PD-1 inhibitors(Programmed cell death protein 1 inhibitors), PDL1 inhibitors(Programmed death-ligand 1 inhibitors), PDL2 inhibitors(Programmed cell death 1 ligand 2 inhibitors)

Therapeutic Areas

NeoplasmsDigestive System DisordersMouth and Tooth Diseases+ [9]

Active Indication

Metastatic melanomaUnresectable MelanomaAdvanced Hepatocellular Carcinoma+ [71]

Inactive Indication

Advanced biliary tract cancerAdvanced cancerAdvanced Gastric Adenocarcinoma+ [17]

Originator Organization

Shanghai Junshi Biosciences Co., Ltd.

Active Organization

Shanghai Junshi Biosciences Co., Ltd.Topalliance Biosciences, Inc.

Cancer Hospital Chinese Academy of Medical Sciences

+ [5]

Inactive Organization

Antengene Corp. Ltd.

License Organization

Dr. Reddy's Laboratories Ltd.

Ascentage Pharma Group International Co., Ltd.Excellmab Pte. Ltd

+ [9]

Drug Highest PhaseApproved

First Approval Date

China (17 Dec 2018),

Melanoma

RegulationPriority Review (United States), Breakthrough Therapy (United States), Fast Track (United States), Orphan Drug (United States), Orphan Drug (European Union), Conditional marketing approval (China), Orphan Drug (Australia), Priority Review (Singapore), Priority Review (China)

Structure/Sequence

Sequence Code 453640391L

Source: *****

Sequence Code 524455911H

Source: *****

634

Clinical Trials associated with Toripalimab

NCT06940180

/ Not yet recruitingPhase 2IIT

Perioperative Chemoimmunotherapy With Toripalimab for Sinonasal Cancer

The aim of this research study is to evaluate the effectiveness and safety of a combination of immunotherapy, using a drug called toripalimab, with chemotherapy drugs, Carboplatin and Docetaxel, as a possible treatment before surgery for sinonasal cancers.
The names of the study drugs used in this research study are:
* Toripalimab (a type of monoclonal antibody)
* Carboplatin (a type of antineoplastic agent)
* Docetaxel (a type of antineoplastic agent)
* Cisplatin (a type of antineoplastic agent)

Start Date01 Oct 2025

Sponsor / Collaborator

Dana-Farber Cancer Institute, Inc.

[+1]

NCT06682780

/ Not yet recruitingPhase 1/2

An Open-label, Dose-escalation, and Dose-expansion Phase I/II Clinical Study of Safety, Tolerability, Pharmacokinetic Profile, and Initial Efficacy of LM-2417 for Injection Alone or in Combination With Other Antitumor Agents in Patients With Advanced Solid Tumors

This study is to assess the safety and tolerability, obtain the recommended phase 2 dose(RP2D)/or Maximum Tolerated Dose (MTD) for LM-2417 as a single agent or in combination with other anti-tumour agents in subjects with advanced solid tumours.

Start Date05 Jun 2025

Sponsor / Collaborator

LaNova Medicines Ltd.

NCT06987123

/ Not yet recruitingPhase 2IIT

Toripalimab in Combination With Capecitabine as Adjuvant Therapy After Surgery for Locally Recurrent Resectable Nasopharyngeal Carcinoma: a Single-arm Clinical Trial

Regarding the application value of capecitabine metronome chemotherapy's regulatory effect on the immune microenvironment in nasopharyngeal carcinoma, many studies in recent years have confirmed that metronome chemotherapy and immunotherapy are safe and effective in resectable recurrent nasopharyngeal carcinoma. Therefore, the investigators plan to conduct a "single-arm clinical study on adjuvant therapy of toripalimab combined with capecitabine after Surgery for locally recurrent resectable nasopharyngeal carcinoma" to explore the efficacy and safety of toripalimab combined with capecitabine as adjuvant therapy after salvage surgery for resectable recurrent nasopharyngeal carcinoma. If this study is confirmed, it is expected to provide a new treatment model for patients with resectable recurrent nasopharyngeal carcinoma.

Start Date18 May 2025

Sponsor / Collaborator

Sun Yat-Sen University

100 Clinical Results associated with Toripalimab

100 Translational Medicine associated with Toripalimab

100 Patents (Medical) associated with Toripalimab

2,041

Literatures (Medical) associated with Toripalimab

01 Dec 2025·Journal of Gastrointestinal Cancer

90Y-Transarterial Radioembolization Combined with Immune Checkpoint Inhibitors in Hepatocellular Carcinoma: A Systematic Review

Review

Author: Shobeiri, Parnian ; Lewandowski, Robert J ; Fowler, Kathryn J ; Behnoush, Amir Hossein ; Hosseini Shabanan, Sedighe ; Haghshomar, Maryam

BACKGROUND:

Transarterial radioembolization with yttrium-90 (⁹⁰Yt-TARE) and immune checkpoint inhibitors (ICIs) are emerging as treatment modalities for intermediate to advanced hepatocellular carcinoma (HCC) based on randomized controlled trials. Herein, we systematically reviewed the published literature on the effects of ⁹⁰Yt-TARE and ICIs combined on clinical outcomes of HCC.

METHODS:

Medical databases of PubMed, Embase, and Cochrane Library were systematically searched for all studies assessing the use of concomitant immunotherapy of ICI with TARE in patients with HCC. Patient characteristics, treatment protocols, treatment outcomes, treatment adverse events, and survival outcomes were extracted after the screening phase. The primary outcomes were overall survival (OS) and patient-free survival (PFS), while the secondary outcomes were imaging objective response (OR) and adverse events.

