On July 29, 2025 Eisai Co., Ltd. (Headquarters: Tokyo, CEO: Haruo Naito, "Eisai") reported that its in-house discovered tyrosine kinase inhibitor, "LENVIMA" (generic name: lenvatinib mesylate), in combination with the anti-PD-1 antibody, pembrolizumab, and transarterial chemoembolization (TACE) has been approved by the National Medical Products Administration (NMPA) of China for unresectable, non-metastatic hepatocellular carcinoma (Press release, Eisai, JUL 29, 2025, View Source [SID1234654572]).

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This approval is based on interim analysis results from the pivotal Phase III LEAP-012 trial. The results of this trial were presented at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Annual Congress 2024 held in September 20241 and published in The Lancet in January 2025.2 In this trial, LENVIMA in combination with pembrolizumab and TACE (the "combination therapy") demonstrated a statistically significant and clinically meaningful improvement in one of the trial’s primary endpoints, progression-free survival (PFS), reducing the risk of disease progression or death by 34% (Hazard Ratio [HR]=0.66 [95% Confidence Interval (CI), 0.51-0.84]; p=0.0002) compared to TACE alone*.2 Median PFS was 14.6 months(95% CI, 12.6-16.7) in the combination therapy and 10.0 months (95% CI, 8.1-12.2) in TACE alone. 2 At this analysis, a trend toward improvement in overall survival (OS), the trial’s other primary endpoint, was observed for the combination therapy versus TACE alone (HR=0.80 [95% CI, 0.57-1.11]; p=0.087). 2

237 patients received the combination therapy and 241 patients received TACE alone.2 Treatment Emergent Adverse Events (TEAEs) occurred in 99.6% (n=236) of patients receiving the combination therapy versus 96.7% (n=233) of patients receiving TACE alone and led to the discontinuation of both study drugs in 13.1% (n=31) versus 4.1% (n=10) of patients, respectively.2 Grade 3, 4, or 5 TEAEs occurred in 82.3% (n=195) of patients receiving the combination therapy versus 47.7% (n=115) for TACE alone and TEAEs led to death in 4.2% (n=10) versus 2.5% (n=6) of patients, respectively.2

Liver cancer is one of the leading causes of cancer-related deaths worldwide.3 In 2022, it was estimated there were more than 865,000 new cases globally and 367,000 in China, with more than 757,000 deaths worldwide, including 316,000 in China.3,4 China is estimated to account for more than 40% of global new cases and deaths.3,4 Hepatocellular carcinoma (HCC) is the most common type of liver cancer, representing approximately 90% of primary liver cancer cases.5 TACE has been a standard of care for patients with unresectable, non-metastatic HCC for many years. However, since many patients experience disease progression within one year6,7,8,9, new treatment options have been sought.

LENVIMA monotherapy has been approved for the treatment of patients with unresectable HCC in more than 80 countries, including in Japan, the U.S., Europe and China and has made contributions to many patients to date. With this approval, it is expected that LENVIMA will further expand its contribution to patients with hepatocellular carcinoma in China.

Eisai positions oncology as a key therapeutic area and is aiming to discover innovative new medicines with the potential to cure cancer. Eisai is committed to expanding the potential clinical benefits of lenvatinib for cancer treatment, as it seeks to contribute to addressing the diverse needs of, and increasing the benefits provided to, patients with cancer, their families and healthcare professionals.

In March 2018, Eisai and Merck & Co., Inc., Rahway, through an affiliate, entered into a strategic collaboration for the worldwide co-development and co-commercialization of lenvatinib.

*TACE alone: In addition to TACE, oral placebo and intravenous placebo corresponding to LENVIMA and pembrolizumab were administered.

On July 28, 2025 CStone Pharmaceuticals reported it will announce the latest clinical data from its 2.0 blockbuster product, CS2009 (a PD-1/VEGF/CTLA-4 trispecific antibody), as well as the design of the global, multicenter Phase Ib study for CS5001 (a ROR1 ADC) at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper), one of the world’s most influential oncology conferences, will be held in Berlin, Germany from October 17 to 21, 2025 (Press release, CStone Pharmaceauticals, JUL 28, 2025, View Source [SID1234656217]).

