On August 14, 2025 Daiichi Sankyo (TSE: 4568) reported it will present new clinical research across its DXd antibody drug conjugate (ADC) portfolio in lung cancer at the IASLC 2025 World Conference on Lung Cancer hosted by the International Association for the Study of Lung Cancer (#WCLC25) (Press release, Daiichi Sankyo, AUG 14, 2025, https://www.businesswire.com/news/home/20250814589664/en/Daiichi-Sankyo-Presents-New-Data-in-Small-Cell-Lung-Cancer-and-Updates-Across-ADC-Portfolio-Highlighting-Progress-in-Creating-New-Standards-of-Care-for-Patients-with-Lung-Cancer-at-WCLC [SID1234655325]).

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

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

                  Schedule Your 30 min Free Demo!

Data at WCLC will showcase the company’s progress towards creating new standards of care for patients with lung cancer, including a late-breaking oral presentation featuring the primary analysis from the dose optimization and dose expansion parts of the IDeate-Lung01 phase 2 trial (OA06.03) of ifinatamab deruxtecan (I-DXd) in patients with pretreated extensive-stage small cell lung cancer (ES-SCLC). Interim data from the dose optimization part of the trial was previously presented at 2024 WCLC.

"The late-breaking data at WCLC continues to demonstrate the potential of ifinatamab deruxtecan to become a first-in-class B7-H3 directed antibody drug conjugate for patients with pretreated extensive-stage small cell lung cancer, where treatment options following platinum-based chemotherapies are limited," said Ken Takeshita, MD, Global Head, R&D, Daiichi Sankyo. "These data along with other important updates across our portfolio continue to demonstrate how our DXd antibody drug conjugate technology is being leveraged to create new medicines for patients with cancer."

Additional data updates at WCLC include an oral presentation featuring a retrospective analysis of the intracranial efficacy of DATROWAY (datopotamab deruxtecan) or docetaxel in patients with non-small cell lung cancer (NSCLC) and baseline brain metastases in the TROPION-Lung01 phase 3 trial (0A10.01), and a poster presentation of the final results of the DESTINY-Lung05 phase 2 trial (P2.10.12) of ENHERTU (trastuzumab deruxtecan) in patients from China with previously treated HER2 mutant NSCLC.

Trials-in-Progress Across DXd ADC Portfolio

Several trials-in-progress poster presentations at WCLC further highlight the Daiichi Sankyo R&D strategy of continuing to expand the DXd ADC portfolio to address a broad spectrum of unmet needs for patients with lung cancer, including the DESTINY-Lung06 phase 3 trial evaluating the efficacy and safety of ENHERTU plus pembrolizumab versus platinum-based chemotherapy plus pembrolizumab as a first-line treatment strategy for patients with HER2 overexpressing, PD-L1 TPS <50% metastatic NSCLC.

Other early phase trials evaluating additional treatment strategies in lung cancer include the KEYMAKER-U01 substudy 01G phase 2 trial evaluating patritumab deruxtecan (HER3-DXd) in combination with pembrolizumab with or without platinum chemotherapy in patients with previously untreated stage 4 NSCLC, and a phase 1b/2 trial evaluating ifinatamab deruxtecan and gocatamig, a DLL3 targeted T-cell engager, in patients with relapsed or refractory ES-SCLC will be highlighted.

Investor Briefing Following WCLC

Daiichi Sankyo will hold a virtual conference call for investors on Wednesday, September 17, 2025 from 7:00 to 8:15 pm EDT / Thursday, September 18 from 8:00 to 9:15 am JST. Executives from Daiichi Sankyo will provide an overview of the late-breaking WCLC data and clinical development plan for ifinatamab deruxtecan.

