Paul G Richardson, Suzanne Trudel, Rakesh Popat, María-Victoria Mateos, Annette J Vangsted, Karthik Ramasamy, Joaquín Martinez-Lopez, Hang Quach, Robert Z Orlowski, Mario Arnao, Sagar Lonial, Chatchada Karanes, Charlotte Pawlyn, Kihyun Kim, Albert Oriol, Jesus G Berdeja, Paula Rodríguez Otero, Ignacio Casas-Avilés, Alessia Spirli, Jennifer Poon, Shaoyi Li, Jing Gong, Lilly Wong, Manisha Lamba, Daniel W Pierce, Michael Amatangelo, Teresa Peluso, Paulo Maciag, Jessica Katz, Michael Pourdehnad, Nizar J Bahlis
BACKGROUND: Despite recent progress, multiple myeloma remains incurable. Mezigdomide is a novel cereblon E3 ubiquitin ligase modulator with potent antiproliferative and tumoricidal activity in preclinical models of multiple myeloma, including those resistant to lenalidomide and pomalidomide. METHODS: In this phase 1-2 study, we administered oral mezigdomide in combination with dexamethasone to patients with relapsed and refractory myeloma. The primary objectives of phase 1 (dose-escalation cohort) were to assess safety and pharmacokinetics and to identify the dose and schedule for phase 2...
September 14, 2023: New England Journal of Medicine