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TuHURA Biosciences Celebrates FDA's Orphan Drug Designation

In a significant advancement in the field of oncology, TuHURA Biosciences, Inc. (NASDAQ: HURA) has announced its receipt of the FDA's Orphan Drug Designation (ODD) for its innovative therapy, IFx-2.0, tailored for patients suffering from stage IIB to IV cutaneous melanoma. This pivotal moment underscores the urgency and need for more effective treatment options in the realm of skin cancers, particularly for those patients who have not responded to existing therapies.

TuHURA Biosciences, established in the immuno-oncology sector and currently in Phase 3 clinical trials, is gaining recognition for developing therapeutics aimed at overcoming the persistent challenges of resistance in cancer immunotherapy. The ODD for IFx-2.0 comes after positive outcomes from a prior Phase 1 study that showcased the drug's potential in enhancing the efficacy of anti-PD1 therapies, notably in patients who qualify as treatment-resistant.

According to the results published in the journal Molecular Therapeutics, led by Dr. Joseph Markowitz at the H. Lee Moffitt Cancer Center, IFx-2.0 appeared safe, with no serious dose-limiting toxicities detected. Encouragingly, the study indicated that patients who were previously unresponsive to checkpoint inhibitor therapy exhibited significant clinical benefits when treated with IFx-2.0 alongside anti-PD1 therapy.

Dr. James Bianco, the President and CEO of TuHURA, stated, “Our immediate goal with IFx-2.0 is to accelerate the enrollment process in our Phase 3 study, where it is being tested in conjunction with Keytruda®, for treating advanced or metastatic Merkel Cell Carcinoma. This ODD designation not only highlights the pressing need for innovative therapies in managing skin cancer but also positions IFx-2.0 as a promising candidate in treating this vulnerable patient demographic.”

The Orphan Drug Designation is particularly noteworthy as it provides numerous incentives for pharmaceutical developers. This designation grants seven years of market exclusivity upon approval, which can greatly enhance the financial feasibility of bringing new drugs to market. Additionally, it includes tax credits for qualifying research expenses, assistance from the FDA, research grants, and a waiver for user fees associated with the New Drug Application process. Such support is vital for companies focused on creating therapies for rare diseases that impact fewer than 200,000 people in the U.S., hence addressing significant medical needs.

TuHURA’s primary focus extends beyond just IFx-2.0. The company is also advancing a Phase 3 randomized, placebo-controlled trial studying IFx-2.0 administered as a complementary treatment to Keytruda® in the first-line management of advanced or metastatic Merkel Cell Carcinoma. This approach illustrates the continued dedication of TuHURA to innovate within the oncological space, amplifying the arsenal against various cancer types.

In addition, following its merger with Kineta Inc. on June 30, 2025, TuHURA has expanded its portfolio to include TBS-2025, which is currently moving into Phase 2 development as a VISTA inhibiting monoclonal antibody aimed at treating mutNPM1 r/r AML (acute myeloid leukemia). Moreover, the company is leveraging its proprietary Delta Opioid Receptor technology to forge new paths in developing bi-specific antibody drug conjugates and peptide conjugates targeting Myeloid Derived Suppressor Cells (MDSCs) to counteract their immunosuppressive effects on tumors, preventing T-cell exhaustion and the recurrence of resistance to therapies.

In conclusion, the recent FDA Orphan Drug Designation granted to TuHURA Biosciences for IFx-2.0 stands as a beacon of hope in the relentless fight against melanoma and other hard-to-treat cancers. As the company continues its mission to break new ground in immuno-oncology, the future looks promising for both TuHURA and the patients that could greatly benefit from this cutting-edge treatment innovation. For further updates, visit TuHURA's official website and stay connected on their social media channels including Facebook, X, and LinkedIn.