Arrowhead presents clinical data from Phase 1 study of ARO-AAT

Arrowhead Pharmaceuticals announced clinical data from a Phase 1 study of ARO-AAT, the company’s second generation subcutaneously administered RNA interference therapeutic being developed as a treatment for a rare genetic liver disease associated with alpha-1 antitrypsin deficiency, will be presented in a late-breaking poster at The Liver Meeting, the Annual Meeting of the American Association for the Study of Liver Disease, being held in San Francisco. In the AROAAT1001 study, 45 normal healthy volunteers received a single dose of ARO-AAT, three monthly doses of ARO-AAT or placebo. Key data presented include the following: ARO-AAT at single- and multiple-doses produced robust and consistent reductions in serum AAT levels. Single-doses of 200 and 300 mg resulted in greater than 91% serum AAT reduction, with 3 of 4 subjects having concentrations below the level of quantitation. In 200 and 300 mg single-dose cohorts, an average serum AAT reduction of greater than 90% was sustained for 6 weeks. In the multiple-dose cohorts of 200 and 300 mg, for subjects receiving all 3 doses, an average of greater than 90% reduction in serum AAT was sustained for longer than 14 weeks. The maximum NADIR reduction is 94%. Monthly serum AAT follow up is ongoing with 9 of 10 subjects at BLQ in the multiple-dose cohorts, including 100% of subjects from the 300 mg cohort. Duration of response indicates that quarterly or less frequent dosing appears feasible. ARO-AAT has been well tolerated at all doses tested given three times every 28 days. The most common adverse events were upper respiratory tract infection and headache.
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