Eikon Therapeutics Presents Data on Clinical-Stage Programs at the 2026 Annual Meeting of the American Society of Clinical Oncology

On May 31, 2026 Eikon Therapeutics, Inc. (Nasdaq: EIKN) (Eikon), a late-stage clinical biopharmaceutical company dedicated to developing innovative medicines to address serious unmet medical needs, reported presentations on several of its lead programs at the 2026 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in Chicago, including updated data for its EIK1001 trial in non-small cell lung cancer (NSCLC) and its highly selective PARP1 inhibitor EIK1003.

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"We are pleased to present six abstracts at ASCO (Free ASCO Whitepaper) reflecting both the progress of our pipeline and the growing body of evidence supporting our lead programs," said Roy Baynes, M.D., Ph.D., Chief Medical Officer of Eikon. "In our most advanced program, EIK1001 demonstrated encouraging response rates and durability in combination with standard of care in first-line NSCLC. We also observed meaningful clinical activity of EIK1003 both as a monotherapy and, potentially a first for the field, in combination with paclitaxel, including responses in heavily pretreated patients. Together, these data continue to reinforce the scientific rationale behind our programs and their potential to deliver meaningful benefit to people living with cancer."

EIK1001 Clinical Update.

Updated data from our ongoing Phase 2 trial evaluating the safety and tolerability of EIK1001 in combination with both pembrolizumab and histology appropriate chemotherapy for the front-line treatment of patients with advanced NSCLC, also known as our TeLuRide-005 trial, provide evidence of a potentially durable effect of EIK1001 in combination with standard of care, and a preliminary tolerability profile supportive of systemic administration in an out-patient setting, a potential key differentiator of EIK1001 from previous TLR7/8 targeted therapies.

EIK1001 is an investigational, systemically administered dual-agonist of Toll-like receptors 7 and 8 designed to stimulate both innate and adaptive immune responses. In Phase 1 trials of EIK1001, single-agent activity was observed in patients with advanced malignancy. This mechanism may complement the antitumor immune response engendered by PD-(L)1 blockade.

TeLuRide-005 is our multicenter, open-label trial of once-weekly (QW) systemically administered EIK1001 in combination with standard of care, once every third week (Q3W) pembrolizumab and histology appropriate chemotherapy in first-line, treatment-naïve patients with stage 4 NSCLC. Enrollment completed in the first quarter of 2026.

As of enrollment cutoff, 72 participants with previously untreated stage 4 NSCLC received intravenous EIK1001 QW combined with standard of care pembrolizumab plus chemotherapy Q3W. After 8 cycles, EIK1001 administration was reduced to Q3W. The maximum treatment duration is 2 years (up to 35 cycles) for EIK1001 in combination with pembrolizumab, with pemetrexed continued at the discretion of the Investigator for non-squamous patients.

At the March 17, 2026 safety data cutoff, among the safety evaluable population (n=72: 39 non-squamous; 33 squamous), the combination of EIK1001 with pembrolizumab and chemotherapy was generally well tolerated, with an adverse event (AE) profile similar to standard of care alone. Most treatment-emergent adverse events (TEAEs) were Grade 1-2, and the most common Grade 3 or higher treatment-related adverse events (TRAEs) were neutropenia (30.6%), anemia (9.7%), and thrombocytopenia (9.7%). All cytokine release syndrome (CRS) AEs were Grade 1 and 2, and all CRS events occurred before Cycle 4 in all but one patient. This profile was sufficient to support administration in an out-patient setting during the ongoing trial.

Among the efficacy-evaluable population (n=65: 36 non-squamous; 29 squamous), EIK1001 treatment in combination with pembrolizumab and chemotherapy resulted in a 63.1% objective response rate (ORR) and 90.8% disease control rate (DCR) at the efficacy data cutoff of May 4, 2026. Among participants in the non-squamous cohort, an ORR of 55.6% and a DCR of 83.3% were observed, respectively, with a median follow-up of 13.6 months. The median duration of response (DOR) in the non-squamous cohort was greater than 11 months at the efficacy data cutoff. Among participants in the squamous cohort, for which the data set was still maturing at the data cutoff due to slower enrollment, an ORR of 72.4% and a DCR of 100% were observed, respectively, with a median follow-up of 8.8 months.

