Arrowhead Pharmaceuticals, Inc. announced that it has dosed the first subjects in a Phase 1/2a clinical trial of ARO-ALK7, the company's investigational RNA interference (RNAi) therapeutic being developed as a potential treatment for obesity. ARO-ALK7 is designed to intervene in a known pathway that signals the body to store fat in adipose tissue. The study initiates in otherwise healthy obese subjects using single and multiple escalating doses of ARO-ALK7 monotherapy and is expected to progress rapidly to investigate combinations of ARO-ALK7 with tirzepatide in obese patients with and without type 2 diabetes.
Both programs have strong genetic validation and promising results in preclinical studies, which suggest that silencing the respective genes may lead to reduced body weight and potentially preserve lean muscle mass resulting in improved body composition.
Arrowhead Pharmaceuticals, Inc. develops medicines that treat intractable diseases by silencing the genes that cause them. The Company's therapies, using a broad portfolio of ribonucleic acid (RNA) chemistries and modes of delivery, trigger the RNA interference (RNAi) mechanism to induce rapid, deep and durable knockdown of target genes. Its Targeted RNAi Molecule (TRiMTM) platform utilizes ligand-mediated delivery and is designed to enable tissue-specific targeting while being structurally simple. It is focused on various therapeutic areas, such as cardiometabolic, pulmonary, liver, neuromuscular and others. It has over 18 discovered drug candidates in clinical trials ranging from early stage (Phase I) to late stage (Phase III). The Company's pipeline products include plozasiran, zodasiran, olpasiran, ARO-RAGE, SRP-1002, GSK4532990, fazirsiran, daplusiran/tomligisiran, ARO-CFB, ARO-INHBE, ARO-C3, ARO-PNPLA3, SRP-1001 (ARO-DUX4), SRP-1003 (ARO-DM1), SRP-1004 (ARO-ATXN2), and others.
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