Zevra Therapeutics, Inc. · NASDAQ:ZVRA
Zevra Therapeutics — Investor Presentation (January 2024)
24 slides from Zevra Therapeutics's investor presentation, Jan 8, 2024
Slide 1: A Rare Approach to Therapeutics
Corporate Presentation January 2024 A Rare Approach to Therapeutics NASDAQ: ZVRA
Slide 2: Cautionary Note Regarding Forward-Looking Statements
a\\A ANN Cautionary Note Regarding Forward-Looking QZEVRA Statements THERAPEUTICS ‘This presentation may contain forward-looking statoments within the meaning of the Private Securities Litigation Reform Act of 1985. Fonvard- looking statements include all statements that do not relate solely to hist
Slide 3: Becoming a Leading Rare Disease Company
aA ANY Becoming a Leading Rare Disease Company SZEVRA y Pil itegi Our Mission: Bringing life-changing Mc j i ‘ ten i therapeutics to people | Gemmercial exeeilencete/ensure/patlentnoresste living with rare diseases Growing pipeline with Potential to bringinew products and deliver value for patient
Slide 4: Experienced Team with Rare Disease Expertise
Experienced Team with Rare Disease Expertise S\ZEVRA THERAPEUTICS RAREDISEASEEHPERIENGE Ec A QEYME BIOMARIN: Matinekrodt Wastellas i Retrophin| <fateaa> a Fearne Adrian Wau £0 an Pron ae RARE PRODUCT EXPERIENCE socover Thiglaeey” INOmax Sekuwes kepaee 1NOmax ‘t ? " eveNITY fGhobamr ign pei : —— ee e
Slide 5: Commercial Excellence to Ensure Patient Access to Rare Disease Therapies
2\h\ ANN Commercial Excellence to Ensure Patient Access GZEVR to Rare Disease Therapies oT Overlap in Prescribers and Centers of Excellence between UCD and NPC indications allow for efficient team approach THERAPEUTICS fe) Rare Disease Patient Services Marketing Medical Affairs and Qu Sales Speciali
Slide 6: Growing Pipeline with Potential to Bring New Products and Deliver Value for Patients
Growing Pipeline with Potential to Bring New QZEVRA KP1077 Phase 2 topline data Idiopathic Hypersomnia (IH) expected HT 2028 P1077 ‘i eer Phase tral complete . ‘and arimociomol Tf : a Receiving royalties: ‘ontrack tobe paid next sales milestone Q1 2028 {Deena onan art drs haan 2H pc ert ly a ou ‘ Z
Slide 7: Urea Cycle Disorders Cause Hyperammonemia Leading to Brain Damage or Death
Urea Cycle Disorders Cause Hyperammonem GQZEVRA 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 : - clinical hallmark of UCDs ‘ perammonemi
Slide 8: Unmet Need in Urea Cycle Disorders
2 Unmet Need in Urea Cycle Disorders SZEVRA THERAPEUTICS Poor treatment adherence can lead to neurocognitive damage, coma and even death Orphan Designation Unmet Need + Incidence: 1 in 35,000 births! + Phenylbutyrates are approved to treat UCDs + Prevalence: * Palatability, odor, route of administra
Slide 9: OLPRUVA Designed to Address Unmet Needs
OLPRUVA® Designed to Address Unmet Needs QZEV + Novel formulation of phenylbutyrate |. adjunctive therapy to standard of care + Physicians attribute improved adherence to: + Dual-coated formulation delays release in water for up to 5 minutes, rapidly dissolves in stomach + Long-term management of ad
Slide 10: Niemann-Pick Disease Type C is a Progressive Lysosomal Storage Disorder
a aN Niemann-Pick Disease Type C is a QGZEVRA Progressive Lysosomal Storage Disorder THERAPEUTICS Cholesterol buildup leads to cell death; arimoclomol may enhance cholesterol metabolism through improved lysosomal function NPC! is alec ight + NPC gene mutations produce abnormal, absent or non-functio
Slide 11: No Approved NPC Treatments in the U.S.
