On October 20, 2025 Sichuan Kelun-Biotech Biopharmaceutical Co., Ltd. (the "Company") reported that the results from the Phase III registrational clinical study OptiTROP-Lung04—led by Professor Zhang Li’s team from Sun Yat-sen University Cancer Center—were published in the New England Journal of Medicine (Impact Factor = 78.5). The study evaluated the efficacy and safety of the TROP2 antibody-drug conjugate (ADC) sacituzumab tirumotecan (sac-TMT) monotherapy versus pemetrexed plus platinum chemotherapy for the treatment of patients with epidermal growth factor receptor (EGFR)-mutant locally advanced or metastatic non-squamous non-small cell lung cancer (NSCLC) who have progressed failed after treatment with EGFR-tyrosine kinase inhibitor (TKI) therapy. The study was also selected as a Late-Breaking Abstract (LBA) at the 2025 European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress and was presented as an oral report in the Presidential Symposium (Presentation # LBA5).

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OptiTROP-Lung04 is a randomized, open-label, multicenter Phase III study designed to evaluate the efficacy and safety of sac-TMT monotherapy versus pemetrexed plus platinum in patients with EGFR-mutated locally advanced or metastatic NSCLC who had progressed on prior EGFR-TKI therapy. Eligible patients were those with histologically and/or cytologically confirmed disease. The primary endpoint was Progression-Free Survival (PFS) assessed by a Blinded Independent Review Committee (BIRC) per RECIST v1.1, and the key secondary endpoint was overall survival (OS). Results showed that compared with platinum-based doublet chemotherapy, sac-TMT monotherapy demonstrated a statistically significant and clinically meaningful improvement in both PFS and OS. Consistent PFS and OS benefits were observed across all prespecified subgroups, including prior EGFR-TKI therapy, presence of liver or brain metastases, and EGFR mutation subtype.

Based on the positive results of the OptiTROP-Lung04 study, sac-TMT has been approved by the National Medical Products Administration (NMPA) in China for the treatment of adult patients with EGFR mutation-positive locally advanced or metastatic non-squamous NSCLC who have progressed after EGFR-TKI therapy. To date, sac-TMT monotherapy remains the only approved treatment option for advanced EGFR-mutant NSCLC patients who have progressed after prior TKI therapy, as well as those who have progressed after prior TKI and platinum-based chemotherapy (either concurrently or sequentially). This achievement provides comprehensive coverage for the entire population of TKI-resistant patients.

Dr. Michael Ge, Chief Executive Officer of Kelun-Biotech, commented: "OptiTROP-Lung04 is a key study that propels sac-TMT from late-line to an earlier-line treatment-setting in lung cancer, achieving a significant improvement in overall survival. We are thrilled that the results have been published in The New England Journal of Medicine, allowing this important finding to be shared widely among medical professionals and providing a reference for future lung cancer research. Together with our partner MSD, we will continue to advance sac-TMT’s clinical development and regulatory approvals, covering broader lung cancer and other indications, with the goal of making sac-TMT a cornerstone therapy for the benefit of patients."

Professor Shengxiang Ren from the Department of Oncology at Shanghai Pulmonary Hospital, Tongji University, stated: "The application of third-generation EGFR-TKIs has significantly improved the overall prognosis for patients with advanced EGFR-mutant NSCLC. However, drug resistance is almost inevitable. Traditional treatment after resistance involves platinum-based doublet chemotherapy, which offers limited overall benefit. In recent years, regimens of chemotherapy plus immune checkpoint inhibitors+anti-angiogenic agents, or EGFR/c-Met bsAb, or PD-1/VEGF bsAb, have been successively approved for this indication. However, all these approaches are chemotherapy-based, highlighting an urgent need to explore novel treatment regimens. The OptiTROP-Lung04 study confirms sac-TMT as the first treatment option as a monotherapy to deliver clear dual benefits in both PFS and OS following EGFR-TKI progression. The sequential application of sac-TMT prior to chemotherapy demonstrates strong competence, establishing itself as a new standard of care for patients with advanced EGFR-mutant lung cancer after TKI resistance."

