On August 18, 2025 Aptose Biosciences Inc. ("Aptose" or the "Company") (OTC: APTOF, TSX: APS), a clinical-stage precision oncology company developing the tuspetinib (TUS)-based triple drug frontline therapy to treat patients with newly diagnosed AML, reported a data update from the Phase 1/2 TUSCANY trial in newly diagnosed AML (Press release, Aptose Biosciences, AUG 18, 2025, View Source [SID1234655342]). The trial was initiated in December 2024, and the growing body of positive data includes the recently completed third cohort of 120 mg TUS in the TUS+VEN+AZA triplet therapy. Data to date from ten (10) patients across all three cohorts, 40 mg, 80 mg or 120 mg TUS dose in TUS+VEN+AZA, support the use of TUS with standard of care treatment across all AML populations, including those carrying mutations that are the most difficult to treat and those with mutated and unmutated (wildtype) FLT3 genes.

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The TUS+VEN+AZA triplet is being developed as a safe and well-tolerated mutation agnostic frontline therapy to treat large, mutationally diverse populations of newly diagnosed AML patients who are ineligible to receive induction chemotherapy. At the 120 mg TUS dose level in combination with VEN+AZA, as with the prior reported 40 mg and 80 mg TUS dose cohorts, no significant safety concerns or dose limiting toxicities (DLTs) have been observed in the TUSCANY trial, including no prolonged myelosuppression in Cycle 1 of subjects in remission, no reports of drug-related QTc prolongation or differentiation syndrome (DS), and no treatment-related deaths. Nine out of ten dosed patients remain on study across all dose cohorts and enrollment is being advanced to the 160 mg TUS dose level following the Cohort Safety Review Committee (CSRC) meeting.

"We already have data from three different TUS dose levels in the TUSCANY trial, and the data continue to strengthen at higher doses of TUS and over time. We are building a strong case for TUS+VEN+AZA as a triplet frontline therapy of choice to address a broad AML population, including subgroups with the most adverse of mutations," said William G. Rice, Ph.D., Chairman, President and Chief Executive Officer of Aptose.

Data highlights:

Comparison of CR/CRh1 Response rates2-5:

VEN+AZA2 TUS+VEN+AZA
All subjects 65% 90% (9/10)
NPM1-mutant 67% 100% (2/2)
FLT3-ITD 61% 100% (2/2)
TP53-mutant 52% 100% (2/2)

Comparison of MRD-negativity6 rates among All Subjects and among CR/CRh Responders3:

VEN+AZA2,3 TUS+VEN+AZA
Among All Subjects 23.4% 70% (7/10)
Among CR/CRh Responders 40.9% 78% (7/9)

Comparison of MRD-negativity rates among more difficult-to-treat Patient Subpopulations defined as Lower Benefit (TP53-mutated) and Intermediate Benefit (FLT3-ITD or RAS-mutated) relative to VEN+AZA5:

VEN+AZA3,5 TUS+VEN+AZA
Intermediate Benefit 27.9% 100% (3/3)
Lower Benefit 14.5% 100% (2/2)

TUS+VEN+AZA – CR/CRh and MRD-negativity rates among Subjects with Adverse Mutations:

TP53, FLT3-ITD, RAS mutations: Achieved CR/CRh and MRD-negativity
100% (5/5)

"As illustrated in the data highlights, the addition of TUS to VEN+AZA appears to boost response rates and MRD-negativity while maintaining favorable safety and tolerability," said Rafael Bejar, M.D., Ph.D., Chief Medical Officer of Aptose, "and the 100% CR/CRh and 100% MRD-negativity rates among the five biallelic TP53-mutant, FLT3-ITD, and RAS-mutant AML cases are exciting to see, as this can correlate with longer overall survival. We have observed a trend towards achieving CRs more quickly at the higher dose levels, so we are keen to see the activity as we advance into the 160 mg TUS dose cohort."

Key messages:

Addition of TUS to VEN+AZA demonstrates excellent CR/CRh rates
100% CR/CRh among all subjects treated at 80 mg and 120 mg TUS dose levels
Appear to be achieving CR earlier with 120 mg TUS than with 40 mg or 80 mg
Addition of TUS to VEN+AZA demonstrates excellent MRD-negativity rates
MRD-negativity in 7 of 9 (78%) already achieved in patients who responded to therapy
Expect patient survival to be extended with continued long-term treatment
Excellent safety and well tolerated with no dose-limiting toxicities (No DLT) at completed dose levels
Broad-spectrum activity including patients with adverse TP53, RAS and FLT3-ITD mutations
No loss of MRD-negativity observed to date, including in one patient with over 7 months of follow up
MRD-negativity and remissions continue to mature over time on therapy
No relapses reported to date and no treatment related deaths
The only non-responder was a patient at the initial TUS dose level (40 mg) that did not achieve TUS exposures previously associated with response
Additional data are included in the new Aptose corporate presentation here.