EIK1003 Clinical Update.

Updated data from EIK1003-001, our Phase 1/2 trial evaluating the safety and efficacy of EIK1003 as monotherapy or in combination with anti-cancer agents in participants with advanced solid tumors, demonstrate that EIK1003 monotherapy (Cohort 1A) was generally well-tolerated across multiple dose levels. In Cohort 1C, signals of antitumor activity were observed with EIK1003 in combination with weekly paclitaxel, with a combination safety profile consistent with paclitaxel’s known toxicities. These data support what appears to be a unique profile for EIK1003 in combination strategies within the evolving PARP inhibitor landscape.

EIK1003 is an investigational, highly-selective PARP1 inhibitor designed to leave PARP2 signaling intact. PARP2 inhibition may be a key driver of the hematological toxicity associated with first generation, non-selective PARP inhibitors.

Cohort 1A: Updated EIK1003 Monotherapy Data

As of the enrollment cutoff, 65 patients with breast, ovarian, prostate, or pancreatic cancer have been treated in Cohort 1A with EIK1003 monotherapy at doses ranging from 10mg to 160mg once daily (QD), using a Bayesian optimal interval dose-escalation design to assess for safety, tolerability, pharmacokinetics and antitumor activity. This represents an expansion of the dataset previously presented at ASCO (Free ASCO Whitepaper) 2025, with additional patients enrolled and longer follow-up.

At the February 27, 2026 safety data cutoff, the updated safety profile in Cohort 1A remained generally consistent with data previously presented at ASCO (Free ASCO Whitepaper) 2025. Treatment-emergent adverse events (TEAEs) were reported in 63 of 65 patients (96.9%). Grade 3 or higher TEAEs occurred in 29 patients (44.6%); the most common were anemia (9.2%), neutropenia (7.7%), and ascites (7.7%). 4 of the 6 patients who developed Grade 3 or higher anemia had Grade 1-2 anemia at study entry. TEAEs led to dose reductions in 7 patients (10.8%) and to treatment discontinuation in 6 patients (9.2%). No TRAEs leading to death were reported.

Among efficacy-evaluable patients in Cohort 1A (n=49), ORR was 14.3% overall and 26.7% in PARP-naïve patients. Objective responses by tumor type were 14.8% (4/27) in ovarian cancer, 12.5% (2/16) in breast cancer, and one patient with prostate cancer. Median duration of response among confirmed responders (n=5) was 7.8 months at the efficacy data cutoff of May 4, 2026.

Cohort 1C: Initial Data on EIK1003 in Combination with Weekly Paclitaxel

As of the February 27, 2026 safety data cutoff, 60 patients with platinum-resistant ovarian cancer or HER2-negative breast cancer that failed chemotherapy or hormonal therapy have been treated in Cohort 1C with EIK1003 at doses ranging from 10mg to 60mg QD in combination with paclitaxel 80 mg/m2 IV QW. Dose-limiting toxicities of febrile neutropenia and tachycardia were reported in one patient each at the highest dose level tested, 60mg. TEAEs were reported in 60 out of 60 patients (100%). Grade 3 or higher TEAEs occurred in 45 patients (75%); the most common were neutropenia (50%) and anemia (13.3%). Neutropenia is a known and expected adverse event associated with weekly paclitaxel chemotherapy. All 8 patients who developed Grade 3 or higher anemia had Grade 1-2 anemia at study entry. TEAEs led to dose reductions in 15 patients (25%) and discontinuation of one or both study drugs in 11 patients (18.3%). No TRAEs leading to death were reported.

