As reported on PharmaBiz, HAYA Therapeutics has reached an early clinical milestone with its lead investigational therapy, HTX-001, announcing that the first cohort of participants has been enrolled and treated in a phase 1 trial targeting nonobstructive hypertrophic cardiomyopathy (nHCM). The study marks the company’s initial step in translating its regulatory genome platform into human testing for cardiac disease.
HTX-001 represents a novel therapeutic strategy aimed at addressing fibrosis, a central driver of disease progression in nHCM. The compound is an antisense oligonucleotide designed to suppress WISPER, a long non-coding RNA associated with cardiac stress and overexpressed in hypertrophic cardiomyopathy. By reducing WISPER activity in cardiac myofibroblasts, the therapy seeks to shift these cells away from a pathological, fibrotic state and toward a healthier phenotype. Preclinical findings suggest that this mechanism may help reduce fibrotic remodeling while improving cardiac performance.
The ongoing phase 1a/b trial is structured to assess safety, tolerability, pharmacokinetics, and pharmacodynamics. It will initially enroll healthy volunteers before expanding into patients diagnosed with nHCM, using a multiple ascending dose design.
Nonobstructive hypertrophic cardiomyopathy accounts for a substantial proportion of hypertrophic cardiomyopathy cases—estimated at 30% to 60%. The condition is characterized by thickened ventricular walls, impaired relaxation of the heart muscle, and significant fibrosis. Although advances have been made in understanding the disease biology, current therapies largely fail to directly address the fibrotic processes or diastolic dysfunction underlying disease progression.
Company leadership emphasized the potential clinical importance of directly targeting fibrosis. Chief Medical Officer Jordan Shin noted that patients with nHCM currently lack therapies that modify the disease at its source, underscoring the unmet need for approaches that intervene at the level of fibrosis. By contrast, HTX-001 is intended to act on a key molecular driver rather than only managing symptoms.
The scientific basis for HTX-001 stems from the discovery of WISPER in 2017 by HAYA co-founder and CEO Samir Ounzain, who identified its role in cardiac remodeling. Further development, led by co-founder and Chief Scientific Officer Daniel Blessing, enabled the translation of this insight into a therapeutic candidate designed for clinical application. The company’s broader platform focuses on targeting the regulatory genome to alter disease-associated cellular behavior.
According to Ounzain, this approach reflects a broader shift toward RNA-guided precision therapies capable of reprogramming pathological cell states. In the context of cardiac fibrosis, fibroblasts play a central role in driving tissue remodeling, making them a key target for intervention. HTX-001 is intended to modify the behavior of these cells at the transcriptional level.
The successful dosing of the first cohort represents a key operational milestone for HAYA, signaling progress from laboratory research into clinical development. Blessing highlighted the achievement as validation of the company’s ability to convert discoveries in non-coding RNA biology into therapeutic candidates.
Despite the promise, HTX-001 remains an investigational product. Its safety profile and clinical efficacy are not yet established, and it has not been approved by regulatory authorities such as the US Food and Drug Administration or the European Medicines Agency.
HAYA Therapeutics is headquartered in Lausanne, Switzerland, with additional research operations in San Diego, California. The advancement of HTX-001 into human trials positions the company among a growing group of biotech firms exploring RNA-targeted approaches to treat complex cardiovascular diseases.
