Arrowhead Pharmaceuticals announced interim results from its ongoing Phase I/II study of ARO-RAGE.  The treatment, an RNA interference therapeutic, is intended to lower production of the receptor for advanced glycation end products (RAGE).

The study (NCT05276570) involved comparing multiple ascending doses of ARO-RAGE in participants with mild-to-moderate asthma compared to a placebo group. Interim results demonstrated a reduction in soluble RAGE as measured in serum (after singular and two dose regime) and bronchoalveolar lavage fluid (after a single dose).

In addition, the duration of pharmacologic effects lasted for at least six weeks after a second dose. The company also reported good safety and no adverse effects. Arrowhead is yet to analyse data from the highest dose level.

RAGE is implicated in the pathogenesis of inflammatory diseases such as asthma and RNAi therapy is designed to reduce RAGE expression with the resultant termination of RAGE-dependent inflammatory pathways.

Arrowhead CEO and president, Christopher Anazolone, said that the data represents clinical validation of Arrowhead’s inhaled pulmonary TRiM platform. In February 2022, the company announced plans to build a $250m new plant to help with commercial-scale manufacturing of the platform.

“Unmet need continues to exist for many patients with severe asthma who suffer from persistent symptoms and exacerbations, despite current therapies,” said Matthias Salathe, M.D., Professor of pulmonary, critical care, and sleep medicine.

“Reducing expression of the RAGE protein in pulmonary epithelial cells to the degree that ARO-RAGE has demonstrated to date in this study has the potential to treat patients with asthma and other inflammatory lung diseases in a fundamentally new way.”

Arrowhead shares opened with a 15% rise on Wednesday morning at $34.47 compared to pre-announcement close.