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Arrowhead Pharmaceuticals (NASDAQ:ARWR) announced Tuesday that patients with a rare form of hypercholesterolemia indicated a 44% – 48% reduction of LDL-Cholesterol, also known as “bad” cholesterol, after receiving its experimental RNAi therapeutic ARO-ANG3.
Citing interim data from its ongoing Phase 2 GATEWAY study, Arrowhead (ARWR) said that the results support its plans to further investigate ARO-ANG3 in a Phase 3 study for the condition known as homozygous familial hypercholesterolemia ((HoFH)).
The company plans to conduct a Phase 3 trial for ARO-ANG3 and intends to engage regulators in H2 2023 to discuss a proposed study design.
“Despite advances in the treatment of cardiovascular disease associated with high LDL-cholesterol, patients with HoFH have a particularly high need for additional therapy,” the chief medical officer of Arrowhead (ARWR) Javier San Martin remarked.
HoFH is a rare form of familial hypercholesterolemia, that can cause early indications of coronary artery disease if untreated.
Arrowhead’s (ARWR) GATEWAY study for 18 patients demonstrated that the administration of ARO-ANG3 at 200 mg or 300 mg on day one and day 84 caused a 48.1% and 44.0% decline in LDL-C at Week 20, respectively.
There were no discontinuations and dose interruptions due to treatment-emergent adverse events. However, there was an incidence of atrioventricular block that investigators classified as a serious adverse event unrelated to ARO-ANG3.
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