Ionis Pharmaceuticals, Inc.

Good morning and welcome to the Ionis Pharmaceuticals fourth quarter and full year 2021 financial results conference call. As a reminder, this call is being recorded. At this time, I would like to turn the call over to Jennifer Capuzzello, Investor Relations, to lead off the call. Please begin.

Thank you, Anthony. Before we begin, I encourage everyone to go to the investor section of the IONIS website to view the press release and related financial tables we will be discussing today, including a reconciliation of GAAP to non-GAAP financials. We believe non-GAAP financial results better represent the economics of our business and how we manage our business. We have also posted slides on our website to accompany today's call. With me this morning are Brett Monia, Chief Executive Officer, Beth Haugen, Chief Financial Officer, and Richard Gehry, Executive Vice President of Development. And joining us for Q&A are Eric Swayze, Executive Vice President of Research, and Oneza Katare, Chief Product Strategy and Operations Officer. I would like to draw your attention to slide three, which contains our forward-looking statement During this call, we will be making forward-looking language statements that are based on our current expectations and beliefs. These statements are subject to certain risks and uncertainties, and our actual results may differ materially. I encourage you to consult the risk factors contained in our SEC filings for additional detail. And with that, I'll turn the call over to Brett.

Thanks, Jennifer. Good morning, everyone, and thanks for joining us for today's call. We made substantial progress last year toward achieving our vision of becoming a leading, fully integrated biotechnology company. We're building our commercial pipeline, advancing and expanding our technology, and moving towards delivering an abundance of new medicines to the market, which together we expect will drive substantial future growth. At our investor date late last year, we introduced our go-to-market strategies for our near-term commercial opportunities, Eflon Thurston, Ola Zarsson, and Donnie DeLorsen. And through our strategic collaboration with AstraZeneca to jointly develop and commercialize Eflon Thurston, which we announced late last year, we bolstered our commercial organization and accelerated preparations for our near-term product launches while also positioning Eflon Thurston to win in the competitive TTR amyloidosis market. The clinical development of efluontursin, olizarcin, and donadilurcin also continued to progress well. The phase three neurotransform study of efluontursin in patients with TTR polyneuropathy completed enrollment in 2021 and remains on track for data around mid-year, followed by regulatory filing by the end of the year, assuming positive data. And the phase three cardiotransform study in patients with TTR cardiomyopathy is also progressing well, with data on track for 2024. We expanded our phase III pipeline late last year when we initiated the core severe hypertriglyceridemia study for olivaziracin. This was an important step as it expands the olivaziracin phase III program beyond FCS to potentially address the more than 3 million patients with severe elevated triglycerides. Importantly, with first mover advantage, we have the potential to deliver a first in-class medicine to this broad patient population. We plan to report data from our Olazarsen program in patients with FCS next year and in patients with severe hypertriglyceridemia the following year in 2024. We also initiated the Phase III OASIS-HAE study of donaglorsen in patients with hereditary angioedema late last year, putting it on track for data also in 2024. Donaglorsen has demonstrated a potential best in-class profile in studies to date. Given the continuing unmet medical need for more effective HAE prophylactic treatments, we see Donald-Dolorsen as a very compelling opportunity for IONIS. This year, we plan to report additional data from the Phase II study for Donald-Dolorsen and the ongoing open-label extension, further demonstrating Donald-Dolorsen's highly favorable profile based on studies to date. Now, looking more broadly at our late and mid-stage programs, beginning with programs from our leading cardiovascular disease franchise, the Phase III Healthy Little A Horizon Study of Pella-Carson continues to progress well. Novartis reached 50% enrollment last summer, and the study remains on track for data in 2025. And coming up this year, we look forward to several Phase IIb data readouts, including three from our cardiovascular franchise. Beginning with data from the Atesian study of ION449, our PCSK9 glycomedicine with AstraZeneca in patients with dyslipidemia at ACC in April. Later this year, we expect data for thezomersin, our Factor XI glycomedicine with Bayer in patients with end-stage renal disease, and Phase IIb data for IONUS-AGT-LRX in patients with treatment-resistant hypertension. Moving to our neurological disease franchise, we further strengthened our leadership position in SMA by advancing a follow-on program to Spinraza with Biogen for the treatment of SMA, which has the potential to reduce dosing frequency. We're also pleased that Biogen continues to actively engage with regulatory authorities, working towards a potential path forward for Tofersen in patients with SOD1 ALS, while continuing to evaluate the data from the Bay Street Valor Study and the Open Label Extensions. The other programs in our ALS franchise also continue to progress well, and this includes our C9 ALS program, which is on track for data in the first half of this year. We are also pleased that, based on new findings from the Generation HD1 study, Roche has identified a path forward for Tominerson in patients with Huntington's disease, with plans to initiate a new phase 2 study in younger patients with less disease burden. We look forward to additional updates from Roche later in the year. And in addition to the Spinraza follow-on, we made other important technology advancements throughout last year, including advancements in microchemistries and in the potential identification of a new backbone chemistry, as we highlighted at our investor day in December. Importantly, we achieved all these pipeline and technology advances while strengthening our overall financial position and substantially exceeding our 2021 financial guidance. And looking ahead, key catalysts that will keep moving us closer to our goal of becoming a leading, fully integrated biotechnology company. And with that, I'll turn the call over to Beth to review our 2021 financial results and our 2022 financial guidance. Then Richard will discuss our recent key pipeline achievements and highlight pipeline catalysts for the year ahead. After Richard, I'll wrap up our prepared remarks before taking your questions. So now over to Beth.