About Sac-TMT

Sac-TMT, a core product of the Company, is a novel human TROP2 ADC in which the Company has proprietary intellectual property rights, targeting advanced solid tumors such as NSCLC, breast cancer (BC), gastric cancer (GC), gynecological tumors, among others. Sac-TMT is developed with a novel linker to conjugate the payload, a belotecan-derivative topoisomerase I inhibitor with a drug-to-antibody-ratio (DAR) of 7.4. Sac-TMT specifically recognizes TROP2 on the surface of tumor cells by recombinant anti-TROP2 humanized monoclonal antibodies, which is then endocytosed by tumor cells and releases the payload KL610023 intracellularly. KL610023, as a topoisomerase I inhibitor, induces DNA damage to tumor cells, which in turn leads to cell-cycle arrest and apoptosis. In addition, it also releases KL610023 in the tumor microenvironment. Given that KL610023 is membrane permeable, it can enable a bystander effect, or in other words kill adjacent tumor cells.

In May 2022, the Company licensed the exclusive rights to MSD (the tradename of Merck & Co., Inc., Rahway, NJ, USA) to develop, use, manufacture and commercialize sac-TMT in all territories outside of Greater China (includes Mainland China, Hong Kong, Macau, and Taiwan).

To date, three indications for sac-TMT have been approved and marketed in China for the treatment of adult patients with unresectable locally advanced or metastatic triple negative breast cancer (TNBC) who have received at least two prior systemic therapies (at least one of them for advanced or metastatic setting), EGFR mutation-positive locally advanced or metastatic non-squamous NSCLC following progression on EGFR-TKI therapy and platinum-based chemotherapy and EGFR mutant-positive locally advanced or metastatic non-squamous NSCLC who progressed after treatment with EGFR-TKI therapy. Sac-TMT is the first TROP2 ADC drug approved for marketing in lung cancer globally. In addition, the new indication application for sac-TMT for the treatment of adult patients with unresectable locally advanced, metastatic hormone receptor positive (HR+) and human epidermal growth factor receptor 2-negative (HER2-) BC who have received prior endocrine therapy and other systemic treatments in the advanced or metastatic setting was accepted by the Center for Drug Evaluation of the NMPA, and was included in the priority review and approval process.

As of today, the Company has initiated 9 registrational clinical studies in China. MSD has initiated 15 ongoing Phase Ⅲ global clinical studies of sac-TMT as a monotherapy or with pembrolizumab1 or other anti-cancer agents for several types of cancer. These studies are sponsored and led by MSD.

(Press release, Kelun, OCT 20, 2025, View Source [SID1234656832])

On October 20, 2025 SunRock Biopharma, a biotechnology company focused on developing next-generation therapeutic antibodies, and Chime Biologics, a global leading contract development and manufacturing organization (CDMO), reported a strategic collaboration for the development of SRB5, a novel anti-CCR9 monoclonal antibody targeting inflammatory bowel disease (IBD), with potential expansion into other immune-mediated inflammatory indications.

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SRB5 is a humanized monoclonal antibody with enhanced ADCC properties, designed to selectively deplete CCR9+ cells involved in chronic intestinal inflammation. It is the latest advancement in SunRock’s anti-CCR9 platform and represents a significant advance in a selective therapeutic approach for treating diseases such as Crohn’s disease and ulcerative colitis. Through this partnership, SunRock will benefit from Chime Biologics’ global-standard GMP manufacturing platform to scale production and accelerate preclinical and regulatory milestones.

"We are proud to collaborate with SunRock Biopharma on this promising anti-CCR9 antibody (SRB5) for inflammatory bowel disease, a condition that significantly impacts patients worldwide. This partnership reflects our commitment to applying our global-standard quality and comprehensive experiences in manufacturing biologics to accelerate the development of innovative therapies. We look forward to contributing to the long-term mission of SunRock Biopharma to develop innovative antibodies against highly invasive tumors with an urgent clinical need in oncology," said Dr. Jimmy Wei, President of Chime Biologics.

Dr. Laureano Simón, CEO of SunRock Biopharma, added "This collaboration with Chime Biologics reinforces our strategy of working with world-class partners across every stage of development. Chime’s technical expertise and industrial quality standards will help us scale SRB5 under optimal conditions. SRB5 has a distinctive biological profile that could transform the therapeutic landscape in IBD and potentially in highly aggressive CCR9-expressing tumors. This agreement is a critical step toward clinical validation."

(Press release, SunRock Biopharma, OCT 20, 2025, View Source [SID1234656848])

On October 20, 2025 Leap Therapeutics, Inc. (Nasdaq:LPTX), a biotechnology company focused on developing targeted and immuno-oncology therapeutics, reported final results from Part B of the DeFianCe study (NCT05480306), a Phase 2 study of sirexatamab (DKN-01), an anti-DKK1 monoclonal antibody, in combination with bevacizumab and chemotherapy (Sirexatamab Arm) compared to bevacizumab and chemotherapy (Control Arm) in patients with microsatellite stable (MSS) colorectal cancer (CRC) who have received one prior systemic therapy for advanced disease. The final clinical results were presented on behalf of the DeFianCe study investigators by Zev Wainberg, MD, Professor of Medicine and Co-Director of the GI Oncology Program at UCLA in a Mini Oral session at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress 2025 in Berlin, Germany.