WCLC Presentation Overview

Presentation Title

Presenter

Abstract

Presentation (CEST)

Ifinatamab Deruxtecan (I-DXd)

Ifinatamab deruxtecan (I-DXd) in extensive-stage small cell lung cancer: primary analysis of the phase 2 IDeate-Lung01 study

M. Ahn

OA06.03

Oral Presentation

Sunday, September 7

4:45– 6:00 pm

A phase 1b/2 study of gocatamig and ifinatamab deruxtecan for relapsed or refractory extensive-stage small cell lung cancer

P. Rocha

P3.18.73

Poster Presentation

Tuesday, September 9

10:00 – 11:30 am

ENHERTU (trastuzumab deruxtecan; T-DXd)

Trastuzumab deruxtecan in patients from China with pretreated HER2 mutant NSCLC: final results from the DESTINY-Lung05 study

B. Wang

P2.10.12

Poster Session

Monday, September 8

10:30 am – 12:00 pm

Trastuzumab deruxtecan + pembrolizumab as first-line treatment in HER2 overexpressing, PD-L1 TPS <50% NSCLC (DESTINY-Lung06)

W.N. William

P3.18.58

Poster Session

Tuesday, September 9

10:00 – 11:30 am

Concordance of HER2 protein overexpression by IHC and ERBB2 gene amplification by NGS in lung cancer

S. Yeramaneni

P1.17.22

Poster Session

Sunday, September 7

10:30 am – 12:00 pm

DATROWAY (datopotamab deruxtecan; Dato-DXd)

Intracranial efficacy of datopotamab deruxtecan (Dato- DXd) in patients with advanced/metastatic NSCLC in TROPION-Lung01

E. Pons-Tostivint

OA10.01

Oral Presentation

Monday, September 8

3:30 – 4:45 pm

Real world assessment of TROP2-NMR by quantitative continuous scoring (QCS) in non-small cell lung carcinoma (NSCLC)

F. Lopez-Rios

OA09.03

Oral Presentation

Monday, September 8

3:30 – 4:45 pm

Patritumab Deruxtecan (HER3-DXd)

KEYMAKER-U01 substudy 01G: pembrolizumab + patritumab deruxtecan ± chemotherapy in previously untreated stage IV NSCLC

V. Velcheti

P3.18.28

Poster Presentation

Tuesday, September 9

10:00 – 11:30 am

About the ADC Portfolio of Daiichi Sankyo

The Daiichi Sankyo ADC portfolio consists of seven ADCs in clinical development crafted from two distinct ADC technology platforms discovered in-house by Daiichi Sankyo.

The ADC platform furthest in clinical development is Daiichi Sankyo’s DXd ADC Technology where each ADC consists of a monoclonal antibody attached to a number of topoisomerase I inhibitor payloads (an exatecan derivative, DXd) via tetrapeptide-based cleavable linkers. The DXd ADC portfolio currently consists of ENHERTU, a HER2 directed ADC, and DATROWAY, a TROP2 directed ADC, which are being jointly developed and commercialized globally with AstraZeneca. Patritumab deruxtecan (HER3-DXd), a HER3 directed ADC, ifinatamab deruxtecan (I-DXd), a B7-H3 directed ADC, and raludotatug deruxtecan (R-DXd), a CDH6 directed ADC, are being jointly developed and commercialized globally with Merck & Co., Inc, Rahway, NJ, USA. DS-3939, a TA-MUC1 directed ADC, is being developed by Daiichi Sankyo.

The second Daiichi Sankyo ADC platform consists of a monoclonal antibody attached to a modified pyrrolobenzodiazepine (PBD) payload. DS-9606, a CLDN6 directed PBD ADC, is the first of several planned ADCs in clinical development utilizing this platform. Ifinatamab deruxtecan, patritumab deruxtecan, raludotatug deruxtecan, DS-3939 and DS-9606 are investigational medicines that have not been approved for any indication in any country. Safety and efficacy have not been established.

