On August 14, 2025 Plus Therapeutics, Inc. (Nasdaq: PSTV) ("Plus" or the "Company"), a clinical-stage pharmaceutical company developing targeted radiotherapeutics with advanced platform technologies for central nervous system (CNS) cancers, reported positive data from a retrospective analysis of the CNSide Cerebrospinal Fluid (CSF) Assay Platform at the 2025 Society for Neuro-Oncology (SNO)/American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) CNS Metastases Conference in Baltimore, Maryland (Press release, Plus Therapeutics, AUG 14, 2025, View Source [SID1234655320]).

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The presentation, titled "The Oncogenic Flip in Patients with Leptomeningeal Metastatic Disease (LMD): Longitudinal Detection in Cerebrospinal Fluid Tumor Cells (CSF-TCs) Reveals Implications for Differential Treatment of the LMD Tumor," was a retrospective, multi-center analysis of 613 CNSide assays ordered by 19 physicians from 5 institutions at 2 health systems for 218 individual patients. 74% of the patients were female and the cancers most analyzed were breast (n=105) and lung (n=65). The research was presented by Priya U. Kumthekar, M.D., Professor of Neurology and Medicine at Northwestern University.

Data Demonstrated:

CSF tumor cells detected in 67% (412/613) patients using CNSide;
66 patients underwent 2 or more CSF draws; 24 patients underwent 5 or more;
20% (13/66) of patients were found to have a flip in immunocytochemistry (ICC) detection; and
88% (58/66) of patients were found to have a flip in FISH probe detection.
"The CNSide CSF Assay Platform can be used to detect gene amplification on CSF tumor cells of patients with LM and, therefore, may provide therapeutic insights to specifically target the LM tumor," said Priya U. Kumthekar, M.D., "Further, longitudinal CSF tumor cell analysis using CNSide may provide insights to modify treatment of the LM tumor over time."

The data builds upon previously announced results, "CSF Tumor Cell (CSF-TC) Detection, Quantification and Biomarker Assessment Helps in Clinical Management of Breast Cancer and Non-Small Cell Lung Cancer Patients Having Leptomeningeal Disease," from the prospective FORESEE study, which was also presented by Dr. Kumthekar, principal investigator. The study met key primary and secondary endpoints and showed that the CNSide CSF Assay platform influenced clinical management decisions in over 90% of LM cases. Further the CNSide CSF Assay demonstrated 2.8 times the diagnostic sensitivity versus standard CSF cytology.

About CNSide Diagnostic, LLC
CNSide Diagnostics, LLC is a wholly owned subsidiary of Plus Therapeutics, Inc. that develops and commercializes proprietary laboratory-developed tests, such as CNSide, designed to identify tumor cells that have metastasized to the central nervous system in patients with carcinomas and melanomas. The CNSide CSF Assay Platform enables quantitative analysis and molecular characterization of tumor cells and circulating tumor DNA in the cerebrospinal fluid that inform and improve the management of patients with leptomeningeal metastases.

About Leptomeningeal Metastases
Leptomeningeal metastases (LM) are a rare but severe complication of advanced cancer, affecting the fluid-lined structures of the central nervous system. LM occurs in approximately 5% of patients with metastatic cancer, but the actual incidence may be higher as it can be difficult to diagnose. Postmortem studies show the frequency of LM to be around 20% or more, highlighting healthcare providers’ need for more sensitive diagnostic options.

On August 14, 2025 Inhibikase Therapeutics, Inc. (Nasdaq: IKT) ("Inhibikase" or "Company"), a clinical-stage pharmaceutical company developing therapeutics to modify the course of cardiopulmonary diseases namely, Pulmonary Arterial Hypertension ("PAH"), reported financial results for the quarter ended June 30, 2025 and highlighted recent developments (Press release, Inhibikase Therapeutics, AUG 14, 2025, View Source [SID1234655303]).

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"During our second quarter of 2025, we continued to position the Company to advance IKT-001 toward a late-stage clinical trial in PAH," said Mark Iwicki, Chief Executive Officer of Inhibikase. "We have now finalized our study protocol, and we expect to initiate our Phase 2b clinical study of IKT-001, our re-engineered prodrug of imatinib mesylate, in PAH in the second half of 2025."

