On April 25, 2025 Rgenta Therapeutics, a clinical-stage biotechnology company pioneering the development of a new class of oral small molecules targeting RNA and RNA regulation for oncology and neurological disorders, reported that preclinical data from its lead program, RGT-61159 were presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) 2025 Annual Meeting, which is being held from April 25-30, 2025, in Chicago, IL (Press release, Rgenta Therapeutics, APR 25, 2025, View Source [SID1234652159]). The data demonstrate robust anti-tumor activity of RGT-61159 in several cell-derived xenograft (CDX) models of AML and synergistic benefit when combined with standard of care for AML highlighting the potential of RGT-61159 to treat a broad AML patient population.

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"The data presented at the AACR (Free AACR Whitepaper) meeting highlight the ability of RGT-61159 to reduce levels of both MYB RNA transcripts and correspondingly MYB protein in a dose dependent fashion, which translates to potent killing activity in cells that overexpress MYB," said Travis Wager, Ph.D. co-founder and chief scientific officer. "Importantly, we see robust antitumor activity with RGT-61159 across a range of AML models that carry genetic alterations that are common in patients with AML highlighting its potential to treat a broad AML patient population."

"Using RGT-61159, which specifically acts at the RNA level, we are able to address MYB which has been shown to function as an oncogenic driver in a variety of cancers including AML, colorectal cancer (CRC) and adenoid cystic carcinoma (ACC), and until now, has been a difficult target to drug," said Simon Xi, Ph.D., cofounder and chief executive officer of Rgenta. "Our ongoing Phase 1a/b clinical trial of RGT- 61159 in patients with relapsed or refractory ACC or CRC is advancing well and we look forward to broadening that program and initiating a new Phase 1/2 study of RGT-61159 in adults with AML/high risk myelodysplastic syndromes the second half of 2025."

In a poster titled RGT-61159, Best-in-class Oral Small Molecule Inhibitor of MYB via Selective RNA Splicing Alteration, Synergistic Anti-Tumor Activity When Combined with Standards of Care in Leukemia Disease Models Harboring AML Common Genetic Lesions, data were presented highlighting the close correlation between the elimination of MYB RNA and protein by RGT-61159 and its potent killing activity against leukemia and lymphoma cancer cell lines that over express MYB, demonstrating on-target therapeutic activity of this potent, orally available small molecule. RGT-61159 also inhibited AML cell proliferation and downregulated the expression of master oncogenes controlled by MYB, including MYC, BCL2, FLT3 and IDH1. Data from several CDX models of AML with genetic alterations seen commonly in AML, demonstrated the robust anti-tumor activity of tolerated doses of RGT-61159 and synergistic activity both in vitro and in vivo when administered in combination with agents used in AML standard of care.

About RGT-61159
RGT-61159 is an orally available small molecule designed to specifically modulate splicing of the transcription factor MYB resulting in the inhibition of the oncogenic MYB protein and potential cell death of the cancer cells that overexpress the MYB protein. MYB acts as a master regulator of cell proliferation, self-renewal, and differentiation processes and its aberrant expression has been demonstrated in multiple forms of human cancer including adenoid cystic carcinoma (ACC), acute myeloid leukemia (AML), T-cell acute lymphoblastic leukemia (T-ALL), colorectal cancer (CRC), small cell lung cancer (SCLC) and breast cancer. Rgenta is evaluating RGT-61159 in an ongoing multi-center, open-label Phase 1a/b clinical trial in patients with advanced relapsed or refractory ACC or CRC. The Phase 1a/b study is designed to evaluate safety, tolerability, pharmacokinetics, target engagement and clinical efficacy of RGT-61159 in patients with ACC or CRC. Additional information about the Phase 1a/b clinical trial can be accessed at ClinicalTrials.gov (NCT06462183).

On April 25, 2025 Akeso, Inc. (9926.HK) ("Akeso" or the "Company") reported that its globally first-in-class PD-1/VEGF bispecific antibody, ivonescimab, has received approval from the National Medical Products Administration (NMPA) for its supplementary New Drug Application (sNDA) for use as a monotherapy for the first-line treatment of PD-L1-positive (TPS ≥ 1%) non-small cell lung cancer (NSCLC) in patients who are negative for epidermal growth factor receptor (EGFR) gene mutations and anaplastic lymphoma kinase (ALK) gene mutations (Press release, Akeso Biopharma, APR 25, 2025, View Source [SID1234652158]). This indication marks Ivonescimab’s second major approval.

