KemPharm Files IND for KP484 for the Treatment of ADHD, An Investigational Prodrug of Methylphenidate

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

CORALVILLE, Iowa, Sept. 20, 2017 -- KemPharm, Inc. (NASDAQ:KMPH), a clinical-stage specialty pharmaceutical company focused on the discovery and development of proprietary prodrugs,  announced today that it has filed an Investigational New Drug (IND) application with the U.S. Food and Drug Administration (FDA) to begin human clinical trials of KP484, the Company’s prodrug product candidate of “super-extended” release d-methylphenidate (d-MPH) for the treatment of attention deficit hyperactivity disorder (ADHD).

“In the process of re-analyzing the Phase 1 data for KP415, our co-lead investigation prodrug of d-MPH for the treatment of ADHD, we observed properties and a drug release profile in KP484 that we believe allows for the possible development of a much longer acting form of the molecule that, if approved, separate and apart from KP415, may enable us to target an underserved group of ADHD patients,” said Travis C. Mickle, Ph.D., President and Chief Executive Officer of KemPharm.  “We believe both KP484 and KP415 have features that could provide certain benefits when compared with other FDA-approved and widely prescribed methylphenidate products. Pharmacokinetic data from our studies 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.”

“Operationally designated KP484,” Dr. Mickle continued, “the data observed suggests that the prodrug may produce a longer duration release of d-MPH relative to comparator products available on the market today.  We believe this longer acting property – or “Super-Extended Release” – may be well-suited for the unique treatment needs of the adult ADHD population.” 

KemPharm’s IND proposal is to develop KP484 along a similar clinical trial pathway as KP415, with efficacy studies of KP484 initiating in 2018.  It is anticipated that data for KP484 will be leveraged from current and ongoing KP415 research, including pharmacokinetic and human abuse liability studies, which could allow an expedited development timeline towards a potential New Drug Application (NDA) submission with the FDA as early as 2019.  KemPharm believes that the ability to develop multiple prodrugs 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 LAT^TM (Ligand Activated Therapy) platform technology.  KemPharm utilizes its LAT^TM platform technology to generate improved prodrug versions of FDA-approved drugs in the high need areas of pain, ADHD 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, 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, the expected timing of the initiation and completion of clinical trials of KP415 and KP484, and the expected timing for any submission of NDAs with the FDA for KP415 and KP484. 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.