Cereno's HDAC inhibitor lowers pulmonary arterial hypertension risks in phase 2 trial

Cereno Scientific has announced promising results from a phase 2 study of its hypertension drug CS1, aimed at treating pulmonary arterial hypertension (PAH). The study involved 21 patients, and after 12 weeks of treatment with CS1, significant improvements were noted. Specifically, nine patients showed an improved risk score, which is used to measure the risk of death associated with PAH. Additionally, six patients maintained a stable risk score, indicating that overall, 71% of the participants experienced either an improvement or stabilization in their condition.

Moreover, two-thirds of the patients experienced a sustained reduction in pulmonary arterial pressure, a critical factor in PAH management. A third of the participants reported an improved functional class, which refers to their symptom severity and physical capacity.

The trial also met its primary endpoint by demonstrating the safety and tolerability of CS1, with no treatment-related serious adverse events reported. The study did not record any significant changes in liver lab values, clinically significant bleedings, or decreases in platelet counts, indicating a favorable safety profile for CS1.

Following the release of these results, Cereno's shares saw a 25% increase on the Stockholm stock exchange, indicating strong market confidence. The company is now considering moving CS1 into a pivotal trial phase and plans to use these encouraging results as the basis for discussions with regulatory authorities.

Rahul Agrawal, Cereno's Head of R&D, expressed excitement about the findings, emphasizing the potential of CS1 to make a meaningful impact on PAH patients. Agrawal highlighted that the CS1-003 trial showed compelling signs of clinical efficacy over just a 12-week period and anticipated even more positive outcomes with prolonged use.

CS1 functions as a histone deacetylase (HDAC) inhibitor. HDACs play a role in regulating gene expression, and the U.S. Food and Drug Administration (FDA) has already approved several HDAC inhibitors to treat blood cancers. Cereno aims to leverage this mechanism to target the root cause of PAH by reversing the pathological vascular remodeling of the small lung arteries.

PAH is a severe, progressive disease characterized by the narrowing of blood vessels in the lungs, leading to increased blood pressure and heart failure. The PAH treatment market is currently led by Johnson & Johnson with its drugs Uptravi and Opsumit. Recently, Merck & Co. entered the market with the approval of Winrevair, an activin signaling inhibitor that targets the underlying cause of PAH.

Despite the competition, Cereno maintains that existing treatment options are insufficient as they do not address the root cause of the disease. The company is hopeful that CS1 will fill this gap and offer a more effective treatment option for PAH.

Cereno's next steps involve preparing for pivotal trials and engaging with regulatory bodies to discuss the future of CS1. The company’s leadership is optimistic about the potential of CS1 to transform PAH treatment and improve patient outcomes.