Pegcetacoplan

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Cat Number

API2019171696

CAS Number

2019171-69-6

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Product Detail Description Scientific Support Customer Q&A

CAS Number

2019171-69-6

Synonyms

Pegcetacoplan acetate; APL-2

Molecular Formula

C62H100N16O23S2

Molecular Weight

1501.7

Smiles

C[C@@H]1CC=N[C@H]1C(=O)NCCCC[C@@H](C(=O)N2CCC[C@H]2C(=O)N[C@@H](CC(C)C)C(=O)N[C@@H](C)C(=O)N[C@@H](CC(=O)N)C(=O)O)NC(=O)[C@H](CS)NC(=O)[C@H](C)NC(=O)[C@H]([C@@H](C)O)NC(=O)[C@H](CCC(=O)O)NC(=O)[C@H](CS)NC(=O)CNC(=O)[C@H](CCC(=O)O)NC(=O)[C@@H]3CCCN3.CC(=O)O

Melting Point

2-8℃

General Description

Pegcetacoplan is a PEGylated, synthetic, cyclic peptide developed as a targeted inhibitor of complement C3. Pegcetacoplan has a prolonged duration of action that is able to provide sustained inhibition of immune-mediated disorders.

Mechanism of Action

Pegcetacoplan targets and binds to complement protein C3 and its activation product C3b to regulate the central node of the complement cascade. Pegcetacoplan blocks the cleavage of C3, which in turn, inhibits both the intravascular and extravascular hemolysis of red blood cells and the downstream inflammatory response. Systemic inhibition of the complement cascade reduces complement-mediated injury to healthy cells and dampens the accumulation of proteins in the kidney and eye tissues.

Application

Pegcetacoplan is indicated to treat adults with PNH and to treat geographic atrophy in patients with age-related macular degeneration. It is also indicated to treat C3 glomerulopathy and primary immune complex membranoproliferative glomerulonephritis in adults and children to reduce proteinuria.

The first C3 inhibitor Pegcetacoplan is approved for adults with paroxysmal nocturnal haemoglobinuria (PNH). Phase III PRINCE (treatment-naïve, anaemic) found Pegcetacoplan stabilised haemoglobin in 86% versus 0% with supportive care, increased mean Hb 2.9 g/dL, normalised LDH in 66% and transfusion-free in 91% at week 26. Phase III PEGASUS (patients anaemic despite eculizumab) found Pegcetacoplan raised Hb 2.4 g/dL while eculizumab alone lowered it by 1.5 g/dL; 85% were transfusion-free versus 15%, and fatigue improved in 73% versus 0%. Benefits were maintained for 48 weeks.
Pegcetacoplan blocks C3 upstream of C5, stopping intra- and extravascular haemolysis. Pegcetacoplan decreases D-dimer levels and reduces thrombosis risk. Pegcetacoplan is cost-effective when compared with C5 inhibitors. It provides a self-administered, complete complement blockade that corrects anaemia, reduces transfusions and improves the quality of life in both treatment-naïve and C5-refractory PNH.

Fig. 1 Mechanism of action of Pegcetacoplan in paroxysmal nocturnal haemoglobinuria. (Heo Y A.; et al. 2022)

References

Heo Y A, et al. Pegcetacoplan: a review in paroxysmal nocturnal haemoglobinuria. Drugs, 2022, 82(18): 1727-1735.

How does Pegcetacoplan work for PNH?

Pegcetacoplan is a complement inhibitor that works by inhibiting the complement C3 protein and preventing both intravascular and extravascular hemolysis of red blood cells.

What are the pharmacological benefits of Pegcetacoplan?

Pegcetacoplan’s PEGylated design allows for sustained activity. Additionally, inhibition of C3 allows for broad control of the complement cascade downstream.

Is Pegcetacoplan a targeted therapy?

Yes, Pegcetacoplan is a targeted biologic therapy.

What grade of Pegcetacoplan do you manufacture?

We supply pharmaceutical-grade Pegcetacoplan API that is manufactured to meet strict quality and regulatory standards.