1. 1 1 November 2021 Management Presentation
25. 25 25 For the Treatment of Idiopathic Hypersomnia (IH) SDX Potential Product Candidate: KP1077
22. 22 22 Agonist Replacement Therapy for the Treatment of Stimulant Use Disorder (SUD) SDX Potential Product Candidate: KP879
28. 28 28 Financial Update As of September 30, 2021
9. 9 9 D - Methylphenidate Prodrug Product for the Treatment of ADHD AZSTARYS ®
4. 4 4 Experienced Management Team in Corporate and Drug Development Travis C. Mickle, PhD President and CEO R. LaDuane Clifton CFO Sven Guenther, PhD EVP of R&D Collective Team Experience
18. 18 18 First - and - Only C - IV Methylphenidate - Based Stimulant Serdexmethylphenidate (SDX)
32. 32 32 Leveraging our LAT ® Prodrug Technology to Create Long - Term Value For additional information please contact: Maureen McEnroe mmcenroe@tiberend.com
15. 15 15 Figure 2 : LS Mean Change in SKAMP - Combined Score from Baseline after Treatment with AZSTARYS or Placebo during Classroom Day in Pediatric Patients (6 to 12 years) with ADHD Section 14 AZSTARYS ® Label IMPORTANT SAFETY INFORMATION, Contraindications, Warnings and Precautions, Adverse Reactions and Drug Interactions may be found within the full Prescribing Information at www.kempharm.com/pipeline - products/#kp415
13. 13 13 Section 1: Indications and Usage • AZSTARYS is indicated for the treatment of Attention Deficit Hyperactivity Disorder (ADHD) in patients 6 years of age and older. Section 2.3: Administration Information • Administer AZSTARYS orally once daily in the morning with or without food. • AZSTARYS capsules may be taken whole, or opened and the entire contents sprinkled into 50 mL of water or over 2 tablespoons of applesauce. Sections 1 and 2.3 AZSTARYS ® Label IMPORTANT SAFETY INFORMATION, Contraindications, Warnings and Precautions, Adverse Reactions and Drug Interactions may be found within the full Prescribing Information at www.kempharm.com/pipeline - products/#kp415
30. 30 30 Uplisting to The Nasdaq Global Select Market • KemPharm’s common stock listing was moved up to The Nasdaq Global Select Market effective October 19, 2021 • Nasdaq Global Select Market recognized as having the highest initial listing standards of any exchange in the world o Consists of 1,450 stocks o Must meet Nasdaq's strict financial and liquidity requirements and corporate governance standards on both an initial and continuing basis “KemPharm’s advancement to The Nasdaq Global Select Market continues a year of tremendous growth and accomplishment for our company during which time we transformed our business, solidified our financial position and, most importantly with AZSTARYS ® , succeeded in bringing forth to market the first truly differentiated ADHD medication in years.” Travis C. Mickle, Ph.D., President and Chief Executive Officer of KemPharm.