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The sNDA approval is based on the breakthrough results of the AK112-303/HARMONi-2 Phase III study, which is a randomized, double-blind, controlled study that directly compared ivonescimab with pembrolizumab in first line PD-L1 positive NSCLC.

Ivonescimab demonstrated a statistically significant improvement in the trial’s primary end point, median progression-free survival (PFS) when compared to pembrolizumab, with a median PFS of 11.14 months, achieving a hazard ratio (HR) of 0.51 reducing the risk of disease progression or death by 49%.
An interim analysis of OS conducted at 39% data maturity (α=0.0001), indicated a clinically meaningful improvement in OS with ivonescimab compared to pembrolizumab, with a hazard ratio (HR) of 0.777, reflecting a 22.3% reduction in the risk of death.
Ivonescimab is the first therapy to achieve statistically significant positive results in a Phase III trial compared with pembrolizumab in a head-to-head setting.
Ivonescimab represents a new, more effective, and safer "chemotherapy-free" option for the first-line treatment of NSCLC.
Ivonescimab is the first bispecific antibody approved globally with a dual "cancer immunotherapy + anti-angiogenesis" mechanism. It has already shown significant positive results in three Phase III trials for lung cancer, including 1) treatment for EGFR-TKI-resistant NSCLC, 2) comparison with pembrolizumab in PD-L1-positive NSCLC, and 3) treatment in combination with chemotherapy versus tislelizumab in combination with chemotherapy in squamous NSCLC.

Professor Zhou Caicun, principal investigator of the HARMONi-2 trial, director of the Department of Oncology at the Shanghai East Hospital, Tongji University, commented:

"The Phase III HARMONi-2 study demonstrated that ivonescimab offers significant improvements in progression-free survival (PFS) and overall survival (OS) compared to pembrolizumab. This breakthrough provides a safer and more effective first-line treatment option for NSCLC, particularly beneficial for patients who need better efficacy or quality of life, or those who are not suitable for chemotherapy due to its side effects.

Ivonescimab has also gained widespread recognition in treating EGFR-TKI-resistant NSCLC. The recent Phase III trial combining ivonescimab with chemotherapy, compared to tislelizumab for squamous NSCLC, showed promising positive results. Ivonescimab is now positioned as a new standard of care for both first- and second-line treatment in lung cancer. We are optimistic about its continued success in global Phase III trials and its potential to redefine cancer treatment standards worldwide."

Dr. Xia Yu, Founder, Chairwoman, President, and CEO of Akeso, commented:

"We are thrilled to announce the approval of ivonescimab as a first-line treatment for PD-L1-positive NSCLC, a major breakthrough in cancer immunotherapy. This milestone is a result of the dedication of investigators, participants, and patients, and we sincerely thank all of them. We also appreciate the regulatory authorities for their efficient and diligent review, which enables us to offer this new treatment to patients in China.

Ivonescimab has shown superior efficacy over pembrolizumab in the HARMONi-2 Phase III trial, becoming the first therapy to achieve this globally. Positive results so far from three pivotal Phase III lung cancer trials further establish ivonescimab as a next-gen IO treatment with strong global potential.

The clinical data highlights ivonescimab’s exceptional efficacy and safety benefits for patients across various cancer types. Beyond lung, ivonescimab has demonstrated promising potential as first-line treatment for other key cancers, including breast, head and neck, biliary tract, and colorectal cancer, supported by, over 12 ongoing Phase III trials. Akeso, along with our partner Summit, are committed to advancing ivonescimab as a cornerstone of global cancer immunotherapy, aiming to provide safer, more effective treatment options for patients worldwide."

On April 25, 2025 BillionToOne, a molecular diagnostics company with a mission to create powerful and accurate tests accessible to all, reported several abstract presentations that will be showcased at the American Association of Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting on April 25–30, 2025, in Chicago, IL (Press release, BillionToOne, APR 25, 2025, View Source [SID1234652157]).

