Zevra Therapeutics, Inc. · NASDAQ:ZVRA
Zevra Therapeutics — Kempharm — Canaccord Genuity 35th Annual Growth Conference 2015
30 slides from Zevra Therapeutics's conference presentation, Aug 12, 2015
Slide 1: Canaccord Genuity 35th Annual Growth Conference
Exhibit 99.1 Canaccord Genuity 35th Annual Growth Conference August 12, 2015
Slide 2: Cautionary Note Regarding Presentation Information
Cautionary Note Regarding Presentation Information This presentation contains forward-looking statements, including statements about our plans to develop and commercialize our product candidates, our planned clinical trials for KP201/APAP and our other prodrug product candidates, the timing of and o
Slide 3: KemPharm Overview
KemPharm Overview • Specialty pharmaceutical company discovering and developing novel prodrugs • Leverage LAT Platform Technology to improve the attributes of approved drugs in large markets o 505(b)(2) pathway reduces risk and expense o Composition-of-matter patent protection • KP201/APAP has the p
Slide 4: Management Team
Management Team Travis C. Mickle, PhD President and CEO Gordon K. Johnson CBO Tracy M. Woody CCO R. LaDuane Clifton CFO Sven Guenther, PhD EVP of R&D 4
Slide 5: Ligand Activated Therapy (LAT) Platform Technology
Ligand Activated Therapy (LAT) Platform Technology LAT LAT LAT Approved Drug with Ligand Prodrug with Cleaved Drug through Sub-Optimal Improved Attributes Human Metabolic Properties Process 1) Select FDA-approved and widely prescribed drug for improvement 2) Chemically modify using a ligand to creat
Slide 6: Pipeline of Multiple Product Candidates
Pipeline of Multiple Product Candidates Selected KemPharm Prodrug Product Candidates Indication / Parent Drug Product Candidate Development Status Key Milestone Pain Hydrocodone (IR) KP201/APAP Clinical Trials NDA Filing – 2H 2015 Hydromorphone (ER) KP511/ER Preclinical Human POC Data – 2016 Oxycodo
Slide 7: Advancing Opioid Abuse-Deterrent Technology
Advancing Opioid Abuse-Deterrent Technology No Tamper Resistance Traditional Opioids Many Formulation- Crushing or Physical Based Abuse-Deterrent Extraction Manipulation Opioids KemPharm’s Molecular Neutralization Crushing or Physical Chemical Based Abuse-Deterrent Extraction and/or Manipulation Hyd
Slide 8: Opioid Abuse-Deterrent Landscape
Opioid Abuse-Deterrent Landscape KemPharm OxyContin TARGINIQ EMBEDA Hysingla Prodrugs Multiple ER ER ER ER Parent Drug IR/ER Opioids Oxycodone Oxycodone Morphine Hydrocodone Resists Physical Tampering Resists Non-Oral Abuse Molecular-Based Deterrence x x x x Prevents Common Solvent Extraction x x x
Slide 9: KP201/APAP Overview
KP201/APAP Overview Treatment of Acute Moderate to Moderately Severe Pain 9
Slide 10: KP201/APAP Product Features
KP201/APAP Product Features Prodrug composed of hydrocodone and a generally regarded as safe (GRAS) ligand Molecular-Based Abuse-Deterrent Technology Composition-of-Matter Patent Protection Until 2031 No Generic Equivalent Product (Benzhydrocodone) Convenient Dosing Successfully completed bioequival
Slide 11: Bioequivalence Study KP201.102 (KP201/APAP vs. Norco)
Bioequivalence Study KP201.102 (KP201/APAP vs. Norco®) Hydrocodone/Hydromorphone Acetaminophen 20,000 500 4.5 1 ×7.5/325 mg Norco 1×7.5/325 mg Norco (HC) 450 4.0 1 ×6.67/325 mg 1×6.67/325 mg KP201/APAP KP201/APAP (HC) 400 3.5 15,000 1 ×7.5/325 mg Norco (HM) 350 3.0 1 ×6.67/325 mg KP201/APAP (HM) 300
Slide 12: KP201/APAP Tamper Resistant Properties – In Vitro Studies
KP201/APAP Tamper Resistant Properties – In Vitro Studies • Extraction of API (KP201) only yields inactive prodrug • Hydrocodone not released through: o Physical manipulation (e.g., grinding) o Common solvent extraction (e.g., “alcohol dose dumping”, “cold water extraction”) • KP201 is chemically st
Slide 13: KP201 Solvent Hydrolysis Studies
KP201 Solvent Hydrolysis Studies Preliminary results suggest that KP201 remains intact and does not hydrolyze or break down into its components in commonly available solvents % Release of Hydrocodone Solvent Ambient Temperature At Boiling Point 0.5 hours 1 hour 4 hours 0.5 hours 1 hour 4 hours Water
Slide 14: KP201 Abuse-Deterrent Properties in Rat Studies
KP201 Abuse-Deterrent Properties in Rat Studies Intranasal Intravenous 500 4,000 HC·BT (2.0 mg/kg HC eq.) HC·BT (25.5 mg/kg HC eq.) KP201 (2.0 mg/kg HC eq.) KP201 (0.2 mg/kg HC eq.) 400 3,000 300 2,000 200 1,000 100 0 0 0.0 0.2 0.4 0.6 0.8 1.0 0.0 0.2 0.4 0.6 0.8 1.0 Time [hours] Time [hours] Note:
Slide 15: Recently Completed KP201.A01 Human Abuse Liability Trial
