Q3 2022 Results
Company overview
Financial review
2022 priorities
Appendix
Financial performance
Innovation: Pipeline overview
Innovation: Clinical trials
Cardiovascular
Immunology
Neuroscience
Oncology
iptacopan - CFB inhibitor
iptacopan - CFB inhibitor
References
Abbreviations
Other
NCT04558918 APPLY-PNH (CLNP023C12302)
Paroxysmal nocturnal haemoglobinuria
Indication
Phase
Phase 3
Patients
91
Primary
Outcome
Measures
Arms
Intervention
Target
Patients
Read-out
Milestone(s)
Publication
Percentage of participants achieving a sustained increase in hemoglobin
levels of ≥ 2 g/dL in the absence of red blood cell transfusions
Percentage of participants achieving sustained hemoglobin levels ≥ 12 g/dL in
the absence of red blood cell transfusions
Arm 1: Drug: LNP023, taken orally b.i.d. dosage supplied: 200 mg dosage
form: hard gelatin capsule Route of Administration: Oral
Arm 2:
Drug: Eculizumab, administered as intravenous infusion every 2 weeks as per
the stable regimen, the maintenance dose is a fixed dose. Dosage supplied:
300 mg/30mL Dosage form: Concentrate solution for infusion
Drug: Ravulizumab, administered as intravenous infusion every 8 weeks, the
maintenance dose is based on body weight. Dosage Supplied: 300 mg/30mL
Dosage form: Concentrate solution for infusion
Adult patients with PNH and residual anemia, despite treatment with an
intravenous Anti-C5 antibody
Primary 2022
Risitano AM, et al. Abstract accepted at the European Hematology
Association (EHA 2021) congress (study design abstract; accepted for
publication only)
NCT04820530 APPOINT-PNH (CLNP023C12301)
Indication
Phase
Paroxysmal nocturnal haemoglobinuria
Phase 3
Patients
40
Primary
Outcome
Measures
Arms
Intervention
Target
Patients
Read-out
Milestone(s)
Publication
Proportion of participants achieving a sustained increase from baseline in
hemoglobin levels of ≥2 g/dL assessed, in the absence of red blood cell
transfusions
Iptacopan (LNP023), taken orally b.i.d. (dosage supplied: 200mg)
PNH patients who are naive to complement inhibitor therapy, including anti-
C5 antibody
2022
Peffault de Latour R, et al. Abstract accepted at the European Hematology
Association (EHA 2021) congress (study design abstract; accepted for
publication only)
66 Investor Relations | Q3 2022 Results
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