Arrowhead Files for Regulatory Clearance to Initiate Phase 1/2a Study of ARO-MUC5AC for Treatment of Muco-Obstructive Lung Diseases

– Company to Host Pulmonary R&D Day on May 26, 2022

PASADENA, Calif.–(BUSINESS WIRE)–$arwr–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-MUC5AC, the company’s investigational RNA interference (RNAi) therapeutic designed to reduce production of the mucin 5AC (MUC5AC) as a potential treatment for asthma and various muco-obstructive pulmonary diseases. Arrowhead also announced plans to host a pulmonary R&D day on May 26, 2022, to be held in New York and webcast online. Additional details about the event and how to register will be released in the coming weeks.

James Hamilton, M.D., MBA, senior vice president of discovery and translational medicine at Arrowhead, said: “We continue to rapidly advance a robust pipeline of product candidates that leverage our inhaled pulmonary TRiMTM platform. The newest candidate, ARO-MUC5AC, is designed to specifically silence pulmonary epithelial MUC5AC expression, which is highly upregulated in the asthmatic airway and implicated in the development of mucus plugging, airflow obstruction, and clinical exacerbations in multiple muco-obstructive diseases. Targeting mucus via inhibition of MUC5AC expression represents a fundamentally new way of treating muco-obstructive diseases, where current standard of care is dominated by bronchodilators and anti-inflammatory modalities. We look forward to sharing additional information about ARO-MUC5AC, ARO-RAGE, and Arrowhead’s pulmonary platform broadly at our upcoming pulmonary R&D day.”

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 AROMUC5AC-1001, a Phase 1/2a study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of ARO-MUC5AC in up to 42 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.


Arrowhead Pharmaceuticals, Inc.

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Brian Ritchie


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Josephine Belluardo, Ph.D.


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