3. 3 3 Specialty pharmaceutical company discovering and developing novel treatments for CNS diseases Two FDA approved and partnered medications, AZSTARYS ® and APADAZ ® , validate approach and science Leveraging LAT ® Platform Technology and extensive CNS expertise to create a pipeline of high - value opportunities KEMPHARM VALUE PROPOSITION
20. 20 20 Serdexmethylphenidate (SDX) Opportunity • SDX provides an opportunity to explore indications outside ADHD o SDX is the only C - IV methylphenidate - based product; all others are C - II o SDX has a unique PK profile allowing for gradual and continuous release throughout the day o No generic equivalent and not substitutable • Recently initiated a trial with SDX under the KP879 IND exploring the pharmacokinetics, safety and exploratory effects of SDX at doses above those studied with AZSTARYS (>240 mg) o Data release should be available prior to year - end 2021 • Goal is to assess the best potential clinical path with SDX in future drug development in such indications like Stimulant Use Disorder (SUD), Idiopathic Hypersomnia (IH) and others
19. 19 19 Serdexmethylphenidate (SDX) – Schedule IV Classification • SDX classified as a Schedule IV Controlled Substance by DEA o AZSTARYS TM classified as a Schedule II controlled substance as it includes a 70:30 mixture of SDX (Schedule IV) and d - MPH (Schedule II), respectively • SDX Schedule IV classification based on eight - factor analysis by HHS, which concluded that, “SDX is related in action and effect to the schedule IV substance phentermine, and can therefore be expected to have a similar potential for abuse.” • HHS also affirmed that, “in clinical studies, SDX demonstrated a lower potential for abuse when compared to d - MPH.” • SDX is the sole API in two other potential product candidates, KP879 and KP1077, that are currently under evaluation
29. 29 29 Q3 2021 Results; Balance Sheet Demonstrates Solid Financial Position • Revenue of $2.0M, derived primarily from consulting service fees • Net loss ($1.8M), or ($0.05) per basic and diluted share • Net operating loss was ($2.2M), which increased by $1.0M compared to Q3 2020 due to an increase in operating expenses o R&D expenses were $2.2M, an increase of $0.5M compared to Q3 2020 due to the ongoing SDX study o G&A expenses were $1.9M, an increase of $0.5M compared to Q3 2020 • Balance sheet details as of Sept. 30, 2021: o Cash and cash equivalents was $131.5M, a decrease of $0.8M compared to June 30, 2021 o 35,317,313 common shares outstanding; no preferred shares outstanding o 46,553,727 fully diluted common shares outstanding, which includes 4,252,600 shares issuable upon exercise of warrants • Strong balance sheet with a cash balance that provides for operating requirements, internal development opportunities, and other potential external investments (in - licensing, acquisition, partnerships, etc.)
10. 10 10 ADHD Market • ~$17.7 billion ADHD market in 2020 with prescription growth of ~1% year - over - year • The branded portion of the ADHD market was ~$7.6B in 2020 and more than 98% of these branded prescriptions are for extended release • Methylphenidate (MPH) accounted for approximately 19 million TRx’s and $4.5 billion in sales in 2020 • Early market research indicates prescribers see the following product benefits as key advantages: o Duration of action (60%) o Lower abuse potential (52%) o Early onset of action (43%) • Market research also indicates that prescribers estimate that MPH is given as the preferred first line of therapy for children under the age of 13 approximately 60% of the time Source: Symphony Health, Metys v5.2, 2021
17. 17 17 License Agreement with Commave (Affiliate of GPC) License agreement with Commave, an affiliate of GPC, was entered into Sept 2019 for AZSTARYS ® and KP484 • Commercial rights assigned to Corium, another affiliate of GPC, led by ex - Shire team o Perry Sternberg (CEO) and key commercial team members led Vyvanse commercial effort at Shire • Total potential regulatory and sales milestone payments including payments already made: $590M o $35M in regulatory milestones already paid o Sales milestones to be paid based on tiers • Royalty rates range from a percentage in the high single digits up to the mid - twenties for U.S. net sales • ROFR and ROFN for SDX - based products • ROFN for amphetamine - based prodrug products
