8/1/2024

speaker
Danielle
Operator

Good morning and welcome to the Iona second 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 Walk, Senior Vice President of Investor Relations, to lead off the call. Please begin.

speaker
Wade Walk
Senior Vice President, Investor Relations

Thank you, Danielle. Before we begin, I encourage everyone to go to the investor section of the Iona's website to view the press release and related financial tables that 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've also posted slides on our website that accompany today's call. With me on this morning's call are Brett Monia, our Chief Executive Officer, Richard Geary, Chief Development Officer, Kyle Genet, Chief Global Project Strategy Officer, and Beth Haugen, our Chief Financial Officer. Eric Swayze, Executive Vice President of Research, Eugene Schneider, Chief Clinical Development Officer, and Jonathan Burchell, Chief Commercial Officer, will also join us for the Q&A portion of our call. I would like to draw your attention to slide three in our presentation, which contains our forward-looking language statements. 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.

speaker
Brett Monia
Chief Executive Officer

Thanks, Wade. Good morning, everyone. Thanks for joining us on today's call. We've achieved a great deal during the first half of this year, delivering many significant milestones as we continue to execute on our vision to bring better futures to people with serious diseases. Today, biomes discovered and developed medicines are reaching more and more people. hosted by two recent launches, WENUA, for people with hereditary APTR polyneuropathy in the U.S., and CALSATI, the first approved treatment for a genetic cause of ALS, currently available in the U.S. and now approved and available in Europe. And with the progress we're making across our pipeline, our medicines are well-positioned to continue reaching more and more patients for years to come. The U.S. launch of our first Ionis co-branded medicine, Wenua, is progressing well with AstraZeneca, and we expect to bring Wenua to even more patients across the globe with the recent approval in Canada, an anticipated approval decision in Europe later this year, and with additional regulatory submissions completed in many other geographies with more coming. Today, we are even more confident in the potential of Wenua to address the larger ATTR cardiomyopathy population. As the largest study ever conducted in this patient group, our ongoing landmark CardioTransform trial is on track to deliver the most comprehensive and the most robust data set in this patient population. We believe Wingoa has the potential to be the treatment of choice for the global ATTR population. Our confidence is based on we knew a strong efficacy profile as demonstrated in hereditary ATTR polyneuropathy and the freedom of simple at-home self-administration, together with AstraZeneca's global cardiovascular commercialization leadership, along with our leadership in VTR amyloidosis. As the Wainoa launch accelerates, we're also preparing for the launch of Olisarcin in FCS later this year, our first independent launch of a whole-immune medicine for Ionis. Olisarcin represents one of the most meaningful opportunities in our pipeline today. as we prepare to first launch an FCS, a severe rare disease with no approved treatments in the U.S., and then to expand to the much larger SHTG patient population. Our NDA submission for olizarcin was recently accepted with priority review. Our PDUFA date is set for December 19th, and we're ready to bring this groundbreaking medicine to people with FCS. We also recently completed enrollment for our SHTG Phase III program, keeping us on track for data in the second half of next year. Based on our significant first-mover advantage and our positive results demonstrated to date, if approved, we believe Olazarsen could be the standard of care for both SCS and SHTG. We're building on the capabilities we've established for Renewal and Olazarsen in preparation for our anticipated launch of Diamond Olazarsen. for HAE prophylaxis next year. In our comprehensive clinical program, Donna Dolorsen has demonstrated strong evidence of meaningful benefit across multiple measures of disease with a favorable safety and tolerability profile. These data, together with the potential for monthly or every two months self-administration using an autoinjector, strengthen our belief that, if approved, Donna Dolorsen has the potential to advance the treatment paradigm for people living with HAE. With positive phase three data in hand, we expect to submit the MDA for Donald Olorson soon as we look to independently bring Donald Olorson to patients in the U.S. Outside the U.S., our commercial partner, Atsuka, is preparing to file for marketing approval in Europe later this year. And with our recently expanded license agreement, Atsuka is also working to bring Donald Olorson to people with HAE in the Asia-Pacific region. Following Renewal, Olorson and Donald Olorson Our next wave of wholly-owned opportunities includes our program for Angelman syndrome. Based on the positive results from the HALOS Phase 1-2 study that we presented last week, we are advancing this potentially transformational medicine into a Phase 3 study, which we anticipate will begin in the first half of next year. ION582 is positioned to become the cornerstone of our wholly-owned neurology franchise. which today includes five wholly-owned medicines and clinical development, with more to come by the end of the year. 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 next-level value for all Iona stakeholders. With that, I'll turn the call over to Richard to discuss our recent pipeline progress and preview key upcoming events. Next, Kyle will review the Wainua launch and our launch plans in progress for Olasarsen and Bonneville-Worson. And Beth will review our financial results. I'll then wrap up things before taking your questions. With that, over to Richard.

Disclaimer

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