IONIS" FY 2020 Financial Results and Business Update February 24, 2021 On Today's Earnings Call | --- | --- | --- | --- | |--------------------------------------------|--------------------|-------------------------------------|--------------------------------------------------------------------| | | | | | | | | | | | Brett Monia, Ph.D. Chief Executive Officer | | Beth Hougen Chief Financial Officer | Richard Geary, Ph.D. Executive Vice President, Development | | | Eric Swayze, Ph.D. | Executive Vice Preside ...

Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) Q4 2020 Earnings Conference Call February 24, 2020 9:00 AM ET Company Participants Wade Walke - VP, IR Brett Monia - CEO Beth Hougen - CFO Richard Geary - EVP of Development Onaiza Cadoret - Chief Corporate Development and Commercial Officer Eric Swayze - EVP of Research Conference Call Participants Yaron Werber - Cowen Alex Thompson - Stifel Jim Birchenough - Wells Fargo Tyler Van Buren - Piper Sandler Vincent Chen - Bernstein Joel Beatty - Citi Yale Jen - Laidlaw ...

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, 2020 ☐ 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 ...

IONIS" Investor Day 2020 December 7, 2020 Forward Looking Language Statement This presentation includes forward-looking statements regarding our business, financial guidance and the therapeutic and commercial potential of SPINRAZA® (nusinersen), TEGSEDI® (inotersen), WAYLIVRA® (volanesorsen) and Ionis' technologies and products in development, including the business of Akcea Therapeutics, Inc., Ionis' wholly owned subsidiary. Any statement describing Ionis' goals, expectations, financial or other projection ...

Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) Q3 2020 Earnings Conference Call November 4, 2020 11:30 AM ET Company Participants Wade Walke – Vice President of Investor Relations Brett Monia – Chief Executive Officer Beth Hougen – Chief Financial Officer Onaiza Cadoret – Chief Corporate Development and Commercial Officer Richard Geary – Executive Vice President of Development Eric Swayze – Executive Vice President of Research Kyle Jenne – Chief Commercial Officer-Akcea Conference Call Participants Do Kim – BMO ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION ☐ 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) 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, 2020 OR Delaware 33-0336973 (State or ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, DC 20549 Form 10-Q (Mark One) ☐ 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. ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 (Exact name of Registrant as specified in its charter) For the Quarterly Period Ended June 30, 2020 Delaware 33-0336973 OR (State or othe ...

Financial Data and Key Metrics Changes - The company ended Q2 2020 with a net income of $8 million on a non-GAAP basis, an increase compared to Q1 results [25] - Cash and investments totaled more than $2.3 billion at the end of June, indicating strong financial health [12][27] - The company reaffirmed its financial guidance for 2020, projecting increased revenue and earnings in the second half of the year [12][26] Business Line Data and Key Metrics Changes - SPINRAZA sales reached $495 million in Q2, generating $72 million in royalty revenue for the company [13] - TEGSEDI and WAYLIVRA reported product sales of $16 million in Q2, showing substantial growth compared to the previous year [17] - R&D revenue exceeded $55 million in Q2, with significant contributions from various therapeutic franchises [23] Market Data and Key Metrics Changes - Over 11,000 patients were on SPINRAZA treatment worldwide by the end of June, reflecting strong market demand [13] - Biogen estimates over 60,000 SMA patients in markets where they have a commercial presence, a nearly 35% increase from previous estimates [14] - TEGSEDI is now commercially available in 15 countries, with ongoing efforts to broaden access in new markets [19] Company Strategy and Development Direction - The company aims to deliver 10 or more marketing applications for various diseases by the end of 2025, positioning itself as a leader in transformative medicines [11][49] - There is a focus on expanding the Ionis-owned pipeline, particularly in rare neurological diseases, while also pursuing strategic partnerships [67][70] - The company is prioritizing its rare neurological disease pipeline, with several drugs expected to enter clinical studies soon [68][70] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in achieving 2020 financial goals and highlighted the strength of the clinical pipeline [10][12] - The company is adapting to the evolving global pandemic while continuing to execute its strategic objectives [44] - There is optimism regarding the potential for significant R&D revenue growth in the second half of the year [26] Other Important Information - The company is advancing its pulmonary franchise with plans to initiate a Phase 2 study of Ionis-ENAC-2.5Rx in COPD patients [41] - The collaboration with AstraZeneca is expected to yield updates on several ongoing programs later this year [38] Q&A Session Summary Question: What level of knockdown is targeted in the inhaled program? - The company targets a 50% reduction in ENaC messenger RNA and protein for the CF program, with confidence in achieving greater reductions [55][56] Question: How should R&D revenue be modeled for the long term? - The company expects sustained and potentially growing R&D revenues due to strong partnerships and increasing milestone payments [60] Question: How does the company view its long-term strategic vision? - The company plans to strategically partner where it makes sense while prioritizing its Ionis-owned pipeline, particularly in neurology [66][70] Question: What is the competitive landscape for the prekallikrein program? - The company believes there is room for more players in the HAE market, focusing on differentiation through dosing and administration [88] Question: What is the timeline for the APOCIII LICA program? - The company plans to initiate the Phase 3 study for APOCIII LICA in FCS patients this year, with potential market availability next year [125]

IONIS® Neurology Webcast July 14, 2020 Forward Looking Language Statement This presentation includes forward-looking statements regarding our business, financial guidance and the therapeutic and commercial potential of SPINRAZA® (nusinersen) and Ionis' technologies and products in development, including Ionis' neurology franchise. Any statement describing Ionis' goals, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk state ...

Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) BMO 2020 Prescriptions for Success Healthcare Conference June 23, 2020 2:00 PM ET Company Participants Eric Swayze - SVP of Research Wade Walke - Head, IR Conference Call Participants Do Kim - BMO Capital Markets Do Kim Good afternoon. My name is Do Kim. I'm one of the Biotech Analyst at BMO. For next fireside chat, we have with us, Eric Swayze, SVP Research at Ionis Pharmaceuticals and Wade Walke, Head of Investor Relations. Could you start by providing us a brief ...