Zevra Therapeutics, Inc. · NASDAQ:ZVRA
Zevra Therapeutics — Kempharm — Canaccord Genuity 38th Annual Growth Conference (August 2018)
30 slides from Zevra Therapeutics's conference presentation, Aug 9, 2018
Slide 1: Canaccord Genuity 38th Annual Growth Conference
K Pharm Canaccord Genuity 38th Annual Growth 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 drugs in large markets + Building a pipeline of product candidates for ADHD, pain and CNS disorders + Potentially utilizing FDA
Slide 4: LAT (Ligand Activated Therapy) Platform Technology
LAT™ (Ligand Activated Therapy) Platform Technology ro) are ap ae ‘Approved Drug 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 c
Slide 5: KemPharm Product Pipeline
KemPharm Product Pipeline Potential Product Development| —_Next Category | candidate | Parent Drug Status Milestone NDA Submission Methylphenidate Oraland IN KP415 pl Clinical pgs 12019 ADHD Methylphenidate Initiation of Kp484 Has Clinical | eae Tal 2019 EEE Potential ‘Apadaz® Hyeecegone! FDAApprove
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 o Modification of pharmacokinetic p
Slide 7: Attention-Deficit/Hyperactivity Disorder: KP415 and KP484
Attention-Deficit/Hyperactivity Disorder: KP415 and KP484 for the Treatment of ADHD Mme =
Slide 8: The ADHD and ER Methylphenidate Market
The ADHD and ER Methylphenidate Market + ~$13 billion ADHD market with prescriptions growing at >5% year-over-year + The branded portion of the ADHD market was ~$6.4B in 2016 and more than 95% of these branded prescriptions are for extended release + Methylphenidate (MPH) accounted for approximately
Slide 9: 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 pred
Slide 10: KP415.E01 Efficacy Trial Overview
KP415.E01 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-week open-l
Slide 11: KP415.E01 Efficacy Trial Met Primary Endpoint
KP415.E01 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 Visit5 Ls Mean (SE) Difference in 95% Confidence LS mean (
Slide 12: Differences in SKAMP-C Change Between KP415 and Placebo
Differences in SKAMP-C Change Between KP415 and Placebo Are Statis' Time Predose 0.5 hours postdose 1 hours postdose 2hours postdose 4hours postdose Bhours postdose 10 hours postdose 12 hours postdose 13 hours postdose ‘ally Significant SKAMP-C Change at Visit 6 from Baseline Baseline = predose Visi
Slide 13: Differences in PERMP-A Change Between KP415 and Placebo
Differences in PERMP-A Change Between KP415 and Placebo Are Statistically Significant PERMP-A Change at Visit 6 from Baseline Baseline = predose Visit 5 | Baseline = predose Visit 6 (prespecified)” {post hoc)" Difference in Difference in LS mean (SE) LS mean (SE) Time KP415~Placebo p-Value KP415~Pla
Slide 14: Differences in PERMP-C Change Between KP415 and Placebo
Differences in PERMP-C Change Between KP415 and Placebo Are Statistically Significant PERMP-C Change at Visit 6 from Baseline Baseline = predose Visit 5 | Baseline = predose Visit 6 (prespecified)” {post hoc)" Difference in Difference in LS mean (SE) LS mean (SE) Time KP415~Placebo p-Value KP415~Pla
Slide 15: KP415.E01 Efficacy Trial Summary
KP415.E01 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 effect wi
Slide 16: Drug Liking VAS After IV Injection of KP415 Prodrug
Drug Liking VAS After IV Injection of KP415 Prodrug was Similar to Placebo At the Moment Drug Liking VAS Scores 85 —*-KP415 Prodrug 75 ed. MPH HCI 2 9 65 8 3 S55 45 36 Time (hours) Note: d-MPH HCI = dexmethyiphenidate hydrochloride Drug Liking was assessed on a bipolar scale (0 = Strong Disiking, 50
Slide 17: 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% year-over-year vs. 4% for the pediatric ADHD market for the last several years’ + Vyva
Slide 18: KP484 Product Overview
KP484 Product Overview + Prodrug of d-MPH (serdexmethylphenidate) with extended release properties + Potential KP484 features and benefits o Once-daily dosing o Longer duration than other super-extended release ADHD products o Active metabolism may offer more predictable therapeutic effect o Lower a
Slide 19: KP484 Steady State PK vs. Mydayis
KP484 Steady State PK vs. Mydayis* KP484 vs Mydayis - AUC... Normalized KP484 vs Mydayis - AUC, ., Normalized + Mydayis (steady state) —e- KPag4 (steady state) 7 lasma Concentration lasma Concentration 0 2 4 6 8 0 2 8 16 18 20 2 0 2 4 6 8 10 12 14 16 18 20 2 2 Time (hours) ‘Time (hours) Disclaimer.
