Aims: 177Lu-PSMA-617 is an emerging targeted therapy that has shown good efficacy and safety in mCRPC. Idronoxil is an inhibitor of external NADH oxidase type 2 that possesses downstream pro-apoptotic actions that confer radio-sensitivity to cancer cells. We aimed to evaluate the efficacy of 177Lu-PSMA-617 monotherapy versus combination 177Lu-PSMA-617/NOX-66 (Veyondra) therapy.
Methods: 30 men with progressive mCRPC despite docetaxel, cabazitaxel and either abiraterone or enzalutamide were enrolled across two therapeutic studies. The first with177Lu-PSMA-617 alone (14 men) and the second 177Lu-PSMA-617 + NOX66 (16 men; 8 receiving 400mg and 8 receiving 800mg daily for 10 days per cycle). Both studies required PSMA uptake on PSMA PET of greater than liver activity with no discordant disease compared with FDG-PET. Each man received 177Lu-PSMA-617 at 6-weekly intervals.
Results: Baseline PSA distributions were similar in the combination and monotherapy trials (median PSA level 147ng/mL [IQR 67-443] versus 88ng/mL [IQR 54-293] respectively). Significantly more patients in the combination trial initiated a fourth treatment cycle compared to the monotherapy trial (69% vs 21%; P < 0.001). PFS was significantly longer in the combination group 8.4 months (IQR 3.6-10.9) versus the monotherapy group 2 months (IQR 0.2-5.0; mean PSA PFS 7.8 vs 3.4 months [p=0.023]). A PSA reduction of ≥50% occurred in 69% (11/16) versus 36% (5/14) of patients treated with combination and monotherapy respectively (P=0.073). Kaplan-Meier survival estimation revealed a significant benefit to combination therapy (p=0.014). Cox regression further revealed that combination treatment and increasing SUVmax were significant predictors of PSA PFS (HR 0.239 [95%CI 0.92-0.623] and HR 0.945 [95%CI 0.93-0.977] respectively). No significant treatment related adverse effects were reported in either study.
Conclusions: Within the limits of a comparison of two separate phase 1 trials, the efficacy of PSMA targeted treatments may be significantly improved through patient selection and use of combination treatments.