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The FDA has granted fast track designation to VT3989, a transcriptional enhanced associate domain (TEAD) autopalmitoylation inhibitor, for the treatment of patients with unresectable malignant nonpleural or pleural mesothelioma who have progressed on prior immune checkpoint inhibition and platinum-based chemotherapy.¹

“We are pleased to receive fast track designation from the FDA for VT3989 in this patient population, which is in desperate need of new and effective therapeutic options,” Sofie Qiao, PhD, president and chief executive officer of Vivace Therapeutics, the developer of VT3989, stated in a news release. “This designation represents another important step in our ongoing development of VT3989 and will offer key advantages as we continue on our path toward potential commercialization of this first-in-class and best-in-class therapy.”

VT3989 Pipeline Update

• The FDA granted fast track designation to VT3989, a transcriptional enhanced associate domain (TEAD) autopalmitoylation inhibitor, for the treatment of patients with unresectable malignant nonpleural or pleural mesothelioma who have progressed on prior immune checkpoint inhibition and platinum-based chemotherapy.

• VT3989 is a novel, investigational small molecule designed to inhibit palmitoylation of members of the TEAD protein family, thereby targeting the Hippo pathway.

• Preliminary efficacy findings presented from the dose-escalation portion of the ongoing phase 1/2 trial showed decreases in the sum of the target lesions from baseline among patients with both pleural and non-pleural mesothelioma, regardless of their NF2 mutation status.

What Is the Mechanism of Action of VT3989?

This novel, investigational small molecule therapeutic is designed to inhibit palmitoylation of members of the TEAD protein family, thus targeting the Hippo pathway. The efficacy and safety of VT3989 are under investigation in an ongoing phase 1/2 clinical trial (NCT04665206).

What Is the Design of the Phase 1/2 Trial?

This multicenter, open-label trial is enrolling patients with mesothelioma and/or metastatic solid tumors that are resistant to standard therapy or for which no effective standard therapy is available.² Patients need to have an ECOG performance status of 0 or 1, as well as adequate organ function.

This trial consists of 3 parts. The dose-escalation part investigated the safety of VT3989 in patients with mesothelioma or metastatic solid tumors. Patients received the agent in a 3+3 design until the maximum tolerated dose or recommended phase 2 schedule and dose are identified.

The dose-expansion part assessed the safety and preliminary antitumor activity of VT3989 at the recommended phase 2 schedule and dose in up to 6 cohorts, including patients with mesothelioma of any site origin regardless of NF2 mutation status (cohorts 1 and 2), patients with non-pleural mesothelioma (cohort 3), patients with solid tumors with clearly inactivating NF2 alterations or mutations or YAP/TAZ gene rearrangements (cohort 4), and patients with pleural mesothelioma (cohort 5).

The combination part will consist of 2 cohorts. Cohort A will enroll patients with mesothelioma who will be treated with VT3989 plus nivolumab (Opdivo) or ipilimumab (Yervoy). Cohort B will enroll patients with non–small cell lung cancer with tumors harboring EGFR exon 19 deletions or EGFR exon 21 L858R mutations who will be treated with VT3989 plus osimertinib.

VT3989 is administered orally in 25-mg, 50-mg, 100-mg, 150-mg, or 200-mg capsules over 21- or 28-day cycles.

The primary end point is the occurrence of dose-limiting toxicities and the occurrence of general toxicities. Secondary end points include tumor response; pharmacokinetic evaluations; overall survival in part 2 cohorts 3, 4, and 5; progression-free survival in part 2 cohorts 3, 4, and 5; and quality of life in part 2 cohorts 3, 4, and 5.

What Is the Efficacy of VT3989 in Patients With Mesothelioma?

Findings from the dose-escalation portion of the trial, presented at the 2023 IASLC World Conference on Lung Cancer, showed decreases in the sum of the target lesions from baseline among patients with both pleural and non-pleural mesothelioma who received the agent across dose levels and schedules.³ Responses were observed regardless of NF2 mutation status.

What Is the Safety Profile of VT3989 in Patients With Mesothelioma?

