Arrowhead Receives Regulatory Clearance to Begin Phase 1 Study of ARO-AAT for Treatment of Alpha-1 Liver Disease
- Category: DNA RNA and Cells
- Published on Thursday, 22 February 2018 17:31
- Hits: 638
PASADENA, CA, USA I February 22, 2018 I Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today announced that it has received approval from the New Zealand Medicines and Medical Devices Safety Authority (MEDSAFE) and from the local Ethics Committee to proceed with a first-in-human study of ARO-AAT, which is being developed as treatment for a rare genetic liver disease associated with alpha-1 antitrypsin deficiency. Arrowhead anticipates that dosing in the Phase 1 study will begin around the end of March.
The study, AROAAT1001 (NCT03362242), 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.
About Arrowhead Pharmaceuticals
Arrowhead Pharmaceuticals develops medicines that treat intractable diseases by silencing the genes that cause them. Using a broad portfolio of RNA chemistries and efficient modes of delivery, Arrowhead therapies trigger the RNA interference mechanism to induce rapid, deep, and durable knockdown of target genes. RNA interference, or RNAi, is a mechanism present in living cells that 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.
SOURCE: Arrowhead Pharmaceuticals