KemPharm Initiates Pivotal Efficacy Trial of KP415

‘Classroom-style’ Pivotal Efficacy Trial of ADHD Prodrug Composition Candidate to Study Onset and Duration of Efficacy and Product Safety

Screening commenced in November; first patient treatment occurred December 20, 2017

CORALVILLE, Iowa, Jan. 08, 2018 (GLOBE NEWSWIRE) -- KemPharm, Inc. (NASDAQ:KMPH), a clinical-stage specialty pharmaceutical company engaged in the discovery and development of proprietary prodrugs, today reported the successful initiation of a “classroom-style” pivotal efficacy trial of KP415, the company’s extended release (ER) d-methylphenidate (d-MPH) prodrug composition product candidate for the treatment of Attention Deficit, Hyperactivity Disorder (ADHD).  The trial is designed to support a potential label for KP415 for both an early onset and long duration of a KP415 treatment effect in ADHD patients.

The trial is a multicenter, dose-optimized, double-blind, randomized, placebo-controlled, parallel efficacy laboratory classroom trial with children aged 6 to 12 years with a diagnosis of ADHD.  Screening for pediatric trial patients began on November 20, 2017.  Treatment with the study drug was initiated in the first patient on December 20, 2017.  Enrollment in the trial is expected to total approximately 140 patients across five clinical sites in the United States, and it is anticipated that all patients will have completed the trial by the end of second quarter of 2018.  Each patient will be treated for a total of four weeks.

“The start of the KP415 pivotal efficacy trial is a significant milestone in our effort to provide the first product with a methylphenidate prodrug to ADHD patients,” said Travis C. Mickle, Ph.D., President and Chief Executive Officer of KemPharm. “The trial is designed to collect evidence for a potential claim in the KP415 label indicating early onset and extended treatment effect, both of which are unmet by current methylphenidate products.”

“With this trial initiation, the KP415 developmental timeline for a possible NDA submission is estimated to be as early as the first quarter of 2019,” added Mickle.  “We expect to have topline data from this trial to share as soon as mid-2018, with a complete trial data set available prior to the end of the year.”

KP415 – Prodrug Composition of d-MPH for ADHD:

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

KP415 Trial Information:

Details of the study design can be found at https://clinicaltrials.gov/ct2/show/NCT03292952.

About KemPharm:

KemPharm is a clinical-stage 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^TM 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 product 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 is advancing Apadaz™, an immediate-release combination product candidate of benzhydrocodone, a prodrug of hydrocodone, and acetaminophen, as well as KP201/IR, an acetaminophen-free immediate-release formulation of the company’s benzhydrocodone. Both Apadaz™ and KP201/IR are designed to deter abuse and 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. These forward-looking statements include statements regarding the expected features and characteristics of KemPharm’s product candidates, including KP415, as well as the expected timing of the completion of the pivotal efficacy trial for KP415 and the expected timing of the NDA submission for KP415. These forward-looking statements are not guarantees of future actions or performance. 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. Actual results and performance could differ materially from those projected in the forward-looking statements as a result of many factors, including, without limitation, the risks and uncertainties associated with: KemPharm's financial resources and whether they will be sufficient to meet KemPharm's business objectives and operational requirements; results of earlier studies and trials may not be predictive of future clinical trial results; the protection and market exclusivity provided by KemPharm's intellectual property; risks related to the drug discovery and the regulatory approval process; the impact of competitive products and technological changes; and the FDA approval process under the Section 505(b)(2) regulatory pathway, including without limitation any timelines for related approval. KemPharm's forward-looking statements also involve assumptions that, if they prove incorrect, would cause its results to differ materially from those expressed or implied by such forward-looking statements. These and other risks concerning KemPharm’s business are described in additional detail in KemPharm's Quarterly Report on Form 10-Q for the quarter ended September 30, 2017, filed with the Securities and Exchange Commission on November 9, 2017, 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.