Financial Performance - Total revenue for 2023 was $787.6 million, a 34% increase from $587.4 million in 2022[470] - Net loss for 2023 was $366.3 million, compared to a net loss of $269.7 million in 2022[470] - Investment income surged to $89.0 million in 2023, up from $25.3 million in 2022, representing an increase of about 251.0%[497] - The company recorded a net loss of $366.3 million for 2023, compared to a net loss of $269.7 million in 2022, indicating an increase in losses of approximately 35.8%[508] Operating Expenses - Total operating expenses for 2023 were $1,141.4 million, up from $997.6 million in 2022, reflecting a 14.4% increase[470] - Operating expenses, excluding non-cash compensation, were $1,035.7 million in 2023, compared to $897.3 million in 2022[478] - SG&A expenses rose to $232.6 million in 2023, compared to $150.3 million in 2022, marking an increase of approximately 54.6%[496] - Medical affairs expenses rose to $22.9 million in 2023, compared to $17.9 million in 2022, reflecting a 27.9% increase[489] - Total drug development expenses for 2023 were $564.8 million, an increase from $457.7 million in 2022[486] - Total R&D support expenses increased to $96.0 million in 2023, up from $73.9 million in 2022, reflecting a rise of about 30.0%[493] - Manufacturing and development chemistry expenses decreased to $74.1 million in 2023 from $86.1 million in 2022, a reduction of approximately 14.5%[491] Revenue Sources - R&D revenue increased to $479.0 million in 2023, up from $284.0 million in 2022, marking a 68.7% increase[472] - WAINUA joint development revenue was $126.4 million in 2023, significantly higher than $76.8 million in 2022[476] - The company recognizes R&D revenue from collaboration agreements, which include technology licenses, R&D services, and manufacturing services[526] Cash and Investments - Cash, cash equivalents, and short-term investments totaled $2,331.2 million as of December 31, 2023, up from $1,986.9 million in 2022[470] - The company raised approximately $575.0 million through the issuance of 1.75% Notes in 2023[503] - The company has raised approximately $2.1 billion from the sale of equity securities since inception[510] Contractual Obligations and Agreements - Total contractual obligations as of December 31, 2023, amounted to $1,587.9 million[513] - The company completed a royalty purchase agreement with Royalty Pharma for $500 million upfront payment in January 2023[517] - The company entered into a royalty purchase agreement with Royalty Pharma in January 2023 to monetize future royalties from SPINRAZA and pelacarsen[532] Tax and Liabilities - Income tax expense increased to $32.3 million in 2023 from $11.7 million in 2022, reflecting an increase of about 175.2%[504] - As of December 31, 2023, a hypothetical 10 percent increase in the liability for preclinical and clinical development costs would result in an increase in loss before income tax benefit of approximately $10.6 million[531] Revenue Recognition Practices - Revenue recognition involves assessing the propriety of revenue recognition and associated deferred revenue, as well as estimating future royalty payments[525] - The company uses an input method to estimate revenue from R&D services with upfront payments, adjusting revenue if estimates change[528] - Milestone payments are recognized based on whether they relate to services performed by the company or its partners, with full recognition for partner-achieved milestones[529] - The company assesses the transaction price for collaboration agreements and allocates it to performance obligations based on relative stand-alone selling prices[526] - Historical accrual estimates for clinical development costs have not materially differed from actual amounts, indicating effective estimation practices[530] Development Pipeline - The company has nine medicines in Phase 3 development and multiple additional medicines in early and mid-stage development[469] Risk Management - The company does not utilize derivative financial instruments to manage exposure to interest rate changes and believes it is not subject to material risks from market changes as of December 31, 2023[534] - Future royalty payments from Novartis for pelacarsen are subject to various factors, including regulatory approval and competing products, which could materially impact estimates[533]

Ionis (IONS) recently announced that the FDA has granted orphan drug designation to its pipeline candidate, olezarsen, for the treatment of familial chylomicronemia syndrome (FCS), a rare genetic disease.The FDA’s orphan drug designation is bestowed on medicines in development for the treatment, prevention or diagnosis of a rare disease that affects fewer than 200,000 people in the United States at the time of designation. The rare disease, FCS, is characterized by extremely high triglyceride levels and rec ...

Analysts on Wall Street project that Ionis Pharmaceuticals (IONS) will announce quarterly loss of $0.78 per share in its forthcoming report, representing a decline of 110.8% year over year. Revenues are projected to reach $183.6 million, increasing 20.8% from the same quarter last year.The consensus EPS estimate for the quarter has been revised 12.6% lower over the last 30 days to the current level. This reflects how the analysts covering the stock have collectively reevaluated their initial estimates durin ...

CARLSBAD, Calif., Feb. 15, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation to the investigational medicine olezarsen for the treatment of familial chylomicronemia syndrome (FCS), a rare, genetic disease characterized by extremely elevated triglyceride levels and recurrent acute pancreatitis (AP). The FDA grants Orphan Drug designation to therapies intended for treatment, prevention or diagnos ...

Wall Street expects a year-over-year decline in earnings on higher revenues when Ionis Pharmaceuticals (IONS) reports results for the quarter ended December 2023. While this widely-known consensus outlook is important in gauging the company's earnings picture, a powerful factor that could impact its near-term stock price is how the actual results compare to these estimates.The stock might move higher if these key numbers top expectations in the upcoming earnings report, which is expected to be released on F ...

Ionis Pharmaceuticals, Inc. (IONS) announced that the FDA has granted a Fast Track designation to eplontersen for the treatment of adult patients with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM).ATTR-CM is a progressive and fatal disease that is caused by the accumulation of misfolded TTR protein in the cardiac muscle.Ionis is developing eplontersen in partnership with British drugmaker AstraZeneca (AZN) .The phase III CARDIO-TTRansform study is currently evaluating eplontersen for treating ATTR ...

CARLSBAD, Calif., Feb. 8, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to Ionis and AstraZeneca's eplontersen, an investigational therapy for the treatment of transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) in adults. The FDA grants development programs Fast Track designation to facilitate the development and expedite the review of drugs that demonstrate the potential to treat serio ...

Webcast scheduled for Wednesday, February 21 at 11:30 a.m. Eastern TimeCARLSBAD, Calif., Feb. 7, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that it will host a live webcast on Wednesday, February 21st at 11:30 a.m. Eastern Time to discuss its fourth quarter and full year 2023 financial results.The live webcast may be accessed at https://ir.ionispharma.com/events-and-presentations/upcoming-events. A replay will be available for a limited time at the same address.About Ion ...

Ionis Pharmaceuticals (IONS) announced positive top-line results from the phase III OASIS-HAE study on its investigational drug donidalorsen in patients aged 12 years and older with hereditary angioedema (HAE).The OASIS-HAE study randomized study participants into three equal groups over a 24-week treatment period — two groups evaluating an 80mg dose of donidalorsen administered once every four weeks (Q4W) and another group wherein the drug was administered once every eight weeks (Q8W). The third group admi ...

Donidalorsen met the primary endpoint with a statistically significant reduction in the rate of HAE attacks in patients treated every 4 weeks or patients treated every 8 weeks  Donidalorsen demonstrated a favorable safety and tolerability profile Ionis is preparing to submit a New Drug Application with U.S. FDA Data to be presented at an upcoming medical congress CARLSBAD, Calif., Jan. 22, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced positive topline results for the Pha ...