Zevra Therapeutics, Inc. · NASDAQ:ZVRA
Zevra Therapeutics — Q2 2025 Earnings Presentation
35 slides from Zevra Therapeutics's earnings presentation, Aug 12, 2025
Slide 1: A Rare Approach to Therapeutics
A Rare Approach to Therapeutics August 2025 1 NasdaqGS: ZVRA
Slide 2: Cautionary Note Regarding Forward-Looking Statements
Cautionary Note Regarding 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-l
Slide 3: Two commercial-stage rare disease products
Two commercial-stage rare disease products Advanced clinical development pipeline Existing infrastructure supportive of future growth Strong financial position: $217.7M of cash, cash equivalents and Adult living with Niemann-Pick disease type C investments As of Jun 30, 2025 3
Slide 4: Pursuing our Mission to Bring Life-Changing Therapeutics to the Rare Disease Community
Pursuing our Mission to Bring Life-Changing Therapeutics to the Rare Disease Community Execute Focus Innovate Resources prioritized across four pillars to achieve our vision Commercial Pipeline and Talent and Corporate Excellence Innovation Culture Foundation Synergistic Potential for growth commerc
Slide 5: Strengthening Zevra as a Rare Disease Company Through Robust Advocacy Partnerships
Strengthening Zevra as a Rare Disease Company Through Robust Advocacy Partnerships 5
Slide 6: Experienced Team with Rare Disease Expertise
Experienced Team with Rare Disease Expertise Neil F. McFarlane R. LaDuane Clifton, CPA Joshua Schafer Alison Peters Rahsaan W. Thompson Adrian W. Quartel, Chief Legal Officer, Chief Executive Officer Chief Financial Officer Chief Commercial Officer Chief People Officer M.D., FFPM Secretary, and Comp
Slide 7: Diversified Portfolio to Deliver Value for Patients
Diversified Portfolio to Deliver Value for Patients iv PHASE 1 PHASE 2 PHASE 3 NDA/MAA APPROVED STATUS AND IP Zevra’s Portfolio FDA Approval: Sep 20, 2024 MIPLYFFATM arimoclomol Launch: Nov 21, 2024 Asset portfolio targeting Niemann-Pick Disease Type C (NPC) U.S. IP through 2031 via ODEi rare diseas
Slide 8: Efficient Team Approach to Providing Patient Access
Efficient Team Approach to Providing Patient Access Patient Account Medical Affairs Rare Disease Marketing Reimbursement Management & & Patient Specialists Services Contracting Advocacy Coordination of efforts between Letter of Medical Necessity Commercial copay assistance insurance companies and (L
Slide 9: U.S. Prescribing Information for MIPLYFFA (arimoclomol capsules)
U.S. Prescribing Information for MIPLYFFA® (arimoclomol capsules) Indication MIPLYFFA is indicated for use in combination with miglustat for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adult and pediatric patients 2 years of age and older. Recommended Dose Re
Slide 10: NPC is a Neurodegenerative Lysosomal Storage Disorder
NPC is a Neurodegenerative Lysosomal Storage Disorder NPC1 is a large, transmembrane protein with specific domains that are involved in the transport of lipophilic substrates such as cholesterol and sphingolipids2,3 NPC causes cholesterol buildup that leads to cell death NPC2 is a small, NPC1 solubl
Slide 11: NPC is an Ultra-Rare, Heterogenous and Fatal Disease
NPC is an Ultra-Rare, Heterogenous and Fatal Disease ~900 Prevalence Individuals in the U.S. live with NPC Of which 300-350 are diagnosed or treated6 Onset at Any Age Heterogeneous Makes NPC particularly difficult to Rate of progression, always fatal diagnose ~13 Years ~80% Of patients who participa
Slide 12: MIPLYFFA is Foundational Treatment for NPC
MIPLYFFA is Foundational Treatment for NPC Proven effectiveness using the NPC Clinical Severity Scale (NPCCSS) MIPLYFFA, used with miglustat, is the only treatment shown to halt disease Efficacy progression by addressing the underlying pathology of NPC -- with improvement seen at the first evaluatio