TUSCANY: TUS+VEN+AZA Triplet Phase 1/2 Study

The 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, durable, and well tolerated.

The TUSCANY triplet Phase 1/2 study, being conducted at 10 leading U.S. clinical sites by elite clinical investigators, is designed to test various doses and schedules of TUS in combination with standard dosing of AZA and VEN for patients with AML who are ineligible to receive induction chemotherapy. A convenient, once daily oral agent, TUS is being administered in 28-day cycles. Multiple U.S. sites are enrolling in the TUSCANY trial with anticipated enrollment of 18-24 patients by late 2025. Data will be released as it becomes available.

More information on the TUSCANY Phase 1/2 study can be found on www.clinicaltrials.gov (here: View Source).

On August 18, 2025 Merck & Co. reported that Ifinatamab deruxtecan (I-DXd) has been granted Breakthrough Therapy Designation (BTD) by the U.S. Food and Drug Administration (FDA) for the treatment of adult patients with extensive-stage small cell lung cancer with disease progression on or after platinum-based chemotherapy (Press release, Merck & Co, AUG 18, 2025, View Source [SID1234655360]).

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Ifinatamab deruxtecan is a specifically engineered, potential first-in-class B7-H3 directed DXd antibody drug conjugate (ADC) discovered by Daiichi Sankyo (TSE: 4568) and being jointly developed by Daiichi Sankyo and Merck (NYSE: MRK), known as MSD outside of the United States and Canada.

The FDA BTD is designed to accelerate the development and regulatory review of potential new medicines that are intended to treat a serious condition and address a significant unmet medical need. The medicine is required to have shown encouraging preliminary clinical results that demonstrate substantial improvement on a clinically significant endpoint over currently available medicines.

The FDA granted the BTD based on data from the IDeate-Lung01 Phase 2 trial, with support from the IDeate-PanTumor01 Phase 1/2 trial. Results from the primary analysis of IDeate-Lung01 will be presented in a late-breaking oral presentation at the IASLC 2025 World Conference on Lung Cancer hosted by the International Association for the Study of Lung Cancer (#WCLC25). This is the first BTD for ifinatamab deruxtecan and represents the first BTD since the start of the Daiichi Sankyo and Merck collaboration.

"This Breakthrough Therapy Designation granted by the FDA to ifinatamab deruxtecan highlights the urgent need for new treatment options for patients with pretreated extensive-stage small cell lung cancer," said Ken Takeshita, MD, global head, R&D, Daiichi Sankyo. "We are committed to advancing this medicine with the goal of bringing the first B7-H3 directed antibody drug conjugate to patients in order to transform the outcomes of those facing this aggressive disease."

"Patients living with extensive-stage small cell lung cancer often have limited therapeutic options following disease progression after standard of care treatments," said Eliav Barr, MD, senior vice president, head of global clinical development and chief medical officer, Merck Research Laboratories. "This Breakthrough Therapy Designation reinforces our confidence in the promise of ifinatamab deruxtecan to play an important role in the treatment of extensive-stage small cell lung cancer, and we are looking forward to sharing data at the upcoming IASLC 2025 World Conference on Lung Cancer that show the potential of this novel option."

About IDeate-Lung01

IDeate-Lung01 is a global, multicenter, randomized, open-label, two-part Phase 2 trial evaluating the safety and efficacy of ifinatamab deruxtecan in patients with extensive-stage small cell lung cancer who were previously treated with at least one prior line of platinum-based chemotherapy and a maximum of three prior lines of therapy. Patients with asymptomatic brain metastases (untreated or previously treated) were eligible.

In the first part of the trial (dose optimization), patients were randomized 1:1 to receive ifinatamab deruxtecan 8 or 12 mg/kg intravenously Q3W. In the second part of the trial (dose expansion), patients received ifinatamab deruxtecan 12 mg/kg intravenously Q3W.

The primary endpoint is objective response rate (ORR) as assessed by blinded independent central review (BICR) per RECIST v1.1. Secondary endpoints included duration of response, progression-free survival, disease control rate, time to response, overall survival, pharmacokinetics and safety. Intracranial ORR was assessed by BICR as an exploratory analysis.

IDeate-Lung01 enrolled 187 patients in Asia, Europe and North America. For more information about the trial, visit ClinicalTrials.gov.

