Amicus Therapeutics, Inc.

Good morning, ladies and gentlemen, and welcome to the Amicus Therapeutics Third Quarter 2022 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. As a reminder, this conference call is being recorded. I would now like to turn the conference over to your host, Mr. Andrew Fonin, Executive Director of Investor Relations. You may begin.

Thank you, Shannon. Good morning. Thank you for joining our conference call to discuss Amicus Therapeutics third quarter 2022 financial results and corporate highlights. Leading today's call, we have Bradley Campbell, President and Chief Executive Officer, Stephanie Creamy, Chief Financial Officer, Sebastian Martel, Chief Business Officer, and Dr. Jessica Stelly, Chief Development Officer. Joining for Q&A is Dr. Mitchell Goldman, Chief Medical Officer, and Ellen Rosenberg, Chief Legal Officer. As referenced on slide 2, 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. We are cautioned not to place undue reliance on any forward-looking statements which speak only to the dates 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 quarterly report on Form 10-Q for the quarter-ended September 30, 2022, filed this morning with the Securities and Exchange Commission. At this time, it's my pleasure to turn the call over to Bradley Campbell, President and Chief Executive Officer. Bradley.

Great. Thank you, Andrew, and welcome, everybody, to our third quarter 2022 results conference call. I'm really pleased today to review the continued progress made across our business this year. And as we did in this morning's press release, let me highlight several key accomplishments for the quarter. First and foremost, Gallifold continues its strong performance and remains the cornerstone of our success. We continue to be very pleased with the uptake of Gallifold globally, despite significant foreign currency headwinds we've seen this year. And the first three quarters of the year represented 8% reported revenue growth or 16% on an operational basis. In the third quarter, our growth and key performance indicators are meeting our objectives in all geographies. And as Sebastian will highlight, we continue to see strong trends in a number of our metrics, including new patient starts and in-person visits between our field team and our physicians. So we were reiterating our guidance that 15 to 20% growth in Gallifold revenues and constant exchange rates. We're also pleased to announce that the Gallifold intellectual property estate continues to grow this quarter. This year, we've strengthened our IP estate through the issuance of 19 new patents. There are now 46 orange book listed issued patents related to Gallifold in the United States, 30 of which provide protection to 2038 and beyond. and importantly includes five composition of matter patents. We see this patent portfolio, along with our orphan drug exclusivities, providing broad and long-term intellectual property rights well into the late 2030s with its novel precision medicine. As we've discussed throughout the year and as anticipated, we saw three abbreviated new drug application or ANDA filers and have received their paragraph four certifications. Given our strong and innovative intellectual property estate, we believe we are well positioned to address any potential generic challenges to our patents. And we announced this morning, later today, we intend to file infringement lawsuits against three antifilers who are requesting approval to market a generic version of Gallifold. We intend to continue to protect and enforce our broad intellectual property rights. And looking ahead, we expect continued growth for Gallifold this year and remain confident that with our strong IP protection, it has a long runway well into the next decade. Second, we continue to make progress on our global regulatory filings and commercial planning for ATGAA, our novel next-generation therapy for Pompe disease. As we just announced last week, the U.S. Food and Drug Administration recently deferred action on the biologics licensing application, or BLA, for sipaglucosidase alpha, the biological component of ATGA, which we also call ATB200. The agency cited that due to restrictions on travel related to COVID-19, it was unable to conduct the required inspection of the Wuxi Biologics manufacturing site in China during the review cycle and is deferring action in the application until the inspection is complete. That being said, the good news is that per FDA guidance, a deferral in such circumstances can only be given, quote, provided that no deficiencies have been identified and the application otherwise satisfies the requirements for approval, end quote. So as we've been saying, we believe this is a matter of when, not if, APGA is approved. Additionally, the company is now actively engaged in the FDA on this step, and at the agency's direction, the company has requested a Type A meeting with the Office of Pharmaceutical Quality to discuss and agree upon plans and logistics for the pre-approval inspections. We at Amicus, along with our manufacturing partner, Wuxi Biologics, are ready for the inspection of the manufacturing facility in China. And as soon as we have more clarity, we will provide an update on when we might expect an approval in the United States. As a reminder, we continue to expect the two components of APGA will be approved together. Importantly, in Europe, we are very far along now in the review by the EMA on the marketing authorization application for APGA. To provide a little more color there, We are announcing today that we are included now in the agenda for an oral explanation in November. And thus, the CHMP opinion is expected at the December meeting, setting us up for a commercial launch in 2023. As a reminder, the EMA has indicated that it does not require an inspection of the WUSHI manufacturing site as a condition of their approval. We're also extremely pleased with the level of interest and participation we're seeing in our expanded access programs globally. We now have programs in place in the United States, United Kingdom, France, Germany, and Japan, with a growing number of patients participating in each. In fact, across all of our ongoing clinical studies and access programs, there are now nearly 190 patients on ATGA today, which we believe represents more than 5% of the total treated Pompe patients around the world. We expect this number to continue to grow as we approach approvals in the United States and Europe. In anticipation of these approvals in the near term, our global launch plans continue to move ahead, including our pre-launch activities, targeted investments, and a handful of additional personnel to support the launch and ongoing investments in building launch inventory. We are close now to reaching this much anticipated milestone of ATGA getting across the regulatory finish line and providing another treatment option for people living with Pompe disease, both in the United States and in Europe, and with further regulatory applications planned in the months ahead. And third, Amicus has maintained a strong financial position as we continue to execute on the global expansion of Gallifold and prepare for the global launch of ATGA. Despite all the headwinds with FX and the delays with ATGA approval in the United States, we will continue on our path to profitability. Our goal is to achieve non-GAAP profitability in the second half of 2023. This is dependent on a number of factors, including the timing of approvals and the launch of ATGA, But based on the current operating plans and projections, we believe this will happen in the second half next year. Additionally, today we entered into an at-the-market or ATM equity offering of up to $250 million. We see this as good financial housekeeping measure, and we have no present intention to use the ATM this year. In 2023 and beyond, we will be judicious about any use of the ATM, and any proceeds will go towards ensuring continued commercial expansion of Gallifold and maximizing anticipated launches of APGA. I'll emphasize that we're sharply focused on our three primary objectives. Number one, continuing to advance Gallifold to as many patients in as many geographies as possible. Number two, securing approvals for and launch of APGA globally. And number three, ensuring the financial strength of Amicus. On slide five, we see that we're well on our way towards achieving our key strategic priorities for this year. including number one, continuing to drive Gallifold to more people living with febrile disease with amenable variants and existing in new markets. As I mentioned, we're on track to achieve our guidance of double digit global product revenue growth of 15 to 20% at constant exchange rates. This reflects the strong momentum and demand behind this precision medicine globally. We remain steadfast in our commitment to advancing APGA regulatory approvals and the anticipated launch of APGA, leveraging our seasoned global commercial, medical, and market access teams and our experience across all areas needed for a successful and effective drug launch, we are fully prepared for and anticipate a successful launch at ATGA. We continue to judiciously invest in the advancement of our best-in-class next-generation genetic medicines and capabilities, as well as our next-generation chaperone for Fabry disease. And again, we'll continue to maintain a strong financial position as we carefully manage our expenses and our investments in the business. With that, let me now hand the call over to Sebastian Martel, our Chief Business Officer, who'll give further highlights on the Gallifold performance for the quarter.