KemPharm Receives Clearance from FDA to Initiate Human Clinical Trials of KP511, an Investigational Prodrug of Hydromorphone

CORALVILLE, Iowa, March 28, 2016 (GLOBE NEWSWIRE) -- KemPharm, Inc. (NASDAQ:KMPH) announced today that its Investigational New Drug (IND) application for KP511, KemPharm’s prodrug of hydromorphone, has been accepted by the U.S. Food and Drug Administration (FDA). KP511 is being developed as an abuse-deterrent, extended-release (ER) prodrug for the management of pain severe enough to require daily, around-the-clock, long-term opioid treatment and for which alternative treatments are inadequate (KP511/ER). KemPharm expects to initiate human clinical trials of KP511 in the second quarter of 2016.

Travis Mickle, Ph.D., President and Chief Executive Officer at KemPharm, said, “We are very pleased that the FDA accepted our IND for KP511 a full week earlier than the required 30 day review cycle and we look forward to initiating human clinical trials of KP511 in the second quarter of 2016. In particular, as part of the clinical trial process, we will investigate whether the molecular structure of KP511 may reduce the risk of oral overdose by a mechanism that is inherent in the prodrug molecule itself.”

KemPharm plans to seek approval of KP511/ER under Section 505(b)(2) of the Federal Food, Drug and Cosmetic Act with an anticipated New Drug Application (NDA) submission as early as 2018. The anticipated development program for KP511 is designed to assess the product candidate’s tamper and extraction resistance, intranasal and intravenous abuse potential, as well as the potential to limit oral abuse and/or overdose. KemPharm believes that the potential to reduce the risk of oral overdose may be possible based on the prodrug overwhelming the metabolic pathways required to convert it to hydromorphone at excessive oral doses. Additionally, KemPharm intends to investigate KP511’s potential to improve or reduce opioid-induced constipation, a common side effect of opioid therapy.

KP511 utilizes KemPharm’s Ligand Activated Therapy (LAT) platform technology to create potential abuse-deterrent properties at the molecular level. KemPharm believes that this proprietary design offers the potential to restrict the release of the active hydromorphone component until the prodrug is metabolized following oral administration. KP511/ER is designed to be bioequivalent to currently available ER hydromorphone products.

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 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 KP511 and the timelines surrounding potential clinical trials for KP511 and the submission of an NDA for KP511/ER. 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 Annual Report on Form 10-K for the fiscal year ended December 31, 2015, 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 Media Contact: Jim Heins Cooney Waters Unlimited 212-886-2221