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Rockville, Maryland, USA and Suzhou, ChinaJune 1, 2026 /PRNewswire/ — Ascentage Pharma (Nasdaq: AAPG; HKEX: 6855), a leading biopharmaceutical company committed to developing innovative drugs in oncology and other fields, announced today that it has presented, for the first time, the latest data from its apoptosis pipeline key asset, the MDM2-p53 inhibitor Alrizomadlin (APG-115), as a monotherapy or in combination with Lisaftoclax (brand name: Lisantuo®; development code: APG-2575), in pediatric patients with relapsed/metastatic rhabdomyosarcoma or other soft-tissue sarcomas, in a Rapid Oral Presentation at the 62nd American Society of Clinical Oncology (ASCO) Annual Meeting.
The annual ASCO Annual Meeting is one of the most important and authoritative academic exchange events in the global oncology field, showcasing the latest cutting-edge clinical oncology research achievements and cancer treatment technologies. This year marks Ascentage Pharma’s ninth consecutive year at the ASCO Annual Meeting, with six studies from three key assets selected, three of which were accepted as Rapid Oral Presentations.
The research progress presented in this Rapid Oral Presentation preliminarily demonstrates the antitumor activity and safety of APG-115 in pediatric patients with solid tumors. The data showed: APG-115 monotherapy showed initial efficacy in pediatric rhabdomyosarcoma, with one pediatric patient achieving a complete response (CR); the combination with Lisaftoclax demonstrated significant antitumor activity, with an objective response rate (ORR) of 23.5% in 17 evaluable patients, including one patient with Ewing sarcoma achieving a CR and three achieving partial responses (PRs); in terms of safety, APG-115 as a monotherapy or in combination with Lisaftoclax was safe and manageable in the pediatric solid tumor population.
APG-115 is an oral, highly selective MDM2-p53 inhibitor independently developed by Ascentage Pharma. It is the first drug targeting this mechanism to enter clinical trials in China and has First-in-class potential. By blocking the MDM2-p53 interaction, the drug restores the tumor-suppressive activity of p53, thereby inducing apoptosis in tumor cells. Recently, APG-115 was officially included in the “Pediatric Antitumor Drug Research and Development Encouragement Pilot Program” (Starlight Program) by the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA), with planned development for pediatric solid tumors such as neuroblastoma, rhabdomyosarcoma, and Ewing sarcoma.
Professor Yizhuo Zhang, Principal Investigator of this clinical study and from the Department of Pediatric Oncology at Sun Yat-sen University Cancer Center, stated: “Relapsed/refractory pediatric sarcomas have a very poor prognosis, and there is an urgent clinical need. The data presented at ASCO this time confirm that APG-115, as a monotherapy and in combination with Lisaftoclax, shows good tolerability and significant antitumor activity, with the CR cases being particularly encouraging. As a key asset in the Starlight Program, its First-in-class potential is expected to fill a treatment gap and bring new hope for long-term survival to pediatric patients.”
Professor Yi Zhang, Investigator of this study and from the Department of Pediatrics at Beijing Tongren Hospital, Capital Medical University, stated: “Clinical treatment options for pediatric solid tumors, especially advanced soft-tissue sarcomas, are very limited. The clinical data presented by the APG-115 combination regimen this time are highly significant. This apoptosis pathway-targeted drug is well-tolerated and shows a considerable ORR, fully confirming the value of dual-target combination therapy in pediatric refractory tumors and pointing a high-quality direction for the future development of precision new drugs for pediatric cancers.”
Dr. Yifan Zhai, Chief Medical Officer of Ascentage Pharma, stated: “There has long been a significant unmet clinical need in the field of pediatric solid tumors. The study presented at ASCO this time marks the first appearance of data for APG-115 in treating pediatric solid tumor patients, and the preliminary efficacy and tolerability are encouraging. It is worth noting that APG-115 was previously included in the CDE’s ‘Starlight Program’ for development in various pediatric solid tumors. The data presented here provide preliminary evidence-based support for this direction. We will accelerate the progress of related research, hoping to bring hope to these pediatric patients in urgent need of new therapies as soon as possible.”
