KP415 End-of-Phase 1 Meeting with FDA Affirms KemPharm’s Development Plan and Potential NDA Submission as
early as late 2018
Conference Call and Live Audio Webcast with Slide Presentation Scheduled for Today at 4:30 p.m. ET
CORALVILLE, Iowa, June 28, 2017 (GLOBE NEWSWIRE) -- KemPharm, Inc. (NASDAQ:KMPH), a clinical-stage specialty pharmaceutical
company focused on the discovery and development of proprietary prodrugs, today announced two significant advances involving its
prodrug development pipeline for the treatment of attention deficit hyperactivity disorder (ADHD). These advances include the
development of a new prodrug product candidate of d-threo-methylphenidate (d-MPH), KP484, for ADHD indications that may benefit
from a super-extended duration of treatment and the successful completion of an End-of-Phase 1 (EOP1) meeting with the U.S. Food
and Drug Administration (FDA) for KP415.
KP484 – Prodrug of d-MPH for Super-Extended Duration Treatment of ADHD
KP484 is KemPharm’s newly developed prodrug product candidate for a super-extended release (SER) d-MPH being designed for the
treatment of ADHD in patients that respond best when a long duration of therapy beyond both KP415 and all current methylphenidate
treatments is required. The new therapeutic application was developed during a data analysis of the KP415 Phase 1 study, in
which KemPharm observed that the prodrug molecule demonstrated an ability to produce a longer duration release of d-MPH relative to
comparator products available on the market today. As a result, KemPharm is now planning to initiate development of KP484 and
anticipates filing an Investigational New Drug (IND) application for KP484 as early as the third quarter of 2017. KemPharm
expects to leverage data from certain clinical and nonclinical trials of KP415 to expedite the development of KP484. KemPharm
believes that this may enable it to realize key cost and R&D efficiencies and target a New Drug Application (NDA) submission as
soon as 2019.
KP415 – Prodrug of d-MPH for Treatment of ADHD with Early On-set and Better Total Duration
KP415 is KemPharm’s extended release (ER) d-MPH prodrug product candidate designed for the treatment of ADHD with patients that
could benefit from both earlier onset as well as better total duration of effect. As previously indicated, KemPharm held an
EOP1 meeting with the FDA to discuss the data from the Phase 1 proof-of-concept clinical trial of KP415 (KP415.101), additional
nonclinical and manufacturing data sets, and the proposed clinical and nonclinical programs required for eventual submission of an
NDA for KP415. Additionally, KemPharm and the FDA discussed the proposed commercial formulation of KP415, which KemPharm
plans to develop with a layer of methylphenidate to potentially support a superior early onset profile. Based on the feedback
from the FDA, KemPharm believes that its ongoing and anticipated research of KP415, including the pivotal efficacy trial, which
KemPharm plans to initiate in the second half of 2017, remains on schedule and in alignment with an NDA submission as soon as late
2018. KemPharm also anticipates that it will initiate a human abuse liability (HAL) program of KP415 beginning in the second
half of 2017 to assess the potential for the prodrug to deter intranasal, intravenous and oral abuse. KemPharm expects data
from the HAL program and the ongoing pharmacokinetic (PK) studies will be available in the second half of 2017 and early
2018. KemPharm plans to utilize the data from both studies in the development KP484, as well.
“We now believe KemPharm’s ADHD prodrug portfolio is the company’s most valuable asset. In our view, the advances that we have
made with KP415 and our newly developed product candidate for a super extended treatment, KP484, further support this belief,”
stated Travis C. Mickle, Ph.D., President and Chief Executive Officer of KemPharm. “We continue to view KP415 as our highest
value product candidate, and we are excited to now take this prodrug to the next phase of its development. Since our quarterly
update in May, we have initiated several pharmacokinetic studies of KP415 with data from these studies expected during the second
half of 2017 and in early 2018. Now, following the End-of-Phase 1 meeting with the FDA, we are prepared to develop the commercial
formulation of KP415 and initiate the pivotal efficacy study and human abuse liability program for KP415 in the second half of
2017; all pointing towards an NDA submission for KP415 as soon as late 2018.”
“The success of our KP415 program is now heightened by the development of a new product we are designating as KP484, which we
are planning to develop for patients that require a product that provides a much longer duration of treatment for their ADHD,” Dr.
Mickle continued. “In contrast to Shire’s recently approved amphetamine-based MYDAYISTM for extended duration of
treatment for ADHD, initial clinical data on KP484 suggest our new methylphenidate product could offer substantially longer
duration when compared to other methylphenidate ADHD drugs which is clearly an unmet need in treatment with methylphenidate. Given
this, we have now activated the development program for KP484 with our first milestone being an IND filing in the third
quarter.”
“We believe physicians need products, such as KP415 and KP484, which are designed to provide solutions for their patients’
individual needs, recognizing that many may respond differently to different treatments. Thus, it is important for patients
to have multiple treatment options,” Dr. Mickle concluded. “For example, some patients need milder therapies such as a
methylphenidate-based option that may be just as effective and have fewer side effects when compared to amphetamine-based
products.”
Conference Call Information:
The company will host a conference call and live audio webcast with slide presentation today, June 28, 2017, at
4:30 p.m. ET. Interested participants and investors may access the conference call by dialing either:
- (866) 395-2480 (U.S.)
- (678) 509-7538 (international)
- Conference ID: 45888929
The live webcast with accompanying slides will be accessible via the Investor Relations section of the KemPharm
website http://investors.kempharm.com/. An archive of the webcast and presentation will remain
available following the call.
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 LATTM (Ligand Activated Therapy) platform technology. KemPharm
utilizes its LATTM 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, an
extended-release prodrug of methylphenidate 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, and the anticipated timelines for any IND or NDA submission or the
availability of clinical trial results. 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; obligations
to third parties regarding the potential commercialization or sale of KP415 or KP484; and the FDA approval process, 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 March 31, 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.
Investor Contacts: Jason Rando / Joshua Drumm, Ph.D. Tiberend Strategic Advisors, Inc. 212-375-2665 / 2664 jrando@tiberend.com jdrumm@tiberend.com Media Contact: Daniel L. Cohen Executive VP, Government and Public Relations KemPharm, Inc. 202-329-1825 dcohen@kempharm.com
