Q2 2021 Results - Reimagining Medicine
Participants
Company overview
Pharmaceuticals
Oncology
Financial review
Conclusion
Appendix
References
Iptacopan reduces LDH and increases hemoglobin in PNH
3 INNOVATION
New data (anti-C5 naive)
presented at EHA 2021
LDH¹ (U/L)
LDH¹ (U/L)
4000-
Mean
3000 (90% CI³)
2000
1000
Week 4
Cohort 1: 25.
→> 100mg BID
Week 12
0
-3
-2
-1
0
1
2
6
8
10
12
14
Week
4000
3000-
Mean
(90% CI3)
2000-
1000-
0-
-2
1.5'ULN
ULN4
Week 4
Cohort 2: 50
→>>
200mg BID
Week 12
6
10
12
Week
1.5 ULN
ULN
Hemoglobin (g/L)
Hemoglobin (g/L)
170-
150-
130
110-
90
Cohort 1: 25
Week 4
100mg BID
Week 12
70-
Mean
(90% CI)
50-
-3
-1
0
4
6
8
10
12
14
Week
170-
150-
130-
Week 4
Cohort 21: 50 → 200mg BID
110-
Mean
(90% C14)
90-
70-
Week 12
50-
-2
-1
0
1
2
6
8
10
12
14
Week
Male LLN
Female LLN
Male LLN
Female LLN
FDA Breakthrough
Therapy designation
■
■
New data (EHA 2021) shows clinically important benefits of monotherapy iptacopan in anti-C5 treatment naive PNH patients
Previous Ph2 shows iptacopan provided clinical benefits as add-on to eculizumab in PNH residual hemolysis (EBMT 2020)
Ph3 APPLY-PNH study to assess superiority of iptacopan vs. anti-C5 therapy ongoing, will support filings worldwide
LDH Lactate dehydrogenase
raising Hb from 71 to 110 g/L.
BID - Twice a day CI - Confidence interval ULN - Upper limit of normal 1. One patient in Cohort 2 was excluded for Hb analyses due to RBC transfusion that occurred between screening and baseline,
Source: Jang JH, et al. Iptacopan Effectively Controls Intra- And Extravascular Hemolysis And Leads To Durable Hemoglobin Increase In Patients With Treatment-Naïve PNH.
14 Investor Relations | Q2 2021 Results
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