FDA Grants Fast Track Designation to HAYA’s HTX-001 for nHCM

HTX-001, an investigational RNA-guided therapy targeting cardiac fibrosis, has received FDA Fast Track designation for symptomatic nonobstructive hypertrophic cardiomyopathy. The designation supports expedited development of a first-in-class approach that targets the disease biology rather than symptom control.

The U.S. Food and Drug Administration (FDA) has granted Fast Track designation to HTX-001, HAYA Therapeutics’ investigational antisense oligonucleotide therapy, for the treatment of symptomatic nonobstructive hypertrophic cardiomyopathy (nHCM). The designation recognizes the therapy’s potential to address a serious condition with significant unmet medical need and enables closer regulatory interactions as the clinical program advances.

HTX-001 Targets the Fibrotic Biology of nHCM

Unlike currently available therapies that primarily relieve symptoms or improve cardiac function, HTX-001 targets the underlying fibrotic remodeling that contributes to disease progression in nHCM.

The investigational therapy is a first-in-class antisense oligonucleotide that downregulates WISPER, a long non-coding RNA (lncRNA) highly expressed in cardiac myofibroblasts. By suppressing WISPER, HTX-001 aims to reprogram disease-driving fibroblasts toward a healthier cellular state, reducing myocardial fibrosis and adverse cardiac remodeling.

Nonobstructive hypertrophic cardiomyopathy is characterized by thickening of the heart muscle without left ventricular outflow tract obstruction. Patients often experience exercise intolerance, shortness of breath, fatigue, arrhythmias, and progressive heart failure. Despite recent advances in hypertrophic cardiomyopathy treatment, disease-modifying therapies specifically targeting fibrosis remain unavailable.

 Phase 1 Trial Is Underway

HTX-001 is currently being evaluated in an ongoing Phase 1a/b clinical trial. The study is assessing the therapy’s safety, tolerability, pharmacokinetics, and pharmacodynamics through multiple ascending-dose cohorts.

The trial initially enrolls healthy volunteers before expanding into patients with symptomatic nHCM. HAYA previously reported that the first study cohort had been fully enrolled and dosed in May 2026, marking the start of clinical evaluation for the investigational therapy.

FDA Designation Supports Development

Fast Track designation allows more frequent interactions with the FDA during drug development and may provide eligibility for rolling review, Priority Review, and Accelerated Approval if applicable regulatory criteria are met. The designation does not constitute marketing approval or establish the therapy’s safety or efficacy.

Jordan Shin, MD, PhD, Chief Medical Officer of HAYA Therapeutics, said patients with nonobstructive HCM continue to need therapies that address more than symptom management. He noted that the designation will strengthen collaboration with the FDA as HTX-001 advances through clinical development.

Samir Ounzain, PhD, Chief Executive Officer and Co-founder of HAYA Therapeutics, said the regulatory milestone supports the company’s strategy of developing RNA-guided therapies that target disease-driving mechanisms within the regulatory genome and reinforces the potential of HTX-001 as a differentiated therapeutic approach for patients with nHCM.

Path Forward

HAYA Therapeutics will continue enrolling participants in the ongoing Phase 1a/b study while working closely with the FDA under the Fast Track program. Early clinical data from the study are expected to guide subsequent development and determine whether HTX-001 can progress into later-stage trials evaluating its ability to reduce cardiac fibrosis and improve outcomes for patients with symptomatic nonobstructive hypertrophic cardiomyopathy.

Reference

HAYA Therapeutics Receives FDA Fast Track Designation for HTX-001 – HAYA Therapeutics