ESMO 2024: Final Analysis of the Phase 3 LITESPARK-005 Study of Belzutifan Versus Everolimus in Participants with Previously Treated Advanced Clear Cell RCC

(UroToday.com) The 2024 ESMO annual meeting included a session on kidney cancer, featuring a presentation by Dr. Brian Rini discussing the final analysis of the phase 3 LITESPARK-005 study of belzutifan versus everolimus in participants with previously treated advanced clear cell RCC. Belzutifan is a first-in-class oral HIF-2alpha inhibitor that blocks heterodimerization with HIF-1beta and downstream oncogenic pathways:

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Belzutifan significantly improved progression-free survival and objective response rate versus everolimus in patients with advanced clear cell RCC after immune checkpoint and antiangiogenic agents in LITESPARK-005 (NCT04195750).1 Based on these results, belzutifan was approved in the US for advanced RCC following a PD-(L)-1 inhibitor and a VEGFR-TKI. At ESMO 2024, Dr. Rini presented the final results of the LITESPARK-005 trial.

Eligible patients were adults with advanced clear cell RCC and 1–3 prior systemic regimens including ≥1 PD-(L)1 inhibitor and ≥1 VEGFR-TKI. Patients were randomized 1:1 to belzutifan 120 mg or everolimus 10 mg QD until progression or unacceptable adverse events. Progression-free survival per RECIST 1.1 by central review and overall survival were the dual primary endpoints. Objective response rate per RECIST 1.1 by central review was a key secondary endpoint. Duration of response and safety were secondary endpoints. The trial design for LITESPARK-005 is as follows:trial design for LITESPARK-005
No formal statistical testing was performed at final analysis for progression-free survival and objective response rate as these endpoints were positive at first interim analysis.

There were 746 patients allocated to belzutifan (n = 374) or everolimus (n = 372). At final analysis (data cutoff: April 15, 2024), the participant disposition in the two arms was as follows:746 patients allocated to belzutifan (n = 374) or everolimus (n = 372)
The median follow-up was 35.8 months (range 26.9–49.2), with 14.5% of patients having ongoing treatment with belzutifan versus 1.4% with everolimus. Dr. Rini highlighted the baseline characteristics in the intention to treat the population, as well as those that were still receiving ongoing treatment with belzutifan:
baseline characteristics in the intention to treat population, as well as those that were still receiving ongoing treatment with belzutifan
The progression-free survival benefit was maintained with belzutifan vs everolimus (median 5.6 months versus 5.6 months; HR 0.75; 95% CI 0.63–0.88), with an estimated progression-free survival rate at 12 months (33.7% vs 17.6%) and at 24 months (17.5% vs 4.1%) favoring belzutifan:Progression-free survival benefit was maintained with belzutifan vs everolimus
Progression-free survival across the subgroups generally showed an excellent benefit for belzutifan versus everolimus:
Progression free survival across the subgroups generally showed an excellent benefit for belzutifan versus everolimus
Median overall survival was 21.4 months with belzutifan versus 18.2 months with everolimus (HR 0.92; 95% CI 0.77–1.10; p = 0.18). Estimated overall survival rate was 67.9% vs 65.8% at 12 months and 45.2% vs 41.2% at 24 months:Median overall survival was 21.4 months with belzutifan versus 18.2 months with everolimus
Objective response rate was consistent with prior reports (22.7% vs 3.5%), and median duration of response was 19.3 months (range: 1.9+–40.1+) versus 13.7 months (range: 3.8–29.5+):
Objective response rate was consistent with prior reports
Among patients with no subsequent therapy, there were 173 in the belzutifan arm (61 alive at final analysis) and 121 in the everolimus arm (12 alive at final analysis):
patients with no subsequent therapy, there were 173 in the belzutifan arm (61 alive at final analysis) and 121 in the everolimus arm
Fewer patients discontinued belzutifan (6.2%) vs everolimus (15.3%) due to any-cause adverse events, and median duration of therapy was longer with belzutifan (7.6 months vs 3.9 months). Rate of grade ≥3 treatment-related adverse events (39.5% vs 40.0%) was similar between arms. The following shows the time to the first onset of common any-grade adverse events attributed to belzutifan (adverse drug reactions): the time to first onset of common any-grade adverse events attributed to belzutifan (adverse drug reactions)
Additionally, the following shows the duration of common any-grade adverse events attributed to belzutifan:Additionally, the following shows the duration of common any-grade adverse events attributed to belzutifan
Dr. Rini concluded his presentation discussing the final analysis of the phase 3 LITESPARK-005 trial with the following take-home points:

  • At final analysis of the phase 3 LITESPARK-005 study, belzutifan continued to show progression-free survival and objective response rate benefits versus everolimus, including durable response lasting > 2 years
  • With >2 years minimum follow-up, more participants remained on treatment with belzutifan versus everolimus
  • Significant improvement in overall survival was not observed
  • There were no new safety signals for belzutifan observed. The most common any-grade adverse drug reactions had a median time to onset of < 2 months.
  • Final analysis results of LITESPARK-005 support belzutifan as a treatment option in advanced clear cell RCC after PD-(L)1 inhibitor and VEGFR-TKI therapy

Presented by: Brian I. Rini, MD, FASCO, Chief of Clinical Trials, Vanderbilt University, Nashville, TN

Written by: Zachary Klaassen, MD, MSc – Urologic Oncologist, Associate Professor of Urology, Georgia Cancer Center, Wellstar MCG Health, @zklaassen_md on Twitter during the 2024 European Society of Medical Oncology (ESMO) Annual Meeting, Barcelona, Spain, Fri, Sept 13 – Tues, Sept 17, 2024.

References:

  1. Choueiri TK, Powles T, Peltola K, et al. Belzutifan versus Everolimus for Advanced Renal-Cell Carcinoma. N Engl J Med. 2024 Aug 22;391(8):710-721.