21. 21 21 Clinical and Business Criteria will Guide Decisions on SDX Opportunities • Once available, KemPharm will analyze the data, along with other business criteria to determine how to best prioritize the SDX - based product candidates in order to maximize shareholder value • Criteria include: RISK COMMERCIAL OPPORTUNITY TIME, COST & NEED o Clinical risk o Development risk o Regulatory risk o Physician and KOL input/research o Payor research o Competitive landscape o Cost of development o Time to approval o Strategic considerations
24. 24 24 KP879 for the Treatment of SUD • Potential KP879 features and benefits: o Stand - alone formulation of serdexmethylphenidate (SDX) o Releases d - methylphenidate (d - MPH), a dopamine reuptake inhibitor (similar pharmacology as abused stimulants) o Very gradual onset of blood concentrations of released d - MPH followed by sustained release o Low oral, IN, and IV abuse potential • Focus of initial clinical studies o High dose PK o High dose safety o Effect size in different treatment populations • May qualify for FDA fast track, breakthrough therapy and/or priority review
16. 16 16 ASTARYS ® - U.S. Commercial Launch • July 2021, Corium, an affiliate of GPC, launched AZSTARYS (serdexmethylphenidate and dexmethylphenidate capsules) in the U.S. o 100 days’ post - launch, momentum building among payors, patients, providers and prescribers: § No push - back from prescribers or patients regarding the clinical profile § Repeat prescribing and refills o Corium estimates that more than 50 million Commercial and Medicaid lives now have payor access to AZSTARYS with more progress to come • AZSTARYS Commercial Launch is a Significant Milestone for KemPharm o Further demonstrates value potential of SDX and KemPharm’s LAT ® platform o License agreement with Commave, an affiliate of GPC, provides significant economic benefits to KemPharm tied to the commercialization of AZSTARYS o Based on the approved label for AZSTARYS, we believe peak market share may be greater than original internal forecast
23. 23 23 Stimulant Use Disorder (SUD) • Stimulant Use Disorder (SUD) is broadly defined as the abuse or misuse of cocaine, methamphetamine, or other stimulants • Although there are therapies for opioid addiction (buprenorphine, methadone), there are currently no approved treatments for SUD • Studies with agonist replacement therapies have shown promising data for treating SUD Stimulant Abuse/Misuse Reported In Last 30 Days Total Stimulant Abuse/Misuse 4,600,000 Cocaine a 1,900,000 Methamphetamine b 1,000,000 Rx Stimulants 1,700,000 Rx Pain Relievers (incl. opioids) 2,900,000 a includes crack cocaine; b includes only illicitly manufactured methamphetamine Source: Substance Abuse and Mental Health Services Administration. (2019). HHS Publication No. PEP19 - 5068, NSDUH Series H - 54. U.S. Prevalence of Abuse in 2018
26. 26 26 Idiopathic Hypersomnia (IH) • Rare disease with an estimated 37,000 US patients • There are no approved therapies for the treatment of Excessive Daytime Sleepiness (EDS) in IH • Narcolepsy treatments have commonly been used for treating IH symptoms o GHB/sodium oxybate for improved sleep; recent approval of XYWAV ® o Stimulants for Excessive Daytime Sleepiness (EDS) are used off - label § Methylphenidate remains the most commonly used stimulant for IH § Amphetamine § Modafinil • Unmet needs are focused on symptom control and safety o No 16 - hour product available for EDS symptom control o PRN (as needed) utilization of IR methylphenidate is very typical for “break - through” sleepiness o Potential for abuse remains a concern of most prescribers
27. 27 27 KP1077 Product Candidate Overview • Once - daily dosing • Potential features and benefits o Single dose allows for symptom control up to 16 hours o Still allows for PRN stimulant use for “break - through” sleepiness o C - IV designation • Orphan drug designation potential o Fast - track eligible o Break - through designation eligible • No generic equivalent; not substitutable • Composition - of - matter based patents expire in 2037; additional applications may extend past that time
31. 31 31 31 KemPharm: Looking Ahead AZSTARYS ® Solid Financial Position Beyond AZSTARYS SDX Opportunity ü Cash on hand as of Sept. 30, 2021 = $131.5M ü Uplisting to The Nasdaq Global Select provides access to larger pool of potential investors ü Potential of licensing revenue from AZSTARYS royalties and sales milestones in 2022 ü Selection of a lead SDX product development candidate as early as January 2022 ü Ongoing evaluation of non - SDX internal pipeline candidates and external opportunities ü Corium’s U.S. launch progressing as planned ü 100 days in, and AZSTARYS has payor access for over 50 million lives and growing ; momentum is building ü Data from SDX trial expected before year - end ü Multiple potential paths forward will be fully assessed based on commercial potential and development pathway
