What Arrowhead Pharmaceutical's Interim Data Reveals About A RNAi Therapeutic For Rare Cholesterol Disorder

• Arrowhead Pharmaceuticals Inc 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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