Among efficacy-evaluable patients in Cohort 1C (n=53), ORR was 24.5%; 12 of 13 responders (92%) had received prior taxane therapy. Objective responses by tumor type were 29.6% (8/27) in platinum-resistant ovarian cancer and 19.2% (5/26) in breast cancer. Duration of response among confirmed responders (n=9) ranged from 1.5 to 11.4 months, with responses ongoing in 3 responders at the efficacy data cutoff of May 4, 2026.

2026 ASCO (Free ASCO Whitepaper) Abstract Titles.

EIK1001

Title: Efficacy, safety and cytokine profiling with addition of the toll-like receptor (TLR) 7/8 dual agonist EIK1001 to Standard of Care First-Line Therapy: the Phase 2 TeLuRide-005 trial in Stage 4 Non-Small Cell Lung Cancer

Title: Adaptive Phase 2/3 Study of EIK1001, a TLR7/8 Dual Agonist, in Combination with Pembrolizumab, as First-Line Therapy in Participants with Advanced Melanoma (TeLuRide-006)

Title: A Phase 2/3 Study of EIK1001 in Combination with Pembrolizumab and Chemotherapy in Participants with Stage 4 Non-Small Cell Lung Cancer (TeLuRide-008)

EIK1003

Title: EIK1003, a PARP1-selective inhibitor, in combination with paclitaxel (PTX): Initial combination and updated monotherapy results from a Phase 1/2 study EIK1003-001 in advanced solid tumors

EIK1005

Title: First-in-Human Study to Evaluate the Safety, Tolerability, and PK of EIK1005, a Novel WRN Inhibitor in Healthy Participants

Title: Phase 1/2 Study of the novel Werner helicase inhibitor EIK1005 as Monotherapy and in Combination with Pembrolizumab in Patients with Advanced Solid Tumors, including MSI-H or dMMR Tumors (Publication only)

Copies of the 2026 ASCO (Free ASCO Whitepaper) presentations will be made available on our website: www.eikontx.com under Scientific Papers & Publications.

(Press release, Eikon Therapeutics, MAY 31, 2026, View Source [SID1234666287])

NeuExcell Therapeutics Announces Encouraging Data of NXL-004 in Recurrent Malignant Glioma at 2026 ASCO

On May 31, 2026 NeuExcell Therapeutics reported that Dr. Yulun Huang will present the first-in-human clinical study of NXL-004 for recurrent malignant glioma as Rapid Oral presentation at the 2026 Annual Meeting of the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) (ASCO 2026). ASCO (Free ASCO Whitepaper) is one of the leading global oncology conferences. With over 8,500 submissions, abstracts selected for oral presentation represents studies of significant scientific and clinical interests.

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"We are honored that our NXL-004 program has been selected for Rapid Oral Presentation at ASCO (Free ASCO Whitepaper) 2026," said Dr. Gong Chen, Founder and Chief Scientific Officer of NeuExcell. "This recognition underscores the potential of our in vivo cell conversion platform to address significant unmet needs in recurrent malignant glioma and potentially other devastating neurological diseases."

The study was conducted in collaboration with Dr. Yulun Huang and his team at the Department of Neurosurgery at the Fourth Affiliated Hospital of Soochow University.

Presentation Details

Title:
First-in-human study of AAV6-NeuroD1 trans-differentiation therapy in recurrent malignant glioma

Presenter:
Dr. Yulun Huang

Session:
Rapid Oral Abstract Session – Central Nervous System Tumors

Date & Time:
May 31, 2026 | 4:30 PM–6:00 PM CT

Abstract Number:
2012

Favorable Safety and Encouraging Efficacy

Recurrent malignant glioma remains one of the most difficult cancers to treat, with median overall survival (OS) at approximately 6-9 months. After recurrence, there is currently no established standard of care, and no therapy has demonstrated a clear survival benefit in randomized controlled studies.

NXL-004 is a first-in-class in vivo cell conversion therapy using an adeno-associated virus (AAV) vector to deliver neural transcription factor NeuroD1. The therapy is designed to reprogram proliferating glioma cells toward non-proliferating neuron-like cells or induce tumor cell apoptosis.

