7/30/2025

speaker
Steve
Conference Call Operator

Good morning and welcome to IONIS second quarter 2025 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 Walke, Senior Vice President of Investor Relations, to lead off the call. Please go ahead.

speaker
Wade Walke
Senior Vice President, Investor Relations

Thanks, Steve. 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've 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, Richard Geary, Chief Development Officer, and Beth Haugen, Chief Financial Officer. Eugene Schneider, our 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 would like to draw your attention to slide three, which contains a 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 on today's call. We have continued to build strong momentum across our business, highlighted by the excellent early commercial performance of our first independent launch, Tringolza, the first and only FDA-approved treatment for familial chydomyconemia syndromes. Tringolza exceeded revenue expectations during its second quarter on the market, underscoring its strong therapeutic profile, our well-executed commercial strategy, as well as the significant unmet need Tringolza is addressing. The revenue and commercial performance of Tringolza to date, along with our confidence in Tringolza's continued commercial success, supports the increase in our financial guidance for 2025, which Beth will cover in more detail shortly. We're also pleased that Tringolza recently received a positive CHMD opinion, paving the way to bring this transformative therapy to patients across Europe. Shifting gears, we anticipate Donna DeLorsen for hereditary angioedema, or HAE, will be our second independent launch by receiving FDA approval next month. Based on the progress of the review, we believe the approval and subsequent launch remains on track. Don DeLorsen has the potential to become a preferred prophylactic therapy for many HAE patients, with strong efficacy and favorable safety and tolerability in the pivotal studies, a patient-friendly autoinjector, and a convenient dosing schedule of monthly or every other month self-administration. We're well-positioned to launch Don DeLorsen following approval. Additionally, our Phase III pipeline continues to advance well, with data expected later this year from two wholly owned Ionis programs. If positive, these results would support the continued steady cadence of independent launches next year, bringing important new treatments to patients. These include a second indication for Olisarsen in severe hypertriglyceridemia, or SHTG, a condition with a large patient population and significant unmet need, and Zilgenersen for Alexander disease of severe rare leukodystrophy with no approved disease-modifying therapies. Together, these programs, in addition to two goals in FCS and Don Dolores and HAE, represent major breakthroughs for patients and represent multibillion-dollar revenue potential for Ionis. Additionally, by the end of 2027, we anticipate four potential launches from our rich late-stage partner pipeline, targeting serious life-threatening conditions for both rare and highly prevalent diseases. These will further expand the impact and reach of Ionis increase total revenue. With our strong momentum across the business, including Tringolza and our upcoming independent and partner launches, Ionis is well positioned to bring transformative medicines to patients for years to come and in turn achieve sustained revenue growth and positive cash flow. And with that, I'll turn the call over to Kyle.

Disclaimer

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Investor presentation