Real-world and clinical trial data underscore long-term safety and effectiveness in pediatric and adult NPC patientsCELEBRATION, Fla., Feb. 04, 2026 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on providing therapies for people living with rare disease, today announced the presentation of four posters highlighting positive new data on MIPLYFFA® (arimoclomol) for the treatment of Niemann-Pick Disease Type C (NPC) at the 22nd Annual ...

CELEBRATION, Fla., Feb. 02, 2026 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on providing therapies for people living with rare disease, today announced that it has been invited to ring the Nasdaq opening bell on Monday, February 9, 2026. Neil F. McFarlane, Zevra's President and Chief Executive Officer, will deliver opening remarks at 9:24 a.m. followed by the bell ringing at 9:30 a.m. ET, signifying the start of the day's trading ...

Core Insights - Zevra Therapeutics announced that four abstracts related to MIPLYFFA® (arimoclomol) have been accepted for presentation at the 22nd Annual WORLDSymposium™, including a podium presentation by Dr. Caroline Hastings on four-year real-world safety and efficacy data in Niemann-Pick Disease Type C (NPC) patients [1][2] Presentation Details - The podium presentation titled "Real-world Safety and Effectiveness of Arimoclomol in Patients with NPC: Outcomes from the U.S. Early Access Program (EAP) Over a 4-Year Period" will take place on February 5, 2026, at 2:30 p.m. PT, presented by Dr. Caroline Hastings [3] - A satellite symposium titled "Charting a Path in Niemann-Pick Disease Type C: Diagnostic Challenges, Therapeutic Innovations, and Real-World Patient Cases" will also be hosted by the company on February 5, 2026, at 12:15 p.m. PT, featuring Dr. Caroline Hastings as the presenter [4] Poster Presentations - Multiple poster presentations will occur on February 5, 2026, including: - "Multi-year Subgroup Analyses of Niemann-Pick Disease Type C Participants Treated with Arimoclomol in the U.S. Early Access Program," presented by Dr. Kristina Julich at 3:30 p.m. PT [5] - "Efficacy of Arimoclomol Combined with Miglustat at Months 3, 6, 9, and 12 of the Double-blind, Randomized, Placebo-controlled NPC002 Trial," presented by Dr. Eugen Mengel at 3:30 p.m. PT [6] - "Long-term Safety and Effectiveness of Arimoclomol in Adult and Pediatric Niemann Pick Disease Type C Patients in the US Early Access Program," presented by Dr. Damara Ortiz at 3:30 p.m. PT [7] Company Overview - Zevra Therapeutics is a commercial-stage company focused on developing therapies for rare diseases, with its lead product MIPLYFFA marketed for Niemann-Pick disease type C (NPC), a rare neurodegenerative disorder [9] - The company aims to broaden access through geographic expansion and has a pipeline of rare disease programs, emphasizing a patient-centric approach guided by values of accountability, integrity, innovation, and courage [9]

Company Overview - Zevra Therapeutics is a commercial-stage company focused on impacting individuals with rare diseases [2] - The company is currently in a growth phase, with the operational capacity to pursue its commercial and development goals [2] Product and Revenue - Zevra is commercializing its lead product, MIPLYFFA, in the U.S. for Niemann-Pick disease type C and has an MAA under review by the EMA [3] - The company is expanding access through geographic opportunities and has a pipeline of rare disease programs [3] - In the first nine months of the previous year, Zevra generated net revenues of $72.3 million and ended Q3 with a strong cash position of $230.4 million [3]

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]

Core Insights - Zevra Therapeutics, Inc. will present at the J.P. Morgan 44th Annual Healthcare Conference on January 15, 2026, at 9:45 a.m. PT [1] - The company is focused on therapies for rare diseases, with its lead product targeting Niemann-Pick disease type C (NPC) [3] Company Overview - Zevra is a commercial-stage company dedicated to providing life-changing therapeutics for individuals with rare diseases [3] - The company has a strong foundation through the commercialization of its lead product and is expanding access via geographic opportunities [3] - Zevra emphasizes a patient-centric approach, guided by values of accountability, integrity, innovation, and courage [3] Conference Participation - Management will be available for one-on-one meetings with registered attendees at the conference [2] - A live webcast of the presentation will be accessible on the Investor Relations section of Zevra's website [2]

Core Insights - Zevra Therapeutics (ZVRA) reported Q3 FY25 earnings on November 5th, 2025, with net revenues of $26.1 million, of which $22.4 million was generated from their primary revenue driver, MIPLYFFA, indicating a revenue increase of 605% compared to the same quarter last year [1] Company Performance - The company achieved a significant revenue growth of 605% year-over-year, highlighting its strong performance in the market [1] - MIPLYFFA remains the primary revenue driver, contributing a substantial portion of the total revenues [1]

Core Viewpoint - Zevra Therapeutics has entered into an exclusive expanded access distribution agreement with Uniphar to provide Niemann-Pick Disease Type C (NPC) patients access to MIPLYFFA (arimoclomol) in select territories outside of Europe [1][3] Company Overview - Zevra Therapeutics is a commercial-stage company focused on therapies for rare diseases, aiming to address unmet needs in this community [14] - The company has developed MIPLYFFA, the only treatment shown to halt disease progression in NPC, with FDA approval received on September 20, 2024 [4][5] Product Information - MIPLYFFA is indicated for use in combination with miglustat for treating neurological manifestations of NPC in patients aged 2 years and older [5] - The drug has demonstrated a durable effect, halting disease progression as measured by the NPC Clinical Severity Scale over a one-year trial [4] - MIPLYFFA has shown long-term clinical outcomes with over 5 years of patient experience across more than 270 NPC patients [4] Distribution Agreement - The agreement with Uniphar aims to expand access to MIPLYFFA, leveraging Uniphar's expertise in global distribution and supply chain management [3][16] - This partnership is expected to initially address a select patient population while Zevra continues to prepare for its U.S. commercial launch and potential EU approval [3] Clinical Significance - MIPLYFFA increases the activation of transcription factors that regulate lysosomal expression and has been shown to reduce unesterified cholesterol in NPC fibroblasts [4] - The pivotal phase 3 trial results indicate significant clinical benefits, although the full clinical significance of these findings is not yet fully understood [4] Regulatory Status - MIPLYFFA is currently approved in the U.S. and is under review by the European Medicines Agency for marketing authorization in Europe [3][4]

Company Overview - Zevra Therapeutics, Inc. is a commercial-stage company focused on providing therapies for rare diseases, aiming to address unmet medical needs and deliver life-changing therapeutics to affected individuals [3]. Board of Directors Update - Alicia Secor has been appointed to the Board of Directors, bringing over thirty years of experience in the life sciences industry, including leadership roles at Atalanta Therapeutics and Juniper Pharmaceuticals [2]. - Wendy Dixon, PhD, has retired from the Board, effective immediately, after providing strategic counsel during her tenure [2][3]. Leadership Insights - The Chair of the Board, Tamara A. Favorito, expressed confidence that Alicia Secor's expertise will support the company's strategic execution and enhance shareholder value [2]. - CEO Neil F. McFarlane highlighted Alicia's experience in rare diseases as a valuable addition to the team and thanked Wendy for her leadership [3].