RESULTS:

Among 3432 reviewed, ten studies were included in this systematic review, including four randomized controlled trials and six retrospective studies. These consisted of 413 patients with HCC, and seven studies included patients with Child-Pugh A or B7 scores. Most studies allowed advanced or intermediate HCC stages, but only two specified BCLC stages (B and C). Median tumor sizes ranged from 56 to 78.5 mm. Various agents with different administration schedules were used as ICIs for immunotherapy by different studies for the combination of ⁹⁰Yt-TARE with ICIs. Median OS ranged from 16.2 to 27 months between different studies while the PFS also ranged from 5.6 to 13.3 months. The OR rates according to imaging-based response assessments were reported between 31 and 89%, and the incidence rate of any grade toxicities was between 50 and 80%.

CONCLUSION:

Concomitant treatment with ⁹⁰Yt-TARE and ICIs has shown promising results in the treatment of patients with HCC. Further studies are required to reach a consensus on the optimal treatment protocol and the outcome of these treatments for patients with intermediate to advanced HCC.

01 Jul 2025·Lancet Gastroenterology & Hepatology

Toripalimab plus bevacizumab versus sorafenib as first-line treatment for advanced hepatocellular carcinoma (HEPATORCH): a randomised, open-label, phase 3 trial

Article

Author: Wang, Tsang-En ; Li, Enxiao ; Liu, Hongming ; Cheng, Ying ; Wen, Tianfu ; Liang, Bo ; Mao, Yilei ; Lv, Jing ; Zhang, Wenhua ; Zhang, Mingjun ; Wang, Wei ; Guo, Wenzhi ; Guo, Yabing ; Zeng, Hui ; Hao, Chunyi ; Shen, Jie ; Jin, Yongdong ; Shi, Yinghong ; Zheng, Jinfang ; Du, Chengyou ; Xu, Hao ; Liu, Rong ; Shi, Xuetao ; Zhou, Fuxiang ; Hua, Xiangdong ; Zhang, Xiao ; Shao, Bingfeng ; Huang, Jee-Fu ; Shan, Yunfeng ; Zhang, Chengwu ; Zhou, Ledu ; Liu, Chao ; Chen, Yajin ; Wu, Jianbing ; Hu, Sheng ; Samol, J ; Han, Guohong ; Liu, Bifeng ; Li, Yong ; Zhang, Tao ; He, Yifu ; Su, Weiwen ; Shen, Yan ; Zhang, Feng ; Bai, Yuxian ; Pin, Choo Su ; Wang, Rui ; Shen, Shunli ; Jia, Weidong ; Chen, Zhiyu ; Fan, Jia ; Hu, Hongtao ; Bai, Aobing ; Zhang, Wu ; Jin, Chunlei ; Xu, Qian ; Li, Xun ; Wang, Xiangcai ; Zhang, Guoping ; Huang, Ming ; Song, Tianqiang ; Zhou, Jian ; Zhang, Bixiang ; Gu, Shanzhi

BACKGROUND:

Although several PD-1 or PD-L1 inhibitors combined with antiangiogenic agents have been approved as first-line treatment of advanced hepatocellular carcinoma, treatment needs remain unmet given the high incidence and mortality of hepatocellular carcinoma and due to factors such as regional approval status, medical insurance restrictions, and cost considerations. In this phase 3 HEPATORCH study, we aimed to compare the efficacy and safety of toripalimab plus bevacizumab versus sorafenib in patients with previously untreated advanced hepatocellular carcinoma.

METHODS:

We did a randomised, open-label, phase 3 study in 57 hospitals across mainland China, Taiwan, and Singapore. Using a central interactive web response system, eligible patients aged 18-75 years with unresectable or metastatic hepatocellular carcinoma were randomly assigned (1:1) through a stratified block randomisation method to receive 240 mg toripalimab (intravenously, once every 3 weeks) plus 15 mg/kg bevacizumab (intravenously, once every 3 weeks) or 400 mg sorafenib (oral, twice daily). Randomisation was stratified by macrovascular invasion or extrahepatic spread (presence vs absence), ECOG performance status score (0 vs 1), and history of locoregional therapy (yes vs no). The co-primary endpoints were progression-free survival (assessed by the Independent Review Committee per Response Evaluation Criteria in Solid Tumors, version 1.1) and overall survival. Efficacy analysis was performed in the intention-to-treat population (ie, all patients randomly assigned to a treatment group). Safety was assessed in all patients who received at least one dose of study treatment. The study is registered with ClinicalTrials.gov, NCT04723004, and is completed.

FINDINGS:

