On March 28. 2025 Bio-Path Holdings, Inc., (OTCQB:BPTH), a biotechnology company leveraging its proprietary DNAbilize antisense RNAi nanoparticle technology to develop a portfolio of targeted nucleic acid cancer and obesity drugs, reported its financial results for the year ended December 31, 2024 and provided an update on recent corporate developments (Press release, Bio-Path Holdings, MAR 28, 2025, View Source [SID1234651571]).

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"We are merely touching the tip of the iceberg in terms of realizing the potential of our DNAbilize platform to change the treatment paradigm in both obesity and oncology," said Peter Nielsen, President and Chief Executive Officer of Bio-Path Holdings. "Throughout the last year, we built on the body of scientific evidence in support of our powerful platform technologies’ therapeutic effects and fortified our intellectual property to protect it from potential competitors. We continue to advance our clinical studies for BP1001-A as a treatment for obesity in Type 2 diabetes patients, where we have shown restored insulin sensitivity in cell models. Beyond this, our ongoing oncology studies continue to advance, and we are reporting ever improving outcomes for the most vulnerable patients battling these life-threatening cancers."

Recent Corporate Highlights

Announced Pre-Clinical Results Signaling Increased Potential for BP1001-A as Treatment for Obesity in Type 2 Diabetes Patients. Scientific evidence suggests that by downregulating growth factor receptor-bound protein 2 (Grb2) expression, BP1001-A could help lower blood glucose level by affecting insulin signaling. In December 2024, Bio-Path reported results from preclinical studies of BP1001-A for obesity demonstrating enhanced insulin sensitivity in myoblast and hepatoma cells. Furthermore, in March 2025, Bio-Path reported preclinical results that BP1001-A attenuated fatty acid-induced insulin resistance and restored insulin sensitivity in muscle progenitor and skeletal muscle fiber cell models. Together these studies signal increased potential for BP1001-A as a treatment for obesity and related metabolic diseases in Type 2 diabetes patients.
Expanded Global Patent Portfolio. In February 2025, Bio-Path announced the receipt of newly issued patents in the United States and New Zealand, and updated investors on the extent of its global intellectual property portfolio. Bio-Path received Notice of Allowance from the United States Patent and Trademark Office for U.S. Patent No. 17/339,366 titled, "P-ethoxy nucleic acids for STAT3 inhibition." The New Zealand Intellectual Property Office has granted Patent No. 741793 titled, "P-ethoxy nucleic acids for liposomal formulation." These new patents build on earlier patents granted that protect the DNAbilize platform technology and the Company’s novel RNAi nanoparticle drugs.
Bio-Path continues to expand its intellectual property portfolio by filing patent applications that are applicable to its technology and business strategy. Bio-Path’s patent portfolio currently includes seven issued patents in the U.S. and 61 issued patents in foreign jurisdictions, providing protection in 26 countries. The Company has three additional pending patent applications in the U.S. and five additional allowed patent applications in foreign jurisdictions.

Provided Update from Phase 1/1b Clinical Trial of BP1002 for Treatment of Refractory/Relapsed Acute Myeloid Leukemia. In February 2025, the Company provided an update from the ongoing Phase 1/1b clinical trial evaluating BP1002 for the treatment of refractory/relapsed acute myeloid leukemia (AML), including venetoclax-resistant patients. The Company announced a meaningful patient response to treatment and that the study has progressed to the fourth, higher dose cohort of 90 mg/m2.
Financial Results for the Year Ended December 31, 2024

The Company reported a net loss of $9.9 million, or $4.12 per share, for the year ended December 31, 2024, compared to a net loss of $16.1 million, or $33.63 per share, for the year ended December 31, 2023.
Research and development expense for the year ended December 31, 2024 decreased to $7.3 million, compared to $11.6 million for the year ended December 31, 2023 primarily due to decreased manufacturing expenses related to drug product releases in 2024 compared to 2023.
General and administrative expense for the year ended December 31, 2024 increased to $4.7 million, compared to $4.2 million for the year ended December 31, 2023 primarily due to increased salaries and benefits expense as well as expenses related to our special shareholder meeting in 2024.
Change in fair value of the Company’s warrant liability for the year ended December 31, 2024 resulted in a non-cash income of $2.1 million compared to a non-cash loss of $0.3 million for the year ended December 31, 2023.
As of December 31, 2024, the Company had cash of $1.2 million, compared to $1.1 million as of December 31, 2023. Net cash used in operating activities for the year ended December 31, 2024 was $10.6 million compared to $11.5 million for the comparable period in 2023. Net cash provided by financing activities for the year ended December 31, 2024 was $10.7 million.

