On May 22, 2025 Taiho Pharmaceutical Co., Ltd. reported that its U.S. subsidiary, Taiho Oncology, Inc., will present the results of a Phase 1/2 clinical trial of the combination regimen of decitabine and cedazuridine plus venetoclax for acute myeloid leukemia (AML) in an oral presentation at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, to be held May 30 through June 3 in Chicago (Press release, Taiho, MAY 22, 2025, View Source [SID1234653319]).

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This Phase 1/2 trial evaluated the safety and efficacy of an oral administration regimen of decitabine-cedazuridine paired with venetoclax in 101 adult　AML patients who were ineligible for first-line induction chemotherapy.

Study results
Complete remission (CR), which was the primary endpoint of the study, was 46.5%, and CR with incomplete hematologic recovery rates was 63.4%. Median time to CR was 2.4 months. Median CR duration was not reached; among patients who achieved it, 80% maintained that status at 6 months and 75.3% at 12 months. Median OS was 15.5 months.

98% of patients reported treatment-emergent adverse events of grade 3 or lower, most commonly febrile neutropenia (49.5%), anemia (38.6%) and neutropenia (35.6%). The 30- and 60-day mortality rates were 3% and 9%, respectively.

About acute myeloid leukemia (AML)

AML is a disease in which the proliferation of leukemia cells inhibits the normal production of blood in the bone marrow. It is the most common type of acute leukemia in adults, and its incidence increases with age. There is a need for less toxic treatment methods for elderly patients who have difficulty receiving first-line induction chemotherapy.

About decitabine-cedazuridine

This combination is the world’s first oral DNA methylation inhibitor combined with a novel metabolic inhibitor that inhibits the breakdown of decitabine when administered orally.
It was approved in the U.S. and Canada in 2020 for the indications of myelodysplastic syndromes (MDS) and chronic myelomonocytic leukemia (CMML), and in Europe in 2023 for the indication of AML.

On May 22, 2025 Accent Therapeutics, a clinical-stage biopharmaceutical company pioneering novel, targeted, small molecule cancer therapeutics, reported that a Trial in Progress poster for its first-in-human, Phase 1/2 study of its first-in-class oral DHX9 inhibitor, ATX-559, will be presented at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, held May 30-June 3 in Chicago, Illinois (Press release, Accent Therapeutics, MAY 22, 2025, View Source [SID1234653335]).

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"As a novel inhibitor of DHX9, a previously undrugged RNA and DNA/RNA helicase, ATX-559 offers potential as a unique therapeutic approach for addressing cancers characterized by high replication stress," said Jason Sager, M.D., Chief Medical Officer of Accent Therapeutics. "We are encouraged by the robust suite of data that continue to support the clinical evaluation of ATX-559 in our ongoing Phase 1/2 study."

Initiated in late 2024, the first-in-human, Phase 1/2, open-label, dose-escalation and expansion study is designed to evaluate multiple doses of ATX-559 in solid tumor patients, with expansion cohorts of patients with advanced or metastatic BRCA-1 and/or BRCA-2-deficient breast cancer and microsatellite instability-high (MSI-H) and/or deficient mismatch repair (dMMR) solid tumors (NCT06625515). Primary endpoints include identification of the recommended Phase 2 dose (RP2D) and assessment of the safety and tolerability of ATX-559. The study additionally aims to characterize the pharmacokinetics (PK), pharmacodynamics (PD), and preliminary anti-tumor activity (as assessed per RECIST v1.1 criteria) of orally administered ATX-559. The study is currently open and enrolling. Additional undisclosed solid tumor indications undergoing replicative stress and representing significant patient populations may be explored either in parallel or in subsequent studies.

Details for the presentation are as follows:

Presentation Title: Trial in Progress: First-in-Human Study of ATX-559, an Oral Inhibitor of DHX9, in Patients with Advanced or Metastatic Solid Tumors, and Molecularly Defined Cancers

Abstract Number: TPS3181
Session Type: Poster Session
Session Title: Developmental Therapeutics—Molecularly Targeted Agents and Tumor Biology
Session Date and Time: Monday, June 2, 1:30 pm – 4:30 pm CDT
Location: Hall A – Posters and Exhibits | On Demand
Presenter: Meredith Pelster, M.D., M.Sc.
The poster will be available on the Accent Therapeutics website following the meeting.

