Halia Therapeutics Receives FDA Orphan Drug Designation for Ofirnoflast in Myelodysplastic Syndromes

Halia Therapeutics, Inc., a clinical-stage biopharmaceutical company focused on therapies that target the root causes of inflammation, announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to ofirnoflast (HT-6184) for the treatment of Myelodysplastic Syndromes (MDS), a group of rare bone marrow disorders that can progress to acute myeloid leukemia (AML).

“This designation underscores the potential of our approach in MDS and supports our commitment to developing new treatment options,” said David Bearss, PhD, CEO of Halia Therapeutics. “Ofirnoflast represents a first-in-class strategy to modulate inflammasome biology, restoring healthy bone marrow function.”

About Ofirnoflast (HT-6184)

Ofirnoflast is a selective NEK7 allosteric modulator designed to prevent formation and promote disassembly of the NLRP3 inflammasome, a central driver of chronic inflammation. In MDS, inflammasome activation contributes to ineffective blood-cell production. By targeting NEK7, ofirnoflast aims to restore immune balance and improve hematopoiesis without broad immunosuppression.

“Ofirnoflast’s approach addresses the underlying inflammatory drivers of MDS, rather than just downstream effects, potentially redefining treatment for inflammation-linked bone marrow failure,” said Alan F. List, MD, member of Halia’s Scientific Advisory Board.

Benefits of Orphan Drug Designation

FDA orphan-drug status provides incentives including tax credits for clinical testing, exemption from FDA user fees, and potential seven-year U.S. market exclusivity upon approval.

About Myelodysplastic Syndromes (MDS)

MDS primarily affects older adults, causing defective blood-cell formation, anemia, infection risk, and bleeding complications. Existing treatments often provide limited benefit and do not address the inflammatory mechanisms driving the disease.

About Halia Therapeutics

Halia Therapeutics leverages the NEK7–NLRP3 inflammasome axis to develop therapies for chronic inflammatory and degenerative diseases, including hematologic disorders, metabolic disease, and neurodegeneration.

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