MB-106 granted orphan drug designation for the treatment of Waldenström’s macroglobulinemia

On June 22, 2022, it was announced that the U.S. Food and Drug Administration had granted orphan drug designation to the CD20-targeted autologous CAR T-cell therapy, MB-106, for the treatment of patients with relapsed or refractory Waldenström’s macroglobulinemia. The safety and efficacy of MB-106 is still being investigated in patients with high-risk B-cell non-Hodgkin lymphoma as part of an ongoing phase I/II clinical trial (NCT03277729). Data obtained from a subset of patients in this trial with follicular lymphoma were shared at the European Hematology Association (EHA) 2022 Congress. These data indicate that MB-106 has a favorable level of efficacy in this population, as well as a tolerable safety profile.¹

Efficacy²

• 94% overall response rate
• 78% complete response rate
• 17% partial response rate

Safety²

• In total, 27.5% of patients experienced cytokine release syndrome of any grade, of which 22% was Grade 1 and 5.5% was Grade 2.
• Other common adverse effects included lymphopenia (100%), neutropenia (94%), anemia (61%), thrombocytopenia (28%), and febrile neutropenia (16.5%).