Zevra Therapeutics, Inc. · NASDAQ:ZVRA
Zevra Therapeutics — Kempharm — Janney Healthcare Conference (September 2018)
34 slides from Zevra Therapeutics's conference presentation, Sep 17, 2018
Slide 1: Janney Healthcare Conference
K Pharm Janney Healthcare Conference
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 our prodrug product candidates, the timing of and our ability to obtain
Slide 3: KemPharm Overview
KemPharm Overview + Specialty pharmaceutical company discovering and developing novel prodrugs + Leveraging our LAT™ Platform Technology to improve the attributes of approved drugsin large markets + Building a pipeline of product candidates for ADHD, pain and CNS disorders + Potentially utilizing FD
Slide 4: LAT Platform Technology
LAT™ (Ligand Activated Therapy) Platform Technology Approved ona with Ligand Prodrugwith 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 create a prodrug o
Slide 5: KemPharm Product Pipeline
KemPharm Product Pipeline Potential Product Development Next category Candidate TOHELINT Status Milestone ne Submission Methylphenidate a NDA KP415 (ER) Clinical Submission Q1 2019 ADHD Methylphenidate ti if jethylphenidate ini jation of KP484 (ER) Clinical Efficacy Trial 2019 | Potential Apadaz® et
Slide 6: Prodrug New Product Development Partnering
Prodrug New Product Development Partnering + Strategy to potentially leverage KemPharm’s proprietary LAT™ Platform Technology through partnering + Partner benefits may include new or improved products based on potential: o Creation of new, long-lived IP protection eo Modification of pharmacokinetic
Slide 7: Attention-Deficit/Hyperactivity Disorder: KP415 and KP484
Attention-Deficit/Hyperactivity Disorder: KP415 and KP484 for the Treatment of ADHD
Slide 8: The ADHD and ER Methylphenidate Market
The ADHD and ER Methylphenidate Market + ~$13 billion ADHD market with TRx growth of ~4% per year on average * The branded portion of the ADHD market was ~$6.1B in 2017 and more than 95% of these branded prescriptions are for extended release + Methylphenidate (MPH) accounted for approximately 19.6
Slide 9: Selected ER Stimulant Products for ADHD
Selected ER Stimulant Products for ADHD Brand Name Dosage Reduced Active Ingredient Form (1) Drug Liking (1) + Prodrug of d-amphetamine 10, 20, 30, 40, + Ages: 6 years and older 50,60 &70 + Onset: 1.5 hours a Vyvanse® (lisdexamfetamine) Chewable, ce |e Discos 13 hours Difference + Indicated for bing
Slide 10: Selected ER Stimulant Products for ADHD (Cont'd)
Selected ER Stimulant Products for ADHD (Cont’d) _ 2017 2017 Brand Name 2017 2017 WAG Unk Plan Avg. Patient Active Ingredient TRx’s(1) | Sales(1) See Approval | Out-of-Pocket rice (2) Rate (1) Expense (1) Vyanse® ey 11.6MM $3.38 40mg: $9.02 94% $41.65 Adderall XR® (mixed salts of AMPH) 36MM ‘$805MM
Slide 11: KP415 Product Overview
KP415 Product Overview Prodrug of d-MPH (serdexmethylphenidate) with extended release properties, co-formulated with immediate release d-MPH Potential KP415 features and benefits o Once-daily dosing o Earlier onset and longer duration of therapeutic effect o Active metabolism may offer more predicta
Slide 12: KP415.E01 Pivotal Efficacy Trial Overview
KP415.E01 Pivotal Efficacy Trial Overview + First patient treatment on December 20, 2017 * Double-blind, placebo-controlled, randomized, parallel, analog classroom trial * Children 6 to 12 years with ADHD (150 completers) + 5U.S. trial sites, 2-3 cohorts each; 5-18 subjects per cohort/per site + 3-w
Slide 13: KP415.E01 Pivotal Efficacy Trial Met Primary Endpoint
KP415.E01 Pivotal Efficacy Trial Met Primary Endpoint + KP415.E01 met the primary endpoint of mean difference in change from baseline (pre-dose Visit 5) across all post-dose time points for the SKAMP- C score (p<0.001) Changein SKAMP-C from Pre-dose Visit 5 Difference in 95% Confidence eS Items) LS
Slide 14: KP415.E01 Pivotal Efficacy Trial: Differences in SKAMP-C
KP415.E01 Pivotal Efficacy Trial: Differences in SKAMP-C Change Between KP415 and Placebo Are Statistically Significant Baseline = predose (prespecified)" (post hoc)" Difference in Difference in LS mean (SE) LS mean (SE) Time KP415— Placebo p-Value KP415— Placebo _p-Value Predose 2.37 (1.18) 0.044 0
