Arrowhead Pharmaceuticals has reported positive data from a preclinical and initial clinical analysis of the AROAAT1001 trial.

AROAAT1001 is a Phase l, single and multiple-ascending dose trial designed to investigate the safety, tolerability, pharmacokinetics, and effect of ARO-AAT for the treatment of a rare genetic liver disease associated with alpha-1 antitrypsin (AAT) deficiency.

The trial features a total of seven cohorts, where 16 subjects receive placebo and 28 subjects receive single or multiple doses of ARO-AAT at doses of 35mg, 100mg, 200mg, or 300mg.

The new findings have showed that a single, open-label dose of 100mg of ARO-AAT in four subjects achieved 93% maximum serum alpha-1 antitrypsin (AAT) knockdown and 87% mean maximum serum AAT knockdown.

After eight weeks of treatment, the mean serum AAT knockdown was reported to have remained at 83%.

It was also found that the single 100mg dose of ARO-AAT is equivalent to an average dose of 1.4mg/kg in the subjects examined, who had an average weight of 72.9kg.

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“Based on our experience in primates and prior human clinical studies, we believe this knockdown level represents near full suppression of the liver production of AAT.”

ARO-AAT was also appeared to be generally well-tolerated and until the data cut-off date of 11 June 2018, no serious or severe adverse events (AEs) were observed in the 40 subjects.

Out of the 40 subjects, 24 received ARO-AAT and 16 received placebo.

Among other results, the trial did not observe any clinically meaningful adverse changes in blood urea nitrogen (BUN), creatinine, alanine aminotransferase (ALT), aspartate aminotransferase (AST) or total bilirubin.

Arrowhead Pharmaceuticals president and CEO Chris Anzalone said: “The 100mg open-label, single-dose cohort showed strong activity with a maximum serum AAT reduction of 93%.

“Based on our experience in primates and prior human clinical studies, we believe this knockdown level represents near full suppression of the liver production of AAT.

“In addition, the duration of effect we’re seeing should enable monthly or less frequent dosing.”

ARO-AAT is Arrowhead’s second-generation subcutaneously administered RNA interference (RNAi) therapeutic being developed as a treatment for a rare genetic liver disease associated with AAT deficiency.