4/29/2026

speaker
Sabrina
Conference Operator

Good day and welcome to IONI's first quarter 2026 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 Wolk, Senior Vice President of Investor Relations, to lead off the call. Please begin.

speaker
Wade Wolk
Senior Vice President, Investor Relations

Thank you, Sabrina. 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 that accompany today's call. With me this morning are Brett Monia, Chief Executive Officer, Kyle Genet, Chief Global Product Strategy Officer, and Beth Haugen, Chief Financial Officer. Holly Kordasiewicz, Chief Development Officer, Eugene Schneider, Chief Clinical Development Officer, and Eric Swayze, Executive Vice President of Research, will also join us for the Q&A portion of the call. I'd 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 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, Wade.

speaker
Brett Monia
Chief Executive Officer

Good morning, everyone, and thank you for joining us today. IONIS entered 2026 with significant momentum, which continued to accelerate through the first quarter of this year. Our performance to date highlights our strong execution across the business, which positions us to fuel substantial growth for years to come. We are very pleased with the continued success of Tringolza. Demand continues to grow, reflecting both a compelling clinical profile and strong launch execution by our team. The launch of Tringolza is now also underway in Europe through our partner Sobe, expanding access to this transformational therapy for people with SCS. We also continue to advance our launch of Donzera. We're very encouraged by the strong early trajectory and the breadth of prescribers across patient segments. Outside the U.S., Donzera received European approval earlier this year, and our partner, Atsuka, has now initiated launch activities across the region. Together, Tringolza and Donzera give us a strong foundation of successful commercial execution as we look ahead to two additional independent launches this year, with more to come. We are on track for the launch of olosarcin and severe hypertriglyceridemia, which represents our first independent launch in a broad patient population. We are pleased to have received priority review by the FDA with the PDUFA date of June 30, reflecting the significant unmet need in SHTG and the groundbreaking results from our landmark CORE and CORE2 studies. In these studies, we demonstrated profound and highly statistically significant reductions in triglycerides and acute pancreatitis events, along with favorable safety and tolerability. Today, based on HCP demand and payer research, we are increasing our annual peak sales estimate for Olazarsen from greater than $2 billion to now greater than $3 billion, positioning Olazarsen to become Ionis' first wholly-owned, multi-billion-dollar medicine as the new standard of care for treating patients with severe hypertriglyceridemia. Zilgenersen for Alexander disease is our second planned independent launch this year and the first from our industry-leading neurology pipeline. Zilgenersen is the first and only medicine to demonstrate clinically meaningful and disease-modifying benefit in Alexander disease, a devastating and often fatal leukodystrophy with no approved treatments today. We submitted our NDA in January, and based on the results of our pivotal study, the FDA accepted our NDA with priority review and a PDUFA date of September 22. Overall, we are on track to have three independent medicines for four indications on the market in 2026. Gringolza for FCS and SHTG, Donzera for HAE, and Zilgenursen for Alexander disease. This marks a major step in Ionis' evolution as a fully integrated commercial biotech company. Complementing our wholly owned portfolio is our partner pipeline, a rich pipeline that continues to advance toward multiple value-driving events this year. Peperaversin, our potential first in-class medicine for chronic hepatitis B, is on track to launch in the U.S. and Japan this year by our partner GSK. And just this week, Peperaversin was granted breakthrough therapy designation and accepted for priority review by FDA with a PDUFA date of October 26. Next month at EASL, GSK will present the unprecedented results of the Phase III program in which Peperaversin achieves statistically significant and clinically meaningful functional cure rates in patients with chronic hepatitis. hepatitis B. We also remain on track for data from two major cardiovascular outcome trials from our partner pipeline this year. The Pella-Carson LP little a Horizon trial in patients with elevated LP little a in cardiovascular disease and the Epilon-Tersen CardioTransform trial in trans-thyroid mediated cardiomyopathy. Including these programs, along with Peperaversin, we expect five partner-led launches by the end of next year. creating a diversified stream of royalties and milestones for IONIS with multibillion-dollar potential well into the next decade. In addition to our commercial and pipeline achievements, we also delivered strong financial results in the first quarter. As a result of this strong Q1 performance and outlook for the balance of the year, today we are announcing a significant improvement to our financial guidance, which Beth will cover in details in a few moments. IONIS has achieved a great deal of late. Our pipeline and our launches are delivering tremendous value, and we continue to strengthen our financial position. But even more importantly, we are well positioned and committed to build on the success to drive far greater value for patients and our shareholders. And with that, I'll turn the call over to Kyle.

Disclaimer

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