On March 28, 2025 Aptose Biosciences Inc. ("Aptose" or the "Company") (NASDAQ: APTO, TSX: APS), a clinical-stage precision oncology company developing a tuspetinib (TUS)-based triple drug frontline therapy to treat patients with newly diagnosed acute myeloid leukemia (AML), reported financial results for the year ended December 31, 2024, and provided a corporate update (Press release, Aptose Biosciences, MAR 28, 2025, View Source [SID1234651570]).

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"During 2024 and into 2025, we continue to advance our lead investigational drug tuspetinib in combination with venetoclax (VEN) and azacitidine (AZA) for frontline treatment of newly diagnosed acute myeloid leukemia (AML)," said William G. Rice, Ph.D., Chairman, President and Chief Executive Officer of Aptose. "Tuspetinib brings favorable safety and broad activity across AML genetic subtypes to the TUS+VEN+AZA triplet therapy, which already has achieved complete remissions (CRs) in difficult-to-treat and underserved TP53-mutated/CK AML and FLT3-wildtype AML patients in our ongoing TUSCANY trial. We look forward to sharing more data as the trial evolves."

Key Corporate Highlights

Tuspetinib Phase 1/2 TUSCANY Trial Well Under Way with Responses Noted – Tuspetinib based TUS+VEN+AZA triplet therapy is being advanced in the TUSCANY Phase 1/2 trial with the goal of creating an improved frontline therapy for newly diagnosed AML patients that is active across diverse AML populations (mutation agnostic triplet frontline therapy), including FLT3-wildtype AML. This activity differentiates TUS from other drugs in development. In January 2025, Aptose announced initiation of dosing in the first TUSCANY trial cohort with the starting dose of 40 mg TUS in combination with standard-of-care doses of VEN+AZA. At 40 mg TUS, the triplet therapy achieved complete remissions (CRs) in difficult-to-treat TP53-muated AML and FLT3-wildtype AML patients, including a measurable residual disease (MRD) negative remission (press release here). In February 2025, amid the promising early results and favorable safety from patients treated at the 40 mg dose level, the Cohort Safety Review Committee (CSRC) monitoring the TUSCANY trial approved escalating to 80 mg TUS in the triplet therapy (press release here). Subjects have now begun treatment at the 80 mg TUS dose level of the triplet therapy and further recruitment is under way. No significant safety concerns have been reported to date, including no prolonged myelosuppression of subjects in remission. This TUS+VEN+AZA triplet therapy study in newly diagnosed AML was supported with robust safety and efficacy data from the TUS single agent dose escalation study and the TUS+VEN doublet APTIVATE study in relapsed or refractory (R/R) AML, both of which were completed during 2024 after treating more than 170 patients.
Financing Activity – During 2024, Aptose completed several financings for a total of approximately $37 million to support the TUS-based TUS+VEN+HMA triplet therapy development for AML. This included a $10 million loan Facility Agreement with Hanmi Pharmaceutical Co. Ltd. ("Hanmi"). Subsequently in March 2025, Hanmi and Aptose executed a Debt Conversion Agreement to convert a portion of the debt into equity, subject to Hanmi owning no more than 19.99% of the issued and outstanding common shares of Aptose. Therefore, an amount of $1.5 million has been converted into 409,063 common shares as of this date. Beyond the $10 million, Aptose and Hanmi are negotiating a new tuspetinib co-development collaboration agreement intended to provide additional funding to accelerate clinical development of tuspetinib. Aptose licensed tuspetinib from Hanmi Pharmaceutical in November 2021.
Aptose Signs CRADA with NCI – In December, Aptose announced that it entered into a Cooperative Research and Development Agreement ("CRADA") with the National Cancer Institute (NCI), part of the National Institutes of Health (press release here). Under the CRADA, the NCI and Aptose will collaborate on the clinical development of TUS, an inhibitor of key signaling kinases involved in myeloid malignancies, in the NCI Cancer Therapy Evaluation Program (CTEP) sponsored myeloMATCH trials employing combinations of targeted therapy for the treatment of molecularly defined AML and myelodysplastic syndromes (MDS) populations. These trials will be conducted by NCI’s National Clinical Trials Network (NCTN), with the participation of the NCI Community Oncology Research Program (NCORP) in the U.S. and Canada.
Aptose Meets Nasdaq Minimum Bid Compliance – Earlier this month, Aptose announced that it received a written notification from the Listing Qualifications Department of The Nasdaq Stock Market, LLC notifying the Company that it is in compliance with Nasdaq’s minimum bid price requirement (press release here). On March 14, 2025, Nasdaq confirmed that, for ten consecutive business days, the closing bid price of the Company’s common shares has been $1.00 per share or greater. Accordingly, the Company has regained compliance with Listing Rule 5550(a)(2). Separately, Aptose is not in compliance with the $2.5 million shareholders equity requirement and is operating under an exception granted by the Nasdaq Hearing Panel, which provides Aptose additional time to regain compliance, although there is no assurance that the Company will successfully achieve full compliance with the Nasdaq shareholders equity requirement.
Completed and Planned Value-Creating Milestones

