New England Journal of Medicine August 11, 2022

Teclistamab in Relapsed or Refractory Multiple Myeloma (MajesTEC-1)

Moreau P, Garfall AL, van de Donk NWCJ, et al.

Bottom Line

In heavily pretreated patients with relapsed or refractory multiple myeloma, the off-the-shelf BCMAxCD3 bispecific antibody teclistamab yielded a 63.0% overall response rate with deep and durable remissions, despite high rates of cytopenias and infections.

Key Findings

1. The overall response rate (ORR) was 63.0%, with 39.4% of patients achieving a complete response (CR) or better.
2. Minimal residual disease (MRD) negativity was achieved in 26.7% of the overall cohort (and 46% among those with a CR or better).
3. With a median follow-up of 14.1 months, the median duration of response (mDOR) was 18.4 months, and median progression-free survival (mPFS) was 11.3 months.
4. Cytokine release syndrome (CRS) was frequent, occurring in 72.1% of patients, but was predominantly low-grade (grade 3 in only 0.6%, with no grade 4 events).
5. Neurotoxic events occurred in 14.5% of patients, including immune effector cell-associated neurotoxicity syndrome (ICANS) in 3.0%.
6. Cytopenias and infections were highly prevalent: grade 3 or 4 neutropenia occurred in 64.2% of patients, and grade 3 or 4 infections occurred in 44.8%.

Study Design

Design
Phase 1/2 Single-Arm Trial
Open-Label
Sample
165
Patients
Duration
14.1 mo
Median
Setting
Multicenter, international
Population Patients with relapsed or refractory multiple myeloma who had received at least 3 prior therapy lines, including triple-class exposure to an immunomodulatory drug, a proteasome inhibitor, and an anti-CD38 antibody.
Intervention Teclistamab 1.5 mg/kg administered via weekly subcutaneous injection, following step-up doses of 0.06 mg/kg and 0.3 mg/kg.
Comparator N/A (Single-arm study)
Outcome Overall response rate (ORR; defined as a partial response or better) assessed by an independent review committee.

Study Limitations

The single-arm, open-label design lacks a direct randomized comparator, limiting definitive comparative efficacy or safety conclusions against standard-of-care regimens.
The high rate of grade 3 or 4 infections (44.8%) reflects substantial on-target, off-tumor immunosuppression (hypogammaglobulinemia) that requires vigilant monitoring and often necessitates prophylactic strategies like intravenous immunoglobulin (IVIG).
Patients enrolled in early-phase clinical trials are typically fitter than the general, real-world population with triple-class refractory myeloma, potentially underestimating toxicity rates in frail patients.
The relatively short initial median follow-up of 14.1 months means long-term survival outcomes and durability of response require further validation.

Clinical Significance

The MajesTEC-1 trial established teclistamab as a highly active, first-in-class T-cell redirecting bispecific antibody for multiple myeloma. By achieving a 63.0% response rate in patients with a median of five prior lines of therapy (and 77.6% with triple-class refractory disease), teclistamab provided an immediately accessible ('off-the-shelf') alternative to CAR-T cell therapies. This pivotal data led to its accelerated FDA approval in late 2022, fundamentally shifting the treatment paradigm for highly refractory multiple myeloma and establishing BCMA-targeted bispecifics as a new pillar of care.

Historical Context

Multiple myeloma remains an incurable plasma cell malignancy. Prior to the advent of BCMA-directed therapies, patients who became refractory to the three main classes of anti-myeloma agents—proteasome inhibitors, immunomodulatory drugs, and anti-CD38 monoclonal antibodies—faced a dismal prognosis, with historical median overall survival of less than a year. While BCMA-directed CAR-T cell therapies (idecabtagene vicleucel and ciltacabtagene autoleucel) demonstrated unprecedented efficacy in this population, they are constrained by complex logistics, apheresis requirements, and multi-week manufacturing delays. Teclistamab was developed to bypass these logistical hurdles by bridging T cells directly to myeloma cells in vivo via an off-the-shelf injection. The MajesTEC-1 study was a landmark moment, proving that a bispecific antibody could achieve CAR-T-like response rates and durations, democratizing access to powerful T-cell redirecting therapies for multiple myeloma patients.

Guided Discussion

High-yield insights from every perspective

Med Student
Medical Student

Teclistamab is a BCMAxCD3 bispecific antibody. What is the physiological role of BCMA in plasma cell biology, and how does engaging CD3 mediate tumor cell death in this context?

Key Response

BCMA (B-cell maturation antigen) is a receptor heavily expressed on plasma cells that promotes their survival and proliferation by binding to BAFF and APRIL. Engaging CD3 on T-cells brings them into close proximity with BCMA-expressing myeloma cells, inducing T-cell activation, proliferation, and perforin/granzyme-mediated apoptosis of the tumor cells independent of MHC restriction.

