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.
Tyra Biosciences doses first patient in TYRA-300 Phase 2 study for bladder cancer
argenx advances ARGX-119 to registrational study for congenital myasthenic syndromes
hVIVO supports Cidara Therapeutics' positive Phase 2b influenza study results
Sanofi's riliprubart receives orphan drug designation in Japan for CIDP
HUTCHMED gains China approval for ORPATHYS and TAGRISSO combination in lung cancer
Hikma Pharmaceuticals USA announces USD1bn of new US investment
UCB reports positive Phase 3 results for fenfluramine in CDKL5 deficiency disorder