Cereno Scientific

“This is an important milestone on our path toward making a difference for patients with the deadly rare disease PAH. I am very pleased that we now have received FDA’s approval for the Extended Access Program (EAP) to CS1 and delighted for the patients who will now be able to continue receiving CS1 as drug therapy providing potential clinical benefits. As reported recently, we have secured the long-term supply of CS1 by entering a scale-up manufacturing agreement with CordenPharma that will ensure that patients in the EAP have access to CS1 and that we have CS1 for the next clinical study,” said Sten R. Sörensen, CEO, Cereno Scientific.

“I’m excited to be able to extend the use of CS1 to patients with this deadly disease, who are in critical need of additional high quality supportive therapy” said Raymond Benza, MD, Chief of Pulmonary Hypertension for the Mount Sinai Health System; PI for the Phase II study of CS1, member of the Clinical Steering Committee and Chair of the Scientific Advisory Board, Cereno Scientific.

The Expanded Access Program (EAP) with CS1 in PAH provides patients who have completed the Phase II study the option to continue with CS1 drug therapy, in consultation with the investigator and subject to approval by the ethics committee at the local hospital. The Expanded Access Program 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 could provide valuable support to later potential applications to the FDA such as fast-track designation/breakthrough therapy as well as support to obtain the IND acceptance to start a Phase IIb/III pivotal study with CS1.

The Phase II study of CS1 in the rare disease PAH is actively running at 10 specialist clinics in the US with one new clinic currently in late-stage start-up process. In 2023, the company reported positive findings from the ongoing study suggesting a potential positive effect of drug candidate CS1 in patients with the severe rare disease PAH. 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 she had next to normal functional physical capacity with CS1. In addition, Cereno reported in October 2023 that a Data Quality Control Review (DQCR) 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. The review included data obtained by the CardioMEMS HF System from the first 16 patients enrolled in the study and the reported findings can be read in full in a previous announcement. A request for expanded access to CS1 (also called “compassionate use”) was submitted to the FDA after requests by investigators in the Phase II study and was approved in January 2024. The study is designed to randomize 30 PAH patients and the top-line result of the Phase II study is estimated to be reported in Q2 2024.

For further information, please contact:

Tove Bergenholt, Director IR & Communications
Email: tove.bergenholt@cerenoscientific.com
Phone: +46 732-366 246

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 Q2 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.