– Numerous value-driving commercial, regulatory and pipeline milestones anticipated – CARLSBAD, Calif., Jan. 8, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced highlights from the Company's 2023 achievements and previewed a number of important milestones expected in 2024. Ionis will provide a business update at the 42nd Annual J.P. Morgan Healthcare Conference on Wednesday, January 10 at 3pm PT; the presentation is available today on the Ionis website. In 2023, Ionis made sig ...

MicroStockHub Investment Overview The biotech sector of the stock market is finishing 2023 on a high after what has been a difficult couple of years. Buoyed by a Big Pharma M&A spree - the globally recognized Pharmas AbbVie (ABBV) and Bristol-Myers Squibb (BMY) have respectively spent a total of $18.8bn acquiring antibody drug conjugate ("ADC") specialist ImmunoGen (IMGN) and neuroscience pioneer Cerevel Therapeutics, and $18.1bn acquiring Karuna Therapeutics (KRTX) and its highly rated schizophrenia dr ...

U.S. FDA approval based on Phase 3 NEURO-TTRansform results showing WAINUA demonstrated consistent and sustained benefit halting neuropathy disease progression and improving neuropathy impairment and quality of life Additional regulatory reviews for WAINUA underway in rest of world WAINUA will be available in the U.S. in January 2024 CARLSBAD, Calif., Dec. 21, 2023 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that the U.S. Food and Drug Administration (FDA) has approved Ion ...

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 (Exact name of Registrant as specified in its charter) Delaware 33-0336973 (State or other jurisdiction of incorporation or organization) (IRS Employer Identification No.) 2855 Gazelle Court, Carlsbad, California 92010 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 SECU ...