About IDeate-PanTumor01

IDeate-PanTumor01 is a global, multicenter, first-in-human, open-label Phase 1/2 trial evaluating the safety and efficacy of ifinatamab deruxtecan in patients with advanced/unresectable or metastatic solid tumors that are refractory or intolerable to standard treatment or for whom no standard treatment exists.

The Phase 1 part of the trial (dose escalation) is assessing the safety and tolerability of increasing doses of ifinatamab deruxtecan to determine the maximum tolerated dose and recommended dose for expansion (RDE). The Phase 2 part of the trial (dose expansion) is evaluating the safety and efficacy of ifinatamab deruxtecan at the RDE of 12 mg/kg in patients with squamous non-small cell lung cancer, metastatic castration-resistance prostate cancer or esophageal squamous cell carcinoma.

The dose escalation part of the trial is evaluating dose-limiting toxicity and safety. The dose expansion part of the trial is evaluating overall response rate, duration of response, disease control rate, progression-free survival, overall survival and safety. Pharmacokinetic endpoints, exploratory biomarker and immunogenicity endpoints will also be assessed.

IDeate-PanTumor01 enrolled approximately 250 patients in Asia and North America. For more information about the trial, visit ClinicalTrials.gov.

About small cell lung cancer

More than 2.48 million lung cancer cases were diagnosed globally in 2022. Small cell lung cancer (SCLC) is the second most common type of lung cancer, accounting for approximately 15% of cases. SCLC is aggressive and progresses rapidly to the distant metastatic stage, which has a low five-year survival rate. While conventional standard of care treatments for patients with advanced SCLC may help improve outcomes, there is a need for additional subsequent treatment approaches.

About B7-H3

B7-H3 is a transmembrane protein that belongs to the B7 family of proteins, which bind to the CD28 family of receptors that includes PD-1. B7-H3 is overexpressed in a wide range of cancer types, including SCLC, and its overexpression has been shown to correlate with poor prognosis, making B7-H3 a promising therapeutic target. There are currently no B7-H3 directed medicines approved for the treatment of any cancer.

About ifinatamab deruxtecan

Ifinatamab deruxtecan (I-DXd) is an investigational potential first-in-class B7-H3 directed ADC. Designed using Daiichi Sankyo’s proprietary DXd ADC Technology, ifinatamab deruxtecan is comprised of a humanized anti-B7-H3 IgG1 monoclonal antibody attached to a number of topoisomerase I inhibitor payloads (an exatecan derivative, DXd) via tetrapeptide-based cleavable linkers.

Ifinatamab deruxtecan has been granted orphan drug designation by the U.S. FDA, European Commission, Japan Ministry of Health, Labour and Welfare and Taiwan Food and Drug Administration for the treatment of SCLC.

About the ifinatamab deruxtecan clinical development program

A comprehensive global clinical development program is underway evaluating the efficacy and safety of ifinatamab deruxtecan monotherapy and in combination with other anticancer medicines across multiple cancers.

On August 15, 2025 Asta Bio and Alpha Nuclide reported that it will combine expertise in targeted alpha radiotherapy and radionuclide manufacturing to accelerate the discovery and development of novel cancer treatments (Press release, Asta Bio, AUG 15, 2025, View Source [SID1234656370]).

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BOSTON, Aug. 15, 2025 /PRNewswire-PRWeb/ — Asta Bio, a biopharmaceutical company developing next-generation targeted alpha radiotherapies, reported a discovery and development partnership with Alpha Nuclide, a leading provider of specialized radionuclide manufacturing and radiopharmaceutical supply chain solutions.

Under the agreement, Alpha Nuclide will support Asta Bio on clinical translation of Asta Bio’s pipeline through coordinated efforts in discovery research and drug product manufacturing for preclinical and clinical studies. The collaboration is designed to ensure accelerated entry into human trials for Asta Bio’s lead 211-Astatine-RadiobodyTM drug candidates targeting solid tumors. This collaboration is part of Alpha Nuclide’s broader mission to provide logistical solutions to enable drug developers worldwide to bring targeted alpha therapies into the clinic and accelerate their discoveries.

"We are honored to partner with Alpha Nuclide, whose team has the deepest technical expertise and track record in 211-Astatine radionuclide production and chemistry and makes them an ideal collaborator for advancing our programs" said Ahmad-Reza Saadat, Co-Founder and CEO of Asta Bio. "This collaboration is a critical step toward bringing our 211-Astatine-Radiobody therapies to patients and ensuring that we can scale production and delivery as we move into clinical development."

Alpha Nuclide brings specialized experience in the production, purification, and delivery of alpha-emitting isotopes, including 211-Astatine, which must be manufactured with precision and coordinated timing for clinical use. The company will provide key infrastructure to support radioisotope supply chain logistics, contract preclinical research services, GMP compliance, and study coordination with clinical sites.

