Arrowhead highlights potential of RNAi in asthma at ERS 23

By Isabel Cameron

- Last updated on GMT

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© Getty Images

Related tags RNAi Asthma Clinical trial Lung

Arrowhead Pharmaceuticals has spotlighted the potential of RNA interference (RNAi) therapeutic candidates in the lungs, with a clinical late breaker and multiple pre-clinical presentations at this year’s European Respiratory Society (ERS) International Congress 2023 in Milan, Italy.

The company’s late-breaking abstract includes new data from its Phase 1 program evaluating ARO-RAGE for the treatment of asthma. Previously, the company has shown that a single inhaled dose of ARO-RAGE led to a 90% reduction in a circulating form of target in patients with asthma.   

At ERS, the company also presented four preclinical posters on multiple candidates across indications, including asthma, idiopathic pulmonary fibrosis, allergic inflammation, and others. 

In an ongoing phase 1/2 clinical trial, ARO-RAGE, Arrowhead’s investigational RNAi therapeutic for the treatment of inflammatory lung diseases, achieved mean target gene knockdown of up to 90% with a maximum of 95% after a single inhaled administration. 

RNAi is a promising strategy to target disease-causing genes. However, delivery to the lung has historically posed a significant challenge for the field.

According to Arrowhead, its continued progress suggests it may have unlocked the potential of RNAi to treat diseases of the lung. 

Interim results from the healthy volunteer portion of the ARORAGE-1001 phase 1/2 clinical trial show that ARO-RAGE reduced soluble RAGE (sRAGE) concentration in bronchoalveolar lavage fluid (BALF) and serum in a dose-dependent manner.

Reduction of serum sRAGE was similar in healthy volunteers and in patients with asthma at the 44 mg dose level.

Based on interim blinded safety results from the ongoing study, ARO-RAGE has been well-tolerated to date in healthy volunteers and asthma patients. 

Javier San Martin, chief medical officer at Arrowhead, said: “Today we announced additional data on multiple programs from our emerging pipeline of first-in-class pulmonary targeted RNAi therapeutic candidates at the European Respiratory Society International Congress. Clinical results from the ARO-RAGE phase 1/2 study are highly encouraging and demonstrate a significant level of target gene knockdown with a duration of effect that may enable a long dosing interval possibly up to once every two months.”

“In addition, the preclinical data presented at ERS suggest that the TRiM platform can achieve compelling results across multiple additional gene targets in the lung, including MUC5AC, MMP7, and our newest program against thymic stromal lymphopoietin (TSLP), a clinically well validated target.” 

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