Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today announced that it has dosed the first subjects in two Phase 1/2a clinical trials of ARO-MUC5AC and ARO-RAGE, the company’s investigational RNA interference (RNAi) therapeutics designed to reduce production the of mucin 5AC (MUC5AC) and the receptor for advanced glycation end products (RAGE), respectively, as potential treatments for various muco-obstructive and inflammatory pulmonary diseases.

“Initiating clinical studies for ARO-MUC5AC and ARO-RAGE, Arrowhead’s next generation pulmonary targeted RNAi therapeutic candidates, is a significant milestone for the company,” said Chris Anzalone, Ph.D., president and chief executive officer of Arrowhead. “These are potentially important new medicines that address targets that have been difficult to drug using other modalities and are designed to treat muco-obstructive and inflammatory lung diseases in fundamentally new ways.”

AROMUC5AC-1001 (NCT05292950) is a Phase 1/2a, randomized, double-blinded, placebo-controlled study in up to 42 normal healthy volunteers (NHV) and up to 16 patients with moderate-to-severe asthma. The single ascending dose portion of the study includes 3 sequentially enrolled NHV cohorts with escalating single-dose levels. The multiple ascending dose portion of the study includes 3 NHV cohorts and 2 asthma patient cohorts. The objectives of the study include the assessment of safety and tolerability, pharmacokinetics, and pharmacodynamics of ARO-MUC5AC in NHVs and patients with asthma.

ARORAGE-1001 (NCT05276570) is a Phase 1/2a, randomized, double-blinded, placebo-controlled study in up to 64 NHVs and up to 16 patients with mild-to-moderate asthma. The single ascending dose portion of the study includes 4 sequentially enrolled NHV cohorts and one optional cohort treated with escalating single-dose levels. The multiple ascending dose portion of the study includes 4 NHV cohorts and 2 asthma patient cohorts. The objectives of the study include the assessment of safety and tolerability, pharmacokinetics, and pharmacodynamics of ARO-RAGE in NHVs and 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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