On August 14, 2025 Tvardi Therapeutics, Inc. ("Tvardi") (NASDAQ: TVRD), a clinical-stage biopharmaceutical company focused on the development of novel, oral, small molecule therapies targeting STAT3 to treat fibrosis-driven diseases, reported its financial and operating results for the second quarter ended June 30, 2025, and provided a business update (Press release, Tvardi Therapeutics, AUG 14, 2025, View Source [SID1234655324]).

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

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

                  Schedule Your 30 min Free Demo!

Second Quarter 2025 Highlights:

Announced completion of enrollment in its REVERT IPF clinical trial, a Phase 2 trial of TTI-101, for patients with idiopathic pulmonary fibrosis (IPF); topline data on track for Q4 2025.
Submitted an Investigational New Drug (IND) application for its second clinical candidate, TTI-109, with the U.S. Food and Drug Administration (FDA) in June 2025.
Announced that an abstract, entitled Single Cell Transcriptomics in A Treatment Status Segregated Cohort Exposes a STAT-3-Regulated Therapeutic Gap in Idiopathic Pulmonary Fibrosis, was presented at the American Thoracic Society (ATS) 2025 Annual Conference.
Completed its merger with Cara Therapeutics, transitioning Tvardi into a publicly traded company.
Imran Alibhai, Ph.D., Chief Executive Officer of Tvardi, stated, "We are on track for topline data in the fourth quarter from our fully enrolled REVERT IPF Phase 2 clinical trial. These data will offer important additional insights into the safety and efficacy of TTI-101, and, if positive, we believe will further validate our approach of targeting STAT3, a central mediator of fibrosis, to treat patients with IPF.

"In parallel, our Phase 2 REVERT Liver Cancer trial continues to enroll patients, and we remain on track to report topline results in the first half of 2026. Prior interim data from this ongoing study demonstrated clinically meaningful activity of TTI-101 as both monotherapy and in combination with established anti-cancer agents across treatment lines.

"Importantly, we are well-financed through these potential value inflection points, and into Q4 of next year. We believe we are very well positioned to bring meaningful innovation to patients living with fibrosis driven-diseases while creating significant value for our company."

Upcoming Milestones:

Data from the company’s ongoing REVERT IPF Phase 2 clinical trial of TTI-101 anticipated in 4Q 2025
Preliminary topline data from the company’s ongoing REVERT Liver Cancer Phase 1b/2 clinical trial of TTI-101 anticipated in 1H 2026
Second Quarter 2025 Financial Results

Research and development expenses for the three months ended June 30, 2025, were $5.8 million as compared to $6.5 million for the comparable period in 2024. The decrease of $0.7 million was primarily driven by clinical, pre-clinical, and CMC costs associated with TTI-101.

General and administrative expenses for the three months ended June 30, 2025, were $3.1 million as compared to $650,000 for the comparable period in 2024. The increase of $2.4 million was primarily driven by increases in professional fees of $1.6 million, attributable to increased legal, accounting and audit fees incurred as a result of the merger. The remaining increase was attributable to increases in personnel costs, insurance costs, and other costs.

Net income for the three months ended June 30, 2025, was $4.2 million as compared to a net loss of $7.0 million for the comparable period in 2024. The improvement in net income was due primarily to a $12.8 million remeasurement gain on Tvardi’s Convertible Notes recognized in the second quarter of 2025.

Basic and diluted net income (loss) per share attributable to common shareholders for the three months ended June 30, 2025, were a net gain of $0.51 and net loss of $1.00, respectively, compared to a net loss of $2.71 on a basic and diluted basis for the comparable period in 2024.

Cash, cash equivalents and short-term investments as of June 30, 2025, were $41.0 million, as compared to $31.6 million as of December 31, 2024.

