KemPharm Announces Positive Topline Results from KP415 Pharmacokinetic Study in Children and Adolescents With ADHD

Results Suggest that Data from Single Classroom-style Efficacy Study May Be Applicable Across Pediatric, Adolescent and Adult ADHD Patient Populations

CORALVILLE, Iowa, March 20, 2018 (GLOBE NEWSWIRE) -- KemPharm, Inc. (NASDAQ:KMPH), a specialty pharmaceutical company focused on the discovery and development of proprietary prodrugs, today announced that topline results from its ongoing pediatric and adolescent pharmacokinetic (PK) study (KP415.105) suggest that following oral administration of its KP415 product candidate, exposure to d-methylphenidate is predictable in patients with attention-deficit/hyperactivity disorder (ADHD) between the ages of 6 and 17 years.

“Two of the benefits of d-methylphenidate (d-MPH) are, according to published literature, drug exposure from a specific product is comparable between age groups after adjusting for dose and body weight, and there is a direct correlation between that exposure and the clinical response,” said Travis Mickle, Ph.D., KemPharm President and Chief Executive Officer.  “These topline data announced today may permit the conduct of a single efficacy study in the established classroom ADHD trial design in pediatrics without resulting in a label indication that is limited to pediatric patients only.”

“This development adds to our excitement around KP415 as we anticipate additional clinical data throughout 2018, including topline results from the on-going pivotal efficacy study (KP415.E01)  by mid-year,” added Mickle. “While awaiting complete analysis, the preliminary results from our KP415.105 trial leave us confident that the data currently being collected in our product efficacy trial will also provide support for effectiveness in the adolescent and adult populations.”

About the KP415.105 Study
Study KP415.105 was single-dose, single-period, PK study with a KP415/d-methylphenidate (d-MPH) capsule in children 6-12 years and adolescents 13-17 years old with ADHD. Each subject received one of two dose levels:  28/6 mg KP415/d-MPH (equivalent to 20 mg total d-MPH) or 56/12 mg KP415/d-MPH (equivalent to 40 mg total d-MPH). PK data were available and analyzed from 30 subjects: 10 children 6-8 years old received the 28/6 mg KP415/d-MPH capsule; 10 children 9-12 years old received the 56/12 mg KP415/d-MPH capsule; 10 adolescents 13-17-year-old received either the 28/6 or 56/12 mg KP415/d-MPH capsule (5 per treatment group). Body weights ranged from 21 kg to 73 kg. Consistent with published studies of methylphenidate pharmacokinetics, exposure of d-MPH decreased with age due to body weight differences between younger children and adolescents based on topline data. When normalized by dose and body weight, d-MPH exposure and PK parameters were comparable between age groups.

About KP415 – KemPharm’s Prodrug Composition of d-MPH for ADHD
KP415 is KemPharm’s d-MPH prodrug composition product candidate designed for the broad treatment needs of the ADHD population.  KemPharm believes the prodrug may also demonstrate a lower abuse potential as well as less variability in the delivery of d-MPH compared to current methylphenidate products.

About KemPharm
KemPharm is a specialty pharmaceutical company focused on the discovery and development of proprietary prodrugs to treat serious medical conditions through its proprietary LAT^TM (Ligand Activated Therapy) platform technology.  KemPharm utilizes its proprietary LAT platform technology to generate improved prodrug versions of FDA-approved drugs in the high need areas of ADHD, pain and other central nervous system disorders. KemPharm’s co-lead clinical development candidates are KP415 and KP484, both based on a prodrug of methylphenidate, but with differing extended-release/effect profiles for the treatment of ADHD.  In addition, the company has received FDA approval for APADAZ™, an immediate-release combination product candidate of benzhydrocodone, a prodrug of hydrocodone, and acetaminophen.  The company is also advancing KP201/IR, an acetaminophen-free immediate-release formulation of the company’s benzhydrocodone prodrug candidate. Both APADAZ and KP201/IR are intended for the treatment of acute pain severe enough to require an opioid analgesic and for which alternative treatments are inadequate. For more information on KemPharm and its pipeline of prodrug product candidates visit www.kempharm.com.

Caution Concerning Forward Looking Statements:

This press release may contain forward-looking statements made in reliance upon the safe harbor provisions of Section 27A of the Securities Act of 1933, as amended, and Section 21E 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, 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 expected features and characteristics of KemPharm’s product candidates, including KP415, and any potential future clinical trial design for KP415, the ability of KemPharm to generalize the data from children in its product efficacy trial to the adolescent and adult populations and any potential indications for which KP415 may be approved. 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 KemPharm's Annual Report on Form 10-K for the year ended December 31, 2016, and KemPharm’s other Periodic and Current Reports filed 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.