HAYA Therapeutics granted Fast Track designation by the FDA for HTX-001

HAYA Therapeutics SA, a clinical-stage biotechnology company based in Lausanne and San Diego, announced that the US Food and Drug Administration (FDA) has granted Fast Track designation to HTX-001, its therapeutic candidate for symptomatic non-obstructive hypertrophic cardiomyopathy (nHCM).

HTX-001 is an antisense oligonucleotide designed to reduce the expression of *WISPER*, a cardiac stress-specific long non-coding RNA (lncRNA) that is overexpressed in hypertrophic cardiomyopathy. By targeting *WISPER* in cardiac myofibroblasts, the treatment aims to reprogramme this fibrotic and pathological cellular state towards a healthy phenotype. It is the first candidate of its kind to target this RNA and is currently being evaluated in a Phase 1a/b trial, the first cohort of which was dosed last May.

“HAYA was founded on the belief that the regulatory genome opens up a new avenue for tackling the underlying biology of the disease,” said Samir Ounzain, Ph.D., CEO and co-founder of HAYA Therapeutics. “This designation recognises the potential of HTX-001 to offer a differentiated therapeutic approach for patients with nHCM.”

Jordan Shin, M.D., Ph.D., the company’s Chief Medical Officer, emphasised the urgency of providing better treatment options: Fast Track designation will enable more frequent interactions with the FDA throughout clinical development.

Fast Track designation aims to accelerate the development and review of treatments addressing unmet medical needs for serious conditions. It paves the way for enhanced interactions with the FDA and potential eligibility for accelerated approval, priority review and rolling review — a major challenge for nHCM, which accounts for 30–60 per cent of cases of hypertrophic cardiomyopathy and for which current treatments do not directly target the fibrosis or diastolic dysfunction underlying the condition.

HTX-001 remains an experimental treatment, not yet approved by the FDA, the European Medicines Agency (EMA) or any other regulatory authority.

➡️ Source: Press Release | 📸 ©Haya Therapeutics