FDA Removes Partial Clinical Hold on TuHURA Biosciences' Phase 3 Accelerated Approval Trial for IFx-2.0 in Advanced or Metastatic Merkel Cell Carcinoma

Rhea-AI Summary

TuHURA Biosciences (NASDAQ:HURA) announced the FDA's removal of a manufacturing-related partial clinical hold on their Phase 3 accelerated approval trial for IFx-2.0. The trial, conducted under Special Protocol Assessment Agreement, will evaluate IFx-2.0 as adjunctive therapy with Keytruda for first-line treatment of advanced/metastatic Merkel cell carcinoma. The removal triggers a $2.23M funding tranche from a recent $12.5M PIPE financing. The trial will enroll 118 patients across 22-25 U.S. sites, with participants randomized 1:1 to receive either Keytruda+IFx-2.0 or Keytruda+placebo. Primary endpoint is Overall Response Rate, with Progression Free Survival as key secondary endpoint. Trial initiation is expected in June 2025.

Positive

• FDA removal of partial clinical hold enables immediate trial initiation
• Unlocks $2.23M additional funding from $12.5M PIPE financing
• Trial design allows for potential regular approval without confirmatory trial if PFS endpoint is met
• Special Protocol Assessment (SPA) Agreement with FDA provides clear regulatory pathway

Negative

• Manufacturing-related issues previously led to clinical hold
• Relatively small trial size of 118 patients may limit statistical power
• Competition from already-approved Keytruda in the same indication

Insights

Biotechnology Regulatory Specialist

FDA's removal of clinical hold unlocks TuHURA's Phase 3 trial and $2.23M funding, significantly advancing their cancer immunotherapy program.

The FDA's decision to lift the manufacturing-related partial clinical hold on TuHURA's Phase 3 trial represents a critical regulatory milestone that removes a significant barrier to the company's development timeline. This regulatory clearance is particularly valuable because it comes with a Special Protocol Assessment (SPA) Agreement, which essentially provides a binding commitment from the FDA regarding the acceptability of the trial design for approval purposes.

What makes this development especially notable is the accelerated approval pathway TuHURA is pursuing. Their trial design is strategically constructed to potentially achieve both accelerated and regular approval in a single trial - an efficient regulatory approach that could significantly compress the timeline to market. The primary endpoint of Overall Response Rate (ORR) could support accelerated approval, while the key secondary endpoint of Progression-Free Survival (PFS) could enable conversion to full approval without requiring a separate confirmatory trial.

The relatively modest trial size of 118 patients across 22-25 sites suggests a manageable clinical development budget and timeline. For Merkel cell carcinoma (MCC), an aggressive but rare skin cancer with limited treatment options, this sample size is appropriate and reflects the targeted nature of this indication. The design as an adjunctive therapy to Keytruda® (the current standard of care) represents a pragmatic approach to improving outcomes in a difficult-to-treat cancer.

The resolution of the manufacturing issues that prompted the clinical hold demonstrates the company's ability to address complex regulatory challenges, suggesting operational competence in navigating the approval process - a critical factor for clinical-stage biotechs.

Biotech Investment Analyst

Clinical hold removal enables Phase 3 trial initiation and unlocks critical financing, significantly derisking TuHURA's development path.

The removal of the partial clinical hold addresses two critical factors for TuHURA: operational progress and financial stability. Most importantly, it triggers the release of $2.23 million from their recently announced $12.5 million PIPE financing. For small biotechs, cash runway is often as important as clinical data, and this milestone-based funding structure provides essential capital while minimizing dilution.

This announcement effectively derisks a significant near-term uncertainty that likely concerned investors. Manufacturing-related clinical holds can sometimes indicate fundamental issues with production processes, but the relatively quick resolution suggests the problems were addressable rather than fundamental flaws. The prompt FDA response time mentioned also suggests constructive regulatory engagement.

