Iptacopan (LNP023) 是一种首创的，有效的，具有口服活性的高选择性的 factor B 抑制剂，IC₅₀ 值为 10 nM。Iptacopan 直接可逆且高亲和力结合 factor B，K_D为 7.9 nM。Iptacopan 可靶向 C3 肾小球病的根本病因。

Iptacopan (LNP023) is a first-in-class, orally bioavailable, highly potent and highly selective factor B inhibitor with an IC 50 value of 10 nM. Iptacopan shows direct, reversible, and high-affinity binding to human factor B with a K D of 7.9 nM. Iptacopan targets the underlying cause of complement 3 glomerulopathy (C3G).

Solid

KD: 7.9 nM (factor B)
IC50: 10 nM (factor B)

Iptacopan (LNP023) demonstrates potent inhibition of alternative complement pathway (AP)-induced membrane attack complex (MAC) formation in 50% human serum (IC50 value of 130 nM).
Iptacopan (LNP023) exhibits excellent selectivity over other proteases affording IC50 values of >30 μM across a panel of 41 human proteases, including the AP protein factor D (>100 μM). has not independently confirmed the accuracy of these methods. They are for reference only.

Iptacopan (LNP023; 20-180 mg/kg; oral administration) prevents KRN (150 μL)-induced arthritis in mice and is effective upon prophylactic and therapeutic dosing in an experimental model of membranous nephropathy in rats.
LNP023 exhibits moderate half-lives (T 1/2 ; Wistar Han rats 3.4 h, beagle dogs 5.5 h) and C max (Wistar Han rats 410 nM, beagle dogs 2200 nM) following oral administration (rat 30 and, dog 10 mg/kg).
Iptacopan exhibits terminal elimination half-lives (T 1/2 ; Wistar Han rats 7 h, beagle dogs 5.6 h) due to high plasma clearance (8, and 2 mL/min/kg respectively combined with large volumes of distribution (2.3, and 0.6 L/kg respectively) following intravenous administration (rat 1.0 and, dog 0.1 mg/kg). has not independently confirmed the accuracy of these methods.

Room temperature in continental US; may vary elsewhere.

Powder -20°C 3 years；4°C 2 years

[1]. Dimitrios C Mastellos, et al. Expanding Complement Therapeutics for the Treatment of Paroxysmal Nocturnal Hemoglobinuria. Semin Hematol. 2018 Jul;55(3):167-175.
[2]. Anna Schubart, et al. Small-molecule Factor B Inhibitor for the Treatment of Complement-Mediated Diseases. Proc Natl Acad Sci U S A. 2019 Apr 16;116(16):7926-7931.

In Vitro: DMSO : 50 mg/mL (118.34 mM; Need ultrasonic)配制储备液