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CS2009 is a potential first-in-class/best-in-class trispecific antibody. Its global multicenter Phase I dose escalation and dose expansion study is actively recruiting patients in Australia and China, and plans to expand to the United States for Phase II enrollment. CStone Pharmaceuticals will announce the Phase Ia clinical study data of CS2009 at the ESMO (Free ESMO Whitepaper) Congress , which will also become the first globalClinical data report of PD-1/VEGF/CTLA-4 trispecific antibody.
CS5001 is the first known ROR1 ADC to demonstrate clinical efficacy in both lymphoma and solid tumors, and its clinical development progress ranks among the top two globally. Currently, a global, multicenter Phase Ib clinical trial of CS5001 is progressing simultaneously in the United States, Australia, and China. CStone Pharmaceuticals will unveil the design of the ongoing Phase Ib study at the ESMO (Free ESMO Whitepaper) Congress.

The following are the key messages presented by the company at the ESMO (Free ESMO Whitepaper) conference:

CS2009, a novel PD-1/VEGF/CTLA-4 trispecific antibody, in patients with advanced solid tumors: an open-label, multicenter, phase I first-in-human study

【Category】 Investigational immunotherapy

【Format】 Poster

【Exhibition Number】 1545P

A phase Ib trial of CS5001 (ROR1-ADC) as a single agent and in combination with systemic therapies in patients with advanced solid tumours and lymphomas

【Category】 Haematological malignancies

【Format】 Electronic poster

【Exhibition Number】 1316eTiP

About CS2009 (PD-1/VEGF/CTLA-4 trispecific antibody)

CS2009 is a novel trispecific antibody targeting PD-1, VEGFA, and CTLA-4, independently developed by CStone Pharmaceuticals from the molecular design perspective. It achieves multi-dimensional anti-tumor effects through synergistic action and holds first-in-class/best-in-class potential. CS2009’s differentiated molecular design combines three clinically validated targets, enabling the reactivation of near-exhausted tumor-infiltrating T cells and demonstrating comparable VEGF neutralization to existing anti-VEGF antibodies. It targets a broad range of diseases, including but not limited to non-small cell lung cancer, liver cancer, gastric cancer, endometrial cancer, ovarian cancer, renal cell carcinoma, and cervical cancer.

About the CS5001 (ROR1 ADC)

CS5001 is an antibody-drug conjugate (ADC) targeting receptor tyrosine kinase-like orphan receptor 1 (ROR1). The drug utilizes a unique design, combining a tumor-specifically activated pyrrolobenzodiazepine (PBD) protoxin payload and a linker. This "dual-control" mechanism of linker plus protoxin effectively mitigates the toxicity issues associated with traditional PBD payloads and significantly extends the safety window. CS5001 has demonstrated complete tumor inhibition in multiple preclinical cancer models and exhibited favorable serum half-life and pharmacokinetic properties, demonstrating its significant clinical development potential and broad application prospects in the treatment of various solid and hematological tumors. Furthermore, CS5001 utilizes targeted conjugation technology to achieve a precise drug-to-antibody ratio (DAR), ensuring homogeneous production and large-scale manufacturing.

In October 2020, CStone Pharmaceuticals and LigaChem Biosciences, Inc. (LCB) entered into a licensing agreement for the development and commercialization of CS5001. CS5001 was originally co-synthesized by LCB and ABL bio, two leading Korean biotech companies. Under the terms of the agreement, CStone Pharmaceuticals obtained exclusive development and commercialization rights for CS5001 worldwide, excluding Korea.

On July 28, 2025 Deciphera Pharmaceuticals, LLC, a biopharmaceutical company focused on discovering, developing, and commercializing important new medicines, and Ono Pharmaceutical Co., Ltd. (Headquarters: Osaka, Japan; President and COO: Toichi Takino; "Ono"), reported that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending the approval of vimseltinib for the treatment of adult patients with symptomatic tenosynovial giant cell tumor (TGCT) associated with clinically relevant physical function deterioration and in whom surgical options have been exhausted or would induce unacceptable morbidity or disability (Press release, Deciphera Pharmaceuticals, JUL 28, 2025, View Source [SID1234654587]).

