Financial Performance - Total revenue for 2024 was $705.1 million, a decrease of 10.5% from $787.6 million in 2023[376] - The company recorded a net loss of $453.9 million in 2024, compared to a net loss of $366.3 million in 2023, indicating an increase of about 23.9%[412] - The income tax benefit for 2024 was $6.2 million, a significant improvement from an income tax expense of $32.3 million in 2023[409] - Investment income increased from $89.0 million in 2023 to $107.0 million in 2024, a growth of about 20.2% driven by higher interest rates[403] - Interest expense increased from $12.7 million in 2023 to $17.0 million in 2024, representing a rise of approximately 33.9% due to new convertible notes[404] Operating Expenses - Total operating expenses increased to $1,180.2 million in 2024 from $1,141.4 million in 2023, resulting in a loss from operations of $475.1 million[376] - Non-cash compensation expense related to equity awards increased to $130.2 million in 2024 from $105.7 million in 2023, driven by higher headcount and stock prices[384] - SG&A expenses rose from $232.6 million in 2023 to $267.5 million in 2024, marking an increase of approximately 15% due to new product launches[402] - Medical affairs expenses increased to $31.9 million in 2024 from $22.9 million in 2023, reflecting advancements in the late-stage pipeline[396] - Total R&D support expenses increased from $96.0 million in 2023 to $101.2 million in 2024, reflecting a rise of about 6.3%[400] Revenue Sources - SPINRAZA royalties decreased to $216.1 million in 2024 from $240.4 million in 2023, impacted by a non-recurring annual order from a single country[378] - WAINUA joint development revenue decreased to $79.4 million in 2024 from $126.4 million in 2023, primarily due to winding down development activities[382] - The company launched WAINUA in the U.S. in late January 2024 and TRYNGOLZA in late December 2024, contributing to new commercial revenue sources[378] - R&D revenue is recognized from various collaboration agreements, including upfront payments, milestone payments, and license fees, requiring significant estimates and judgments[432] - Revenue from R&D services is recognized based on an input method estimating total costs to complete performance obligations, which may require adjustments if estimates change[433] Cash and Investments - Cash, cash equivalents, and short-term investments were $2,297.7 million as of December 31, 2024, down from $2,331.2 million in 2023[376] - The company raised approximately $489 million from a public stock offering in September 2024, contributing to increased cash available for investments[403] Development Costs - Total drug development expenses were $568.5 million in 2024, compared to $564.8 million in 2023, indicating stable development costs[392] - Manufacturing and development chemistry expenses decreased from $74.1 million in 2023 to $67.1 million in 2024, a reduction of approximately 9.4%[398] - As of December 31, 2024, a hypothetical 10% increase in liability for preclinical and clinical development costs would increase loss before income tax benefit by approximately $7.7 million[436] - The company maintains an accrual for preclinical and clinical development costs, estimating liabilities based on contracted fees and expected expenses[435] Future Estimates and Risks - Factors affecting future royalty payments include regulatory approvals and competition, which could lead to significant changes in revenue estimates[439] - The allocation of transaction price to performance obligations involves estimating relative stand-alone selling prices, which may require significant judgments[431] - The company assesses the propriety of revenue recognition and deferred revenue, along with estimating future royalty payments under its royalty purchase agreement[430] - The company recognizes revenue related to milestone payments based on the probability of achievement and the estimated period of performance[437] - In 2023, the company entered into a royalty purchase agreement with Royalty Pharma to monetize future royalties from SPINRAZA and pelacarsen, with estimates subject to change based on sales performance[438] Contractual Obligations - Total contractual obligations as of December 31, 2024, amounted to $1,513.4 million, with $31.6 million due within one year[417] - The company does not utilize derivative financial instruments to manage exposure to interest rate changes, believing it is not subject to material risks from such changes as of December 31, 2024[440] Historical Performance - The company has generated approximately $7.9 billion in revenue since its inception through December 31, 2024[413]

Group 1 - Brendan completed a Ph.D. at Stanford University in organic synthesis in 2009 [1] - He worked for Merck from 2009 to 2013 and has experience in biotech startups such as Theravance and Aspira [1] - Brendan is a co-founder of 1200 Pharma, which spun out of Caltech and received significant investment in the 8 figures [1] Group 2 - Brendan remains an avid investor focused on market trends, particularly in biotechnology stocks [1]

