Financial Data and Key Metrics Changes - The company reported net revenue of $20.4 million for Q1 2025, with $17.1 million from MyPlifa, $0.1 million from Olpruva, and $2.3 million in net reimbursements from the French EAP for Aramcholamol [29] - Operating expenses decreased to $22.8 million, down $0.6 million compared to the same quarter last year [30] - The net loss for Q1 2025 was $3.1 million, or $0.06 per share, compared to a net loss of $16.6 million, or $0.40 per share, for the same quarter a year ago [30] Business Line Data and Key Metrics Changes - MyPlifa's launch has exceeded expectations, with 122 prescription enrollment forms received since launch, indicating that approximately one-third of diagnosed NPC patients in the U.S. have been enrolled [19][20] - Olpruva received a total of 28 prescription enrollment forms since its initial availability, with five forms in Q1 2025 [12][26] Market Data and Key Metrics Changes - The company achieved 38% of covered lives for MyPlifa by the end of Q1 2025, which aligns with expectations for this stage of the launch [20][64] - The European market for MyPlifa is being targeted, with an estimated 1,100 people living with NPC, and regulatory submissions are planned for the second half of the year [11] Company Strategy and Development Direction - The company is focused on commercial excellence, pipeline innovation, talent and culture, and corporate foundation as its four strategic pillars [5][6] - The recent monetization of the Pediatric Rare Disease Priority Review Voucher added $148.3 million of non-dilutive capital to the balance sheet, enhancing financial strength [8][30] - The company aims to establish MyPlifa as a cornerstone treatment for NPC globally and maximize commercial opportunities with Olpruva [16][32] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the early success of MyPlifa and the refined marketing strategy for Olpruva, indicating a strong position for future growth [33] - The company is well-positioned to support strategic priorities independent from capital markets, with ample resources for execution [32] Other Important Information - The company has made a strategic decision to withdraw the NDA for Apodas, reducing regulatory costs [16] - The ongoing Phase III trial for saliprolol is being actively managed to drive enrollment, with 32 patients currently enrolled [14][72] Q&A Session Summary Question: Details on the number of patients on MyPlifa and reimbursement status - Management indicated that the majority of the 122 enrollments are on the drug, with some undergoing benefits investigation [36] Question: Top reasons for reimbursement denials for MyPlifa - Initial denials are common, primarily due to prior authorization requirements, but the company has successfully secured coverage for most patients [39] Question: Visibility on patient sources and enrollment cadence - Approximately 600 undiagnosed patients exist, and efforts are ongoing to raise awareness and enroll both diagnosed and undiagnosed patients [48] Question: Inventory levels at the end of the quarter - The company is managing inventory closely and maintaining target levels as they support patient needs [51] Question: Qualitative metrics on refill rates and patient retention - Most patients receiving active drug have continued with refills, reflecting the clinical benefit of MyPlifa [55] Question: Coverage details and impact on patient access - 38% of covered lives means that many plans have reviewed MyPlifa, with successful reimbursement through medical exception pathways [64][66]