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FDA Grants Orphan Drug Status to Experimental Melanoma Therapy After Phase 1 Results

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Source: Streetwise Reports (2/6/26)

TuHURA Biosciences Inc. (HURA:NASDAQ) said the FDA granted Orphan Drug Designation to IFx-2.0 for stage IIB to stage IV cutaneous melanoma. The designation was based on data from the companys previously completed Phase 1 clinical study.

TuHURA Biosciences Inc. (HURA:NASDAQ) announced that 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.

According to the company, results from the Phase 1 study were published in the journal Molecular Therapeutics under the title "First-in-Human Stage II/IV Melanoma Clinical Trial of Immune Priming Agent IFx-Hu2.0," authored by Dr. Joseph Markowitz of the Department of Cutaneous Oncology at H. Lee Moffitt Cancer Center and Research Institute. The study reported that IFx-Hu2.0 was safe with no serious dose-limiting toxicities and that patients refractory to checkpoint inhibitor therapy experienced clinical benefit upon subsequent anti-PD1-based treatment.

Dr. James Bianco, President and Chief Executive Officer of TuHURA Biosciences, said in a company news release, "Our current focus with IFx-2.0 is targeting completion of enrollment in our Phase 3 study of IFx-2.0 in combination with Keytruda® for the first-line treatment of advanced or metastatic Merkel Cell Carcinoma." He added, "We believe receiving ODD in advanced cutaneous melanoma demonstrates not only the significant need for new treatments in skin cancer but also highlights IFx-2.0 as a potential new therapeutic approach in this patient population."

The company stated that 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 grants orphan status to drugs intended for the treatment of rare diseases affecting fewer than 200,000 individuals in the United States.

Immuno-Oncology in Focus as Biopharma Regains Momentum

BioPharma Dive reported on January 29 that the biopharmaceutical sector regained momentum toward the end of 2025 following a prolonged downturn. The article stated that a wave of dealmaking, financings, and positive clinical trial readouts helped lift drug companies and revive investor interest. However, it also noted ongoing challenges, including an unpredictable U.S. regulatory environment and intensifying competition from China, with industry participants debating the durability of the sector's rebound during the January J.P. Morgan Healthcare Conference.

According to a February 3 report from BioProcess Insider, immuno-oncology continued to be a significant focus within the biotechnology industry, particularly for companies advancing complex biologic platforms. The article highlighted the importance of manufacturing scale and execution in this segment, quoting HanchorBio founder and CEO Scott Liu as saying that "execution speed, manufacturing reliability, and scalability are critical" for advancing multiple immunotherapy programs. The report also noted that antibody-based biologics, including bi-specific antibodies and antibody-drug conjugates, represented a substantial portion of current development activity in the sector.

Precedence Research wrote on February 4, that the global biopharmaceutical industry continued to experience strong growth, with oncology identified as one of the leading areas of innovation. The research firm stated that companies were investing heavily in biologics and immuno-oncology, with efforts focused on developing more targeted and personalized cancer treatments. The report also noted that biologics played "an increasingly important role" across oncology, immunology, and rare diseases, reflecting broader industry emphasis on immune-based and precision medicine approaches.

Analysts Assign Buy Ratings and Price Targets Up to $10 Following Orphan Drug Designation

In a December 22 report, Zacks Investment Research described TuHURA Biosciences as "a clinical-stage, oncology-focused biotechnology company advancing innate immune agonists, checkpoint inhibitors & antibody-drug conjugates (ADCs)." Zacks stated that "IFx-2.0 uses pDNA to encode the production of Emm55 on cancer cells to elicit an immune response." The firm assigned a valuation of US$7.50 per share and wrote that its model applied "a success probability of 60% to the IFx-2.0 program in MCC and 15% to the anti-VISTA program in AML, generating a 50% blended rate."

Zacks also reported on TuHURA's delta opioid receptor program following presentations at the American Society of Hematology Annual Meeting in December 2025. According to Zacks, data presented by the company showed that "pharmacological antagonism of the DOR reduced the suppressive activity of MDSCs." Zacks further noted that a poster presentation concluded that "targeting the DOR may be a strategy to address several vulnerabilities," including reprogramming suppressive tumor-associated macrophages and alleviating T-cell dysfunction.

In a February 3 research note, H.C. Wainwright & Co. reiterated its Buy rating on TuHURA Biosciences with a 12-month price target of US$10.00 per share. The firm wrote that "prior data showed a 50% response rate in anti-PD1 refractory stage III/IV melanoma patients treated with IFx-2.0 followed by anti-PD1 therapy." H.C. Wainwright stated, "We reiterate our Buy rating and 12-month price target of $10." The firm also noted that its valuation applied "a 45% probability of approval for IFx-2.0 in MCC and a 20% probability of approval for the company's anti-Vista mAb in AML," and added, "We do not currently include risk-adjusted revenue projections for IFx-2.0 in melanoma in our discounted cash flow (DCF)-based valuation."

Clinical and Development Milestones

TuHURA reported that IFx-2.0 is being evaluated in a randomized, placebo-controlled Phase 3 registration trial in combination with Keytruda® compared to Keytruda® plus placebo in the first-line treatment of advanced or metastatic Merkel Cell Carcinoma. The trial is being conducted under a Special Protocol Assessment agreement with the FDA.

The January 2026 corporate presentation indicated that the Phase 3 study of IFx-2.0 in Merkel Cell Carcinoma is ongoing, with topline results expected in the first half of 2027. The presentation also stated that Phase 1 and Phase 2 data for IFx-2.0 are planned to be presented at ASCO 2026.

Streetwise Ownership Overview*

TuHURA Biosciences Inc. (HURA:NASDAQ)

^*Share Structure as of 2/5/2026

In addition to IFx-2.0, TuHURA disclosed that its pipeline includes TBS-2025, a VISTA-inhibiting monoclonal antibody that entered Phase 2 development for mutNPM1 acute myeloid leukemia following the company's merger with Kineta Inc. on June 30, 2025. The presentation noted that initiation of the Phase 2 trial for TBS-2025 was scheduled for the first quarter of 2026.

The corporate deck also outlined ongoing preclinical work related to the company's Delta Opioid Receptor technology, which is being used to develop bi-specific antibody drug conjugates and antibody peptide conjugates targeting myeloid-derived suppressor cells. Proof-of-concept studies for this program were identified in the presentation as occurring in 2026.

TuHURA stated that it is a Phase 3 immuno-oncology company developing technologies intended to address resistance to cancer immunotherapy and that its development programs span multiple clinical stages across oncology indications.

Ownership and Share Structure¹

25.37% of TruHura is held by management and insiders, with Vijay Patel holding the most at 13.17%. Strategic Entities hold 1.5% and Institutions hold 10.01%. The rest is retail.

TruHura Biosciences has 60.72 million outstanding shares, a market cap of US$28.16 millon, and a 52-week trading range of US$0.50 and US$5.27.

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Important Disclosures:

James Guttman wrote this article for Streetwise Reports LLC and provides services to Streetwise Reports as an employee.
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1. Ownership and Share Structure Information

The information listed above was updated on the date this article was published and was compiled from information from the company and various other data providers.

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