As previously reported, SVB Leerink analyst Mani Foroohar initiated coverage of Arrowhead (ARWR) with an Underperform rating and $32 price target. The analyst notes that safety profile and regulatory path for lead asset ARO-AAT remains uncertain, and competitor data from Vertex (VRTX) is an existential threat to the program. Non-dilutive financing and potential royalties from the Johnson & Johnson (JNJ) partnership are more than fully valued at current levels, he adds. With a tough catalyst path and current valuation baking in substantial technology value despite very few patient-years of exposure to the TRiM platform, Foroohar sees the next 12 months as far more challenging than the past two years as Arrowhead faces sky-high expectations in the face of increasingly clear competitive headwinds.

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.

Vertex Pharmaceuticals and TreeFrog Therapeutics announced that Vertex has obtained an exclusive license to TreeFrog's proprietary cell manufacturing technology, C-Stem, to optimize production of Vertex's cell therapies for type 1 diabetes, or T1D. TreeFrog and Vertex will collaborate to scale-up TreeFrog's process to produce and amplify cells for Vertex's T1D therapies. TreeFrog's proprietary technology platform, C-Stem, is designed to mimic the natural microenvironment, allowing cells to grow exponentially in 3D. The technology will enhance Vertex's ability to generate large amounts of fully differentiated cells for its portfolio of T1D cell therapies.