Slide 13: MIPLYFFA Shown to Halt Disease Progression by Addressing the Underlying Pathology of NPC
MIPLYFFA Shown to Halt Disease Progression by Addressing the Underlying Pathology of NPC in Patients who also Received Miglustat Improvement seen at the first evaluation at Week 12 and which continued for up to 7 years data that confirms its foundational long-term effectiveness, safety, and durabili
Slide 14: MIPLYFFA Status
MIPLYFFA Status U.S. Launch Metrics as of June 30, 2025i ▪ 129 prescription enrollment forms received ▪ 7 of which were enrollments in Q2 ▪ All active U.S. EAP participants enrolled to receive MIPLYFFA ▪ U.S. EAP closure, including locking the database and closing study centers ahead of previous gui
Slide 15: U.S. Prescribing Information for OLPRUVA (sodium phenylbutyrate) for Oral Suspension
U.S. Prescribing Information for OLPRUVA® (sodium phenylbutyrate) for Oral Suspension Indication OLPRUVA is a nitrogen-binding agent indicated as adjunctive therapy to standard of care, which includes dietary management, for the chronic management of adult and pediatric patients weighing 20 kg or gr
Slide 16: UCDs are Rare Inherited Metabolic Disorders Characterized by Hyperammonemia
UCDs are Rare Inherited Metabolic Disorders Characterized by Hyperammonemia Defect in one of the 6 enzymes or 2 transporters in urea cycle leads to accumulation of ammonia9,15 Elevated ammonia can be neurotoxic, leading to neurocognitive damage and even death10-13 Signs and symptoms of hyperammonemi
Slide 17: Poor Treatment Adherence Remains an Unmet Need in UCDs
Poor Treatment Adherence Remains an Unmet Need in UCDs ~1,100 Individuals diagnosed in the U.S.18 ~80% Of patients have deficiencies in the CPS, OTC, or AS enzymesi >800 Patients are currently receiving treatment in the U.S.18 >25% Of hyperammonemic crises stem from poor treatment Person living with
Slide 18: Potential to Improve Compliance with OLPRUVA through Formulation Designed for Convenience and Palatability
Potential to Improve Compliance with OLPRUVA through Formulation Designed for Convenience and Palatability Established efficacy profile through 505(b)(2) filing Efficacy ➢ OLPRUVA is indicated as adjunctive therapy to standard of care for long-term management of adults and children in UCDs involving
Slide 19: Unmet Medical Needs in UCDs
Unmet Medical Needs in UCDs Important reasons to reassess therapy for patients currently on a nitrogen scavenger therapy 1 Poor Adherence 2 Recurrent Hospital Visits Not on Optimalcrises) (hyperammonemic Treatment 3 Mild Undiagnosed UCD Adult living with urea cycle disorder Not on Optimal Treatment
Slide 20: Celiprolol
Celiprolol Potential Treatment for Vascular Ehlers-Danlos Syndrome (VEDS) 20 Celiprolol is investigational, safety and efficacy have not been established
Slide 21: VEDS Impairs Connective Tissue
VEDS Impairs Connective Tissue Abnormal Collagen Matrix Impairs Vascular Most severe Ehlers-Danlos syndrome Integrity & Elasticity subtype20 Inherited connective tissue disorder caused by COL3A1 gene mutations Abnormal Collagen III Altered Proteostasis Leads to defect in type III procollagen in vess
Slide 22: Significant Unmet Need in VEDS with No Approved Treatments
Significant Unmet Need in VEDS with No Approved Treatments ~7,500 Individuals in the U.S. diagnosed and live with VEDS22 95% of diagnoses are genetically confirmed (COL3A1)20, 21 ~25% ~90% Of patients experience an Of patients experience an event by the event before the age of 2020 age of 4020 51 Ye
Slide 23: Celiprolol is a Potential Treatment of Patients with COL3A1+ VEDS
