Amicus Therapeutics, Inc.

Good morning, ladies and gentlemen, and welcome to the Amicus Therapeutics first quarter 2021 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. If anyone should require assistance during the conference, please press star and then zero on your touchtone telephone. As a reminder, this conference call is being recorded. I would now like to turn the conference over to your host, Mr. Andrew Fonin, Head of Investor Relations. You may begin.

Thank you, Mary. Good morning. Thank you for joining our conference call to discuss Amicus Therapeutics first quarter 2021 financial results and corporate highlights. Speaking on today's call, we have John Crowley, Chairman and Chief Executive Officer, Bradley Campbell, President and Chief Operating Officer, Daphne Queamy, Chief Financial Officer, and Dr. Jeff Castelli, Chief Development Officer. Joining for Q&A, we'll have Dr. Mitchell Goleman, Senior Vice President of Clinical Research, joining. As referenced on slide two, 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 of 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 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 31, 2020, and the quarterly report on Form 10-Q for the quarter-ended March 31, 2021, to be filed later today with the Securities and Exchange Commission. At this time, it is my pleasure to turn the call over to John Crowley, Chairman and Chief Executive Officer.

John? Great. Thank you, Andrew. Good morning and welcome, everyone, to our first quarter 2021 results conference call. I'm pleased to report that the first quarter of 2021 reflects execution across the key strategic priorities that we outlined at the beginning of the year. As we did in this morning's press release, I'd like to highlight several key accomplishments. First, Gallifold continues its strong launch performance and remains the cornerstone of our success. With $66.4 million in first quarter revenue, we're very pleased with the continued momentum of the Gallifold uptake globally. The number of patients on Gallifold at the end of the quarter continued to exceed our internal expectations, despite continued headwinds from COVID-19, as well as uneven ordering patterns. Second, our key regulatory timelines for ATGAA remain on track. and our R&D pipeline of gene therapies continues to advance. Following a successful pre-BLA meeting and written communication with the US FDA, we continue with our plans to complete the rolling BLA submission of ATGAA in the second quarter of this year and anticipate additional regulatory submissions in the European Union and in other geographies throughout 2021. We continue to have great confidence in ATGAA to benefit people living with Pompe disease, and we intend to move it rapidly through regulatory submissions onward towards approval. Within our gene therapy pipeline, we continue to further our lead batten disease programs for CLN6 and CLN3, as well as our most advanced preclinical gene therapy programs. Through our broad research collaboration with Dr. Jim Wilson and the University of Pennsylvania, we are highly encouraged by the preclinical data and progress from our Febre and Pompe disease gene therapy clinical candidates. We are pleased to share that new preclinical data from both programs will be presented at ASGCT this week. Third, the amicus cash position is sufficient to achieve self-sustainability without the need for any future dilutive financings. Our continued revenue growth, prudent expense management, and growth potential has allowed us to reach this important milestone as we continue to realize our vision of delivering groundbreaking and potentially curative new medicines for people living with rare diseases around the world. And finally, as we announced just a few days ago, I'm very pleased to welcome our new Senior Vice President of Strategy and Business Development, Sebastian Martell, to the Amicus team. Sebastian served most recently as global head of rare diseases at Sanofi Genzyme and brings deep expertise in corporate strategy, business development, and commercialization. We look forward to Sebastian's contributions to advancing Amicus's mission for patients as a leading global biotechnology company. Turning to slide four, we are well on track to achieve our five key strategic priorities for 2021. These include Gallifold, our precision medicine for February. We will continue to drive Gallifold to more people living with February disease with amenable variants in existing and in new markets. We look to achieve this year global product revenue of $300 million to $315 million, which reflects the strong momentum and demand behind this precision medicine globally, despite some COVID-related disruptions that we saw in the latter part of 2020 and which continued into early this year. Second, we remain steadfast and passionate in our commitment to advancing ATGAA through global regulatory submissions for the benefit of as many people living with Pompe disease and as quickly as possible. Three, we are advancing our industry-leading rare disease gene therapy portfolio, again stemming from our global research and gene therapy center of excellence in Philadelphia. We will be advancing the clinical development, manufacturing, and regulatory discussions across multiple programs in our gene therapy pipeline. Four, in addition, we are progressing our manufacturing capabilities and capacity to build world-class technical operations to support all gene therapy programs. And five, finally, again, we continue to maintain a strong financial position as we carefully manage our expenses and investments. and we remain fully funded through all major milestones. So with that introduction this morning, let me now hand the call over to Bradley Campbell, our President and Chief Operating Officer, to further highlight the Gallifold performance. Bradley. Bradley Campbell Great.