Drug that treats nonobstructive HCM gains FDA Fast Track designation
The U.S. Food and Drug Administration (FDA) has granted Fast Track designation for a new type of drug that treats nonobstructive hypertrophic cardiomyopathy (nHCM). These patient accounts for 30-60% of all HCM cases, and there are currently no therapies that directly target the fibrotic pathology or diastolic dysfunction that drives the disease.
Haya Therapeutics SA announced the designation this week for its HTX-001 agent. The first-in-class, investigational, long non-coding RNA (lncRNA)-targeting therapy is current being tested in a Phase 1a/b trial, with the first cohort of patients dosed in May.
The company said its RNA-guided therapeutics target the regulatory genome to reprogram disease-driving cell states. HTX-001 is a potentially disease-modifying antisense oligonucleotide engineered to down-regulate WISPER, (Wisp2 super-enhancer-associated RNA), which is a cardiac fibroblast-enriched lncRNA that regulates cardiac fibrosis. Heart stress-specific lncRNA is overexpressed in hypertrophic cardiomyopathy, including nHCM. By targeting WISPER in cardiac myofibroblasts, the company said HTX-001 is intended to promote the precision reprogramming of this fibrotic and pathological cell state toward a healthy phenotype.
“Patients with nonobstructive HCM are seeking treatments that go beyond management of symptoms. This designation reflects the urgency of delivering better therapeutic options,” said Jordan Shin, MD, PhD, CMO of Haya Therapeutics in a statement. “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, our investigational precision RNA-guided therapy designed to reprogram disease-driving cardiac fibroblasts, the sentinel effector cells which act to drive fibrosis and remodeling in the myocardium.”
The FDA’s Fast Track designation is intended to expedite the development and review of drugs that treat serious conditions and fill an unmet medical need. This designation may enable more frequent interactions with the FDA throughout the drug development process and potentially provides eligibility for accelerated approval, priority review and rolling review.
HTX-001 is an investigational therapy candidate that has not gained clearance from any other regulatory authority. Its safety and ability to translate into clinical benefit is still under investigation.
Dave Fornell has covered healthcare for more than 17 years, with a focus in cardiology and radiology. Fornell is a 5-time winner of a Jesse H. Neal Award, the most prestigious editorial honors in the field of specialized journalism. The wins included best technical content, best use of social media and best COVID-19 coverage. Fornell was also a three-time Neal finalist for best range of work by a single author. He produces more than 100 editorial videos each year, most of them interviews with key opinion leaders in medicine. He also writes technical articles, covers key trends, conducts video hospital site visits, and is very involved with social media. E-mail: [email protected]