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
NCT03439839 (CLNP023X2201)
Paroxysmal nocturnal hemoglobinuria (PNH)
Phase 2
16
Reduction of chronic hemolysis, based on LDH level at Week 13
10 patients receiving LNP023 high dose daily over up to approximately 3 years
5 patients receiving LNP023 low dose daily over up to approximately 3 years
Patients with PNH, showing signs of active hemolysis despite treatment with SoC
(defined as an antibody with anti C5 activity).
Primary: Q2-2020 (actual)
Extension: 2023
Antonio M. Risitano, MD, PhD1 et al. Presented at EBMT 2020 congress
Jan 2021Pubs: Addition of iptacopan, an oral factor B inhibitor, to eculizumab in
patients with paroxysmal nocturnal haemoglobinuria and active haemolysis: an
open-label, single-arm, phase 2, proof-of-concept trial, Risitano, Antonio M et al.
The Lancet Haematology, Volume 8, Issue 5, e344 - e354
NCT03896152 (CLNP023X2204)
Paroxysmal nocturnal hemoglobinuria (PNH)
Phase 2
13
Reduction of PNH associated hemolysis, based on percentage of patients with 60%
reduction in LDH or LDH below upper limit of normal up to 12 weeks of treatment.
approximately 2 year Treatment with low LNP023 dose
approximately 2 year Treatment with higher LNP023 dose
Patients with PNH, showing signs of active hemolysis, not treated with any other
complement inhibitor less than 3 months prior to study start Day 1
Primary: Q2-2020 (actual)
Extension: 2022
-Jang JH, et al. Presented at Korean Society of Hematology International
Conference and 62nd Annual Meeting (ICKSH 2021)
-Presented as an oral presentation (encore) at the European Haematology
Association (EHA 2021) congress
-Planned manuscript submission in Q3 2021
135 Investor Relations | Q4 2021 Results
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