On April 28, 2025 Actinium Pharmaceuticals, Inc. (NYSE AMERICAN: ATNM) (Actinium or the Company), a pioneer in the development of targeted radiotherapies, reported data presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting supporting Actimab-A’s mutation agnostic antileukemic effect and backbone therapy potential in preclinical acute myeloid leukemia (AML) models (Press release, Actinium Pharmaceuticals, APR 28, 2025, View Source;302439223.html [SID1234652271]). The preclinical data demonstrate that the combination of Actimab-A with standard of care AML therapies including menin and FLT3 inhibitors and the hypomethylating agent (HMA) azacitidine resulted in significant antileukemic activity in AML cells lines with FLT3, NPM1, KMT2A and TP53 mutations. Additionally, in animal models, Actimab-A significantly enhanced tumor growth inhibition, prolonged the duration of response and survival when combined with the menin inhibitor revumenib (Syndax Pharmaceuticals, Inc.), and potentiated AML cell killing in combination with the FLT3 inhibitor gilteritinib (Astellas Pharma US, Inc.) and HMA azacitidine.

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Sandesh Seth, Actinium’s Chairman and CEO, said, "In multiple clinical trials, Actimab-A has demonstrated potent single agent activity, synergy with other therapeutic modalities and efficacy in patients with high-risk features such as a TP53 mutation. To our knowledge, Actimab-A is the only AML therapy to achieve all of these outcomes, which we attribute to its mutation agnostic, backbone therapy profile. The data presented at AACR (Free AACR Whitepaper) further support Actimab-A’s mutation agnostic mechanism of action across several of the most commonly expressed mutations and synergy with the targeted therapies approved for patients with these mutations. With multiple clinical trials underway and being planned across the AML treatment settings, we are focused on generating strong clinical outcomes starting in the second half of this year and committed to realizing Actimab-A’s potential for patients who have significant unmet needs."

Actimab-A is Actinium’s lead radiotherapy that delivers Actinium-225, a potent alpha-emitter radioisotope payload that can produce lethal double strand DNA breaks to kill targeted cells that express CD33. CD33 is expressed ubiquitously in AML and in other myeloid malignancies such as myeloid dysplastic syndromes (MDS), which Actinium estimates to be an addressable market of over 100,000 patients in the U.S. and EU5. Actimab-A is also being advanced into a pivotal Phase 2/3 trial in combination with the chemotherapy regimen CLAG-M in patients with relapsed or refractory AML and in newly diagnosed AML in combination with Venetoclax and ASTX-727 (Taiho Oncology, an Otsuka holdings company) an oral hypomethylating agent (HMA) under a cooperative research and development agreement (CRADA) with the National Cancer Institute (NCI). Actimab-A has demonstrated a mutation agnostic profile with positive clinical results in high-risk relapsed and refractory (r/r) AML patients including those with a TP53 gene mutation, prior Venetoclax treatment and prior bone marrow transplant (BMT).

The Actimab-A AACR (Free AACR Whitepaper) presentation is available for viewing on the Presentations& Webinars page of Actinium’s website HERE.

Title: Actimab-A (Lintuzumab-Ac-225) has potent mutation agnostic antileukemic activity in preclinical models of AML
Abstract Number: 594

On April 28, 2025 GlycoNex, Inc. (4168, hereinafter referred to as GNX), a clinical stage biotechnology company focused on the development of glycan-directed cancer immunotherapies, reported the presentation of preclinical data on GNX1021, its lead antibody-drug conjugate (ADC) program, at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2025 (Press release, GlycoNex, APR 28, 2025, View Source [SID1234652270]). The presentation is part of a poster session held on April 28, 2025, at McCormick Place in Chicago.

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GNX1021 is a novel ADC that targets branched Lewis B/Y (bLeB/Y), a tumor-associated glycan highly expressed in gastric cancer and other solid tumors. The target has a distinct expression profile compared to HER2 and CLDN18—two well-known markers in gastric cancer—allowing GNX1021 to potentially treat patients who are not eligible for current HER2- or CLDN18-targeted therapies.

The poster presents preclinical data showing GNX1021’s antitumor activity in gastric cancer models with high bLeB/Y expression, along with a favorable safety profile in nonclinical studies. Findings suggest that GNX1021 may offer a differentiated therapeutic option for gastric cancer patients with limited targeted treatment choices.

