After Alnylam (ALNY) announced that it was discontinuing one of its Phase III RNAi drugs due to poor clinical outcomes, RBC Capital analyst Michael Yee does not thinks that the news indicates problems with all RNAi drugs. Rather, he thinks that RNAi was not the proper treatment for the disease that Alnylam was targeting. Other companies with RNAi treatments include The Medicines Company (MDCO0, Ionis Pharmaceuticals (IONS), Arrowhead (ARWR), and Arbutus Biopharma (ABUS).

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.

ProQR Therapeutics (PRQR) announced the Annual General Meeting, AGM, of Shareholders will take place on Wednesday, May 22, 2024 at the offices of Allen & Overy LLP, in Amsterdam, the Netherlands. As part of the AGM, Martin Maier, PhD, is nominated for appointment as a non-executive Board Member for a period of four years. Dr. Maier is Senior Vice President Research heading the Oncology group at Alnylam Pharmaceuticals (ALNY).