SUMMARY  

• BLC2001 (NCT02365597) was a phase 2, multicenter, open-label study in evaluating the use of BALVERSA in adult patients (age ≥18 years; N=99) with locally advanced and unresectable or metastatic urothelial carcinoma (UC) and prespecified fibroblast growth factor receptor (FGFR) genetic alterations (FGFR3 mutation or FGFR2/3 fusion) who received prior chemotherapy or were chemotherapy-naïve due to cisplatin ineligibility.^1-4The median age was 68 years (range, 36-87 years) for the overall study population, with 16 patients aged ≥75 years and 83 patients aged <75 years.^1,2
  • Park et al (2019)¹ conducted a post hoc subgroup analysis of patients with high-risk disease from the BLC2001 study. The investigator-assessed objective response rate (ORR) was 50.0% (8/16; 95% confidence interval [CI], 24.7-75.3) in those aged ≥75 years. No differences in the proportion of grade 3/4 serious adverse events (AEs) were identified between age-based subgroups (eg, age ≥75 years and age <75 years).

CLINICAL DATA

Park et al (2019)¹ conducted a post hoc subgroup analysis of patients with high-risk disease, including patients aged ≥75 years, in the BLC2001 study.

Study Design/Methods

• Phase 2, multicenter, open-label study evaluating the use of BALVERSA in adult patients (N=99) with locally advanced and unresectable or metastatic UC who had prespecified FGFR genetic alterations (FGFR3 mutation or FGFR2/3 fusion).^1,2
• Patients who had progressed during or following ≥1 line of prior systemic chemotherapy or within 12 months of neoadjuvant or adjuvant chemotherapy (n=87) or were chemotherapy-naïve due to cisplatin ineligibility (n=12) were included.^1,2
• Patients had tumor tissues with one of the following FGFR3 gene mutations (R248C, S249C, G370C, Y373C) or FGFR gene fusions (FGFR3-TACC3, FGFR3-BAIAP2L1, FGFR2-BICC1, FGFR2-CASP7).⁵
• Efficacy and safety analyses were reported for patients who received an initial BALVERSA dose of 8 mg once daily, with up-titration to 9 mg once daily if the serum phosphate levels on day 14 were below the target of 5.5 mg/dL and there were no treatment-related AEs.¹

Results

Patient Characteristics

• The median age was 68 years (range, 36-87 years) for the overall study population, with 16 patients aged ≥75 years and 83 patients aged <75 years.^1,2

Efficacy

• Investigator-assessed ORR was 40.4% (95% CI, 30.7-50.7) in the primary analysis of all patients.
  • The ORR was 50.0% (8/16; 95% CI, 24.7-75.3) in patients aged ≥75 years and 38.6% (32/83; 95% CI, 28.1-49.9) in patients aged <75 years.
• The median duration of response (DOR) was 5.59 months (95% CI, 4.24-7.23) in the primary analysis of all patients.
  • The median DOR was 13.37 months (8/16; 95% CI, 2.56-13.37) in patients aged ≥75 years and 5.59 months (32/83; 95% CI, 4.21-7.00) in patients aged <75 years.
• The median progression-free survival (PFS) was 5.52 months (95% CI, 4.17-5.95) in the primary analysis of all patients.
  • The median PFS was 5.65 months (95% CI, 1.38-14.75) in patients aged ≥75 years and 5.52 months (95% CI, 4.04-6.64) in patients aged <75 years (hazard ratio [HR], 0.71; P=0.28).
• The median overall survival (OS) was 13.80 months (95% CI, 8.82-not evaluable [NE]) in the primary analysis of all patients.
  • The median OS was 14.03 months (95% CI, 3.98-NE) in patients aged ≥75 years and 12.02 months (95% CI, 8.97-NE) in patients aged <75 years (HR, 0.76; P=0.54).

Safety

• Rates of grade 3/4 serious treatment-emergent AEs, dose reduction, dose interruption, treatment discontinuation, and death in the primary analysis population and age-stratified subgroups are given in Table: Safety Analysis for the Primary Analysis Population and Age Subgroups.

Literature Search

A literature search of MEDLINE^®, Embase^®, BIOSIS Previews^®, and DERWENT Drug Files (and/or other resources, including internal/external databases) was conducted on 07 October 2024.