New positive donidalorsen data to be presented at 2024 American College of Allergy, Asthma & Immunology (ACAAI) Annual Scientific Meeting

Core Insights - Ionis Pharmaceuticals announced new three-year data from the Phase 2 open-label extension study and pivotal Phase 3 studies of donidalorsen, demonstrating its potential to significantly reduce hereditary angioedema (HAE) attacks with sustained effects over time [1][2] Group 1: Donidalorsen Efficacy - Donidalorsen has shown significant reductions in HAE attacks, leading to high levels of disease control and clinically meaningful improvements in quality of life for a vast majority of patients, sustained for up to three years [2] - The presentations at the 2024 ACAAI Annual Scientific Meeting will cover various aspects of donidalorsen, including patient-reported outcomes and safety and efficacy after switching to donidalorsen [2] Group 2: Background on HAE - Hereditary angioedema (HAE) is a rare genetic condition characterized by recurrent severe swelling attacks, affecting over 20,000 patients in the U.S. and Europe [3] - Prophylactic treatment approaches are commonly used by doctors in the U.S. to prevent and reduce the severity of HAE attacks [3] Group 3: About Donidalorsen - Donidalorsen is an investigational RNA-targeted medicine aimed at reducing prekallikrein (PKK) production, which is crucial in activating inflammatory mediators associated with HAE attacks [4] - If approved, donidalorsen could serve as an effective prophylactic treatment for HAE [4] Group 4: Company Overview - Ionis Pharmaceuticals has been innovating medicines for serious diseases for three decades, with a leading pipeline in neurology, cardiology, and other high-need areas [5] - The company is recognized as a pioneer in RNA-targeted medicines and continues to advance new approaches in gene editing [5]