Zevra Therapeutics, Inc. · NASDAQ:ZVRA
Zevra Therapeutics — Investor Presentation (March 2024)
27 slides from Zevra Therapeutics's investor presentation, Mar 5, 2024
Slide 1: Corporate Presentation March 2024
SZEVRA THERAPEUTICS Corporate Presentation March 2024 A Rare Approach to Therapeutics NasdaqGS: ZVRA
Slide 2: Cautionary Note Regarding Forward-Looking Statements
a. 0 SaA\ Cautionary Note Regarding Forward-Looking SIZEV Statements This presentation may contain forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward- looking statements include all statements that do not relate solely to historical or curr
Slide 3: Becoming a Leading Rare Disease Company
Becoming a Leading Rare Disease Company SIZEV Focused on Key Pillars for Strategic Growth Experienced team with rare disease expertise Our Mission: Bringing life-changing therapeutics to people ‘ ) therapeutics living with rare diseases ize a Commercial excellence to ensure patient access to Growing
Slide 4: Experienced Team with Rare Disease Expertise
ACA SSSSSANN Experienced Team with Rare Disease Expertise SAZEV Our Mission: Bringing life-changing therapeutics to people living with rare diseases SESE SS Neil F. McFarlane CEO and President peg Christal Mickle Chief Development Officer & Co-Founder Adrian W. Quartel, MD FFPM Chief Medical Officer
Slide 5: Establishing Zevra as a Rare Disease Company Through Robust Advocacy Partnership
Establishing Zevra as a Rare Disease Company WZEV Z Through Robust Advocacy Partnership i C B 9 les Prrsicunss Cycle CG f npdf S | eeR Az MEDICAL Ri Disorders — wmovnemmwprocoiseaserounoaon —- CONSOrtium <{ a P soa ones Foundation Yd —— aE, lam N Cm) firefly” INGE svinins I Pveersomnie FUND FOR RARE
Slide 6: Diversified Portfolio with Potential to Bring New Products and Deliver Value for Patients
Diversified Portfolio with Potential to Bring New QZEV Products and Deliver Value for Patients Dieter’ Perens FDA J _rases | sane aca } mm | aD Ean Zevra’s Portfolio Full scale U.S. commercial launch Jan 2024 2@ Asset portfolio targeting Arimoclomol PDUFA rare diseases Niemann Sept 21, 2024 Celiprol
Slide 7: Commercial Excellence to Ensure Patient Access
Commercial Excellence to Ensure Patient Access
Slide 8: Launch Excellence to Ensure Patient Access to Rare Disease Therapies
aC. ANY Launch Excellence to Ensure Patient Access to Rare Disease Therapies SZEVRA THERAPEUTICS Overlap in Prescribers and Centers of Excellence for OLPRUVA® and arimoclomol allow for efficient team approach Rare Disease Sales Specialists callingon prescribing physicians and Centers of Excellence P
Slide 9: Urea Cycle Disorders Cause Hyperammonemia, Leading to Brain Damage or Death
Urea Cycle Disorders Cause Hyperammonemia, Wd RA Leading to Brain Damage or Death THERAPEUTICS OLPRUVA® is a nitrogen scavenger that removes excess ammonia « Defect in one of the 6 enzymes or 2 transporters in the urea cycle leads to accumulation of ammonia * Aclinical hallmark of UCDs is hyperammon
Slide 10: Unmet Need in Urea Cycle Disorders
2. ANY SZEVRA Unmet Need in Urea Cycle Disorders SLE VISES Poor treatment adherence can lead to neurocognitive damage, coma and even death Orphan Designation + Incidence: 1 in 35,000 births" + Prevalence: » ~2,100 estimated patients in US" * ~1,100 patients diagnosed? » >800 treated? « About 80% of
Slide 11: OLPRUVA Designed to Address Unmet Needs in Treatment of UCDs
OLPRUVA® Designed to Address Unmet Needs in Treatment of UCDs Unique formulation in single-dose envelopes for ”ammonia control on the go” UNIQUE FORMULATION DRIVES PALATABILITY AND ADHERENCE + Novel formulation of phenylbutyrate + Dual-coated formulation delays release in water for up to 5 minutes,
Slide 12: Growing Pipeline in Rare Diseases
Growing Pipeline in Rare Diseases
Slide 13: Niemann-Pick Disease Type C is a Progressive Lysosomal Storage Disorder
Niemann-Pick Disease Type C is a QWZEVRA A a, Progressive Lysosomal Storage Disorder Tipetunwe erties Cholesterol buildup leads to cell death; arimoclomol may enhance cholesterol metabolism through improved lysosomal function e « NPC gene mutations produce abnormal, ul absent or non-functional NPC p
Slide 14: Unmet Need in Niemann-Pick Disease Type C
Orphan Designation yN * Incidence: 1 in 120,000 live births’ + Prevalence: * 1,800 patients estimated in EU and US ° 900 patients estimated in US? * 300-350 US patients currently diagnosed or treated? No Approved NPC Treatments in the U.S. Ultra-rare, relentlessly progressive and fatal neurodegenera
