Arrowhead Pharmaceuticals has completed patient enrolment in the Phase l AROAAT1001 trial of ARO-AAT for Treatment of Alpha-1 Liver Disease.
The single and multiple-ascending dose clinical trial is designed to investigate the safety, tolerability, pharmacokinetics, and effect of ARO-AAT on serum alpha-1 antitrypsin levels in healthy adult subjects.
It comprises 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.
Arrowhead also planned to include additional cohorts at a dose of 400mg, but terminated the plan after the maximal activity appeared to take place at lower doses than expected.
The primary endpoint of the AROAAT1001 trial is the number of participants with adverse events (AEs) possibly or probably related to treatment.
The secondary endpoints include pharmacokinetics (PK), percentage change in serum alpha-1 antitrypsin (aat) levels and duration of response of serum AAT levels.
The trial is expected to be completed by February next year.
Arrowhead Pharmaceuticals president and CEO Chris Anzalone said: “The ARO-AAT Phase l study provides the first readout on pharmacologic activity for a therapeutic leveraging our proprietary Targeted RNAi Molecule, or Trim , platform.
“We have escalated above a dose that we believe achieves maximal activity, and all doses to date appear to be generally well-tolerated.”
ARO-AAT is an investigational subcutaneously administered RNA interference (RNAi) therapeutic that could be used as a treatment for a rare genetic liver disease associated with alpha-1 antitrypsin deficiency.