Arrowhead Presents Data on Pulmonary Pipeline at ERS 2023
- Arrowhead Expands the
- Late-Breaking Clinical Data for ARO-RAGE for Treatment of Asthma Demonstrate Deep and Durable Gene Silencing
- Promising Preclinical Data Across Multiple Gene Targets Suggest Potentially Broad Applications for Arrowhead’s TRiM™ Platform
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. Additional dose cohorts are ongoing. Based on interim blinded safety results from the ongoing study, ARO-RAGE has been well-tolerated to date in healthy volunteers and asthma patients.
“Today we announced additional data on multiple programs from our emerging pipeline of first-in-class pulmonary targeted RNAi therapeutic candidates at the
Arrowhead Abstracts at ERS 2023
A Clinical-Stage Inhaled RNAi Therapeutic for Pulmonary Inflammation Mediates Durable RAGE Silencing in Nonhuman Primates
Poster Session 146,
A Lung-Targeted RNAi Therapeutics Delivery Platform Compatible with Subcutaneous Administration Mediates Pulmonary RAGE Silencing in Rodents and Nonhuman Primates
Poster Session 146,
Silencing TSLP Expression with a Lung-targeted RNAi Molecule Suppresses Pulmonary Allergic Inflammation
Poster Session 146,
A First-in-human Study of ARO-RAGE, an RNAi Therapy Designed to Silence Pulmonary RAGE Expression
Late Breaking Abstract,
A Clinical-stage RNAi Therapeutic Candidate for IPF Mediates Durable MMP7 Silencing in Nonhuman Primates and Human
Poster Session 383,
Presentation slides and posters may be accessed on the Events and Presentations page under the Investors section of the Arrowhead website.
About Pulmonary TRiM™ Platform
Arrowhead’s pulmonary Targeted RNAi Molecule (TRiM™) delivery platform facilitates selective delivery of therapeutic small interfering RNAs (siRNAs) to the lung epithelium via an integrin αvβ6 targeting moiety, mediating durable gene silencing upon inhalation. The pulmonary TRiM™ delivery platform is also compatible with subcutaneous administration and is capable of effectively silencing gene expression in the lung in rodents and non-human primates. While inhaled dosing offers a convenient local delivery solution, systemic administration would offer additional flexibility for some therapeutic applications. Subcutaneous administration in humans is currently being evaluated in a clinical trial.
ARO-RAGE is an investigational RNAi therapeutic targeting the receptor for advanced glycation end-products (RAGE) as a potential treatment for inflammatory lung diseases. RAGE is implicated as an upstream mediator of Type-2 and non-Type-2 inflammatory cascades and is involved in the pathogenesis of asthma and numerous inflammatory diseases. Silencing RAGE expression via RNAi is designed to reduce the amount of RAGE protein expressed on pulmonary epithelial cells. 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.
About the ARORAGE-1001 Phase 1/2 Study
ARORAGE-1001 (NCT05276570) is a Phase 1/2a, randomized, double-blinded, placebo-controlled study in normal healthy volunteers (NHV), Part 1, and in patients with inflammatory lung disease, Part 2. The single ascending dose portion of the study includes 5 sequentially enrolled NHV cohorts with escalating single-dose levels. The multiple ascending dose portion of the study includes 5 NHV cohorts, 3 asthma patient cohorts, and 1 cystic fibrosis patient cohort. The objectives of the study include the assessment of safety and tolerability, pharmacokinetics, and pharmacodynamics of ARO-RAGE in NHVs and patients with inflammatory lung disease.
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