Q4 2021 Results
Participants
Company overview
Pharmaceuticals
Oncology
Financial review
Conclusion
Appendix
Financial performance
Innovation: Pipeline overview
Innovation: Clinical trials
CRM
IHD
Neuroscience
Ophthalmology
Respiratory & Allergy
Oncology: Solid Tumors
Hematology
Biosimilars
Global Health
Abbreviations
iptacopan - CFB inhibitor - HEM
Study
Indication
Phase
Patients
Primary Outcome
Measures
Arms Intervention
Target Patients
Read-out Milestone(s)
Publication
NCT04558918 APPLY-PNH (CLNP023C12302)
Paroxysmal nocturnal haemoglobinuria
Phase 3
91
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 f
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)
Paroxysmal nocturnal haemoglobinuria
Phase 3
40
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
2023
Peffault de Latour R, et al. Abstract accepted at the European Hematology
Association (EHA 2021) congress (study design abstract; accepted for publication
only)
136 Investor Relations | Q4 2021 Results
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