Results from the first-in-human study in recurrent malignant glioma showed a favorable safety profile and encouraging efficacy:

Baseline and Treatment
Eleven patients were enrolled, with a median age of 48.0 years; 81.8% male.
All patients had recurrent WHO grade 4 astrocytoma, including 10/11 with IDH-wildtype tumors and 6/11 with MGMT-unmethylated tumors.
Ten patients received repeat tumor resection plus intracavitary injection of NXL-004, and one received biopsy plus intra-tumoral injection.
Safety:
No drug-related serious adverse events (SAEs) were reported.
All AEs related to NXL-004 were grade 1–2. The most common treatment-related AEs included fever and headache.
Efficacy:
Median overall survival (mOS) of all treated recurrent glioma patients was over 13.2 months, comparing favorably with historical benchmarks of 6–9 months.
The observed one-year survival rate reached 77.9%.
All five patients enrolled between February and June 2025 were in high-dose group and remained alive at the time of this report:
Among these five patients, four already achieved OS exceeding 12 months.
One complete response (CR) and one durable stable disease (SD) were observed in the high-dose group, with ongoing progression-free survival (PFS) of 13.1 and 11.0 months.
"This study represents the first clinical evaluation of an AAV-NeuroD1 based gene therapy for glioma," said Dr. Yulun Huang, the principal investigator of the study. "The findings provide encouraging evidence supporting both the safety and therapeutic potential of this novel approach."

About NXL-004

NXL-004 is NeuExcell’s investigational gene therapy candidate based on its proprietary in vivo cell conversion platform. The therapy utilizes AAV-mediated delivery of NeuroD1 to reprogram tumor cells directly within the brain microenvironment.

(Press release, NeuExcell Therapeutics, MAY 31, 2026, View Source [SID1234666286])

Precede Biosciences Presents New ASCO 2026 Data Demonstrating Ability of Precede Bio Insight™ to Decode MET-Driven Biology and Resistance in Advanced NSCLC

On May 31, 2026 Precede Biosciences, a company powering next-generation precision medicine through its functional liquid biopsy platform, reported two new data presentations at the 2026 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, being held May 29 to June 2 in Chicago, Illinois.

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The data highlight how functional information beyond genomics in patients with advanced non-small cell lung cancer (NSCLC), including target expression, pathway activity, and cell-state changes associated with response and resistance to targeted therapies, can be revealed from only 1 mL of plasma using Precede Bio Insight.

"Patients with advanced NSCLC in whom MET is an important disease driver often require multiple lines of therapy, underscoring the need to better understand the biology driving response and resistance," said J. Carl Barrett, PhD, Chief Scientific Officer of Precede Biosciences. "For drug developers working to advance MET-targeted therapies, these data demonstrate the ability of our​ platform to provide a broad functional view of tumor biology from plasma that can help guide patient selection and rational therapeutic development strategies."

ASCO 2026 Presentations

Comprehensive Epigenomic Profiling of Plasma for Non-invasive Detection of MET Activation Uncovers MET-associated Biology in Patients with EGFR-mutated Advanced NSCLC and Progression on Osimertinib (Abstract 8642)
Plasma Epigenomic Profiling Identifies Mechanisms of Sensitivity and Resistance to Tepotinib in METex14 Skipping Metastatic NSCLC (Abstract 8637)

(Press release, Precede Biosciences, MAY 31, 2026, View Source [SID1234666285])

Ascentage Pharma Presents Updated Clinical Data for Olverembatinib as Second-Line Therapy in CML-CP at ASCO 2026

On May 31, 2026 Ascentage Pharma Group International (NASDAQ: AAPG; HKEX: 6855), a global, commercial-stage, integrated biopharmaceutical company engaged in the discovery, development and commercialization of novel, differentiated therapies to address unmet medical needs in cancer, reported updated efficacy and safety data from a clinical study of its first approved product, olverembatinib (HQP1351), as a second-line therapy in patients with chronic-phase chronic myeloid leukemia (CML-CP) were presented in a rapid oral presentation at the 2026 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting.