Slide 15: KP415.E01 Pivotal Efficacy Trial: Differences in PERMP-A
KP415.E01 Pivotal Efficacy Trial: Differences in PERMP-A Change Between KP415 and Placebo Are Statistically Significant Baseline = predose (prespecified)" (post hoc)" Difference in Difference in LS mean (SE) LS mean (SE) Time KP415— Placebo p-Value KP415— Placebo _p-Value Predose -0.43 (5.18) 0.933,
Slide 16: KP415.E01 Pivotal Efficacy Trial Summary
KP415.E01 Pivotal Efficacy Trial Summary + KP415.E01 efficacy trial met its primary endpoint + SKAMP-C trial data using Visit 6 as the pre-dose baseline demonstrate a 30-minute onset of effect with duration up to 13 hours *« PERMP-A and PERMP-C trial data are also supportive of a 30-minute onset of
Slide 17: KP415 Prodrug Drug Liking Emax After Oral Administration
KP415 Prodrug Drug Liking E,,., After Oral Administration was Significantly Lower vs. Focalin XR and Phentermine Maximum Drug Liking Scores (E,,2x) 4 abc de ae 815 802 | KP 415 Prodrug, 120 mg 3 KP415 Prodrug, 240 mg mmm Focalin XR, 80 mg t= Phentermine, 60 mg lm Placebo peer Neutral Score Drug Liki
Slide 18: KP415 Prodrug At-the-Moment Drug Liking After Oral Administration vs. Focalin XR
KP415 Prodrug At-the-Moment Drug Liking After Oral Administration vs. Focalin XR At the Moment Drug Liking VAS 80 -=-SDX, 240 mg == Focalin XR, 80 mg 70 ~=-Placebo g Ca 2 £60 5 2 a a 8 40 Time (hours) Note: SDX = serdexmethylphenidate = KP415 Prodrug Drug Liking was assessed on a bipolar scale (0 =
Slide 19: KP415 Prodrug At-the-Moment Drug Liking After Oral Administration vs. Phentermine
KP415 Prodrug At-the-Moment Drug Liking After Oral Administration vs. Phentermine At the Moment Drug Liking VAS 80 -=- SDX, 240 mg -=-Phentermine, 60 mg 70 Placebo g ea 2 = 60 i 2 a 50 40 Time (hours) Note: SDX = serdexmethylphenidate = KP415 Prodrug Drug Liking was assessed on a bipolar scale (0 =
Slide 20: KP415 Prodrug Drug Liking Emax After Intranasal Administration
KP415 Prodrug Drug Liking E,,,, After Intranasal Admin- istration was Significantly Lower vs. D-Methylphenidate Maximum Drug Liking Score (E,,3x) 100 9 a2 == KP415 Prodrug, 80 mg, IN mmm d-MPH HCl, 40 mg, IN mm Placebo, IN - Neutral Score Drug Liking + significantly higher vs KP415 Prodrug by at lea
Slide 21: KP415 Prodrug At-the-Moment Drug Liking After Intranasal Administration
KP415 Prodrug At-the-Moment Drug Liking After Intranasal Administration vs. D-Methylphenidate At the Moment Drug Liking VAS 90 —=-SDX, 80 mg » —a—d-MPH HCI, 40 mg —s—Placebo 9 Sof Ne Neutral Score tz 2 a 360 a 50 j-#4eeHe--8L-22. 40 0 2 4 8 $ e ° Time (hours) Note: SDX = serdexmethylphenidate = KP41
Slide 22: KP415 Prodrug Drug Liking Emax After Intravenous Administration
KP415 Prodrug Drug Liking E,,,, After Intravenous Admin- istration was Significantly Lower vs. D-Methylphenidate Maximum Drug Liking Scores (E,,2x) 100 tt 90 84.3 =33KP415, 30 mg, IV mama d-MPH HCl, 15 mg, IV @amPlacebo cael Neutral Score Drug Liking * significantly non-inferior to placebo within at
Slide 23: KP415 Prodrug At-the-Moment Drug Liking After Intravenous Administration
KP415 Prodrug At-the-Moment Drug Liking After Intravenous Administration vs. D-Methylphenidate At the Moment Drug Liking VAS 80 —"—SDX, 30 mg —#—d-MPH HCl, 15mg N 3 —™-—-Placebo — ‘Neutral Score 2 8 Drug Liking VAS Pa 8 40 Time (hours) Note: SDX = serdexmethylphenidate = KP415 Prodrug Drug Liking wa
Slide 24: KP415 Prodrug Human Abuse Potential Trials Summary
KP415 Prodrug Human Abuse Potential Trials Summary * KP415 Prodrug HAP trials were required by FDA to help it determine a recommendation for controlled substance scheduling of the KP415 Prodrug * Statistically significant differences in key HAP endpoints were observed between KP415 Prodrug and activ
Slide 25: The Adult ADHD Market
The Adult ADHD Market + Over 4% of U.S. adults, or approximately 10.5 million adults have ADHD and are now the largest part of the ADHD market, comprising 53% of total TRx'2 * The adultADHD market has grown at ~11% per year on average vs. 4% for the pediatric ADHD market for the last several years?
