Pen-RMA for PCNSL: Phase II study results

Results from a phase II study (NCT05347641), evaluating penpulimab, a humanized immunoglobulin G1 (IgG1) monoclonal antibody targeting programmed death-1 (PD-1), in combination with rituximab + high-dose methotrexate + cytarabine (Pen-RMA) in 23 patients with newly diagnosed primary central nervous system lymphoma (PCNSL), were published in Blood Cancer Journal by Shen et al. The primary endpoint was 2-year progression-free survival (PFS). Secondary endpoints included overall response rate (ORR) and 2-year overall survival (OS).

Key data: At the end of induction therapy, the ORR was 95.7% (95% confidence interval [CI], 83.2–99.8) and the complete response rate (CRR) was 91.3% (95% CI, 81.2–99.9). At a median follow-up of 29.4 months, the 2-year PFS was 70.7% (95% CI, 50.1–91.3) and the 2-year OS was 75.0% (95% CI, 50.9–90.1). The median PFS, OS, and duration of response (DoR) were not reached. Grade ≥3 treatment-related adverse events (TRAEs) occurred in 30.4% of patients. 

Key learning: The Pen-RMA regimen demonstrated antitumor activity with a tolerable safety profile in patients with newly diagnosed PCNSL, warranting further investigation in this patient population.