On August 14, 2025 Summit Therapeutics Inc. (NASDAQ: SMMT) ("Summit," "we," or the "Company") reported that data from the Phase III HARMONi trial featuring the novel, potential first-in-class investigational bispecific antibody, ivonescimab, will be presented as part of the Presidential Symposium at the International Association for the Study of Lung Cancer’s (IASLC) 2025 World Conference on Lung Cancer (WCLC 2025) in Barcelona on Sunday, September 7, 2025, at 8:15am CET (2:15am ET) (Press release, Summit Therapeutics, AUG 14, 2025, View Source [SID1234655323]). This is the second consecutive year ivonescimab has been featured in the WCLC Presidential Symposium.

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

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

                  Schedule Your 30 min Free Demo!

HARMONi is a multiregional, double-blinded, placebo-controlled, Phase III study sponsored by Summit evaluating ivonescimab plus platinum-doublet chemotherapy compared to placebo plus platinum-doublet chemotherapy in patients with epidermal growth factor receptor (EGFR)-mutated, locally advanced or metastatic non-squamous non-small cell lung cancer (NSCLC) who have progressed after treatment with a 3rd generation EGFR tyrosine kinase inhibitor (TKI). This is a clinical setting with a patient population where PD-1 monoclonal antibodies have previously been unsuccessful in Phase III global clinical trials in showing either a progression-free survival (PFS) or overall survival (OS) benefit.

On May 30, 2025, we announced, via a press release, topline results from our multiregional, double-blinded, placebo-controlled, Phase III study, HARMONi. At the prespecified primary data analysis, ivonescimab in combination with chemotherapy demonstrated a statistically significant and clinically meaningful improvement in progression-free survival (PFS), with a hazard ratio of 0.52 (95% CI: 0.41 – 0.66; p<0.00001). PFS was measured by blinded independent central radiology review committee (BICR) compared to placebo in combination with chemotherapy. Ivonescimab in combination with chemotherapy showed a positive trend in overall survival (OS) in the primary analysis without achieving a statistically significant benefit with a hazard ratio of 0.79 (95% CI: 0.62 – 1.01; p=0.057).

The trial results will be presented by Jonathan Goldman, MD, Professor of Medicine at UCLA in the Hematology/Oncology Division, UCLA Director of Clinical Trials in Thoracic Oncology, Associate Director of Early Drug Development, and Chair of University of California Lung Cancer Consortium.

About the WCLC 2025 Presidential Symposium Presentation

Presidential Symposium Presentation
Presentation Title: Ivonescimab vs Placebo Plus Chemo, Phase 3 in Patients with EGFR+ NSCLC Progressed with 3rd gen EGFR-TKI Treatment: HARMONi
Presenter: Jonathan Goldman, MD, Professor of Medicine at UCLA in the Hematology/Oncology Division
WCLC Presentation No.: PL02.12
Session Date & Time: Sunday, September 7, 2025, 8:15am CET (2:15am ET)

About Ivonescimab

Ivonescimab, known as SMT112 in Summit’s license territories, North America, South America, Europe, the Middle East, Africa, and Japan, and as AK112 in China and Australia, is a novel, potential first-in-class investigational bispecific antibody combining the effects of immunotherapy via a blockade of PD-1 with the anti-angiogenesis effects associated with blocking VEGF into a single molecule. Ivonescimab displays unique cooperative binding to each of its intended targets with multifold higher affinity to PD-1 when in the presence of VEGF.

This could differentiate ivonescimab as there is potentially higher expression (presence) of both PD-1 and VEGF in tumor tissue and the tumor microenvironment (TME) as compared to normal tissue in the body. Ivonescimab’s tetravalent structure (four binding sites) enables higher avidity (accumulated strength of multiple binding interactions) in the TME (Zhong, et al, SITC (Free SITC Whitepaper), 2023). This tetravalent structure, the intentional novel design of the molecule, and bringing these two targets into a single bispecific antibody with cooperative binding qualities have the potential to direct ivonescimab to the tumor tissue versus healthy tissue. The intent of this design, together with a half-life of 6 to 7 days after the first dose (Zhong, et al, SITC (Free SITC Whitepaper), 2023), is to improve upon previously established efficacy thresholds, in addition to side effects and safety profiles associated with these targets.

