Pipeline & Products

KemPharm’s development pipeline is led by KP1077, which the company is developing as a treatment for idiopathic hypersomnia, a rare neurological sleep disorder. KemPharm also possesses a portfolio of two FDA-approved products, highlighted by AZSTARYS®, once-daily product for the treatment of attention deficit hyperactivity disorder (ADHD) in patients aged six years and older.

Selected KemPharm® Prodrug Product Candidates

Indication Product Candidate Phase of Development Anticipated Timing of Next Milestone
Rare Sleep Disorders
Idiopathic Hypersomnia (IH) KP1077 Phase 2 Year-end 2022
Narcolepsy Type I and II KP1077 Phase 3 Post-IH Trial
Preclinical Candidate TBD Lead Identification Q2 2022
First-in-Class Therapy
Stimulant Use Disorder (SUD) KP879 External Funding/Collaboration TBD
In-licensed or Acquired Product(s)
Niemann-Pick Disease Type C (NPC) Arimoclomol NDA Q3 2023

Approved KemPharm® Prodrug Products

Product Name Parent Drug Commercial Partner Key Milestone
Approved Products
APADAZ® Hydrocodone/APAP KVK Tech, Inc. Initial Adoption Milestone from KVK—2021
AZSTARYS® Methylphenidate (ER) Corium Inc. Royalties and Sales Milestones—2022

Stimulant Use Disorder

KemPharm is developing KP879 as an extended-duration, agonist replacement therapy for the treatment of Stimulant Use Disorder (SUD).  KP879 utilizes serdexmethylphenidate (SDX), KemPharm’s prodrug of d-MPH which is also the primary active pharmaceutical ingredient of KP415 and KP484, the company’s co-lead clinical development product candidates which are intended for the treatment of ADHD.

SUDs include those marked by abuse/misuse of cocaine, methamphetamines, prescription stimulant products that contain methylphenidate or amphetamine, and numerous designer stimulants including, for example, 3,4-methylenedioxypyrovalerone (MDPV) and 4-methylmethcathinone (mephedrone) (“bath salts”). According to the Substance Abuse and Mental Health Services Administration (SAMHSA), in 2016, among Americans older than 12 years, there were approximately 1.9 million current users of cocaine, 667,000 users of methamphetamine, and 1.7 million current misusers of prescription stimulants.  To date, there are no FDA-approved medications for the treatment of SUD.

Among the many potential drug therapies that have been studied for the treatment of SUD, medications with CNS stimulant-like properties have shown the most benefit. It is believed that by enhancing dopamine signaling in key brain areas, agonist replacement therapies may decrease craving and use of illicit stimulants, promote restorative sleep, and increase retention time of patients in substance abuse therapy. However, prior agonist replacement therapies have been limited in part by concerns of their own abuse potential among the stimulant-abusing population.

Based on information gathered during the KP415 and KP484 development programs, KemPharm believes that SDX has pharmacokinetic properties that are desirable as an agonist replacement therapy for the treatment of SUD. Specifically, KemPharm observed that SDX provided a gradual onset followed by long duration of d-MPH exposure, while also exhibiting the potential for a reduction in abuse-related effects. Given these properties, KemPharm is advancing KP879 as a potential first-ever therapeutic treatment for SUD.

Idiopathic Hypersomnia

KemPharm is advancing KP1077 as a potential first-ever therapeutic treatment for Idiopathic Hypersomnia (IH).  IH is an incapacitating neurological disorder marked by significant detrimental effects on nighttime sleep as well as daytime sleepiness/wakefulness.  IH is considered a rare disease (11/100,000), sometimes confused with narcolepsy due to the similarity in daytime symptoms.  There are currently no approved therapies for IH but narcolepsy treatments are commonly employed off-label to manage symptoms.  These off-label treatments include GHB, methylphenidate, amphetamine, and modafinil.  However, there remains significant unmet needs with currently available treatment options.

KemPharm has identified a prodrug of an undisclosed parent drug that it believes has significant potential for treating IH while also addressing unmet needs with currently available therapies that are employed off-label in this patient population.  As with other prodrugs developed by KemPharm, this product candidate has the potential for long-lived composition of matter patent protection.

Approved Products

APADAZ® (benzhydrocodone and acetaminophen) is a combination of benzhydrocodone, a prodrug of the opioid agonist hydrocodone, and acetaminophen that was approved by the U.S. Food and Drug Administration (FDA) on February 23, 2018.  KemPharm announced its entry into a definitive collaboration and license agreement with KVK Tech, Inc. for the U.S. commercial rights of APADAZ on October 30, 2018.  The commercial launch for APADAZ is planned for the second half of 2019.

Click here to learn more about Apadaz.

AZSTARYS® (serdexmethylphenidate and dexmethylphenidate capsules, for oral use, CII) is an FDA-approved, once-daily product for the treatment of attention deficit hyperactivity disorder (ADHD) in patients age six years or older.  AZSTARYS® consists of serdexmethylphenidate, KemPharm’s prodrug of d-methylphenidate (d-MPH), co-formulated with immediate release d-MPH.

The complete approved prescribing information for AZSTARYS® is provided below:

Full Prescribing Information