Amicus Therapeutics, Inc.

Good morning, ladies and gentlemen. Welcome to the Amicus Therapeutics Third 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 touch-tone 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, Operator, and good morning, everyone. Thank you for joining our conference call to discuss Amicus Therapeutics' third quarter of 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 Creamy, Chief Financial Officer, and Dr. Jeff Costelli, Chief Development Officer. Joining for Q&A will have Dr. Mitchell Goleman, Chief Medical Officer, and Sebastian Martel, Chief Business Officer, 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 the forward-looking statements made on this call are may turn out to be wrong and can be affected by inaccurate assumptions we might make or by known or unknown risks and uncertainties. Your caution 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 factor 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 September 30, 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, everyone. Good morning and welcome to our third quarter 2021 results conference call. I am pleased to report that the third quarter reflected broad execution across our business as we remain on track to achieve our key strategic priorities for the year. As we did in this morning's release, I'd like to go ahead and highlight several key updates. First, Gallifold continues its strong performance and remains the cornerstone of our success with 79.4%. $5 million in third quarter revenue. We are very pleased with the continued momentum of Gallifold globally as we added new patients from both switch and naive populations throughout the quarter. Second, we continue to make tremendous progress on our global regulatory filings for ATGAA, our novel next generation therapy for Pompe disease. In September, the US Food and Drug Administration accepted the review accepted for review the biologics license application for Cipaglucosidase-alpha and the new drug application for Miglostat, the two components of ATGAA. The U.S. review is underway. The FDA has set a PDUFA action date of May 29, 2022, for the NDA related to the enzyme stabilizer, and July 29, 2022, for the BLA of the biologics. The two regulatory filings significantly cross-reference each other, and we expect that they will be reviewed together. In the European Union, following our previously announced positive rapporteur and co-rapporteur meeting, I am pleased to share for the first time this morning that the marketing authorization applications, or MAA, for ATGAA have been submitted to the European Medicines Agency. We are now closer to having another potential treatment option for people living with Pompe disease both in the United States and in Europe, with further regulatory applications planned in the months ahead. We are confident in the potential benefits of ATGAA and what it can bring to people living with Pompe disease around the world, and we look forward to working with regulators to get this important medicine to as many patients as quickly as possible. On the gene therapy front, we continue to make excellent progress in advancing and developing the industry's leading portfolio of next generation gene therapies for people living with rare genetic diseases. Our teams are preparing and making great progress toward the business combination in which the amicus gene therapy business will be acquired by ARIA IV, a special purpose acquisition company. Through this business combination, the amicus gene therapy programs Technologies, intellectual property, key personnel, and relationships will form a new company, Caritas Therapeutics. We strongly believe that separating our business into two highly focused standalone companies is the best way to unlock value for amicus shareholders and to properly fund and focus our gene therapy efforts. In a single stroke, we will have created what we believe will be one of the world's preeminent next-generation genetic medicine and gene therapy companies in Caritas, while significantly strengthening Amicus for its future. Caritas, which is the Latin word for compassion, will launch with approximately $400 million in capital. Amicus will become the largest shareholder in Caritas with a 36% ownership stake and retain co-development and commercialization rights to the Fabre and Pompe gene therapy programs, as well as negotiation rights on select future muscular dystrophy programs. As we disclosed at the time of announcement, I will lead Caritas as its chairman and CEO, while remaining the chairman emeritus and chief strategic advisor for amicus. In this amicus role, I will work closely with Bradley and the board of directors at amicus to ensure that we get ATGAA approved and launched in all the major regions around the world, as well as to support major strategic initiatives. Bradley Campbell will succeed me at Amicus as Chief Executive Officer. The Caritas Form S4 is now filed with the Securities and Exchange Commission, and the launch of Caritas Therapeutics is expected in late 2021 or early 2022. dependent on customary closing conditions. I'd also like to provide an update to our CLN6 Batten gene therapy program. We have very recently learned of the loss of two children with CLN6 in this study during the long-term follow-up extension period of this study. As deemed by the investigator, the two participants passed away from disease-related complications unrelated to this study drug. We will of course continue to assess the data in this extension study. This is a further reminder of the cruel nature of this disease and the urgency for therapies that may extend and enhance the lives of these children. And finally, under the current plan, which includes the intended Caritas transaction, the amicus cash position is sufficient to achieve self-sustainability and profitability in 2023. During the quarter, the balance sheet of amicus was strengthened through a $200 million private investment from a syndicate of leading biotechnology investors. 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 new medicines for people living with rare diseases. 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 in the quarter. Bradley.