TuHURA gains FDA orphan drug designation for IFx-2.0 in cutaneous melanoma

TuHURA Biosciences said the U.S. Food and Drug Administration's Office of Orphan Products Development granted Orphan Drug Designation to IFx-2.0 for the treatment of stage IIB to stage IV cutaneous melanoma. The designation was based on data from the company's previously completed Phase 1 study of IFx-2.0, while the company said its Phase 3 study of IFx-2.0 in combination with Keytruda for the first-line treatment of advanced or metastatic Merkel cell carcinoma continues to enroll.

The company said the Phase 1 study results were published in Molecular Therapeutics under the title "First-in-Human Stage II/IV Melanoma Clinical Trial of Immune Priming Agent IFx-Hu2.0." The study demonstrated IFx-Hu2.0 to be safe with no serious dose limiting toxicities and showed that patients refractory to checkpoint inhibitor therapy (anti-PD1) experienced clinical benefit upon subsequent anti-PD1 based treatment.

TuHURA said orphan drug designation provides seven years of market exclusivity benefits, increased engagement and assistance from the FDA, tax credits for certain research, research grants and a waiver of the New Drug Application user fee. The FDA's Office of Orphan Products Development grants orphan status to drugs intended for the safe and effective treatment, diagnosis or prevention of rare diseases or conditions affecting fewer than 200,000 individuals in the United States.

In a corporate update on April 1, 2026, the company said its IFx-2.0 Phase 3 study in front-line Merkel cell carcinoma continues to enroll and that it anticipates completing enrollment in mid-2027. TuHURA also said TBS-2025, a VISTA inhibiting mAb acquired in its merger with Kineta on June 30, 2025, is moving into Phase 2 development in mutNPM1 r/r AML, and that it continues to advance preclinical work in its bi-specific antibody drug conjugates program.

In first-quarter 2026 results reported on May 15, 2026, TuHURA said a $50 million credit facility and royalty transaction announced in April extends its projected cash runway into 2028 and provides flexible, non-equity operating capital. The company reported cash and cash equivalents of $6.3 million as of March 31, 2026, and approximately 63.6 million common shares outstanding.

For the full year ended December 31, 2025, TuHURA reported cash and cash equivalents of $3.6 million, with an additional $7.5 million received in the first quarter of 2026 from the 2025 registered direct offering. Research and development expenses were $20.5 million for 2025 versus $13.3 million for 2024, general and administrative expenses were $7.6 million versus $3.9 million, net cash outflows from operating activities were $27.7 million versus $14.7 million, and net cash flows from financing activities were $19.9 million versus $29.7 million.