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The positive CHMP opinion is a scientific recommendation for marketing authorization and one of the final steps before the European Commission (EC), which has the authority to approve medicines in the European Union (EU), issues a decision on Deciphera’s marketing authorization application (MAA) for vimseltinib. This decision is expected in the second quarter of the fiscal year ending March 31, 2026.

"CHMP’s positive opinion is an important milestone for the TGCT community in the European Union (EU), where there are currently no approved treatments for TGCT, and for vimseltinib, which is now one step closer to potential EU regulatory approval," said Ryota Udagawa, President and Chief Executive Officer of Deciphera. "We look forward to building upon vimseltinib’s positive CHMP opinion as we work to bring this medicine to TGCT patients around the world in need of new treatment options."

The positive CHMP opinion is supported by compelling efficacy and safety results from the pivotal Phase 3 MOTION study of vimseltinib in patients with TGCT not amenable to surgery with no prior anti-CSF1/CSF1R therapy (prior therapy with imatinib or nilotinib allowed), compared to placebo, as well as the Phase 1/2 study of vimseltinib. In MOTION, vimseltinib demonstrated a statistically significant and clinically meaningful objective response rate (ORR) at Week 25 in the intent-to-treat (ITT) population, as assessed by blinded independent radiologic review (BIRR) per Response Evaluation Criteria in Solid Tumors version 1.1 (RECIST v1.1), versus placebo (40% in vimseltinib arm vs 0% in placebo arm, p <0.0001). The primary endpoint was supported by statistically significant and clinically meaningful improvements in active range of motion, patient-reported physical functioning, and patient-reported pain observed in the vimseltinib arm compared to the placebo arm at week 25. The safety profile of vimseltinib is manageable and consistent with results previously disclosed in the Phase 1/2 clinical trial.

About vimseltinib

Vimseltinib is an oral switch-control tyrosine kinase inhibitor specifically designed to selectively and potently inhibit CSF1R. Vimseltinib has been developed using Deciphera’s proprietary switch-control kinase inhibitor platform. ROMVIMZA (vimseltinib) is a kinase inhibitor approved in the United States for adult patients with symptomatic tenosynovial giant cell tumor (TGCT) for which surgical resection will potentially cause worsening functional limitation or severe morbidity.

About Tenosynovial Giant Cell Tumor (TGCT)

TGCT is caused by a translocation in colony-stimulating factor 1 (CSF1) gene resulting in overexpression of CSF1 and recruitment of colony-stimulating factor 1 receptor (CSF1R)-positive inflammatory cells into the lesion. TGCT is a rare, non-malignant tumor that develops inside or near joints. TGCT is caused by dysregulation of the CSF1 gene leading to overproduction of CSF1. TGCT is also known as giant cell tumor of the tendon sheath (GCT-TS) or pigmented villonodular synovitis (PVNS), a diffuse-type of TGCT. TGCT is a locally aggressive neoplasm that can grow and cause damage to surrounding tissues and structures inducing pain, swelling, and limitation of movement of the joint. Surgery is the main treatment option; however, these tumors tend to recur, particularly in diffuse-type TGCT. If untreated or if the tumor continually recurs, damage and degeneration may occur in the affected joint and surrounding tissues, which may cause significant disability. For a subset of patients, surgical resection will potentially cause worsening functional limitation or severe morbidity, systemic treatment options are limited and a new therapeutic option for TGCT is needed.

On July 28, 2025 MAIA Biotechnology, Inc. (NYSE American: MAIA) ("MAIA", the "Company"), a clinical-stage biopharmaceutical company focused on developing targeted immunotherapies for cancer, reported that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for ateganosine (THIO, 6-thio-dG or 6-thio-2’-deoxyguanosine) for the treatment of non-small cell lung cancer (NSCLC) (Press release, MAIA Biotechnology, JUL 28, 2025, View Source [SID1234654586]). Ateganosine is currently being evaluated in a pivotal Phase 2 THIO-101 clinical trial evaluating its anti-tumor activity when followed by a checkpoint inhibitor.