The Phase 3 trial design warrants attention for its capital efficiency. By pursuing accelerated approval with a simultaneous path to full approval through the PFS secondary endpoint, the company has structured a program that could potentially avoid the significant expense of a mandatory post-approval confirmatory trial. This approach demonstrates strategic regulatory planning that could conserve capital and accelerate time to market.

The first-line treatment positioning in combination with Keytruda® represents a commercially advantageous approach. Rather than competing with an established therapy, IFx-2.0 is positioned as an enhancer of the standard of care, which typically faces fewer market adoption barriers. While Merkel cell carcinoma is a relatively small indication, success here could provide validation for the company's technology platform and potentially enable expansion into more prevalent cancer types.

With trial initiation expected later this month, investors can anticipate a series of enrollment updates and, eventually, data readouts as key value-driving catalysts.

Company anticipates initiating its Phase 3 accelerated approval trial of IFx-Hu2.0 as adjunctive therapy with Keytruda^® (pembrolizumab) as first-line treatment for advanced and metastatic Merkel cell carcinoma (MCC), conducted under Special Protocol Assessment (SPA) Agreement with U.S. Food and Drug Administration (FDA), later in June 2025

Resolution of partial clinical hold unlocks second tranche of funds from $12.5 million PIPE financing announced on June 3, 2025

TAMPA, Fla., June 9, 2025 /PRNewswire/ -- TuHURA Biosciences, Inc. (NASDAQ:HURA) ("TuHURA" or the "Company"), a Phase 3 immune-oncology company developing novel technologies to overcome resistance to cancer immunotherapy, today announced that the FDA has removed the manufacturing-related partial clinical hold on the Company's Phase 3 accelerated approval trial for IFx-2.0, thereby allowing the trial to proceed as agreed to under the previously announced SPA Agreement with the FDA.

"We are grateful for the collaborative interaction with the reviewers at the Office of Therapeutic Products (OTP) and the Oncology Center of Excellence (OCE), including their quick response time and, importantly, their helpful recommendations going forward," stated James Bianco, M.D., President and Chief Executive Officer of TuHURA Biosciences.

"The removal of the partial clinical hold allows TuHURA to begin the trial's initiation and activation of clinical sites for the Phase 3 accelerated approval trial of IFx-2.0," continued Dr. Bianco. "We are also pleased that the removal of the partial clinical hold represents the achievement of the second milestone funding condition under our recently announced private placement, thereunder trigging the payment to the Company of an additional $2.23 million under the financing."

The Company's Phase 3 accelerated approval trial of IFx-2.0, will be conducted under an SPA Agreement with the U.S. FDA, and will evaluate IFx-2.0 as an adjunctive therapy administered weekly for three weeks concurrent with the approved dose and schedule for Keytruda^® compared to Keytruda^® plus placebo in the first line treatment of patients with advanced or metastatic MCC. Keytruda^® is currently approved in MCC under accelerated approval based on Overall Response Rate (ORR). The pivotal trial for IFx-2.0 is expected to enroll 118 across approximately 22 to 25 U.S. sites. Trial participants will be randomized on a 1:1 basis and receive Keytruda^® in both arms, for up to two years, or until disease progression or Keytruda^® related toxicities. The primary endpoint for the trial is ORR with a key secondary endpoint of Progression Free Survival (PFS). Other secondary endpoints are safety, duration of response, and overall survival. Accelerated approval is based on the successful achievement of the ORR primary endpoint. PFS, the key secondary endpoint, if successfully achieved, without a detrimental effect on overall survival, could satisfy the requirement for regular approval without the requirement for a post approval confirmatory trial (in contrast to most accelerated approval trials).

About TuHURA Biosciences, Inc.

TuHURA Biosciences, Inc. (Nasdaq: HURA) is a Phase 3 immuno-oncology company developing novel technologies to overcome primary and acquired resistance to cancer immunotherapy, two of the most common reasons cancer immunotherapies fail to work or stop working in the majority of patients with cancer.

