Full Press Release Details
Ascentage Pharma Presents Its First Dataset
Inhibitor Alrizomadlin (APG-115) in Pediatric Solid Tumors at ASCO 2026
ROCKVILLE, MD and SUZHOU, China, 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, today
announced 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) Annual Meeting.
The ASCO 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. 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 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
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 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 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
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
* Alrizomadlin is currently under investigation
and has not yet been approved by the US FDA.
About Ascentage Pharma
Ascentage Pharma Group International (NASDAQ:
AAPG; HKEX: 6855) ("Ascentage Pharma" or the "Company") is 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. The Company has built a rich pipeline of innovative drug products and candidates that include inhibitors targeting key proteins
in the apoptotic pathway, such as Bcl-2 and MDM2-p53, next-generation kinase inhibitors, and protein degraders.
The Company's first approved product, olverembatinib,
is the first novel third-generation BCR-ABL1 inhibitor approved in China for the treatment of patients with CML in chronic phase (CML-CP)
with T315I mutations, CML in accelerated phase (CML-AP) with T315I mutations, and CML-CP that is resistant or intolerant to first and
second-generation TKIs. It is covered by the China National Reimbursement Drug List (NRDL). Ascentage Pharma is currently conducting an
FDA- and EMA-cleared registrational Phase III trial, called POLARIS-2, of olverembatinib for CML, as well as an FDA- and EMA-cleared registrational
Phase III trials for patients with newly diagnosed Ph+ ALL, called POLARIS-1, and SDH-deficient GIST patients, called POLARIS-3.
The Company's second approved product, lisaftoclax,
is a novel Bcl-2 inhibitor for the treatment of various hematologic malignancies. Lisaftoclax 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 previously received at least one systemic therapy including Bruton's tyrosine kinase (BTK) inhibitors. The Company
is currently conducting four global registrational Phase III trials: the FDA- and EMA- cleared GLORA study of lisaftoclax in combination
with BTK inhibitors in patients with CLL/SLL previously treated with BTK inhibitors for more than 12 months with suboptimal response;
the GLORA-2 study in patients with newly diagnosed CLL/SLL; the GLORA-3 study in newly diagnosed, elderly and unfit patients with AML;
and the FDA- and EMA-cleared GLORA-4 study in patients with newly diagnosed higher risk MDS.
Leveraging its robust R&D capabilities, Ascentage
Pharma has built a portfolio of global intellectual property rights and entered into global partnerships and other relationships with
numerous leading biotechnology and pharmaceutical companies, such as Takeda, AstraZeneca, Merck, Pfizer, and Innovent, in addition to
research and development relationships with leading research institutions, such as Dana-Farber Cancer Institute, Mayo Clinic, National
Cancer Institute and the University of Michigan. For more information, visit https://ascentage.com/
Cautionary Note Regarding Forward-Looking Statements
This press release includes forward-looking statements
within the meaning of the Private Securities Litigation Reform Act of 1995 and Section 27A of the Securities Act of 1933, as amended,
and Section 21E of the Securities Exchange Act of 1934, as amended. All statements, other than statements of historical facts, contained
in this press release may be forward-looking statements, including statements that express Ascentage Pharma's opinions, expectations,
beliefs, plans, objectives, assumptions or projections regarding future events or future results of operations or financial condition.
These forward-looking statements are subject to a number of risks and uncertainties as discussed in Ascentage Pharma's filings with
the SEC, including those set forth in the sections titled "Risk factors" and "Cautionary note regarding forward-looking
statements" in its Annual Report on Form 20-F for the year ended December 31, 2025, filed with the SEC on April 29, 2026, the sections
headed "Forward-looking Statements" and "Risks Factors" in the prospectus of the Company for its Hong Kong initial
public offering dated October 16, 2019, and other filings with the SEC and/or The Stock Exchange of Hong Kong Limited where the Company's
ordinary shares are listed it has made or it makes from time to time that may cause actual results, levels of activity, performance or
achievements to be materially different from the information expressed or implied by these forward-looking statements. The forward-looking
statements contained in this presentation do not constitute profit forecast by the Company's management.
As a result of these factors, you should not rely
on these forward-looking statements as predictions of future events. The forward-looking statements contained in this press release are
based on Ascentage Pharma's current expectations and beliefs concerning future developments and their potential effects and speak
only as of the date of such statements. Ascentage Pharma does not undertake any obligation to update or revise any forward-looking statements,
whether as a result of new information, future events or otherwise.