Zevra Therapeutics(ZVRA) - 2024 Q4 - Earnings Call Transcript

Financial Data and Key Metrics Changes - In Q4 2024, the company reported net revenue of $12 million, including$ 10.1 million from MIPLYFFA, marking a record level of quarterly product revenue [41][42] - The net loss for Q4 2024 was $35.7 million, compared to a net loss of$ 19.6 million in the same quarter a year ago [44] - For the full year 2024, net revenue was $23.6 million, with a net loss of$ 105.5 million, compared to a net loss of $46 million in 2023 [46][48] Business Line Data and Key Metrics Changes - MIPLYFFA's early launch exceeded expectations, with 109 patient enrollment forms received in Q4, reflecting strong demand in the NPC community [10][23] - OLPRUVA saw an increase in enrollments to four in Q4, compared to three in Q3, with market access remaining consistent at 76% of covered lives [35][36] Market Data and Key Metrics Changes - The company estimates approximately 900 people with NPC in the U.S., with 300 to 350 diagnosed, indicating a significant market opportunity [24][102] - The European market is expected to have about 1,100 patients living with NPC, with plans to submit a marketing authorization application in the second half of 2025 [11][53] Company Strategy and Development Direction - The company is focused on expanding MIPLYFFA's availability outside the U.S. and enhancing commercial capabilities for both MIPLYFFA and OLPRUVA [11][12] - A strategic review led to the decision to outsource in-house discovery efforts, allowing the company to focus investments where there is the greatest potential for impact [17] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the momentum from 2024 and the strength of the company's position to execute on strategic priorities in 2025 [20][48] - The management team emphasized the importance of building financial strength and disciplined capital allocation to support long-term transformation [19][47] Other Important Information - The company entered into an agreement to monetize its priority review voucher for$ 150 million, which will provide non-dilutive capital for future growth [20][46] - The company has a cash runway extending into 2029, excluding potential proceeds from the sale of the PRV [48] Q&A Session Summary Question: Can you provide guidance on the European filing timeline? - Management indicated plans to file for MIPLYFFA in Europe in the second half of 2025, with confidence in the acceptance of the filing [53][56] Question: How do you expect the European market dynamics to affect patient identification? - The management noted that the European market is more mature due to the long-standing presence of miglustat, which could facilitate patient identification and treatment [53][54] Question: Can you provide more details on MIPLYFFA sales and patient enrollment? - The company reported $10.1 million in MIPLYFFA revenue for Q4, with 109 total prescription enrollment forms received [41][23] Question: What is the average time from enrollment to reimbursement? - Management stated it is too early to provide specifics on the average time from enrollment to reimbursement but noted that many patients received their first fill and refill quickly [104] Question: How are reimbursement conversations progressing? - The management reported that most patients are getting covered through direct formulary or medical exceptions, with ongoing discussions with payers about the clinical benefits of MIPLYFFA [91][93] Question: What are the expected discontinuation rates for MIPLYFFA? - Management indicated it is too early to assess discontinuation rates but noted high continuity of care observed in previous programs [113][115]