Amicus Therapeutics(US:FOLD)2024-05-09 19:14Financial Data and Key Metrics Changes - Total revenue for Q1 2024 reached $110 million, representing a 28% increase year-over-year [17][72] - Galafold revenue was $99 million globally, reflecting a 16% growth year-over-year on a constant currency basis [57][58] - Pombiliti and Opfolda reported revenue of $11 million for Q1 2024, a 30% increase compared to Q4 2023 [22] Business Line Data and Key Metrics Changes - Galafold continues to capture over 60% of the global amenable market, with potential to reach 85%-90% in mature markets [18][78] - The number of patients on Pombiliti and Opfolda treatment reached 155, with a significant increase in new commercial patients [5][75] - Non-GAAP operating expenses increased to $86 million in Q1 2024, compared to $81 million in the same period last year [27] Market Data and Key Metrics Changes - 66% of total revenue in Q1 2024 was generated outside the U.S., with 34% from within the U.S. [33] - The overall time from prescription to infusion has decreased to around 70 days, with improvements expected [8][134] - Pombiliti and Opfolda launched in Spain, with a fast reimbursement process enhancing access [25] Company Strategy and Development Direction - The company aims to maximize the number of patients on therapy by year-end 2024, with a focus on expanding patient access through reimbursement discussions [10][93] - The strategy includes sustaining double-digit growth for Galafold and executing multiple commercial launches for Pombiliti and Opfolda [96] - The company is committed to achieving non-GAAP profitability in 2024 while managing expenses carefully [38][62] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the successful launch of Pombiliti and Opfolda, anticipating it to become the standard-of-care for Pompe disease [11][49] - Positive trends in patient demand and physician feedback were highlighted, indicating strong momentum for the therapies [60][134] - The company expects to achieve full-year revenue growth of 25% to 30% in 2024, driven by Galafold and Pombiliti/Opfolda sales [37][38] Other Important Information - The company has submitted over 10 reimbursement dossiers and is in discussions for broader patient access across the EU [9][12] - Cash, cash equivalents, and marketable securities totaled $240 million as of March 31, 2024, down from $286 million at the end of December 2023 [36] Q&A Session Summary Question: What is the biggest disconnect between guidance for Pombiliti and Opfolda and Street models? - Management noted that understanding the timing for new patients through the insurance process was crucial, which led to a divergence in revenue expectations [44][46] Question: What are the expectations for patient switching from previous therapies? - Management indicated that trends show patients are switching from both Nexviazyme and Lumizyme, with a significant proportion of new patients coming from the naive population [23][126] Question: What are the goals for the Amicus Pompe registry? - The registry aims to collect real-world evidence and fulfill post-marketing commitments, with hundreds of patients expected to be enrolled [116][145] Question: How is the company addressing supply chain challenges? - Management expressed confidence in navigating supply challenges and highlighted progress in moving product out of China to central depots in Europe [85][106] Question: What proportion of revenue is expected from U.S. versus ex-U.S. markets? - The majority of revenue is expected to come from Europe initially, with a shift towards a more balanced distribution as more countries launch [109][110]