Ivonescimab was engineered by Akeso Inc. (HKEX Code: 9926.HK) and is currently engaged in multiple Phase III clinical trials. Over 2,800 patients have been treated with ivonescimab in clinical studies globally.

Summit began its clinical development of ivonescimab in non-small cell lung cancer (NSCLC), commencing enrollment in 2023 in two multiregional Phase III clinical trials, HARMONi and HARMONi-3. Additionally, in early 2025 the Company began enrolling clinical trial sites in the United States for HARMONi-7.

HARMONi is a Phase III clinical trial which intends to evaluate ivonescimab combined with chemotherapy compared to placebo plus chemotherapy in patients with EGFR-mutated, locally advanced or metastatic non-squamous NSCLC who have progressed after treatment with a 3rd generation EGFR TKI (e.g., osimertinib). Enrollment in HARMONi was completed in the second half of 2024, and top-line results were announced in May of 2025.

HARMONi-3 is a Phase III clinical trial which is intended to evaluate ivonescimab combined with chemotherapy compared to pembrolizumab combined with chemotherapy in patients with first-line metastatic, squamous or non-squamous NSCLC, irrespective of PD-L1 expression.

HARMONi-7 is a Phase III clinical trial which is intended to evaluate ivonescimab monotherapy compared to pembrolizumab monotherapy in patients with first-line metastatic NSCLC whose tumors have high PD-L1 expression.

In addition, Akeso has recently had positive read-outs in three single-region (China), randomized Phase III clinical trials for ivonescimab in NSCLC: HARMONi-A, HARMONi-2, and HARMONi-6.

HARMONi-A was a Phase III clinical trial which evaluated ivonescimab combined with chemotherapy compared to placebo plus chemotherapy in patients with EGFR-mutated, locally advanced or metastatic non-squamous NSCLC who have progressed after treatment with an EGFR TKI.

HARMONi-2 is a Phase III clinical trial evaluating monotherapy ivonescimab against monotherapy pembrolizumab in patients with locally advanced or metastatic NSCLC whose tumors have positive PD-L1 expression.

HARMONi-6 is a Phase III clinical trial evaluating ivonescimab in combination with platinum-based chemotherapy compared with tislelizumab, an anti-PD-1 antibody, in combination with platinum-based chemotherapy in patients with locally advanced or metastatic squamous NSCLC, irrespective of PD-L1 expression.

Ivonescimab is an investigational therapy that is not approved by any regulatory authority in Summit’s license territories, including the United States and Europe. Ivonescimab was initially approved for marketing authorization in China in May 2024. Ivonescimab was granted Fast Track designation by the US Food & Drug Administration (FDA) for the HARMONi clinical trial setting.

On August 14, 2025 Nanjing Leads Biolabs Co., Ltd. ("Leads Biolabs" or the "Company," Stock Code: 9887.HK) reported the successful completion of patient enrollment in the ongoing single-arm, pivotal registrational clinical trial (CTR20213023) for Opamtistomig (LBL-024), a novel PD-L1/4-1BB bispecific antibody (Press release, Nanjing Leads Biolabs, AUG 14, 2025, View Source [SID1234655322]).

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

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

                  Schedule Your 30 min Free Demo!

This marks the world’s first registrational clinical trial evaluating an immunotherapy monotherapy for extrapulmonary neuroendocrine carcinoma (EP-NEC). The study, led by Professor Shen Lin from Peking University Cancer Hospital and conducted across multiple medical centers, aims to evaluate the efficacy and safety of Opamtistomig in patients with advanced EP-NEC who failed at least two lines of chemotherapy.

EP-NEC is a highly aggressive, immunologically "cold" tumor. The current first-line standard treatment for advanced EP-NEC remains platinum-based chemotherapy, with an objective response rate (ORR) of 30%-50% and a median overall survival (mOS) of approximately 1 year. For patients with platinum-resistant, no established second-line standard of care exists, representing a pressing unmet need for new therapeutic options.

