Arrowhead Pharmaceuticals said that interim data from the ARORAGE-1001 Phase 1/2a trial of its ARO-RAGE inhaled RNAi therapeutic, which it is developing for the treatment of inflammatory lung diseases, demonstrate reductions in soluble receptor for advanced glycation end products (RAGE) as high as 90% in healthy volunteers at the 4th highest dose with no indications of safety or tolerability issues and with effects lasting at least 6 weeks post-dose. Arrowhead said that no data are available yet for the highest dose of the inhalation solution.
The trial is expected to enroll a total of 80 subjects, with the SAD portion of the study evaluating 5 dose levels from 10 mg to 184 mg in healthy volunteers and the MAD portion of the study including asthma patients in addition to healthy volunteers.
According to Arrowhead, “Reduced RAGE expression in the pulmonary epithelium may result in reduction of RAGE-dependent inflammatory pathways, leading to decreased exacerbation frequency and improved airflow in patients with asthma.” The company’s web site also lists RNAi therapeutics targeted at the epithelial sodium channel alpha subunit, mucin 5AC, matrix metalloproteinase 7, and coronaviruses in its pipeline and says that it aims to have 20 drugs in clinical development or marketed by 2025.
Arrowhead President and CEO Christopher Anzalone commented, “We think these interim data with ARO-RAGE represent clinical validation of Arrowhead’s inhaled pulmonary TRiM platform and, specifically, of ARO-RAGE as a potential new therapy to treat patients with inflammatory lung diseases. The high level of target gene knockdown, the long duration of effect, and the promising safety and tolerability results are all very encouraging signs for our growing pipeline of RNAi therapeutic candidates that leverage this same platform. We look forward to providing additional data at our upcoming R&D Day on June 1, 2023, and at future medical meetings.”
Read the Arrowhead Pharmaceuticals press release.
