FDA provides positive feedback in end of phase II meeting for BNZ-1 for patients with refractory cutaneous T-cell lymphoma

On January 4, 2021, it was announced that the U.S. Food and Drug Administration (FDA) provided positive feedback in the end of phase II meeting for BNZ-1, a multi-cytokine inhibitor, for the treatment of refractory cutaneous T-cell lymphoma.¹ The completed phase II trial data were also presented at the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition and are an update on those previously reported on Lymphoma Hub.²

NCT03239392 was a multicenter, open label, phase I/II study to evaluate the efficacy and safety of BNZ‑1 in patents with refractory cutaneous T-cell lymphoma that had failed up to seven prior therapies. Initial dose escalation (n = 15) resulted in selection of the 2 mg/kg dose, based on the pharmacokinetic/pharmacodynamic relationship and clinical efficacy, and the cohort was subsequently expanded (n = 19).²

The primary endpoint of the study was overall safety after 4 weeks of treatment. A treatment extension of 3 months was required to further evaluate safety and clinical response at week 17, and long-term extension of over a year was available for those who benefited from BNZ-1 treatment (n = 12).²

The overall response rate for the expanded cohort was 63.2%, as measured by the global response score. Positive response typically occurred soon after treatment initiation, which persisted for the duration of treatment, with 10% of patients reaching a partial response as early as 4 weeks after the initial dose. Stable disease was seen in 37% of patients. No disease recurrence or relapse was observed during the study period. BNZ-1 treatment was well tolerated with no dose-limiting toxicities or drug-related serious adverse effects. BNZ-1 also demonstrated a dose-dependent reduction of regulatory T‑cells.²

Based on these results and the FDA’s feedback, a phase III clinical trial will be initiated in this subset of patients. The FDA provided clear guidance for the design of this pivotal trial; therefore, enrollment is expected to begin in the second half of 2021.¹