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"Circulating DKK1 is a negative prognostic factor and elevated in patients with advanced, metastatic CRC. The data presented at ESMO (Free ESMO Whitepaper) demonstrate that sirexatamab, which binds to and removes free DKK1, has significant potential to provide a survival benefit for CRC patients who have high DKK1 levels and who are likely to have poor outcomes receiving the current standard of care alone," said Dr. Wainberg. "Sirexatamab has the potential to be a valuable addition to the CRC treatment paradigm as a targeted therapeutic for patients with high DKK1 and should move forward to be evaluated in a biomarker-focused registrational trial."

The DeFianCe study was a two part, open-label, multi-country study. Part A of the DeFianCe study enrolled 33 patients, including a significant number of patients who had early progression on first-line therapy, previous exposure to bevacizumab, tumors with RAS mutations, or liver and lung metastases. The study expanded into a 188 patient Part B randomized controlled trial. The primary objective of the study was progression-free survival PFS. Secondary objectives included objective response rate (ORR), duration of response, and overall survival (OS). A key pre-defined exploratory population was those patients who had high levels of circulating DKK1, as measured by a biomarker assay.

Key Part B DeFianCe Study Findings:

· Across the DKK1-high (upper median) patients (n=88):
o ORR was 38.0% in the Sirexatamab Arm compared to 23.7% ORR in the Control Arm.
o mPFS was 9.03 months in the Sirexatamab Arm compared to 7.06 months in the Control Arm, Hazard Ratio (HR) 0.61, p-value = 0.0255.
o mOS was not reached in the Sirexatamab Arm compared to 14.39 months in the Control Arm, HR 0.42, p-value = 0.0118.

· Across the DKK1-high (upper quartile) patients (n=44):
o ORR was 44.0% in the Sirexatamab Arm compared to 15.8% ORR in the Control Arm.
o mPFS was 9.36 months in the Sirexatamab Arm compared to 5.88 months in the Control Arm, HR 0.46, p-value = 0.0168.
o mOS was not reached in the Sirexatamab Arm compared to 9.66 months in the Control Arm, HR 0.17, p-value < 0.001.

· In the full intent-to-treat population (n=188):
o ORR was 35.1% in the Sirexatamab Arm compared to 26.6% ORR in the Control Arm.
o mPFS was 9.2 months in the Sirexatamab Arm compared to 8.3 months in the Control Arm, HR 0.84, p-value = 0.1712.
o Event-free rate favors Sirexatamab Arm beginning at month 9 (53 vs 47%) with further separation at month 12 (34 vs 23%).

· Sirexatamab, in combination with chemotherapy and bevacizumab, was safe and well tolerated
o Overall treatment-emergent adverse effects (TEAE) profile was similar between the Sirextamab and Control Arms, suggesting sirexatamab did not impact the safety profile when combined with the standard of care.

"The DeFianCe study results demonstrate the significant potential of sirexatamab in patients with advanced CRC. Patients with this aggressive cancer, particularly those with high DKK1 levels, have poor overall survival outcomes and few promising second-line or later options," said Douglas E. Onsi, President and Chief Executive Officer of Leap. "Sirexatamab has repeatedly demonstrated its potential as a novel, first-in-class antibody targeting DKK1 that provides deep and durable benefit for patients in desperate need of new therapies. With support from a recently completed financing, Leap plans to engage with regulatory authorities over the registrational path for sirexatamab in CRC and to optimize the DKK1 biomarker diagnostic test that could be used to identify these CRC patients with poor prognosis."

(Press release, Leap Therapeutics, OCT 20, 2025, View Source [SID1234656817])

On October 20, 2025 Remegen reported a Phase III clinical study on disitamab vedotin plus toripalimab versus chemotherapy as first-line treatment for HER2-expressing locally advanced or metastatic urothelial carcinoma (RC48-C016) was presented at the Presidential Symposium by Professor Jun Guo from Beijing Cancer Hospital at the 2025 European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress. It’s the first time a research led by Chinese scholars in the field of urological oncology has ever been selected in this honorable session. The full manuscript was simultaneously published online in The New England Journal of Medicine (NEJM). This marks the first time that the results of a Chinese evidence-based medical research in the field of urothelial carcinoma have been recognized by both an authoritative international academic conference and a top-tier journal.

