Selpercatinib is a highly selective RET kinase inhibitor that has demonstrated improved progression-free survival (PFS) in patients with first-line RET fusion-positive NSCLC. This study examines selpercatinib outcomes with variable exposures resulting from dose adjustment.
NSCLC patients treated with selpercatinib in 2 prospective trials (LIBRETTO-001/LIBRETTO-431) were started at 160mg BID; dose reductions to 120mg, 80mg, and 40mg BID were permitted. Relationship between selpercatinib and efficacy endpoints (overall response rate [ORR] and PFS) were determined using exposure-response models. Steady-state exposure parameters selected for analysis (area under the plasma concentration-time curve over 24 hours at steady state [AUC24] and maximum and minimum selpercatinib concentrations) represented average selpercatinib exposure over time. Additionally, average exposure over the last 10 doses was considered.
Of 504 treated NSCLC patients (LIBRETTO-431, n=150; LIBRETTO-001, n=354), 266 underwent dose reduction. Median time to first dose reduction was 2 months. Patients with dose reduction tended to be older (median age 61 vs 59 years) and had lower body weight (median weight 63 vs 66 kg) and longer time on therapy (median time on treatment [95% confidence interval]: 31.7 vs 21.2 months) versus patients without dose reduction. AST/ALT elevation, QT prolongation, and hypertension were the most common adverse events leading to dose adjustments. Focusing on 502 patients included in the ORR exposure-response analysis, the response rate was 69%; a stepwise multivariate logistic regression showed that probability of response increased with increasing selpercatinib AUC24 (P<0.05). Exploratory analysis of PFS in 504 patients (254 events) by exposure metrics showed no significant relationship with PFS across exposure quartiles.
No correlation found between drug exposure and PFS but did suggest that higher exposure was associated with better response rates. For patients experiencing toxicity on selpercatinib, dose adjustment to reduce exposure may allow ongoing clinical benefit without a decremental impact on PFS.
Previously presented at WCLC 2024.