Multiple studies including both Phase 2 and the Phase 3 IMPRES study previously demonstrated that imatinib mesylate ("imatinib"), an anti-proliferative tyrosine kinase inhibitor, was highly efficacious in PAH. Notably in the IMPRES study, patients that maintained 400 mg of imatinib for greater than 50% of the treatment period showed a placebo adjusted 45-meter improvement in 6-minute walk distance ("6MWD") which represents best-in-class improvements for patients afflicted by PAH. More recently, a contemporary study in the American Journal of Respiratory and Critical Care Medicine demonstrated that higher exposures of imatinib were associated with a larger improvement in total pulmonary resistance ("TPR"). The 400 mg dose of imatinib exhibited the greatest impact on TPR and, even though the majority of patients completed the study at 200 mg or less, the magnitude of the hemodynamic change for the study was noted to compare favorably with recent studies of novel therapies added to background treatment. The Company believes this supports its thesis that IKT-001 has the potential to minimize GI side effects while maximizing the highly efficacious outcomes observed at 400 mg across multiple studies.

Recent Developments:

Advancement of IKT-001 as a therapy in PAH:
During the first half of 2025, the Company evaluated potential study designs and obtained feedback from various key opinion leaders before finalizing a clinical study protocol for its forthcoming Phase 2b study, known as IMPROVE-PAH.
IMPROVE-PAH is a multi-center, randomized, double-blind, placebo-controlled study of approximately 150 PAH participants. Participants under IMPROVE-PAH will be randomized 1:1:1 to receive 300 mg IKT-001, 500 mg IKT-001, or placebo once daily for 26 weeks, in addition to stable background PAH therapy. The Company’s bioequivalence studies previously confirmed that 500 mg of IKT-001 has comparable exposure in humans to 380 mg of imatinib. The primary efficacy endpoint is change in pulmonary vascular resistance at Week 26. Secondary endpoints include 6MWD, World Health Organization functional class, and pharmacokinetics. The study protocol also includes an interim safety review for study continuance by the Data Safety Monitoring Board with at least 50 patients at 12-weeks of follow-up.
The Company expects to initiate IMPROVE-PAH in the second half of 2025.
Financial Results

Cash Position: As of June 30, 2025, cash, cash equivalents and marketable securities were $87.7 million as compared to $97.5 million as of December 31, 2024.

Net Loss: Net loss for the quarter ended June 30, 2025, was $9.9 million, or $0.11 per share, compared to a net loss of $5.0 million, or $0.66 per share in the quarter ended June 30, 2024. Net loss for the six months ended June 30, 2025, was $23.6 million, or $0.26 per share, compared to a net loss of $9.6 million, or $1.38 per share, for the six months ended June 30, 2024.

R&D Expenses: Research and development expenses were $5.3 million for the quarter ended June 30, 2025, compared to $3.1 million for the quarter ended June 30, 2024. Research and development expenses were $15.8 million for the six months ended June 30, 2025, which includes a non-cash write-off of in-process research and development of $7.4 million and $1.0 million of stock-based compensation expense, both associated with the Company’s acquisition of CorHepta in February 2025, compared to $5.8 million for the six months ended June 30, 2024.

SG&A Expenses: Selling, general and administrative expenses for the quarter ended June 30, 2025 were $5.9 million, compared to $2.0 million for the quarter ended June 30, 2024. Selling, general and administrative expenses for the six months ended June 30, 2025 were $11.2 million, which includes $1.0 million of severance expenses resulting from the transition of senior executives in the Company during the year, compared to $4.0 million for the six months ended June 30, 2024.

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]).

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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).

On August 14, 2025 IO Biotech (Nasdaq: IOBT), a clinical-stage biopharmaceutical company developing novel, immune-modulatory, off-the-shelf therapeutic cancer vaccines, reported financial results and business highlights for the second quarter of 2025 (Press release, IO Biotech, AUG 14, 2025, View Source [SID1234655304]).

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"In the second quarter, we achieved a significant milestone advancing our pipeline of novel cancer therapies with the readout of the topline results of the Phase 3 trial for Cylembio, our potentially first-in-class, immune-modulatory, off-the-shelf therapeutic cancer vaccine, being investigated in advanced melanoma," said Mai-Britt Zocca, PhD, President and CEO of IO Biotech. "With the notable safety profile and clinical improvement observed in this trial, we are focused on discussing the results with the FDA and determining the next steps for a potential submission of a Biologics License Application (BLA) for the treatment of advanced melanoma."