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The results showed that the RC48-C016 study met the dual primary endpoints of Progression-Free Survival (PFS) and Overall Survival (OS). The Blinded Independent Central Review (BICR)-assessed median PFS reached 13.1 months, and the median OS reached 31.5 months, demonstrating both statistical and clinical significance.

The RC48-C016 trial is by now the only randomized controlled study from China providing high-level evidence for HER2-ADC as a first-line treatment of HER2-expressing (IHC 1+/2+/3+) locally advanced/metastatic urothelial carcinoma (la/mUC). The results garnered high attention and sparked lively discussion among global scholars upon release.

Comprehensive Superiority: Meeting Both PFS and OS Primary Endpoints

On the afternoon of October 19 (local time), a pivotal moment arrived at the ESMO (Free ESMO Whitepaper) Congress: Professor Guo Jun, the principal investigator of the RC48-C016 study, delivered a featured oral presentation at the Presidential Symposium, unveiling the breakthrough results globally for the first time.

The RC48-C016 study is a randomized controlled, multi-center, phase III clinical trial. It compares the efficacy and safety of disitamab vedotin combined with toripalimab versus gemcitabine combined with cisplatin or carboplatin in patients with locally advanced or metastatic urothelial carcinoma (la/mUC) who have not received systemic treatment and have HER2 expression (HER2 IHC 1+/2+/3+). The study was initiated in June 2022, with 76 clinical research centers across the country participating, and a total of 484 patients were enrolled. The dual primary endpoints of the study are PFS and OS, and the secondary endpoints include objective response rate (ORR), disease control rate (DCR), and safety, etc.

Results as of March 31, 2025, showed:

Regarding PFS, the median PFS in the disitamab vedotin combination group reached 13.1 months, more than doubling the 6.5 months in the chemotherapy group and reducing the risk of disease progression or death by 64% (Hazard Ratio [HR] =0.36, 95% CI: 0.28 – 0.46, P < 0.0001).
The OS data were equally exciting. In this interim survival analysis, the median OS in the disitamab vedotin combination group was 31.5 months, compared to 16.9 months in the platinum-based chemotherapy group. The delayed disease progression has been well translated into long-term survival benefit as the overall survival time almost doubled as compared to chemotherapy, with a 46% reduction in the risk of death (HR = 0.54, 95% CI: 0.41 – 0.73, P < 0.0001).
Regarding tumor response, the ORR assessed by BICR reached 76.1% in the disitamab vedotin combination group, far exceeding the 50.2% in the chemotherapy group. For disease control, the DCR in the disitamab vedotin combination group reached 91.4%, significantly higher than the 77.6% in the chemotherapy group.
In key subgroup analyses, significant improvements in median PFS and median OS were observed in the disitamab vedotin combination group compared to the platinum-based chemotherapy across subgroups, regardless of cisplatin eligibility, HER2 expression status, or primary tumor site.
Furthermore, the combination regimen demonstrated a better safety profile. The overall incidence of Grade ≥3 treatment-related adverse events was only 55.1% in the disitamab vedotin combination group, significantly lower than the 86.9% in the chemotherapy group.
"This is the world’s first large-scale Phase III randomized controlled clinical study in the first-line treatment of HER2-expressing Chinese mUC patients, which demonstrated that HER2-ADC combined with immunotherapy was superior to standard platinum-based chemotherapy, potentially establishing the combination therapy of disitamab vedotin and immunotherapy as the standard of care in the first-line treatment of advanced HER2-expressing mUC. This not only signifies that China’s innovative drug development is leading transformations in cancer treatment globally but also provides critical rationale for exploring similar combination strategies in treating other cancers." As Professor Guo Jun commented, the result of the RC48-C016 study confirmed that the efficacy of disitamab vedotin + toripalimab combination regimen surpassed that of traditional chemotherapy as first-line treatment for advanced mUC, potentially marking a major shift in the treatment landscape for urothelial carcinoma. It promotes innovation in Chinese diagnostic and treatment pathways while providing valuable evidence for global clinical practice.

During the subsequent expert commentary session, Professor Andrea Necchi from Italy’s IRCCS San Raffaele Hospital spoke highly of RC48-C016 study. He pointed out that the study represents a significant breakthrough in the treatment of HER2-positive urothelial carcinoma. Compared to chemotherapy, the combination therapy of disitamab vedotin and toripalimab significantly improves patients’ PFS and OS, demonstrating substantial statistical significance and clinical value, thereby offering a novel treatment option for this patient population.