Celiprolol is a Potential Treatment of Patients with COL3A1+ VEDS Celiprolol designed to reduce mechanical stress on collagen fibers within arterial wall New Chemical Entity in the U.S., Orphan Drug Designation and Breakthrough Therapy Designation, & IP to 2038 • Selective adrenergic modulator (SAM)
Slide 24: Strong Corporate Foundation Demonstrating Financial Discipline
Strong Corporate Foundation Demonstrating Financial Discipline 24
Slide 25: Financial Position is a Source of Strength
Financial Position is a Source of Strength Based on the Company’s current operating forecast and adding the non-dilutive capital from the PRV sale, we have ample resources and financial flexibility to execute on our strategic priorities independent from the capital markets Q2 2025 Income Statement D
Slide 26: Zevra has a Unique Opportunity to Impact People Living with Rare Diseases
Zevra has a Unique Opportunity to Impact People Living with Rare Diseases Two commercial-stage rare disease products ▪ Launch of MIPLYFFA for people living with NPC ▪ OLPRUVA launched for the treatment of UCDs involving deficiencies of CPS, OTC and AS Advanced clinical development pipeline ▪ Submitt
Slide 27: A Rare Approach to Therapeutics
A Rare Approach to Therapeutics August 2025 Children living with Niemann-Pick disease type C 27 NasdaqGS: ZVRA
Slide 28: Appendix
Appendix SZEVRA
Slide 29: Driving Adoption of OLPRUVA through Patient Experience
Driving Adoption of OLPRUVA through Patient Experience Diagnosed Adult Patients (Dx'd) • Only managed by diet, newly diagnosed, • With busy lifestyles not diagnosed, or misdiagnosed • Adult onset, OTC deficient • Need to take their medication at work • Taking medication was inconvenient • Have jobs
Slide 30: KP1077 (Serdexmethylphenidate)
KP1077 (Serdexmethylphenidate) Potential Treatment for Idiopathic Hypersomnia (IH) 30 KP1077 is investigational, safety and efficacy have not been established
Slide 31: IH Causes Brain Fog, Excessive Daytime Sleepiness, & Sleep Inertia
IH Causes Brain Fog, Excessive Daytime Sleepiness, & Sleep Inertia Idiopathic Hypersomnia Severity Scale Rare, debilitating, chronic neurologic disorder • 14 questions on a scale of 0 to 3 or 0 to 4, totaling 50 points 28 • Higher scores, indicate more severe/frequent symptoms28 Unknown pathophysiol
Slide 32: Remaining Unmet Need in IH
Remaining Unmet Need in IH ~37,000 Individuals in the U.S. diagnosed and live with IH, though the total population may be much larger32 ~66% Report IH symptoms as High Impact including not feeling refreshed, fatigue, excessive daytime sleepiness, and brain fog33 ~85% Require more than a first-line t
Slide 33: KP1077 has a Differentiated Profile Addresses Unmet Needs in IH
KP1077 has a Differentiated Profile Addresses Unmet Needs in IH Results from Ph. 2 Clinical Trialiii; All patients received active KP1077 for IH drug in Weeks 1-5; (decrease = improvement) • SDXi is a proprietary prodrug of d-MPH A. Epworth Sleepiness Scale (ESS) • Two dosing regimens being explored
Slide 34: KP1077 Ph. 2 Trial Produced Clinically Meaningful Improvements at All Endpoints
KP1077 Ph. 2 Trial Produced Clinically Meaningful Improvements at All Endpoints All patients received active drug in Weeks 1-5; (decrease = improvement) A. Epworth Sleepiness Scale (ESS) B. Idiopathic Hypersomnia Severity Score (IHSS) QD = once daily BID = twice daily QD = once daily BID = twice dai
Slide 35: Sources
SOURCES 1. Geberhiwot T, et al. Orphanet J Rare Dis. 2018 Apr 6;13(1):50. 2. Carstea ED et al. Science. 1997;277:228-231. 3. Platt FM et al. Annu Rev Genomics Hum Genet. 2014;15:173-194. 4. Ingemann L, Kirkegaard T J Lipid Res. 2014;55:2198-2210. 5. MRC-2400139-ASHG-2024-Arimoclomol-In-Vitro-Poster-