Amicus Therapeutics, Inc.

Good morning, ladies and gentlemen, and welcome to the Amicus Therapeutics 4-Year 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 then 1 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 Farnan, Executive Director of Investor Relations. You may begin.

Thank you. Good morning, everyone. Thank you for joining our conference call to discuss Amicus Therapeutics' full year 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. 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. 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 factors section of our annual report on Form 10-K for the full year ended December 31st, 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, and welcome, everyone, to our full year 2021 results conference call. I am pleased to highlight the progress that we've made at Amicus throughout 2021 and now into the start of 2022. a reflection of the broad execution across our business. As we did in this morning's press release, I'd like to highlight several key points. First, Gallifold continues its strong performance and is the cornerstone of our success. For the full year 2021, Gallifold revenue is $306 million and in line with our full year revenue guidance. We are very pleased with the execution of Gallifold globally which was largely driven by new patient accruals from both the switch and naive populations, as well as sustained patient compliance and adherence rates. We expect significant growth for Gallifold in 2022, and we believe that Gallifold has the potential to be a $1 billion medicine at peak revenue. Second, we continue to make great progress on our global regulatory filings for ATGAA, our novel next-generation therapy for Pompe disease. The U.S. and EU regulatory reviews are progressing very well, and we are extremely pleased with the level of engagement from the regulatory agencies. We recently completed the mid-cycle review with the FDA, and we remain confident in ATGAA's approvability and its potential to become the next standard of care in Pompe disease. As a reminder, 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 VLA of the biologic. In the EU, the marketing authorization applications for ATGAA were submitted to the European Medicines Agency in the fourth quarter of 2021, and we are actively engaged in the EMA reviews, and we expect the CHMP opinion later this year. Our global launch plans continue to accelerate for APGAA, including targeted investments in additional personnel to support the launch and significant investments in launch inventory preparations. We are now closer to having another potential treatment for people living with Pompe disease, both in the United States and in Europe, with further regulatory applications planned in the months ahead. And third, and importantly, Amicus has maintained our strong financial position and remains committed to achieving profitability in 2023 as we continue to execute on the global expansion of Gallifold and prepare for the global launch of ATGAA. We have no need for any equity or equity-linked financings to achieve profitability. Turning to slide five, as announced today and highlighted on this slide, we have made the strategic decision not to spin off our gene therapy programs and technologies. The decision here results from the current unfavorable market conditions affecting the biotech sector broadly, as well as an increasingly challenging environment for standalone gene therapy companies. Again, Amicus remains strongly committed to achieving profitability in 2023 without the need for any further dilutive equity or equity-related financings. In order to accomplish this, we will concentrate the vast majority of our efforts and investments in our priority growth franchises in Febre disease and Pompe disease. We will do this through, one, the continued global growth and commercialization of Gallifold, two, by advancing the activities to secure global regulatory approvals and commercial launch for ATGAA, and thirdly, by making judicious strategic investments in next-generation therapies in Febre and Pompei, and in core science and platform technologies in the field of genetic medicine. This strategic prioritization of our pipeline, as well as the reorganization of our internal R&D team, is expected to result in approximately $400 million in net cost savings through 2026. This is a similar amount in savings associated with the previously announced spinoff. This decision to streamline and prioritize our gene therapy pipeline portfolio and overall R&D operations is designed to create a more efficient organization that is highly focused on delivering for patients and for shareholders. We are able to achieve this $400 million in cost savings ahead by taking three principal actions with respect to our R&D. First, we are prioritizing our pipeline portfolio including the decision to focus on core platform and enabling technologies. As such, we will not be moving multiple gene therapy programs into the clinic in the years ahead. This prioritization will significantly reduce R&D-associated expenses in clinical operations, manufacturing, and supporting activities. Second, we are maintaining overall amicus employee count flat at about 500 employees for the next several years. To achieve this, we today are reducing our overall workforce by 7%, about 35 individuals, mostly all in our R&D organization. This will allow us to continue to hire key personnel for the global growth of Gallifold and the coming launches of ATGAA, again, while keeping overall headcount at Amicus flat at about 500 employees globally. And third, we will suspend indefinitely our plans to build an internal gene therapy manufacturing organization and facility. These portfolio and organizational changes will strengthen amicus by sharpening our focus, investing appropriately and very judiciously in our pipeline, and ensuring our continued financial strength. We at amicus are rapidly approaching two pivotal inflection points. First, the global launch of a second rare disease medicine in ATGAA. And secondly, profitability. A second product in profitability. These are rare feats that we believe will enable amicus to truly enter the upper echelon of biotechnology. Turning now to slide six, key strategic priorities for 2022. Here, as we laid out last month, we are focusing on achieving our key strategic priorities for this year. These include One, we will continue to drive Gallifold to more people living with febrile disease with amenable variants in existing and in new markets. We look to achieve double-digit global product revenue growth of 15 to 20 percent with revenue of $350 to $365 million for the year. This growth reflects the strong momentum and demand behind this precision medicine globally. Second, we remain steadfast in our commitment to advancing ATGAA regulatory filing and initiating the anticipated launches of ATGAA in the United States. Leveraging our seasoned global commercial team and experience across all areas needed for an effective drug launch, we are fully prepared and anticipate another successful launch with ATGAA. Third, we are strategically advancing our best-in-class next generation genetic medicines and capabilities. Four, again, we will continue to maintain a strong financial position as we carefully manage our expenses and investments, and we remain fully funded through all major milestones. Before I hand the call over, I'd like to provide an update to our previously announced leadership transition. Again, as we previously announced in September 2021, Brad Campbell will be succeeding me as Chief Executive Officer. With that transition now taking place effective August 1, 2022, I will also now become at that time the full-time executive chairman of amicus for a two-year term and expect to continue as the non-executive chairman of the board at amicus thereafter. This transition will enable Bradley and I to continue our tremendous long-term partnership in very clearly defined roles ahead. As I've highlighted on past calls, there is no one more capable to succeed in this role And both Bradley and I remain steadfast in our drive to achieve our strategic goals and priorities laid out here today and our mutual vision for Amicus for years ahead. So with that, Bradley, I'll turn the call over to you.