KemPharm, Inc. Announces Top-Line Results from Clinical Trial Evaluating the Safety and Pharmacokine
Tuesday, December 14, 2021
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KemPharm, Inc. Announces Top-Line Results
from Clinical Trial Evaluating the Safety and Pharmacokinetics of “Higher-Dose
SDX”Data reveal serdexmethylphenidate
(SDX) delivered at doses higher than those studied with AZSTARYS® is well-tolerated, yields dose-proportional
d-MPH exposure, and produces targeted biological effects
Celebration, FL – December 14, 2021 – KemPharm, Inc. (NASDAQ: KMPH), a specialty
pharmaceutical company focused on the discovery and development of proprietary
prodrugs, announced today top-line results from its clinical trial exploring
the safety and pharmacokinetics of serdexmethylphenidate (SDX) delivered at
doses higher than those studied as part of the AZSTARYS® development
program. AZSTARYS is a once-daily
product approved for the treatment of attention deficit hyperactivity disorder
(ADHD) in patients ages six years and older which is being commercialized in
the U.S. by Corium, Inc., a portfolio company of Gurnet Point Capital
(GPC). SDX is KemPharm’s proprietary
prodrug of d-methylphenidate (d-MPH) and the primary active pharmaceutical
ingredient in AZSTARYS. The U.S. Drug
Enforcement Agency (DEA) has classified SDX as a Schedule IV controlled
substance, which is a lower schedule than all other currently available
methylphenidate-based products. The dose-ascending Phase 1 clinical trial enrolled 14
subjects who were administered up to four increasing single oral doses of SDX,
each at least 14 days apart. Doses
ranged from 240 mg to 600 mg. Of those individual subjects administered more
than one dose of SDX, 10 received 360 mg, seven received 480 mg, and two
received 600 mg. Doses greater than 240
mg were above those studied under the AZSTARYS development program. Data from the study indicated that the 240 mg
and 360 mg doses of SDX were well-tolerated and produced d-MPH exposure
generally proportional to the dose.
Consistent with previous studies, after dosing d-MPH plasma concentrations demonstrated a gradual
increase followed by a slow decline resulting in prototypical broad d-MPH
exposure peak observed after oral administration of SDX. Additionally,
data suggested that the higher SDX doses produced targeted biological effects
that potentially align with the treatment of idiopathic hypersomnia (IH) and
other sleep disorders, as well as stimulant use disorder (SUD). Specifically, increased wakefulness,
alertness, excitability and insomnia effects were observed in the study. Modest increases in Drug Liking, which was
expected given the SDX’s status as a Schedule IV controlled substance, coupled
with the stable PK profile predicted at “steady-state,” are factors thought to
be predictive of a potentially successful maintenance therapy for SUD and
related disorders. Based on these results, KemPharm is assessing the
development programs, approval pathways and commercial potential of two product
candidates based on SDX, KP1077 for the treatment of IH, and KP879 for the
treatment of SUD, and expects to provide an update on its plans to expand its
pipeline in early Q1 2022. KemPharm is
also exploring other disease indications that may benefit from SDX-based
treatments. “The intent of this clinical trial was to determine if
higher doses of SDX could be administered safely and produce biological effects
consistent with the dosing and in alignment with disease indications that we
believe could benefit from the unique properties of SDX. In short, the results were exactly what we
were hoping to achieve, and we now expect to finalize our SDX development plan
and commercial value assessment in early 2022,” said Travis Mickle, Ph.D.,
President and Chief Executive Officer of KemPharm. “We believe the status of SDX as a Schedule
IV controlled substance, combined with the unique pharmacokinetic profile of
SDX, offers multiple treatment opportunities in a variety of disease
indications, which has the potential to be the basis for a portfolio of
SDX-based products.” About
AZSTARYS®: AZSTARYS is an FDA-approved, once-daily product for
the treatment of attention deficit hyperactivity disorder (ADHD) in patients
age six years or older. AZSTARYS consists of SDX, KemPharm’s prodrug of
d-methylphenidate (d-MPH), co-formulated with immediate release d-MPH. Corium, Inc., a portfolio company of Gurnet
Point Capital, is leading all commercialization efforts for AZSTARYS in the U.S. The complete approved prescribing information for
AZSTARYS may be downloaded in PDF format here: https://kempharm.com/wp-content/uploads/2021/03/AZSTARYS-Master-Label-Final_20210302.pdf About KemPharm: KemPharm is a specialty
pharmaceutical company focused on the discovery and development of proprietary
prodrugs to treat serious medical conditions through its proprietary LAT®
(Ligand Activated Therapy) technology.
KemPharm utilizes its proprietary LAT® technology to generate
improved prodrug versions of FDA-approved drugs as well as to generate prodrug
versions of existing compounds that may have applications for new disease
indications. KemPharm’s prodrug product
candidate pipeline is focused on the high need areas of attention deficit
hyperactivity disorder, or ADHD, stimulant use disorder (SUD) and CNS rare
diseases, including idiopathic hypersomnia (IH). In addition, the U.S. Food and Drug
Administration (FDA) has approved AZSTARYS®, a new once-daily
treatment for ADHD in patents age six years and older containing KemPharm’s
prodrug, serdexmethylphenidate (SDX), and APADAZ®, an
immediate-release combination product containing benzhydrocodone, KemPharm’s
prodrug of hydrocodone, and acetaminophen.
For more information on KemPharm and its pipeline of prodrug product
candidates visit www.kempharm.com or connect with us on Twitter, LinkedIn, Facebook and YouTube. 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.
Forward-looking statements are not guarantees of future actions or
performance. These forward-looking
statements, including the continued commercialization of AZSTARYS® and
the further development of KemPharm’s pipeline of product candidates, or the
suitability of SDX for any specific disease indication, 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.
Risks concerning KemPharm’s business are described in detail in
KemPharm’s Quarterly Report on Form 10-Q for the quarter ended September 30,
2021, and KemPharm’s other filings 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. KemPharm Contacts: Tiberend Strategic Advisors, Inc. Jason Rando/Maureen
McEnroe, CFA (212) 375-2665 / 2664 [email protected] [email protected]
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