Cereno Scientific has announced the enrollment of nine additional patients in its Expanded Access Program (EAP) for CS1, a drug candidate targeting pulmonary arterial hypertension (PAH). This brings the total number of patients in the EAP to ten, offering a valuable opportunity to gather long-term safety and efficacy data on the drug in this rare disease.
The EAP, an extension of the Phase IIa trial for CS1, allows patients who completed the initial trial to continue receiving the treatment, provided their physicians deem it appropriate. This program is particularly significant as it enables Cereno Scientific to monitor the long-term effects of CS1 and strengthen the drug's documentation for regulatory purposes.
Long-Term Data Collection
According to Sten R. Sørensen, CEO of Cereno Scientific, the high interest from patients and physicians in continuing CS1 treatment after the Phase IIa study is encouraging. He emphasized the value of collecting long-term clinical data as the company advances its clinical program.
In collaboration with Fluidda, an innovative medical company, Functional Respiratory Imaging (FRI) will be employed on select EAP patients. This non-invasive imaging technology will visualize how long-term CS1 use affects changes in pulmonary arteries, providing critical insights into the drug's mechanism of action and potential for disease modification.
CS1: An HDAC Inhibitor for PAH
CS1 is an HDAC inhibitor (HDACi) that modulates epigenetic processes and is being developed as a disease-modifying treatment for PAH. The drug aims to reverse the pathological vascular remodeling of small lung arteries, addressing the root cause of the disease.
In the Phase IIa study, CS1 successfully met its primary endpoint of safety and tolerability. Exploratory clinical efficacy parameters also showed a positive impact over the 12-week treatment period. Preclinical data further supports the dose-dependent positive impact of the HDACi program on reverse remodeling in small lung arteries, suggesting CS1's potential to modify the course of PAH.
Preclinical models of cardiovascular disease have demonstrated that HDAC inhibitors exhibit anti-fibrotic, anti-inflammatory, pulmonary pressure-reducing, and anti-thrombotic effects. CS1's efficacy profile aligns with the underlying mechanisms driving PAH progression, positioning it as a potential solution to the unmet need for more effective treatment options. CS1 has been granted orphan drug status in both the US and EU.
