Ionis Pharmaceuticals, Inc.

Our Company invents medicines for people with serious diseases, focusing on RNA-targeted therapies. We operate as a fully integrated commercial-stage biotechnology company with seven marketed medicines. TRYNGOLZA (olezarsen) is approved in the U.S. as an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome (FCS), in the EU for patients with genetically confirmed FCS, and in Canada. It is the first FDA-approved treatment to significantly reduce triglyceride levels and provide clinically meaningful reduction in acute pancreatitis events in adults with FCS when used with an appropriate diet (≤20 grams of fat per day). We independently commercialize TRYNGOLZA in the U.S. DAWNZERA (donidalorsen) is approved in the U.S. for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients 12 years of age and older, and in the EU for the routine prevention of recurrent attacks of HAE in the same age group. It is the first and only approved RNA-targeted prophylactic therapy for HAE. We independently commercialize DAWNZERA in the U.S. WAINUA (eplontersen) is approved in the U.S., EU, China, and numerous other countries for the treatment of the polyneuropathy of hereditary transthyretin-medicated amyloidosis (ATTRv-PN) in adults. WAINUA is the only approved medicine for ATTRv-PN that can be self-administered via an auto-injector. We and AstraZeneca co-develop WAINUA globally and co-commercialize it in the U.S. Our other marketed medicines include SPINRAZA (nusinersen), QALSODY (tofersen), TEGSEDI (inotersen), and WAYLIVRA (volanesorsen).