Between Nov 23, 2020, and Jan 21, 2022, 545 patients were screened for study inclusion, of whom 219 did not meet the screening criteria. 326 patients were randomly assigned to receive an intervention: 162 patients were assigned to the toripalimab plus bevacizumab group and 164 were assigned to the sorafenib group, with median age 58·0 years (IQR 50·0-66·0) and 56·0 years (49·0-61·0) years, respectively. All 326 patients were included in the intention-to-treat population and the safety population. 282 (87%) patients were male and 44 (14%) were female. At the primary analysis of progression-free survival (data cutoff Aug 10, 2022), median follow-up was 9·4 months (IQR 7·0-12·0). Toripalimab plus bevacizumab significantly prolonged progression-free survival compared with sorafenib (median 5·8 months [95% CI 4·6-7·2] vs 4·0 months [2·8-4·2]; hazard ratio [HR] 0·69 [95% CI 0·53-0·91; p=0·0086). At the final analysis of overall survival (May 31, 2024), median follow-up was 16·4 months (IQR 7·1-29·5). Toripalimab plus bevacizumab significantly improved overall survival compared with sorafenib (median 20·0 months [95% CI 15·3-23·4] vs 14·5 months [11·4-18·8]; HR 0·76 [95% CI 0·58-0·99; p=0·039). Grade 3 or higher adverse events occurred in 102 (63%) patients in the toripalimab plus bevacizumab group compared with 100 (61%) in the sorafenib group, and led to discontinuation of treatment in 21 (13·0%) participants in the toripalimab plus bevacizumab group and 20 (12%) participants in the sorafenib group. The incidence of treatment-related fatal adverse events (two [1%] vs one [1%]) was similar between the toripalimab plus bevacizumab and sorafenib groups. The most common (incidence ≥5% in the toripalimab plus bevacizumab group) grade 3-4 adverse events were hypertension (26 [16%] in the toripalimab plus bevacizumab group vs 19 [12%] in the sorafenib group), thrombocytopenia (16 [10%] vs four [2%]), upper gastrointestinal haemorrhage (ten [6%] vs one [1%]), anaemia (nine [6%] vs seven [4%]), and abnormal hepatic function (nine [6%] vs five [3%]). The most common (incidence ≥2% in the toripalimab plus bevacizumab group) serious adverse events were upper gastrointestinal haemorrhage (12 [7%] vs one [1%]), abnormal hepatic function (eight [5%] vs five [3%]), ascites (six [4%] vs three [2%]), and gastrointestinal haemorrhage (four [2%] vs three [2%]).

INTERPRETATION:

Among patients with previously untreated advanced hepatocellular carcinoma, toripalimab plus bevacizumab resulted in significantly longer progression-free survival and overall survival than did sorafenib, with an acceptable safety profile. Based on these results, the regimen has been approved for use in China by the National Medical Products Administration.

FUNDING:

Shanghai Junshi Biosciences.

TRANSLATION:

For the Chinese translation of the abstract see Supplementary Materials section.

01 Jun 2025·CRITICAL REVIEWS IN ONCOLOGY HEMATOLOGY

A comprehensive review on targeting diverse immune cells for anticancer therapy: Beyond immune checkpoint inhibitors

Review

Author: Liu, Dequan ; Chen, Xiaochi ; Zhao, Xinming ; Wu, Guangzhen ; Zhang, Xiaoman ; Che, Xiangyu ; Liu, Lei

Although immune checkpoint inhibitors (ICIs) have revolutionized cancer treatment, primary resistance and acquired resistance continue to limit their efficacy for many patients. To address resistance and enhance the anti-tumor activity within the tumor immune microenvironment (TIME), numerous therapeutic strategies targeting both innate and adaptive immune cells have emerged. These include combination therapies with ICIs, chimeric antigen receptor T-cell (CAR-T), chimeric antigen receptor macrophages (CAR-Ms) or chimeric antigen receptor natural killer cell (CAR-NK) therapy, colony stimulating factor 1 receptor (CSF1R) inhibitors, dendritic cell (DC) vaccines, toll-like receptor (TLR) agonists, cytokine therapies, and chemokine inhibition. These approaches underscore the significant potential of the TIME in cancer treatment. This article provides a comprehensive and up-to-date review of the mechanisms of action of various innate and adaptive immune cells within the TIME, as well as the therapeutic strategies targeting each immune cell type, aiming to deepen the understanding of their therapeutic potential.