Recently Completed KP201.A01 Human Abuse Liability Trial • Oral Human Abuse Liability Trial o Compared the drug likability, exposure levels and safety of KP201/APAP compared to Norco after oral administration o Single-center, randomized, double-blind, active- and placebo-controlled crossover trial (
Slide 16: Ongoing Human Abuse Liability and PK Trials
Ongoing Human Abuse Liability and PK Trials • Intranasal Human Abuse Liability Trial o Assesses the drug likability, exposure levels and safety of KP201/APAP compared to Norco after crushing and intranasal administration o Single-center, randomized, double-blind trial (n=40) o Data in 3Q 2015 • Intr
Slide 17: KP201/APAP Potential Abuse-Deterrent Label Claims
KP201/APAP Potential Abuse-Deterrent Label Claims FDA Guidance • The FDA outlines 3 types of studies that may translate into 4 categories of label claims • KemPharm’s abuse liability 1. Laboratory manipulation and extraction studies (Category 1) program is consistent with the FDA Guidance: 2. Pharma
Slide 18: Large Market Opportunity
Large Market Opportunity • Hydrocodone is associated with more drug abuse and diversion than any other licit or illicit opioid(1) • IR hydrocodone in combination with acetaminophen is the most frequently prescribed opioid in the U.S.(2) o IR hydrocodone/APAP products accounted for 127.4 million pres
Slide 19: KP201/APAP Commercial Strategy
KP201/APAP Commercial Strategy KemPharm has many potential avenues to commercialize KP201/APAP, including but not limited to, a collaboration or establishing a specialist U.S. sales force Collaboration Specialist U.S. Sales Force • Potential global / U.S.-based • Specialist sales force targeting dea
Slide 20: State Legislative Initiatives
State Legislative Initiatives WA ME MT ND OR MN NH* ID WI NY VT SD MI MA WY RI IA PA NE CT NV OH UT** IN NJ IL WV* DE CA CO** KS MO VA* MD KY NC TN** OK AZ AR SC NM* AL GA MS TX LA To be filed FL Introduced Passed by one or both Houses * Bill would require a study of AD inclusion on formularies ** B
Slide 21: KP201/APAP Milestones
KP201/APAP Milestones Human Bioequivalence Preclinical Abuse Deterrence / Tamper Resistance Oral Human Abuse Liability Trial Intranasal Human Abuse Liability Trial 3Q 2015 Intranasal PK Trial 3Q 2015 NDA Submission 2H 2015 NDA Approval (with priority review) As Early As Mid-2016 DEA Scheduling and P
Slide 22: KP511/ER Overview
KP511/ER Overview Treatment of Moderate to Severe Pain 22
Slide 23: KP511/ER Product Overview
KP511/ER Product Overview • KP511/ER is an ER formulation of KP511, a prodrug of hydromorphone • IR bioequivalent release of hydromorphone demonstrated in rats • Potential valuable properties based on preclinical data o Significantly reduced IN and IV bioavailability (abuse deterrence) o Highly tamp
Slide 24: KP511/ER Reduced Abuse Potential
KP511/ER Reduced Abuse Potential Intranasal PK Curves Intravenous PK Curves 1,200 HM (compiled) 60 HM KP511 (compiled) KP511 1,000 50 800 40 600 30 400 20 200 10 0 0 0 0.2 0.4 0.6 0.8 1 0 0.5 1 1.5 2 Time [h] Time [h] • 2.0 mg/kg (hydromorphone eq.) • 0.2 mg/kg (hydromorphone eq.) • Average data fro
Slide 25: KP511/ER Potential Oral Overdose Protection
KP511/ER Potential Oral Overdose Protection AUC 3,500 HM·HCl extrapolated 35 3,000 30 HM·HCl (HM) 25 20 KP511 (HM) 2,500 15 10 5 2,000 0 0 1 2 3 4 Time [h] 1,500 1,000 500 0 0 4 8 12 16 20 24 28 32 36 40 44 48 52 56 60 64 Dose [mg/kg HM] Note: HM refers to hydromorphone hydrochloride. Studies conduc
Slide 26: KP415 Overview
KP415 Overview Treatment for ADHD 26
Slide 27: KP415 Overview
KP415 Overview • Prodrug of methylphenidate • Branded formulations of methylphenidate (Concerta, Focalin and Ritalin) accounted for sales of $1.1 billion in 2014(1) • Potential features and benefits o Controlled release methylphenidate o Reduced abuse potential o Suitable for more patient compliant
Slide 28: KemPharm Expected News Flow
KemPharm Expected News Flow Product Event Date KP201/APAP Intranasal Human Abuse Liability Trial – Clinical Results 3Q 2015 KP201 (API) Intranasal PK Trial – Clinical Results 3Q 2015 KP201/APAP NDA Submission 2H 2015 KP201/APAP NDA Approval (Priority Review) As Early As Mid-2016 KP201/APAP DEA Sched
Slide 29: 1Q 2015 Update
1Q 2015 Update • Cash of $10.3 million as of March 31, 2015 • Net proceeds from IPO in April/May 2015 of $59.9 million, net of underwriting discounts and commissions • 1Q 2015 net loss was $6.0 million vs. $1.9 million for 1Q 2014 o Increase in net loss year-over-year primarily due to IPO expenses a
Slide 30: For additional information please contact:
For additional information please contact: Gordon K. “Rusty” Johnson rjohnson@kempharm.com 319-665-2575
Kempharm — Canaccord Genuity 35th Annual Growth Conference 2015 on 6ix