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5/1/2025
Thursday, May 1st, 2025. I would now like to turn the conference over to El Nilem. Please go ahead.
Good morning. I'm Christine Lindenboom, Chief Corporate Communications Officer at El Nilem. With me today are Vaughn Greenstreet, Chief Executive Officer, Tolga Tangular, Chief Commercial Officer, Pushbull Garg, Chief Medical Officer, and Jeff Fulton, Chief Financial Officer. For those of you participating via conference call, the accompanying slides can be accessed by going to the events section of the investors page of our website investors.alnylam.com slash events. During today's call, as outlined in slide two, Yvonne will offer introductory remarks and provide some general context. Tolga will provide an update on our global commercial progress, Pushkal will review pipeline updates and clinical progress, and Jeff will review our financials and guidance, followed by a summary of our upcoming milestones before we open the call to your questions. I would like to remind you that this call will contain remarks concerning Alnylam's future expectations, plans, and prospects, which constitute forward-looking statements for the purposes of the safe proper provision under the Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including those discussed in our most recent periodic report on file with the SEC. In addition, any forward-looking statements represent our views only as of the date of this recording and should not be relied upon as representing our views as of any subsequent date. We specifically disclaim any obligation to update such statements. With that, I'd like to turn the call over to Yvonne. Yvonne?
Thanks, Christine, and thank you, everyone, for joining the call today. 2025 is off to a strong start at Sound Island. In the first quarter, we made notable progress towards our goal of becoming the global TTR leader, which will play a critical role in driving sustainable growth and value creation this year and beyond. Commercially, we delivered $469 million in combined net product revenues, and this represents 28% growth year-over-year. This was driven primarily by the robust 45% year-over-year growth of our US TTR franchise, which in the first quarter consisted solely of continued uptake in the hereditary polyneuropathy population. We also had several exciting portfolio advancements in the quarter, most notably the expanded indication from VUTRA in ATTR cardiomyopathy. with approvals in the US and Brazil. And earlier this week, we received a positive CHMP opinion in the EU. We also saw approval of the sixth Ireland-discovered RNA therapeutic, QFITLIA, for haemophilia A or B, secured by our partners at Sanofi. Lastly, we are reiterating our guidance for the year, underscoring our confidence in continued growth and a strong launch in ATTR cardiomyopathy. This also includes our goal of achieving sustainable non-gap profitability in 2025. Now, before I move on, I'd like to take just a brief moment to acknowledge the uncertainty that exists related to potential evolutions in certain policies, including tariffs, FDA personnel reorganization, and other biotech industry initiatives being discussed. While much has yet to be clarified and finalized, We've taken a comprehensive look across our business, and we believe that our 9M is well positioned and pretty well insulated from any potential impacts associated with the key initiatives that have been announced to date. We feel that we're completely able to focus on the key tasks at hand, executing on a strong launch in ATTR cardiomyopathy and continuing to advance our robust, high-value pipelines. Our achievements in early 2025 underpin the three core elements of our business that we believe will drive sustainable growth and value creation for years to come. The first is TTR leadership. Tolga will provide color on our launch of Ambutra in ATTRCM, but at a high level, we're very pleased with our early progress, which is focused on securing broad access, driving awareness, and supporting treatment initiation for patients in the initial few weeks since approval. The next is growth through innovation, including a pipeline with numerous multi-billion dollar opportunities and an R&D engine set up to deliver sustainable innovation and value creation. The third element is strong financial performance with robust commercial delivery and a disciplined approach to capital allocation to enable us to sustain profitability going forward. And of course, All of this is underpinned by a best-in-class team and our award-winning culture. And this all represents tremendous progress against our Nilem piece of 5x25 goals. And we continue to work to turn these goals into reality by the end of the year. Doing so will further establish our Nilem as a unique, top-tier biotech company, delivering sustainable innovation to patients for years to come. With that, Let me now turn the call over to Tolga for a review of our commercial performance. Tolga?
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