HAYA Therapeutics' investigational antisense oligonucleotide HTX-001 has been granted FDA Fast Track designation for symptomatic non-obstructive hypertrophic cardiomyopathy (nHCM). The therapy targets the WISPER lncRNA in cardiac myofibroblasts to reprogramme fibrotic, disease-driving cellular states. A Phase Ia/b trial is underway with the first cohort dosed in May, and HAYA raised $65 million in Series A funding in May 2025 to accelerate development. HTX-001 remains unapproved and its safety and efficacy are still being evaluated.
FDA Grants Fast Track To HAYA’s HTX-001 — Precision RNA Therapy For Non-Obstructive HCM
By targeting WISPER in cardiac myofibroblasts
HAYA Therapeutics has received Fast Track designation from the US Food and Drug Administration (FDA) for HTX-001, its investigational antisense oligonucleotide for symptomatic non-obstructive hypertrophic cardiomyopathy (nHCM).
What HTX-001 Does
HTX-001 targets a heart stress-specific long non-coding RNA (lncRNA) called WISPER, which is overexpressed in hypertrophic cardiomyopathy, including non-obstructive forms. By directing the antisense oligonucleotide to cardiac myofibroblasts, the therapy is intended to reprogramme disease-driving fibrotic and pathological cellular states that contribute to myocardial remodelling.
Clinical Progress
The investigational therapy is being evaluated in a Phase Ia/b clinical trial; HAYA reports the first cohort was dosed in May. HTX-001 remains investigational and its safety and clinical benefit have not yet been established.
Comments From Management
Samir Ounzain, CEO and Co-Founder: "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."
Jordan Shin, Chief Medical Officer: "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."
Regulatory Implications
Fast Track designation is intended to expedite the development and review of therapies for serious conditions with unmet medical need. It can enable more frequent interactions with the FDA and may make the programme eligible for certain accelerated development and review pathways, although it does not guarantee approval.
Funding And Next Steps
In May 2025, HAYA Therapeutics raised $65 million in a Series A financing round to accelerate clinical development of HTX-001, including plans for indications related to heart failure. The company will continue its Phase Ia/b trial while engaging with regulators under the Fast Track programme.
Note: HTX-001 has not been approved by the FDA, the European Medicines Agency (EMA), or any other regulatory authority. Ongoing clinical trials will determine its safety and therapeutic benefit.
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