HAYA Therapeutics has secured fast track designation from the US Food and Drug Administration (FDA) for its investigational therapy, HTX-001, to treat symptomatic non-obstructive hypertrophic cardiomyopathy (nHCM).
Targeting long non-coding ribonucleic acid (lncRNA), HTX-001 is being evaluated in a Phase Ia/b clinical trial. The first cohort in this trial was dosed in May.
This antisense oligonucleotide has been developed to suppress WISPER, a heart stress-specific lncRNA that is overexpressed in hypertrophic cardiomyopathy, including nHCM.
By targeting this lncRNA in cardiac myofibroblasts, the therapy is intended to facilitate the precise reprogramming of these fibrotic and pathological cell states.
HAYA Therapeutics CEO and co-founder Samir Ounzain said: “HAYA was founded on the belief that the regulatory genome offers a new way to address the underlying biology of disease. This regulatory designation recognises the potential of HTX-001 to offer a differentiated therapeutic approach for patients with nHCM.”
HAYA Therapeutics chief medical officer Jordan Shin said: “Patients with non-obstructive HCM are seeking treatments that go beyond management of symptoms. This designation reflects the urgency of delivering better therapeutic options.
“With our Phase I clinical trial already underway, fast track designation allows us to work more closely with the FDA as we advance HTX-001, our investigational precision RNA-guided therapy designed to reprogramme disease-driving cardiac fibroblasts—the sentinel effector cells that act to drive fibrosis and remodelling in the myocardium.”
The designation seeks to expedite the development and review of drugs intended for serious conditions where there is an unmet medical need.
It may result in more frequent interactions with the FDA and eligibility for various expedited review processes.
HTX-001 has not received approval from the FDA, European Medicines Agency (EMA), or any other regulatory authority. Its safety and potential to provide clinical benefit are yet to be determined.
In May 2025, Haya Therapeutics procured $65m in Series A funding to expedite the clinical development of HTX-001 for treating heart failure.