Slide 26: KP484 Product Overview
KP484 Product Overview + Prodrug of d-MPH (serdexmethylphenidate) with extended release properties + Potential KP484 features and benefits eo Once-daily dosing o Longer duration than other super-extended release ADHD products o Active metabolism may offer more predictable therapeutic effect o Lower
Slide 27: KP484 Steady State PK vs. Mydayis
KP484 Steady State PK vs. Mydayis® KP484 vs Mydayis - AUC. 2, Normalized KP484 vs Mydayis - AUC. 2, Normalized 9 8 —e— KPA84 (steady state) 4 Mydayis (steady state) & & = = 5 5 8 8 § § 3 2 2 1 o o 0 2 4 6 8 1012 4 16 18 20 2 0 2 4 6 8 1012 4 16 18 20 2 Time (hours) Time (hours) Disclaimer: Mydayis s
Slide 28: Next Steps
Next Steps + KP415 NDA remains on track for submission in Q1 2019 *« KP484 NDA submission expected to follow later in 2019 « Strategic partnering discussions for KP415 and KP484 continue based on: o Significant, near-term ADHD prodrug commercial opportunities o Differentiated onset and duration effi
Slide 29: Apadaz: FDA Approved for the Short-Term Treatment of Acute Pain
Apadaz® FDA Approved for the Short-Term Treatment of Acute Pain
Slide 30: Apadaz Overview
Apadaz® Overview + First prodrug of hydrocodone (benzhydrocodone) combined with acetaminophen to be approved by the FDA + IR fixed-dose combination comprised of 6.67 mg benzhydrocodone HCI (equivalent to 7.5 mg hydrocodone bitartrate) and 325 mg APAP « Apadaz DEA product scheduling and quota allocat
Slide 31: Apadaz Commercialization Strategy
Apadaz® Commercialization Strategy KemPharm is pursuing two potential strategies for commercializing Apadaz. Neither strategy requires KemPharm to establish its own sales force. Non-traditional PBM Partnerships Traditional Pharma Partnership * Collaborative partnerships with + Partnership with a US-
Slide 32: KemPharm Expected Milestones
KemPharm Expected Milestones Product Event Date KP484 IND Filing 2017 v KP415 Initiate Pivotal Efficacy Study 2017 v Apadaz® FDA Approval 02/23/18 v KP415/ KP484 IV Human Abuse Potential (HAP) Data Q2 2018 v KP415 Pivotal Efficacy Study Results Mid-2018 ¥ KP415/ KP484 Oral HAP Data 2H 2018 v KP415/
Slide 33: Q2 2018 Financial Results
Q2 2018 Financial Results * Q2 2018 net loss of $10.0 million, or $0.65 per basic share and $0.91 per diluted share, vs. Q2 2017 net loss of $6.5 million, or $0.44 per basic and diluted share o Net loss for Q2 2018 was primarily due to loss from operations of $13.9 million and net interest expense a
Slide 34: For additional information please contact
K Pharm For additional information please contact: jdrumm@tiberend.com
Kempharm — Janney Healthcare Conference (September 2018) on 6ix