Question · Q3 2025
Jeroen Werber questioned whether the reduction in acute pancreatitis (AP) events for Olzarsen would primarily be in patients with a history of AP or also prevent new events. He also asked for clarification on the estimated U.S. patient population for hereditary angioedema (HAE), noting discrepancies with other companies' estimates.
Answer
Brett Monia, CEO, explained that higher triglycerides correlate with more AP events, and patients with prior AP events have a higher chance of recurrence, consistent with the data. He emphasized that the 12-month study duration makes the results even more remarkable. Kyle Jenne, Chief Global Product Strategy Officer, confirmed Ionis's estimate of 7,000 HAE patients in the U.S., with 75% on prophylactic therapy, and stated the focus is on the switch market, not being aware of an 11,000 patient estimate.