Slide 15: Arimoclomol is Positioned to Become First-Line Treatment for NPC Patients
AAA SANN Arimoclomol is Positioned to Become First-Line Treatment for NPC Patients Evidence indicates that arimoclomol acts on multiple fronts to help reduce lipid build-up in cells with improved lysosomal function SZEVRA THERAPEUTICS FIRST-IN-CLASS, ORAL TREATMENT - Potential to be the foundational
Slide 16: Vascular Ehlers-Danlos Syndrome Impairs Connective Tissue and Leads to Vascular Ruptures
Vascular Ehlers-Danlos Syndrome Impairs QZEV Connective Tissue and Leads to Vascular Ruptures THERAPEUTICS Celiprolol designed to reduce the mechanical stress on collagen fibers within the arterial wall * vEDS (EDS type IV) is the severe subtype: * Characterized by aneurysms, dissections and/or rupt
Slide 17: Unmet Need in Vascular Ehlers-Danlos Syndrome
AVA SANN Unmet Need in Vascular Ehlers-Danlos Syndrome Orphan Designation + Incidence: 1 in 50,00 to 200,000 people’ » Prevalence 7,500 diagnosed patients in U.S. 2 4. hitos:/www.orpha net SZEVRA THERAPEUTICS Mutation in COL3A1 gene impairs connective tissue and leads to vascular ruptures Significan
Slide 18: Celiprolol is a Selective Adrenergic Modulator for Potential Treatment of COL3A1+ vEDS
VAAN Celiprolol is a Selective Adrenergic Modulator for Potential Treatment of Patients with COL3A1+ vEDS CELIPROLOL FOR VEDS Mechanism of action in VEDS patients | - is thought to be through vascular dilatation and smooth muscle relaxation May reduce the mechanical stress on | > collagen fibers wit
Slide 19: Idiopathic Hypersomnia Causes Excessive Daytime Sleepiness, Sleep Inertia and Brain Fog
Idiopathic Hypersomnia (IH) Causes Excessive QZEV Daytime Sleepiness, Sleep Inertia and Brain Fog EVE Mtone ie nies KP1077 may provide optimal exposure of methylphenidate to better address these unmet needs Idiopathic Hypersomnia Severity Scale ° IHis a rare, debilitating, chronic neurologic disorde
Slide 20: Unmet Need in Idiopathic Hypersomnia
Orphan Designation * Incidence: 10.3 per 100,000 people in the US* * Prevalence: ~37,000 patients diagnosed? * Total population may be much larger Unmet Need in Idiopathic Hypersomnia S\ZEVRA THERAPEUTICS IH is a rare, debilitating, chronic neurologic disorder with an unknown pathophysiology Current
Slide 21: KP1077 is a Novel Approach to Treating IH
VAAN KP1077 is a Novel Approach to Treating IH SZEVRA THERAPEUTICS Unique PK profile and dosing regimen designed to address EDS and sleep inertia KP1077 FOR IH + Proprietary prodrug of d-MPH + Potential to address primary IH symptoms. + Two dosing regimens being explored + Once daily at bedtime * 2x
Slide 22: Focused on Key Pillars for Strategic Growth
Focused on Key Pillars for Strategic Growth SZEV Our Mission: Bringing life-changing therapeutics to people living with rare diseases Rare Disease Team Commercial Excellence Growing Pipeline + Strong experience in rare disease + Arimoclomol: PDUFA is Sep 21, 2024 commercial launches * Celiprolol: On
Slide 23: Thank You
5 hank Yo ZEVRA \ THERAPEUTICS I AN SS
Slide 24: Appendix
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Slide 25: Arimoclomol NDA Resubmitted to FDA
AVA SANN Arimoclomol NDA Resubmitted to FDA SZ RA Complete Response Letter (CRL) and FDA feedback gathered through multiple interactions/meetings has provided added clarity on resubmission package. ® ® CRL ZEVRA’S ONGOING RESPONSE Niemann-Pick type C Clinical Severity the NPCCSS as the primary instr
Slide 26: Two Dosing Regimens Being Explored to Achieve Sustained Symptom Management in IH
AVA SANN Two Dosing Regimens Being Explored to WZ Achieve Sustained Symptom Management in IH d-MPH Concentration (ng/mL) PK after Multiple Oral Doses of KP1077 Analyte = d-MPH a Peak Occurs Around Exposure Remains Waking Until Bedtime 25 20 45: —Once Daily (10 pm) —BID (10 pmand 8 am) 10 5 0 10PM12A
Slide 27: Phase 2 Clinical Trial of KP1077 in IH
Phase 2 Clinical Trial of KP1077 in IH SWZEVRA THERAPEUTICS Multi-center, dose-optimizing, double-blind, placebo-controlled, randomized-withdrawal study to evaluate safety of KP1077, as well as potential efficacy endpoints INTERIM DATA: To inform the design of the Saul? (eu Teleel Sir PHASE 2 TRIAL
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