Zevra Therapeutics(ZVRA) - 2025 Q3 - Earnings Call Transcript

Financial Data and Key Metrics Changes - In Q3 2025, the company reported net revenue of $26.1 million, with $22.4 million attributed to Miplyf, $2.4 million from the French Expanded Access Program, $1.2 million from royalties, and $100,000 from Olpruva [6][18][20] - Operating expenses decreased by 6.8% year-over-year to $20.4 million, while R&D expenses decreased by 7.5% to $3.4 million [19] - The net loss for Q3 2025 was $500,000, significantly improved from a net loss of $33.2 million in Q3 2024 [20] Business Line Data and Key Metrics Changes - Miplyf's performance remains strong, with 137 total prescription enrollment forms received since its launch, indicating approximately 40% of the diagnosed patient population [6][29] - The company has seen a total of eight new prescription enrollment forms in Q3 2025, reflecting ongoing efforts in disease awareness and patient identification [12][29] - Apruva's market penetration remains limited, leading to a decision to scale back sales and marketing efforts for this product [9] Market Data and Key Metrics Changes - The company is preparing to expand Miplyf into the European market, where an estimated 1,100 individuals are living with Niemann-Pick Disease Type C [6][7] - The Marketing Authorization Application for Miplyf in Europe has been validated and is under review, with confidence in the submission based on an expanded data set [7][8] - Coverage for Miplyf has increased to 66%, up from 52%, indicating improved access for patients [16][45] Company Strategy and Development Direction - The company aims to build a leading rare disease company, focusing on Miplyf as a foundational treatment for Niemann-Pick Disease Type C [4][11] - Strategic priorities include expanding into Europe and maintaining a disciplined approach to capital allocation while investing in high-impact opportunities [22][23] - The company is actively recruiting patients for the Discover Trial, evaluating salicylol for vascular Ehlers-Danlos syndrome [9][10] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the continued success of Miplyf and the potential for geographic expansion and patent term extension [11][23] - The company is focused on maintaining cost efficiency and operational accountability to support long-term value creation [22] - Management highlighted the importance of ongoing disease awareness initiatives to identify undiagnosed patients and improve treatment access [14][15] Other Important Information - The company has established a solid patent position for Miplyf and requested a patent term extension with the U.S. Patent and Trademark Office [8] - The Expanded Access Program in Europe is critical for building relationships with physicians and clinics, with 92 patients enrolled by the end of Q3 [8][9] Q&A Session Summary Question: Have any of the new start forms been from newly diagnosed patients? - Management confirmed that many new enrollment forms are from newly diagnosed patients, largely due to disease awareness campaigns and partnerships with genetic testing collaborators [25][27] Question: How is the company thinking about its robust balance sheet? - Management indicated that they are focused on executing the Miplyf launch and exploring opportunities for expansion while maintaining a disciplined approach to capital allocation [30][32] Question: What percentage of patients are getting refills? - Management reported that the majority of patients are receiving refills, with 66% of covered lives today, and emphasized the effectiveness of their reimbursement processes [39] Question: What are the key drivers for the increase in coverage? - The increase in coverage is attributed to the strength of Miplyf's clinical data, which has resonated with payers, and the targeting of Centers of Excellence and community-based physicians [43][45] Question: What are the expectations for the European market? - Management is confident in the MAA submission for Europe and anticipates a favorable reception based on the comprehensive data package [48][49] Question: What is the status of the KP1077 program? - Management remains open to strategic alternatives for KP1077 and is currently in discussions, but no specific updates were provided [58]