PASADENA, CA, USA I April 24, 2024 I Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) announced today that it has dosed the first subjects in a Phase 1/2a clinical trial (NCT06209177) of ARO-CFB, the company’s investigational RNA interference (RNAi) therapeutic, in up to 66 healthy volunteers and patients with complement mediated kidney disease.

James Hamilton, M.D., MBA, Chief of Discovery and Translational Medicine at Arrowhead, said: “In preclinical studies, ARO-CFB achieved deep and durable reductions in liver production of complement factor B, which is involved in alternative complement pathway activation and associated with pathogenesis of diseases involving complement activation. ARO-CFB is our second clinical program designed to address diseases associated with activation of the complement pathway, the first being ARO-C3, our Phase 1 program targeting complement component 3. We look forward to further progressing both programs to investigate whether these candidates can address the substantial unmet medical need that remains in the treatment of multiple complement mediated diseases.”

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 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