Press Release Details
Arrowhead Pharmaceuticals Presents Interim Clinical Data on ARO-CFB for the Treatment of Complement Mediated Diseases
- Interim data from Phase 1/2a study demonstrate near complete inhibition in hemolytic activity and functional activity of alternative complement pathway
“Dysregulated activation of the complement system can lead to progression of certain renal diseases, either by playing a directly pathogenic role, or by amplifying or exacerbating the inflammatory and damaging impact of non-complement disease triggers. In a Phase 1/2a clinical study, ARO-CFB treatment in healthy volunteers achieved deep and durable reductions in the liver production of complement factor B (CFB), which is involved in alternative complement pathway activation and associated with pathogenesis of diseases involving complement activation. Circulating levels of CFB protein were reduced by a mean of up to 90% to date, with additional data from higher doses levels pending, and a duration of response greater than 3 months,” said
Select ARO-CFB Results
In the ongoing AROCFB-1001 study, ARO-CFB achieved the following key results in normal healthy volunteers as of the interim data cutoff -
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ARO-CFB led to dose dependent reductions in circulating CFB protein by up to 90% with greater than 3 months duration
- 90% mean reduction achieved after a single dose of 400 mg
- 90% mean reduction achieved after two doses of 100 mg
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Single and multiple doses of ARO-CFB led to near complete inhibition of alternative pathway activity based on Wieslab AP
- 100% mean reduction achieved by week 4 after a single dose at both 200 mg and 400 mg doses
- 92% and 100% mean reductions were achieved after two doses at 100 mg and 200 mg, respectively
- Single and multiple doses of ARO-CFB led to near complete inhibition of alternative pathway hemolytic activity, measured by AH50
Safety and Tolerability Results
ARO-CFB has been generally well-tolerated to date with safety data supportive of further clinical development. There have been no treatment emergent adverse events (TEAE) leading to study or study drug discontinuation with most TEAEs being mild in severity.
About ARO-CFB
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. ARO-CFB is being developed as a potential treatment for complement mediated kidney diseases 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.
About the AROCFB-1001 Phase 1/2 Study
AROCFB-1001 (NCT06209177) is an ongoing Phase 1/2a dose-escalating study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of ARO-CFB in up to 66 normal healthy volunteers (NHV) and patients with complement mediated kidney disease. In Part 1 of the study, NHVs will receive either one or two doses of ARO-CFB or placebo. In Part 2 of the study, adult patients with IgAN will receive 3 open-label doses of ARO-CFB. The study is designed to assess safety and tolerability and key pharmacodynamic parameters, including the change and percent change from baseline over time in serum CFB, and alternative complement pathway activity via AH50 and Wieslab AP.
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