"These data demonstrate the potential of GNX1021 to expand the treatment landscape for gastric cancer by targeting a glycan biomarker not addressed by existing therapies," said Dr. Mei-Chun Yang, CEO of GlycoNex. "As a first-in-class anti-glycan ADC, GNX1021 exemplifies our approach to developing next-generation oncology therapeutics designed to overcome tumor heterogeneity and drug resistance."

GlycoNex is advancing GNX1021 through IND-enabling preclinical development, with an IND submission planned for Q1 2026 and Phase 1 trial initiation expected in Q2 2026.

Presentation Details:

Event:

American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2025

Session Category:

Experimental and Molecular Therapeutics

Session:

Growth Factor Receptors and Other Surface Antigens as Targets for Therapy 1

Abstract Title: (#2932)

GNX1021, a novel ADC targeting glycans with branched Lewis B/Y, demonstrated preclinical tumor control activity in gastric cancer models and favorable safety

Date and Time:

April 28, 2025; 2:00 pm CDT

Location:

McCormick Place Convention Center, Chicago

Poster Section 17, Poster Board 6

On April 28, 2025 AVEO Oncology, an LG Chem company ("AVEO"), a biopharmaceutical company committed to providing differentiated solutions to improve cancer patients lives, reported that three abstracts were accepted for presentation at the upcoming American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2025 annual meeting this May 30-June 3, 2025, in Chicago, IL (Press release, AVEO, APR 28, 2025, View Source [SID1234652269]).

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Presentation Details

Title: Efficacy of second line (2L) treatment with tivozanib (Tivo) as monotherapy or with nivolumab (Nivo) in patients (pts) with metastatic renal cell carcinoma (mRCC) previously treated with an immune checkpoint inhibitor (ICI) combination of ipilimumab (Ipi)/Nivo or vascular endothelial growth factor receptor-tyrosine kinase inhibitor (VEGFR-TKI)/ICI in the Phase 3 TiNivo-2 study
First Author: Alexander Chehrazi-Raffle, MD, City of Hope Cancer Center
Abstract Number: 4540
Poster Session: Genitourinary Cancer—Kidney and Bladder
Poster Board: 340
Date and Time: Monday, June 2, 2025, 9:00am – 12:00 PM CDT

Title: FIERCE-HN: A multicenter, randomized, double-blind, placebo-controlled, phase 3 study of ficlatuzumab (HGF/cMET Mab) in combination with cetuximab in participants with recurrent or metastatic (R/M) HPV negative head and neck squamous cell carcinoma (HNSCC)
First Author: Julie E. Bauman, MD, MPH, George Washington University Cancer Center
Abstract Number: TPS6115
Poster Session: Head and Neck Cancer
Poster Board: 520a
Date and Time: Monday, June 2, 2025, 9:00 AM – 12:00 PM CDT

Title: A phase1b dose escalation study of AV-380 (anti-GDF15 monoclonal antibody) in combination with standard-of-care therapy in cancer patients with cachexia
First Author: Eric Roeland, MD, FAAHPM, Oregon Health & Science University
Abstract Number: TPS12142
Poster Session: Symptom Science and Palliative Care
Poster Board: 159a
Date and Time: Monday, June 2, 2025, 1:30-4:30 PM CDT

On April 28, 2025 Boehringer Ingelheim reported new and updated data from the Beamion LUNG-1 trial evaluating zongertinib in previously treated patients with HER2 (ERBB2)-mutant advanced non-small cell lung cancer (NSCLC) (Press release, Boehringer Ingelheim, APR 28, 2025, View Source [SID1234652268]). The data was featured in the official press program at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2025 and simultaneously published in The New England Journal of Medicine.

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"These data presented at AACR (Free AACR Whitepaper) 2025 suggest that zongertinib may offer a new approach to treating patients with non-small cell lung cancer with activating HER2 mutations," said the trial’s coordinating investigator, Dr. John Heymach, MD, PhD, chair of Thoracic/Head and Neck Medical Oncology at The University of Texas MD Anderson Cancer Center. "Notably, more than 70% of patients experienced a tumor response, which is highly meaningful for those with this subtype of lung cancer. If approved by the FDA, zongertinib would be the first oral, targeted treatment option that addresses an unmet need for these patients."