481

News (Medical) associated with Toripalimab

05 Jun 2025

·www.prnewswire.com

Highlights in Oral Presentation of Mabwell's 9MW2821 at 2025 ASCO

SHANGHAI, June 5, 2025 /PRNewswire/ -- At the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting held in Chicago, USA, from May 30 to June 3, the results of the Phase Ib/II clinical study of 9MW2821 in combination with Toripalimab for the treatment of patients with locally advanced or metastatic urothelial carcinoma (la/mUC) were presented by Prof. Xinan Sheng, Chief Physician of the Department of Urologic Oncology, Beijing Cancer Hospital, on behalf of the research team, with key highlights as below: Oral Presentation Background: Urothelial carcinoma (UC) is a common malignant tumor worldwide. Global cancer statistics released in 2024 showed that bladder cancer is the ninth most commonly diagnosed cancer in the world, with about 614,000 new cases and 220,000 deaths annually. The burden and morbidity of bladder cancer is significantly higher in men than in women. It is the sixth most common cancer and the ninth leading cause of cancer death in men [1]. 9MW2821 (Bulumtatug Fuvedotin, BFv) is a next-generation Nectin-4 targeting antibody-drug conjugate. Previous study of BFv has shown promising efficacy in la/mUC patients who progressed on/after platinum-based chemotherapy and immune checkpoint inhibitors [2]. A pivotal phase 3 study of BFv is ongoing in China (NCT06196736, CTR20234024). Toripalimab is a novel recombinant humanized anti-PD-1 monoclonal antibody that has been approved in multiple countries including China and the USA. It is the first time to report in detail on the efficacy and safety of 9MW2821 in combination with Toripalimab in patients with la/mUC. Method: This is a phase Ib/II, open-label, multicenter study of BFv and Toripalimab in la/mUC in China. Every 21 days per cycle, patients received BFv (1.0 or 1.25mg/kg, on day 1 and 8) and Toripalimab (240mg, on day 1). Treatment until disease progression or unacceptable toxicity. Results: As of Apr. 30, 2025, 52 patients with la/mUC were enrolled. Among the 40 assessable patients (treatment-naive for la/mUC) with the median age of 67, 70% of patients were male, 72.5% had an Eastern Cooperative Oncology Group (ECOG) score of 1, 55% with tumor primary site at upper urinary tract, 15% had liver metastases, 82.5% were Nectin-4 positive and 20% were PD-L1 positive. The Objective Response Rate (ORR) was 87.5% (95% CI: 73.2, 95.8), confirmed ORR was 80%, complete response (CR) rate was 12.5%, Disease Control Rate (DCR) was 92.5% (95% CI: 79.6, 98.4). 97.5% of assessable patients had tumor reduction, 57.5% of assessable patients had tumor reduction of 50% above. The ORR in selected subgroups was consistent with results in overall population. Different subgroups of treatment-naive patients could benefit from BFv and Toripalimab regardless of primary tumor site, liver metastasis, expression of Nectin-4 and PD-L1. The ORR was 100% (24/24) in patients older than 65, 94.44% (17/18) in patients with tumor primary site at lower urinary tract, 83.33% (5/6) in patients with liver metastasis, 100% (7/7) in nectin-4 negative patients, and 100% (8/8) in PD-L1 positive patients. As of April 30, 2025, 45% (18/40) of subjects experienced disease progression or death, with a median progression-free survival (mPFS) of 12.5 months (95% CI: 6.47-NA), and a median duration of response (mDoR) that has not been reached. Median follow-up time was 10.8 months, thus mPFS and mDoR was not mature. The incidence rate of treatment-related adverse events (TRAE) was 98.1%, and most of the TRAEs were grade 1-2. The incidence rate of TRAEs ≥grade 3 was 42.3%, mainly characterized by neutrophil counts decreased (11.5%), alanine aminotransferase increased (5.8%), and white blood cell decreased (5.8%). The incidence rate of serious TRAEs was 28.8%, and no TRAEs leading to death occurred. BFv and Toripalimab showed well-tolerated safety profile. No new safety signals of BFv or Toripalimab were observed in this study. Conclusion: BFv combined with Toripalimab has demonstrated encouraging efficacy and manageable safety profile in la/mUC. Patients with advanced age, liver metastases and negative expression of Nectin-4 also achieved considerable response in this study, suggesting that BFv in combination with Toripalimab may provide a new treatment option for patients with advanced UC who are intolerant of or potentially have a poor prognosis with platinum-based chemotherapy. A pivotal phase 3 study of BFv combined with Toripalimab vs platinum-based chemotherapy is ongoing in China (NCT06592326, CTR20242828). About 9MW2821 9MW2821 is a novel Nectin-4 targeting ADC, which achieves site-specific modification of antibody through proprietary conjugate technology linkers and optimized ADC conjugation process of Mabwell. It stands out as the first clinical-stage drug candidate among Chinese companies for this specific target. And it's the first Nectin-4 targeting ADC to disclose clinical efficacy data in cervical, esophageal, and breast cancers. 3 Phase III pivotal clinical trials are ongoing. Its monotherapy and combination therapy with Toripalimab for UC have been granted Breakthrough Therapy Designation (BTD) by the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) in China. It has also been granted Fast Track Designations (FTD) for 3 indications and Orphan Drug Designation (ODD) for 1 indication by the U.S. Food and Drug Administration (FDA). About Mabwell Mabwell (688062.SH) is an innovation-driven biopharmaceutical company with the entire value chain of the pharmaceutical industry. We provide more effective and accessible therapy and innovative medicines to fulfill global medical needs. Since 2017, an advanced R&D system which covers target discovery, early discovery, druggability, preclinical, clinical research and manufacturing transformation was established. Mabwell has 16 pipeline products in different stages based on a world-class and state-of-the-art R&D engine, including 12 novel drug candidates and 4 biosimilars. We focus on the therapeutic areas of oncology, immunology, bone disorders, ophthalmology, hematology and infectious diseases, etc. Of these, 4 products have been approved and commercialized, 1 product filed for pre-NDA meeting, 2 products are in pivotal trials. We have also undertaken 1 national major scientific and technological special project for "Significant New Drugs Development", 2 projects for National Key R&D Programmes, and multiple provincial and municipal science and technological innovation projects. Mabwell's Taizhou factory possesses robust in-house manufacturing capability compliant with international GMP standards regulated by the NMPA, FDA and EMA, and has passed the EU QP Audit. The large-scale manufacturing base in Shanghai and the ADC commercialized manufacturing base in Taizhou are under construction. Our mission is "Explore Life, Benefit Health" and our vision is "Innovation, from ideas to reality". For more information, please visit . Forward-Looking Statements This press release contains forward-looking statements including, but not limited to, the potential safety, efficacy, regulatory review or approval and commercial success of our product candidates and those relating to the Company's product development, clinical studies, clinical and regulatory milestones and timelines, market opportunity, competitive position, possible or assumed future results of operations, business strategies, potential growth opportunities and other statements that are predictive in nature. "Forward-looking statements" are statements that are not historical facts and involve a number of risks and uncertainties, which may cause actual results to be materially different from any future results expressed or implied in the forward-looking statements. These statements may be identified by the use of forward-looking expressions, including, but not limited to, "expect", "anticipate", "intend", "plan", "believe", "estimate", "potential", "predict", "project", "should", "would", and similar expressions and the negatives of those terms. Forward-looking statements are based on the Company's current expectations and assumptions. Forward-looking statements are subject to a number of risks, uncertainties, and other factors, many of which are beyond the Company's control, including, but not limited to: environment; politic; economy; society; legislation; our dependence on our product candidates, most of which are still in preclinical or various stages of clinical development; our reliance on third-party vendors, such as contract research organizations and contract manufacturing organizations; the uncertainties inherent in clinical testing; our ability to complete required clinical trials for our product candidates and obtain approval from regulatory authorities for our product candidates; our ability to protect our intellectual property; the potential impact of COVID-19; the loss of any executive officers or key personnel. In case one or more of these risks or uncertainties deteriorate, or any assumptions are incorrect, the actual results may be seriously inconsistent with the stated results. The Company cautions all the persons not to place undue reliance on any such forward-looking statements, which speaks only as of the date of this press release. The Company disclaims any obligation, except as specifically required by law and the rules of the applicable Stock authority to publicly update or revise any such statements to reflect any change in expectations or in events, conditions, or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. All forward-looking descriptions, figures and assumptions in this press release are applicable to this statement. SOURCE Mabwell WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Clinical ResultBreakthrough TherapyPhase 3Fast TrackASCO

