FDA grants Orphan Drug Designation to STRO-001 for the treatment of multiple myeloma

On 12 October 2018, the US Food and Drug Administration (FDA) granted Orphan Drug Designation to STRO-001, a first-in-class antibody-drug conjugate (ADC), for the treatment of patients with multiple myeloma (MM).¹

CD74, a transmembrane glycoprotein involved in the formation and transport of MHC proteins, has been reported to have a high expression in up to 90% of B-cell malignancies, including MM, with minimal expression in normal tissues.  STRO-001 is a novel ADC targeting CD74 glycoprotein. Preclinical data have shown that STRO-001 demonstrates a potent cytotoxicity in MM cell lines and reduces tumor burden in MM xenograft models, including significant prolongation of survival in the MM.1S model.²

A first-in-human phase I, open-label, multicenter, dose-escalation study  (NCT03424603) evaluating the safety, tolerability, and preliminary anti-tumor activity of STRO-001 in adult subjects with B-cell malignancies including MM and non-Hodgkin lymphoma who are refractory to, or intolerant of all established therapy known to provide clinical benefit for their conditions is currently ongoing.