William Blair analyst Y. Katherine Xu upgraded Arrowhead Pharmaceuticals to Outperform from Market Perform and raised her fair value estimate for the shares to $6 from $2. The stock closed Friday up 51c to $3.30. Last week's R&D day revealed the TRiM platform for developing novel RNA interference medicines and promised five investigational new drug or clinical trial application filings in 2018, Xu tells investors in a research note. The analyst highlights "versatility" of the company's TRiM platform for designing decorated RNAi drugs.

Arrowhead Pharmaceuticals announced that it has dosed the first subjects in a Phase 1/2a clinical trial, NCT06209177 or 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.