03 Jun 2025

·www.prnewswire.com

2025 ASCO ｜Oral Presentation: Disitamab Vedotin Achieves Stellar Efficacy as First-Line Therapy for HER2-Expressing Locally Advanced or Metastatic Gastric Cancer

YANTAI, China, June 2, 2025 /PRNewswire/ -- On June 2 (Chicago time), in an oral presentation at the 2025 ASCO Annual Meeting, Dr. Lin Shen from Beijing Cancer Hospital presented the results of a Phase 2 clinical study conducted in China evaluating the efficacy and safety of disitamab vedotin (DV), developed by Remegen Co., Ltd., and toripalimab (PD-1) combined with CAPOX or trastuzumab as the first-line therapy for HER2-expressing patients with locally advanced or metastatic (la/m) gastric cancer. Comparing to the control group who received standard-of-care therapy, the DV combination therapy demonstrated clinically meaningful efficacy improvement, potentially to benefit patients who are non-responders to traditional targeted therapies. The data presented are from the Phase 2 part of a randomized, multi-cohort, seamlessly connecting Phase 2/3 study, which enrolled systematic chemotherapy-native patients with different HER2 expression levels. As of April 7, 2025, the results showed: Among HER2-overexpressing gastric cancer patients, compared to the PD-1-trastuzumab-CAPOX combination therapy, DV and PD-1 + chemotherapy as well as DV and PD-1 + trastuzumab both demonstrated statistically significant efficacy and favorable safety profiles. Objective response rate (ORR): 66.7% vs 82.4% vs 68.8%; Median progression-free survival (mPFS): NR vs NR vs 14.1 months, with risk of disease progression decreasing by 54%（HR=0.46）and 41% (HR: 0.59); 12-month PFS rate: 66.3%, 67% and 53.6%； Common TRAEs of grade 3-5: diarrhea, neutrophil count decreased, platelet count decreased, etc. In patients with HER2-low-expressing gastric cancer, promising efficacy was observed with DV + PD-1 + CAPOX comparing to PD-1 + CAPOX, with a manageable safety profile. ORR: 72.0% vs 47.8%; mPFS: 9.9 vs 7.2 months, with risk of disease progression decreasing by 31% (HR: 0.69); Common TRAEs of grade 3-5: diarrhea, neutrophil count decreased, platelet count decreased, etc. Dose optimization conducted in patients with HER2-median/low-expressing gastric cancer. Compared to PD-1 + CAPOX, DV at 2.5 mg/kg or 2.0 mg/kg combined with PD-1 + reduced-dose CAPOX showed significant efficacy, and better safety over the full-dose chemotherapy. ORR: 71.4% vs 66.7% vs 56.3%; 6-month PFS rate: 71.4%，72.7% and 53.3%. Globally, this is the first study to explore the triple combination therapy of "HER2 ADC + PD-1 + targeted medication" as first-line treatment of patients with la/m gastric cancer, pioneering a new mode of synergistic therapy. The multi-cohort design of this study provides precision treatment regimen for gastric cancer patients with different level of HER2 expression. For the HER2-overexpressing gastric cancer patients, DV + PD-1 + trastuzumab has the potential to become the new standard first-line treatment; for the HER2-low-expressing gastric cancer patients, DV + PD-1 + chemotherapy has the potential to fill the treatment gap of these patients. Based on the data obtained from the phase 2 study, the phase 3 clinical study of the triple combination therapy in patients with HER2-median/low-expressing gastric cancer has been initiated in April, 2025, in which 616 participants were planned to be enrolled, to further validate the efficacy of the DV combination therapy. Gastric cancer is the fifth most common malignant tumor in the world, and China accounts for about 42.6% of new cases and 45.0% of deaths worldwide. HER2 is an important target in the treatment of gastric cancer, while the traditional targeted drug trastuzumab is only effective in the population with high expression (IHC 3+ or IHC 2+/FISH+), and can easily become resistant. There is a lack of effective targeted therapy options for patients with low/median HER2 expression (IHC 1+ or IHC 2+/FISH-), and the efficacy of chemotherapy combined with immunotherapy is not satisfactory. As the first domestic HER2-targeted ADC drug in China, DV not only precisely kills tumor cells with HER2 over expression but also attacks adjacent cells with HER2 low expression through the bystander effect. Preclinical studies have also shown that the combination of DV with PD-1 inhibitor and trastuzumab can enhance anti-tumor activity. SOURCE RemeGen Co., Ltd WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Clinical ResultPhase 2Phase 3ImmunotherapyADC

02 Jun 2025

·www.einpresswire.com

Live from ASCO 2025 | Ascentage Pharma Releases Promising Clinical Data on Alrizomadlin Monotherapy and Combinations in Solid Tumors

