Cereno cleared to launch Phase 1 trial of potential PH-ILD treatment CS014
Upcoming study expected to eliminate need for Phase 2a clinical trial
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Cereno Scientific has been cleared to launch a Phase 1 clinical trial in Sweden to evaluate the pharmacological properties of CS014, its experimental oral treatment for pulmonary hypertension associated with interstitial lung disease (PH-ILD).
The upcoming study in healthy volunteers was designed based on feedback from the U.S. Food and Drug Administration (FDA) and is expected to eliminate the need for additional safety studies and a Phase 2a clinical trial.
If all goes as planned, findings will inform the design of a Phase 2b study of CS014 in people with PH-ILD, which is expected to start in early 2027.
“This study represents a regulatory aligned and strategically important step in the continued development of CS014,” Sten R. Sörensen, Cereno’s CEO, said in a company press release. “By incorporating FDA feedback early, we have designed a focused pharmacokinetic program that supports efficient progression toward our planned Phase IIb study in PH-ILD and enables a more streamlined development pathway toward potential marketing approval.”
CS014 inhibits key proteins that regulate gene activity within cells
PH-ILD is characterized by high blood pressure in the vessels that carry blood from the heart to the lungs in people with underlying interstitial lung disease, a group of disorders in which lung tissue becomes inflamed and scarred.
CS014 is a new chemical compound that aims to reduce lung disease processes by inhibiting histone deacetylases (HDACs), proteins that regulate gene activity within cells. These proteins affect how DNA is packaged, which influences which genes are turned on or off in cells.
Preclinical studies have indicated that, by modulating the genetic activity of lung cells, the therapy has potent effects on pathways involved in tissue scarring, blood vessel abnormalities, and abnormal clotting, which are key drivers of disease progression in many lung diseases.
CS014 has completed a Phase 1 study in healthy volunteers, where it showed an acceptable safety profile at multiple doses, either given once or daily for seven days. It also achieved levels in the bloodstream predicted to have a maximal effect on tissue scarring and blood vessel abnormalities.
CS014 builds on our strong expertise in HDAC inhibition.
The upcoming Phase 1 study will again assess the therapy’s safety, but its main goal will be to evaluate CS014’s pharmacokinetic profile — that is, how much of it is absorbed and reaches the bloodstream, how it is distributed throughout the body and metabolized, and how it is excreted.
The study will compare the profile of CS014 against that of valproic acid, a well-studied anti-seizure medication that also works by inhibiting HDACs. A total of 14 healthy volunteers will be enrolled and randomly assigned to receive either CS014 or valproic acid for seven days before switching to the other drug for another seven days.
“CS014 builds on our strong expertise in HDAC inhibition,” said Rahul Agrawal, Cereno’s chief medical officer and head of research and development. “By generating comparative pharmacokinetic data with [valproic acid], this study allows us to leverage the extensive clinical experience of this HDAC inhibitor class while advancing CS014 toward Phase II development in PH-ILD, a serious condition with limited treatment options.”
Cereno is also developing CS014 for idiopathic pulmonary fibrosis, a type of interstitial lung disease. It is also advancing a reformulation of valproic acid called CS1 as a potential treatment for pulmonary hypertension. CS1 has shown positive results in early clinical testing, and an expanded access program is ongoing.
