Ionis Pharmaceuticals, Inc.

Good morning and welcome to the IONIS Third Quarter 2024 Financial Results Conference Call. As a reminder, this call is being recorded. At this time, I would like to turn the call over to Wade Wach, Senior Vice President of Investor Relations, to lead off the call. Please begin.

Thank you, Danielle. 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 financials results better represent the economics of our business and how we manage our business. We've also posted the slides on our website that accompany today's call. With me on this morning's call are Brett Monia, our Chief Executive Officer, Eugene Schneider, Chief Clinical Development Officer, Kyle Genet, Chief Global Product Strategy Officer, and Beth Haugen, Chief Financial Officer. Richard Geary, Chief Development Officer, Eric Swayze, Executive Vice President of Research, and Jonathan Birchall, Chief Commercial Officer, will also join us for the Q&A portion of the call. I would like to draw your attention to slide three, which contains our forward-looking language statement. During this call, we will be making forward-looking statements that are based on our current expectations and beliefs. These statements are subject to certain risks and uncertainties, and our action 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, Wade. Good morning, everybody, and thank you for joining us on today's call. This is a pivotal time for Ionis. Nearly five years ago, we set out to execute on a new vision focused on bringing our innovative medicines directly to patients in need. And today, we're on the cusp of achieving that vision with our first planned independent launch just a few weeks away. Furthermore, we anticipate three additional initiatives independent launches over the next three years, positioning Ionis to enter a new era, marked by delivering a steady cadence of important new medicines to people with serious diseases. Olazarsen represents one of the most meaningful, wholly-owned opportunities in our late-stage pipeline today. It has the potential to address two important patient populations. With an FDA action date next month, we're ready to first bring Olazarsen to people with familial chylomicronemia syndrome, or FCS, a serious and rare disease that today has no approved treatments in the United States. And with phase three data and people with severe hypertriglyceridemia, or SHCG, planned for the second half of next year, we expect to bring olzarsen to a much larger patient population in 2026. With significant first mover advantage in both populations and compelling results already demonstrated in FCS, coupled with our expectation for similarly positive data in SHTG, we believe Olazarsen could be the standard of care for both disease indications. In parallel, we're leveraging the capabilities established for Wainua and Olazarsen in our preparations to launch Donadilorsen next year, our first in-class treatment for HAE prophylaxis. We're pleased that our NDA submission was recently accepted for review by FDA with no plan for an adcom. Our PDUFA date is set for August 21st, 2025. And outside the U.S., our commercial partner, Otsuka, expects to file for marketing approval in Europe soon. We've generated important and compelling positive data from our comprehensive Donald Lawson clinical program, including encouraging data from our OASIS Plus switch cohort. These results, together with the potential for monthly or every two-month self-administration using an auto-injector, strengthen our belief that if approved, Donna Delorsen has the potential to advance the treatment paradigm for people living with HAE. Looking further ahead, our next wave of wholly-owned opportunities is progressing rapidly and includes our program for Angelman syndrome, ION582. Based on the positive results from the HALOS Phase 1-2 study and our alignment with FDA on the Phase 3 study design, we're on track to advance this potentially transformational medicine into Phase 3 development in the first half of next year. ION582 is an important program in our growing wholly-owned neurology franchise, which now includes seven medicines in clinical development. On top of the great progress we're making across our wholly-owned pipeline, our partner programs are also advancing very well, allowing more IONs discovered and developed medicines to reach more and more people, as demonstrated this year with the successful launches of Wainua and CalSati. The launch of our first Ionis co-branded medicine this year, Wainua, for people with hereditary ATTR polyneuropathy, continues to progress well with AstraZeneca. Wainua is now approved and available in major markets, including the US, Canada, and the UK, and recently received a positive CHMP opinion in Europe. And we're confident in the potential of Wainua to address the larger ATTR cardiomyopathy population with our ongoing landmark CardioTransform trial on track to deliver the most comprehensive and most robust data set in these patients in the second half of 2026. We believe Wainuiwa has the potential to be the treatment of choice for the global ATTR population based on its strong efficacy profile and hereditary ATTR polyneuropathy and the freedom of simple at-home self-administration together with AstraZeneca's global cardiovascular leadership and our leadership in TTR amyloidosis. And we're also pleased that CALSATi, the first approved treatment for a genetic cause of ALS, a medicine that was conceived and discovered by Ionis and commercialized by our partner Biogen, is now launched and available in Europe and China, in addition to the U.S. Our other partner programs are also progressing well. This includes the ongoing Phase III Horizon Study of Pellicarsin for LpA-driven cardiovascular disease, being developed by Novartis with data expected next year. and Depuraversin in Phase 3 development with GSK for chronic HPV infection now fully enrolled with data expected in 2026. Our accomplishments so far this year and the investments we're making move us closer to achieving our goal of bringing a steady cadence of new transformational medicines to patients for years to come and generating increased value for all Iona stakeholders. And with that, I'll turn the call over to Eugene.