13 March 2018 - - US-based RNAi-based therapeutics developer Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) has dosed the first subjects in a Phase 1 clinical study of ARO-AAT, which is being developed as treatment for a rare genetic liver disease associated with alpha-1 antitrypsin deficiency, the company said.
ARO-AAT is the first clinical candidate to utilize Arrowhead's proprietary Targeted RNAi Molecule (TRiM) technology. The second candidate, ARO-HBV, which is being developed as a potentially curative therapy for patients with chronic hepatitis B virus infection, is also on schedule to dose the first subjects in a Phase 1/2 study at the end of March.
The AROAAT1001 study is a Phase 1 single- and multiple-ascending dose study to evaluate the safety, tolerability, pharmacokinetics, and effect of ARO-AAT on serum alpha-1 antitrypsin levels in healthy adult volunteers.
The study is designed to include up to 5 cohorts of 8 subjects per cohort who will receive placebo or ARO-AAT at doses of 35, 100, 200, 300, or 400 mg.
Arrowhead Pharmaceuticals develops medicines that treat intractable diseases by silencing the genes that cause them. The company's therapies trigger the RNA interference, or RNAi, mechanism to induce rapid, deep, and durable knockdown of target genes.
RNAi, present in living cells, inhibits the expression of a specific gene, thereby affecting the production of a specific protein. Arrowhead's RNAi-based therapeutics leverage this natural pathway of gene silencing.