Slide 1: Management Presentation
K Pharm Management Presentation
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 « Utilizing FDA's 505(b)(2
Slide 4: Ligand Activated Therapy (LAT) Platform Technology
Ligand Activated Therapy (LAT) Platform Technology -@ a o~ aD a@i6 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 cr
Slide 5: KemPharm Prodrug Product Pipeline
KemPharm Prodrug Product Pipeline Phase1 Next Milestone Parent Drug | Feasibility Methylphenidate (ER) Proof-of Concept Data Apadaz™ Hydrocodone FDRR Submission ' KP201/IR Hydrocodone IND Submission : I PAIN KPS11/ER | Hydromorphone Phase Initiation KP6O6/IR Oxycodone Ae Development Preclinical KP74
Slide 6: ADHD: KP415
ADHD: KP415 For the Treatment of ADHD
Slide 7: KP415 Product Overview
KP415 Product Overview « Prodrug of methylphenidate « Potential features and benefits o Extended release methylphenidate = Time to maximum plasma concentration is approximately three times longer than IR methylphenidate o Active metabolism may offer more consistent drug delivery o Suitable for more
Slide 8: ADHD and ER Methylphenidate Market
ADHD and ER Methylphenidate Market >$15 billion ADHD market « ADHD market prescriptions are growing at ~5% year-over-year ¢ Methylphenidate accounted for approximately 19.7 million TRx’s and $4.2 billion in sales in 2015 « Many physician specialties have increased their prescribing of ADHD products
Slide 9: KP415 Oral PK Profile in Rats
KP415 Oral PK Profile in Rats Oral PK Curves 140 Sbetmax + MPH-HC! 100% 120 e-KPA15 283% 100 80 60 40 Methylphenidate Concentration {ngimL] 20 a 1 2 3 4 Time th] Note: MPH-HCI refers to methylphenidate hydrochloride. Studies conducted in rats. we ... .}§©= eee
Slide 10: KP415 Reduced Abuse Potential
KP415 Reduced Abuse Potential Intranasal PK Curves Intravenous PK Curves 2600 360 —eMPHHCL — —e-KPAI5 Se MPHHCL —e-KPA15 a ZF 30 = 2000 = 2 "3 260 = 1500 Foon 5 3 1.000 2 (0 3: 3 100 2 500 = = 3 50 3 = = a a 0 02 o4 O68 o8 1 0 05 1 18 2 Time [h] Time [h] %AUC —_%Cmae %AUC —_%Cmae KPAIS 7% 5% KPAIS 2
Slide 11: KP415 Clinical Update and Development Timeline
KP415 Clinical Update and Development Timeline Completion of a Pre-Investigational New Drug (Pre-IND) review of KP415 announced on July 14, 2016 « KP415 IND filing announced on September 19, 2016 « Human clinical trials of KP415 expected to begin in 4Q 2016 « Results from KP415 Phase 1 proof-of-conc
Slide 12: Pain: Apadaz End of Review Meeting Update
Pain: Apadaz™ End of Review Meeting Update
Slide 13: End of Review Meeting with FDA
End of Review Meeting with FDA « Primary goals for the End of Review meeting were to gain greater clarity into the issues identified by the FDA in the Apadaz NDA and to achieve a potential path forward for an Apadaz product label that could include abuse deterrence claims « Discussed fundamental que
Slide 14: Formal Dispute Resolution Request (FDRR)
Formal Dispute Resolution Request (FDRR) « The intent of a FDRR submission is to provide a pathway by which applicants seek to resolve scientific and/or medical policy disputes that cannot be resolved at the Division level within the FDA « If an issue is not resolved at the Division level, the appli
Slide 15: Pain: KP201/IR (APAP-free)
Pain: KP201/IR (APAP-free) For the Short-Term Treatment of Acute Pain
Slide 16: KP201/IR (APAP-free) Product Overview
KP201/IR (APAP-free) Product Overview « IR formulation of benzhydrocodone without APAP « Potentially the first IR hydrocodone-related product without APAP in the U.S. « An abuse-deterrent opioid that offers comparable efficacy to Vicodin, Norco and Lortab, but with the potential safety advantage of
Slide 17: KP201/IR Intranasal PK Trial
KP201/IR Intranasal PK Trial « KP201/IR (benzhydrocodone API) compared to hydrocodone bitartrate (HB) after intranasal administration for drug exposure levels, safety and drug likability in single-center, randomized, double-blind trial (n=51) « Significant differences for IN KP201/IR vs. IN HB: o Si
Slide 18: KP201 (API) Demonstrated Lower Hydrocodone Release IN Administration
of KP201 (API) Demonstrated Lower HC IN Administration Release 40 5 » | | Mean + SE 20 4 a Hydrocodone H } Concentration ! y KP201 (API) L 7 (ng/ml) 4g 1} y i 4 a 0 4 : 0 1 2 3 4 Time (hours) Study AO3: Intranasal HAP Study of APIs (N=24) me ... .}©§©§©=—h—eeeee
