Zevra Therapeutics Conference Call Summary Company Overview - **Company**: Zevra Therapeutics (NasdaqGS: ZVRA) - **Focus**: Commercial-stage organization specializing in rare diseases, particularly Niemann-Pick disease type C (NPC) and vascular Ehlers-Danlos syndrome Key Points MIPLYFFA Performance - **Launch Success**: MIPLYFFA, the only disease-modifying therapy for NPC, launched successfully with a robust commercial performance [5][12] - **Revenue Growth**: Total revenue reached $106.5 million, marking a 30% quarter-over-quarter growth [12] - **Patient Enrollment**: 24 enrollment forms were submitted in Q4, indicating strong demand [12] Patient Demographics and Diagnosis - **Prevalence and Diagnosis**: Approximately 900 patients in the U.S. are estimated to have NPC, with 300-350 diagnosed [10][18] - **New Diagnoses**: Efforts in genetic testing and disease awareness are leading to an increase in newly diagnosed patients [13][18] - **Patient Journey**: The organization is focused on understanding the unique journey of each patient to improve diagnosis rates [20][23] Clinical Profile and Treatment Impact - **Disease Modification**: MIPLYFFA shows the ability to halt disease progression within 12 weeks, with long-term data supporting efficacy over 4-6 years [11][12] - **Safety Profile**: The therapy has an acceptable safety profile, which is crucial for patient acceptance [11] Geographic Expansion - **International Opportunities**: Zevra is exploring geographic expansion beyond the U.S. and Europe, with plans to increase access to MIPLYFFA [6][39] - **European Market**: The company is seeing a pull for the product in Europe, with 92 patients added in Q3, representing about 10% of the diagnosed population [39] Celiprolol Development - **Ongoing Trials**: Celiprolol is currently enrolling patients for vascular Ehlers-Danlos syndrome, with a focus on accelerating clinical development [6][52] - **Market Potential**: The U.S. prevalence for this condition is approximately 7,500 patients, indicating a significant market opportunity [58] Financial Strategy - **Capital Allocation**: The company emphasizes disciplined capital allocation, allowing for independent operation without reliance on capital markets [54][57] - **Investment Focus**: Investments are being made in MIPLYFFA, Celiprolol, and European market expansion [57] Awareness and Education Efforts - **Healthcare Provider Engagement**: Zevra is actively educating healthcare providers about NPC and MIPLYFFA to improve diagnosis and treatment rates [15][30] - **Genetic Testing Initiatives**: Collaborations with organizations like GeneDx are aimed at facilitating earlier diagnosis through genetic testing [15][16] Future Outlook - **Long-term Vision**: The company aims to continue focusing on MIPLYFFA's growth while developing Celiprolol and expanding its market presence [52][57] - **Market Strategy**: A flexible go-to-market strategy is being developed for Europe and other international markets, considering local dynamics and patient needs [40][43] Additional Insights - **Patient Retention**: High persistency rates are observed, with most patients remaining on therapy unless they progress to a point where therapy is no longer needed [26][29] - **Market Share**: Zevra has captured approximately 40%-50% market share in its first full year of MIPLYFFA's launch [24] This summary encapsulates the key insights from the conference call, highlighting Zevra Therapeutics' strategic focus, market performance, and future growth opportunities in the rare disease sector.

Financial Performance - In Q4 2025, total net revenue was $34.1 million, including $26.4 million from MIPLYFFA sales, $400,000 from OLPRUVA, $5.6 million from global EAP reimbursements, and $1.8 million from royalties under the AZSTARYS license [22][23] - For the full year 2025, net revenue reached $106.5 million, with $87.4 million from MIPLYFFA sales, $800,000 from OLPRUVA, $13 million from global EAP reimbursements, and $5 million from royalties [24] - Operating expenses for Q4 were $23 million, a decrease of $1.5 million year-over-year, while full-year operating expenses were $90.4 million, down $6.6 million from 2024 [22][24] - Net income for Q4 was $12.2 million, compared to a net loss of $35.7 million in Q4 2024, and full-year net income was $83.2 million, compared to a net loss of $105.5 million in 2024 [23][24] Business Line Performance - MIPLYFFA generated $87.4 million in revenue for the full year 2025, with strong commercial performance reflected in 52 prescription enrollment forms received in the first year of commercialization [5][6] - In Q4, 24 new prescription enrollment forms were received, contributing to a total of 161 enrollments since launch [6][14] - The company is expanding access to MIPLYFFA through a global expanded access program, with 113 patients enrolled by the end of 2025 [8] Market Data - The estimated U.S. prevalence of Niemann-Pick disease type C (NPC) is approximately 900 individuals, with 300-350 currently diagnosed [7] - In Europe, NPC prevalence is estimated at around 1,100 individuals, with the current EAP supporting nearly 10% of this population [9] - Diagnosis rates in Europe are higher due to the approval of miglustat, leading to stronger physician awareness and higher patient identification [10] Company Strategy and Industry Competition - The company aims to redefine therapies for rare diseases, focusing on patient-centricity, integrity, accountability, innovation, and courage [5] - MIPLYFFA has exclusivity in the U.S. through 2031 under the Orphan Drug Designation, and the company is pursuing a patent term extension [7] - The company is also advancing celiprolol for the treatment of vascular Ehlers-Danlos syndrome, with ongoing Phase III studies [10][11] Management Commentary on Operating Environment and Future Outlook - Management expressed confidence in the growth potential of MIPLYFFA, driven by both previously diagnosed and newly diagnosed patients [15][16] - The company is focused on expanding access and strengthening relationships with key opinion leaders to maximize commercial impact [10] - Management highlighted the importance of ongoing education and awareness campaigns to facilitate early diagnosis and treatment [16][18] Other Important Information - The company moved its headquarters to Boston to enhance collaboration and access to specialized talent [12][13] - The new CFO, Justin Renz, officially started on the call date and will support the company's long-term value creation [4][28] Q&A Session Summary Question: Can you provide details on the split of new diagnoses versus previously identified patients? - Management noted that there has been meaningful growth in newly diagnosed patients, attributed to clinical data strength and disease awareness campaigns [33][34] Question: What is the expected timeline for diagnosis confirmation? - Management indicated that the timeline for diagnosis can vary significantly due to the nature of rare diseases, but efforts are being made to streamline the process [37][40] Question: Can you discuss persistency rates for patients on therapy? - Management stated that it is too early to provide meaningful data on persistency but expressed encouragement from refill rates observed [45][46] Question: How is the initial experience with the Uniphar collaboration? - Management expressed satisfaction with the collaboration, noting that it has facilitated rapid product delivery to patients [47][48] Question: What is the expected growth of EAP revenue? - Management clarified that EAP revenue is based on individual patient shipments and will have variability quarter-over-quarter [54][55] Question: How is the gross to net evolving in the U.S.? - Management noted that gross to net has evolved and is dynamic, making it difficult to provide specific guidance [58] Question: What is the average age of patients on MIPLYFFA? - Management indicated that approximately half of MIPLYFFA patients are adults, reflecting the patient mix seen in the expanded access program [64][66] Question: What are the next steps for the EMA application? - Management confirmed readiness to respond to the EMA's questions and emphasized the robustness of the data package submitted [69]

Core Insights - XOMA Royalty Corporation has made significant acquisitions, including mezagitamab royalty rights from BioInvent International and partnered assets through the acquisition of LAVA Therapeutics, enhancing its portfolio of early- and late-stage assets [1][2][5]. Business Development - XOMA Royalty acquired future royalty and milestone rights for mezagitamab from BioInvent for $20 million, with an additional $10 million contingent on regulatory milestones, potentially earning up to $16.25 million in milestones from Takeda and mid-single digit royalties on future sales [5]. - The acquisition of LAVA Therapeutics will secure economic interests in two partnered assets, PF 08046052 (developed by Pfizer) and JNJ-89853413 (developed by Johnson & Johnson) [5]. Company Acquisitions - XOMA Royalty has entered into definitive agreements to acquire Turnstone Biologics for $0.34 per share plus a contingent value right (CVR) [7]. - The acquisition of HilleVax is set at $1.95 per share, with additional CVR payments based on future cash distributions and potential sales of norovirus programs [7][8]. - The acquisition of LAVA Therapeutics is expected to close in Q4 2025, with a cash payment between $1.16 and $1.24 per share plus a CVR for future proceeds [8]. Key Pipeline Advancements - Rezolute completed enrollment in the Phase 3 sunRIZE study for ersodetug, receiving a $5 million milestone payment [3][9]. - The Marketing Authorization Application (MAA) for tovorafenib by Day One Biopharmaceuticals was accepted for review by the EMA, resulting in a $4 million milestone payment to XOMA Royalty [3][9]. - Zevra Therapeutics submitted an MAA to the EMA for arimoclomol as a treatment for Niemann-Pick Type C [3]. Financial Performance - In the first half of 2025, XOMA Royalty received $29.6 million in royalties and milestones, with $11.7 million in the second quarter [4][12]. - Income for the second quarter of 2025 was reported at $13.1 million, an increase from $11.1 million in the same period of 2024 [12]. - General and administrative expenses decreased to $7.8 million in Q2 2025 from $11.0 million in Q2 2024, primarily due to prior exit packages [14]. Cash Position - As of June 30, 2025, XOMA Royalty had cash and cash equivalents of $78.5 million, down from $106.4 million at the end of 2024 [20][32]. - The company repurchased approximately 81,700 shares for $1.8 million in Q2 2025, totaling over 107,500 shares repurchased in 2025 [21].