Amicus Therapeutics, Inc.

Good morning, ladies and gentlemen, and welcome to the AMACUS Therapeutic Full Quarter 2020 Fiscal Year Results Conference Call and Webcast. At this time, all participants are in 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 phone. As a reminder, this conference is being recorded. I would now like to turn the conference over to your host, Mr. Andrew Fawning, head of investor relations. You may begin.

Good morning. Thank you for joining our conference call to discuss Amicus Therapeutics' full year 2020 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 Creamy, chief financial officer, and Dr. Jeff Castelli, Chief Development Officer. Joining for Q&A will have Dr. Hung Do, Chief Science Officer, and Dr. Mitchell Goleman, Senior Vice President of Clinical Research. 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 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 risks and uncertainties that may impact them, we refer you to the forward-looking statements and risk factors section on our annual report on Form 10-K for the year-ended December 31st, 2020, 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, and welcome, everyone, to our full year 2020 results conference call. Yesterday, February 28th, marked the 14th annual World Rare Disease Day. The goal of this day each year is to raise awareness with policymakers and the public of rare diseases and their impact on the lives of the millions of people and their families living with any one of the more than 7,000 rare diseases. If you're on this call, you care about amicus and our mission, and you have an interest in rare diseases. I'd encourage you to go to the website, rarediseaseday.org, and learn more. including a moving and creating video of six heroes from six continents, all living with a rare disease. The impact of these diseases is always so personal. The need for new or newer and better medicines is so great. Yesterday morning, I sat down at my computer to edit these remarks today. I opened first an email from our team in Europe that let me know of the deaths earlier yesterday of a brave young woman living with Pompe disease. Her name was Rosella Passero. She was 21 years old and living in Naples, Italy. I first came to know of Rosella when she was two years old in the fall of 2002. Her parents had chained themselves to the front gate of a hospital in Naples, Italy, begging for compassionate use of the then experimental enzyme replacement therapy that we were developing at Genzyme. I was running the Pompe program at the time, And Henry Tremere, with his compassion and wisdom, allowed us to send the experimental enzyme to Rosella. She was the first Pompe patient in the world to receive this ERT on compassionate use. And then in December 2018, while live on Italian television in front of an audience of millions a few days before Christmas, I had the honor and the privilege to meet Rosella in person for the first time. It was a moving experience, as you might imagine. We offer our prayers and our thoughts for Rosella and her family. This is the very human side of this business that we have chosen. Rosella's life, her spirit, sadly her passing, remind us of the great urgency with which we need new treatment options for everyone living with Pompe disease and all rare diseases, and we need them as soon as possible. So with that very important reminder of our mission, we'll turn now to Amicus Business. For Amicus, 2020 was a year of excellent growth and execution across all aspects of our business, including science, clinical, regulatory, and as you see, our commercial effort. As we continue to build one of the next great global biotechnology companies poised to impact people around the world living with rare diseases. 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 $261 million in 2020 revenue, the Gallifold launch continues to exceed expectations and coming in above our guidance range that was set out last year. The full year revenue represents performance across the global business. including new patient starts from both switch and treatment-naive patients throughout the year. Second, our key regulatory and R&D timelines remain on track. Following results from the Phase III PROPEL study of ATGAA in late-onset Pompe disease, we plan to complete the rolling BLA submission 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 continue to move it rapidly toward its regulatory submissions for approval. Within our gene therapy pipeline, we also continue to move forward our lead batten disease programs for CLN6 and CLN3, as well as our most advanced preclinical gene therapy programs. Announced for the first time this morning, our CLN6 program has been granted fast-track designation by the US FDA. This follows the EMA granting the prime designation to this CLN program in late 2020. Through our broad and important research collaboration with Dr. Jim Wilson and the University of Pennsylvania, we are highly encouraged by the preclinical data and progress from our Fabry Disease Gene Therapy clinical candidates. Given the data we have seen to date, this is the second program to highlight the capabilities and potential this collaboration can bring to people living with rare diseases. And third, the amicus cash position is sufficient to achieve self-sustainability without the need for any future dilutive financing. 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. Next on slide five, we'll set, again, our key strategic priorities for this year. We are well on track to achieve our five key strategic priorities for 2021, including 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 new markets. We look to achieve global product revenue of $300 million to $315 million this year, 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 continue into early this year. Second, we remain steadfast and passionate in our commitment to advancing ATGAA to global regulatory submissions as quickly as possible for the benefit of all people living with Pompe disease. 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. Fourth, in addition, we are progressing our manufacturing capabilities and capacity to build world-class technical operations to support all gene therapy programs. And five, 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, let me now hand the call over to Bradley Campbell, our President and Chief Operating Officer, to further highlight the Gallifold performance, Brad.