Tagraxofusp (SL-401) in Participants With Chronic Myelomonocytic Leukemia (CMML) and Myelofibrosis (MF)

Summary

This multicenter, multi-arm trial evaluated the safety and efficacy of tagraxofusp, a cell division cycle protein 123 homolog-targeted therapy, in participants with either CMML or MF. There were 2 CMML cohorts, 1 enrolled participant with CMML (CMML-1 or CMML-2) who were refractory/resistant or intolerant to hypomethylating agents (HMA), hydroxyurea (HU), or intensive chemotherapy and 1 enrolled treatment-naive participants with CMML (CMML-1 or CMML-2) with molecular features associated with poor prognosis. The MF cohort enrolled participants who were resistant/refractory or intolerant to approved Janus kinase (JAK) therapy (JAK1/JAK2 or JAK2).

Timeline

Start

2016-02-10

Primary completion

2023-03-27

Completion

2023-03-27

Publications

• Yacoub A, Ali H, Gupta V, Wang ES, Patnaik MM, Schiller GJ, Taparia M, Mughal TI, Lindsay R, Galleu A, Gupta I, Pemmaraju N. Final safety and efficacy results from a phase 1/2 study of tagraxofusp, a CD123-targeted therapy, for myelofibrosis. Blood Neoplasia. 2025 Aug 25;2(4):100165. doi: 10.1016/j.bneo.2025.100165. eCollection 2025 Nov.
• Patnaik MM, Ali H, Wang ES, Yacoub A, Foran JM, Gupta V, Schiller GJ, Stevens DA, Talpaz M, Wall S, Lasho TL, Mangaonkar AA, Badar T, Lindsay R, Galleu A, Gupta I, Pemmaraju N, Garcia-Manero G. Tagraxofusp, a CD123-targeted therapy, for chronic myelomonocytic leukemia: final results of a phase 1/2 study. Blood Neoplasia. 2025 May 11;2(4):100115. doi: 10.1016/j.bneo.2025.100115. eCollection 2025 Nov.

Drugs

Indications