Slide 19: Differences in Drug Liking Over Time Mirrored PK Findings
Differences in Drug Liking Over Time Mirrored PK Findings 100 4 ~® Hydrocodone Bitartrate 90 J --KP201 (API) 80 4 Mean+sE 7° Drug Liking 60 7 50 40 T T T 1 0 0.5 1 15 2 Time (hours) Study AO3: Intranasal HAP Study of APIs (N=51) WR .}©= = —eestsi—i‘i—™OSOS
Slide 20: Differences Observed in Drug Liking Emax and Ease of Insufflation
Differences Observed in Drug Liking E,,,, and Ease of Insufflation of KP201 (API) vs. Hydrocodone Bitartrate Mean Hydrocodone Parameter KP201 (API) Bitartrate Drug Liking E,,2. 674 73.2 0.004 Ease of Insufflation 79 66 0.004 Study AO3: Intranasal HAP Study of APIs (N=51) me ti‘
Slide 21: KP201/IR Clinical Update and Development Timeline
KP201/IR Clinical Update and Development Timeline Completed KP201/IR End-of-Phase 1 (EOP1) meeting with the FDA on June 23, 2016 « KP201/IR IND filing expected in Q4 2016 « Human clinical trials of KP201/IR expected to begin in Q1 2017 « KP201/IR NDA expected to be filed in 2018 « Priority Review st
Slide 22: Pain: KP511/ER
Pain: KP511/ER For the Treatment of Severe Pain
Slide 23: KP511/ER Product Overview
KP511/ER Product Overview « KP511/ER is an ER formulation of KP511, a prodrug of hydromorphone « Demonstrated comparable hydromorphone exposure vs. equimolar dose of Dilaudid™ in oral human proof-of-concept trial « Potential valuable properties based on preclinical data o Significantly reduced IN an
Slide 24: Hydromorphone ER Market
Hydromorphone ER Market ¢ Nearly a $350 million dollar hydromorphone market in 2015 « Almost 3.2 million total prescriptions in 2015 « Prescription data suggests an increased writer base since a generic hydromorphone ER product was launched in Q2 2014 « Hydromorphone prescribers: o ~3,500 branded pr
Slide 25: Clinical Study KP511.101 — Oral PK in Humans
Clinical Study KP511.101 — Oral PK in Humans bad a o N a —=—KP511, 8mg —+— Dilaudid Oral Liquid, 4 mg a HM Concentration (ng/mL) N 0.5 ie) 0.5 1 1.5 2 25 3 3.5 4 Time (hours) Note: Dilaudid Oral Liquid is an oral solution of hydromorphone hydrochloride at a concentration of 1 mg/ml. KPS11,6 mg is th
Slide 26: KP511/ER Reduced Abuse Potential
KP511/ER Reduced Abuse Potential Intranasal PK Curves Intravenous PK Curves 1.200 —+-HM (compiled) 60 HM = = KPS11 (compiled) = KPa 2 1 p00 Z 00 e800 ~ 40 2 soo 2 30 & & = 400 22 = 20 = 10 = 2 o ——————————* a 02 o4 08 08 1 if 08 1 18 2 Time [h] Time [h] + 2.0 mgvkg (hydromorphane eq.) + 0.2 mg/kg (h
Slide 27: KP511/ER Potential Oral Overdose Protection
KP511/ER Potential Oral Overdose Protection 3600 3,000 = 2500 z = 2000 2 2 1500 mre Note: HM refers to hydromorphane hydrochloride Studies conducted in rats. -HM-HCI extrapolated HMC! HM) ——kP511 (HM) ‘concentaton tng sme Dose [mg/kg HM]
Slide 28: KP511/ER Clinical Update and Development Timeline
KP511/ER Clinical Update and Development Timeline « IND application accepted by the FDA in March 2016 and “Fast Track” designation granted in May 2016 « Positive results reported from Phase 1 proof-of-concept trial in June 2016 o Comparable hydromorphone exposure between 4 mg Dilaudid ™ Oral Liquid
Slide 29: CNS: KP303
CNS: KP303 For the Treatment of Schizophrenia and Other CNS Disorders
Slide 30: KP303 Product Overview
KP303 Product Overview ¢ Prodrug of quetiapine (Seroquel®) « Potential for utilization of the 505(b)(2) regulatory pathway *« Composition-based patent expires in 2030, and is potentially NCE eligible
Slide 31: KemPharm Expected News Flow
KemPharm Expected News Flow Product Even Date KP201/IR IND Filing 4Q 2016 KP415, Human POC Data 4Q 2016 KP511 (API) Intranasal PK and HAL Study Data 1Q 2017 KP415, Initiate Phase 3 Trials 2H 2017 KP201/IR Intranasal HAL Study Data 2H 2017 KP415 Phase 3 Trial Results 1H 2018 KP415, NDA Submission 201
Slide 32: Q2 2016 Financial Update
Q2 2016 Financial Update Total cash and cash equivalents, restricted cash, marketable securities and long-term investments of $102.6M as of June 30, 2016 o Represents a decrease of $8.4M from March 31, 2016 * Q2 2016 net income of $9.8M, or $0.59 per basic share, and ($0.58) net loss per diluted sha
Slide 33: For additional information please contact
K Pharm For additional information please contact: jdrumm@tiberend.com