HAYA Therapeutics announced that the first cohort has been fully enrolled and dosed in its Phase 1 clinical trial of HTX-001, an investigational antisense oligonucleotide that targets the WISPER long non-coding RNA, according to the company.
The molecule is designed to downregulate WISPER in cardiac myofibroblasts to promote cell-state reprogramming away from a fibrotic phenotype; the company claims preclinical studies show reductions in pathological cardiac fibrosis and improvements in heart function.
The Phase 1a/b study will evaluate safety, tolerability, pharmacokinetics and pharmacodynamics across multiple ascending-dose cohorts, beginning in healthy volunteers and proceeding to patients with nonobstructive hypertrophic cardiomyopathy, or nHCM.
The company notes that nHCM is estimated to account for 30 to 60 percent of hypertrophic cardiomyopathy cases and is characterized by marked fibrosis and diastolic dysfunction.
HTX-001 remains investigational and is not approved by the U.S. Food and Drug Administration, the European Medicines Agency or other regulatory authorities.
