Amicus Therapeutics, Inc.

Good morning, ladies and gentlemen, and welcome to the Amicus Therapeutics Full Year 2022 Financial Results Conference Call-In 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 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, Operator. Good morning, everyone. Thank you for joining our conference call to discuss Amicus Therapeutics' full year 2022 financial results and corporate highlights. Leading today's call, we have Bradley Campbell, President and Chief Executive Officer, Daphne Cleamy, Chief Financial Officer, Sebastian Martel, Chief Business 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 2, we may 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. We 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, 2022 to be filed to the Securities and Exchange Commission today. At this time, it is my pleasure to turn the call over to Bradley Campbell, President and Chief Executive Officer. Bradley.

Great. Thanks, Andrew, and welcome everyone to our full year 2022 results conference call. I'm pleased today to highlight the progress we've made across our business throughout 2022 and now into the start of 2023. As we did in this morning's press release, let me highlight several key points. First, Gallifold continues its strong performance and remains the cornerstone of our success. We continue to be very pleased with the strong demand for and growth of Gallifold globally, with now over 2,000 people living with Fabry disease taking Gallifold as their only treatment for Fabry. What an amazing milestone for this precision medicine. And despite the significant foreign currency headwinds experienced throughout last year, in 2022, we reported Gallifold revenue growth of 8% or 16% on an operational or constant currency basis. Throughout last year and into this year, we continue to see strong trends across a number of our key performance indicators, including increasing demand through new patient starts from both the switch and naive populations, continued growth in in-person visits between our field teams and Fabrate treaters throughout the world, and sustained patient compliance and adherence rates of over 90%. In 2023, we expect to see continued double-digit Gallifold growth fueled by significant demand from patients and physicians. We're also pleased by the growing Gallifold intellectual property estate through the issuance of 19 new patents over the last year. There are now a total of 46 Orange Book listed issued patents related to Gallifold in the United States, 30 of which provide protection to 2038 and beyond. And importantly, this includes five composition of matter patents. We see this patent portfolio, along with our orphan drug exclusivity, providing broad and long-term intellectual property rights well into the late 2030s for this novel precision medicine. Second, we continue to make progress on our global regulatory filings and commercial launch planning for ATGAA, our novel two-component therapy for Pompe disease. We are thrilled today to announce the US FDA has scheduled the required pre-approval inspection of the Wuxi Biologics manufacturing site in China during the second quarter this year. Pending completion of a successful inspection, We anticipate the regulatory approval and launch of ATGAA in the United States will be during the third quarter of 2023. Additionally, in Europe, we're on track with the regulatory reviews by both EMA and MHRA on the marketing authorization applications for ATGAA. As a reminder, in December, the CHMP adopted a positive opinion of Sipic Glucosidase Alpha to be marketed as POMbility, with the European Commission approval expected this quarter. We anticipate a CHMP opinion from Miglustat, the enzyme stabilizer component of ATGA, in the second quarter of this year. And as a reminder, the EMA has indicated that it does not require an inspection of the WUSHI manufacturing site as a condition of their approval. In the UK, the regulatory submission process for ATGA was initiated in December and is on track for an expected approval in the third quarter this year. As we've observed throughout 2022, we continue to see significant interest in participation in our expanded access mechanisms for ATGA globally. Programs are now in place in the US, United Kingdom, France, Germany, and Japan, with a growing number of patients participating in each. Across all of our ongoing clinical studies and expanded access programs, we're now over 195 patients on ATGA today, which we believe represents more than 5% of the total treated Pompe patients around the world. We expect this number to continue to grow as we approach approvals in the United States and Europe and beyond. In anticipation of these approvals in the near term, our global launch plans continue to move ahead, including our pre-launch activities, targeted investments, and additional personnel and capabilities to support the launch and ongoing investments in building launch inventory. We're closer to reaching this much anticipated milestone of getting ATGA across the regulatory finish line. and providing another treatment option for people living with Pompe disease in the United States, Europe, and the United Kingdom with further regulatory applications planned in the months ahead. And third, Amicus has maintained a strong financial position as we continue to execute in the global expansion of Gallifold and prepare for the global launch of ATGA. Importantly, based on the projected strong growth of Gallifold, our latest assumptions on approvals and launch for ATGA, our current operating plan and continued careful management of expenses, we remain on track to achieve non-GAAP profitability, which we continue to expect in the second half of 2023. And finally, just to round out the key highlights and progress we've made across our Fabry and Pompe disease programs, I do want to mention that we once again had a significant presence at the 19th Annual World Symposium held this February in Orlando, Florida. Anacus had two oral presentations and 11 posters highlighting data from our development programs in Fabry and Pompe disease. Most notably, As Jeff will go into more detail later in the call, we represented for the, excuse me, we presented for the first time encouraging long-term efficacy and safety data from the phase three open-label extension study of ATGA in late-onset Pompe disease, demonstrating consistency and durability of effect in patients now out to two years. Also noteworthy, we provided the first results from our own Follow Me Fabray Pathfinders Registry, showing stability in EGFR out to three years in that population. And this will be a key part of our efforts to continue to build the body of evidence supporting Gallifold. Finally, in slide four, as we laid out last month, we are focused on achieving our key strategic priorities for 2023, including number one, sustaining double-digit Gallifold revenue growth of 12 to 17% at constant exchange rates, while delivering Gallifold to more people living with Fabry disease with amenable variants in existing and in new markets. Number two, we remain laser-focused on securing ATGA regulatory approvals by the FDA, EMA, and MHRA later this year, as well as continuing to advance preparations for the respective anticipated launches. Given our experience and success with Gallifold and our seasoned global commercial, medical, and market access teams, we are fully prepared for and anticipate a successful launch of ATGA. Number three, we will continue to judiciously invest in the advancement of our best-in-class next-generation Fabre and Pompe genetic medicines and capabilities, as well as our next-generation chaperone for Fabre disease. And number four, as always, we will maintain a strong financial position as we carefully manage our expenses and investments on our path to non-GAAP profitability. With that, let me now hand the call over to Sebastian Martell, our Chief Business Officer, to further highlight the Gallifold performance.