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The ASCO (Free ASCO Whitepaper) Annual Meeting is the world’s most prominent scientific gathering in the clinical oncology community, showcasing cutting-edge research in clinical oncology and advanced cancer therapies. This year marks Ascentage Pharma’s ninth consecutive appearance at the ASCO (Free ASCO Whitepaper) Annual Meeting. A total of six studies involving three of the Company’s key assets were selected for presentation, including three rapid oral presentations.

Data presented in this rapid oral presentation showed that, at cycle 24, patients with CML-CP treated with olverembatinib achieved a complete cytogenetic response (CCyR) rate of 91.3% and a major molecular response (MMR) rate of 60.9%. Responses deepened over time with longer treatment duration. Olverembatinib demonstrated a stable and manageable safety profile during long-term treatment, with no new safety signals identified. This longer follow-up study has generated more mature and encouraging results, further supporting the efficacy and safety of olverembatinib in patients with CML without the T315I mutation and reinforcing its potential role as a second-line treatment option for patients with CML-CP that failed first-line TKI therapy.

Olverembatinib is a novel drug developed by Ascentage Pharma and represents the first third-generation BCR-ABL1 inhibitor approved in China. Olverembatinib is currently being jointly commercialized in China by Ascentage Pharma and Innovent Biologics. The drug is currently approved in China for adult patients with tyrosine kinase inhibitor (TKI)-resistant CML-CP or accelerated-phase CML (CML-AP) harboring the T315I mutation; and adult patients with CML-CP resistant to and/or intolerant of first- and second-generation TKIs, with all approved indications now covered by the China National Reimbursement Drug List (NRDL). Ascentage Pharma is currently conducting three global registrational Phase III studies to evaluate olverembatinib in multiple indications, including CML-CP, newly diagnosed Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL), and succinate dehydrogenase (SDH)-deficient gastrointestinal stromal tumors (GIST), with two of these studies having been cleared by the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA). Ascentage Pharma has signed an exclusive option agreement to enter into an exclusive license agreement with Takeda for olverembatinib. In the event that Takeda exercises the option, Takeda would license the global rights to develop and commercialize olverembatinib in all territories outside of, among others, mainland China, Hong Kong, Macau, and Taiwan, China.

Professor Weiming Li, the Principal Investigator of this study from Union Hospital of Tongji Medical College, Huazhong University of Science and Technology, stated: "Overall, with longer treatment duration, olverembatinib not only helped patients achieve deeper responses, but also continued to provide durable clinical benefit while maintaining a favorable safety and tolerability profile, resulting in good patient adherence. These findings further support its role as a potential second-line treatment option for patients with CP-CML without the T315I mutation and provide stronger evidence for clinical practice. We also look forward to additional long-term follow-up data to further validate its efficacy and safety, and to provide stronger evidence-based support for the standardized use and guideline recommendations of olverembatinib in the second-line treatment setting, helping to deliver more optimized treatment options for patients and clinicians."

Yifan Zhai, MD, Chief Medical Officer of Ascentage Pharma, said: "The updated data presented at ASCO (Free ASCO Whitepaper) once again demonstrated the consistent performance of olverembatinib in the second-line treatment of CML, further strengthening our confidence in advancing this therapy into earlier lines of treatment. We look forward to continuously accumulating evidence from second-line and earlier-line settings to further optimize the treatment pathway for patients with CML and help to deliver greater clinical benefit, longer survival, and improved quality of life. Moving forward, we will continue to uphold our mission of addressing unmet clinical needs for patients around the world by accelerating clinical development and bringing more safe and effective therapies to patients as soon as possible."

