Antengene Presents Four Posters at AACR 2025 Highlighting Focuses on AnTenGagerTM TCEs and Synthetic Lethality

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.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

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.

Talus Bio Presents Preclinical Data on Transcription Factor Therapeutic Programs at the AACR Annual Meeting 2025

On April 25, 2025 Talus Bioscience, a technology-enabled therapeutics company, reported new preclinical data from programs targeting transcription factors in chordoma, non-small cell lung cancer, and advanced prostate cancer (Press release, Talus Bioscience, APR 25, 2025, View Source [SID1234652172]). The data, presented at the 2025 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting, held April 25-30, support Talus Bio’s first-in-class approach to target previously undruggable transcription factors using regulome-scale discovery in live human cells.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We built our platform to systematically address transcription factors, a target class long considered out of reach for small-molecule therapeutics," said Alex Federation, PhD, CEO and Co-Founder of Talus Bio. "The data we’re presenting at AACR (Free AACR Whitepaper) show that the approach is working. Our platform has already logged over 50 million protein-compound interactions this year. These results validate our strategy in two difficult oncology indications. Our Brachyury molecules are on track for candidate nomination, and new compounds targeting NONO and AR-V7 show real promise for metastatic castration-resistant prostate cancer. We now have a repeatable model for discovering tractable starting points across transcription factor targets once thought undruggable."

Talus Bio’s technology profiles the regulome, providing a quantitative readout of active transcription factors and other DNA-bound regulators in live, unmodified human cells. The platform can measure over 10,000 regulomes per month and is being deployed to discover and optimize small molecules that modulate transcription factor activity in their native context.

Data presented at AACR (Free AACR Whitepaper) on lead drug candidates highlight the strength of this approach. By combining high-throughput transcription factor profiling with AI-guided chemistry, Talus Bio is accelerating the discovery of modulators for transcription factor targets.

Presentation Highlights

Poster 4036 / 2: Selective inhibition of the transcription factor Brachyury in chordoma and non-small cell lung cancer

Preclinical data presented by Gaelle Mercenne at AACR (Free AACR Whitepaper) showcase the ability of a novel covalent small molecule, TAL61, to effectively modulate Brachyury activity in patient-derived xenograft (PDX) models of chordoma:

TAL61 demonstrated significant efficacy in reducing tumor burden in PDX chordoma models with durable tumor suppression post-treatment
TAL61 exhibited low toxicity and high tolerability with daily dosing
Additionally, TAL61 decreased tumor burden by more than 50% in in vivo models of Brachyury-positive non-small cell lung cancer (NSCLC)
Poster 6991 / 20: Targeting NONO as a therapeutic strategy for metastatic castration-resistant prostate cancer (CRPC)

Preclinical data presented by Brian McEllin support an emerging modality targeting NONO to disrupt both AR and ARv7 transcription as an emerging treatment for metastatic castration-resistant prostate cancer:

A targeted search of Talus Bio’s proprietary compound database identified covalent small molecules for lead optimization
Compound treatment reduced mRNA and protein levels of AR and ARv7 in prostate cancer cells
Active compounds show abrogate AR and ARv7 gene expression programs in castration-resistant prostate cancer models
Poster 4496 / 7: Small molecule inhibition of previously "undruggable" transcription factors with AI-guided functional proteomics

Data presented by Alex Federation at AACR (Free AACR Whitepaper) highlight the regulome sequencing platform as a drug discovery engine for previously intractable transcription factors:

The assay provides a quantitative, time-resolved readout of drug-induced changes in transcription factor activity, capturing protein:genome interactions for thousands of proteins simultaneously
This approach enables small molecule screening in physiologically relevant contexts, where transcription factors fold, assemble, and function as they do in human disease
AI-guided hit discovery using this technology has enabled discovery and optimization for inhibitors of Brachyury, AR-V7, and STAT3
Talus Bio’s foundational AI model has helped identify over 30 tractable compounds against validated transcription factor targets in cancer and other diseases. The company is actively pursuing co-development opportunities with partners to accelerate these discoveries into the clinic. Connect with the team to learn more about partnerships and collaborations.

CatalYm to Present New Data Demonstrating GDF-15 Blockade Enhances Efficacy of Antibody-Drug Conjugates at AACR 2025

On April 25, 2025 CatalYm reported the presentation of the first preclinical data demonstrating that the company’s clinical-stage antibody visugromab can improve the anti-tumor activity of antibody-drug conjugates (ADCs). The data will be presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting, taking place in Chicago, IL, April 25-30, 2025 (Press release, Catalym, APR 25, 2025, View Source [SID1234652140]). The poster will highlight the potential of targeting Growth Differentiation Factor 15 (GDF-15) to enhance the activity and overcome resistance to ADC therapies, which are increasingly used as frontline treatments in several solid tumor indications. The full abstract is available on the AACR (Free AACR Whitepaper) Annual Meeting website.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The study showed that several clinically validated ADCs, including topoisomerase I and microtubule inhibitor-based regimens, consistently triggered GDF-15 expression, suggesting a potential resistance mechanism to ADC therapy. Across in vitro and in vivo models of multiple solid tumor types, ADC treatment induced GDF-15 release from tumor cells, leading to elevated serum levels and reduced ADC efficacy. Combining ADC treatment with GDF-15 neutralization improved anti-tumor responses, resulting in reduced tumor growth and enhanced infiltration and activation of T cells and myeloid cells in the tumor microenvironment.