Data from the most recent analysis showed durable response and clinically meaningful results with zongertinib in previously treated patients with advanced NSCLC who have HER2 mutations within the tyrosine kinase domain (TKD) (N=75). The ORR was 71% (95% CI: 60-80), with 7% complete response, 64% partial response, and 96% disease control in previously treated patients. Additionally, zongertinib had intracranial activity in previously treated patients (n=27, who were evaluable) with brain metastases, with 41% achieving response and 81% disease control. At AACR (Free AACR Whitepaper) 2025, the median DoR of 14.1 and median PFS of 12.4 months were presented for the first time.

Itziar Canamasas, Global Head of Oncology at Boehringer Ingelheim, said: "Zongertinib has the potential to reset the benchmark for patients with HER2-mutant advanced non-small cell lung cancer, a patient population that has historically faced a poor prognosis. At Boehringer, we take cancer care personally; these updated data reaffirm our approach of addressing areas with high unmet need and letting our research guide us to where we can have the biggest impact for patients."

Additional analyses of previously treated patients with HER2 mutations demonstrated zongertinib’s clinically meaningful results

Initial results in patients with advanced NSCLC with HER2 mutations in the TKD, who were previously treated with both platinum-based chemotherapy and subsequent HER2-directed antibody drug conjugates (ADC) (N=31), demonstrated an ORR of 48% (95% CI: 32-65) with 97% (95% CI: 15-52) of patients achieving disease control. An exploratory cohort (n=20) that included previously treated patients with advanced NSCLC with HER2 mutations outside of the TKD demonstrated an investigator-assessed ORR of 30% (95% CI: 15-52) and a DCR of 65% (95% CI: 43-82). This is the largest known dataset of patients with previously treated advanced NSCLC who have HER2 mutations outside the TKD. Both of these data sets were presented at AACR (Free AACR Whitepaper) 2025 and are included in The New England Journal of Medicine publication.

The data presented at AACR (Free AACR Whitepaper) 2025 demonstrated a manageable safety profile for zongertinib with no drug-related deaths, cases of interstitial lung disease (ILD) or cardiotoxicity reported. The most commonly reported adverse event (AE) was grade 1 diarrhea, with low incidence of grade ≥3 drug-related events (17%) in patients with TKD mutations (N=75).

AACR 2025 presentation: summary of key efficacy endpoints

Endpoint

Patients with TKD mutations (N = 75)

Patients with TKD mutations and
prior HER2-directed ADC treatment (N = 31)

Patients with non-TKD mutations (n = 20)

ORR, %

95% CI

71*

60-80

48*

32–65

30**

15-52

CR, %

7*

3*

0**

PR, %

64*

45*

30**

DCR, %

95% CI

96*

89-99

97*

84-99

65**

43-82

Median DoR

95% CI

14.1 months***

6.9–NE

n/a

n/a

Median PFS

95% CI

12.4 months***

8.2–NE

n/a

n/a

*Confirmed response by BICR according to RECIST v1.1
**Confirmed response by investigator review according to RECIST v1.1
*** Median DoR and median PFS are based on Kaplan Meier estimates

About non-small cell lung cancer (NSCLC)
Lung cancer claims more lives than any other cancer type1 and the incidence is set to increase to over 3 million cases worldwide by 2040.2 NSCLC is the most common type of lung cancer.3 The condition is often diagnosed at a late stage,4 and fewer than 3 in 10 patients are alive five years after diagnosis.5 People living with advanced NSCLC can experience a detrimental physical, psychological, and emotional impact on their daily lives. There remains a high unmet need for additional treatment options for people living with advanced NSCLC. Up to 4% of lung cancers are driven by HER2 mutations (or gene alterations).6 Mutations in HER2 can lead to overexpression and overactivation, which can in turn result in uncontrolled cell production, inhibition of cell death and promotion of tumor growth and spread.7

About zongertinib
Zongertinib is an investigational irreversible tyrosine kinase inhibitor (TKI) that selectively inhibits HER2 while sparing wild-type EGFR, thereby limiting associated toxicities. This orally administered, targeted therapy was granted FDA Fast Track Designation in 2023, followed by Breakthrough Therapy Designation in the U.S. and China for the treatment of adult patients with unresectable or metastatic NSCLC whose tumors have activating HER2 mutations and who have received prior systemic therapy. An application for accelerated approval was granted Priority Review status by the FDA in February 2025. In addition, Japan’s Pharmaceuticals and Medical Devices Agency granted Orphan Drug Designation to zongertinib.

