Balversa (erdafitinib)

P2, N=35, Active, not recruiting, National Cancer Institute (NCI) | Trial completion date: Dec 2025 --> Jan 2027

The incidence of FGFRi-associated nail toxicities varies by agent and can affect quality of life and treatment adherence. The pathogenesis remains unclear, and no predictive biomarkers exist. Further research into optimized management and preventative strategies is needed. Early recognition and proactive multidisciplinary management are essential to minimizing complications and maintaining oncologic treatment continuity. &nbsp.

P2, N=20, Active, not recruiting, National Cancer Institute (NCI) | Trial completion date: Dec 2025 --> Dec 2026

In 21 patients who received erdafitinib after testing, the median progression-free survival is 7.5 months, and one patient with a ctDNA-exclusive FGFR alteration remained on erdafitinib for 33 months. Our results support clinical uptake of ctDNA FGFR testing in combination with tissue-based approaches in mUC.

A sonic hedgehog (SHH)-MB model, including a Gorlin syndrome patient neuroepithelial stem cell line (NES) and its tumor derivative (tNES), was used to evaluate single and combined treatments of PI3K, AKT, FGFR, and CDK4/6 inhibitors (BYL719, AZD5363, JNJ-42756493, and PD-0332991, respectively). This study illustrates that single and combined administrations of PI3K, FGFR, CDK4/6, and AKT inhibitors in a NES/tNES model have dose-dependent and additive/synergistic anti-MB activity impacting tumor growth. Their effects on tNES cells were generally more pronounced than on NES; however, the difference in proliferative capacity between the cells should be considered.

Recovery studies in spiked human serum and urine samples yielded excellent results (95.7-104.8% recovery, RSD ≤ 3.65%), validating the sensor's real-world applicability. Importantly, the sensor offers scalable fabrication, requires no toxic reducing agents, and exhibits strong selectivity and long-term stability, making it a novel and sustainable platform for therapeutic drug monitoring, pharmacokinetics, and point-of-care diagnostics.

This has broadened the treatment landscape of the disease to include novel agents, such as antibody-drug conjugates (e.g., enfortumab vedotin) and targeted therapies, including the pan-fibroblast growth factor receptor (FGFR) inhibitor erdafitinib. Equally important is the standardization and timely implementation of FGFR3 testing in clinical practice to optimize treatment planning. This review addresses key considerations in FGFR3 testing in mUC and discusses how it can be routinely incorporated into clinical practice.

Furthermore, [68Ga]Ga-DOTA-cHW8 validated FGFR1 dynamic modulation during erdafitinib treatment in CDX and PDX models, establishing its efficacy for noninvasive UM treatment response assessment. This study reports a cyclic peptide-based radiotracer, [68Ga]Ga-DOTA-cHW8, for FGFR1 PET imaging in UM. Through rational design and preclinical validation in UM models, we establish its high specificity, favorable pharmacokinetics properties, and capability to monitor FGFR1 dynamics during targeted therapy.

P2, N=11, Terminated, M.D. Anderson Cancer Center | Trial completion date: Jun 2026 --> Feb 2026 | Active, not recruiting --> Terminated | Trial primary completion date: Jun 2026 --> Feb 2026; <75% participation

Drug-gene mapping revealed candidates spanning already in clinical trials for HNC (e.g. Afatinib, Cabozantinib, Dasatinib, Brigatinib, Lenvatinib, Capivasertib, Erdafitinib) and emerging or repurposing candidates (Amuvatinib, XL765 (Voxtalisib), Golotimod, Artenimol, Quercetin, and Acetylsalicylic Acid), offering opportunities for precision repurposing...These included targeted therapies such as Fostamatinib, Nintedanib, Brigatinib, Regorafenib, and Lenvatinib, as well as emerging compounds like Artenimol, Quercetin, and Acetylsalicylic Acid (Aspirin). Through a combination of genomic analysis, network expansion, and literature validation, the GARD pipeline offers a powerful way to accelerate personalized cancer treatments while reducing cost and development time.

P2, N=239, Active, not recruiting, Janssen Research & Development, LLC | Trial completion date: Dec 2026 --> Mar 2027