"We are building a broader understanding of GDF-15 as a key resistance mechanism activated by targeted approaches like ADCs as well as the leading immunotherapies like PD-1 inhibitors," said Christine Schuberth-Wagner, PhD, Chief Scientific Officer at CatalYm. "By blocking GDF-15, visugromab restores anti-tumor immune activity and enhances treatment efficacy, underscoring its potential as a versatile combination partner across a broad spectrum of cancer therapies beyond immunotherapies."

"ADCs continue to demonstrate potent and specific therapeutic impact, nevertheless resistance is a significant challenge for long-term patient benefit. In addition to our Phase 2 clinical development program, CatalYm will continue to investigate how our GDF-15 neutralizing antibody visugromab can create a novel path to address cancer resistance effectively," added Scott Clarke, Chief Executive Officer at CatalYm.

The preclinical findings build on clinical evidence from the GDFATHER-1/2a trial (GDF-15 Antibody-mediaTed Human Effector T Cell Relocation Phase 1/2a Trial; NCT04725474), that was recently published in Nature. The data show that visugromab induces deep and durable responses in patients with relapsed or refractory solid tumors who had progressed on prior anti-PD-1/PD-L1 therapies. In addition to restoring anti-tumor immune responses, visugromab also demonstrated the potential to mitigate cancer-associated cachexia. CatalYm is currently conducting a broad, global Phase 2b clinical development program for visugromab, evaluating its effectiveness in earlier treatment lines of non-squamous non-small-cell lung cancer and hepatocellular carcinoma, as well as neoadjuvant treatment in urothelial cancer in combination with standard of care.

Poster details

Session title: "PO.IM01.03 – Antibodies and Antibody-Drug Conjugates"

Abstract title: "GDF-15 neutralization enhances the therapeutic activity of antibody-drug conjugates"

Abstract number: 4777/13

Location: Section 36

Date and time: April 29, 2025, 9:00 AM – 12 PM CT/ 4:00 PM – 7:00 PM CEST

About Visugromab
Visugromab is a monoclonal antibody that neutralizes the tumor-derived Growth Differentiation Factor-15 (GDF-15), a locally acting immunosuppressant fostering immunotherapy resistance. Neutralizing GDF-15 with visugromab reverses key cancer resistance mechanisms to reinstate an efficient anti-tumor response by re-enabling immune cell activation, proliferation and induction of interferon-γ.

Innovent Announces NMPA Approval of Limertinib, a Third-generation EGFR TKI Collaborated with ASK Pharma, for the First-line Treatment of Lung Cancer

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.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

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.

Onchilles Pharma Presents New Data at AACR 2025 Highlighting N17350, a Next-Generation Cytotoxic Therapeutic Leveraging the ELANE Pathway

On April 25, 2025 Onchilles Pharma, a private biotech company pioneering next-generation cytotoxic therapeutics that harness the ELANE pathway, reported the presentation of new preclinical data for its lead drug candidate, N17350, at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2025 ("AACR") (Press release, Onchilles Pharma, APR 25, 2025, View Source [SID1234652173]). The presentation highlights N17350’s superior and selective tumor-killing ability compared to standard-of-care chemotherapies in ovarian cancer models, including chemotherapy-resistant tumors, derived from patients.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

This data builds on the growing body of evidence supporting N17350’s novel mechanism of action and its potential to become a next-generation cytotoxic therapeutic. Unlike traditional chemotherapies, which indiscriminately damage both cancer and healthy immune cells, N17350 leverages elevated levels of histone H1 isoforms found in cancer cells to selectively kill tumors while sparing healthy cells. The results show that N17350 maintains its efficacy across various tumor types and treatment histories.

"We are excited to share this compelling data at AACR (Free AACR Whitepaper), which further reinforces the potential of N17350 to address unmet needs across cancers, with the potential to make a significant impact in chemotherapy-resistant disease," said Lev Becker, Ph.D., Founder and Chief Scientific Officer of Onchilles Pharma. "These results demonstrate the strength of leveraging the ELANE pathway to therapeutically target tumor heterogeneity and overcome limitations of current cytotoxic therapies."

Highlights from the AACR (Free AACR Whitepaper) Presentation Include:

Broad Efficacy: N17350 was effective in killing cancer cells derived from treatment-naïve, neoadjuvant chemotherapy-treated (NACT), and recurrent ovarian tumors.
Selective Cytotoxicity: While standard chemotherapies were equally toxic to both cancer and immune cells, N17350 preserved immune cell viability while inducing immunogenic cell death in cancer cells.
In Vivo Superiority: In cell-derived xenograft (CDX) models from ovarian cancer patients, N17350 outperformed carboplatin in inducing tumor regression.
No Resistance: N17350 maintains potency following repeat dosing and effectively kills chemotherapy-resistant cancer cells.
"These new data exemplify the power of the ELANE pathway, and we believe therapeutics such as N17350 could represent a fundamentally new class of cancer treatments," said Court R. Turner, J.D., Co-Founder and Chief Executive Officer of Onchilles Pharma. "We remain focused on translating these compelling preclinical results into clinical benefit as we prepare to enter the clinic later this year."

N17350 is currently advancing toward first-in-human clinical trials, with an initial focus on skin, head and neck, triple-negative breast, and lung cancers. These new data support its continued development and highlight its promise as a differentiated cytotoxic modality with potential applications across a broad range of solid tumors.

The AACR (Free AACR Whitepaper) poster presentation, titled "N17350 outperforms cytotoxic agents in killing tumors of treatment-naïve and chemotherapy-treated ovarian carcinoma patients," will be available in Poster Section 21 (Poster Board Number: 30, Presentation Number: 861) from 2pm to 5pm CT on Monday, April 28th. The poster will also be available to meeting attendees via the AACR (Free AACR Whitepaper) virtual platform beginning at 1pm ET today.