2 No Approved NPC Treatments in the U.S. SIZEVRA THERAPEUTICS Ultra-rare, relentlessly progressive and fatal neurodegenerative disease Orphan Designation Significant Unmet Need + Incidence: 1 in 100,000-120,000 live births* + Neurocognitive decline adversely impacts + Prevalence: daily living + 1,80
Slide 12: Arimoclomol is Positioned to Become First-Line Treatment for NPC Patients
Arimoclomol is Positioned to Become First-Line QZ EVRA ‘Eesatmenrt for NPC Patients HERAPEUTICS Evi ates that arimoclomol acts on multiple fronts to help reduce lipid build-up in cells with prekes: lysosomal function ADVANTAGEOUS REGULATORY DESIGNATION EXPERIENCE WITH DEMONSTRATED SAFETY * Potential
Slide 13: Growing Pipeline in Rare Diseases
Growing Pipeline in Rare Diseases
Slide 14: Vascular Ehlers-Danlos Syndrome Impairs Connective Tissue and Leads to Vascular Ruptures
Vascular Ehlers-Danlos Syndrome Impairs QZEVRA 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: NE charactorized by ancurysme,cissoctons andlor rup
Slide 15: Unmet Need in VEDS
7 Unmet Need in VEDS SZEVRA THERAPEUTICS Mutation in COL3A1 gene impairs connective tissue and leads to vascular ruptures Orphan Designation + Incidence: 1 in 50,00 to 200,000 people" - Prevalence 7,500 diagnosed patients in U.S. ? Significant Unmet Need + No approved options in the U.S. + Current t
Slide 16: Celiprolol is a Selective Modulator for Potential Treatment of COL3A1+ vEDS
Celiprolol is a Selective Ad Modulator for S\ZEVRA Z oA, THERAPEUTICS Potential Treatment of patients with COL3A1+ vEDS Phase 3 primary endpoint: time to first occurrence of primary cardiac or arterial clinical event ‘CELIPROLOL FOR VEDS CLINICAL EXPERIENCE REGULATORY & IP ADVANTAGES: + Mechanism of
Slide 17: Idiopathic Hypersomnia Causes Excessive Daytime Sleepiness, Sleep Inertia and Brain Fog
Idiopathic Hypersomnia Causes Excessive QGZEVRA Daytime Sleepiness, Sleep Inertia and Brain Fog HE RABSiCnicls KP1077 may provide optimal exposure of methylphenidate to better address these unmet needs Idiopathic Hypersomnia Severity Scale + IHis a rare, debilitating, chronic neurologic disorder wit
Slide 18: Unmet Need in Idiopathic Hypersomnia
2 ANN Unmet Need in Idiopathic Hypersomnia SZEVRA THERAPEUTICS IH Is a rare, debilitating, chronic neurologic disorder with an unknown pathophysiology Orphan Designation Current Treatments Don’t Address Needs * Incidence: 10.3 per 100,000 people in the US* + Prevalence: ~37,000 patients diagnosed? +
Slide 19: KP1077 is a Novel Approach to Treating IH
KP1077 is a Novel Approach to Treating IH SZEVRA HERAPEUTICS Unique PK profile and dosing regimen designed to address EDS and sleep inertia IMPROVED SAFETY & REGULATORY & IP KP1077 FOR IH TOLERABILITY OVER EXISTING ADVANTAGES TREATMENTS + Proprietary prodrug of d-MPH + Unique pharmacokinetic profile
Slide 20: Focused on Key Pillars for Strategic Growth
Focused on Key Pillars for Strategic Growth SZEVRA HERAPEUTICS Our Vision: To give every promising rare disease therapy a fighting chance to improve the lives of the patients we serve Commercial Excellence Growing Pipeline + Growing capabilities in-line with vision for a bespoke patient services app
Slide 21: Appendix
Appendix SZEVRA THERAPEUTICS
Slide 22: NDA Resubmission
| NDA Resubmi Complete Response Letter (CRL) and FDA feedback gathered through multi interactions/meetings has provided added clarity on resubmissior RL Sufficiency of validation and reliability of the Niemann-Pick type © Clinical Severity Scale (NPCCSS) instrument Appropriateness of how to handle d
Slide 23: Two Dosing Regimens Being Explored to Achieve Sustained Symptom Management in IH
Two Dosing Regimens Being Explored to SIZEVRA Achieve Sustained Symptom Management in IH -MPH Concentration (ngimL) THERAPEUTICS PK after Multiple Oral Doses of KP1077 Analyte = d-MPH o Peak Occurs Around Exposure Remains king Until Bedtime once Daily (10 pm) BID (10 pmard 8 am) Plasma concentration
Slide 24: Phase 2 Clinical Trial of KP1077 in IH
Phase 2 Clinical Trial of KP1077 in IH SIZEVRA THERAPEUTICS Multi-center, dose-optimizing, double-blind, placebo-controlled, randomized-withdrawal study to evaluate safety of KP1077, as well as potential efficacy endpoints INTERIM DAT/ To inform the design of the PRIMARY ENDPOINT PHASE 2 TRIAL (N=48
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