HAYA Therapeutics has received FDA Fast Track designation for HTX-001, an investigational therapy being developed for symptomatic nonobstructive hypertrophic cardiomyopathy.

The RNA-based therapy is currently being evaluated in a Phase 1a/b clinical trial, with the first patient cohort dosed in May.

HTX-001 is an antisense oligonucleotide designed to target WISPER, a long non-coding RNA that HAYA said is overexpressed in hypertrophic cardiomyopathy.

The company is developing the therapy to target fibrosis and pathological changes in cardiac tissue associated with nonobstructive hypertrophic cardiomyopathy, or nHCM.

The FDA Fast Track designation is intended to support the development and review of therapies for serious conditions where there is an unmet medical need.

The designation can provide opportunities for more frequent interactions with the FDA during development and may allow eligible therapies to benefit from other regulatory pathways, including rolling review and priority review.

HTX-001 targets fibrosis in nonobstructive HCM

Nonobstructive hypertrophic cardiomyopathy is a form of hypertrophic cardiomyopathy in which the heart muscle becomes abnormally thick and stiff without an obstruction that limits blood flow from the heart.

The condition can involve impaired diastolic function and fibrosis, which can affect the heart’s ability to function effectively.

HAYA estimates that nHCM accounts for between 30% and 60% of hypertrophic cardiomyopathy cases.

The company said current treatment approaches do not directly target the fibrotic pathology or diastolic dysfunction associated with nHCM, creating a potential opportunity for therapies aimed at the underlying disease biology.

HTX-001 is designed to reduce the activity of WISPER in cardiac myofibroblasts. HAYA said this approach is intended to alter the pathological cell state associated with fibrosis and cardiac remodelling.

Samir Ounzain, CEO and co-founder of HAYA Therapeutics, said: “This regulatory designation recognizes the potential of HTX-001 to offer a differentiated therapeutic approach for patients with nHCM.”

The Phase 1a/b study is evaluating the investigational therapy in patients with nHCM, with the first cohort receiving treatment in May.

Jordan Shin, chief medical officer of HAYA Therapeutics, said: “With our Phase 1 clinical trial already underway, Fast Track designation allows us to work more closely with the FDA as we advance HTX-001.”

The company said the designation could support interactions with the FDA as clinical development progresses.

FDA designation supports early clinical development

Fast Track is one of several regulatory mechanisms available to support the development of therapies intended to address serious conditions and unmet medical needs.

It does not mean that a therapy has been shown to be effective or safe, nor does it guarantee regulatory approval.

HTX-001 remains investigational and has not been approved by the FDA, the European Medicines Agency or any other regulatory authority.

The safety of HTX-001 and its ability to provide a clinical benefit in patients with nHCM have yet to be established.

HAYA is developing HTX-001 as a potential disease-modifying approach based on targeting the regulatory genome, with the company focusing on long non-coding RNA as a potential therapeutic target.

The company’s approach is intended to address biological processes involved in disease rather than focusing solely on symptom management.

The Phase 1a/b trial will provide the first clinical data to assess the safety and tolerability of HTX-001 and help inform its future development.

The Fast Track designation comes as HAYA advances the therapy through early-stage clinical development, with further data from the Phase 1 programme expected to determine whether the approach can progress into later-stage testing.

The company said its work is focused on developing RNA-guided therapies for chronic and age-related diseases by targeting regulatory mechanisms involved in disease-driving cell states.

For patients with nHCM, the development of treatments aimed at the fibrotic component of the disease could represent a potential new therapeutic approach if HTX-001 is shown to be safe and effective in clinical studies.