Table 3 Interim results of clinical trials of BCMA-targeted CAR-T cell products

Idecabtagene vicleucel; Bb2121

Phase 1b NCT02658929

[71]

R/R MM who received or were refractory to ≥ 3 prior lines, including PI, IMiD

33 pts (21 in dose-escalation; 12 in dose expansion);

median age 60;

median prior lines 7;

high-risk cytogenetics 13 (45%);

mDOF 11.3 mo

CTX + FAMP

150 or 450 × 10⁶ cells/pt

Expansion at all dose level, persist up to 1 yr

ORR 85%; CR 15 (45%); mPFS 11.8 mo

G3+ neutropenia (85%), leukopenia (58%), thrombocytopenia (45%), anemia (45%);

CRS 23 (70%) G1–2, 2 (6%) G3+;

NTX 14 (42%) G1–2, 13 (39%) G3+

Phase 2 NCT0336174

[103]

R/R MM who received or were refractory to ≥ 3 prior lines, including PI, IMiD, and CD38 mAb

128 pts (54 received 450 × 10⁶ cells);

median age 61;

median prior lines 6; triple-refractory 108 (84%); penta-refractory 33 (26%);

mDOF 11.3 mo

CTX + FAMP

150–450 × 10⁶ cells/pt

Peak on d11, detectable in 29/49 (59%) pts at 6 mo and 4/11 (36%) pts at 12 mo

ORR 73.4%; CR 31.3%; mPFS 11.3 mo

450 × 10⁶ cells dose cohort: ORR 81.5%; CR 35.2%; mPFS 11.3 mo

All grades cytopenia (94%);

CRS 107 (83.6%) G1–2, 7 (5.5%) G3+;

NTX 19 (14.9%) G1–2, 4 (3.1%) G3+

Bb21217

Phase 1 NCT03274219

[104]

R/R MM who received or were refractory to ≥ 3 prior lines, including PI, IMiD; ≥ 50% cell-surface BCMA expression

22 pts;

median age 63;

median prior lines 7; ASCT 18 (82%);

high-risk cytogenetics 7 (32%);

mDOF 23 wk

CTX + FAMP

150 or 350 or 450 × 10⁶ cells/pt

6/8 detectable at 6 mo, 2/2 detectable at 12 mo

ORR 83%; PD 6

CRS 7 G1–2, 1 G3+;

NTX 3 G1–2, 2 G3+

LCAR-B38M

Phase 1/2 NCT03090659

[105]

R/R MM who received or were refractory to ≥ 1 prior lines

57 pts;

median age 54

median prior lines 3;

ASCT 10 (18%);

mDOF 19 mo

CTX

Avg 0.5 × 10⁶ cells/kg 3 split infusions

Detectable till 4 mo, at most 10 mo

ORR 88%; CR 42 (74%), VGPR 2 (4%), PR 6 (11%); mPFS 20 mo; 18-mo PFS 50%; 18-mo OS 68%

G3+ leukopenia (30%), thrombocytopenia (23%), increased AST (21%);

CRS 46 (82%) G1–2, 4 (7%) G3+;

NTX 1 (2%) G1–2

Phase 1/2 NCT03090659

[106]

R/R MM who received or were refractory to ≥ 3 prior lines,

17 pts;

median age 55

median prior lines 5;

ASCT 8 (47%);

mDOF 22 mo

CTX ± FAMP

Avg 0.7 × 10⁶ cells/kg 1 infusion or 3 split infusions

Peak on d6–30, detectable till up to 9 mo

ORR 88%; CR 14 (82%), VGPR 1 (4%); mPFS 12 mo; 1-yr PFS 52.9%; 1-yr OS 82.3%

G3+ cytopenia 10 (59%); G3+ liver toxicity 5 (29%);

CRS 10 (59%) G1–2, 7 (41%) G3+;

NTX 0

JNJ-4528

Phase 1b/2 NCT03548207

[107]

R/R MM who received or were refractory to ≥ 3 prior lines, including PI, IMiD, CD38 mAb

29 pts;

median age 61;

median prior lines 5; 76% penta-exposed, 86% triple-refractory, 31% penta-refractory;

mDOF 9 mo

CTX + FAMP

Avg 0.75 × 10⁶ cells/kg

Peak on d10-14, detectable till 6 mo, memory CD8+ CAR-T

ORR 100%; sCR 22 (76%), VGPR 6 (21%), PR 1 (3%); 6-mo PFS 93%; best mPFS 15 mo

G3+ neutropenia (100%), thrombocytopenia (69%), leukopenia (59%);

