Phase III trial of Lumoxiti shows complete response rate in hairy cell leukemia.- Innate Pharma

Innate Pharma shared new, long-term data from the pivotal Phase III trial of Lumoxiti (moxetumomab pasudotox-tdfk) in relapsed or refractory hairy cell leukemia patients at the 61st American Society of Hematology (ASH) Annual Meeting. The trial expands on the efficacy results and affirms the manageable safety profile of the medicine. The final analysis showed that 36 percent (29/80) of the relapsed or refractory hairy cell leukemia patients achieved durable complete response (CR) with Lumoxiti at Day 181 of patients� respective evaluation, compared to the primary analysis in which 30 percent durable CR rate was reported.

In addition, there was a 61 percent probability that patients who achieved a CR would maintain it after five years. The single-arm, multi-center, open-label Phase III �1053� clinical trial assessed the efficacy, safety, immunogenicity and pharmacokinetics of Lumoxiti monotherapy in 80 patients with relapsed or refractory hairy cell leukemia who had received at least two prior therapies, including one purine nucleoside analog. The primary endpoint of durable CR was defined as CR with hematologic remission (HR) for >180 days. The final analysis shows that the risk-benefit profile of Lumoxiti is maintained.

There were no new serious adverse events and no change in hemolytic uremic syndrome or capillary leak syndrome. Per the primary analysis on the 1053 study, the most frequent treatment-related adverse events (AEs) were peripheral edema (39%), nausea (35%), fatigue (34%), headache (33%), and pyrexia (31%). Treatment-related grade 3/4 AEs were reported in 24 patients (30%) and treatment-related serious AEs in 14 patients (18%). Grade 3/4 CLS events occurred in two patients (2.5%) and any grade of HUS occurred in six patients (7.5%). CLS and HUS events were manageable and reversible with appropriate supportive care and monitoring. Treatment-emergent AEs led to study drug discontinuation in eight patients (10.0%): hemolytic uremic syndrome (HUS), n = 4 (5.0%); capillary leak syndrome (CLS), n = 2 (2.5%); increased blood creatinine, n = 2 (2.5%); renal failure, n =1 (1.3%); vomiting, n =1 (1.3%); and chills, n =1 (1.3%). There were four deaths reported (including the three reported during the primary analysis): two due to disease progression and two due to an AE (1 each of pneumonia and septic shock). No death was considered treatment related.