First patients dosed in hypertrophic cardiomyopathy trial

HAYA Therapeutics has dosed the first cohort in a Phase I trial evaluating a treatment for nonobstructive hypertrophic cardiomyopathy (nHCM). 

The Swiss biotechnology company is studying HTX-001, a differentiated and potentially disease-modifying approach to treating patients with nHCM.  

The investigational molecule is an antisense oligonucleotide designed to downregulate WISPER, a heart stress-specific long non-coding RNA (lncRNA) overexpressed in patients with hypertrophic cardiomyopathy. 

By decreasing WISPER expression in cardiac myofibroblast cells, HTX-001 aims to promote cell-state reprogramming of this fibrotic and pathological cell population back toward a healthy state.  

Preclinical studies have demonstrated that HTX-001 reduces pathological cardiac fibrosis and improves heart function. 

nHCM accounts for an estimated 30-60% of all hypertrophic cardiomyopathy cases. The condition is characterised by increased wall thickness in the heart, hypertrophy in the left ventricular cavity, impaired diastolic function and marked fibrosis.  

While significant progress has been made in understanding the cellular biology underlying nHCM, currently available therapeutic approaches do not directly address the fibrotic pathology or diastolic dysfunction that drive the disease. 

“Our approach is built on our belief that the regulatory genome holds the instructions that drive diseased cell states,” said Dr Samir Ounzain, Co-Founder and CEO of HAYA Therapeutics. 

“With HTX-001, we are translating that biology into an investigational precision RNA-guided therapy that is designed to enable the reprogramming of cardiac fibroblasts, the sentinel effector cells of cardiac fibrosis and pathological remodeling of the myocardium.  

“For patients living with nHCM, where fibrosis remains a driver of disease and current options are limited, our goal is to bring forward a tractable, causal therapeutic approach for an area of significant unmet need.”

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