Zevra Therapeutics, Inc. · NASDAQ:ZVRA
Zevra Therapeutics — Q4 2025 Earnings Presentation
20 slides from Zevra Therapeutics's earnings presentation, Mar 9, 2026
Slide 1: A Rare Approach to Therapeutics
A Rare Approach TO THERAPEUTICS March 2026 Adult living with Niemann-Pick Nasdaq: ZVRA disease type C CRC-ZEV-26-0006-4
Slide 2: Forward Looking Statements
Forward Looking Statements Presentation may contain forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements relate to future events or our future financial performance. We generally identify forward-looking statements by termi
Slide 3: We are a Purpose Driven Company
We are a Purpose Driven Company OUR VALUES OUR MISSION Patient Centricity We redefine what is possible prioritizing patient needs in bringing life-changing therapies to people living with rare diseases Accountability delivering on commitments consistently Integrity always doing what is right OUR VIS
Slide 4: Unique Opportunity to Impact People Living with Rare Diseases
Unique Opportunity to Impact People Living with Rare Diseases Commercial-stage rare disease products Late-stage clinical development assets MAA under review by EMA Existing infrastructure can be leveraged for future growth Multiple Exited Q4 25 Growth mode; revenue sources with a strong operating ru
Slide 5: Diversified Portfolio Delivering Value for Patients
Diversified Portfolio Delivering Value for Patients PHASE 1 PHASE 2 PHASE 3 NDA/MAAi APPROVED STATUS & IP FDA Approval: Sep 20, 2024 MIPLYFFA® arimoclomol Niemann-Pick Disease Type C (NPC) Protection through 2031 via U.S ODEii Commercial OLPRUVA® sodium phenylbutyrate for oral suspension FDA Approva
Slide 6: Fulfilling Our Mission to Bring Life-Changing Therapies to People Living with Rare Disease
Fulfilling Our Mission to Bring Life-Changing Therapies to People Living with Rare Disease Focus Execute Innovate Resources Prioritized Across Four Pillars to Achieve our Vision Marketed Pipeline Talent Corporate Therapies and Innovation and Culture Foundation Growth mode for Synergistic growth Expe
Slide 7: Execution in 2025 Paved the Way for Multiple Growth Drivers in 2026
Execution in 2025 Paved the Way for Multiple Growth Drivers in 2026 Marketed Pipeline Talent Corporate Products and Innovation and Culture Foundation Established Significant Becoming Strengthened MIPLYFFA as Growth “Partner of Capital Position Foundational Opportunities Choice” for Rare Through $150
Slide 8: Marketed Therapies
Marketed Therapies Niemann-Pick disease type C (NPC) is an ultra-rare genetic disorder that leads to premature mortality. Adult living with NPC For full prescribing information, visit MIPLYFFA.com
Slide 9: NPC is a Devastating and Fatal Lysosomal Storage Disorder
NPC is a Devastating and Fatal Lysosomal Storage Disorder ~900 people in the U.S. with NPC, 300–350 diagnosed or treated1 Cholesterol Buildup Leads to Cell Death Heterogenous Presentation NPC gene mutations produce abnormal, absent, or non-functional NPC proteins2 Age of Onset Split 50/50 (children/
Slide 10: Multiple Trials Demonstrate MIPLYFFA's Impact on NPC Progression
Multiple Trials Demonstrate MIPLYFFA’s Impact on NPC Progression Proven effectiveness using the NPC Clinical Severity Scale in combination with miglustat i,3, 4 R4DNPCCSS over 48 months OLE with MIPLYFFA + miglustat ~80% Of patients who participated in the Phase 2/3 clinical trial took miglustat3 Mo
Slide 11: Establishing MIPLYFFA as Foundational Treatment for NPC
Establishing MIPLYFFA as Foundational Treatment for NPC Demonstrating Clinical Increasing Identification of Driving Patient Access Differentiation Patients • Unique mechanism of action • Payor coverage at 68% of • 161 patient enrollments; 52 in improves cholesterol trafficking covered lives as of Q4
Slide 12: Capturing the Next Wave of Growth for MIPLYFFA
Capturing the Next Wave of Growth for MIPLYFFA U.S. Prevalence EU Prevalence Expanded Access ~900 individuals ~1,100 individuals living with Geographic expansion to in the U.S. live with NPC NPC in Europe5 broaden access outside the of which 300-350 are diagnosed Diagnosis rates estimated to U.S. an
Slide 13: Pipeline and Innovation
Pipeline and Innovation Vascular Ehlers-Danlos Syndrome (VEDS) is a severe autosomal, dominant, genetic, connective tissue disorder.
Slide 14: VEDS is a Life-threatening Connective Tissue Disorder with No Approved Treatments
VEDS is a Life-threatening Connective Tissue Disorder with No Approved Treatments VEDS is the most severe Ehlers-Danlos syndrome subtype6 Inherited connective tissue Leads to defect in type III procollagen in Characterized by arterial aneurysms and disorder caused by COL3A1 gene vessel walls and hol
Slide 15: Celiprolol is a Potential Treatment of Patients with COL3A1+ VEDS
Celiprolol is a Potential Treatment of Patients with COL3A1+ VEDS • Celiprolol addresses VEDS by reducing mechanical stress on collagen fibers within arterial wall9 • New chemical entity in the U.S., Orphan drug & breakthrough therapy designation • Selective adrenergic modulator (SAM) Consistent Red
Slide 16: Strong Corporate Foundation
BALANCE SHEET AS OF SEPT 30, 2025: • Available cash, cash Strong Corporate Foundation equivalents and investments of $230.4M Demonstrating Financial Discipline
Slide 17: Financial Position is a Source of Strength
Financial Position is a Source of Strength 2025 Income Statement Detailsi Net revenue of $ 106.5Mii Balance Sheet as of EAP net Dec. 31, 2025: reimbursements: • Available cash, cash $13.0M equivalents and investments AZSTARYS license of $238.9M agreement: $5.0M – Debt of $61.9M MIPLYFFA: $87.4M OLPR
Slide 18: Growth Drivers to Maximize the Opportunity in 2026 and Beyond
Growth Drivers to Maximize the Opportunity in 2026 and Beyond Commercial Execution Possible Patent Term MAA Submitted; Potential to Further Unlock NPC Extension for MIPLYFFA for EMA Approval of Opportunity in the U.S. Arimoclomol • Continue successful • Any exclusivity beyond current • Continue to d
Slide 19: A Rare Approach to Therapeutics
A Rare Approach to Therapeutics Nasdaq: ZVRA Children living with NPC
Slide 20: Sources
Sources 1. Burton et.al., Molecular Genetics and Metabolism Volume 134, Issues 1–2, September–October 2021, Pages 182-187 2. Geberhiwot T, et al. Orphanet J Rare Dis. 2018 Apr 6;13(1):50. 3. MIPLYFFA Full Prescribing Information. Celebration, FL, US, Zevra Therapeutics Inc.; 09/2024. 4. Mengel et al