IPTACOPAN ASH UPDATE
Iptacopan monotherapy was well tolerated and had a favorable
safety profile
Most common TEAEs (24 patients in either arm)1
n (%)
Iptacopan
200mg bid N=62
Any TEAE
51 (82.3)
Anti-C5 SoC
N=35
28 (80.0)
No deaths
No discontinuations due to TEAES
Mild/Moderate / Severe, %
32.3/45.2/4.8
37.1 34.3/8.6
Headache
10 (16.1)
1 (2.9)
Diarrhea
9 (14.5)
2 (5.7)
Nasopharyngitis
7 (11.3)
2 (5.7)
Nausea
6 (9.7)
1 (2.9)
COVID-19
5 (8.1)
9 (25.7)
Urinary tract infection
5 (8.1)
1 (2.9)
Arthralgia
5 (8.1)
1 (2.9)
Abdominal pain
4 (6.5)
1 (2.9)
Increased blood LDH
4 (6.5)
3 (8.6)
Dizziness
4 (6.5)
0
Serious TEAES:
9.7% vs 14.3%
Hemolysis serious TEAEs:
Iptacopan: None
SoC: Breakthrough hemolysis (n=1)
and extravascular hemolysis (n=1)
No serious infections caused
Breakthrough hemolysis
2 (3.2)
6 (17.1)
by encapsulated bacteria
TEAE = treatment-emergent adverse event 1. Organized by descending frequency in the iptacopan arm
19
IPTACOPAN ASH UPDATE | DECEMBER 13, 2022
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