Amicus Therapeutics, Inc.

Good morning, ladies and gentlemen, and welcome to the Amicus Therapeutics Full Year 2023 Financial Results Conference Call and Webcast. At this time, all participants are in a listen-only mode. Later, we will conduct a question and answer session, and instructions will follow at that time. As a reminder, this conference call is being recorded. I would now like to turn the conference over to your host, Mr. Andrew Fonin. Vice President of Investor Relations. You may begin.

Great. Thank you, Didi. Good morning. Thank you for joining our conference call to discuss Amicus Therapeutics' full year 2023 financial results and corporate highlights. Leading today's call, we have Bradley Campbell, President and Chief Executive Officer, Sebastian Martel, Chief Business Officer, Simon Harford, Chief Financial Officer, and Dr. Jeff Castelli, Chief Development Officer. Joining for Q&A is Dr. Mitchell Goldman, Chief Medical Officer, and Ellen Rosenberg, Chief Legal Officer. As referenced on slide two, we might make forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 relating to our business as well as our plans and prospects. Our forward-looking statements should not be regarded as representation by us that any of our plans will be achieved. Any or all the forward-looking statements made on this call may turn out to be wrong and can be affected by inaccurate assumptions we might make or by known or unknown risks and uncertainties. You are cautioned not to place undue reliance on any forward-looking statements which speak only to the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement, and we undertake no obligation to revise or update this presentation and conference call to reflect events or circumstances after the date hereof. For a full discussion of such forward-looking statements and the risks and uncertainties that may impact them, we refer you to the forward-looking statements and risk factors section of our annual report on Form 10-K for the year ended December 31st, 2023, to be filed today with the Securities and Exchange Commission. At this time, it's my pleasure to turn the call over to Bradley Campbell, President and Chief Executive Officer. Bradley?

Great. Thanks, Andrew, and welcome everybody to our full year 2023 conference call. This year, our call falls a day before Rare Disease Day, so before I dive into our results, I just want to take a minute to acknowledge this important day for the rare disease community. This year's theme is achieving true health equity for the nearly 300 million people around the world living with a rare condition. Since our inception, Amicus has been dedicated to serving the needs of those in the rare disease community in extraordinary ways, and we are committed to advocating for patient communities and fighting on behalf of those whose voices have not been heard. Rare Disease Day serves as a global call to action to collectively work towards equity and social opportunity, healthcare, and perhaps most importantly, access, to medicines for people living with rare diseases. So I just ask that all of you join me in commemorating this important day for the global rare disease community. So with that, let me just shift into our financial results and outlook, and I am very pleased to highlight what tremendous progress we made across our global business last year and into the start of 2024. As you saw in this morning's press release, I'll highlight several key points before I turn it over to the team to go through more detail. So first Gallifold continues its strong performance and it remains the cornerstone of our success. We are very pleased with the continued uptake of Gallifold globally. We now have over 2,400 people living with Fabry disease who take Gallifold or taking Gallifold at the end of 2023. For the full year that translated to Gallifold revenue of $388 million globally representing 18% growth year over year. outperforming our expectations and our initial guidance for the year. Throughout 2023, we continued to observe strong trends across our key performance indicators in all of our key geographies, including continued demand through new patient starts from both switch and naive populations in all of our leading markets. We saw steady growth of in-person visits between our field team and Fabry treaters, and we continue to see sustained patient compliance and adherence rates of over 90%. Growth in 2023 was driven primarily by patient demand from that new patient starts, continuing to switch patients in our newer launch markets and continued penetration into the diagnosed and untreated population, which we expect to continue to be a major driver of growth in 2024 and beyond as the Fabry market continues to see improved diagnosis and medical education and finding patients. Just to put that in perspective for a minute, as a reminder, just over eight years ago, We estimated there were 5,000 treated Fabry patients and 5,000 patients who were diagnosed with untreated. Today, there are more than 11,000 treated patients, so the treated market has more than doubled in that time, while the untreated market has also increased to almost 6,000 patients. These underlying market dynamics will provide the opportunity to grow Gallifold for many, many years to come, including in 2024 when we expect another great year with 11% to 16% projected growth at constant exchange rates. Second, the global commercial launch of Pompility and Opfolda is underway, and it's off to a great start in the three largest Pompei markets. Our teams have made significant progress in the initial commercial launch in Germany, the UK, and the US, and we remain incredibly pleased with the launch so far. Seven weeks ago at JPMorgan, we shared that 120 patients were on treatment or scheduled for treatment, including 15 new commercial patients. And as we said at the conference, we're not going to give mid-quarter updates, but I can share more color on how incredibly pleased we are with continued patient and physician demand for this new therapy. The key performance indicators that we talked about last time continue to improve and continue to give us confidence in the strength of this launch. Specifically, the rate of new commercial patients coming onto Pombility and Opfolda continues to increase in all three markets as we progress through the early weeks of the year. We anticipate launches in additional countries. We'll add to that growth through the latter part of the year. And importantly, for our new commercial patients, we continue to see prescriptions proportionally coming from Nexviazime and Lumazime, as well as naive patients in XUS markets. So that means in the U.S., the majority of new patients are switching off of Nexviazime and coming on to Pompility and Upfolda. Whereas in Europe, where Lumazime has the majority market share, most of our switch patients are coming from that therapy, and we're seeing an increasing number of naive patients as well. So all the patient segments are performing as we would expect. In a moment, Sebastian will provide further color on the ongoing launch of these key performance indicators, but overall, we're very pleased by the launch and the great momentum we're seeing across each of our markets. Throughout 2024, we'll continue to focus on increasing patient access by expanding into additional European countries as we navigate the country-by-country pricing and reimbursement process and focus on additional regulatory submissions as well. We are incredibly pleased to be providing a real choice for patients and challenging therapeutic options for both physicians and people living with Pompe disease. Third, Anacus has maintained an incredibly strong financial position as we continue to execute on the expansion of Gallifold and advance the global launch of Pompility and Upsolda. We are pleased to share today that as we promised, we delivered our first quarter of non-GAAP profitability in the fourth quarter of 2023. This is obviously a significant milestone as we now look to deliver our first full year of non-GAAP profitability in 2024, supported for the first time by well over half a billion in combined product sales, as well as prudent expense management, two really significant milestones in the evolution of Anikus as a biotechnology company in the rare disease space. Our key strategic priorities for 2024 are laid out in the slide here. As we laid out last month, we're focused on achieving our key strategic priorities for 2024, including Number one, again, sustaining that double-digit Gallifold revenue growth of 11% to 16% in constant exchange rates. Continuing our successful launch of Pompility and Opfolda and executing multiple successful new commercial launches throughout the year. Advancing our ongoing studies to support our medical and scientific leadership in Fabry and Pompe disease. And finally, maintaining our strong financial position as we carefully manage our expenses and investments in order to achieve a full year of non-GAAP profitability. With that, let me now hand the call over to Sebastian, who can give further highlights on our commercial performance. Sebastian?