Dr. Charles Cai, Chief Medical Officer of Leads Biolabs, stated:" As the first PD-L1/4-1BB-targeted bispecific antibody globally to have reached pivotal single-arm registrational stage, Opamtistomig has demonstrated compelling antitumor activity and favorable safety in early trials. Completing enrollment months ahead of schedule accelerates clinical development timelines and reflects both strong confidence of investigators in the trial design and the urgent patient need for effective, safer therapies. We remain committed to advancing rigorous clinical development across multiple indications, including EP-NEC."

Dr. Xiaoqiang Kang, Founder, Chairman and CEO of Leads Biolabs, added, "This achievement was made possible by the exceptional execution of our clinical team, the dedication of investigators, and the trust of patients and their families. Every data point in this study represents hope for patients facing a devastating disease. Guided by our mission-care for life, focus on innovation, and win win cooperation-we will continue to invest in R&D to accelerate Opamtistomig’s global development and deliver transformative therapies worldwide."

About Opamtistomig

Opamtistomig (LBL-024) is a potential first-in-class bispecific antibody simultaneously targeting PD-L1 and the co-stimulatory receptor 4-1BB. It is the first 4-1BB-targeting bispecific antibody globally to reach the single arm pivotal trial stage as a monotherapy and holds promise to become the first approved treatment specifically for extrapulmonary neuroendocrine carcinoma (EP-NEC), a malignancy with significant unmet medical need.

Developed using Leads Biolabs’ proprietary X-Body bispecific platform, Opamtistomig features a 2:2 format with two binding domains each for PD-L1 and 4-1BB, and an optimized affinity ratio. This design allows Opamtistomig to both reverse PD-L1–mediated immune suppression and selectively enhance T cell activation, resulting in potent, synergistic anti-tumor effects.

In Phase I/II clinical trials in China, Opamtistomig has demonstrated promising efficacy and a favorable safety profile in patients with advanced EP-NEC, both as monotherapy and in combination with chemotherapy. The lack of a standard of care in EP-NEC supports the pursuit of accelerated approval through a single-arm pivotal study.

In recognition of its clinical potential, Opamtistomig received Breakthrough Therapy Designation (BTD) from the National Medical Products Administration (NMPA) in China (October 2024), and Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA) for neuroendocrine carcinoma (November 2024).

Notably, 4-1BB agonism can reactivate exhausted T cells and drive robust proliferation, making it particularly promising for PD-1/PD-L1-resistant or immunologically "cold" tumors. Beyond EP-NEC, Opamtistomig has been approved for clinical trial across multiple cancer types with high unmet need, including small cell lung cancer (SCLC), biliary tract cancer (BTC), ovarian cancer (OC), non-small cell lung cancer (NSCLC), esophageal squamous cell carcinoma (ESCC), hepatocellular carcinoma (HCC), gastric cancer (GC), triple-negative breast cancer (TNBC), and malignant melanoma. Encouraging clinical activity has already been observed in SCLC, BTC, OC, and other cancer types, supporting Opamtistomig’s potential as a broad-spectrum oncology therapy.

About EP-NEC

Neuroendocrine carcinoma (NEC) is a class of poorly differentiated, high-grade neuroendocrine neoplasms (NENs), which originate in the diffuse neuroendocrine cell system and can occur in many different sites. NEC can be categorized into pulmonary NEC and EP-NEC. EP-NEC exhibits similar highly aggressive and metastatic characteristics to small cell lung cancer (SCLC). Most patients are diagnosed at a later stage or already have distant metastases, resulting in rapid disease progression and a poor prognosis.

Currently, the primary first-line treatment for advanced EP-NEC involves platinum-based chemotherapy, achieving an overall response rate (ORR) of approximately 30% to 50%, with a median overall survival (mOS) of only approximately one year. Treatment options for patients with platinum-resistant are limited, and there are no established second-line standard of care for patients who progress after first-line therapy. These underscore the urgency to develop novel therapeutic approaches.