Among evaluable patients with mesothelioma (n = 44), the most common grade 1 to 4 treatment-related adverse effects included albuminuria (61.6%), proteinuria (59.1%), fatigue (31.8%), peripheral edema (29.5%), nausea (20.5%), increased alanine aminotransferase levels (13.6%), increased aspartate aminotransferase levels (13.6%), anemia (11.4%), and decreased appetite (11.4%).

References

1. Vivace Therapeutics’ VT3989 granted fast track designation by the U.S. Food and Drug Administration for the treatment of mesothelioma. News release. Vivace Therapeutics, Inc. October 8, 2025. Accessed October 8, 2025. https://www.prnewswire.com/news-releases/vivace-therapeutics-vt3989-granted-fast-track-designation-by-the-us-food-and-drug-administration-for-the-treatment-of-mesothelioma-302577269.html
2. Study to evaluate VT3989 in patients with metastatic solid tumors.ClinicalTrials.gov. Updated August 11, 2025. Accessed October 8, 2025. https://clinicaltrials.gov/study/NCT04665206
3. Yap T, Desai J, Dagogo-Jack I, et al. First-in-human phase 1 trial of VT3989, a first-in-class YAP/TEAD inhibitor in patients with advanced mesothelioma. Presented at: 2023 IASLC World Conference on Lung Cancer. September 9-12, 2023; Singapore.

China’s biopharmaceutical innovation is experiencing an unprecedented period of growth. Thanks to sustained policy support, capital investment, and expanding research capabilities, the country is rapidly increasing its reputation as a global hub of innovation. According to the WHO, in 2024 China ranked second in the world for number of registered clinical trials, and over the past decade, its new pharmaceutical and medical technology patents have nearly quadrupled.

Against this backdrop, the 28th Annual Meeting of the Chinese Society of Clinical Oncology (CSCO) was held this month in Jinan, Shandong Province. As one of the most influential academic platforms in the field, CSCO has become a key venue for showcasing China’s oncology research to the world, and this year’s meeting shined a spotlight on the latest advances in cancer prevention, diagnosis, and treatment across both research and clinical practice.

Partnering with leading experts in oncology, molecular pathology, and drug development, Illumina hosted a special session at the conference that facilitated in-depth exchanges on biomarker applications in solid tumors, cancer drug innovation, and precision medicine in the Guangdong–Hong Kong–Macao Greater Bay Area.

Advances in next-generation sequencing and multiomics

Cancer therapy is undergoing a profound transformation, as subtypes are increasingly defined on a molecular basis rather than on organs or histology. With advances in next-generation sequencing (NGS), researchers can now integrate genomic, transcriptomic, proteomic, and other layers of biological information to achieve a comprehensive view of tumors. Large-scale genomics and multiomics projects are also enriching our understanding of cancer initiation and progression, opening new possibilities for precise tumor classification and translational research.

Professor Zhang Xuchao of Guangdong Provincial People’s Hospital noted that “NGS and multiomics, as core tools in the life sciences, are enabling us to more precisely identify disease drivers and transition from single-target to multi-biomarker-driven approaches. By analyzing diseases at the molecular level, we can trace their evolutionary pathways, identify potential therapeutic targets, and accelerate the translation of scientific discoveries into clinical applications.”

From the frontiers of research to clinical practice

As clinical practice increasingly adopts NGS, improving the accessibility and efficiency of precision oncology remains a top priority for clinicians. Comprehensive genomic profiling (CGP) is a core tool for detecting cancer biomarkers and enabling a thorough analysis of genomic alterations in tumors. It helps identify variants linked to disease progression and drug sensitivity, significantly improving the accuracy of targeted therapy matching.

“China urgently needs breast cancer clinical research that is more tailored to local populations,” said Professor Cao Wenming of Zhejiang Cancer Hospital. “Systematic and highly sensitive genomics testing can guide clinical practice, significantly improve patient outcomes, and open up new therapeutic pathways.”