About ATX-559
ATX-559 is a first-in-class potent and selective inhibitor of DHX9, a novel and previously undrugged RNA and DNA/RNA helicase, shown to play a critical role in tumors with high levels of replication stress (including breast, ovarian, colorectal, endometrial, gastric, and others), representing large patient populations with significant unmet medical need. DHX9 has been reported to play important roles in replication, transcription, translation, RNA splicing, RNA processing, and maintenance of genomic stability, making it a compelling novel oncology target. In addition to exploiting key tumor vulnerabilities in DNA repair deficient backgrounds (e.g., BRCA) and hyper-mutated states (e.g., MSI-H/dMMR), Accent is exploring the sensitivity of other tumor types to DHX9 inhibition, and the potential to combine DHX9 inhibitors with other cancer treatments to maximize its full potential for helping patients. Accent retains full worldwide rights to ATX-559, currently being evaluated in a Phase 1/2 clinical trial (NCT06625515), and the DHX9 program.

On May 22, 2025 NeoGenomics, Inc. (NASDAQ: NEO), a leading provider of oncology testing services, reported the commercial launch of c-MET CDx for NSCLC, its c-MET companion diagnostic immunohistochemistry (IHC) assay (Press release, NeoGenomics Laboratories, MAY 22, 2025, View Source [SID1234653351]). The test is now available to oncologists and pathologists nationwide, supporting treatment selection for patients with advanced non-small cell lung cancer (NSCLC) with a 48-hour turnaround time.

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The c-MET CDx for NSCLC assay detects c-Met protein overexpression, a biomarker observed in up to 50% of patients with advanced NSCLC.¹ It is designed to help identify patients who may be eligible for newly approved targeted therapies, including EMRELIS (telisotuzumab vedotin-tllv), which was recently approved by the U.S. Food and Drug Administration (FDA).*

"Accurate and timely biomarker testing is critical in lung cancer, where targeted therapies can meaningfully change the course of a patient’s treatment," said Dr. Nathan Montgomery, Vice President of Medical Services at NeoGenomics. "The c-MET CDx for NSCLC assay adds an important tool to our testing portfolio, helping oncologists quickly identify patients who may benefit from MET-directed therapies. It also complements our PanTracer suite, enabling comprehensive biomarker profiling for NSCLC."

Key features of NeoGenomics’ assay include:

Companion Diagnostic Indication: Developed in accordance with FDA guidance and validated for use with MET-targeted therapies.
Fast Turnaround: Delivers results within 48 hours to enable timely, informed clinical decisions.
Validated Performance: Designed for use with tumor tissue samples to detect MET protein overexpression.
Integrated NSCLC Offering: Complements NeoGenomics’ broader PanTracer portfolio, including genomic and immuno-oncology markers.
The c-MET CDx for NSCLC assay is now available as part of NeoGenomics’ comprehensive NSCLC testing portfolio. Its addition supports the growing use of MET-directed therapies and reflects ongoing efforts to align diagnostic services with emerging standards in precision cancer care.

For more information or to order the test, visit www.neogenomics.com/cmetcdx.

*EMRELIS (telisotuzumab vedotin-tllv) was approved by the U.S. FDA on May 14, 2025, for adults with previously treated advanced NSCLC with high c-MET protein overexpression.

On May 22, 2025 Genmab A/S (Nasdaq: GMAB) reported that it will present new research from its comprehensive development program evaluating its late-stage portfolio at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, taking place from May 30 to June 3 in Chicago, Illinois (Press release, Genmab, MAY 22, 2025, View Source [SID1234653305]).

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The presentations will include the first disclosure of results from a Phase 1/2 trial evaluating rinatabart sesutecan (Rina-S), an investigational folate receptor-alpha (FRa)-targeted, TOPO1-inhibor antibody-drug conjugate (ADC), in patients with recurrent/advanced endometrial cancer. Additionally, results from an analysis of the Phase 1/2 EPCORE NHL-1 study of epcoritamab, a T-cell–engaging bispecific antibody administered subcutaneously, including long-term follow-up in adult patients with relapsed/refractory diffuse large B-cell lymphoma (DLBCL) who remain in complete response (CR) at 2 years, will be presented.

"Our presence at ASCO (Free ASCO Whitepaper) reflects our commitment to advancing our antibody science for patients in need of alternative treatment options, including women with endometrial cancers that have progressed following treatment with existing available therapies," said Dr. Judith Klimovsky, Executive Vice President and Chief Development Officer of Genmab. "We’re particularly encouraged by Rina-S as a potential treatment option for endometrial cancer, one of the few cancers with rising mortality rates and few treatment options. Additionally, together with AbbVie, we are continuing our commitment to evaluating epcoritamab as a potential core therapy across B-cell malignancies."

