Piper Jaffray analyst Joshua Schimmer says that while Ionis Pharmaceuticals' (IONS) Phase 3 results for the NEURO-TTR study met both primary endpoints, the safety findings confirmed concerns about thrombocytopenia sequelae and also brought to light renal serious adverse event. The renal toxicity has been a platform concern, historically, "but we are surprised to see it pop up again considering the company's dismissal of it as a concern," Schimmer tells investors in a research note. He notes, however, that no other events occurred since Ionis implemented more intensified platelet and renal monitoring. Schimmer believes the safety issues are "not insurmountable" and that IONIS-TTRrx is still approvable. The analyst maintains an Overweight rating on Ionis with a $51 price target. The stock in early trading is down $4.05 to $43.03. Competitor Alnylam (ALNY) is trading up 14% to $64.58.

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).