Zevra Therapeutics (ZVRA) Q2 2025 Earnings Call August 12, 2025 04:30 PM ET Speaker0Good afternoon and thank you for joining Zebra's Second Quarter twenty twenty five Financial Results and Corporate Update Conference Call. Today's call is being recorded and will be available via the Investor Relations section of the company's website later today. The host for today's call is Nicole Osher, Zebra's Vice President of Investor Relations and Corporate Communications. Please go ahead.Speaker1Thank you, and welcom ...

Financial Data and Key Metrics Changes - The company reported a strong cash position with $217 million in the bank, allowing it to operate independently of capital markets [47] - The enrollment forms for MyPlifa have reached 122 since its launch, indicating a successful initial uptake [8][12] Business Line Data and Key Metrics Changes - MyPlifa, the recently launched product for Niemann Pick disease type C, is in its launch phase and has shown promising enrollment numbers in the U.S. market [5][8] - Olpruva, the other commercial product, is targeting specific urea cycle disorders and has seen five enrollments in line with its revised strategy [42] Market Data and Key Metrics Changes - In the U.S., there are approximately 900 patients diagnosed with Niemann Pick disease type C, with 300 to 350 diagnosed patients currently identified [7][8] - In Europe, the market is more mature with about 1,100 patients, but many are already on treatment, presenting a challenge for new product uptake [9] Company Strategy and Development Direction - The company is focused on executing the launch of MyPlifa and expanding its European filing for the product in the second half of the year [37][40] - There is an emphasis on increasing disease state awareness and product awareness to drive patient diagnosis and treatment [18][36] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the future, citing the successful launch of MyPlifa and the potential for increased patient identification through awareness efforts [18][36] - The company is committed to supporting patients through the reimbursement landscape and ensuring access to therapies [27][32] Other Important Information - The company is exploring various avenues for its go-to-market strategy in Europe, considering both direct and partnership approaches [40] - The enrollment in the saliprolol program for vascular Ehlers Danlos syndrome has reached 32 patients, with ongoing efforts to increase participation [44] Q&A Session Summary Question: Can you provide an overview of Zebra and its products? - The company is a commercial stage rare disease company with two products in the U.S. market and a pipeline in various stages of development [4] Question: How is the launch of MyPlifa progressing? - The launch is exceeding expectations, with significant patient enrollment already achieved [8][12] Question: What are the challenges in diagnosing Niemann Pick disease type C? - The heterogeneous nature of the disease complicates diagnosis, leading to many undiagnosed patients [14][15] Question: How does the company plan to penetrate the market further? - The company is leveraging successful strategies from Europe to increase awareness and diagnosis in the U.S. [18][33] Question: What is the current status of reimbursement levels? - The company is pleased with the current reimbursement efforts and the willingness of physicians and patients to navigate the process [26][28] Question: How does the company support patients transitioning from EAP to commercial therapy? - The company has a robust support system in place to assist patients through the transition [29][32] Question: What is the strategy for the European market? - The company is focused on the MAA filing and is exploring various market entry strategies [37][40]

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]

Financial Data and Key Metrics Changes - In Q1 2025, the company reported net revenue of $20.4 million, 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][30] - Operating expenses for Q1 were $22.8 million, a decrease of $0.6 million compared to the same quarter last year [30] - The net loss for Q1 2025 was $3.1 million, compared to a net loss of $16.6 million in the same quarter a year ago [30][31] - Total cash, cash equivalents, and investments as of March 31, 2025, were $68.7 million, a decrease of $6.8 million from December 31, 2024 [30][31] 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 [20][21] - Olpruva received a total of 28 prescription enrollment forms since its initial availability, with five forms received in Q1 2025 [12][27] - The company has achieved 38% of covered lives for MyPlifa, which is in line with expectations at this stage of the launch [21][22] Market Data and Key Metrics Changes - The company is focusing on expanding MyPlifa's availability outside the U.S., with plans for a marketing authorization application in Europe in the second half of the year [11] - The European market for NPC is well established, with approximately 1,100 people estimated to be living with the disease [11] Company Strategy and Development Direction - The company is executing on four strategic pillars: commercial excellence, pipeline and innovation, talent and culture, and corporate foundation [5][8] - The focus is on bringing innovative therapies to patients with rare diseases, with a strong emphasis on MyPlifa as a cornerstone treatment for NPC [17][33] - The company is also assessing strategic alternatives for its Phase III ready asset KP1077 for rare sleep disorders [6] 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 believes it is well positioned to support its strategic priorities and execute on its long-term vision, independent from capital markets [31][32] Other Important Information - The company has successfully monetized its Pediatric Rare Disease Priority Review Voucher, adding $148.3 million of non-dilutive capital to its balance sheet [8][31] - The company is committed to patient access and comprehensive patient services through its in-house program, Amplify Assist [27] Q&A Session Summary Question: Details on the number of patients on MyPlifa and reimbursement status - The company received 122 enrollments since launch, with the majority of patients actively on the drug, while some are in the benefits investigation process [36] Question: Reasons for reimbursement denials for MyPlifa - Initial denials are common for rare disease products, primarily due to prior authorization requirements, but the company has been successful in securing coverage through medical exception processes [39][40] Question: Visibility on patient cohorts and enrollment cadence - The company estimates around 600 undiagnosed patients and 300 diagnosed patients, with ongoing efforts to raise awareness and facilitate treatment [47][59] Question: Inventory levels at the end of the quarter - The company is managing inventory closely and maintaining target levels as it supports patient needs during the early launch phase [50] Question: Qualitative metrics on refill rates and patient retention - The majority of patients receiving active drug have continued to refill, reflecting the clinical benefit and durability of MyPlifa [54] Question: Coverage details and impact on patient access - 38% of covered lives have access to MyPlifa, with ongoing efforts to increase this percentage as more plans evaluate the product [63][64]