5. 5 5 Approved Drug with Sub - Optimal Properties Prodrug with Improved Attributes Cleaved Drug through Human Metabolic Process Ligand KemPharm Leverages its LAT ® Prodrug Technology to Improve the Attributes of Approved Drugs Select FDA - approved and widely prescribed drug for improvement, seek indications with few options or significant unmet need Chemically modify using a ligand to create a prodrug o Ligands – GRAS or demonstrated to be safe o Prodrugs generate composition - based patents Following ingestion, normal human metabolic processes cleave the ligand and release the active drug • Generates long - lived composition - of - matter patent protection • Proprietary to KemPharm 1 2 3
8. 8 8 8 KemPharm: Recent Highlights AZSTARYS ® Solid Financial Position Corporate Updates SDX Opportunity ü Cash on hand as of Sept 30, 2021 = $131.5M ü Q3 2021 net loss of $(1.8)M, or ($0.05) per basic and diluted share ü Uplisted to The Nasdaq Global Select Market; affirms strong financial/corporate framework ü Richard W. Pascoe appointed Executive Chairman ü Tamara A. Seymour named to Board of Directors ü Commercial launch on July 21, 2021; just 100 days into launch ü Payor access significantly improving; over 50 million lives now have access to AZSTARYS ü Multiple presentations for AZSTARYS and SDX at recent medical conferences ü Clinical trial with SDX underway; data expected prior to year - end 2021 ü Multiple potential paths forward to be fully assessed based on commercial potential and development pathway
11. 11 11 AZSTARYS ® Product Highlights • 70% prodrug of d - MPH (serdexmethylphenidate, or SDX) co - formulated with 30% immediate release d - MPH • AZSTARYS ® features and benefits o Indicated for the treatment of ADHD in patients 6 years of age and older o Can be administered with or without food o Capsule can be opened and sprinkled in applesauce or water o In a 12 - month study, no clinically significant changes in height or weight compared to normal growth o SDX is a Schedule IV compound; the first - and - only C - IV methylphenidate - based compound o LS mean change in SKAMP - C Score from baseline was different at all timepoints from 30 minutes to 13 hours post - dose for AZSTARYS vs. placebo • No generic equivalent product • Composition - based patent expires in 2037; NCE status granted; PTE and pediatric exclusivity possible as well IMPORTANT SAFETY INFORMATION, Contraindications, Warnings and Precautions, Adverse Reactions and Drug Interactions may be found within the full Prescribing Information at www.kempharm.com/pipeline - products/#kp415
7. 7 7 7 Evolution of the KemPharm Strategy Creates Risk Mitigated Value Product and Revenue Focused CNS Drug Development Expertise APADAZ Licensing and Partnerships AZSTARYS CNS Drug Development Expertise APADAZ LAT ® Platform Technology Tech Focused CNS Drug Development Expertise APADAZ Licensing and Partnerships AZSTARYS Innovative, Niche or Rare Disease Product Candidates License Revenue and High - Value Product Candidates Higher Value Product Candidates, Licensing Revenue and Potential to Commercialize LAT ® Platform Technology LAT ® Platform Technology LAT ® Platform Technology
6. 6 6 6 Candidate Indication Partner Status AZSTARYS ® Methylphenidate ER ADHD Gurnet Point Capital/Corium • Commercially available • Royalties/sales milestones expected to start in 2022 and beyond KP484 Methylphenidate ER ADHD Gurnet Point Capital/Corium • Licensed • Ready to enter clinical phase • Timing TBD with partner SDX - Candidates Methylphenidate Various (SUD, IH) Gurnet Point Capital/Corium • IND cleared for KP879 • Pre - clinical not needed, clinical underway 2021 • GPC has Right of First Negotiation after POC, then one - time ROFR up to NDA acceptance KP922 Amphetamine TBD Gurnet Point Capital/Corium • Timing TBD • GPC has Right of First Negotiation after POC, but no ROFR APADAZ ® Hydrocodone IR Pain KVK Tech • Licensed • Commercial launch Q4 2020 • Potential for $3.4M in m/s and reimb, plus profit share up to 50% Partnered Assets Create the Potential For Near and Long - Term Value