Investor Update at ASCO (Free ASCO Whitepaper) 2025
On Monday, June 2 at 4:00 PM CDT (5:00 PM EDT/11:00 PM CEST), Genmab will host a review of the Rina-S data presented at ASCO (Free ASCO Whitepaper). The event will be virtual and webcast live. Details, including the webcast link and registration, will be available on View Source This meeting is not an official program of the ASCO (Free ASCO Whitepaper) Annual Meeting.

All abstracts accepted for presentation have been published and may be accessed online via the ASCO (Free ASCO Whitepaper) Meeting Library. Abstracts accepted for presentation at ASCO (Free ASCO Whitepaper) include:

Rinatabart sesutecan (Rina-S)

Abstract Number Abstract Title Type of Presentation Date/Time of Presentation
3039 Winer et al. Rinatabart sesutecan (Rina-S) for patients with advanced endometrial cancer: First disclosure from dose expansion cohort B2 of the GCT1184-01 study. Poster June 2; 1:30 p.m.-4:30 p.m. CDT

TPS5627 Secord et al. A phase 3, open-label, randomized study of rinatabart sesutecan (Rina-S) vs investigator’s (IC) of chemotherapy in patients with platinum-resistant ovarian cancer Poster June 1, 9:00 a.m.-12:00 p.m. CDT
Epcoritamab

Abstract
Number Abstract Title Type of Presentation Date/Time of Presentation
7043 Karimi et al. Novel analysis of 3-y results from the pivotal EPCORE NHL-1 study: Outcomes in patients with relapsed/refractory large B-cell lymphoma and complete response at 2 y with epcoritamab monotherapy Poster June 1; 9:00 a.m.-12:00 p.m. CDT
e19001 Zhao et al. First data from phase 1b/2 EPCORE NHL-4: epcoritamab (epcor) in Chinese patients (Pts) with relapsed or refractory diffuse large B-cell lymphoma Publication NA
Non-Asset

Abstract
Number Abstract Title Type of Presentation Date/Time of Presentation
6046 Maghsoudi et al. Fusion of Radiomic, Pathomic, and Clinical Biomarkers Reveals Multi-scale Tumor Biology, Improves OS
Stratification in HNSCC receiving Standard of Care (SOC) Poster June 2; 9:00 a.m.-12:00 p.m. CDT
The safety and efficacy of investigational products and uses have not been established.

About Rinatabart Sesutecan (Rina-S; GEN1184)
Rinatabart sesutecan (Rina-S; GEN1184) is a FRα-targeted, TOPO1 ADC, currently being evaluated for the potential treatment of ovarian cancer and other FRα-expressing cancers. A Phase 3 trial (RAINFOLTM-02, NCT06619236) evaluating Rina-S in patients with platinum resistant ovarian cancer compared to treatment of investigator’s choice is ongoing. In January 2024, the U.S. Food and Drug Administration granted Fast Track designation to Rina-S for the treatment of patients with FRα-expressing high-grade serous or endometrioid platinum-resistant ovarian cancer.

The safety and efficacy of rinatabart sesutecan has not been established. Please visit www.clinicaltrials.gov for more information.

About Epcoritamab
Epcoritamab is an IgG1-bispecific antibody created using Genmab’s proprietary DuoBody technology and administered subcutaneously. Genmab’s DuoBody-CD3 technology is designed to direct cytotoxic T cells selectively to elicit an immune response toward target cell types. Epcoritamab is designed to simultaneously bind to CD3 on T cells and CD20 on B cells and induces T-cell-mediated killing of CD20+ cells.i

Epcoritamab (approved under the brand name EPKINLY in the U.S. and Japan, and TEPKINLY in the EU) has received regulatory approval in certain lymphoma indications in several territories. Epcoritamab is being co-developed by Genmab and AbbVie as part of the companies’ oncology collaboration. The companies share commercial responsibilities in the U.S. and Japan, with AbbVie responsible for further global commercialization. Both companies will pursue additional international regulatory approvals for the investigational R/R FL indication and additional approvals for the R/R DLBCL indication.

