Clinical-stage biotechnology company Halia Therapeutics Inc announced on Monday that the US Food and Drug Administration (FDA) has granted Fast Track designation to ofirnoflast (HT-6184) for the treatment of adult patients with lower-risk myelodysplastic syndromes (LR-MDS).

Fast Track is an FDA process designed to facilitate the development and expedite the review of therapies intended to treat serious conditions and address an unmet medical need. A drug that receives Fast Track designation is eligible for more frequent interactions with the FDA and may be eligible for Accelerated Approval, Priority Review, and Rolling Review if relevant criteria are met.

Halia says that ofirnoflast is a first-in-class oral NEK7 allosteric modulator designed to prevent formation and promote disassembly of the NLRP3 inflammasome, a key regulator of innate immune signalling and chronic inflammation. In lower-risk MDS, NLRP3 inflammasome activation contributes to ineffective haematopoiesis, bone marrow dysfunction, and anaemia. By modulating NEK7, ofirnoflast is designed to address an underlying driver of disease.

"Receiving Fast Track designation is an important milestone for Halia and reflects the significant unmet need that remains for patients with lower-risk MDS," said David J. Bearss, Ph.D., Halia Therapeutics chief executive officer. "The designation follows encouraging Phase 2 results demonstrating durable transfusion independence and multilineage haematologic improvement and provides an important opportunity to work closely with the FDA as we advance ofirnoflast toward pivotal development. Patients who no longer respond to erythropoiesis-stimulating agents or erythroid maturation agents often face limited options and a substantial burden from chronic anaemia and transfusion dependence."

Halia is currently planning the next stage of clinical development for ofirnoflast in lower-risk MDS, and intends to leverage the benefits associated with Fast Track designation as the programme advances.