Full Press Release Details
Except as otherwise specified
or indicated by the context, references to "we," "us," "our" or the "company" are to TuHURA Biosciences, Inc. and its wholly owned subsidiaries.
We are a clinical stage
immuno-oncology company with three distinct technologies focused on the development of novel therapeutics designed to overcome primary and acquired resistance to cancer immunotherapies.
Our proprietary Immune FxTM technology platform, or IFx, is an innate immune agonist technology designed to "trick" the
body's immune system to attack tumor cells by making tumor cells look like bacteria. Our lead product candidate, IFx2.0, is an innate immune agonist designed to overcome primary resistance to checkpoint inhibitors. In June 2025, we initiated a
single randomized placebo-controlled Phase 3 registration trial of IFx-2.0 administered as an adjunctive therapy to Keytruda (pembrolizumab) in first
line treatment for patients with advanced or metastatic Merkel cell carcinoma who are checkpoint inhibitor na ve utilizing the FDA's accelerated approval pathway.
In addition to our IFx technology platform, in June 2025 we acquired the rights to TBS-2025, a novel
VISTA-inhibiting monoclonal antibody formerly known as KVA1213, through our acquisition of Kineta, Inc. ("Kineta") on June 30, 2025. VISTA (otherwise referred to as V-domain Ig suppressor
of T cell activation) is an immune checkpoint highly expressed on myeloid cells that is believed to be a strong driver of immunosuppression in the tumor microenvironment and is believed to be a primary mechanism by which leukemic blasts escape
immune recognition contributing to low relapse rates and high rates of recurrence in acute myeloid leukemia, or AML. Following our acquisition of Kineta, we are currently planning on investigating TBS-2025 in
a randomized Phase 2 trial in combination with a menin inhibitor vs menin inhibitor alone in mutated NPM1 (mutNPM1) AML.
addition to our IFx and TBS-2025 technologies, we are leveraging our Delta Opioid Receptor technology to develop tumor microenvironment modulators in the form of first-in-class bi-specific antibody-peptide conjugates ("APCs") and antibody-drug conjugates ("ADCs") targeting Myeloid Derived Suppressor Cells
("MDSCs"). Our APCs and ADCs are being developed to inhibit the immune-suppressing effects of MDSCs on the tumor microenvironment to prevent T cell exhaustion and acquired resistance to checkpoint inhibitors and cellular therapies.
IFx Innate Immune Agonist Development Program
We have developed Immune FxTM, or IFx, as an innate immune agonist technology designed to "trick" the body's immune system to
attack tumor cells by making tumor cells look like bacteria and to thereby harness the natural power of innate immunity by leveraging natural mechanisms conserved throughout evolution to recognize threats from foreign pathogens like bacteria or
viruses. Our innate immune agonist product candidates are delivered either via intratumoral injection (in the case of the Company's pDNA innate immune agonist) or tumor targeted via intravenous or autologous whole-cell administration (in the
case of our mRNA innate immune agonist).
Our IFx-2.0 innate immune agonist, our company's
lead product candidate, is comparatively simple to administer and involves only the injection into a patient's tumor, or lymph node, of a relatively small amount of pDNA that is designed to encode for an immunogenic gram positive bacterial
protein that gets expressed on the surface of the patient's tumor so that the surface of the tumor looks like a bacterium.
