEMS implantation, of which 24 patients have already been randomized to CS1 drug therapy and one is still in baseline period prior to randomization. To date 20 patients have completed the study.  

"We appreciate the hard work and valuable contribution from investigators and all other stakeholders in supporting the recruitment of patients to finalize the Phase II study," said Rahul Agrawal, CMO and Head of R&D at Cereno Scientific.

EAP approval follows positive findings from the PAH study with CS1 drug therapy reported since 2023   

Cereno Scientific has during the last few months reported positive findings from the ongoing study suggesting a potential positive effect of drug candidate CS1 in patients with the severe rare disease PAH. Study completion and top-line results are expected during Q3 2024.

Remarkable Patient Case with CS1 reported June 2023

First, a patient case study performed on the first patient having completed the study at a specific clinic showed remarkable efficacy data. In 12 weeks of treatment with CS1, the patient showed a 30% reduction in pulmonary pressure and a 20% increase in cardiac output. The patient's overall functional status was changed from NYHA/WHO functional class II to I at the end of the treatment period, meaning that the patient had next to normal functional physical capacity with CS1 added to stable conventional therapy.

Data Quality Control Review reported October 13th 2023

In addition, Cereno reported in October 2023 that a Data Quality Control Review (DQCR), of data obtained by the CardioMEMS HF System from the first 16 patients, was concluded with positive findings. The data quality of the CardioMEMS measurements was found satisfactory with adherence to study protocol and with timely data transfers from the patient's home to the clinic. Efficacy findings showed a clinically meaningful reduction of pulmonary pressure in several patients, included in the data quality control, of a similar or greater magnitude as in the Patient Case.

EAP for CS1 Approved Jan 30th 2024

Further, the Expanded Access Program (EAP) ("Compassionate Use") approved by the FDA on 31st January 2024 is progressing according to plan, propelled by investigator and patient interest. Investigators indicate that close to two-thirds of the patients, having completed the study or are currently on therapy, have been judged to be interested in continued access to CS1 following study completion. Currently site-specific and IRB approvals are being progressed. The EAP will provide Cereno with the opportunity to, under a formal FDA-approved protocol, collect safety and efficacy data from long-term exposure to CS1 in patients with PAH. The EAP will thus 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.

"We have seen great progress in the CS1 study over the last year, and two initiatives run during the ongoing Phase II study have shown positive findings, indicating a potential clinical benefit of CS1 in PAH patients. Since our FDA approval from January 30 for Expanded Access to CS1, followed by investigators indicating that a majority of patients would be interested in continued access to CS1 following study completion, we are making great strides towards reaching the milestone of completing our Phase II study of CS1 in PAH", said Sten R. Sörensen, CEO, Cereno Scientific.

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, CS1 has been available under FDA's Expanded Access Program ("compassionate use") for continued CS1 treatment in patients who have completed the Phase II study. 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.