"We are excited to collaborate with Asta Bio as they pioneer a new generation of targeted alpha therapies" said Dr. Yutian Feng, CEO of Alpha Nuclide. "Their Radiobody platform represents a truly novel way to harness the power of 211-Astatine, and we are committed to supporting their mission to improve outcomes for patients with aggressive cancers."

The collaboration between Asta Bio and Alpha Nuclide will support preclinical studies and clinical readiness for the first human trials.

On August 15, 2025 AIM ImmunoTech Inc. (NYSE American: AIM) ("AIM" or the "Company") reported its financial results for the second quarter 2025 (Press release, AIM ImmunoTech, AUG 15, 2025, View Source [SID1234655331]).

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"Through the second quarter, we continued to prioritize and accelerate our development efforts for Ampligen, particularly in our Phase 2 trial for locally advanced pancreatic cancer in collaboration with Erasmus Medical Center and AstraZeneca. We remain encouraged by the growing body of positive clinical data demonstrated to date and believe that as we build momentum, we are well positioned to execute on a clear path toward government approval. Pancreatic cancer is one of the most aggressive and difficult-to-treat cancers, and our mission remains focused on making a meaningful difference in this space for patients," commented AIM Chief Executive Officer Thomas K. Equels.

Recent Highlights

Resumed trading on the NYSE American;
Closed public equity offering in July, raising $8.0 million in gross proceeds, which is expected to fund operations for approximately 12 months;
Reported positive data in a mid-year update from the ongoing Phase 2 DURIPANC clinical study evaluating Ampligen in combination with AstraZeneca’s anti-PD-L1 immune checkpoint inhibitor Imfinzi (durvalumab) in the treatment of metastatic pancreatic cancer patients with stable disease post-FOLFIRINOX (See: NCT05927142);
Announced upcoming presentations at the International 5th Annual Marie Sklodowska-Curie Symposium on Cancer Research and Care;
Ampligen oncology data was presented at scientific congresses including the Annual Meeting of the American Association of Immunologists and U.S.-Poland Science and Technology Symposium 2025.
For more information, please visit the Company’s website at aimimmuno.com.

Summary of Financial Highlights for Second Quarter 2025

As of June 30, 2025, AIM reported cash, cash equivalents and marketable investments of $835,000.
Research and development expenses for the three months ended June 30, 2025 were $1.2 million, compared to $1.1 million for the same period in 2024.
General and administrative expenses were $1.5 million for the three months ended June 30, 2025, compared to $2.6 million for the same period 2024.
The net loss from operations for the three months ended June 30, 2025 was $2.8 million, or $(3.68) per share, compared to $1.8 million, or $(3.00) per share, for the three months ended June 30, 2024.
Please refer to the full 10-Q for complete details.

On August 15, 2025 Propanc Biopharma, Inc. (OTC Pink: PPCB) ("Propanc" or the "Company"), a biopharmaceutical company developing novel cancer treatments for patients suffering from recurring and metastatic cancer, reported the pricing of an underwritten public offering of 1,000,000 shares of its common stock, par value $0.001 per share, at a price of $4.00 per share (Press release, Propanc, AUG 15, 2025, View Source [SID1234655332]). The offering is expected to yield approximately $4 million in gross proceeds before offering expenses and underwriting discounts and commissions. Propanc intends to use the net proceeds from the offering for its working capital and general business purposes.

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In connection with the offering, the Company has also granted the underwriter a 45-day option to purchase up to 150,000 additional shares of common stock. The offering is expected to close on August 18, 2025, subject to customary closing conditions. On August 14, 2025, the Company received approval to list its common stock on the Nasdaq Capital Market stock exchange ("Nasdaq"). Trading on Nasdaq is expected to commence on August 15, 2025, subject to continued compliance with the exchange rules.

The Company’s shares of common stock were previously quoted on the OTC Markets, Pink Tier and ceased to be quoted at the close of business on August 14, 2025. Stockholders are not required to take any action as a result of the uplisting, and the Company’s ticker symbol "PPCB" will remain unchanged.

D. Boral Capital LLC and Craft Capital Management LLC are acting as book running managers for the offering.

A registration statement on Form S-1 relating to these securities was declared effective by the U.S. Securities and Exchange Commission (the "SEC") on August 13, 2025. These securities may not be sold, nor may offers to buy these securities be accepted, prior to the time the registration statement becomes effective. This press release shall not constitute an offer to sell or the solicitation of an offer to buy, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

The proposed offering will be made only by means of a prospectus. Electronic copies of the preliminary prospectus supplement and accompanying prospectus will also be available on the SEC’s website at View Source