Bacteria, like all pathogens, have molecular patterns or motifs that are conserved through
evolution and that are recognized by specific pattern-recognition receptors on immune cells of our innate immune system. This is an individual's primary line of defense against pathogens that the individual is born with, and the innate immune
system has no choice but to recognize the tumor as it would a gram-positive bacteria or any pathogen. Gram-positive bacterial proteins are recognized by Toll Like Receptor-2
(TLR-2) on antigen presenting cells, or APCs, which engulf and ingest the entire intact tumor cell packaging all the foreign tumor neoantigens presenting them to and educating tumor killing T cells and B
cells. In doing so, IFx-2.0 harnesses the power of the innate immune response to produce activated tumor-specific T cells where they previously didn't exist overcoming primary resistance to checkpoint
We have entered into a Special Protocol Assessment agreement with the FDA for a single Phase 3 randomized placebo and
injection-controlled trial for IFx-2.0, our lead innate immune agonist, as an adjunctive therapy to pembrolizumab (Keytruda ) in the first line
treatment of patients with advanced or metastatic Merkel cell carcinoma, who are checkpoint inhibitor-na ve utilizing the FDA's accelerated approval pathway. A Special Protocol Assessment agreement
is a binding written agreement between the U.S. Food and Drug Administration (FDA) and a trial sponsor that indicates the FDA has agreed to the study's design, charters, and statistical analysis plan, and if the study endpoints are met within
the context of the SPA Agreement, such results would be adequate to support accelerated and regular approval. A Special Protocol Assessment agreement does not increase the likelihood of marketing approval for the product and may not lead to a faster
or less costly development, review, or approval process. We initiated the Phase 3 trial in June 2025.
In designing the Phase 3 trial for IFx-2.0, we worked with the deputy director of the FDA's Oncology Center of Excellence (OCE) on what we believe is a unique trial design. Consistent with the FDA's Project Front Runner initiative, the
FDA recommended investigating IFx-2.0 in the front-line treatment setting rather than in patients who are progressing on checkpoint inhibitor therapy. In doing so, data from a primary endpoint of objective
response rate, or ORR, that is of sufficient magnitude and duration and with a favorable risk/benefit profile could be sufficient to support accelerated approval. Furthermore, OCE requested that the Company consider incorporating a key secondary
endpoint that is of clinical benefit such that results from a key secondary endpoint of progression-free survival, or PFS, that is adequately powered with statistical assumptions in the statistical analysis plan provided to the FDA, if achieved
without a detrimental effect on overall survival, or OS, could be adequate to support conversion to regular approval satisfying the requirement for a confirmatory trial.
We anticipate that enrollment for the Phase 3 will take approximately 14 - 18 months from the initiation of the trial, with top-line data potentially being available 6 to 7 months following the last patient enrolled. If successful, this Phase 3 trial would form the basis of a Biologics License Application, or BLA.
We previously announced that we were pursuing development of a product candidate referred to as
IFx-3.0, an mRNA innate immune agonist candidate for intravenous or autologous whole cell administration for blood- related cancers. However, with the acquisition of Kineta, we have determined not to
advance the development of IFx-3.0 until the results of the IFx-2.0 Phase 3 trial in Merkel cell carcinoma are known and have reallocated resources to the
below-described planned trial for TBS-2025.
As a result of our acquisition of Kineta in June 2025, we acquired the rights to
TBS-2025, a novel VISTA- inhibiting monoclonal antibody formerly known as KVA1213. Unlike other checkpoints, which are mostly present on activated T cells, VISTA is predominately expressed on myeloid cells,
notably MDSCs, and on quiescent T cells. Research has demonstrated that when mutated, NPM1 and DNM3TA, two of the most common mutations in AML and typically co-mutated in myelodysplasia (MDS), result in high
expression of VISTA on the surface of leukemic blasts. The presence of VISTA on these cells is believed to be the primary mechanism by which leukemic cells escape immune recognition and attack, resulting in a low treatment response rate and a high
level of relapse in AML.
TBS-2025 was previously investigated in a dose escalation Phase 1/2
trial, both as a monotherapy and in combination with pembrolizumab, in patients with relapsed and/or treatment-refractory advanced solid tumors.