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Each abstract highlights BillionToOne’s relentless focus in advancing the field of liquid biopsy technology:

Title: "Tumor fraction estimation and tissue copy number inference using copy number signal from a liquid biopsy assay"
Session Time: 4/29/25 at 9:00 AM – 12:00 PM
Location: Poster Section 20

Title: "Detection of a novel GNA11 processed pseudogene from cfDNA and implications for liquid biopsy"
Session Time: 4/29/25 at 2:00 – 5:00 PM
Location: Poster Section 31

Title: "Circulating cell-free methylated tumor DNA measurements correlate with plasma VAF-based tumor fraction estimates"
Session Time: 4/29/25 at 2:00 – 5:00 PM
Location: Poster Section 32

"These data presented at AACR (Free AACR Whitepaper) represent our dedication in advancing liquid biopsy testing with our proprietary single molecule precision technology and setting new standards in what can be achieved with plasma-based tests," said Dr. Gary Palmer, Chief Medical Officer, Oncology at BillionToOne. "Together, these innovations enhance the accuracy and robustness of ctDNA-based cancer profiling and monitoring, which will ultimately enable more precise treatment decisions for patients."

On April 25, 2025 Innovent Biologics, Inc. ("Innovent") (HKEX: 01801), a world-class biopharmaceutical company that develops, manufactures and commercializes high-quality medicines for the treatment of oncologic, autoimmune, cardiovascular and metabolic, ophthalmologic and other major diseases, reported that China’s National Medical Products Administration (NMPA) has approved the New Drug Application (NDA) for limertinib as the first-line treatment for adult patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) harboring EGFR exon 19 deletions or exon 21 L858R mutations (Press release, Innovent Biologics, APR 25, 2025, View Source [SID1234652156]). Innovent and ASK Pharm entered into a commercial collaboration agreement for limertinib in Mainland China in 2024.

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The approval of this new indication is supported by positive results from a randomized, double-blind, positive-controlled Phase 3 clinical trial. A total of 337 treatment-naïve patients with EGFR-sensitive mutation-positive locally advanced or metastatic NSCLC were enrolled and randomized 1:1 to receive either limertinib or gefitinib. The primary endpoint was progression-free survival (PFS), as assed by an independent review committee (IRC).

The data showed that limertinib significantly prolonged median PFS compared to gefitinib (20.7 months vs. 9.7 months), representing a 56% risk reduction in disease progression or death (hazard ratio [HR] 0.44; 95% CI: 0.34–0.58; p < 0.0001). In patients with central nervous system (CNS) lesions at baseline, median CNS PFS was also significantly longer with limertinib (20.7 months vs. 7.1 months), corresponding to a 72% risk reduction for CNS progression or death (HR 0.28; 95% CI: 0.10–0.82; p = 0.0136), underscoring its robust intracranial activity and clinical utility in such population with high-unmet-needs.

The safety profile of limertinib was consistent with that of known EGFR-targeted therapies. Adverse events were predominantly mild to moderate and well-tolerated, with no new safety signals identified during the clinical trial. Full data and analysis from this pivotal Phase 3 study will be published in academic journals.

"Limertinib has demonstrated exceptional efficacy and safety as first-line therapy in patients with EGFR-muted locally advanced or metastatic NSCLC, including notable efficacy in those with brain metastases. The approval of this first-line indication introduces a new treatment option for Chinese patients, addressing a critical clinical need in this population." said Professor Shi Yuankai, MD, Department of Medical Oncology at Chinese Academy of Medical Sciences and Principal Investigator of the Phase 3 Clinical Study.

Dr. Hui Zhou, Senior Vice President of Innovent, stated："We are delighted that both the first-line and second-line indications for limertinib have been successively approved. As a next-generation EGFR TKI, limertinib is poised to significantly improve survival outcomes for mutation-positive patients. Innovent has built a rich portfolio of precision therapies for lung cancer—including limertinib, Retsevmo(selpercatinib), Dupert(fulzerasib) and DOVBLERON (taletrectinib)—with ongoing efforts to enhance their synergistic value. We will continue to work closely with Ask Pharm to ensure that limertinib brings a new hope to the broad population of patients with EGFR-mutated NSCLC."

