KemPharm’s KP484 for the Treatment of ADHD, An Investigational Prodrug of Methylphenidate, Allowed to Proceed to Clinical Studies

KP484 Designed to be a Predictably Dosed, “Super-Extended” Release, Methylphenidate Product Candidate

CORALVILLE, Iowa, Nov. 02, 2017 -- KemPharm, Inc, a clinical-stage specialty pharmaceutical company focused on the discovery and development of proprietary prodrugs, announced today that the U.S. Food and Drug Administration (FDA) has completed its safety review of the Investigational New Drug (IND) application for KP484, the Company’s prodrug product candidate of “super-extended” release d-methylphenidate (d-MPH), and concluded that the company may proceed with its planned clinical investigation for the treatment of attention deficit hyperactivity disorder (ADHD).

“We are gratified that the FDA has allowed us to proceed to the clinic after completing their review of the IND application for KP484.  This developmental product candidate is one we believe allows for the possible development of a much longer acting methylphenidate product using our prodrug molecule that may enable us to target an underserved group of adult ADHD patients similar to the recently launched Mydayis^® product (mixed salts of a single entity amphetamine product), except for the methylphenidate treatment space,” said Travis C. Mickle, Ph.D., President and Chief Executive Officer of KemPharm.  

“We believe the features of KP484 could provide certain benefits when compared with other FDA-approved and widely prescribed methylphenidate products,” Mickle added.  “Pharmacokinetic (PK) data from preclinical studies conducted to date suggest that the time to maximum plasma concentration of our prodrug of methylphenidate after oral administration is approximately three times longer compared to immediate release methylphenidate, which may allow for convenient, once-daily dosing in the adult population.”

Submitted under Section 505(i) of the Federal Food, Drug, and Cosmetic Act, KemPharm’s IND proposes to develop KP484 along a similar clinical trial pathway as its current co-lead investigation prodrug of d-MPH for the treatment of ADHD, KP415.  Efficacy studies of KP484 are anticipated to be initiated in 2018.  Data for KP484 will be leveraged from current and ongoing KP415 research, including PK and human abuse liability studies, which could potentially allow an expedited development timeline towards a possible New Drug Application (NDA) submission with the FDA as early as 2019.  KemPharm believes that the ability to develop multiple prodrug products from the same Active Pharmaceutical Ingredient with unique treatment benefits and commercial profiles is the hallmark of its Ligand Activated Therapy (LAT™) prodrug discovery platform.

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 pain, attention deficit hyperactivity disorder 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 profiles for the treatment of ADHD.  In addition, the company is advancing Apadaz™, an immediate-release, abuse-deterrent hydrocodone/acetaminophen combination product candidate, and KP201/IR, an acetaminophen-free formulation of the company’s immediate release abuse deterrent hydrocodone product candidate, KP201.  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 KP415 and KP484, and the ability of KemPharm to meet its developmental milestones for KP415 and KP484, including any regulatory filings with the FDA related thereto. 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 period ended June 30, 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.