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Ateganosine is a first-in-class small molecule that compromises telomere structure and function in cancer cells, leading to rapid tumor cell elimination and specific immune memory. Through telomerase-mediated action, ateganosine reverses intrinsic or acquired resistance to immune checkpoint inhibitors (ICIs).

"FDA’s Fast Track Designation recognizes ateganosine’s potential as a new therapeutic paradigm in cancer treatment science. Ateganosine is the first and only anticancer treatment of its kind that we are aware of in clinical development," stated MAIA Chairman and CEO Vlad Vitoc, M.D. "If we are successful in the Fast Track regulatory pathway, ateganosine could qualify for accelerated FDA approval and robust exclusivity in NSCLC, with a potential FDA decision as early as next year. If approved, ateganosine would have a first-to-market competitive position within a $34 billion NSCLC treatment market with significant unmet medical need."

NSCLC represents one of the largest global oncology indications. The market was valued at $34.1B in 2024 and is projected to reach $68.8B by 2033 with a projected CAGR of 8.1%.1

"This is an important milestone for MAIA’s clinical development program. Ateganosine has demonstrated robust preclinical efficacy and superior clinical median overall survival compared to other FDA-approved treatments for NSCLC patients with prior disease progression on platinum-based chemotherapy and anti-PD-(L)1 antibody. Additionally, advanced NSCLC is a devastating disease that clearly meets the criteria for a serious condition with unmet medical need. Both are key criteria for the Fast Track designation," said K. Robinson Lewis, Vice President, Head of Regulatory and Quality at MAIA. "We intend to utilize the incentives of the Fast Track Program to expedite the development and review of ateganosine and bring patient access sooner."

The FDA Fast Track is a process designed to facilitate development and expedite the review of drugs for treating serious conditions and filling an unmet medical need, as in providing a therapy where none exists or which may be potentially better than available therapy. If relevant criteria are met during the Fast Track process, a drug will be eligible for FDA Accelerated Approval and Priority Review (FDA decision within six months).

MAIA’s most recent data from the pivotal Phase 2 THIO-101 clinical trial of ateganosine as of May 15, 2025 showed median overall survival (OS) of 17.8 months in a heavily pre-treated population. As of the data cut-off date, the patient with the longest survival in the trial had completed 32 cycles of therapy and had 24.3 months survival. Studies of standard-of-care chemotherapy treatments for NSCLC in a similar setting have shown overall survival of 5 to 6 months.

About Ateganosine

Ateganosine (THIO, 6-thio-dG or 6-thio-2’-deoxyguanosine) is a first-in-class investigational telomere-targeting agent currently in clinical development to evaluate its activity in non-small cell lung cancer (NSCLC). Telomeres, along with the enzyme telomerase, play a fundamental role in the survival of cancer cells and their resistance to current therapies. The modified nucleotide 6-thio-2’-deoxyguanosine induces telomerase-dependent telomeric DNA modification, DNA damage responses, and selective cancer cell death. Ateganosine-damaged telomeric fragments accumulate in cytosolic micronuclei and activates both innate (cGAS/STING) and adaptive (T-cell) immune responses. The sequential treatment of ateganosine followed by PD-(L)1 inhibitors resulted in profound and persistent tumor regression in advanced, in vivo cancer models by induction of cancer type–specific immune memory. Ateganosine is presently developed as a second or later line of treatment for NSCLC for patients that have progressed beyond the standard-of-care regimen of existing checkpoint inhibitors.

About THIO-101 Phase 2 Clinical Trial

THIO-101 is a multicenter, open-label, dose finding Phase 2 clinical trial. It is the first trial designed to evaluate ateganosine’s anti-tumor activity when followed by PD-(L)1 inhibition. The trial is testing the hypothesis that low doses of ateganosine administered prior to cemiplimab (Libtayo) will enhance and prolong immune response in patients with advanced NSCLC who previously did not respond or developed resistance and progressed after first-line treatment regimen containing another checkpoint inhibitor. The trial design has two primary objectives: (1) to evaluate the safety and tolerability of ateganosine administered as an anticancer compound and a priming immune activator (2) to assess the clinical efficacy of ateganosine using Overall Response Rate (ORR) as the primary clinical endpoint. The expansion of the study will assess overall response rates (ORR) in advanced NSCLC patients receiving third line (3L) therapy who were resistant to previous checkpoint inhibitor treatments (CPI) and chemotherapy. Treatment with ateganosine followed by cemiplimab (Libtayo) has shown an acceptable safety profile to date in a heavily pre-treated population. For more information on this Phase II trial, please visit ClinicalTrials.gov using the identifier NCT05208944.

