ARWR - Arrowhead's RNAi therapy for complement disorders shows promise in interim trial data

Arrowhead Pharmaceuticals ( NASDAQ: ARWR ) reported interim data from Part 1 of an ongoing phase 1/2 trial of ARO-C3 in healthy people as a potential therapy for complement mediated diseases.

The complement system is a part of the body's immune system.

According to the company ARO-C3, an RNA interference (RNAi) therapy, is designed to reduce production of complement component 3 (C3).

The company said that the results from part 1 of the study, dubbed AROC3-1001, showed a dose-dependent reduction in serum C3, with 88% average reduction at the highest dose which was evaluated.

A dose-dependent reduction in AH50, a marker of alternative complement pathway hemolytic activity, with 91% average reduction at the highest dose, the company added.

Hemolysis is the destruction of red blood cells faster than they can be created.

Arrowhead noted that the duration of pharmacologic effect supports a quarterly or less frequent subcutaneous dose administration.

The company added that there were no dose limiting toxicity, serious or severe adverse events or study discontinuation due to adverse events. The most common adverse events included, headache, COVID-19, mild injection site reactions and seasonal allergy.

"These data in healthy volunteers provide us with further confidence as we begin Part 2 of the study, which includes patients with various complement mediated diseases," said James Hamilton, chief of discovery and translational medicine at Arrowhead.

Part of the trial evaluated ARO-C3 in up to 42 healthy adults, while part 2 will evaluate the drug in up to 42 adult patients with paroxysmal nocturnal hemoglobinuria (PNH) or with complement-mediated kidney disease.

The company expects to begin dosing in part 2 in H1 2023.

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