Core Insights - Ionis Pharmaceuticals reported better-than-expected revenue and a smaller-than-expected loss for Q4 2024, indicating strong operational performance [2][6]. Financial Performance - Q4 2024 revenue reached $227 million, significantly exceeding analysts' expectations of $135 million, marking a 68.1% increase [3][6]. - The earnings loss was $0.66 per share, which was better than the anticipated loss of $1.11 per share, reflecting effective operational execution [3][7]. - Total revenue decreased by 30.2% compared to Q4 2023, where it was $325 million [3]. Operational Highlights - Ionis' non-GAAP operating expenses were $301 million, a slight decrease of 1.3% from the previous year [3][7]. - The successful launch of Wainua contributed to the positive revenue surprise, while royalty revenue from Spinraza remained a significant revenue source [6][7]. Strategic Focus - The company is expanding its drug offerings and enhancing the reach of its marketed medicines, with a focus on collaborations to mitigate development risks [5][8]. - Ionis has formed strategic alliances with Theratechnologies and Otsuka, which have provided financial support through milestones and royalties [8]. Future Outlook - Ionis anticipates revenue exceeding $600 million in 2025, with a planned operating loss of less than $495 million [9]. - The company has cash reserves of $2.3 billion, which will support continued investments in product launches and R&D [9]. - Three independent product launches are planned for the year, including donidalorsen for hereditary angioedema [10].

YE:2024 Business Update and Financial Results February 19, 2025 Nasdaq: IONS 1 On Today's Earnings Call Beth Hougen Chief Financial Officer 2 Brett Monia, Ph.D. Chief Executive Officer Kyle Jenne Chief Global Product Strategy Officer Richard Geary, Ph.D. Chief Development Officer Eric Swayze, Ph.D. Executive Vice President, Research Forward-Looking Statements This presentation includes forward-looking statements regarding our business, financial guidance and the therapeutic and commercial potential of our c ...

On Wednesday, Ionis Pharmaceuticals Inc IONS reported a fourth-quarter EPS loss of $0.66, up from a loss of $0.06, beating the consensus of a $1.10 loss.The company reported an adjusted net loss of $68 million versus a net income of $17 million.Ionis reported quarterly sales of $227 million, down from $325 million a year ago, beating the consensus of $137.6 million.Ionis says 2024 revenue of $705 million exceeded guidance as the company continued to generate revenue from diverse sources. The company added n ...

Exhibit 99.1 Ionis reports fourth quarter and full year 2024 financial results - First independent launch underway following TRYNGOLZATM approval - - Second independent launch on track with donidalorsen PDUFA August 21, 2025 - - WAINUATM U.S. launch delivering accelerating sequential growth - - Ionis exceeds 2024 financial guidance and provides full year 2025 guidance - CARLSBAD, Calif., February 19, 2025 – Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) (the "Company") today reported financial results for the f ...

For the quarter ended December 2024, Ionis Pharmaceuticals (IONS) reported revenue of $227 million, down 30.2% over the same period last year. EPS came in at -$0.66, compared to -$0.06 in the year-ago quarter.The reported revenue represents a surprise of +65.19% over the Zacks Consensus Estimate of $137.42 million. With the consensus EPS estimate being -$1.12, the EPS surprise was +41.07%.While investors scrutinize revenue and earnings changes year-over-year and how they compare with Wall Street expectation ...

Core Viewpoint - Ionis Pharmaceuticals is expected to report a significant quarterly loss and a substantial decline in revenues compared to the previous year [1] Financial Performance - Analysts predict a quarterly loss of $1.12 per share, reflecting a decline of 1766.7% year-over-year [1] - Revenues are forecasted to be $137.47 million, representing a year-over-year decrease of 57.7% [1] Earnings Estimates Revisions - Over the last 30 days, there has been an upward revision of 3.7% in the consensus EPS estimate for the quarter [2] - Revisions to earnings estimates are crucial indicators for predicting potential investor actions regarding the stock [3] Revenue Breakdown - Revenue from Spinraza royalties is estimated to reach $60.73 million, indicating a year-over-year change of -2% [5] - Licensing and royalty revenue is projected at $5.23 million, reflecting a year-over-year change of -34.7% [5] - Total commercial revenue is estimated at $80.55 million, showing a change of +2% from the prior-year quarter [6] - Revenue from Tegsedi and Waylivra is expected to be $7.06 million, indicating a change of -21.6% from the prior-year quarter [6] - Collaborative agreement revenue is projected at $43.79 million, reflecting a year-over-year change of -75.5% [7] Market Performance - Shares of Ionis Pharmaceuticals have experienced a change of -1.2% in the past month, compared to a +4.7% move of the Zacks S&P 500 composite [7] - The company holds a Zacks Rank 3 (Hold), indicating it is expected to mirror overall market performance in the near future [7]