Recent Business Highlights

•
The company announced topline results of its Phase 3 pivotal trial (IOB-013/KN-D18) evaluating the company’s lead investigational vaccine, Cylembio (imsapepimut and etimupepimut, adjuvanted), in combination with Merck’s (known as MSD outside of the United States and Canada) anti-PD-1 therapy KEYTRUDA (pembrolizumab) for the treatment of advanced melanoma. The trial evaluated Cylembio in combination with pembrolizumab vs. pembrolizumab alone as a first-line treatment in 407 patients with unresectable or metastatic (advanced) melanoma. In the study, Cylembio plus pembrolizumab demonstrated clinical improvement in progression free survival (PFS) compared to pembrolizumab alone, but statistical significance was narrowly missed on the PFS primary endpoint. Based on these results, IO Biotech plans to meet with the United States (US) Food and Drug Administration (FDA) this fall to discuss the totality of data and determine next steps for a potential submission of a Biologics License Application (BLA) for the treatment of advanced melanoma.

•
The company continues to expect initial data from the perioperative Phase 2 solid tumor basket trial (IOB-032/PN-E40), studying treatment with Cylembio in combination with pembrolizumab in patients with resectable squamous cell carcinoma of the head and neck (SCCHN) and melanoma that completed enrollment in January, as well as longer-term data from the company’s other ongoing Phase 2 basket trial, IOB-022/KN-D38, in patients with advanced SCCHN or non-small cell lung cancer (NSCLC), to be available in the second half of 2025 and presented at a medical meeting in 2026.

Upcoming Investor Conferences Participation

•
Morgan Stanley 23rd Annual Global Healthcare Conference: Mai-Britt Zocca, PhD, President and CEO, Amy Sullivan, CFO, and Qasim Ahmad, MD, CMO, will participate in a fireside chat beginning at 7:45 AM ET on September 9, 2025.

•
H.C. Wainwright 27th Annual Global Investment Conference: Mai-Britt Zocca, PhD, President and CEO, will give a company presentation beginning at 8:00 AM ET on September 10, 2025.

The webcasts for these two upcoming conferences will be available from the Investors section of the company’s website at View Source

Second Quarter 2025 Financial Results

•
Net loss for the three months ended June 30, 2025, was $26.2 million, compared to $20.7 million for the three months ended June 30, 2024.

•
Research and development expenses were $16.7 million for the three months ended June 30, 2025, compared to $15.8 million for the three months ended June 30, 2024. The company recognized $0.6 million in research and development equity-based compensation for the three months ended June 30, 2025, compared to $0.7 million for the three months ended June 30, 2024.

•
General and administrative expenses were $6.5 million for the three months ended June 30, 2025, compared to $5.7 million for the three months ended June 30, 2024. The company recognized $1.0 million in general and administrative equity-based compensation for the three months ended June 30, 2025 and 2024, respectively.

•
Cash and cash equivalents as of June 30, 2025 were $28.1 million, compared to $60.0 million at December 31, 2024. During the three months ended June 30, 2025, the company used cash, cash equivalents and restricted cash of $9.0 million. The company has drawn down on the Tranche A and Tranche B loan facility in principal amount of €10.0 million and €12.5 million, respectively, on May 6, 2025 and July 4, 2025, each before payment of certain fees and transaction related expense. With the funds received from the second tranche of the EIB loan facility on July 4th, the company expects that it will have sufficient cash to run the company into the first quarter of 2026.

