news/AZN/2023.02.23/Ionis reports fourth quarter and full year 2022 financial results.txt

CARLSBAD - 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 Highlights2022 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 HighlightsSubmitted 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 extension study of donidalorsen in patients with hereditary angioedema treated for one year, including new data showing clinically meaningful improvement in angioedema quality of life score; Phase 3 study expected to complete enrollment in 2023GSK advanced bepirovirsen into Phase 3 development in patients with chronic hepatitis BRecent Additional Pipeline HighlightsBiogen initiated a Phase 2 study of IONIS-MAPTRx (BIIB080) in patients with mild cognitive impairment or mild dementia due to Alzheimer's diseaseRoche initiated a Phase 2 study of tominersen in patients with prodromal or early manifest Huntington's diseaseBiogen initiated a Phase 1 study of ION306 (BIIB115) for the treatment of spinal muscular atrophy with the potential for long interval dosingRecent Technology Advancement HighlightsPartnered with Metagenomi to add gene editing capabilities to Ionis' technology platformAdvanced programs incorporating muscle LICA technology and MsPA backbone chemistry into preclinical development'We made substantial progress in 2022, marked by important achievements, including the December submission of the eplontersen NDA for people with ATTRv-PN. We also delivered multiple positive data readouts, enabling us to advance and expand our rich late- and mid-stage pipeline. And we took important steps to expand and diversify our technology, including our Metagenomi collaboration to add DNA editing to our platform,' said Brett P. Monia, Ph.D., chief executive officer of Ionis. 'As we start 2023, we are positioned to build upon our achievements by bringing our first near-term commercial opportunities to the market. We look forward to reporting the 66-week results from the eplontersen NEURO-TTRansform study in the first half of this year. Importantly, we are prepared to co-commercialization eplontersen with our partner, AstraZeneca. We also look forward to Phase 3 data from olezarsen in FCS patients, positioning us for our first independent launch. With the talent and resources we have today, we anticipate a highly productive year that will enable us to drive increasing value for all stakeholders.'About Ionis Pharmaceuticals, Inc.For more than 30 years, Ionis has been the leader in RNA-targeted therapy, pioneering new markets and changing the standards of care with its novel antisense technology. Ionis currently has three marketed medicines and a premier late-stage pipeline highlighted by industry leading cardiovascular and neurological franchises. Our scientific innovation began and continues with the knowledge that sick people depend on us, which fuels our vision of becoming the leader in genetic medicine, utilizing a multi-platform approach to discover, develop and deliver life-transforming therapies.Ionis Forward-looking StatementThis press release includes forward-looking statements regarding Ionis' business, financial guidance and the therapeutic and commercial potential of SPINRAZA (nusinersen), TEGSEDI (inotersen), WAYLIVRA (volanesorsen), eplontersen, olezarsen, donidalorsen, ION363, tofersen, pelacarsen, bepirovirsen, Ionis' technologies and Ionis' other products in development. 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 statement. Such statements are subject to certain risks and uncertainties including those inherent in the process of discovering, developing and commercializing medicines that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such medicines. Ionis' forward-looking statements also involve assumptions that, if they never materialize or prove correct, could cause its results to differ materially from those expressed or implied by such forward-looking statements. Although Ionis' forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Ionis. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Ionis' programs are described in additional detail in Ionis' annual report on Form 10-K for the year ended December 31, 2021, and most recent Form 10-Q, which are on file with the Securities and Exchange Commission.In this press release, unless the context requires otherwise, 'Ionis,' 'Company,' 'we,' 'our' and 'us' all refer to Ionis Pharmaceuticals and its subsidiaries.Ionis Pharmaceuticals is a registered trademark of Ionis Pharmaceuticals, Inc. Akcea Therapeutics is a registered trademark of Akcea Therapeutics, Inc. TEGSEDI is a registered trademark of Akcea Therapeutics, Inc. WAYLIVRA is a registered trademark of Akcea Therapeutics, Inc. SPINRAZA is a registered trademark of Biogen.Contact:Email: info@ionisph.comTel: 760-603-2331(C) 2023 Electronic News Publishing, source ENP Newswire