CRS 27 (93%) G1–2, 2 (9%) G3+;

NTX 3 G1–2, 1 G3+;

DLT 1; TRD 1

CT053

Phase 1 NCT03716856

[108]

R/R MM who received or were refractory to ≥ 2 prior lines

24 pts;

median age 60

median prior lines 4.5

high-risk cytogenetics 9 (38%);

mDOF 333 d

CTX + FAMP

1.5 × 10⁸ cells/pt

Peak on d7–21, detectable till 172d, at most 341d

ORR 87.5%; sCR 14 (71%), CR 5 (21%), VGPR 1 (4%); mPFS 281 d; PD 9

G3+ leukopenia (87.5%), neutropenia (66.7%), lymphopenia (79.2%), thrombocytopenia (25%);

CRS 15 (62.5%) G1–2;

NTX 2 (8%) G1–2, 1 (4%) G3+

P-BCMA-101

Phase 2 NCT03288493

[109]

R/R MM who received or were refractory to ≥ 3 prior lines, including PI, IMiD, CD38 mAb

12 pts;

prior lines 3–9

mDOF 3 wk

CTX + FAMP

0.75–15 × 10⁶ cells/kg

Peak at 2–3 wks, remain detectable at 3 mo

6 pts in higher dose cohort: ORR 83%; 1 sCR 1 VGPR 3 PR

G3+ cytopenia and febrile neutropenia;

CRS 1 (8%) G1–2;

NTX 0

CART-BCMA

Phase 1b NCT02546167

[110]

R/R MM who received or were refractory to ≥ 3 prior lines, including PI, IMiD,

25 pts;

median age 58;

median prior lines 7;

high-risk cytogenetics 24 (94%); ASCT 23 (92%); panta-refractory 11 (44%);

mDOF 24 mo

None or CTX

1–5 × 10⁷ or 1–5 × 10⁸ cells/pt

Peak on d10–14, remain detectable at 6 mo

ORR 48%; CR 2 (8%), VGPR 5 (20%), PR 5 (20%); best mPFS 125 d; PD 22

G3+ leukopenia (44%), neutropenia (44%), lymphopenia (36%);

CRS 14 (56%) G1–2, 8 (32%) G3+;

NTX 5 (20%) G1–2, 3 (12%) G3+

CT103A

Phase 1 ChiCTR 1800018137

[111]

R/R MM who received or were refractory to ≥ 3 prior lines, including PI, IMiD,

16 pts;

median prior lines 4;

mDOF 195 d

CTX + FAMP

1–8 × 10⁶ cells/kg

Peak at 2 wks, remain detectable at 6 mo

ORR 100%; CR/sCR 12 (75%), VGPR 2 (12.5%)

CRS 15 (94%) G1–2, 1 (6%) G3+;

NTX 0;

DLT 1; TRD 1

JCARH125

Phase 1/2 NCT03430011

[112]

R/R MM who received or were refractory to ≥ 3 prior lines, including PI, IMiD, CD38 mAb, ASCT

8 pts;

median age 53;

median prior lines 10; ASCT 8 (88%); panta-refractory 4 (50%);

mDOF 5 wk

CTX + FAMP

50 or 150 × 10⁶ cells/pt

–

ORR 100%; sCR/CR 3 (37.5%), VGPR 2 (25%), PR 2 (25%); PD 0

CRS 6 (75%) G1–2;

NTX 2 (25%) G1–2, 1 (12.5%) G3+

MCARH171

Phase 1 NCT03070327

[113]

R/R MM who received or were refractory to ≥ 2 prior lines, including PI, IMiD

11 pts;

median prior lines 6

CTX + FAMP

72, 137, 475, 818 × 10⁶ cells/pt 1 to 2 doses

Peak expansion found in dose cohort 475, 818 × 10⁶

ORR 64%; mDOR 106 d

CRS 4 (40%) G1–2, 2 (20%) G3+;

NTX 1 (9%) G1–2

BCMA CAR-T

Phase 1 NCT03093168

[114]

R/R MM who received or were refractory to ≥ 2 prior lines, including PI, IMiD; ≥ 5% cell-surface BCMA

44 pts

median prior lines ≥ 2

mDOF ≥ 1 mo

CTX + FAMP

9 × 10⁶ cells/kg

Expansion and persistence throughout the DOF

ORR 79.6%; sCR 2 (4.5%), CR 16 (36%), VGPR 8 (18%), PR 8 (18%); mPFS 15 mo; 2-yr PFS 49.16%; 2-yr OS 53.95%

CRS 10 (22.7%) G1–2, 3 (6.8%) G3+