On August 14, 2025 CARsgen Therapeutics Holdings Limited (Stock Code: 2171.HK), a company focused on developing innovative CAR T-cell therapies, reported its 2025 Interim Results (Press release, Carsgen Therapeutics, AUG 14, 2025, View Source [SID1234655321]).

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

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

                  Schedule Your 30 min Free Demo!

Business Highlights

Cash and bank balances were around RMB1,261 million as of June 30, 2025. Cash and cash equivalents and deposits at the end of 2025 are expected to be not less than RMB1,100 million. We expect to have adequate cash into the 2028 excluding subsequent cash inflows.
During H1 2025, certification and regulatory filings for zevor-cel have been completed in more than 20 provinces or cities. CARsgen has received a total of 111 confirmed orders from its commercialization partner Huadong Medicine.
The Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) of China has accepted the New Drug Application (NDA) for satri-cel.
The results of satri-cel confirmatory Phase II trial in China have been published in The Lancet and at the 2025 ASCO (Free ASCO Whitepaper) Annual Meeting.
Multiple allogeneic CAR-T products are in development, covering treatment areas such as hematologic malignancies, solid tumors, and autoimmune diseases.
CARsgen introduced Zhuhai SB Xinchuang to accelerate allogeneic CAR-T development in mainland China.
Dr. Zonghai Li, Founder, Chairman of the Board, Chief Executive Officer, and Chief Scientific Officer of CARsgen Therapeutics, said, "In the first half of 2025, we made significant strides across technology innovation, product development, and commercialization. Zevor-cel sales surged, while satri-cel became the world’s first CAR-T targeting solid tumors to file an NDA. We are also advancing multiple allogeneic CAR-T therapies to enhance clinical outcomes and expand patient access."

Financial Highlights

CARsgen’s revenue was around RMB51 million for the six months ended June 30, 2025, mainly from zevor-cel, which was calculated on the basis of ex-works price, rather than end-of-market prices. Our revenue is recognized upon completion of ex-works delivery of products. Due to the inherent time cycle of CAR-T manufacturing, there is a discrepancy between the number of orders obtained from Huadong Medicine and number of ex-works deliveries. CARsgen’s gross profit was around RMB29 million for the six months ended June 30, 2025. In the commercialization stage, we are demonstrating a strong cost competitive advantage, which is mainly due to self-manufacture for plasmids and vectors with stable output and high yield per batch.

Cash and bank balances were around RMB1,261 million as of June 30, 2025, representing a decrease of around RMB218 million from around RMB1,479 million as of December 31, 2024. The decrease was mainly due to the payment of research and development expenses, administrative expenses and investment of capital expenditure. Cash and cash equivalents and deposits at the end of 2025 are expected to be not less than RMB1,100 million. We expect to have adequate cash into the 2028 excluding subsequent cash inflows.

Zevor-cel Demonstrates Rapid Sales Growth

Zevorcabtagene autoleucel (zevor-cel, R&D code: CT053) is an autologous fully human CAR T-cell product against B-cell maturation antigen (BCMA) approved by the NMPA of China for the treatment of adult patients with relapsed or refractory multiple myeloma (R/R MM) who have progressed after at least 3 prior lines of therapy (including a proteasome inhibitor and an immunomodulatory agent).

CARsgen entered into a collaboration agreement with Huadong Medicine (000963.SZ) for the commercialization of zevor-cel in mainland China. In terms of commercialization, Huadong Medicine has established a dedicated, professional, and comprehensive commercial team to promote the use of zevor-cel and has been utilizing China’s multi-layered insurance system to improve patient accessibility. During the first half of 2025, certification and regulatory filings for zevor-cel have been completed in more than 20 provinces or cities and we have received a total of 111 confirmed orders from Huadong Medicine. We anticipate that growth of sales revenue of zevor-cel will further accelerate with continuous marketing activities and broader insurance coverage.

