FDA Grants Fast Track Designation to Cereno for PAH Therapy

Rare Daily Staff

The U.S. Food and Drug Administration has granted Cereno Scientific’s lead experimental therapy, CS1, Fast Track designation for the treatment of pulmonary arterial hypertension.

Fast Track designation is designed to accelerate the development and regulatory review of new therapies for serious conditions with high unmet medical need. The designation highlights CS1’s potential as a differentiated treatment approach for PAH, a rare and progressive disease where safer, disease-modifying therapies are urgently needed.

Pulmonary arterial hypertension (PAH) is a rare, progressive disease that affects the blood vessels in the lungs, leading to high blood pressure in the pulmonary circulation. In most cases, the cause is unknown. The disease is marked by thickening and narrowing of the small arteries in the lungs, including the development of characteristic plexiform lesions, which restrict blood flow from the right side of the heart to the lungs. Over time, these changes, combined with increased tissue scarring, reduce the elasticity of the blood vessels and increase resistance to blood flow. This process, known as vascular remodeling, raises the pressure in the pulmonary arteries and impairs circulation. In later stages, small blood clots may form locally, further worsening the condition. Ultimately, most patients develop right heart failure because the heart can no longer cope with the strain, which leads to death in most patients with PAH.

CS1 is an oral HDAC inhibitor with a unique mechanism of action through epigenetic modulation. In a phase 2a trial in PAH, CS1 met its primary endpoint of safety and tolerability while showing encouraging efficacy signals, including improvement of REVEAL risk score, functional class, quality of life, and early signs of reverse vascular remodeling, and improvement of right heart function.

A global phase 2b trial is being prepared to further evaluate CS1 as a potential disease-modifying treatment for PAH.

“The Fast Track designation for CS1 underscores the FDA’s recognition of its potential to address the significant unmet need in PAH,” said Rahul Agrawal, head of research and development at Cereno Scientific. “Fast Track designation will enable closer interaction with the FDA, enabling timely feedback on our development plans as we advance CS1 into its phase 2b trial and beyond.”

Photo: Rahul Agrawal, head of research and development at Cereno Scientific