Business
Ionis reports fourth quarter and full year 2022 financial results
Eplontersen NDA submitted to FDA; tofersen under regulatory review for marketing approval in U.S. and EU Phase 3 data planned for eplontersen and olezarsen;
About this update from Ionis Pharmaceuticals, Inc.
[{"type":"text","content":"Eplontersen NDA submitted to FDA; tofersen under regulatory review for marketing approval in U.S. and EU\nPhase 3 data planned for eplontersen and olezarsen; robust late-stage pipeline expanding with two new Phase 3 programs \nIonis provides full year 2023 financial guidance\nCARLSBAD, Calif., Feb. 22, 2023 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) (the \"Company\"), a genetic medicines company, today reported financial results for the fourth quarter and full year ended December 31, 2022. Financial results are summarized below:\n\n \n \n \n \n \n \n\n \nThree months ended\nDecember 31,\nYear ended\nDecember 31,\n2022\n2021\n2022\n2021\n(amounts in millions)\nTotal revenue\n$152\n$440\n$587\n$810\nOperating expenses\n$360\n$219\n$998\n$840\nOperating expenses on a non-GAAP basis\n$335\n$196\n$898\n$695\nNet (loss) income\n($52)\n$225\n($270)\n($29)\nNet (loss) income on a non-GAAP basis\n($168)\n$248\n($311)\n$116\nCash, cash equivalents and short-term investments1\n$1,987\n$2,115\n1 Balance at December 31, 2022 does not include $500 million received in January 2023 under royalty monetization transaction.\nFinancial Highlights\n2022 revenue was in line with expectations, reflecting revenue from numerous diverse sources. The prior year fourth quarter and full year benefited from the $200 million earned from AstraZeneca to jointly develop and commercialize eplontersen2022 operating expenses increased as planned compared to the prior year, reflecting investments in pipeline, technology and go-to-market activities for eplontersen, olezarsen and donidalorsenFurther strengthened financial position with royalty monetization and sale and leaseback transactions worth up to $1.5 billion, including more than $700 million already receivedInitiated construction of a new manufacturing facility to support Ionis' goal to sustainably deliver genetic medicines to the marketRecent Late-Stage Pipeline Highlights\nSubmitted the eplontersen NDA in the U.S. for patients with polyneuropathy caused by hereditary TTR amyloidosisFDA Advisory Committee meeting planned for March 22, 2023 to review Biogen's NDA for tofersen for patients with SOD1-ALS (PDUFA date of April 25, 2023); EMA accepted MAA for reviewFDA granted olezarsen Fast Track designation for the treatment of patients with FCSReported positive Phase 2 data from the open label ex...