Professor Song Wei, director of Clinical Genomics and Molecular Pathology at UC San Diego Health, shared, “I’m deeply focused on advancing in-house genomics testing to help pathologists in classifying tumors more rapidly. CGP assays provide streamlined end-to-end workflow and integrated analytics, delivering rapid, high-quality results that meet the dual demands of speed and accuracy for in-house testing. By building local projects and databases, we are also advancing real-world research, using data to enhance both accessibility and clinical value of precision medicine.”

As NGS and multiomics technologies generate large-scale datasets, Illumina is empowering deeper analyses through bioinformatics software and AI. Recently, the UK Biobank identified 1.5 billion variants from nearly 500,000 whole genomes with Illumina software. The dataset was published in Nature and is now openly accessible to pharmaceutical partners worldwide.

Synergy between research and medicine

In China, multiomics, AI, and other advanced tools are increasingly embedded across the cancer care continuum. During a live discussion at the CSCO meeting, Professor Gao Chenyan, leading scientist at Changping Laboratory, emphasized that clinical research is a key driver of innovative drug development and the optimization of treatment pathways. Professor Ma Jun, director of the Harbin Institute of Hematology and Oncology (HIHO), elaborated: “Hematologic tumors are highly heterogeneous. Cutting-edge technologies such as NGS have greatly enhanced our ability to monitor tumor heterogeneity. NGS-based ctDNA assays offer high specificity and sensitivity, making them reliable tools for detecting minimal residual disease in B-cell lymphoma and for assessing treatment response. Driving innovation in cancer diagnosis and treatment requires close collaboration across research, clinical practice, and industry—all for the benefit of patients.”

A “pilot-first” model for innovation

The Guangdong–Hong Kong–Macao Greater Bay Area (GBA) has become an important bridge for introducing innovative diagnostic and therapeutic solutions into mainland China, thanks to a Hong Kong Department of Health measure announced in November 2020. By June 2025, the measure had covered 45 designated hospitals, introduced 51 drugs and 63 devices, and benefited tens of thousands of patients in the GBA.

Kang Wei, former managing director of the R&D-Based Pharmaceutical Association Committee, noted that “China has become an important source of drug innovation, with growing impact in international clinical trials. GBA’s biopharmaceutical industry is uniquely positioned to leverage the measure, offering an open window for applying innovative medical resources. This has attracted leading global partners to explore practical solutions and accelerate the translation of new drugs and devices from bench to bedside.”

Illumina continues to invest in research and clinical solutions to expand advanced tumor profiling and meet diverse biomarker testing needs. In August 2024, the FDA approved TruSight Oncology Comprehensive, the first United States FDA–approved distributable comprehensive genomic profiling in vitro diagnostic with pan-cancer companion diagnostic claims.

Au Siu Kie, a professor at the Hong Kong Integrated Oncology Centre and coauthor of the JCO Precision Oncology paper “Consensus Statements on Precision Oncology in the China Greater Bay Area,” also spoke about how the measure, and expert consensus, are transforming cancer care: “This year, FDA-approved products such as TSO Comprehensive are expected to become available in Hong Kong, expanding access to advanced CGP options for patients.” He added that this broadens the reach of precision medicine and helps improve the affordability of internationally approved therapies under local reimbursement systems, “which ultimately benefits more patients.”

Deepening local innovation

Illumina continues to expand local investment and strengthen customer service and support in China. As of this June, the registration of certain Illumina products by its commercial partner Berry Genomics enabled high-throughput clinical sequencing for applications in genetic disease, reproductive health, and oncology. This year, Illumina also launched its Beijing Customer Engagement Center and its official e-commerce platform on WeChat, creating a full-channel service ecosystem.

Jenny Zheng, global senior vice president and the company’s general manager of Greater China, said: “We will work hand in hand with experts at home and abroad to accelerate translation of scientific discoveries, ensuring that cutting-edge technologies truly benefit patients. As oncology research in China continues to deepen and innovative technologies gain wider adoption, the synergistic development of NGS and multiomics, combined with our localization strategy, will contribute China’s wisdom and solutions to global cancer prevention and treatment.”