Key highlights from the study presented at the 2026 ASCO (Free ASCO Whitepaper) Annual Meeting are as follows:
Updated efficacy and safety of Olverembatinib (HQP1351) as second-line therapy in patients with chronic-phase chronic myeloid leukemia (CP-CML)
Abstract #: 6510
Format: Rapid oral presentation
Session Title: Hematologic Malignancies—Leukemia, Myelodysplastic Syndromes, and Allotransplant
First Author: Weiming Li, MD, Department of Hematology, Union Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, China
Key Highlights:

Research Background: This is a single-arm, multicenter, open-label study conducted in China, evaluating the efficacy and safety of olverembatinib as a second-line treatment.
Efficacy Data: Among 42 evaluable patients, olverembatinib demonstrated significant and progressively deepening anti-tumor activity, with a best CCyR rate of 76.2% and a best MMR rate of 47.6% at study cutoff. Responses continued to improve with longer treatment duration, reaching 91.3% and 60.9%, respectively, at cycle 24. High response rates were also observed in patients previously treated with second-generation TKIs.
Safety Data: In terms of safety, the overall incidence of treatment-related adverse events was 89.4%, most of which were manageable low-grade events, primarily including but not limited to skin hyperpigmentation, hyperuricemia, and increased creatine phosphokinase. Although some grade ≥3 hematologic toxicities were observed, they were all recoverable through supportive treatment.
Conclusion: Overall, olverembatinib demonstrated durable and progressively deepening efficacy while maintaining a manageable safety profile, highlighting its promising clinical potential.
* Olverembatinib is currently under investigation and has not yet been approved by the US FDA.

(Press release, Ascentage Pharma, MAY 31, 2026, View Source [SID1234666284])

Ascentage Pharma Presents Its First Dataset on MDM2-p53 Inhibitor Alrizomadlin (APG-115) in Pediatric Solid Tumors at ASCO 2026

On May 31, 2026 Ascentage Pharma Group International (NASDAQ: AAPG; HKEX: 6855), a global, commercial-stage, integrated biopharmaceutical company engaged in the discovery, development and commercialization of novel therapies to address unmet medical needs in cancer, reported that the Company presented its first dataset of alrizomadlin (APG-115), an MDM2-p53 inhibitor from the Company’s apoptosis-targeted pipeline, as monotherapy or in combination with lisaftoclax (APG-2575) in pediatric patients with relapsed/metastatic rhabdomyosarcoma (RMS) or other soft-tissue sarcomas (STSs), in a rapid oral presentation at the 62nd American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting.

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The ASCO (Free ASCO Whitepaper) Annual Meeting showcases cutting-edge research in clinical oncology and advanced cancer therapies and is the world’s largest gathering of the clinical oncology community. This year marks Ascentage Pharma’s ninth consecutive appearance at ASCO (Free ASCO Whitepaper). A total of six studies involving three of the Company’s key assets were selected for presentation, including three rapid oral presentations.

The data presented demonstrated preliminary antitumor activity and a manageable tolerability profile of alrizomadlin in pediatric solid tumors. Results showed that alrizomadlin monotherapy demonstrated initial clinical benefit in pediatric rhabdomyosarcoma (RMS), with one pediatric patient achieving a complete response (CR). In combination with investigational selective Bcl-2 inhibitor lisaftoclax, encouraging antitumor activity was observed, with an objective response rate (ORR) of 23.5% among 17 response-evaluable patients, including one complete response in a patient with Ewing sarcoma and three partial responses (PRs). In terms of safety, alrizomadlin, either as monotherapy or in combination with lisaftoclax, demonstrated a manageable safety profile in pediatric patients with solid tumors.

Alrizomadlin is an orally administered, highly selective MDM2-p53 inhibitor independently developed by Ascentage Pharma. It is the first investigational agent of its class to enter clinical development in China and has global first-in-class potential. By blocking the MDM2-p53 protein-protein interaction, alrizomadlin restores the tumor suppressor activity of p53 and induces apoptosis in tumor cells. Recently, alrizomadlin was officially included by the Center for Drug Evaluation (CDE) of China’s National Medical Products Administration (NMPA) in the Pilot Program for the Support of Anti-tumor Drugs R&D for Kids, also known as the "SPARK Plan," for development in pediatric solid tumors including neuroblastoma, rhabdomyosarcoma, and Ewing sarcoma.

