HAYA Therapeutics has announced full enrolment and dosing of the first cohort in its Phase I clinical trial of HTX-001, a long non-coding ribonucleic acid (lncRNA)-targeting therapy developed for non-obstructive hypertrophic cardiomyopathy (nHCM).
HTX-001 represents an approach aimed at modifying disease by targeting Wisper, a heart stress-specific lncRNA overexpressed in hypertrophic cardiomyopathy patients, including those with nHCM.
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The antisense oligonucleotide intends to decrease Wisper expression in cardiac myofibroblast cells, promoting the reprogramming of the fibrotic and pathological cells toward a healthier state.
Preclinical results have shown that HTX-001 reduces pathological cardiac fibrosis and improves heart function.
The move to clinical evaluation for HTX-001 follows the foundational research led by Haya Therapeutics co-founder and CEO Samir Ounzain, who identified the role of Wisper in cardiac fibrosis and remodelling in 2017.
The company’s co-founder and CSO, Daniel Blessing, led the research and development to advance HTX-001 from concept to clinical trial.
Haya Therapeutics CMO Jordan Shin said: “For patients diagnosed with nHCM, this clinical study marks a major milestone. A targeted anti-fibrotic therapy for nHCM offers the potential to address an important unmet medical need, as currently available treatments fail to address the underlying fibrotic process that drives disease.”
nHCM is characterised by thickening of the heart wall, hypertrophy in the left ventricular cavity, impaired diastolic function and significant fibrosis.
Dr Blessing said: “This is a meaningful milestone for the entire Haya team as we advance our science from the laboratory into clinical development. Dosing of the first cohort reflects the significant progress we have made in translating our RNA-guided regulatory genome platform into an investigational first-in-class therapeutic candidate for patients.”
The Phase Ia/b study will assess the tolerability, safety, pharmacodynamics, and pharmacokinetics of HTX-001 in healthy volunteers and nHCM patients across multiple ascending-dose cohorts.
HTX-001 remains investigational and is not approved by the US Food and Drug Administration, European Medicines Agency, or any other regulatory authority. Its safety and clinical benefits are yet to be established.