ROCKVILLE, Md. and SUZHOU, China, June 02, 2025 (GLOBE NEWSWIRE) -- Ascentage Pharma (NASDAQ: AAPG; HKEX: 6855), a global biopharmaceutical company dedicated to addressing unmet medical needs in cancers, announced that it has released the latest clinical data from its Phase II study of the MDM2-p53 inhibitor alrizomadlin (APG-115) as a single agent or in combination with PD-1 inhibitor toripalimab in patients with advanced adenoid cystic carcinoma (ACC) or other solid tumors in a poster presentation at the 61 st American Society of Clinical Oncology (ASCO) Annual Meeting. Alrizomadlin is the first MDM2-p53 inhibitor to enter clinical development in China and a key investigational drug candidate in Ascentage Pharma’s apoptosis-targeted pipeline with global first-in-class potential. The ASCO Annual Meeting showcases the most cutting-edge research in clinical oncology and state-of-the-art advanced cancer therapies and is the world’s most influential and prominent scientific gathering of the clinical oncology community. Returning to the ASCO Annual Meeting for the eighth consecutive year, Ascentage Pharma has garnered growing interest from the global research community. This year, two studies of the Bcl-2 inhibitor lisaftoclax (APG-2575) and the MDM2-p53 inhibitor alrizomadlin, key drug candidates in the company’s apoptosis-targeted pipeline, have been selected for presentations, including an oral presentation, at the ASCO Annual Meeting. These clinical data on alrizomadlin in ACC and other solid tumors demonstrated the antitumor activity of alrizomadlin monotherapy in patients with advanced ACC or malignant peripheral nerve sheath tumor (MPNST). Moreover, alrizomadlin in combination with toripalimab was well tolerated and showed therapeutic potential in MPNST, biliary-tract cancer (BTC), and liposarcoma (LPS). Prof. Ye Guo, MD, a Principal Investigator of the study from the Department of Medical Oncology, Shanghai East Hospital , noted, “ACC is a rare cancer type that lacks effective treatment options, and the treatment with antiangiogenic tyrosine kinase inhibitors faces certain limitations and safety concerns. At ASCO 2025, our team presented data of alrizomadlin in patients with ACC that demonstrated an objective response rate (ORR) of 16.7% and a disease control rate (DCR) of 100%. These results suggest that the targeted inhibition of the MDM2-p53 pathway has antitumor activity in ACC; therefore, it can potentially offer a new treatment strategy to patients with ACC.” Prof. Ning Li, MD, a Principal Investigator of the study from the Chinese Academy of Medical Sciences Cancer Hospital , commented, “Currently, there are limited treatment options for patients with sarcomas such as MPNST and LPS. In this study, alrizomadlin both as a monotherapy and in combination with a PD-1 therapy, showed favorable antitumor activity in MPNST. The clinical benefit was particularly notable in patients who received the combination regimen, with two patients with MPNST achieving long-term responses that lasted more than 60 and 96 weeks, respectively. Furthermore, alrizomadlin in combination with a PD-1 therapy also showed clinical activity in LPS. These results suggest that alrizomadlin monotherapy and combination regimens may bring clinical benefit to more patients with sarcoma.” Dr. Yifan Zhai, Chief Medical Officer of Ascentage Pharma , said, “Alrizomadlin is an investigational drug with global first-in-class potential. The clinical data presented at this year’s ASCO Annual Meeting demonstrated the therapeutic potential of alrizomadlin, both as a monotherapy and in combinations, in ACC and other solid tumors, and underscored the synergistic effects between alrizomadlin and immunotherapies, thus suggesting a promising therapeutic strategy for various solid tumors. We are focused on addressing unmet clinical needs in China and around the world, and intend to further accelerate our clinical programs to advance more novel treatment options for patients as soon as possible.” Highlights of this abstract selected for presentation at ASCO 2025 are as follows: A Phase 2 Study of Novel MDM2 Inhibitor Alrizomadlin (APG-115) With or Without Toripalimab in Patients with Advanced Adenoid Cystic Carcinoma (ACC) or Other Solid Tumors Abstract #: 6102 Format: Poster Presentation Session Title: Head and Neck Cancer Principal Authors: Ye Guo, MD, Department of Medical Oncology, Shanghai East Hospital, China; Ning Li, MD, Chinese Academy of Medical Sciences Cancer Hospital, China; Xing Zhang, MD, Melanoma and Sarcoma Medical Oncology Unit, Sun Yat-sen University Cancer Center, China; Meiyu Fang, MD, Department of Rare Cancer & Head and Neck Medical Oncology, Cancer Hospital of the University of Chinese Academy of Sciences, China; Shuhang Wang, MD, Chinese Academy of Medical Sciences Cancer Hospital, China, et al. Highlights: Alrizomadlin is a novel investigational oral MDM2 inhibitor that has shown a manageable safety profile with initial clinical activity in ACC. As of the data cutoff date of February 13, 2025, 57 patients with advanced ACC, malignant peripheral nerve sheath tumor (MPNST), liposarcoma (LPS), biliary-tract cancer (BTC), and other tumors were enrolled. Alrizomadlin monotherapy showed encouraging antitumor activity in patients with advanced ACC or MPNST. Alrizomadlin in combination with toripalimab was also well tolerated and demonstrated antitumor activity in patients with MPNST, BTC, and LPS. In the monotherapy arm, 17 patients were efficacy-evaluable. The ORR was 16.7%, and the DCR was 100% in 12 patients with ACC. The DCR was 80% in 5 patients with MPNST, 4 of whom achieved stable disease (SD). In 24 safety-evaluable patients, 33.3% reported grade 3 or higher treatment-related adverse events (TRAEs). Three patients (12.5%) experienced treatment-related serious adverse events (SAEs). One patient discontinued treatment because of TRAE. In the combination arm, 29 patients were efficacy-evaluable. The ORR was 16.7% and the DCR was 100% in 6 patients with BTC. The ORR was also 16.7% and the DCR was 66.7% in 6 patients with LPS. Two patients with MPNST had confirmed partial response (PR) with prolonged progression free survival (PFS) of 60 + weeks and 96 + weeks, respectively. In 27 safety-evaluable patients treated with alrizomadlin at the 150 mg dose level, 12 (44.4%) experienced grade 3 or higher TRAEs. Treatment-related SAEs were reported in 8 patients (29.6%). One patient discontinued treatment because of TRAE. * Alrizomadlin (APG-115) is an investigational compound and has not been approved by the US FDA. About Ascentage Pharma Ascentage Pharma (NASDAQ: AAPG; HKEX: 6855) is a global biopharmaceutical company dedicated to addressing unmet medical needs in cancers. The company has built a rich pipeline of innovative drug candidates that includes inhibitors targeting key proteins in the apoptotic pathway, such as Bcl-2 and MDM2-p53 and next-generation kinase inhibitors. The lead asset, olverembatinib, is the first 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). The Company is currently conducting an FDA-cleared, global registrational Phase III trial, or POLARIS-2, of olverembatinib for CML, as well as global registrational Phase III trials for newly diagnosed Ph+ ALL patients and SDH-deficient GIST patients. The second lead asset, lisaftoclax, is a novel Bcl-2 inhibitor for the treatment of various hematological malignancies. The NDA for the treatment of relapsed and/or refractory CLL and SLL was accepted with Priority Review designation by China’s National Medical Products Administration. The Company is currently conducting an FDA-cleared, global registrational Phase III trial, or GLORA, of lisaftoclax in combination with BTK inhibitors for patients with CLL/SLL previously treated with BTK inhibitors for more than 12 months with sub-optimal response, as well as global registrational Phase III trials for newly diagnosed CLL/SLL, AML and MDS patients. 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/ 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 “Special note regarding forward-looking statements and industry data” in its Registration Statement on Form F-1, as amended, filed with the SEC on January 21, 2025, and the Form 20-F filed with the SEC on April 16, 2025, the sections headed “Forward-looking Statements” and “Risk 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 we made or make 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. Contacts Investor Relations: Hogan Wan, Head of IR and Strategy Ascentage Pharma Hogan.Wan@ascentage.com +86 512 85557777 Stephanie Carrington ICR Healthcare Stephanie.Carrington@icrhealthcare.com +1 (646) 277-1282 Media Relations: Sean Leous ICR Healthcare Sean.Leous@icrhealthcare.com +1 (646) 866-4012 Legal Disclaimer: EIN Presswire provides this news content "as is" without warranty of any kind. We do not accept any responsibility or liability for the accuracy, content, images, videos, licenses, completeness, legality, or reliability of the information contained in this article. If you have any complaints or copyright issues related to this article, kindly contact the author above.