TBS-2025 was well tolerated when administered every 2 weeks at doses up to 1,000mg both in the monotherapy arm (n=24) or in the pembrolizumab combination
therapy arm (n=16). Pharmacokinetic and pharmacodynamic data demonstrated greater than 90% receptor occupancy across the every two- week dosing interval. Immunocytokine analysis was consistent with the mechanism of action for VISTA inhibition on
We believe that, in a relatively inexpensive, small Phase 2 study, we can determine if
TBS-2025 can augment the response rates seen with menin inhibitors and decrease the rate of relapse in patients with mutNMP1 relapsed or refractory AML where menin inhibitors are the current standard of
care. Accordingly, we are currently planning on investigating TBS-2025 in a Phase 2 trial in combination with a menin inhibitor in mutNMP1 AML.
DOR Technology Development Program
addition to its innate immune agonist product candidates, we are using proprietary Delta Opioid Receptor (DOR) technology to develop peptidomimetic or small molecule
bi-specific/bi-functional immune modulating APCs and ADCs designed to inhibit the immune suppressing effects of tumor associated MDSCs on the tumor microenvironment to
prevent T cell exhaustion and acquired resistance to checkpoint inhibitors. The Company's DOR technology was developed by scientists at Moffitt Cancer Center and TuHURA Biopharma, Inc., a separate company whose intellectual property assets we
acquired in January 2023 ("TuHURA Biopharma") We believe the DOR represents a novel target to inhibit the immunosuppressive capacity of MDSCs through its control of the production of multiple immunosuppressive soluble factors, chemokines
and direct cell-cell interactions.
The tumor microenvironment is the tissue surrounding a tumor, including the normal cells, blood
vessels, and molecules that surround and feed a tumor cell and shield it from immune attack and eradication. MDSCs are a heterogeneous group of immature myeloid cells, which when recruited from the bone marrow to the tumor microenvironment, they
transform to tumor-associated MDSCs which are characterized by their ability to suppress both innate and adaptive immune responses. Tumor associated MDSCs are generally believed to be a major contributor to T cell exhaustion (which is the loss of
ability of T cells to proliferate and to kill cancer cells) and for acquired resistance to checkpoint inhibitors and cellular therapies like T cell therapies. The presence of tumor associated MDSCs in the tumor microenvironment or circulating in the
bloodstream is highly correlated with poor prognosis and outcome in a wide variety of solid tumors and blood related cancers.
we are the first company developing immune modulating APC/ADCs targeting the Delta Opioid Receptor on MDSCs. We are developing peptidomimetic or small molecule DOR-selective inhibitors to incorporate into our bi-specific, bi-functional APCs and ADCs, which we believe represents a paradigm shift from conventional APCs or ADCs that are currently in development or being marketed.
Traditional ADCs are a class of drugs in which a monoclonal antibody is chemically linked to a cancer-fighting substance. The antibody carries the cancer fighting payload to the tumor cell improving the selectivity of the resulting anti-cancer
activity. Next generation ADCs incorporate non-chemotherapeutic technologies to interfere with tumor cell cycle growth or to carry with the antibody a checkpoint inhibitor (so called "checkpoint
ADCs"). In contrast, our APCs or ADCs do not target tumor associated receptor targets but rather target the Delta Opioid Receptor on MDSCs while carrying with them an immune effector to target a second receptor target like VISTA with a VISTA
inhibiting antibody or other checkpoint inhibitor(s) producing novel bi-specific, bi-functional conjugates. These two functions are intended to work together with the
goal of overcoming acquired resistance, preventing T cell exhaustion and allowing checkpoint inhibitors and cellular therapies to be safer and more effective while interfering with the tumor's ability to invade and spread throughout the body.
Our pipeline focuses on acquiring and developing technologies designed to overcome tumor-intrinsic mechanisms underlying primary resistance to
checkpoint inhibitors. We also focus on technologies to overcome acquired resistance to cancer immunotherapies related to the immune suppressing characteristics of the tumor microenvironment. We are leveraging our technology platforms to advance
several diversified product candidates, including principally the following, each of which is described in more detail below.
