Cereno Scientific Reports Promising Phase IIa Results for CS1 in Pulmonary Arterial Hypertension
Key Insights
Cereno Scientific (search)'s Phase IIa trial of CS1 for pulmonary arterial hypertension (search) met its primary endpoint of safety and tolerability with no drug-related safety concerns reported.
After 12 weeks of treatment, 40.9% of patients showed improved REVEAL Risk Score 2.0, while 31.8% of patients improved their NYHA/WHO functional class, and 71% reported better quality of life.
The company is now planning a larger, placebo-controlled Phase IIb trial to further evaluate CS1's potential as a first-in-class, disease-modifying therapy targeting the root mechanisms of PAH (search).
Cereno Scientific (search) has presented promising results from its Phase IIa clinical trial evaluating CS1 in patients with pulmonary arterial hypertension (search) (PAH (search)) at the 5th Baltic Pulmonary Hypertension Conference held on May 9, 2025, in Kaunas, Lithuania.
The data, presented in an abstract titled "Exploratory outcomes of CS1 in Pulmonary Arterial Hypertension (search): Phase 2A, Prospective, Randomized, Open-Label, Multicenter Trial," demonstrated that CS1 successfully met its primary endpoint of safety and tolerability with no drug-related safety concerns identified.
Encouraging Efficacy Signals
After 12 weeks of treatment, the study revealed several positive exploratory efficacy outcomes:
- REVEAL Risk Score 2.0 improved from baseline in 40.9% of patients and remained stable in an additional 31.8%
- 31.8% of patients showed improvement in NYHA/WHO functional class, while 54.5% maintained their classification
- 71% of patients reported improved quality of life as measured by the Minnesota Living with Heart Failure Questionnaire (MLHFQ)
"I am pleased that we have now presented the results from our Phase IIa trial of CS1 in PAH (search) to the scientific community in a formal setting," said Rahul Agrawal, CMO and Head of R&D at Cereno Scientific (search). "The Phase IIa trial demonstrated that CS1 is well-tolerated and has a favorable safety profile. Importantly, we observed encouraging signals of reverse vascular remodeling. These exploratory efficacy findings are promising and we are now planning a larger, placebo-controlled Phase IIb trial in PAH to further evaluate these effects."
Addressing Unmet Needs in PAH Treatment
PAH (search) is a rare and progressive disease characterized by high blood pressure in the arteries of the lungs, leading to right heart failure and potentially death. Current treatments primarily focus on symptom management rather than addressing the underlying disease mechanisms.
CS1, an HDAC inhibitor working through epigenetic modulation, is being developed as a potential first-in-class, disease-modifying therapy that targets the root mechanisms driving PAH (search)—pathological vascular remodeling and right heart dysfunction.
"There remains a profound unmet need in PAH (search) for treatments that do more than just manage symptoms," explained Sten R. Sörensen, CEO of Cereno Scientific (search). "With CS1, we are developing a potential first-in-class, disease-modifying therapy that targets the root mechanisms driving PAH—pathological vascular remodeling and right heart dysfunction. The favorable safety and tolerability profile, along with encouraging efficacy signals observed in our clinical development to date, reinforce CS1's potential to transform how PAH is treated."
Expanded Access and Future Plans
Following the completion of the Phase IIa trial, Cereno Scientific (search) has established an Expanded Access Program to enable patients who participated in the study to continue receiving CS1. The company is now preparing for a larger Phase IIb trial to further evaluate the drug's efficacy.
A scientific publication detailing the complete Phase IIa trial results is currently in development and expected to be published during the second half of 2025.
About Cereno Scientific
Cereno Scientific (search) is a biotechnology company pioneering treatments for rare cardiovascular and pulmonary diseases. Beyond CS1, the company's pipeline includes CS014, a new chemical entity with disease-modifying potential currently in Phase I development for conditions such as idiopathic pulmonary fibrosis (search) (IPF (search)). The company is also advancing CS585, a preclinical candidate that is an oral, potent and selective prostacyclin receptor (search) agonist with potential applications in thrombosis prevention and pulmonary hypertension.
Headquartered in Gothenburg, Sweden, with a U.S. subsidiary in Boston, Massachusetts, Cereno Scientific (search) is listed on the Nasdaq First North Growth Market under the ticker symbol CRNO B.