Slide 20: 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 continue based on: o Significant, near-term ADHD prodrug commercial opportunity o Differentiated onset and duration efficacy profile
Slide 21: Apadaz: FDA Approved for the Short-Term Treatment of Acute Pain
Apadaz® FDA Approved for the Short-Term Treatment of Acute Pain — xyx_—————EET |
Slide 22: 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 23: Differences Observed in Drug Liking, Feeling High and Take Drug Again
Differences Observed in Drug Liking, Feeling High and Take Drug Again of IN and Oral Apadaz® vs. Norco (Study A02) LS Mean IN vs. Oral Parameter INNorco _ Oral Norco P-value Drug Liking Emax 79.0 771.9 0.6881 Feeling High Ema, 59.1 60.3) 0.8049 Take Drug Again 74.5 74.0 0.6835 LS Mean IN vs. Oral Pa
Slide 24: Oral and IN Drug Liking of Apadaz vs. Norco
Oral and IN Drug Liking of Apadaz® Apadaz™ vs. Norco (Study A02)! Norco Drug Liking: Oral vs Intranasal ‘Apadaz Drug Liking: Oral vs intranasal 100 100 <-0--2 Tabi - Sates ~-a--2Tablts- Sorted 0 2 Taba - Or “0 2 Tablets - ra Eo Ea Z z rag 8 n § H ary 2° so ‘0 Z 2 ot Pal . o 7 ws 2 . 0 4 2 Tine (ous)
Slide 25: Apadaz Label — Key Areas of Differentiation
Apadaz® Label — Key Areas of Differentiation Section 9.2 (Abuse) Intranasal Clinical Abuse Potential Study + Over the first 2 hours post-dosing (AUCp.95, AUCp.,, and AUC,.2), the cumulative hydrocodone exposure was lower following intranasal APADAZ compared to intranasal hydrocodone/acetaminophen +
Slide 26: Apadaz Label — Key Areas of Differentiation (cont'd)
Apadaz® Label — Key Areas of Differentiation (cont’d) Section 9.2 (Abuse) (cont’d) In Vitro Testing + The efficiency of extracting benzhydrocodone from APADAZ was similar compared to the efficiency of extracting hydrocodone from the non-abuse- deterrent hydrocodone/acetaminophen control. Further con
Slide 27: 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 28: KemPharm Expected Milestones
KemPharm Expected Milestones Product KP484 KP415 Apadaz® KP415/KP484 KP415 KP415/KP484 KP415/ KP484 KP484 KP415 KP484 KP484 [€—6WT PAD Event IND Filing Initiate Pivotal Efficacy Study FDA Approval IV Human Abuse Potential (HAP) Data Pivotal Efficacy Study Results Oral HAP Data IN HAP Data Initiate P
Slide 29: Q1 2018 Financial Update
Q1 2018 Financial Update + Q2 2018 results to be announced today, August 9, 2018, at 4:30pm ET * Total cash’ of $37.2 million as of March 31, 2018 o Cash decreased by $11.4M compared to December 31, 2017 * Q1 2018 net loss of $26.2 million, or $1.77 per basic and diluted share vs. Q1 2017 net loss o
Slide 30: For additional information please contact
K Pharm For additional information please contact: jdrumm@tiberend.com
Kempharm — Canaccord Genuity 38th Annual Growth Conference (August 2018) on 6ix