IPF treatment shows early promise in trial for reversing scarring
CS014 found safe, well tolerated in healthy volunteers in Phase 1 study
CS014, a treatment candidate for idiopathic pulmonary fibrosis (IPF), was found to be safe and well tolerated in healthy volunteers in a Phase 1 clinical trial, according to new data from developer Cereno Scientific.
Further, the experimental medication reached levels in the bloodstream that the company predicts will be effective in reversing both scarring, called fibrosis, and structural alterations to blood vessels in the lungs, known as vascular remodeling.
Cereno now says it plans to advance CS014 to a Phase 2 clinical trial in the first half of 2026.
“We are very pleased with the outcome of this study,” Rahul Agrawal, Cereno’s chief medical officer and head of research and development, said in a company press release. “These findings position CS014 as a promising candidate in IPF and other rare diseases involving vascular remodeling and fibrosis.”
The Phase 1 trial, which took place in Sweden, evaluated the safety of single and multiple doses of CS014 in people without IPF, a respiratory disease with no known cause. The trial also tested the medication’s pharmacokinetics, or how it moves into, through, and out of the body, and its pharmacodynamics, or the drug’s effects on the body.
In IPF, the most common form of pulmonary fibrosis, scarring in the lungs occurs for unknown reasons. It leads to symptoms like shortness of breath and a dry, hacking cough. Fibrosis can also lead to problems with blood clotting, excessive inflammation, and vascular remodeling, or structural changes in blood vessels.
With CS014, Cereno aims to reverse fibrosis through an epigenetic process — that is, changing gene expression without altering the underlying sequence of DNA. The therapy targets a class of proteins called histone deacetylases (HDACs). HDACs impact how cells pack genetic material, influencing which genes are turned into proteins.
“CS014 is a novel HDAC inhibitor with a unique mechanism of action through epigenetic modulation that could represent a differentiated treatment approach for IPF,” said Sten R. Sörensen, Cereno’s CEO. In preclinical models, the therapy had a significant impact on both fibrosis and vascular remodeling, according to the company, which noted that those findings suggest CS014 could be disease-modifying.
Trial tested effects of IPF treatment in 48 healthy volunteers
A total of 48 healthy volunteers took single or multiple ascending doses of CS014 in two study parts. The multiple ascending doses of CS014 were given over seven days.
Results showed the investigational treatment was safe, with no serious adverse events occurring. All treatment-related side effects were mild and temporary, and all participants completed the study.
These positive Phase [1] results, combined with strong nonclinical data, give us confidence as we advance into Phase [2] clinical development.
The therapy reached and exceeded concentrations that the company predicts will have therapeutic effects.
“Notably, CS014 was safe and well tolerated at exposure levels that are expected to be sufficient to impact pathological [disease-related] pulmonary vascular remodeling and reduction of fibrosis — key drivers in several rare cardiovascular and pulmonary diseases,” Agrawal said.
For his part, Sörensen noted that the company is “excited to continue pioneering treatments to improve the lives of patients in these diseases with high unmet medical needs.”
“These positive Phase I results, combined with strong nonclinical data, give us confidence as we advance into Phase II clinical development,” Sörensen said.
In addition to CS014, Cereno is developing CS1, another HDAC inhibitor intended to treat pulmonary arterial hypertension. The U.S. Food and Drug Administration recently aligned with the company on plans for a CS1 Phase 2b trial.
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