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Light-chain amyloidosis (AL amyloidosis) is the most common type of systemic amyloidosis caused by plasma cell dyscrasia and is characterized by a build-up of immunoglobulin light chains, which will progressively generate amyloid fibril deposits in various organ sites, including the heart.1,2 These deposits cause organ failure and, eventually, death.2 The current standard-of-care regimen is aimed at targeting the malignant plasma cell to reduce the formation of these fibrils.1 However, there are no current therapies that remove existing deposits from organs.2
CAEL-101 is a novel, chimeric monoclonal antibody that targets fibrils directly and has the potential to remove them from organ sites. Belantamab mafodotin targets B-cell maturation antigen plasma cells expressed in AL amyloidosis. During the European Hematology Association Congress 2023, Michaela Liedtke presented the results from a phase II trial of CAEL-101 in patients with AL amyloidosis,1,2 and Efstathios Kastritis shared the findings from a phase II trial of belantamab mafodotin.3 We are pleased to summarize the key data from these trials here.
Table 1. Baseline patient characteristics*
Characteristic (% unless otherwise stated) |
N = 25 |
---|---|
Median age, years |
65.2 |
Male |
72 |
Race |
|
White/Caucasian |
92 |
Black/African American |
8 |
Mayo stage at screening |
|
I |
8 |
II |
76 |
IIIa |
16 |
Patients with cardiac involvement |
88 |
Prior anti-PCD therapy |
80 |
PCD, plasma cell dyscrasia. *Adapted from Valent.1 |
Figure 1. Response rates in patients receiving CAEL-101 therapy after 18 months*
*Adapted from Liedtke.1
Figure 2. Adverse events of any grade experienced by ≥25% of patients treated with CAEL-101*
*Adapted from Liedtke.1
The phase II trial showed that CAEL-101 was well tolerated among patients with AL amyloidosis and presented no evidence of organ toxicity. Most TEAEs were mild or moderate in severity. Further data on the efficacy and safety of CAEL-101 will become available from the two ongoing phase III trials.
Table 2. Baseline patient characteristics*
Characteristic (% unless otherwise stated) |
N = 25 |
---|---|
Median age, years (range) |
66 (46–80) |
Sex |
|
Male |
60 |
Female |
40 |
NYHA class |
|
I |
24 |
II |
76 |
NT-proBNP, ng/L |
1,197 |
Median dFLC, mg/L (range) |
117.1 (37.8–2791) |
Median number of previous treatments, n (range) |
3 (1–10) |
Prior treatments |
|
Daratumumab |
72 |
Borterzomib |
92 |
Organ involvement |
|
Heart |
76 |
Kidney |
64 |
Peripheral nervous system |
24 |
Liver |
16 |
Soft tissue |
20 |
GI tract |
12 |
dFLC, difference between involved free light-chain and uninvolved free light-chain; NT-proBNP, N-terminal pro-brain |
Figure 3. Response rates of patients treated with belantamab mafodotin*
dFLC, difference between involved free light-chain and uninvolved free light-chain; ORR, overall response rate; PR, partial response; VGPR, very good partial response.
*Adapted from Kastritis.3
†Patients experienced a response to treatment but had persistence of small, non-measurable amounts of monoclonal protein that could still affect outcomes.
Figure 4. Ocular AEs in patients treated with belantamab mafodotin*
AE, adverse event.
*Adapted from Kastritis.3
The phase II trial of belantamab demonstrated a high hematologic response rate in heavily pre-treated patients with relapsed or refractory AL amyloidosis. The trial did not identify any new toxicity signals. Despite the prolonged administration period of belantamab, ocular toxicity could not be prevented. These results highlight a potential treatment option for patients with relapsed or refractory AL amyloidosis, particularly those with prior exposure to daratumumab and bortezomib.
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