Revenue and Financial Performance - Total revenue for Q2 2023 was $188.4 million, a 40.8% increase compared to $133.8 million in Q2 2022[17] - Total revenues for Q2 2023 were $188.4 million, a 40.8% increase compared to $133.8 million in Q2 2022[108] - Total revenue for Q2 2023 was $188.4 million, compared to $133.8 million in Q2 2022, with a net loss of $85.3 million[105] - Total revenue for the six months ended June 30, 2023, was $318.9 million, compared to $275.7 million in the same period in 2022[17] - Total revenues for Q2 2023 were $188.4 million, a significant increase from $133.8 million in Q2 2022, driven by increased payments from partnered programs[108] - Total revenue for Q2 2023 was $188.4 million, compared to $133.8 million in Q2 2022, and $318.9 million for the first half of 2023, compared to $275.7 million in the same period of 2022[105] - The company has earned approximately $6.7 billion in revenue from inception through June 30, 2023, primarily from research and development collaborative agreements and commercial revenue[144] SPINRAZA Royalties - SPINRAZA royalties generated $61.0 million in Q2 2023, a 2.3% increase from $59.6 million in Q2 2022[17] - SPINRAZA royalties contributed $61.0 million in Q2 2023, slightly up from $59.6 million in Q2 2022[108] - SPINRAZA royalties generated $61.0 million in revenue for the three months ended June 30, 2023, compared to $59.6 million in the same period in 2022[17] - SPINRAZA royalties contributed $61.0 million in Q2 2023, with global SPINRAZA product sales reaching $437 million, showing resilience against competition[109] - SPINRAZA collaboration generated over $1.9 billion in revenues since inception, including $1.5 billion in royalties[99] Research and Development (R&D) Revenue and Expenses - Research and development revenue surged to $110.5 million in Q2 2023, a 98.7% increase from $55.6 million in Q2 2022[17] - R&D revenue for Q2 2023 was $110.5 million, up from $55.6 million in Q2 2022, primarily due to milestone payments of $51.1 million and license fees of $20.0 million[108] - R&D revenue doubled in Q2 2023 and increased over 35% in the first half of 2023 compared to the same periods in 2022[110] - Research and development revenue for the six months ended June 30, 2023, was $173.3 million, compared to $125.2 million in the same period in 2022[17] - R&D revenue for Q2 2023 essentially doubled compared to the same period in 2022, and increased more than 35% for the first half of 2023[110] - Eplontersen joint development revenue reached $19.5 million in Q2 2023, up from $17.4 million in Q2 2022[114] - Eplontersen joint development revenue for Q2 2023 was $19.5 million, compared to $17.4 million in Q2 2022, and $43.9 million for the first half of 2023, compared to $37.2 million in the same period of 2022[114] - Eplontersen Phase 3 development expenses for Q2 2023 were $38.3 million, compared to $35.0 million in Q2 2022, and $85.4 million for the first half of 2023, compared to $71.1 million in the same period of 2022[114] - Eplontersen development expenses rose to $28.7 million in Q2 2023, up from $24.3 million in Q2 2022[123] - Olezarsen development expenses surged to $31.3 million in Q2 2023, compared to $12.7 million in Q2 2022[123] - Donidalorsen development expenses increased to $7.2 million in Q2 2023 from $1.9 million in Q2 2022[123] - Drug development expenses for Q2 2023 were $145.5 million, compared to $103.2 million in Q2 2022, and $269.9 million for the first half of 2023, compared to $201.8 million in the same period of 2022[123] - Manufacturing and development chemistry expenses were $24.4 million in Q2 2023, down from $25.7 million in Q2 2022[128] - R&D support expenses increased to $23.0 million in Q2 2023, up from $17.7 million in Q2 2022, primarily due to higher occupancy and personnel costs[130] - Manufacturing and development chemistry expenses for the six months ended June 30, 2023, were $41.2 million, down from $44.7 million in the same period in 2022[128] - R&D support expenses for the six months ended June 30, 2023, were $45.8 million, up from $34.9 million in the same period in 2022, primarily due to increased occupancy and personnel costs[130] Operating Expenses and Net Loss - Net loss for Q2 2023 was $85.3 million, an improvement from $105.1 million in Q2 2022[17] - Total operating expenses increased to $278.6 million in Q2 2023, up 27.0% from $219.3 million in Q2 2022[17] - Operating expenses, excluding non-cash compensation, increased to $252.1 million in Q2 2023 from $194.8 million in Q2 2022[115] - Net loss for the six months ended June 30, 2023, was $209.6 million, compared to $170.3 million in the same period in 2022[17] - Total operating expenses for Q2 2023 were $278.6 million, up from $219.3 million in Q2 2022, and $523.3 million for the first half of 2023, compared to $418.7 million in the same period of 2022[115] - Net loss for Q2 2023 was $85.3 million, compared to $105.1 million in Q2 2022, and $209.6 million for the first half of 2023, compared to $170.3 million in the same period of 2022[105] Investment Income and Financial Assets - Investment income rose significantly to $20.8 million in Q2 2023, compared to $3.4 million in Q2 2022[17] - The company held $2.32 billion in fair value securities as of June 30, 2023, including $809.1 million in U.S. Treasury securities[62] - Investment income increased to $20.8 million in Q2 2023, up from $3.4 million in Q2 2022, driven by higher interest rates and increased cash available for investment[134] - Investment income for Q2 2023 was $20.8 million, compared to $3.4 million in Q2 2022, and $39.4 million for the first half of 2023, compared to $5.4 million in the same period of 2022[134] - The fair value of cash equivalents held by the company as of June 30, 2023, was $316.4 million[62] - Gain on investments for the three months ended June 30, 2023, was $0.7 million, compared to a loss of $6.3 million in the same period in 2022[137] - Investment gains for the three months ended June 30, 2023, were $0.7 million, compared to a loss of $6.3 million in the same period in 2022[137] Royalty Pharma Agreement - Company received an upfront payment of $500 million from Royalty Pharma for future SPINRAZA and pelacarsen royalties, with potential for up to $625 million in additional milestone payments[78] - Royalty Pharma will receive 25% of SPINRAZA royalty payments from 2023-2027, increasing to 45% in 2028, on up to $1.5 billion in annual sales[78] - Estimated effective interest rate for the royalty agreement was 13.5% as of June 30, 2023[80] - Net liability related to sale of future royalties was $510.174 million as of June 30, 2023[80] - The company received an upfront payment of $500 million from Royalty Pharma and is eligible to receive up to $625 million in additional milestone payments[78] - The estimated effective interest rate under the Royalty Pharma agreement was 13.5% as of June 30, 2023[80] - Interest expense related to the sale of future royalties was $17.7 million in Q2 2023, as part of the $500 million upfront payment from