2024 Accomplishments

Completed $10 million loan from Hanmi as Advance on Collaboration
Completed $8 million S-1 financing
Executed CRADA with NCI MyeloMATCH for tuspetinib in AML/MDS
Initiated dosing of TUS+VEN+AZA triplet therapy in newly diagnosed AML patients in TUSCANY trial
ASH: Reported CR/Safety from APTIVATE TUS and TUS+VEN trial
ASH: Reported dosing accrual from TUS+VEN+AZA triplet therapy trial
2025: 1H

Demonstrated safety and efficacy with 40mg TUS+VEN+AZA in triplet therapy trial
Dosing 80mg TUS in TUS+VEN+AZA dose cohort in triplet therapy trial
Executed Debt Conversion agreement with Hanmi
Expect to report CR/MRD/Safety data from TUS+VEN+AZA triplet therapy trial
Expect to execute Hanmi/Aptose Collaboration
EHA2025 Congress – Report maturing data readout from TUS+VEN+AZA triplet therapy trial
2025: 2H

Select optimal TUS doses for TUS+VEN+HMA triplet therapy Ph 2/3 pivotal trials
Prepare for Ph 2 portion of Ph 2 / Ph 3 pivotal program
American Society of Hematology (ASH) (Free ASH Whitepaper) Update
FINANCIAL RESULTS OF OPERATIONS
Aptose Biosciences Inc.
Statements of Operations Data
(unaudited)
($ in thousands, except for share and per share data)

Year ended
December 31,
2024 2023
Expenses:

Research and development $ 15,103 $ 36,765
General and administrative 11,154 15,591
Operating expenses 26,257 52,356
Other income, net 827 1,149
Net loss $ (25,430 ) $ (51,207 )

Net loss per share, basic and diluted $ (36.38 ) $ (227.43 )
Weighted average number of common shares outstanding used in computing net loss per share, basic and diluted 698,980 225,154

Net loss for the year ended December 31, 2024 decreased by $25.8 million to $25.4 million, as compared to $51.2 million for the comparable period in 2023.

Aptose Biosciences Inc.
Balance Sheet Data
(unaudited)
($ in thousands)

December 31, December 31,
2024
2023
Cash, cash equivalents and restricted cash equivalents $ 6,707 $ 9,252

Working capital 5,071 (3,375 )
Total assets 10,127 12,989
Long-term liabilities 10,211 621
Accumulated deficit (540,967 ) (515,537 )

Shareholders’ deficit (4,543 ) (2,901 )

Total cash, cash equivalents and restricted cash equivalents as of December 31, 2024, were $6.7 million. Based on current operations, the Company expects that cash on hand and available capital provides the Company with sufficient resources to fund planned Company operations including research and development until April 2025.
As of March 21, 2025, we had 2,552,429 Common Shares issued and outstanding. In addition, there were 39,219 Common Shares issuable upon the exercise of outstanding stock options and there were 1,267,585 Common Shares issuable upon the exercise of the outstanding warrants.
RESEARCH AND DEVELOPMENT EXPENSES

The research and development expenses for the years ended December 31, 2024 and 2023 were as follows:

Year ended
December 31,
(in thousands) 2024
2023
Program costs – Tuspetinib $ 9,606 $ 24,925
Program costs – Luxeptinib 422 3,510
Program costs – APTO-253 (19 ) 40
Personnel related expenses 4,735 6,878
Stock-based compensation 346 1,373
Depreciation of equipment 13 39
Total $ 15,103 $ 36,765

Research and development expenses decreased by $21.7 million to $15.1 million for year ended December 31, 2024, as compared to $36.8 million for the comparable period in 2023. Changes to the components of our research and development expenses presented in the table above are primarily as a result of the following events:

Program costs for tuspetinib were $9.6 million for the year ended December 31, 2024, compared with $24.9 million for the comparable period in 2023. The lower program costs for tuspetinib in the current year were due to reduced activity in our APTIVATE clinical trial, reduced manufacturing costs, and related expenses. In the comparable period in 2023, Tuspetinib program costs included the healthy volunteer study, which was completed in the same year.
Program costs for luxeptinib decreased by approximately $3.1 million primarily due to lower clinical trial and manufacturing activities.
Program costs for APTO-253 decreased by approximately $59 thousand. This reduction was due to the Company’s decision to discontinue further development of APTO-253.
Personnel-related expenses decreased by $2.1M due to lower headcount 2024.
Stock-based compensation decreased by approximately $1.0 million in the year ended December 31, 2024, primarily due to stock options granted with lower grant date fair values when compared to the options granted in the prior period, coupled with option forfeitures recorded in the current year.

On March 28, 2025 Merck, a leading science and technology company, reported it has exercised its option with Abbisko Therapeutics Co. Ltd, Shanghai, China for commercialization of pimicotinib in the U.S. and rest of world and will pay Abbisko an option fee of $85 million (Press release, Merck KGaA, MAR 28, 2025, View Source [SID1234651542]). Under the agreement signed in 2023 for the commercialization rights in Mainland China, Hong Kong, Macau, and Taiwan, Merck now holds worldwide commercialization rights for pimicotinib. The companies recently reported positive topline results from the global Phase III MANEUVER study, demonstrating pimicotinib significantly improved the primary endpoint of objective response rate versus placebo (54.0% vs. 3.2% at week 25, p<0.0001) in the treatment of patients with tenosynovial giant cell tumor (TGCT). The companies also will explore pimicotinib in additional indications, such as chronic graft-versus-host disease (cGvHD).

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"Today marks a significant milestone in our partnership with Abbisko as we work together to deliver a potentially best-in-class therapy for patients with TGCT around the world," said Andrew Paterson, Chief Marketing Officer for the Healthcare business sector of Merck. "This collaboration underscores our commitment to advancing new treatment options in rare oncology for patients who need them. With this important step forward, we aim to transform the treatment landscape and offer hope to those living with TGCT, who today have very limited treatment options."

TGCT is a non-malignant and often recurring tumor of the joints that can cause high morbidity associated with swelling, pain, stiffness, and limited mobility of the affected joints, significantly impacting daily activities and quality of life in the primarily working-age population that it affects.

If left untreated or in recurrent cases, TGCT can result in irreversible damage to the bone, joint and surrounding tissues. This highlights the need for well-tolerated and effective systemic treatments that can impact tumor growth while relieving the symptoms of the disease.

About MANEUVER

The pivotal Phase III MANEUVER study is a three-part, randomized, double-blind, placebo-controlled study to assess the efficacy and safety of pimicotinib in patients with TGCT, who are eligible for systemic therapy and have not received prior anti-CSF-1/CSF-1R therapy. The study is being conducted in China (n=45), Europe (n=28), and the US and Canada (n=21).

In the double-blind Part 1, 94 patients were randomized 2:1 to receive either 50 mg QD of pimicotinib (n=63) or placebo (n=31) for 24 weeks. The primary endpoint is objective response rate (ORR) at week 25, as measured by Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1 by blinded independent central review in the intent-to-treat (ITT) population. Secondary endpoints include tumor volume score, active range of motion, stiffness by Numeric Rating Scale (NRS), pain by Brief Pain Inventory (BPI), and physical function measured by Patient-Reported Outcomes Measurement Information System (PROMIS).

After the double-blind Part 1, eligible patients may continue to the open-label Part 2 for up to 24 weeks of dosing, results of which are expected in mid-2025. Patients who complete Part 2 may then enter the open-label extension phase (Part 3) for extended treatment and safety follow-up.

About Pimicotinib (ABSK021)

Pimicotinib (ABSK021), which is being developed by Abbisko Therapeutics, is a novel, orally administered, highly selective and potent small-molecule inhibitor of CSF-1R. Pimicotinib has been granted breakthrough therapy designation (BTD) for the treatment of inoperable TGCT by China National Medical Products Administration (NMPA) and the US Food and Drug Administration (FDA), and priority medicine (PRIME) designation from the European Medicines Agency (EMA).

