Teclistamab Earns Breakthrough Therapy Designation for R/R Multiple Myeloma


Based on data from a phase 1 trial, the FDA granted breakthrough therapy designation to teclistamab for certain patients with pretreated multiple myeloma.

The FDA granted breakthrough therapy designation (BTD) to the off-the-shelf bispecific antibody teclistamab as treatment for patients with relapsed or refractory (R/R) multiple myeloma, according to the company responsible for developing the therapy, The Janssen Pharmaceutical Companies of Johnson & Johnson.

The designation from the agency follows a priority medicines, or PRIME, designation from the European Medicines Agency earlier this year and marks the 11th BTD received by Janssen’s Oncology Therapeutic Area.

“We are pleased to have received Breakthrough Therapy and PRIME Designations for our novel bispecific antibody, teclistamab,” Peter Lebowitz, MD, PhD, Global Therapeutic Area Head of Oncology of Janssen Research & Development, LLC, said in a press release. “This program exemplifies our commitment to advancing science for patients living with multiple myeloma, and it builds upon our robust portfolio in this disease.”

Preliminary results of the open-label, multicenter, phase 1 MajesTEC-1 trial (NCT03145181) of teclistamab in 78 patients with R/R multiple myeloma were presented at the 2020 American Society of Clinical Oncology (ASCO) Scientific Program, demonstrating safety and efficacy of the regimen.

Part 1 was a dose-escalation phase, with a primary objective of dose finding for the expansion portion. Doses ranged from 0.3-720 µg/kg weekly, and there was a 67% overall response rate (ORR) at 270 µg/kg.

The most common all-grade adverse effects (AEs) were cytokine release syndrome (CRS) in 56% of patients, typically occurring during the initial doses, all of which were grade 1/2 in severity. Additionally, neutropenia occurred in 26% of patients, anemia in 23%, treatment-related neurotoxicity in 8% (grade 3 or higher, 3%), and infusion-related reactions in 9%.

A total of 61% of patients experienced an infection-related AE, 21% of which were grade 3 or higher. Reported dose-limiting toxicities were delirium (resolving after 16 days) and thrombocytopenia (resolving after 1 day).

At the time of data reporting, both the dose-escalation and -expansion portions of the trial were ongoing with immature efficacy data for the 270 µg/kg. Updated data are expected for presentation at the upcoming 2021 ASCO Annual Meeting.

Teclistamab is also being examined in a phase 2 trial (NCT04557098), which will evaluate efficacy of the agent at the recommended dose from the phase 1 trial. The primary end point is ORR, with secondary outcome measures including response duration, rate of very good partial response or better, rate of complete response or better, stringent complete response rate, time to response, progression-free survival, overall survival, minimal residual disease negativity, and safety.

Teclistamab is a T-cell redirecting antibody with dual inhibition of B-cell maturation antigen and CD3 receptors, inducing tumor cell death. Preclinical data have indicated that the agent is capable of killing both myeloma cell lines and bone marrow–derived myeloma cells from heavily pretreated patients.

The BTD, which is reserved for therapeutics which demonstrate an ability to substantially improve outcomes versus available therapies, will allow for the expedited development of teclistamab.


Janssen Announces U.S. FDA Breakthrough Therapy Designation Granted for Teclistamab for the Treatment of Relapsed or Refractory Multiple Myeloma. News release. The Janssen Pharmaceutical Companies of Johnson & Johnson. June 1, 2021. Accessed June 2, 2021. https://bit.ly/3yUSBHW

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