A recent study has shown pre-clinically that the investigational compound zongertinib has potential for further clinical study in HER2 dependent solid cancers as monotherapy and as concurrent treatment with ADC therapy. In addition, zongertinib is being evaluated in Beamion LUNG-2 (NCT06151574)4, a global Phase III trial, compared to standard of care as first-line treatment in patients with unresectable, locally advanced or metastatic NSCLC who have activating HER2 TKD mutations.

About the Beamion clinical trial program
Beamion LUNG-1 (NCT04886804): An open-label, Phase I dose escalation trial, with dose confirmation and expansion, of zongertinib as monotherapy in people with advanced or metastatic solid tumors and NSCLC with activating HER2 alterations. The study has 2 parts. The first part is open to adults with different types of advanced cancer (solid tumors with changes in the HER2 gene) for whom previous treatment was not successful. The second part is open to people with advanced non-small cell lung cancer with a specific mutation in the HER2 gene. Beamion LUNG-2 is a phase 3, open label, randomized, active-controlled study in patients with unresectable, locally advanced or metastatic non-squamous NSCLC harboring HER2 TKD mutations to evaluate zongertinib compared with standard of care.

On April 28, 2025 SOPHiA GENETICS (Nasdaq: SOPH), a cloud-native healthcare technology company and global leader in data-driven medicine, reported the expansion of its ongoing collaboration with AstraZeneca (LSE/STO/Nasdaq: AZN) to accelerate the deployment of MSK-ACCESS powered with SOPHiA DDM globally (Press release, AstraZeneca, APR 28, 2025, View Source [SID1234652267]). Building on the initial collaboration announced in October 2024, this new phase will extend the test’s reach to a total of 30 clinical institutions worldwide in 2025.

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Developed in collaboration with Memorial Sloan Kettering Cancer Center, MSK-ACCESS powered with SOPHiA DDM is an innovative liquid biopsy testing application designed to detect actionable genomic alterations from a single blood draw using proprietary, state-of-the-art algorithms which analyze circulating tumor DNA (ctDNA). The application supports real-time cancer monitoring and treatment selection when traditional tissue biopsies are not feasible due to cost, turnaround time, insufficient tissue, or the invasiveness of the procedure.

The expanded rollout will continue to contribute to AstraZeneca’s global real-world evidence initiatives and help further validate the clinical impact of decentralized liquid biopsy testing across diverse healthcare systems. By increasing the availability of MSK-ACCESS powered with SOPHiA DDM, SOPHiA GENETICS and AstraZeneca aim to understand further how liquid biopsy testing can complement solid tissue testing and, in some cases, provide greater benefit for labs and patients.

The announcement coincides with SOPHiA GENETICS’s presentation at AACR (Free AACR Whitepaper), in which the company presented data demonstrating the robust transferability of the decentralized MSK-ACCESS powered with SOPHiA DDMTM solution. Historically, site-to-site discordance has been a major barrier to the widespread adoption of liquid biopsy testing. SOPHiA GENETICS presented real-world data highlighting the consistent accuracy and precision of MSK-ACCESS powered with SOPHiA DDMTM across various laboratory settings. Interim results from the multi-center study demonstrated the high analytical performance of the decentralized test in line with the original single-site test at Memorial Sloan Kettering Cancer Center in New York.

"Our collaboration with AstraZeneca represents a significant step toward scaling next-generation oncology diagnostics globally," said Ross Muken, President, SOPHiA GENETICS. "This expanded deployment reflects the growing momentum behind liquid biopsy and our shared ambition to make these technologies more accessible and impactful worldwide."

Visit SOPHiA GENETICS at the American Association for Cancer Research (AACR) (Free AACR Whitepaper)’s Annual Meeting in Chicago, Illinois from April 25th–30th to learn more about how SOPHiA GENETICS is collaborating with AstraZeneca to transform patient care with data-driven medicine.