On March 27, 2025 Ascentage Pharma Group International (Ascentage Pharma) (NASDAQ: AAPG; HKEX: 6855) (referred hereinto as "Ascentage Pharma," the "Company," "we," "us" or "our"), a global, integrated biopharmaceutical company engaged in discovering, developing and commercializing therapies to address global unmet medical needs primarily in hematological malignancies, reported its unaudited financial results for the year ended December 31, 2024, and provided updates on key clinical and commercial developments (Press release, Ascentage Pharma, MAR 27, 2025, View Source [SID1234654252]).

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Dr. Dajun Yang, Chairman and Chief Executive Officer of Ascentage Pharma, said, "As we reflect on our achievements in 2024, I am delighted to report that Ascentage Pharma has made remarkable strides in advancing our mission to deliver innovative therapies to patients worldwide. The commercialization of olverembatinib in China has gained significant traction in 2024 and is poised for growth in 2025 as all approved indications of olverembatinib are now covered under China’s National Reimbursement Drug List (NRDL), markedly enhancing affordability and accessibility for patients across China."

He continued, "Our momentum continued with the advancement of lisaftoclax. In November 2024, the New Drug Application (NDA) for lisaftoclax for the treatment of relapsed and/or refractory chronic lymphocytic leukemia (R/R CLL) and small lymphocytic lymphoma (SLL) was accepted by the Center of Drug Evaluation (CDE) of China’s National Medical Products Administration (NMPA) with Priority Review designation. This acceptance marks a pivotal step toward bringing this novel therapy to patients in need."

"Our clinical development programs also achieved significant progress over the past year. In February 2024, olverembatinib received clearance by the U.S. Food and Drug Administration (FDA) to initiate a global registrational Phase III clinical trial (POLARIS-2), for patients with Chronic Myeloid Leukemia in Chronic Phase (CML-CP) with or without T315I mutation who have previously failed tyrosine kinase inhibitor (TKI) treatment. In 2024, we also received clearance to commence two registrational Phase III clinical trials for APG-2449, a focal adhesion kinase (FAK), third generation anaplastic lymphoma kinase (ALK) and receptor tyrosine kinase C-ros oncogene 1 (ROS1) inhibitor, for treatment of patients with non-small cell lung cancer (NSCLC). At the moment, we are conducting ten global registrational trials, including two that were cleared by the FDA, for our three late-stage products, olverembatinib, lisaftoclax and APG-2449. These milestones highlight our commitment to addressing unmet medical needs through rigorous clinical innovation."

"We believe Ascentage Pharma is on a transformative path to becoming a global leader in oncology innovation. The commercialization of olverembatinib in China, the progress of lisaftoclax, the continued development of our other clinical-stage small molecule drug assets, and our strategic agreement with Takeda Pharmaceuticals International AG (Takeda) reflect the strength of our pipeline and our ability to execute on our goals. In 2025, we remain focused on accelerating the development and delivery of life-changing therapies, expanding our global footprint, and creating sustainable value for all stakeholders."

On March 27, 2025 Havah Therapeutics, a clinical stage biopharmaceutical company developing innovative therapies for prevention and treatment of breast cancers, reported the filing of a new provisional patent application directed to the use of HAV-088 for treating Ductal Carcinoma In-Situ (Press release, HavaH Therapeutics, MAR 27, 2025, View Source [SID1234651553]).

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Backed by human in vivo data demonstrating on-target effect, the patent application covers the potential application of HAV-088, a novel androgen receptor (AR) agonist, in treating hormone receptor positive (HR+) disease.

"The filing of this provisional patent application marks an important step in our broader strategy to strengthen our intellectual property portfolio with additional disease-specific claims," said Matthew Brewer, CEO of Havah Therapeutics. "This week’s release of 20-year survival data from a Mayo Clinic trial showed that just one year of adjuvant therapy with an AR agonist in early breast cancer led to a statistically significant reduction in recurrence or death.

"Endocrine therapy has been the foundation of treatment for hormone receptor-positive cancers for over 40 years, yet few advances have focused on the androgen receptor pathway. HAV-088, through AR agonism, is designed to reduce estrogen-driven gene transcription, thereby lowering estrogen receptor expression and limiting the tumor’s ability to respond to estrogen. Over time, we believe this could offer meaningful benefits to patients whose disease is resistant to standard endocrine therapies."

"The data supporting this provisional patent may represent a significant advancement in the treatment of HR-positive DCIS and builds upon the foundational research conducted by Dr. Wayne Tilley and myself," said Stephen Birrell, MD, PhD, Founder and Chair of Havah’s Clinical Advisory Board.