Arrowhead Pharmaceuticals Initiates Phase 1/2a Study of ARO-INHBE for the Treatment of Obesity

-       ARO-INHBE targets a known pathway that signals the body to store fat in adipose tissue

-       In preclinical studies ARO-INHBE reduced body weight and fat mass with a novel mechanism of action that may better preserve lean muscle mass compared to currently approved obesity therapies

Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced that it has dosed the first subjects in a Phase 1/2a clinical trial of ARO-INHBE, the company’s investigational RNA interference (RNAi) therapeutic being developed as a potential treatment for obesity. Arrowhead also filed recently a request for regulatory clearance to initiate a clinical trial for its second obesity candidate, ARO-ALK7. Both ARO-INHBE and ARO-ALK7 are designed to intervene in a known pathway that signals the body to store fat in adipose tissue.

“ARO-INHBE is an important program for Arrowhead that complements our strategic focus on developing and commercializing important RNAi-based therapies for cardiometabolic diseases. Further, our preclinical studies have yielded promising results for this novel mechanism to reduce body weight and potentially preserve lean muscle mass resulting in improved body composition,” said James Hamilton, M.D., Chief of Discovery and Translational Medicine at Arrowhead. “The Phase 1/2 study will evaluate ARO-INHBE as a monotherapy in part 1 and as a combination therapy with tirzepatide in part 2, with both parts enrolling patients with obesity.”

About ARO-INHBE

ARO-INHBE is designed to reduce the hepatic expression of the INHBE gene and its secreted gene product, Activin E. INHBE is a promising genetically validated target in which loss-of-function INHBE variants in humans are associated with improved fat distribution and lower risk of metabolic diseases, such as type 2 diabetes. Activin E acts as a ligand in a pathway that regulates energy homeostasis in adipose tissue. Inhibiting this pathway with investigational ARO-INHBE treatment has the potential to increase lipolysis, and reduce adipose hypertrophy and dysfunction, visceral adiposity, and insulin resistance.

About the AROINHBE-1001 Phase 1/2 Study

AROINHBE-1001 (NCT06700538) is a Phase 1/2a dose-escalating study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of ARO-INHBE in up to 78 adult volunteers with obesity. Part 1 of the study is designed to assess single and multiple doses of ARO-INHBE monotherapy, and Part 2 of the study is designed to assess ARO-INHBE in combination with tirzepatide, a subcutaneously administered GLP-1/GIP receptor co-agonist that has been approved in the United States and the European Union for management of type 2 diabetes mellitus since 2022 and weight management since 2023/2024 respectively.

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.

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Source: Arrowhead Pharmaceuticals, Inc.

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