Mr. Jingfei Ma, CEO of ASK Pharm, stated: "Within a few months, limertinib has obtained approvals for both second-line and first-line indications. The approval of the first-line indication further expands its clinical applicability. Meanwhile, ASK Pharm is advancing a clinical trial of limertinib in combination with the cMET inhibitor ASKC202 for patients with NSCLC resistant to third-generation EGFR-TKIs. We look forward to working with our partner Innovent to accelerate the accessibility of limertinib for more patients in need."

About EGFR mutation-positive non-small-cell lung cancer (NSCLC)

Lung cancer remains one of the deadliest and most common cancers globally[1], with NSCLC accounting for approximately 85% of cases. Around 70% of NSCLC patients are diagnosed at locally advanced or metastatic stages that are not amenable to surgical resection. EGFR mutations are particularly prevalent among Asian NSCLC patients, affecting 30% to 50% of cases. EGFR-TKIs are the recommended standard of care in the first-line setting, with third-generation EGFR-TKIs offering the broadest treatment applicability.

About Limertinib

Limertinib is an orally-administrated, third-generation EGFR TKI with proprietary rights. It has been approved by the China’s NMPA for: 1) the treatment of adult patients with locally advanced or metastatic EGFR T790M-mutated non-small cell lung cancer (NSCLC), who have previously experienced disease progression during or after treatment with EGFR TKI; and 2) the first-line treatment of adult patients with locally advanced or metastatic NSCLC carrying EGFR exon 19 deletions or exon 21 L858R mutations.

In October 2024, Innovent and ASK Pharm entered into a strategic collaboration and license agreement for limertinib in Mainland China.

On April 25, 2025 Antengene Corporation Limited ("Antengene", SEHK: 6996.HK), a leading innovative, commercial-stage global biopharmaceutical company dedicated to discovering, developing and commercializing first-in-class and/or best-in-class medicines for hematologic malignancies and solid tumors, reported that it has released results from four preclinical studies in poster presentations at the 2025 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting (AACR 2025) (Press release, Antengene, APR 25, 2025, View Source [SID1234652155]). These four posters feature Antengene’s four highly differentiated and high-potential programs, including ATG-201 (CD19 x CD3 TCE) and ATG-042 (MTAPnull-selective PRMT5 Inhibitor), which are poised to enter clinical development in the second half of 2025; ATG-110 (LY6G6D x CD3 TCE), which was developed on the AnTenGagerTM TCE 2.0 platform for the treatment of microsatellite stable colorectal cancer; and a companion diagnostic antibody developed to assess CD24 expression and guide clinical studies of CD24-targeted therapies.

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Details of the Poster Presentations:
ATG-201 (CD19 x CD3 T cell engager)
Title: ATG-201, a novel T-cell engager (TCE) effectively depletes B cells with reduced risk of CRS for the treatment of B cell malignancies and B cell related autoimmune diseases
Abstract Number: 7326
Session Category: Immunology
Session Title: T Cell Engagers and Novel Antibody-Based Therapies
Date: April 30, 2025
Time: 9:00 AM – 12:00 PM (Central Time)
10:00 PM, April 30, 2025 – 1:00 AM, May 1, 2025 (Beijing Time)
Location: Poster Section 40
• Introduction: By depleting autoreactive B cells, CD19-targeted CAR-T have shown early yet promising efficacy in treating patients with B cell-driven autoimmune diseases. However, the clinical application of TCE continues to be greatly hindered by the unfavorable pharmacokinetics and toxicity associated with cytokine release syndrome. To overcome these limitations, Antengene developed ATG-201, a "2+1" CD19 x CD3 TCE, which was evaluated in a series of in vitro studies for binding affinity, T cell dependent cytotoxicity (TDCC) cytokine release, and drug developability. The in vivo anti-lymphoma efficacy and pharmacodynamic effect were evaluated in Raji xenograft model. The tissue resident B cell depletion was assessed in CD34+ hematopoietic stem cells humanized mice. Pharmacokinetic parameters of ATG-201 were evaluated in normal Balb/c mice.
• Results: ATG-201 demonstrated high affinity binding to CD19, limited T cell binding before CD19 crosslinking, highly potent CD19-dependent T cell cytotoxicity against CD19+ B cells, as well as enhanced naïve B cell depletion with reduced cytokine release compared to clinical benchmarks. In lymphoma models, the study observed potent in vivo efficacy with reduced cytokine release. In CD34+ hematopoietic stem cells humanized mice, ATG-201 was able to induce complete B cell depletion with reduced cytokine release. ATG-201 has a mAb-like pharmacokinetic profile in wild type mice and good drug developability. Moreover, surrogate CD19 x CD3 AnTenGager TCE displayed potent efficacy in MRL/lpr spontaneous systemic lupus erythematosus (SLE) mouse models and MOG-Induced EAE models.
• Conclusions: ATG-201 demonstrated CD19-dependent CD3 binding and activation, inducing effective B cell depletion in vitro and in vivo with low cytokine release, which provides potential for the treatment of B cell malignancies and B cell related autoimmune diseases. ATG-201 is poised to enter clinical development in the second half of 2025.

