Arrowhead Pharmaceuticals has submitted an application seeking regulatory clearance in New Zealand to commence a Phase I/IIa clinical trial of ARO-CFB, to treat complement mediated renal disease.
The filing was made to a local Ethics Committee and the New Zealand Medicines and Medical Devices Safety Authority to be reviewed by the Standing Committee on Therapeutic Trials.
An investigational RNA interference (RNAi) therapeutic, ARO-CFB is in development to treat complement mediated renal disease, including immunoglobulin A nephropathy (IgAN).
Subject to regulatory clearance, the company plans to advance the dose-escalating trial by enrolling up to 66 healthy subjects and complement mediated kidney disease patients.
Dubbed AROCFB-1001, the trial will assess the safety, tolerability, pharmacokinetics and pharmacodynamics of ARO-CFB.
Arrowhead Pharmaceuticals Discovery and Translational Medicine chief James Hamilton 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 TRiM 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.”
Apart from renal diseases, ARO-CFB could possess potential for clinical applications in various non-renal ailments that include complement activation.
ARO-CFB works by cutting down complement factor B's (CFB) hepatic expression.
CFB has a key role in boosting complement alternative pathway activation and was detected to be an encouraging therapeutic target.
In November this year, the company sought regulatory clearance to commence a Phase I/IIa trial of ARO-DM1 to potentially treat type 1 myotonic dystrophy (DM1).