About Cylembio

Cylembio (imsapepimut and etimupepimut, adjuvanted) is an investigational, immune-modulatory, off-the-shelf therapeutic cancer vaccine candidate designed to kill both tumor cells and immune-suppressive cells in the tumor microenvironment (TME) by stimulating activation and expansion of T cells against indoleamine 2,3-dioxygenase 1 (IDO1) positive and/or programmed death-ligand 1 (PD-L1) positive cells. The company is currently conducting a pivotal Phase 3 trial (IOB-013/KN-D18; NCT05155254) investigating Cylembio in combination with Merck’s anti-PD-1 therapy, KEYTRUDA (pembrolizumab) versus pembrolizumab alone in patients with advanced melanoma, a Phase 2 basket trial (IOB-022/KN-D38; NCT05077709) investigating Cylembio in combination with pembrolizumab as first line treatment in patients with advanced solid tumors, and a Phase 2 basket trial (IOB-032/PN-E40; NCT05280314) investigating Cylembio in combination with pembrolizumab as neo-adjuvant/adjuvant treatment of patients with solid tumors. Enrollment in the Phase 3 trial was completed rapidly by December 2023 with topline results from this trial reported in the third quarter of 2025. Enrollment in the two ongoing company-sponsored Phase 2 clinical trials is now complete.

The clinical trials are sponsored by IO Biotech and conducted in collaboration with Merck, which is supplying pembrolizumab. IO Biotech maintains global commercial rights to Cylembio.

Cylembio is a registered trademark of IO Biotech ApS, a subsidiary of IO Biotech.

KEYTRUDA is a registered trademark of Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc., Rahway, NJ, USA (known as MSD outside of the US and Canada).

About the IOB-013/KN-D18 Pivotal Phase 3 Clinical Trial

IOB-013/KN-D18 (ClinicalTrials.gov: NCT05155254) is an open label, randomized Phase 3 pivotal clinical trial evaluating Cylembio in combination with Merck’s anti-PD-1 therapy, KEYTRUDA (pembrolizumab) versus pembrolizumab alone in patients with previously untreated, unresectable or metastatic (advanced) melanoma. Enrollment in the trial was completed by December 2023 with a total of 407 patients enrolled from more than 100 centers across the United States, Europe, Australia, Turkey, Israel and South Africa. The primary endpoint of the study was progression free survival. Secondary endpoints include overall response rate, overall survival, durable objective response rate, complete response rate, duration of response, time to complete response, disease control rate, and incidence of adverse events and serious adverse events (safety and tolerability). Biomarkers in the blood and tumor tissue will also be assessed as exploratory endpoints. The company reported topline results from this trial in the third quarter of 2025. IO Biotech is sponsoring the Phase 3 trial and Merck is supplying pembrolizumab.

About IOB-022/KN-D38 Phase 2 Solid Tumor Basket Trial

IOB-022/KN-D38 (NCT05077709) is a non-comparative, open label trial to investigate the safety and efficacy of Cylembio in combination with Merck’s anti-PD-1 therapy, KEYTRUDA (pembrolizumab) in the first-line treatment of metastatic non-small cell lung cancer (NSCLC) or metastatic squamous cell carcinoma of the head and neck (SCCHN) at sites in the United States, Spain, and the United Kingdom. IO Biotech is sponsoring the Phase 2 trial and Merck is supplying pembrolizumab.

About IOB-032/PN-E40 Phase 2 Solid Tumor Basket Trial

IOB-032/PN-E40 (NCT05280314) is a multicenter Phase 2 basket trial investigating Cylembio in combination with Merck’s anti-PD-1 therapy, KEYTRUDA (pembrolizumab) as neo-adjuvant/adjuvant treatment of patients with solid tumors at sites in Australia, the United States, France, Germany, Spain, and Denmark. The study completed enrollment in all cohorts: 18 patients with melanoma in cohort A and 16 patients with SCCHN in cohort B, both as single arm cohorts receiving combination of Cylembio with pembrolizumab. In cohort C, 61 melanoma patients were randomized 1:1 to either the combination of Cylembio with pembrolizumab or pembrolizumab alone. In the neo-adjuvant period, for all cohorts, treatment is every 3 weeks (Q3W) for 3 cycles (melanoma) or 2-3 cycles (SCCHN). Patients entering the study will be scheduled for surgery and begin neoadjuvant treatment 4-9 weeks prior. Surgery will be followed by adjuvant treatment with the same regimen for 15 cycles. Cohort C patients with poor pathological response to pembrolizumab alone in the neo-adjuvant phase (>10% residual viable tumor) may cross over to combination treatment post-surgery. The primary endpoint is major pathological response at surgery (≤10% residual viable tumor; central assessment). IO Biotech is sponsoring the Phase 2 trial and Merck is supplying pembrolizumab.

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]).

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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

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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).