Royalty Pharma[136] Convertible Notes and Debt - Company completed a $575.0 million offering of 1.75% convertible senior notes in June 2023, with an effective interest rate of 2.3%[81][82] - Outstanding principal balance of 0% convertible senior notes was $632.5 million as of June 30, 2023, with an effective interest rate of 0.5%[84] - Company repurchased $434.1 million of 0.125% Notes in June 2023, resulting in a remaining principal balance of $114.8 million[86] - Effective conversion price for 0.125% Notes with call spread was $123.38 per share[87] - Company recorded a $11.3 million gain on the early retirement of 0.125% Notes[86] - The company completed a $575.0 million offering of convertible senior notes in June 2023, with an effective interest rate of 2.3% and a conversion price per share of $53.73[82] - The company repurchased $434.1 million of its 0.125% Notes in June 2023, resulting in a remaining principal balance of $114.8 million as of June 30, 2023[86] - The company recorded a $11.3 million gain on the early retirement of its 0.125% Notes in June 2023[86] - The company borrowed approximately $2.7 billion under long-term debt arrangements from inception through June 30, 2023[144] Stock-Based Compensation - Stock-based compensation expense totaled $26.6 million in Q2 2023, up 8.4% from $24.5 million in Q2 2022[74] - The weighted-average grant date fair value of RSUs granted to employees for the six months ended June 30, 2023, was $39.50 per share[69] - The weighted-average grant date fair value of PRSUs granted to executive officers for the six months ended June 30, 2023 and 2022 were $58.99 and $42.28 per share, respectively[74] - Total stock-based compensation expense for the six months ended June 30, 2023 was $53.51 million, compared to $50.74 million for the same period in 2022[74] - As of June 30, 2023, total unrecognized estimated stock-based compensation expense related to non-vested stock options, RSUs and PRSUs was $50.5 million, $70.7 million and $8.4 million, respectively[74] Tax and Valuation Allowance - Income tax expense increased to $7.8 million in Q2 2023 from $2.3 million in Q2 2022, primarily due to the Royalty Pharma transaction[76] - The company maintains a full valuation allowance on all net deferred tax assets[77] - The company recorded income tax expense of $7.8 million and $19.2 million for the three and six months ended June 30, 2023, respectively, compared to $2.3 million and $3.4 million for the same periods in 2022[76] Product Development and Approvals - QALSODY received FDA accelerated approval in April 2023 for SOD1-ALS treatment[97] - Eplontersen's PDUFA date for ATTRv-PN is set for December 22, 2023[97] - Phase 3 BALANCE study for olezarsen is on track for data release in H2 2023[103] - Eplontersen's Phase 3 NEURO-TTRansform study showed positive results, halting neuropathy disease progression and improving quality of life through 85 weeks[103] - QALSODY received accelerated FDA approval in April 2023 for SOD1-ALS treatment, with the EMA currently reviewing its Marketing Authorization Application in the EU[102] - The company has eight medicines in Phase 3 studies, including eplontersen, olezarsen, donidalorsen, and ulefnersen, targeting various indications[103] - Olezarsen received FDA fast track designation for FCS treatment in January 2023, with Phase 3 BALANCE FCS study data expected in H2 2023[103] - Donidalorsen's Phase 3 OASIS-HAE study completed enrollment in June 2023, with data expected in H1 2024[103] Corporate Strategy and Partnerships - Corporate partnering remains a key strategy, with potential revenue impacts if collaborative partners fail to fund development programs[210] - AstraZeneca is funding 55% of the Phase 3 development costs for eplontersen, with Ionis recognizing this as R&D revenue[111] Cost of Sales and Medical Affairs - Cost of sales decreased to $2.5 million in Q2 2023, down from $4.7 million in Q2 2022[118] - Medical affairs expenses were $5.4 million in Q2 2023, slightly up from $5.2 million in Q2 2022, with expectations of further increases as the pipeline advances[126] - Cost of sales for the three months ended June 30, 2023, were $2.5 million, a decrease from $4.7 million in the same period in 2022[118] - Medical affairs expenses for the six months ended June 30, 2023, were $10.7 million, up from $8.3 million in the same period in 2022[126] - Commercialization expenses for eplontersen for Q2 2023 were $2.5 million, compared to $0.5 million in Q2 2022, and $3.8 million for the first half of 2023, compared to $0.7 million in the same period of 2022[114] - Medical affairs expenses for eplontersen for Q2 2023 were $1.1 million, compared to $0.5 million in Q2 2022, and $1.8 million for the first half of 2023, compared to $0.8 million in the same period of 2022[114] Contractual Obligations and Capital Expenditures - Total contractual obligations as of June 30, 2023, amounted to $1,673.7 million, with $31.0 million due within one year[147] - The company has raised approximately $2.1 billion from the sale of equity securities since its inception through June 30, 2023[144] - The company borrowed approximately $2.7 billion under long-term debt arrangements from inception through June 30, 2023[144] - The company purchased its primary R&D facility for $79.4 million and its manufacturing facility for $14.0 million in July 2017, financed with $60.4 million in mortgage debt[149] - The company had outstanding purchase orders as of June 30, 2023, for services, capital equipment, and materials as part of its normal course of business[154] - Total contractual obligations as of June 30, 2023, were $1,673.7 million, with $31.0 million due within one year and $1,642.7 million due after one year[147] Legal and Settlement - The Court approved the Revised Settlement and dismissed the Shumacher and Cohen Actions on April 24, 2023, with no admission of wrongdoing by any defendant[93]

Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) Q1 2023 Earnings Conference Call May 3, 2023 11:30 AM ET Company Participants Wade Walke - SVP, IR Brett Monia - CEO Richard Geary - Chief Development Officer Beth Hougen - CFO Eugene Schneider - Chief Clinical Development Officer Onaiza Cadoret - Chief Global Product Strategy & Operations Officer Eric Swayze - EVP, Research Conference Call Participants Joseph Stringer - Needham and Company Jessica Fye - JPMorgan Yanan Zhu - Wells Fargo Mike Ulz - Morgan Stanley Yal ...

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 | --- | --- | --- | |------------------------------------------------------------|---------------------------------------------------------------------------------|----------------------------- ...

Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) Q4 2022 Results Conference Call February 22, 2023 11:30 AM ET Company Participants 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 Conference Call Participants Yanan Zhu - Wells Fargo Securities Gary Nachman - BMO Capital Markets Do Kim - Piper Sandler Gena Wang - Barclays Yale Jen - Laidlaw & Co. Paul Matte ...