On Today's Earnings Call Q3:23 Business Update and Financial Results 2 Brett Monia, Ph.D. Chief Executive Officer Beth Hougen Chief Financial Officer Eugene Schneider, M.D. Chief Clinical Development Officer Eric Swayze, Ph.D. Executive Vice President, Research IONIS Forward-Looking Statements 3 This presentation includes forward-looking statements regarding our business, financial guidance and the therapeutic and commercial potential of QALSODYTM (tofersen), SPINRAZA® (nusinersen), TEGSEDI® (inotersen), WA ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, DC 20549 Form 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the Quarterly Period Ended September 30, 2023 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF SECURITIES EXCHANGE ACT OF 1934 For the transition period from _____ to _____ Commission file number 000-19125 Ionis Pharmaceuticals, Inc. (Exact name of Registrant as specified in its charter) Delaware 33-0336973 (State or ...

Eugene Schneider Sure. Happy to. We're obviously going to also look at responder -- a variety of responder analysis. So looking at proportions of patients that get below particular clinically meaningful thresholds, thresholds like 80 for instance, [is the classic 400, 500]. So again, achieving those reductions that are thought to be meaningful in terms of their risk for having acute pancreatitis events is something that we're -- and future prescribers will be very keen to see. We're also looking at quality ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, DC 20549 Form 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the Quarterly Period Ended June 30, 2023 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF SECURITIES EXCHANGE ACT OF 1934 For the transition period from _____ to _____ Commission file number 000-19125 Ionis Pharmaceuticals, Inc. (Exact name of Registrant as specified in its charter) Delaware 33-0336973 (State or othe ...

Robust Efficacy Anticipated with Pelacarsen in Phase 3 Lp(a) HORIZON Study QALSODY SOD1-ALS (SOD1) SPINRAZA SMA (SMN2) ION859 Parkinson's disease (LRRK2) ION464 Parkinson's disease and Multiple System Atrophy (alpha-synuclein) ION363 FUS-ALS (FUS) ION541 ALS (ATXN2) ION373 Alexander disease (GFAP) Eplontersen ATTR (TTR) ION582 Angelman syndrome (UBE3A-ATS) Tominersen Huntington's disease (HTT) ION260 Spinocerebellar Ataxia Type 3 (ATXN3) ION306 SMA (SMN2) IONIS-MAPTRx Alzheimer's disease (Tau) 24 MAA Accept ...

Onaiza Cadoret - Chief Global Product Strategy & Operations Officer Wade Walke We also continue to make really good progress in our pivotal SHTG studies of olezarsen. With donidalorsen, we're on track to complete enrollment in the Phase 2 OASIS HAE study soon, keeping us on track for data in the first half of next year. Additionally, our robust late-stage pipeline continues to expand, now at seven programs advancing in nine separate indications with the initiation of Phase 3 development of bepirovirsen in c ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, DC 20549 Form 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the Quarterly Period Ended March 31, 2023 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF SECURITIES EXCHANGE ACT OF 1934 For the transition period from _____ to _____ Commission file number 000-19125 Ionis Pharmaceuticals, Inc. (Exact name of Registrant as specified in its charter) Delaware 33-0336973 (State or oth ...

• Advanced new MsPA backbone chemistry with enhanced duration into preclinical development • Advance new routes of delivery 1. Non-GAAP – please see reconciliation to GAAP in FY 2022 press release 2. $500M from royalty monetization transaction in January 2023 15 million Establish an integrated commercial organization | --- | --- | --- | |------------------------------------------------------------|---------------------------------------------------------------------------------|----------------------------- ...

Wade Walke - SVP, IR Brett Monia - CEO Beth Hougen - CFO Eugene Schneider - Chief Clinical Development Officer Onaiza Cadoret - Chief Global Product Strategy & Operations Officer Eric Swayze - EVP, Research Company Participants Operator At this time, I would like to turn the call over to Wade Walke, Senior Vice President of Investor Relations, to lead off the call. Please begin, sir. Thank you, [Vishnari], Navin. Before we begin, I encourage everyone to go to the Investors section of the Ionis website to vi ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, DC 20549 FORM 10-K ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2022 ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from ___________ to ___________ Commission file number 000-19125 Ionis Pharmaceuticals, Inc. (Exact name of Registrant as specified in its charter) Delaware 33-0336973 (State or other ...