EMA grants anti-BCMA CAR T, JNJ-4528, PRIME designation in multiple myeloma 

It has been announced that JNJ-4528, an anti-B-cell maturation antigen (BCMA) CAR T-cell therapy has been granted priority medicines (PRIME) designation by the European Medicines Agency (EMA) for use in multiple myeloma (MM).¹

JNJ-4528, was previously named LCAR-B38M, and the PRIME designation is the result of the phase I/II LEGEND-2 study of LCAR-B38M, previously reported by the Multiple Myeloma Hub here.¹

LEGEND-2 study^1,2

• Interim results of phase I multicenter study in China
• Reported enrollment of 57 patients with relapsed/refractory MM (RRMM)
  • Patients received at least three prior treatments including; a proteasome inhibitor (PI), an immunomodulatory drug (IMiD) and daratumumab
  • Documented disease progression ≤12 months after starting most recent therapy or double-refractory to a PI and IMiD
  • Median age: 54 years (27–72)
  • Stage III disease: 37%
• Data cut-off: 6 February 2018

• Safety:

  • Patients experiencing ≥1 adverse event (AE): 100% (57/57)
  • Cytokine release syndrome (CRS) occurred in 90% patients (51/57)
    • CRS ≥ grade 3: 7% (4/57)
    • One grade 5 fatal event (1/57)
  • Grade ≥3 AEs were reported in 65% of patients (37/57)
  • Most common AEs grade ≥3 (in >20% of patients):
    • Leukopenia: 30% (17/57)
    • Thrombocytopenia: 23% (13/57)
    • Aspartate aminotransferase increase: 21% (12/57)

• Efficacy:

  • Follow-up: 8 months
  • Overall response rate: 88% (50/57, 95% CI, 76–95)
    • Complete response (CR): 68% (39/57, 95% CI, 55–80)
    • Very good partial response (VGPR): 5% (3/57, 95% CI, 1–15)
    • Partial response (PR): 14% (8/57, 95% CI, 6–26)
  • Median progression-free survival (PFS): 15 months (95% CI, 11–not estimable)
  • Median time to initial response: 1 month
  • Median overall survival was not reached

The PRIME designation will speed-up the evaluation of JNJ-4528 and will aim to bring this therapy to patients as soon as possible based on the high unmet medical need.³