CS1 increases the production of t-PA

The lead drug candidate CS1 is a key reformulation of valproic acid (VPA) and acts as an epigenetic modulator with anti-thrombotic, anti-inflammatory, anti-fibrotic, and pressure reducing properties. CS1’s epigenetic mechanism is expressed through histone deacetylase (HDAC) inhibition and brings a novel treatment approach to cardiovascular diseases.

The current body of evidence supporting CS1’s properties has been provided through a successful Phase I study, but also through in vitro studies, animal models, human physiological data, and independent epidemiology studies. In preclinical studies, CS1 showed an improvement in the endogenous fibrinolytic system by supporting thrombolysis only at the site of the injury with few side effects. With the clinical phase I study, CS1 demonstrated good safety and tolerability, robust reduction of PAI-1 and no problems with bleeding.

Combined, CS1 shows strong promise for a four-fold efficacy:

• Anti-thrombotic
• Anti-inflammatory
• Anti-fibrotic
• Pulmonary pressure reduction


Clinical phase II program

A clinical phase IIa study with CS1 in rare disease pulmonary arterial hypertension (PAH) is expected to be initiated in September 2021. CS1's unique effect profile forms a good match with PAH’s pathogenetic mechanisms and could address the unmet clinical needs within the treatment of the rare disease.

The clinical development program for CS1 in PAH is anchored in the Orphan Drug Designation (ODD) that was granted by the US Food and Drug Administration (FDA) in March 2020. The US FDA grants orphan drug designations to entice the development of products that are intended for the treatment of rare diseases that affect fewer than 200,000 people in the US. Several incentives are associated with ODDs to facilitate the drug development for rare diseases, such as seven years of market exclusivity in the US if the drug is approved, FDA assistance in clinical trial design, and tax credits for qualified clinical trial costs.

With the granted ODD request, CS1 has fulfilled the criteria of showing a potential to provide significant benefit to patients suffering from PAH.

Cereno’s development program for CS1 in thrombotic indication VTE/SPAF is deferred to follow after the Phase II study program in PAH.