ATG-042 (MTAPnull-selective PRMT5 Inhibitor)
Title: Preclinical characterization of ATG-042, a novel MTAPnull-selective PRMT5 inhibitor
Abstract Number: 4230
Session Category: Experimental and Molecular Therapeutics
Session Title: HDAC and Methyltransferase Inhibitors
Date: April 29, 2025
Time: 9:00 AM – 12:00 PM (Central Time)
10:00 PM, April 29, 2025 – 1:00 AM, April 30, 2025 (Beijing Time)
Location: Poster Section 16
• Introduction: Targeting the PRMT5-MTA complex has become a promising strategy for treating MTAPnull cancer in a synthetically lethal manner, avoiding on-target-off-tumor hematological toxicity when using first-generation, non-selective PRMT5 inhibitors. Herein, Antengene developed ATG-042, a novel MTAPnull-selective PRMT5 small molecule inhibitor with good brain penetration. In this study, the in vitro activity and MTAP selectivity of ATG-042 were profiled using HCT116 MTAP wild type (wt) cells, HCT116 MTAP knock out (ko) cells and multiple endogenous MTAPnull cell lines. The in vivo efficacy was tested in cell derived xenograft (CDX) mouse models with HCT116 MTAP wt cells, HCT116 MTAP ko cells, LU99 cells (MTAPnull) and U87MG-luc (MTAPnull). The pharmacokinetic and toxicological properties were assessed with corresponding assay methods.
• Results: ATG-042 showed excellent anti-proliferative activities on multiple endogenous MTAPnull cell lines with IC50 values between 10nM and 100nM. ATG-042 demonstrated high permeability, good metabolic stability, and low risk of drug-drug interaction. In vivo PK study shows that ATG-042 is well absorbed, with a dose-dependent increase in plasma distribution and high oral bioavailability in mice, SD rats and beagle dogs. Furthermore, ATG-042 is brain-penetrable (B/P ratio=51% in mice; KPuubrain=0.73 in rats). ATG-042 showed robust in vivo efficacy in both subcutaneous CDX models (HCT116 -MTAP ko, LU99) and orthotopic CDX model (U87MG-luc) as a single agent. In addition, ATG-042 also exhibited potential synergy in combination with other drugs for antitumor therapy.
• Conclusions: ATG-042 is an oral MTAPnull-selective PRMT5 inhibitor with potent efficacy against MTAPnull tumor. It also demonstrated good tolerability and brain penetrability. ATG-042 is poised to enter clinical development in the second half of 2025.