Our History and Team
We were originally incorporated in Nevada on June 24, 2009 under the name Berry Only Inc. On January 25, 2013, TuHURA entered into
and closed an exchange agreement, with Del Mar Pharmaceuticals (BC) Ltd. ("Del Mar (BC)"), 0959454 B.C. Ltd. ("Callco"), and 0959456 B.C. Ltd. ("Exchangeco") and the security holders of Del Mar (BC). Upon
completion of the exchange agreement, Del Mar (BC) became a wholly-owned subsidiary of TuHURA. On August 19, 2020, TuHURA completed its merger with Adgero Biopharmaceuticals Holdings, Inc., a Delaware corporation ("Adgero"), in
which Adgero continued its existence under Delaware law and became a direct, wholly-owned subsidiary of TuHURA. Following the completion of the merger, TuHURA changed its name from Del Mar Pharmaceuticals, Inc. to Kintara Therapeutics, Inc. and
began trading on Nasdaq under the symbol "KTRA."
On October 18, 2024, TuHURA completed a reverse merger transaction
contemplated by its Agreement and Plan of Merger, dated April 2, 2024 (the "TuHURA-Kintara Merger Agreement"), with TuHURA Biosciences, Inc., a Delaware corporation ("Legacy TuHURA"), and Kayak Mergeco, Inc., a Delaware
corporation and wholly-owned subsidiary of TuHURA ("Kintara Merger Sub"). Pursuant to the TuHURA-Kintara Merger Agreement, Kintara Merger Sub merged with and into Legacy TuHURA with Legacy TuHURA surviving the merger (the "Kintara
Merger") and becoming TuHURA's direct, wholly-owned subsidiary. TuHURA completed the merger on October 18, 2024, and changed its name on such date from Kintara Therapeutics, Inc. to "TuHURA Biosciences, Inc." and begin
trading on Nasdaq under the symbol "HURA."
Legacy TuHURA's predecessor company was formed as Morphogenesis, Inc. in
1995 by Drs. Patricia and Michael Lawman. Our IFx technology was developed in the laboratory of Dr. Michael Lawman at the Walt Disney Memorial Cancer Institute, where Dr. Michael Lawman was formerly a Director of the Institute, and
Dr. Patricia Lawman was formerly Division Director of Cancer Molecular Biology at the Institute. Dr. Michael Lawman is a Fellow of the Royal Society of Biology, former Associate Professor at University of South Florida, and former
Scientific Research Director of Pediatric Hematology/Oncology at St. Joseph's Children's Hospital. Dr. Patricia Lawman also serves as an Adjunct Professor at University of South Florida. Drs. Patricia and Michael Lawman are each
inventors on numerous U.S. and foreign patents.
Our Delta Opioid Receptor APC and ADC technology was developed in the laboratory of
Dr. Mark McLaughlin at the Moffitt Cancer Center and at the West Virginia University Research Corporation. Dr. McLaughlin was previously a Senior Member of the Drug Discovery Department at the Moffitt Cancer Center and previously Professor
of Medicinal Chemistry and Member WVU Cancer Institute, where his research focused on protein-protein interaction inhibitor design and molecular targeted immunotherapy. The discovery that the Delta receptor is highly expressed on MDSCs was jointly
discovered by scientists at Moffitt Cancer Center and TuHURA Biopharma, a separate company whose intellectual property assets we acquired in January 2023.
Our CEO, Dr. James Bianco, is a 33-year veteran of the biopharmaceutical industry.
Dr. Bianco is the principal founder of CTI Biopharma, where he served as its CEO from 1992 to October 2016. Dr. Bianco's experience spans all aspects of drug development from phase I-IV
clinical trials, regulatory approval, and pricing reimbursement to sales and marketing. He has extensive experience in financing, negotiating and execution of pharmaceutical development and commercial license agreements. During his tenure at CTI
Biopharma, Dr. Bianco was responsible for strategic portfolio development and identifying, acquiring, licensing, purchasing, or acquiring through international merger and acquisition, five drug candidates, four of which have since been approved