Phase 1 study of CS014 in healthy volunteers moves to last part
Therapy has shown it can reverse PAH fibrosis, ease blood vessel remodeling
A Phase 1 clinical trial testing Cereno Scientific’s candidate therapy for idiopathic pulmonary fibrosis (IPF) in healthy volunteers has moved to its final part, the company has announced.
This first-in-human, open-label trial is testing CS014 at single and multiple ascending doses in about 48 participants. The part that evaluated the safety, tolerability, and pharmacokinetics of single ascending doses in 30 participants is now complete. Pharmacokinetics refers to the movement of a medicine into, through, and out of the body.
According to Cereno, CS014 showed no safety concerns and the data supports its clinical development. The trial is now in its multiple ascending dose phase, which is expected to be completed by June.
“We are pleased with the progress of the CS014 Phase I trial and eagerly anticipate its completion in mid-2025. The successful completion of the single ascending dose (SAD) part provides a strong initial validation of [CS014] favorable safety profile,” Rahul Agrawal, MD, Cerenoo’s chief medical officer and head of research and development, said in a company press release.
What does CS014 do in IPF?
CS014 works by inhibiting the activity of enzymes called histone deacetylases (HDAC), which are responsible for removing acetyl groups from histones, that is, proteins involved in packaging DNA within cells.
Preclinical studies support the therapeutic potential of HDAC inhibitors (HDACi) in IPF models, according to Cereno. Specifically, CS014 has shown it can reverse scarring (fibrosis) and ease blood vessel remodeling — in a dose-dependent manner — in an established preclinical model of pulmonary arterial hypertension. Also, it’s been shown to regulate blood clotting, which becomes deregulated in pulmonary fibrosis due to a combination of abnormal coagulation, an imbalance in blood clot breakdown, called fibrinolysis, and chronic inflammation.
CS014 was shown to regulate the activity of platelets, tiny cell fragments involved in blood clotting, local fibrinolysis, and blood clot stability. Its activity overall helps prevent the risk of thrombosis, or blood clot formation, without increasing the risk of bleeding.
“We believe that our novel HDACi CS014 has the potential to become an important treatment, meeting high unmet clinical needs in the rare disease IPF,” Sten R. Sörensen, Cereno’s CEO, said. “There is a void in the market for safe and well-tolerated novel drugs with a profile addressing the underlying pathophysiology of the IPF disease and its progression. CS014 aims to be a safe, well tolerated oral drug with disease-modifying capacity and, as such, targets a large market potential.”
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