Cereno Scientific has taken a significant step forward in the development of its lead drug candidate CS1 by submitting a Type C meeting request to the U.S. Food and Drug Administration (FDA). This strategic move follows encouraging results from the company's Phase IIa trial investigating CS1 as a treatment for pulmonary arterial hypertension (PAH).
Phase IIa Success Drives Development Forward
The Phase IIa trial demonstrated that CS1, an HDAC inhibitor (HDACi), successfully met its primary endpoint of safety and tolerability. More importantly, the drug showed compelling positive impacts on exploratory parameters of clinical relevance during the 12-week treatment period. These results, combined with preclinical data, suggest CS1's potential for reverse remodeling in small lung arteries, indicating possible disease-modifying capabilities.
"We look forward to a discussion with the FDA following our successfully completed Phase IIa trial with CS1," stated Rahul Agrawal, CMO & Head of R&D at Cereno Scientific. "This meeting will be a valuable opportunity as it helps ensure that CS1's clinical development program is aligned with FDA expectations."
Innovative Mechanism of Action
CS1 represents a novel approach to PAH treatment through epigenetic modulation. The drug targets the root mechanism of the disease, aiming to reverse the pathological vascular remodeling of small lung arteries. Preclinical studies have shown that HDAC inhibitors demonstrate multiple beneficial effects, including anti-fibrotic, anti-inflammatory, pulmonary pressure-reducing, and anti-thrombotic properties.
Expanded Access Program Provides Additional Data
Currently, ten patients are enrolled in an FDA-approved Expanded Access Program (EAP) as an extension of the Phase IIa trial. This program allows Phase IIa trial completers to continue CS1 treatment when deemed appropriate by study physicians. The company has partnered with Fluidda to utilize their Functional Respiratory Imaging (FRI) technology, enabling visualization of how long-term CS1 use affects pulmonary arteries.
Strategic Importance for PAH Treatment
"This is another major milestone for our lead program CS1 in the rare disease PAH," said Sten R. Sörensen, CEO at Cereno Scientific. "CS1 has the potential to transform the treatment approach of PAH by providing a safe and well-tolerated therapeutic alternative with disease-modifying capacity."
The FDA Type C meeting, scheduled to occur within 75 days of the request submission, will evaluate CS1's risk-benefit profile and provide specific feedback on the development plan. This guidance will be crucial for planning future Phase IIb or pivotal Phase III trials.
Regulatory Recognition
The drug's potential has already been recognized by regulatory authorities, with CS1 receiving orphan drug status in both the US and EU. This designation acknowledges the significant unmet need in PAH treatment and supports the development of more effective therapeutic options for patients with this rare cardiovascular disease.