Satri-cel NDA Accepted for Review in China

Satricabtagene autoleucel (satri-cel, R&D code: CT041) is an autologous humanized CAR T-cell product against Claudin18.2 (CLDN18.2). In June 2025, the CDE of NMPA of China has accepted the New Drug Application (NDA) for satri-cel for the treatment of Claudin18.2-positive advanced gastric/gastroesophageal junction adenocarcinoma (G/GEJA) in patients who have failed at least two prior lines of therapy. Satri-cel is the first and only CAR T-cell product globally for which an NDA submitted for the treatment of solid tumors. Satri-cel was granted Priority Review in May 2025 and Breakthrough Therapy Designation (BTD) in March 2025 by the CDE.

The results of satri-cel confirmatory Phase II trial (NCT04581473) in China have been published in The Lancet and were orally presented at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting. Satri-cel demonstrated significant progression-free survival (PFS) improvement and a clinically meaningful overall survival (OS) benefit with a manageable safety profile, compared to standard therapy.

Multiple Allogeneic CAR-T Product Candidates in Development

CARsgen has been advancing differentiated allogeneic CAR T-cell products utilizing the proprietary THANK-uCAR platform. CARsgen has recently developed the THANK-u PlusTM platform as an enhanced version of THANK-uCAR to address the potential impact of NKG2A expression levels on therapeutic efficacy of the allogeneic CAR T-cells.

CT0596 is an allogeneic BCMA-targeted CAR T-cell product utilizing THANK-u Plus platform for the treatment of R/R MM and R/R plasma cell leukemia (PCL). The investigator-initiated trials (IITs) are ongoing in China. Preliminary clinical data for CT0596 were released in May 2025 on CARsgen’s official website. Based on the preliminary safety and efficacy data, CT0596 demonstrated favorable tolerability and encouraging efficacy signals in R/R MM patients across all predefined dose levels, with CAR-T expansion observed.

In addition, there are several allogeneic CAR T-cell products under development:

KJ-C2219: Targeting CD19/CD20, for the treatment of hematologic malignancies and autoimmune diseases. An IIT for relapsed/refractory B-cell non-Hodgkin lymphoma (R/R B-NHL) has been initiated at the end of 2024. A separate IIT for systemic lupus erythematosus (SLE) and systemic sclerosis (SSc) has been initiated in the first half of 2025.
KJ-C2320: Targeting CD38, for the treatment of acute myeloid leukemia (AML). An IIT has been initiated at the end of 2024.
KJ-C2114: For the treatment of solid tumors.
KJ-C2526: Targeting NKG2DL, for the treatment of AML, other malignancies, and senescence.
On February 25, 2025, CARsgen has entered into the agreements (the "Agreements") with an investment fund (the "Investor") managed by Zhuhai Hengqin SB Xinchuang Equity Investment Management Enterprise (Limited Partnership), pursuant to which, among others, the Investor has agreed to subscribe to additional registered capital of UCARsgen Biotech Limited ("UCARsgen") at a cash consideration of RMB80,000,000, representing 8% stake of the enlarged registered capital of UCARsgen. Upon the completion of the capital increase, CARsgen’s share in UCARsgen will be diluted from 100% to 92%.

UCARsgen is a China-based new drug discovery biotechnology company focused on allogeneic CAR T-cell therapies for the treatment of hematologic malignancies. Under the Agreements, UCARsgen has secured the exclusive rights in mainland China for the research, development, manufacture, and commercialization of the following allogeneic CAR T-cell products from CARsgen: the BCMA-targeted allogeneic CAR T-cell therapy for the treatment of multiple myeloma and plasma cell leukemia and the CD19/CD20 dual-targeted allogeneic CAR T-cell therapy for the treatment of B-cell malignancies (excluding indications for the treatment of autoimmune diseases).