Genmab and AbbVie continue to evaluate the use of epcoritamab as a monotherapy, and in combination, across lines of therapy in a range of hematologic malignancies. This includes five ongoing Phase 3, open-label, randomized trials including a trial evaluating epcoritamab as a monotherapy in patients with R/R DLBCL compared to investigators choice chemotherapy (NCT04628494), a trial evaluating epcoritamab in combination with R-CHOP in adult patients with newly diagnosed DLBCL (NCT05578976), a trial evaluating epcoritamab in combination with rituximab and lenalidomide (R2) in patients with R/R FL (NCT05409066), a trial evaluating epcoritamab in combination with rituximab and lenalidomide (R2) compared to chemoimmunotherapy in patients with previously untreated FL (NCT06191744), and a trial evaluating epcoritamab in combination with lenalidomide compared to chemoimmunotherapy in patients with R/R DLBCL (NCT06508658). The safety and efficacy of epcoritamab has not been established for these investigational uses. Please visit www.clinicaltrials.gov for more information.

On May 22, 2025 Volastra Therapeutics, a clinical-stage cancer biotechnology company focused on chromosomal instability, reported preliminary safety and efficacy data from its ongoing, first-in-human Phase I/II trial of its novel, oral KIF18A inhibitor, VLS-1488 (Press release, Volastra Therapeutics, MAY 22, 2025, View Source [SID1234653320]). These results will be featured in an oral presentation at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting on Monday, June 2.

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"We are excited by these results from our VLS-1488 KIF18A inhibitor program, demonstrating a favorable safety profile at clinically active doses, with tumor shrinkage in patients with heavily pre-treated ovarian cancer" said Scott Drutman, M.D., Ph.D., Chief Medical Officer and Head of R&D of Volastra Therapeutics. "These initial data represent a major milestone for the field of chromosomal instability and provide a clear path for dose and patient population selection for the next phase of development."

As of the January 10, 2025 data cutoff, 52 patients with advanced solid tumors were enrolled to the dose escalation portion of the trial across dose levels ranging from 50 mg to 800 mg. Drug exposures exceeded preclinically-defined efficacious thresholds for anti-tumor activity. No dose limiting toxicities were observed and a maximum tolerated dose was not reached. Less than 45% of patients experienced treatment-related adverse events (TRAEs) of any grade and less than 16% of all patients experienced G3 TRAEs. No patients experienced >G3 TRAEs.

Of the 20 patients with advanced high grade serous ovarian cancer, the majority were platinum resistant and heavily pretreated with a median of 5 prior lines of therapy. At the time of data cutoff, 7 of the 17 response-evaluable patients demonstrated a reduction in tumor size, including 3 Partial Responses per RECIST, with 5 patients remaining on therapy.

"These early data show VLS-1488 to be very well tolerated, with promising initial efficacy in ovarian cancer." said Ecaterina Dumbrava, M.D., Assistant Professor of Investigational Cancer Therapeutics at The University of Texas MD Anderson Cancer Center and investigator on the trial. "The data that will be presented demonstrate the potential of KIF18A as a novel, relevant therapeutic target for hard-to-treat cancers and I look forward to continued development."

Volastra continues to enroll patients in this study and explore the broad potential of its two KIF18A inhibitors, VLS-1488 and sovilnesib in ovarian and other cancers.

Details of the ASCO (Free ASCO Whitepaper) Rapid Oral Abstract Session are as follows:
Title: Preliminary results from a first-in-human, phase I/II study of VLS-1488, an oral KIF18A inhibitor, in patients with advanced solid tumors
Presenter: Ecaterina Dumbrava, M.D., Assistant Professor of Investigational Cancer Therapeutics, The University of Texas MD Anderson Cancer Center
Session Title: Developmental Therapeutics—Molecularly Targeted Agents and Tumor Biology – Small Molecules
Session Date and Time: Monday, June 2, 2025, 8:00 AM- 9:30 AM CT
Location: McCormick Place Convention Center, S406
About VLS-1488

VLS-1488 is a novel, oral small molecule inhibitor of KIF18A, a kinesin protein essential for cancer cell division and a synthetic lethal target in chromosomally unstable cancers. VLS-1488 was granted Fast Track Designation from the FDA in October 2024 for the treatment of patients with platinum-resistant high-grade serous ovarian cancer.

About VLS-1488-2201 Trial (NCT05902988)

The Phase 1/2 trial is evaluating the safety, tolerability, PK, and antitumor activity of VLS-1488 in patients with advanced solid tumors, including high grade serious ovarian cancer. The study consists of two parts, Dose Escalation and Dose Expansion. Enrollment to the dose expansion portion of this study is ongoing.

About Ovarian Cancer and Chromosomal Instability

In the U.S. alone, there are more than 20,000 new cases of ovarian cancer each year, over 75% of which are advanced. Most of these patients will experience disease progression on platinum-based therapy.

High grade serous ovarian cancer accounts for approximately 75% of all ovarian cancers, about 80% of all deaths and is almost universally chromosomally unstable.