Phase 2Phase 3ASCOClinical ResultPriority Review

100 Deals associated with Toripalimab

External Link

KEGG	Wiki	ATC	Drug Bank
-	-	L01XC	DB15043

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Metastatic melanoma	China	Shanghai Junshi Biosciences Co., Ltd.	22 Apr 2025
Unresectable Melanoma	China	Shanghai Junshi Biosciences Co., Ltd.	22 Apr 2025
Advanced Hepatocellular Carcinoma	China	Shanghai Junshi Biosciences Co., Ltd.	18 Mar 2025
Metastatic Esophageal Squamous Cell Carcinoma	European Union	Topalliance Biosciences, Inc.	19 Sep 2024
Metastatic Esophageal Squamous Cell Carcinoma	Iceland	Topalliance Biosciences, Inc.	19 Sep 2024
Metastatic Esophageal Squamous Cell Carcinoma	Liechtenstein	Topalliance Biosciences, Inc.	19 Sep 2024
Metastatic Esophageal Squamous Cell Carcinoma	Norway	Topalliance Biosciences, Inc.	19 Sep 2024
Nasopharyngeal Neoplasms	European Union	Topalliance Biosciences, Inc.	19 Sep 2024
Nasopharyngeal Neoplasms	Iceland	Topalliance Biosciences, Inc.	19 Sep 2024
Nasopharyngeal Neoplasms	Liechtenstein	Topalliance Biosciences, Inc.	19 Sep 2024
Nasopharyngeal Neoplasms	Norway	Topalliance Biosciences, Inc.	19 Sep 2024
Oesophageal squamous cell carcinoma recurrent	European Union	Topalliance Biosciences, Inc.	19 Sep 2024
Oesophageal squamous cell carcinoma recurrent	Iceland	Topalliance Biosciences, Inc.	19 Sep 2024
Oesophageal squamous cell carcinoma recurrent	Liechtenstein	Topalliance Biosciences, Inc.	19 Sep 2024
Oesophageal squamous cell carcinoma recurrent	Norway	Topalliance Biosciences, Inc.	19 Sep 2024
Unresectable Esophageal Squamous Cell Carcinoma	European Union	Topalliance Biosciences, Inc.	19 Sep 2024
Unresectable Esophageal Squamous Cell Carcinoma	Iceland	Topalliance Biosciences, Inc.	19 Sep 2024
Unresectable Esophageal Squamous Cell Carcinoma	Liechtenstein	Topalliance Biosciences, Inc.	19 Sep 2024
Unresectable Esophageal Squamous Cell Carcinoma	Norway	Topalliance Biosciences, Inc.	19 Sep 2024
Triple Negative Breast Cancer	China	Shanghai Junshi Biosciences Co., Ltd.	18 Jun 2024