12. 12 12 Selected Extended - Release Stimulant Products for ADHD (1) Brand Name Active Ingredient Sponsor Dosage Form Dosage Strengths Features Reduced Drug Liking Vyvanse ® (lisdexamfetamine) Shire Capsule & Chewable 10, 20, 30, 40, 50, 60 & 70 (cap only) mg • Prodrug of d - amphetamine • Ages: 6 years and older • Onset: 1.5 hours • Duration: 13 hours • Indicated for binge eating disorder Some Difference Adderall XR ® (mixed salts of AMPH) Shire ER Capsule 5, 10, 15, 20, 25 & 30 mg • Comprised of both L - AMPH & D - AMPH • Ages: 6 years and older • Extended - release profile • No duration indicated No Focalin XR ® (dexmethylphenidate) Novartis ER Capsule 5, 10, 15, 20, 25, 30, 35 & 40 mg • Ages: 6 years and older • Onset: 0.5 hours • Duration: 12 hours No Concerta ® (methylphenidate) Janssen ER Ta b l e t 18, 27, 36 & 54 mg • Ages: 6 years to 65 years • Onset: 2 hours • Duration: 12 hours Small Difference (1) Information located within each respective package insert
14. 14 14 AZSTARYS ® Label Section 6.1: Clinical Trials Experience To adjust for normal growth, z - scores were derived (measured in standard deviations [SD]) ; z - scores normalize for the natural growth of children and adolescents by comparisons to age - and sex - matched population standards . A z - score change less than 0 . 5 SD is considered not clinically significant . In this study, the mean increase in weight from baseline to Month 12 was 3 . 4 kg among study completers . The mean change in z - score from baseline to Month 12 was - 0 . 20 , indicating a lower than expected increase in body weight compared to children of the same age and sex, on average . Most of the weight z - score decline occurred in the first 4 months of treatment . The mean increase in height from baseline to Month 12 was 4.9 cm among completers. Using the same z - score analysis for height, the mean change in z - score from baseline to Month 12 was - 0.21, indicating a lower than expected increase in height compared to pediatric patients of the same age and sex, on average. Section 6 IMPORTANT SAFETY INFORMATION, Contraindications, Warnings and Precautions, Adverse Reactions and Drug Interactions may be found within the full Prescribing Information at www.kempharm.com/pipeline - products/#kp415
2. 2 2 This presentation may contain forward - looking statements made in reliance upon the safe harbor provisions of Section 27 A of the Securities Act of 1933 , as amended, and Section 21 E of the Securities Exchange Act of 1934 , as amended . Forward - looking statements include all statements that do not relate solely to historical or current facts, including without limitation and can be identified by the use of words such as “may,” “will,” “expect,” “project,” “estimate,” “anticipate,” “plan,” “believe,” “potential,” “should,” “continue” or the negative versions of those words or other comparable words . Forward - looking statements are not guarantees of future actions or performance . These forward - looking statements include statements regarding the market outlook for AZSTARYS ® , potential regulatory and sales milestone and royalty payments pursuant to the License Agreement with an affiliate of Gurnet Point Capital, the potential benefits of AZSTARYS, the clinical development of KP 879 and KP 1077 , the potential benefits of SDX being classified as a Schedule IV controlled substance, and KemPharm’s forecasted cash runway . These forward - looking statements are based on information currently available to KemPharm and its current plans or expectations and are subject to a number of uncertainties and risks that could significantly affect current plans . Risks concerning KemPharm’s business are described in detail in the “Risk Factors” section of KemPharm’s Annual Report on Form 10 - K for the year ended December 31 , 2020 , KemPharm’s Quarterly Report for the quarter ended September 30 , 2021 , and KemPharm’s other filings with the Securities and Exchange Commission . KemPharm is under no obligation to, and expressly disclaims any such obligation to, update or alter its forward - looking statements, whether as a result of new information, future events or otherwise . This presentation also may contain estimates and other statistical data made by independent parties and by us relating to market size and other data about our industry . This data involves a number of assumptions and limitations, and you are cautioned not to give undue weight to such estimates . In addition, projections, assumptions and estimates of our future performance and the future performance of the markets in which we operate are necessarily subject to a high degree of uncertainty and risk . Cautionary Note Regarding Presentation Information