Key highlights of this study presented at the 2026 ASCO 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)
Alrizomadlin (APG-115) as a monotherapy 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 Number: 10012
Presentation Format: Rapid Oral Presentation
Session Title: Pediatric Oncology II
First Author: Professor Yizhuo Zhang, 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:
- Background: This is a domestic multicenter clinical trial evaluating the safety and preliminary efficacy of alrizomadlin (APG-115) as a monotherapy or in combination with Lisaftoclax in heavily pretreated pediatric patients with relapsed/metastatic rhabdomyosarcoma, Ewing sarcoma, neuroblastoma, and other solid tumors.
- Efficacy Data: In the monotherapy group, one patient with refractory rhabdomyosarcoma achieved a CR; in the combination group, among 17 evaluable pediatric patients with relapsed/refractory solid tumors, the ORR reached 23.5%, including one patient with Ewing sarcoma achieving a CR, and two patients with rhabdomyosarcoma and one with neuroblastoma achieving PRs; the disease control rate (DCR) reached 70.6%.
- Safety Data: Results showed no dose-limiting toxicities (DLTs) in either the monotherapy or combination groups. Adverse events were primarily gastrointestinal and hematological toxicities, with serious adverse events being rare. There were no treatment-related deaths or discontinuations.
- Conclusion: The regimen has a manageable safety profile and shows preliminary antitumor activity in pediatric solid tumors, warranting further investigation.
About Ascentage Pharma
Ascentage Pharma (Nasdaq: AAPG; HKEX: 6855) is a comprehensive global biopharmaceutical company dedicated to the research, development, production, and commercialization of innovative drugs to address unmet clinical needs of patients worldwide in the oncology field. The company has built a rich pipeline of innovative drug candidates, including inhibitors targeting key proteins in the apoptosis pathway such as Bcl-2 and MDM2-p53, next-generation inhibitors targeting kinase mutations arising in cancer treatment, and protein degraders.
The company’s core asset, Nailike®, is China’s first approved third-generation BCR-ABL inhibitor, approved for the treatment of adult patients with chronic myeloid leukemia in chronic phase (CML-CP) and accelerated phase (CML-AP) harboring the T315I mutation, as well as adult patients with CML-CP resistant and/or intolerant to first- and second-generation TKIs. All approved indications for this drug have been included in China’s National Reimbursement Drug List (NRDL). Currently, Ascentage Pharma is conducting three global registrational Phase III clinical studies for Nailike®: the POLARIS-1 study, authorized by the U.S. FDA and European EMA, evaluating Nailike® in newly diagnosed Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL) patients; the POLARIS-2 study, authorized by the U.S. FDA and European EMA, evaluating Nailike® in pretreated adult CML-CP patients; and the POLARIS-3 study evaluating Nailike® in patients with SDH-deficient GIST.
Another key asset of the company, Lisantuo®, is a novel Bcl-2 inhibitor for the treatment of various hematologic malignancies. Lisantuo® has been approved by China’s National Medical Products Administration (NMPA) for the treatment of adult patients with chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) who have received at least one prior systemic therapy, including a Bruton’s tyrosine kinase (BTK) inhibitor. Currently, Ascentage Pharma is conducting four global registrational Phase III clinical studies for Lisantuo®: the GLORA study, authorized by the U.S. FDA and European EMA, evaluating Lisantuo® in combination with a BTK inhibitor in CLL/SLL patients who have received prior BTK inhibitor therapy for more than 12 months and had a suboptimal response; the GLORA-2 study evaluating Lisantuo® as a first-line treatment for treatment-naïve CLL/SLL patients; the GLORA-3 study evaluating Lisantuo® as a first-line treatment for newly diagnosed elderly or unfit AML patients; and the GLORA-4 study, authorized by the U.S. FDA and European EMA, evaluating Lisantuo® as a first-line treatment for newly diagnosed intermediate- and high-risk MDS patients.
Leveraging its strong R&D capabilities, Ascentage Pharma has established a global intellectual property portfolio and entered into global collaborations with numerous leading biopharmaceutical companies, including Takeda, AstraZeneca, Merck Sharp & Dohme, Pfizer, and Innovent, as well as research partnerships with academic institutions such as the Dana-Farber Cancer Institute, Mayo Clinic, the National Cancer Institute, and the University of Michigan. For more information, please visit https://ascentage.com/
Forward-Looking Statements
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