TuHURA's lead innate immune agonist, IFx-2.0, is designed to overcome primary resistance to checkpoint inhibitors. TuHURA is preparing to initiate a single randomized placebo-controlled Phase 3 registration trial of IFx-2.0 administered as an adjunctive therapy to Keytruda^® (pembrolizumab) compared to Keytruda^® plus placebo in first-line treatment for advanced or metastatic Merkel Cell Carcinoma.

Following its proposed merger with Kineta, Inc., if completed, the Company expects to advance Kineta's novel VISTA inhibiting antibody into a randomized Phase 2 trial in NPM1 mutated r/r AML in combination with a menin inhibitor.

In addition to its innate immune agonist product candidate, TuHURA is leveraging its Delta Opioid Receptor technology to develop first-in-class, bi-specific antibody drug conjugates and antibody peptide conjugates targeting Myeloid Derived Suppressor Cells to inhibit their immune-suppressing effects on the tumor microenvironment to prevent T cell exhaustion and acquired resistance to checkpoint inhibitors and cellular therapies.

For more information, please visit www.tuhurabio.com and connect with TuHURA on Facebook, X, and LinkedIn.

CAUTIONARY STATEMENT REGARDING FORWARD-LOOKING STATEMENTS

This press release contains certain "forward-looking statements" within the meaning of, and subject to the safe harbor created by, Section 27A of the Securities Act, Section 21E of the Exchange Act of 1934, as amended, and the Private Securities Litigation Reform Act of 1995. These forward-looking statements are based only on our current beliefs, expectations and assumptions regarding the future of our business, future plans and strategies, projections, anticipated events and other future conditions. In some cases you can identify these statements by forward-looking words such as "believe," "may," "will," "estimate," "continue," "anticipate," "intend," "could," "should," "would," "project," "plan," "expect," "goal," "seek," "future," "likely" or the negative or plural of these words or similar expressions. Examples of such forward-looking statements include but are not limited to express or implied statements regarding TuHURA's expectations, hopes, beliefs, intentions or strategies regarding the future and include, without limitation, statements regarding, TuHURA's IFx-Hu2.0 product candidate and anticipated Phase 3 trial, its tumor microenvironment modulators development program, its potential acquisition by merger of Kineta Inc. and the statements about Kineta's VISTA-101 development program, and any developments or results in connection with the foregoing and the anticipated regulatory pathway and timing of the foregoing development programs, studies and trials. In addition, any statements that refer to projections, forecasts or other characterizations of future events or circumstances, including any underlying assumptions, are forward-looking statements. You are cautioned that such statements are not guarantees of future performance and that actual results or developments may differ materially from those set forth in these forward-looking statements. Factors that could cause actual results to differ materially from these forward-looking statements are described in detail in our registration statements, reports and other filings with the SEC, which are available on the combined company's website, and at www.sec.gov.

The forward-looking statements and other information contained in this press release are made as of the date hereof, and TuHURA does not undertake any obligation to update publicly or revise any forward-looking statements or information, whether as a result of new information, future events or otherwise, unless so required by applicable securities laws. Nothing herein shall constitute an offer to sell or the solicitation of an offer to buy any securities.

Investor Contact:

Monique Kosse
Gilmartin Group
Monique@GilmartinIR.com

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SOURCE TuHURA Biosciences, Inc.

FAQ

What caused the FDA to remove HURA's clinical hold for IFx-2.0?

The FDA removed a manufacturing-related partial clinical hold after collaborative interaction with reviewers at the Office of Therapeutic Products and Oncology Center of Excellence

How many patients will be enrolled in HURA's Phase 3 trial for IFx-2.0?

The Phase 3 trial will enroll 118 patients across 22-25 U.S. clinical sites

What is the primary endpoint for HURA's Phase 3 trial of IFx-2.0?

The primary endpoint is Overall Response Rate (ORR), with Progression Free Survival (PFS) as a key secondary endpoint

How much additional funding did HURA receive after the clinical hold removal?

The removal triggered a $2.23 million second tranche payment from their recent $12.5 million PIPE financing

When will HURA begin its Phase 3 trial for IFx-2.0?

TuHURA Biosciences anticipates initiating the Phase 3 trial later in June 2025