Cereno Scientific AB 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 documentaMon 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 Phase IIb/III pivotal study with CS1. 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 30 2024.