Cereno Scientific publishes update and study protocol for the FDA approved Expanded Access Program for CS1 in PAH

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

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. 

EAP study named CS-004, is a prolongation of the CS-003 Phase II study of CS1 in PAH, with the primary objective being to evaluate the long-term safety and tolerability of continued treatment with CS1 and exploratory efficacy as secondary objective with the same methods as in the parent study (timepoints being at start, 4, 8 and 12 months every year). Further details will be published on clinicaltrials.gov later.

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 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.

"The EAP will inform us further about longer term safety and clinical aspects of CS1 in patients with PAH”, said Rahul Agrawal, CMO and Head of R&D at Cereno Scientific.

“I am happy to share progress in the Expanded Access Program. This program is important not only for the patients that can, if judged by investigators to benefit from treatment, continue to have access to the drug after the study has been completed, but also for Cereno as it allows us to gather longer term safety and efficacy data of CS1 in PAH. We see the interest shown by investigators and patients in the trial as a testimony to possible CS1 clinical benefits experienced by patients, “ said Sten R. Sörensen, CEO, Cereno Scientific.

The approval of the Expanded Access Program (EAP) comes on the heels of encouraging reports emerging from the Phase II study of CS1 in PAH, actively running at 10 specialist clinics in the US. In June, an investigator highlighted a notable case of patient improvement with CS1. Further, Cereno reported in October 2023 that a Data Quality Control Review (DQCR), of data obtained by the CardioMEMS HF System from the first sixteen patients, was concluded with positive findings. In November, another investigator urged Cereno to submit an Expanded Access request to the FDA, seeking permission to continue administering the investigational drug, CS1, to patients post the conclusion of the study treatment. Cereno promptly submitted the request on January 3rd, which was granted by the FDA on January 30th 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 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.