– Accelerating value creation with numerous commercial, regulatory and pipeline milestones –– Ionis outlines clear path to sustained positive cash flow –CARLSBAD, Calif., Jan. 13, 2025 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced highlights from the Company's 2024 achievements and previewed important milestones expected in 2025. Ionis will provide a business update at the 42nd Annual J.P. Morgan Healthcare Conference on Wednesday, January 15 at 10:30am PT; the presentation is a ...

Portfolio Updates and Anticipated Milestones - Praxis has three blockbuster programs in late-stage development, with potential for four product launches between 2026 and 2028 [1] - The Essential3 program for ulixacaltamide in essential tremor (ET) includes two Phase 3 studies, with over 100,000 patients showing interest in participating since recruitment began in November 2023 [2] - Relutrigine is being evaluated in the EMBOLD study for developmental and epileptic encephalopathies (DEEs), with plans for the EMERALD study in a broader patient population [3] - Praxis shared an update at the 2024 AES Annual Meeting for an emergency use patient who achieved extended benefit after receiving relutrigine [4] - Ulixacaltamide is a highly selective small molecule inhibitor of T-type calcium channels, currently in late-stage development for essential tremor [5] - Praxis has completed discussions with global regulatory agencies to harmonize a registrational study design for elsunersen, with trial design and timelines expected in Q1 2025 [6] - UCB exercised its option to in-license global development and commercialization rights for a KCNT1 small molecule candidate, with Praxis eligible for up to $100 million in milestone payments and tiered royalties [6] - Praxis is on track to nominate development candidates for its early-stage ASO therapeutic initiatives in 2025, including PRAX-080, PRAX-090, and PRAX-100 [6] Clinical Trial Progress and Results - Vormatrigine demonstrated strong competitive differentiation in Phase 1 studies, with full results to be shared at an upcoming medical conference [10] - The EMPOWER observational study has enrolled over 2,000 patients, with early results shared at the 2024 AES Annual Meeting [10] - The RADIANT Phase 2 study for focal onset seizures (FOS) and generalized epilepsy is enrolling patients, with topline results expected in H1 2025 [10] - The POWER1 Phase 2/3 registrational study for treatment-resistant FOS is progressing, with topline results anticipated in H2 2025 [10] - Relutrigine showed a 46% placebo-adjusted reduction in monthly motor seizures in the EMBOLD study, with 77% reduction in motor seizures for patients continuing into the open-label extension [17] - Over 30% of patients achieved seizure freedom status while on relutrigine, with a median of 46 days of seizure freedom compared to 3 days at baseline [17] Regulatory and Commercial Developments - Praxis anticipates filing the NDA for ulixacaltamide in 2025, following positive results from the Essential3 program [9] - The company plans to re-initiate a study of ulixacaltamide in Parkinson's disease, addressing unmet needs for non-dopaminergic treatment options [9] - Praxis received a third rare pediatric drug designation (RPDD) for relutrigine in Dravet Syndrome, in addition to previous designations for SCN2A and SCN8A DEEs [17] - The company expects to initiate the pivotal trial for elsunersen in H1 2025, following regulatory feedback [8] Financial and Strategic Outlook - Praxis has a cash and investment position of approximately $470 million at the end of 2024, supporting a runway into 2028 [15] - The company is well-positioned for a readout-rich 2025, with results anticipated for ulixacaltamide, vormatrigine, and relutrigine, as well as regulatory submissions [8] - Praxis plans to provide a thorough update across its portfolio at the Investor R&D Day in Q2 2025 [8]