Summary of Zevra Therapeutics FY Conference Call Company Overview - **Company**: Zevra Therapeutics (NasdaqGS: ZVRA) - **Industry**: Rare Disease Therapeutics - **Lead Product**: MIPLYFFA for Niemann-Pick disease type C (NPC) - **Financial Position**: Net revenue of $72.3 million in the first nine months of 2025, with a cash position of $230.4 million at the end of Q3 2025 [2][5][30] Core Points and Arguments Company Mission and Vision - Zevra aims to redefine possibilities in rare disease therapies, focusing on patient-centricity and accountability to stakeholders [4][5] - The company is in growth mode with a late-stage pipeline and geographic expansion plans [5][10] Product and Market Performance - MIPLYFFA received FDA approval in September 2024 and has orphan drug exclusivity until 2031 [5][6] - The product has achieved approximately 40% penetration of the diagnosed patient population within the first 12 months post-launch, a notable achievement in the rare disease space [10][19] - The company is expanding access to MIPLYFFA in Europe, with an MAA under review by the EMA [5][11] Pipeline and Future Growth - Zevra has a diversified portfolio including OLPRUVA for urea cycle disorders and AZSTARYS, with various products under development [6][28] - Celiprolol is in a pivotal phase 3 trial for vascular Ehlers-Danlos syndrome (VEDS) [28] - The company is leveraging AI for patient identification and diagnosis, enhancing its market reach [22][39] Financial Discipline and Strategy - Strong balance sheet with $230.4 million in cash and $61 million in debt [30] - The company monetized a pediatric priority review voucher for $150 million, adding non-dilutive capital [13] - Focus on sustainable growth through disciplined investments and prioritization of high-value opportunities [9][30] Important but Overlooked Content - The company is actively working on expanding its geographic reach beyond the U.S. and Europe, with distribution agreements in place for markets outside these regions [25][50] - The potential for patent term extension could significantly impact the company's valuation, with a ruling expected in 2026 [52][53] - The total addressable market (TAM) for NPC is estimated between 300 to 900 patients in the U.S., with ongoing efforts to identify undiagnosed patients [21][23] Conclusion Zevra Therapeutics is positioned for significant growth in the rare disease therapeutics market, driven by its innovative product MIPLYFFA, a strong financial foundation, and strategic expansion efforts. The company is focused on enhancing patient access and leveraging its pipeline to solidify its market presence.

Financial Performance & Cash Position - Zevra exited Q3 2025 with a strong cash position of $230.4 million[8], and total debt of $61.3 million[44] - The company generated net revenue of $72.3 million in the 9-month period[8], and $26.1 million for Q3 2025[43] - Q3 2025 net loss was $(0.5) million, or $(0.01) per basic and diluted share[43] Product Portfolio & Pipeline - MIPLYFFA adoption achieved in approximately 40% of diagnosed Niemann-Pick Disease Type C (NPC) patients within the first year of launch[13] - Payor coverage for MIPLYFFA reached 66% of covered lives as of Q3 2025[24] - Approximately 80% of patients who participated in the Phase 2/3 clinical trial for MIPLYFFA took miglustat[20] - Celiprolol Phase 3 DiSCOVER trial is ongoing for Vascular Ehlers-Danlos Syndrome (VEDS), with 44 of 150 patients enrolled as of Q3 2025[40] - For VEDS patients on celiprolol, the annual major vascular event rate is approximately 5% compared to approximately 12% in untreated patients[40] Market & Regulatory - Approximately 900 individuals in the U S live with NPC, of which 300-350 are diagnosed or treated[27] - Approximately 1,100 individuals are living with NPC in Europe[27] - MAA for Arimoclomol is under review by the EMA[8, 13]