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Neoplasms	NDA/BLA	Canada	Apotex, Inc.	01 Dec 2024
Nasopharyngeal Cancer, Recurrent	NDA/BLA	European Union	Tmc Pharma (Eu Ltd.	14 Nov 2022
Recurrent Lung Non-Squamous Non-Small Cell Carcinoma	Phase 3	China	Shanghai Junshi Biosciences Co., Ltd.	11 Jul 2024
Refractory Classic Hodgkin Lymphoma	Phase 3	China	Shanghai Junshi Biosciences Co., Ltd.	28 Dec 2023
Small cell lung cancer limited stage	Phase 3	United States	Shanghai Junshi Biosciences Co., Ltd.	15 Nov 2023
Small cell lung cancer limited stage	Phase 3	China	Shanghai Junshi Biosciences Co., Ltd.	15 Nov 2023
Small cell lung cancer limited stage	Phase 3	Belgium	Shanghai Junshi Biosciences Co., Ltd.	15 Nov 2023
Small cell lung cancer limited stage	Phase 3	France	Shanghai Junshi Biosciences Co., Ltd.	15 Nov 2023
Small cell lung cancer limited stage	Phase 3	Georgia	Shanghai Junshi Biosciences Co., Ltd.	15 Nov 2023
Small cell lung cancer limited stage	Phase 3	Germany	Shanghai Junshi Biosciences Co., Ltd.	15 Nov 2023

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT06592326 (ASCO2025 \| PRNewswire) Manual	Phase 1/2	Transitional Cell Carcinoma First line	40	9MW2821 + toripalimab	gpkuqphrbe(crkcoheeic) = teiagwijag tdnujkxltt (lqqruxvsbx ) View more	Positive	30 May 2025
NCT04025931 (ASCO2025) Manual	Phase 2	Soft Tissue Sarcoma	69	Chidamide + toripalimab	scywfnegzu(taldzoywsa) = aofxxxzpje nshmibdaxv (pnlrxvdygk ) View more	Positive	30 May 2025
NCT05635643 (AACR2025 \| PipelineReview) Manual	Phase 1	Advanced Malignant Solid Neoplasm	30	CHS-114 (solid tumors + Arm 1a: CHS-114 dose escalation from 5 to 1200 mg)	cjonxmsfyg(xpushijmpm) = None anjxbsvqno (ddewzgzfpx ) View more	Positive	28 Apr 2025
				CHS-114 (HNSCC + Arm 1b: 2 dose levels [DL1 or DL2] of pharmacologically active CHS-114)
NCT04844385 (GASTO 1071, Literature) Manual	Phase 2	Locally Advanced Esophageal Squamous Cell Carcinoma Neoadjuvant	124	Neoadjuvant toripalimab plus chemotherapy followed by CCRT (60Gy)	tziufjfimg(lrhyyssvkk) = wlcoqzcduz npxgdvnequ (rebogzyrot, 53.9 - 78.3) View more	Positive	03 Apr 2025
				Neoadjuvant toripalimab plus chemotherapy followed by CCRT (50Gy)	tziufjfimg(lrhyyssvkk) = ugvirmgsbr npxgdvnequ (rebogzyrot, 53.7 - 78.8) View more
NCT04475016 (Pubmed \| Cancer Cell) Manual	Phase 2	Squamous Cell Carcinoma of the Penis Neoadjuvant	29	Toripalimab + Nimotuzumab + Taxol-based chemotherapy	lpzdrnwrzq(jaaqxviyte) = jhdborpdko hbvvscopil (kemezvifdz, 29.4 - 67.5) View more	Positive	01 Apr 2025
ESMO_ELCC2025	Not Applicable	Non-small cell lung cancer stage III	-	Toripalimab plus chemotherapy	ujxuubuqdh(hnmuutmcvx) = No other grade 3-5 TRAEs were further reported bagprssafv (tyhocbkeqg ) View more	-	26 Mar 2025
				Adjuvant toripalimab
NEWS Manual	Not Applicable	Renal Cell Carcinoma Neoadjuvant	21	特瑞普利单抗+阿昔替尼+手术	dpyugfuaal(tytertjaha) = rvxoooudpo esxadshbqp (mtjqgnyyxa ) View more	Positive	25 Mar 2025
				手术	dpyugfuaal(tytertjaha) = arwblzyhde esxadshbqp (mtjqgnyyxa ) View more
ESMO_SRC2025	Phase 2	Thymic Epithelial Tumor First line \| Maintenance	24	Toripalimab + Paclitaxel + Carboplatin	patctkyzbz(xyjtxbjkux) = efigwrnamg kvbrjevqjm (lczauovwfm, 14.0 - 32.0) View more	Positive	21 Mar 2025
ASCO_GU2025 Manual	Not Applicable	HER2 Positive Muscle Invasive Bladder Carcinoma Neoadjuvant HER2 Positive	28	Toripalimab + Disitamab vedotin	egcvcewwdc(qevbhhynac) = yigvpsgoxn gozaaopuik (jtrvkmmkby ) View more	Positive	13 Feb 2025
NCT05297552 (RC48-C017, ASCO_GU2025) Manual	Phase 2	HER2 Positive Muscle Invasive Bladder Carcinoma Adjuvant \| Neoadjuvant HER2 Expression	47	Disitamab vedotin+toripalimab	wqguvqvaut(irhnpwoprk) = jfzgnyrryo dxoflgtvxe (bibwznovto, 45.1 - 79.6) View more	Positive	13 Feb 2025
				Disitamab vedotin+toripalimab (PD-L1-positive)	wqguvqvaut(irhnpwoprk) = hbpaylvaru dxoflgtvxe (bibwznovto )

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