Professor Yizhuo Zhang, principal investigator of the study from the Department of Pediatric Oncology at Sun Yat-sen University Cancer Center, said: "Relapsed/refractory pediatric sarcomas are associated with extremely poor prognosis and substantial unmet medical needs. The data presented at the ASCO (Free ASCO Whitepaper) meeting demonstrated a favorable tolerability profile and promising anti-tumor effect for alrizomadlin both as monotherapy and in combination with lisaftoclax, with the complete response (CR) cases being particularly encouraging. As a key candidate included in the SPARK Plan, alrizomadlin has the potential to become a first-in-class therapy, address unmet medical needs, and bring new hope for long-term survival to pediatric patients."

Professor Yi Zhang, investigator of the study from the Department of Pediatrics at Beijing Tongren Hospital, Capital Medical University, said: "Treatment options for pediatric solid tumors, especially advanced soft-tissue sarcomas, remain very limited. The clinical data generated by the alrizomadlin combination regimen are therefore particularly meaningful. This apoptosis pathway-targeting therapy demonstrated favorable tolerability and encouraging objective response rates, further supporting the therapeutic potential of dual-target combination approaches in refractory pediatric tumors and providing valuable direction for future precision drug development in pediatric oncology."

Yifan Zhai, MD, Chief Medical Officer of Ascentage Pharma, said: "Pediatric solid tumors continue to represent an area of significant unmet medical need. The data presented at ASCO (Free ASCO Whitepaper) mark our first presentation of alrizomadlin clinical data in pediatric solid tumor patients and demonstrated encouraging preliminary clinical benefit and tolerability. Importantly, alrizomadlin has already been included by the CDE in the SPARK Plan for potential development in multiple pediatric solid tumors. The data presented provide initial clinical evidence supporting this development strategy. We will continue to advance the related clinical studies with the goal of bringing new treatment options to pediatric patients in urgent need."

Key highlights from the study presented at the 2026 ASCO (Free ASCO Whitepaper) Annual Meeting are as follows:

Alrizomadlin (APG-115) alone or in combination with Lisaftoclax (APG-2575) for the treatment of pediatric patients with relapsed/metastatic rhabdomyosarcoma (RMS) or other soft-tissue sarcomas (STSs)
Abstract #: 10012
Presentation Type: Rapid Oral Presentation
Session Title: Pediatric Oncology II
First Author: Yizhuo Zhang, MD, Department of Pediatric Oncology, Sun Yat-sen University Cancer Center, State Key Laboratory of Oncology in South China, Collaborative Innovation Center for Cancer Medicine
Key Highlights:

Research Background: This multicenter clinical trial conducted in China evaluated the safety and preliminary efficacy of alrizomadlin (APG-115) as monotherapy or in combination with lisaftoclax in heavily pretreated pediatric patients with relapsed/metastatic RMS, Ewing sarcoma (EWS), neuroblastoma (NB), and other solid tumors.
Efficacy Data: In the monotherapy arm, 1 patient with refractory RMS achieved CR. In the combination arm, among 17 response-evaluable pediatric patients with relapsed/refractory solid tumors, the ORR was 23.5%, including 1 CR in a patient with EWS, as well as PRs in 2 patients with RMS and 1 patient with NB. The disease control rate (DCR) was 70.6%.
Safety Data: No dose-limiting toxicities (DLTs) were observed in either the monotherapy or combination arm. Adverse events were primarily gastrointestinal and hematologic, with few serious adverse events and no treatment-related deaths or discontinuations.
Conclusion: The regimen demonstrated a manageable safety profile and preliminary antitumor activity in pediatric solid tumors, supporting further investigation.

* Alrizomadlin is currently under investigation and has not yet been approved by the US FDA.

(Press release, Ascentage Pharma, MAY 31, 2026, View Source [SID1234666283])