- Arrowhead Pharmaceuticals Inc (NASDAQ:ARWR) presented interim data from the ongoing Phase 2 GATEWAY study of ARO-ANG3, the company’s investigational RNAi therapeutic designed to reduce expression of angiopoietin-like protein 3 (ANGPTL3), in patients with homozygous familial hypercholesterolemia (HoFH).
- HoFH is a rare condition characterized by markedly elevated circulating levels of low-density lipoprotein cholesterol (LDL-C) and accelerated, premature atherosclerotic cardiovascular disease.
- The company is planning a Phase 3 study to investigate ARO-ANG3 further and intends to meet with regulatory authorities in 2H of 2023 to discuss the proposed study design.
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- At study week 20, administration of 200 mg or 300 mg ARO-ANG3 on day one and day 84 led to the following changes:
- Mean reductions in LDL-C (Martin-Hopkins) of 48.1% and 44.0%, respectively.
- ANPTL3 inhibition with ARO-ANG3 also reduced HDL-C, non-HDL-C, and triglycerides, consistent with published human genetic data
- Overall, no newly identified patterns of adverse events in HoFH patients.
- One serious adverse event of the second-degree atrioventricular block was reported in a patient with extensive atherosclerotic cardiovascular disease history, considered unrelated to ARO-ANG3.
- ARWR Price Action: Arrowhead Pharmaceuticals shares are down 0.19% at $36.40 at publication Tuesday.
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