On July 28, 2025 RenovoRx, Inc. ("RenovoRx" or the "Company") (Nasdaq: RNXT), a life sciences company developing innovative targeted oncology therapies and commercializing RenovoCath, a patented, FDA-cleared drug-delivery device, reported the launch of the PanTheR Post-Marketing Registry Study (NCT06805461) (Press release, Renovorx, JUL 28, 2025, View Source [SID1234654585]).

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The initiation of this study demonstrates RenovoRx’s commitment to innovation and RenovoCath’s current and future potential. The study will serve as a critical tool for understanding RenovoCath’s safety and effectiveness in a real-world setting, providing valuable insights into long-term effectiveness and patient outcomes. Patient enrollment is expected to commence before the end of September 2025. Each cancer center participating in the registry study will purchase RenovoCath devices for use in the study from RenovoRx.

A registry study, or post-approval study, is a clinical study that involves collecting data on the long-term use and performance of a medical device, such as RenovoCath, after it has been cleared for market by the FDA. PanTheR is a multi-center, post-marketing observational registry study designed to evaluate the long-term safety of and survival outcomes for patients diagnosed with solid tumors who are treated using RenovoCath for targeted drug delivery. PanTheR will capture real-world data on the utilization of RenovoCath and generate additional safety information across a broader range of solid tumors. This data may be used to inform future clinical trial designs.

The first of multiple clinical sites to initiate patient enrollment in the PanTheR study is the University of Vermont (UVM) Cancer Center, with Dr. Conor O’Neill, Assistant Professor at the UVM Larner College of Medicine and surgical oncologist at the UVM Medical Center, serving as Principal Investigator. Additional clinical sites in the post-marketing registry study are expected to initiate enrollment soon.

"PanTheR marks a significant step forward in our commitment to better understand and demonstrate the long-term safety and therapeutic potential of our RenovoCath device," said Leesa Gentry, Chief Clinical Officer of RenovoRx. "By collaborating with leading cancer centers across the U.S, this is a low-cost study that will yield valuable data. By gathering real-world data across diverse cancer types and clinical environments, PanTheR aims to advance innovation and inform evidence-based treatment strategies, which will ultimately enhance care and potentially improve outcomes for future patients facing solid tumors."

"We are very pleased that the UVM Cancer Center has been initiated to begin enrollment in the PanTheR study," Ms. Gentry continued. "The UVM Cancer Center offers leading-edge care, provided by highly skilled oncologists priding themselves on using the latest research and education for informed care. We believe our study will be an excellent fit within University of Vermont’s oncology program."

"We are proud to be part of this important study that holds the potential to transform the way we treat solid tumors," said Dr. Conor O’Neill of the University of Vermont Cancer Center. "I believe the RenovoCath device offers a novel approach for drug delivery, which may have the potential to improve patient outcomes. This study emphasizes our strong commitment to continually advance treatment options offered to our patients by offering access to the latest innovations that have the potential to transform the treatment paradigm for solid tumors."

To learn more about PanTheR (NCT06805461), visit clinicaltrials.gov for details: View Source;rank=1.

About RenovoCath

Based on its FDA clearance, RenovoCath is intended for the isolation of blood flow and delivery of fluids, including diagnostic and/or therapeutic agents, to selected sites in the peripheral vascular system. RenovoCath is also indicated for temporary vessel occlusion in applications including arteriography, preoperative occlusion, and chemotherapeutic drug infusion. For further information regarding our RenovoCath Instructions for Use ("IFU"), please see: IFU-10004-Rev.-G-Universal-IFU.pdf.