PASADENA, CA, USA I December 21, 2023 I Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today announced that it has filed an application for clearance to initiate a Phase 1/2a clinical trial of ARO-CFB, the company’s investigational RNA interference (RNAi) therapeutic being developed as a potential treatment for complement mediated renal disease, such as immunoglobulin A nephropathy (IgAN), which is the most common glomerular disease worldwide and carries a high lifetime risk of progression to end-stage renal disease. Additionally, ARO-CFB may have clinical applications in non-renal diseases involving complement activation.

ARO-CFB is designed to reduce hepatic expression of complement factor B (CFB), which plays an important regulatory role in amplifying complement alternative pathway activation and has been identified as a promising therapeutic target.

James Hamilton, M.D., MBA, Chief of Discovery and Translational Medicine at Arrowhead, said: “IgAN is the most common glomerular disease worldwide, accounting for up to 40% of all cases of glomerulonephritis. Our preclinical studies have demonstrated that ARO-CFB can achieve deep and durable reductions in liver production of CFB, which plays a key role in the activation of the alternative complement pathway associated with the pathogenesis of renal disease in IgAN. As the liver is the main site of CFB production and our TRiMTM system has consistently demonstrated the ability to silence liver production of target gene products across several candidates, we believe ARO-CFB has the potential to be a powerful treatment option for complement mediated renal disease.”

An application for approval of the clinical trial was submitted to a local Ethics Committee and to the New Zealand Medicines and Medical Devices Safety Authority for review by the Standing Committee on Therapeutic Trials. Pending clearance, Arrowhead intends to proceed with AROCFB-1001, a Phase 1/2a dose-escalating study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of ARO-CFB in up to 66 healthy volunteers and patients with complement mediated kidney disease.

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