Cereno Scientific receives FDA Fast Track for PAH treatment

Pulmonary arterial hypertension (PAH) is a serious and progressive disease characterized by high blood pressure in the pulmonary arteries, leading to heart failure and premature death. Despite available treatments, there is still a great need for safer and more effective therapies that can modify the course of the disease.

Unique mechanism of action through epigenetic modulation

CS1 is an oral HDAC (histone deacetylase) inhibitor that works through epigenetic modulation, meaning the drug affects how genes are expressed without changing the DNA sequence itself. This mechanism differs from existing PAH treatments that focus primarily on widening blood vessels.

In a phase IIa study, CS1 showed promising efficacy signals, including regression of vascular changes and improvement of right ventricular function in PAH patients.

Sten R. Sörensen, CEO of Cereno Scientific, comments on the FDA announcement:

- We are very pleased to have achieved Fast Track designation for CS1, which both validates the strength of our CS1 program and supports our mission to bring pioneering treatments to patients with devastating rare diseases like PAH.

Fast Track status accelerates development

The Fast Track program is designed to facilitate the development and expedite the review of drugs that address serious diseases with high medical needs. The status provides Cereno Scientific with several benefits, including more frequent communication with the FDA, the opportunity for an accelerated approval process, and priority review of the marketing authorization application.

For patients with PAH, this potentially means faster access to a new treatment that could improve quality of life and survival. The Fast Track status also signals to investors and partners that the FDA sees potential in CS1 as a treatment option.

Preparing for a global phase IIb study

Cereno Scientific is now preparing a global phase IIb study to further evaluate CS1 as a treatment for PAH. The planned phase IIb study will be a global, multicenter, placebo-controlled study designed to further evaluate the promising efficacy signals observed in the phase IIa study.

Rahul Agrawal, MD, CMO & Head of R&D at Cereno Scientific, explains:

- The Fast Track designation for CS1 underscores the FDA’s recognition of its potential to address the significant unmet need in PAH. Fast Track designation will enable closer interaction with the FDA, enabling timely feedback on our development plans as we advance CS1 into its Phase IIb trial and beyond. This designation marks an important step in the development journey for CS1 and supports our goal of making innovative treatment options available to PAH patients as efficiently as possible.