Aptose's AML Hope Faces Funding Reality: Promising Trial Data Amidst Financial Strain

NEW YORK, NY – November 13, 2025

Aptose Biosciences is navigating a precarious path. Recent data from its Phase 1/2 TUSCANY trial offers a glimmer of hope for patients battling acute myeloid leukemia (AML), with promising complete response rates observed in combination with standard care. However, this progress is overshadowed by deepening financial pressures and a heavy reliance on funding from Hanmi Pharmaceutical Co. Ltd., raising serious questions about the company’s long-term viability.

Promising Early Data for Tuspetinib

The TUSCANY trial, investigating the oral kinase inhibitor tuspetinib (TUS) in AML patients, has yielded encouraging initial results. Data presented at medical conferences, including the European School of Haematology (ESH) and the upcoming American Society of Hematology (ASH) meeting, demonstrate a 100% complete response rate (CR/CRh) in patients treated with a combination of tuspetinib, venetoclax, and azacitidine at the 80mg and 120mg dose levels. The overall CR/CRh response rate across multiple cohorts reached 90%.

“These results suggest that adding tuspetinib to the standard venetoclax and azacitidine regimen could significantly improve outcomes for AML patients, particularly those ineligible for intensive chemotherapy,” said one hematologist familiar with the trial data, speaking anonymously. “The ‘mutation agnostic’ activity – meaning it appears effective across a broad range of genetic mutations – is particularly noteworthy.” The current standard of care, venetoclax and azacitidine alone, achieves a CR/CRh rate of approximately 66%, suggesting a potentially substantial improvement with the addition of tuspetinib.

The therapy has demonstrated a favorable safety profile to date, with no dose-limiting toxicities or concerning adverse events reported. This is crucial, as many AML treatments are associated with significant side effects that can limit their use. A key differentiator for tuspetinib is its potential to address venetoclax resistance mechanisms, offering hope for patients who have failed other therapies.

Financial Strain and Funding Dependence

Despite the encouraging clinical data, Aptose is facing a significant financial hurdle. The company reported a net loss of $5.1 million for the third quarter of 2025, and cash and equivalents stood at just $1.6 million as of September 30th. Aptose has negative working capital and a substantial shareholders’ deficit, signaling a precarious financial position.

“The company is actively pursuing all available options to secure additional financing,” a source close to the company stated, requesting anonymity. “But the current market environment for biotech funding is challenging, and securing sufficient capital to fund ongoing operations and clinical development is a major concern.”

Aptose’s financial lifeline is currently a loan agreement with Hanmi Pharmaceutical, providing access to up to $8.5 million in funding. As of the second quarter of 2025, the company had received $5.6 million under this agreement. However, reliance on a single funding source exposes Aptose to significant risk. Any disruption in funding from Hanmi could severely jeopardize the company's ability to continue operations and bring tuspetinib to market.

Navigating a Competitive AML Landscape

The AML treatment landscape is evolving rapidly, with a growing number of targeted therapies and immunotherapies becoming available. While venetoclax and azacitidine remain a cornerstone of treatment for many patients, particularly those ineligible for intensive chemotherapy, several other options are available, including FLT3 inhibitors for patients with specific genetic mutations and IDH1/IDH2 inhibitors for those with corresponding mutations.

Tuspetinib differentiates itself through its broad activity across multiple genetic subtypes of AML. While many targeted therapies focus on specific mutations, tuspetinib appears effective regardless of the underlying genetic profile, offering a potential advantage for patients who don’t fit into a specific treatment category. This broad activity is also valuable in the relapsed/refractory setting, where patients often develop resistance to multiple therapies.

“The ability to address a wider range of AML subtypes is a significant benefit,” stated an independent oncology consultant. “It expands the potential patient population and increases the likelihood of finding a treatment option that works.” However, bringing a new drug to market requires substantial investment in clinical trials, manufacturing, and commercialization. For Aptose, securing the necessary funding remains a critical challenge.

While the positive data from the TUSCANY trial offer a glimmer of hope for AML patients, the future of tuspetinib – and Aptose Biosciences – hangs in the balance. The company must navigate a complex financial landscape and secure the necessary funding to continue its clinical development and bring this potentially life-changing therapy to those who need it most.