Amicus Therapeutics, Inc.

Good morning, ladies and gentlemen, and welcome to the Amicus Therapeutics Second 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 Fongan, Head of Investor Relations. You may begin.

Thank you, Operator. Good morning. Thank you for joining our conference call to discuss Amicus Therapeutics second 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 Queeney, Chief Financial Officer, and Dr. Jeff Costelli, Chief Development Officer. Joining for Q&A will have Dr. Mitchell Goldman, Chief Medical Officer as well. 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 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, 2020, and the quarterly report on Form 10Q for the quarter ended June 30th, 2021, to be filed later today with the Securities and Exchange Commission. At this time, it's my pleasure to turn the call over to John Crowley, Chairman and Chief Executive Officer. John?

Great. Thank you, Andrew, and welcome everyone to our second quarter 2021 results conference call. Before we begin, I just want to note yesterday the sudden death of Dr. Tachi Yamada. Tachi, as many of you know, is a giant in our industry, and among his many contributions, he was a passionate advocate for research for people living with rare diseases, and I came to know Tachi well as he chaired the Orkin Disease Center with Jim Wilson at the University of Pennsylvania, where I had the honor to serve with him, and our deepest consolances to his family. I am pleased to report for Amicus that the second quarter of 2021 reflects execution across our 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 performance and remains the cornerstone of our success. With $77 million in second quarter revenue, we are very pleased with the continued momentum of the Gallifold uptake globally. The number of patients on Gallifold at the end of the quarter continues to exceed our internal expectations as we continue to add new patients from both switch and naive populations throughout the quarter. And exciting news for Gallifold. On Monday this week, we announced that the European Commission has approved the expanded EU label for Gallifold for the long-term treatment of February disease in adolescents aged 12 and older with an amenable mutation. This is a transformative moment for the February community in the European Union, where pediatric patients now aged 12 and above have a new treatment option for the first time in over 15 years. Second, our key regulatory initiatives for ATGAA remain on track, and our R&D pipeline of gene therapies continues to advance. I am pleased to share today that the rolling BLA and the NDA submissions for ATGAA, our novel next-generation two-component therapy for Pompe disease, have been completed. We are also pleased to share that following a positive rapporteur and co-rapporteur meeting, the EU regulators are supportive of marketing authorization application, or MAA, submissions for ATGAA. We expect to complete these EMA submissions in the second half of this year. We continue to have great confidence in ATGAA to benefit people living with Pompe disease, and we intend to move it rapidly through these regulatory submissions towards approval. Within our gene therapy pipeline, we continue to further lead batten programs for CLN3 and CLN6, as well as our most advanced preclinical gene therapy programs. And through our broad research collaboration with Dr. Jim Wilson and the University of Pennsylvania, we are highly encouraged by the continued preclinical data and the progress we have seen thus far from our Febre and Pompe disease gene therapy clinical candidates. And third, the amicus cash position is sufficient to achieve self-sustainability under the current plan 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. 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 disease, We will continue to drive Gallifold's availability to more people living with Febre disease with amenable variants in existing and in new markets. We look to achieve global product revenue of $300 million to $315 million this year, which reflects strong momentum and demand behind this precision medicine globally. 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. Third, we are advancing our industry-leading rare disease gene therapy portfolio. 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. In addition, we are progressing our manufacturing capabilities and capacity to build world-class technical operations to support all amicus gene therapy programs. And finally, again, we continue to maintain a strong financial position as we carefully manage our expenses and investments. Again, we remain fully funded through all major milestones. So with that introduction, let me go ahead now and hand the call over to Bradley Campbell, our President and Chief Operating Officer to further highlight the Gallifold performance. Bradley.