Resident
Resident

Given the high rates of cytokine release syndrome (CRS) and infections seen in the MajesTEC-1 trial, what are the initial management steps for a patient presenting with fever and hypotension shortly after step-up dosing of teclistamab?

Key Response

Fever and hypotension post-dosing indicate Grade 2 or higher CRS, but sepsis must be simultaneously ruled out. Initial management requires drawing blood cultures and starting broad-spectrum antibiotics, while concurrently treating the CRS with the IL-6 receptor antagonist tocilizumab, IV fluids, and potentially corticosteroids and vasopressors if refractory.

Fellow
Fellow

For a triple-class refractory myeloma patient, how do you decide between recommending a BCMA-directed CAR-T cell therapy versus an off-the-shelf BCMA bispecific like teclistamab, considering the efficacy, toxicity profile, and logistical constraints highlighted in MajesTEC-1?

Key Response

CAR-T offers a 'one-and-done' treatment with potentially deeper remissions but has high manufacturing wait times, risk of disease progression during bridging, and severe CRS/ICANS. Teclistamab is immediately available ('off-the-shelf') and highly effective for rapidly progressing disease, but requires continuous dosing and carries a sustained risk of severe infections and hypogammaglobulinemia.

Attending
Attending

The MajesTEC-1 trial reported high rates of severe, sometimes fatal, opportunistic infections and hypogammaglobulinemia. As continuous teclistamab therapy becomes integrated into our practice, how should we systematically adapt our prophylactic strategies to mitigate this chronic infectious risk?

Key Response

Continuous T-cell redirection and B-cell depletion lead to profound, lasting immune suppression. Attending physicians must proactively implement IVIG replacement for hypogammaglobulinemia, along with rigorous antimicrobial prophylaxis (e.g., PJP, HSV/VZV, and possibly antibacterial/antifungal cover) and ensure vaccination optimization prior to therapy initiation.

Scholarly Review

Critical appraisal through the lens of expert reviewers and guideline development

PhD
PhD

MajesTEC-1 is a phase 1/2 single-arm study. In heavily pretreated populations, how can researchers methodologically establish robust synthetic control arms or utilize real-world data (RWD) to contextualize the 63 percent ORR, avoiding the biases inherent in historical cross-trial comparisons?

Key Response

Researchers can use techniques like propensity score matching or inverse probability of treatment weighting (IPTW) on prospective observational registries (like the LocoMMotion cohort) to match baseline characteristics. Critiquing single-arm designs requires addressing unmeasured confounding, selection bias, and temporal shifts in standards of care.

Journal Editor
Journal Editor

When reviewing the progression-free survival (PFS) and duration of response (DOR) curves in this single-arm study, what specific aspects of patient censoring and early dropouts (e.g., due to COVID-19 or toxicity) would a peer reviewer flag as potential threats to the validity of the survival estimates?

Key Response

High early dropout rates or informative censoring (e.g., dropping out due to adverse events like infections rather than random loss to follow-up) can artificially inflate Kaplan-Meier estimates for PFS and DOR. Editors must demand sensitivity analyses that treat toxicity-related dropouts as events to ensure the efficacy isn't overstated.

Guideline Committee
Guideline Committee

Based on the MajesTEC-1 data, teclistamab received accelerated approval. How should guideline committees position teclistamab within current algorithms for RRMM relative to existing options, and what evidentiary gaps must be filled before assigning it a Category 1 recommendation?

Key Response

Current guidelines recommend teclistamab for patients with 4 or more prior lines including an IMiD, PI, and anti-CD38 mAb. To upgrade to a Category 1 recommendation, committees require Phase 3 randomized controlled trial data comparing teclistamab directly to standard-of-care regimens, specifically evaluating overall survival, patient-reported outcomes, and long-term cumulative toxicity.

Clinical Landscape

Noteworthy Related Trials

2021

KarMMa Trial

n = 128 · NEJM

Tested

Idecabtagene vicleucel (ide-cel)

Population

Triple-class exposed relapsed/refractory multiple myeloma

Comparator

None (single-arm)

Endpoint

Overall Response Rate (ORR)

Key result: Ide-cel demonstrated deep and durable responses with an ORR of 73% and a complete response rate of 33% in heavily pretreated patients.
2021

CARTITUDE-1 Trial

n = 97 · Lancet

Tested

Ciltacabtagene autoleucel (cilta-cel)

Population

Triple-class exposed relapsed/refractory multiple myeloma

Comparator

None (single-arm)

Endpoint

Overall Response Rate (ORR)

Key result: Cilta-cel yielded an exceptionally high ORR of 97%, with 67% of patients achieving stringent complete response and durable progression-free survival.
2023

MagnetisMM-3 Trial

n = 123 · Nat Med

Tested

Elranatamab

Population

BCMA-naive relapsed/refractory multiple myeloma

Comparator

None (single-arm)

Endpoint

Overall Response Rate (ORR)

Key result: Elranatamab demonstrated an ORR of 61% with a manageable safety profile, validating the use of step-up dosing to mitigate severe CRS.

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