KemPharm Receives Clearance from FDA to Initiate Clinical Program for KP415, an Investigational Prodrug of D-Threo-Methylphenidate for the Treatment of ADHD

CORALVILLE, Iowa, Oct. 11, 2016 -- KemPharm, Inc. (NASDAQ:KMPH), a clinical-stage specialty pharmaceutical company focused on the discovery and development of proprietary prodrugs, today announced that its Investigational New Drug (IND) application for KP415 has been accepted by the U.S. Food and Drug Administration (FDA). KP415, KemPharm’s co-lead product candidate, is an extended release (ER) d-threo-methylphenidate (d-MPH) prodrug for the treatment of attention deficit hyperactivity disorder (ADHD).  KemPharm expects to commence and complete proof of concept human trials prior to the end of 2016, with additional human clinical trials initiating during the first half of 2017.

“We are very pleased that the FDA accepted our IND request for KP415, our co-lead product candidate, and expect to advance this program through proof-of-concept before year-end and into human clinical trials,” stated Travis C. Mickle, Ph.D., President and Chief Executive Officer of KemPharm. “Based on our estimates, in 2015, methylphenidate accounted for approximately 19.7 million prescriptions and $4.2 billion in sales in the ADHD market.” 

“However, patent expiration in the ADHD space is eroding the revenues of branded products,” Mickle continued.  “We believe KP415 offers the opportunity to address this market dynamic with a prodrug technology that, in pre-clinical studies, has demonstrated both extended release properties and a pharmacokinetic profile that could enable a more predictable therapy compared with currently available, immediate-release d-MPH products.”

KemPharm anticipates submitting a New Drug Application (NDA) submission for KP415 as early as 2018. In preclinical studies of KP415, KemPharm observed features that it believes could provide significant benefits when compared with other FDA-approved and widely prescribed methylphenidate products. Pharmacokinetic data from those preclinical studies suggest that the time to maximum plasma concentration of methylphenidate after oral administration of KP415 is approximately three times longer compared to immediate release methylphenidate.  As a prodrug that requires metabolism, KP415 may be able to deliver d-MPH more consistently than the current formulation based approaches. This improved delivery may provide more consistent therapy.

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 Ligand-Activated Therapy (LAT) platform technology.  KemPharm utilizes its LAT platform technology to generate improved prodrug versions of FDA-approved drugs in the high need areas of pain, ADHD and other CNS disorders. 

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 KP415, the expected development timeline of KP415, and potential submission of an NDA 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 June 30, 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.

Investor Contacts:	
Jason Rando / Joshua Drumm, Ph.D.
Tiberend Strategic Advisors, Inc.
212-375-2665 / 2664
[email protected]
[email protected]

Media Contact:
Daniel L. Cohen
Executive VP, Government and Public Relations
KemPharm, Inc.
202-329-1825
[email protected]