Efficacy, Safety, Pharmacokinetics and Pharmacodynamics Study, Assessing Multiple LNP023 Doses in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria

Summary

This was a Phase II randomized, open-label, multicenter, efficacy, safety, pharmacokinetic and pharmacodynamic study assessing four iptacopan doses in adult Paroxysmal nocturnal hemoglobinuria (PNH) patients with active hemolysis who were not on eculizumab or any other complement inhibitor less than 3 months prior to first iptacopan dose. Active hemolysis was defined by a lactate dehydrogenase (LDH) value ≥ 1.5 × ULN.

Timeline

Start

2019-04-05

Primary completion

2020-04-07

Completion

2022-02-09

Outcome

Met primary endpoint

paper all 12 evaluable for efficacy achieved the primary endpoint of reduction in serum lactate dehydrogenase (LDH) levels by ≥60% by week 12 compared with baseline PMID 35561315 ↗

release “achieved the primary endpoint of at least a 60% reduction in their lactate dehydrogenase (LDH) levels” novartis.com ↗

Publications

• Results Jang JH, Wong L, Ko BS, Yoon SS, Li K, Baltcheva I, Nidamarthy PK, Chawla R, Junge G, Yap ES. Iptacopan monotherapy in patients with paroxysmal nocturnal hemoglobinuria: a 2-cohort open-label proof-of-concept study. Blood Adv. 2022 Aug 9;6(15):4450-4460. doi: 10.1182/bloodadvances.2022006960.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Iptacopan	Small molecule	100 mg	Oral
Subject	Iptacopan	Small molecule	200 mg	Oral

Indications