ATG-110 (LY6G6D x CD3 T cell engager)
Title: ATG-110, a novel "2+1" LY6G6D-targeted T-cell Engager (TCE) with high potency for the treatment of MSS colorectal cancer
Abstract Number: 3509
Session Category: Immunology
Session Title: T Cell Engagers
Date: April 28, 2025
Time: 2:00 PM – 5:00 PM (Central Time)
3:00 AM – 6:00 AM, April 29, 2025 (Beijing Time)
Location: Poster Section 38
• Introduction: Colorectal cancer (CRC) is one of the most common cancers worldwide and requires more effective and safer therapies to improve the poor survival outcome, particularly in patients with microsatellite stable (MSS) colorectal cancer, who exhibit primary resistance to immune checkpoint inhibitors and lack effective treatment options. T cell engagers have shown encouraging efficacy in treating hematological malignancies, while exhibiting suboptimal clinical efficacies in solid tumors. The risk of cytokine release syndrome (CRS) remains as a significant challenge clinically. ATG-110 is a novel "2+1" LY6G6D x CD3 TCE developed by Antengene. In this study, ATG-110 was evaluated in a series of preclinical in vitro studies for binding affinity, T cell activation and cytokine release, T cell dependent cytotoxicity (TDCC), and drug developability. The in vivo anti-tumor efficacy was evaluated in PBMC-humanized immunodeficient NDG mice engrafted with LY6G6Dmedium-expression HT55 or LY6G6Dvery low-expression SW480 MSS CRC cells.
• Results: ATG-110 binds to LY6G6D-positive cell lines, including LY6G6D-overexpression 293T and HT55 with the nanomolar grade EC50. The CD3 binding site of ATG-110 is concealed by the LY6G6D Fab arm before binding to LY6G6D, due to the steric hindrance. Therefore, ATG-110 demonstrated limited binding capability to CD3+ cells before LY6G6D crosslinking. It activates T cells and induces cytokine release only in the presence of LY6G6D+ cells. In vitro, ATG-110 resulted in potent T cell dependent cytotoxicity with single-digit pM IC50 values on HT55 cells. ATG-110 also showed highly potent in vitro efficacy against LY6G6Dlow-expression cells. ATG-110 exhibited a low risk of inducing cytokine release syndrome. ATG-110 demonstrated potent anti-tumor activity in PBMC-humanized HT55 xenograft model. Furthermore, ATG-110 also demonstrated good drug developability.
• Conclusions: ATG-110 demonstrated LY6G6D-dependent CD3 binding and activation with low risk of CRS. It showed powerful in vitro and in vivo anti-tumor efficacy against colorectal cancer, which warrants further clinical evaluation.

ATG-1144 (CD24 CDx Antibody)
Title: Development of a diagnostic antibody for CD24 targeted therapy
Abstract Number: 671
Session Category: Clinical Research
Session Title: Diagnostic Biomarkers 2
Date: April 27, 2025
Time: 2:00 PM – 5:00 PM (Central Time)
3:00 AM – 6:00 AM, April 28, 2025 (Beijing Time)
Location: Poster Section 29
• Introduction: CD24 has emerged as a promising therapeutic target for anti-cancer treatment. Several clinical trials are being conducted to evaluate the safety and efficacy of CD24-targeted therapies. Here, Antengene developed and characterized an anti-CD24 diagnostic antibody to enhance the screening and selection of patients based on CD24 expression. In this study, the authors described the discovery of the diagnostic antibody, and the evaluation of accuracy, sensitivity (selectivity), specificity, and assay precision of the antibody.
• Results: Monoclonal antibody clone ATG-1144 binds to the hCD24 core peptide in ELISA with an EC50 of 0.06 nM. Distinct membrane staining on human normal esophageal tissue FFPE specimens can also be observed with IHC staining using ATG-1144. For accuracy assessment, six CDX and twenty human specimens, comprising both positive and negative specimens (including solid tumors and B-cell non-Hodgkin lymphomas), were validated. Samples exhibiting high, medium, and low CD24 expression levels were evaluated for sensitivity and specificity, and the interpreted results aligned with the reference outcomes. FFPE tissues from three distinct patients were evaluated for assay precision assessment. The TMA IHC staining result revealed that 50-80% of patients with lung, breast, bladder, ovarian, or liver cancer have CD24 expression on tumor cell surface with low expression in the para-cancerous normal tissue.
• Conclusions: ATG-1144 specifically binds to human CD24 with high sensitivity as demonstrated by IHC staining. The development and validation of the method have been finalized using Leica Bond III platforms. These data suggest a potential diagnostic use of ATG-1144 for identifying CD24+ patients.