Arrowhead Files for Regulatory Clearance to Initiate Phase 1/2a Study of ARO-RAGE for Treatment of Asthma

Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today announced that it has filed an application for clearance to initiate a Phase 1/2a clinical trial of ARO-RAGE, the company’s investigational RNA interference (RNAi) therapeutic designed to reduce production of the receptor for advanced glycation end products (RAGE) as a potential treatment for asthma and other inflammatory pulmonary diseases.

James Hamilton, M.D., MBA, senior vice president of discovery and translational medicine at Arrowhead, said: “ARO-RAGE is a new clinical candidate using the pulmonary targeted, inhaled TRiM™ platform. We believe RAGE is a promising target as it represents an upstream mediator of the inflammatory cascade in asthma. Importantly, a soluble form of RAGE known as sRAGE can be assessed as a useful serum biomarker to measure gene target knockdown. We hope to further investigate the potential of ARO-RAGE and our pulmonary platform broadly in the upcoming clinical study, including assessments of safety, pharmacokinetics, and target engagement in a non-invasive way. These insights can potentially help to inform the development path of future pulmonary candidates.”

An application for approval of the clinical trial was submitted to a local Ethics Committee and to the New Zealand Medicines and Medical Devices Safety Authority for review by the Standing Committee on Therapeutic Trials. Pending clearance, Arrowhead intends to proceed with ARORAGE-1001, a Phase 1/2a study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of ARO-RAGE in up to 64 healthy volunteers and in up to 16 patients with asthma.

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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