Precision Treatment, Benefiting the Full Spectrum of HER2-Expressing Population (IHC 1+/2+/3+)

Urothelial carcinoma is the most common malignant tumor of the urinary system, representing a significant area of clinical unmet need.

Chinese researchers have been at the forefront of exploring HER2-ADC therapy for UC. The HER2-ADC disitamab vedotin was first approved in China in 2021 for the treatment of HER2-overexpressing (IHC 2+/3+) advanced or metastatic urothelial carcinoma, demonstrating excellent efficacy and consistent safety with that observed in prior clinical trials.

RC48-C016 study prospectively extended its clinical design to the full HER2-expressing population (IHC 1+/2+/3+). Among 765 patients tested for HER2, 632 patients had HER2-expressing characteristics (IHC 1+/2+/3+), accounting for approximately 82.6% of the tested population. The study enrolled 484 patients, who were randomized in a 1:1 ratio to groups. Stratification factors were pre-specified in the study design, primarily including cisplatin eligibility, HER2 expression status (IHC 1+ vs. IHC 2+/3+), and presence of visceral metastasis.

The results showed that the efficacy observed in the disitamab vedotin plus toripalimab regimen were consistent across all pre-specified subgroups. Regardless of HER2 expression status, cisplatin eligibility, or whether the tumor originated in the upper or lower urinary tract, disitamab vedotin combined with toripalimab significantly improved PFS and OS compared to chemotherapy, robustly demonstrating its benefit for the full demonstrating its benefit for the full HER2-expressing population (IHC 1+/2+/3+) with urothelial carcinoma.

"The significant survival benefit achieved by disitamab vedotin combined with toripalimab is not subject to the level of HER2 expression or cisplatin eligibility status, delivering benefit to populations with different characteristics," Professor Guo Jun stated, noting that its benefit for the full HER2-expressing population (IHC 1+/2+/3+) is one of the most groundbreaking findings of this study.

Disitamab Vedotin Combined with Immunotherapy, Establishing a New Benchmark in UC First-Line Treatment

Undoubtedly, RC48-C016 study is a milestone in the global first-line treatment landscape for advanced urothelial carcinoma, signifying a fundamental shift in the treatment paradigm for HER2-expressing patients:

Firstly, RC48-C016 study is the world’s first Phase III head-to-head trial to demonstrate that the combination of HER2-ADC and immunotherapy is significantly superior to traditional platinum-based chemotherapy in the first-line treatment of HER2-expressing (IHC 1+/2+/3+) advanced urothelial carcinoma. The release of its outstanding data on PFS, OS, ORR, and safety provides strong decision-making basis for clinicians, which will promote this combination to become a new first-line treatment standard for Chinese mUC patients and is expected to rewrite clinical guidelines.

Secondly, the study successfully implements the concept of precision medicine in urothelial carcinoma based on significant biomarker characteristics. By extending the beneficiary population from the traditional HER2-high (IHC 2+/3+) to the full HER2-expressing (IHC 1+/2+/3+) population, it enables over 80% of urothelial carcinoma patients to potentially benefit significantly from this treatment regimen. In the current clinical practice in China where HER2 routine detection in urothelial carcinoma has been achieved, the study provides a preferred precise treatment plan for the majority of patients and precise basis for anti-HER2 treatment, promoting a comprehensive upgrade in the global treatment concept and strategy for urothelial carcinoma and leading the continuous exploration of this combination therapy in different disease stages of urothelial carcinoma.

Thirdly, this combination regimen substantially reduces the toxicity associated with traditional platinum-based chemotherapy, significantly improving patient treatment tolerance and compliance, and providing a foundation for subsequent continuous therapy, achieving an optimized balance between tumor control, long-term survival, and quality of life.

Since its marketing four years ago, disitamab vedotin, relying on its outstanding efficacy, has achieved a progress from late-line to first-line treatment in the field of urothelial carcinoma, comprehensively breaking through the bottlenecks of traditional chemotherapy and continuously expanding the boundaries of its clinical value. The revelation of RC48-C016 study data once again demonstrates its huge clinical treatment potential.

Based on the breakthrough results of the RC48-C016 study, RemeGen Co., Ltd. submitted a New Drug Application (NDA) in China in July of this year for the new indication of disitamab vedotin combined with toripalimab for the first-line treatment of HER2-expressing locally advanced or metastatic urothelial carcinoma. This will not only address domestic clinical needs but also holds the potential to become a "Chinese Solution" influencing the global treatment landscape.

It is believed that with extended follow-up, disitamab vedotin will continue to yield clinical benefit data for broader treatment prospects.

(Press release, RemeGen, OCT 20, 2025, View Source;disitamab-vedotin-achieves-major-breakthrough-as-first-line-treatment-for-urothelial-carcinoma-302588752.html [SID1234656833])

On October 20, 2025 CatalYm, a world leader in neutralizing GDF-15 in cancer and cachexia, reported updated long-term data from its ongoing GDFATHER-1/2a trial (NCT04725474) at the European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress 2025. The data provide further evidence that Growth Differentiation Factor-15 (GDF-15) blockade by visugromab can reverse resistance to PD-(L)1 treatment in patients with advanced solid tumors and deliver durable responses in patients who have relapsed or progressed on prior checkpoint inhibitor treatment.

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Visugromab is a humanized monoclonal antibody designed to neutralize the tumor-derived cytokine GDF-15, which plays a key role in immune suppression and resistance to therapy. The updated dataset highlights visugromab’s potential to reinvigorate immune responses in difficult-to-treat tumor types and patient populations with limited treatment alternatives.

"The durability of responses we are seeing with visugromab in this heavily pretreated, late-stage population is particularly remarkable," said Prof. Dr. Ignacio Melero, Co-Director of Immunology and Immunotherapy at CIMA, Universidad de Navarra, and Principal Investigator of the trial. "To observe ongoing responses extending beyond two and sometimes even three years in patients who had progressed on prior checkpoint inhibition is both encouraging and remarkable in this setting. These data underscore the potential of GDF-15 blockade to establish immune sensitivity in resistant tumors."

Key trial results

Out of 199 patients enrolled, 77 patients with either non-squamous NSCLC (n=22), urothelial cancer (n=27) or hepatocellular carcinoma (n=28) received visugromab in combination with nivolumab, all with progression on prior anti-PD-(L)1 therapy.
Confirmed objective response rates (RECIST 1.1) were 18.2% (4/22) in non-squamous NSCLC, 18.5% (5/27) in urothelial cancer, and 14.3% (4/28) in hepatocellular carcinoma, including 5 complete responses and multiple deep partial responses across cohorts.
Median duration of response reached 32.2 months in non-squamous NSCLC, 28.8 months in urothelial cancer, and 19.4 months in hepatocellular carcinoma. At data cut-off, 53.8% (7 of 13) of responses were ongoing across all three cohorts.
In 76.9% (10/13) of responders, the duration of response on visugromab plus nivolumab exceeded that of their prior checkpoint inhibitor treatment; 61.5% of responders also achieved a deeper response than previously recorded.
The combination was well tolerated, with treatment-related adverse events (TRAEs) reported in 58.4% of patients. Most TRAEs were Grade 1 or 2 and manageable. Grade ≥3 TRAEs occurred in 13% of patients and included expected immune-related events.
In cachectic patients, treatment was associated with clinically meaningful weight gain, indicating potential quality-of-life benefit.
"Our long-term follow-up data suggest that GDF-15 blockade with visugromab may offer meaningful and sustained benefit to patients who progress or do not respond to immunotherapy," said Sujata Rao, MD, Chief Medical Officer at CatalYm. "The duration and depth of responses we continue to observe support further development of visugromab in earlier treatment lines, where maintaining immune sensitivity is critical."

"The growing body of clinical data for visugromab confirms our conviction that GDF-15 neutralization provides a novel strategy to overcome immune resistance and support immune reactivation," said Scott Clarke, Chief Executive Officer at CatalYm. "With several Phase 2b trials now underway, we are focused on translating this approach into improved outcomes across different patient populations and tumor types."

GDFATHER-1/2a (NCT04725474) is a multicenter, open-label Phase 1/2a trial evaluating visugromab in combination with an anti-PD-1 inhibitor in patients with advanced solid tumors. Phase 1 dose escalation comprised one cycle of monotherapy followed by combination treatment, while Phase 2a focused on expansion cohorts e.g. in non-squamous NSCLC, urothelial cancer, and hepatocellular carcinoma that had progressed on prior anti-PD-(L)1 therapy. Patients received visugromab at the recommended Phase 2 dose plus nivolumab every two weeks. Key endpoints included objective response rate, duration of response, and translational markers of immune activation.

(Press release, Catalym, OCT 20, 2025, View Source [SID1234656849])