Data demonstrate higher doses of SDX were
generally well-tolerated and produced targeted pharmacodynamic effects
that may be beneficial for the treatment of idiopathic hypersomnia (IH)
and other sleep disorders
Upcoming
anticipated development milestones for KP1077 include: IND filing as
early as Q2 2022; Phase 2 trial initiation in IH patients and announce
results of study to examine safety related to cardiovascular effects
expected in 2H 2022
CELEBRATION, Fla.,
March 21, 2022 (GLOBE NEWSWIRE) -- KemPharm, Inc. (NasdaqGS: KMPH)
(the Company or KemPharm), a specialty pharmaceutical company focused on
the discovery and development of proprietary prodrugs, today announced
that the Company has completed its analysis of the full data set from
its Phase 1 clinical trial exploring the safety and pharmacokinetics
(PK) of serdexmethylphenidate (SDX) delivered at doses higher than those
previously studied. SDX, KemPharm’s proprietary prodrug of
d-methylphenidate (d-MPH), is the sole active pharmaceutical ingredient
(API) in KP1077, which KemPharm is developing as a treatment for
idiopathic hypersomnia (IH).
The full data set,
which builds upon top-line data previously reported on December 14,
2021, affirmed that 240 mg and 360 mg doses of SDX were well-tolerated
and produced d-MPH exposure that appeared to increase proportionally
with dose. Mean d-MPH plasma concentrations demonstrated a gradual
increase after SDX administration, reaching a broad peak from eight to
twelve hours post-dose, followed by a shallow decline thereafter.
Increased wakefulness, alertness, hypervigilance, and insomnia effects
were reported by study participants, which suggests that SDX produced
targeted pharmacodynamic effects that could benefit patients with IH and
other sleep disorders.
“The full data set from
the Phase 1 clinical trial of ‘higher-dose’ SDX affirms our decision to
select KP1077 as a treatment for IH as our lead development candidate
and informs further development of SDX in related disorders,” said
Travis C. Mickle, Ph.D., President and CEO of KemPharm. “IH is a rare
neurological sleep disorder characterized by excessive daytime
sleepiness, extreme difficulty waking, and severe brain fog, despite
adequate or even prolonged nighttime sleep. Unfortunately, the treatment
options currently available only address a limited range of these
symptoms and are known to cause adverse events. The data generated in
the Phase 1 trial of higher-dose SDX suggest that KP1077 is
well-tolerated and possesses unique properties that could potentially
address excessive daytime sleepiness as well as other debilitating
symptoms associated with IH.”
KemPharm also
provided an update on several important upcoming milestones for the
KP1077 development program. The Company anticipates filing an
Investigational New Drug (IND) application for KP1077 as early as the
second quarter of 2022 and subsequently initiating a Phase 2 trial in
patients with IH in the second half of 2022. Additionally, we expect to
receive results from an additional trial to assess the relative
cardiovascular safety of SDX vs. current stimulant treatments also in
the second half of 2022.
About the Higher-Dose SDX Phase 1 Clinical Trial:
The
Higher-Dose SDX Phase 1 clinical trial was a dose-escalation study to
determine the PK, pharmacodynamic stimulant effects, and safety of
single oral doses of SDX in subjects with a history of high-dose
stimulant use. Following screening, subjects were treated with single,
ascending doses of SDX (240, 360, 480, and 600 mg), with each dose
separated by a minimum of 14 days. A total of 14 subjects received at
least one dose of SDX with 14, 10, 7 and 2 subjects dosed with 240, 360,
480, and 600 mg, respectively.
Subject-rated
pharmacodynamic effects of Drowsiness/Alertness and Energized were
scored on a Visual Analogue Scale (VAS) at several timepoints post-dose.
The results indicate that the VAS scores for Drowsiness/Alertness
(bipolar scale: 0 to 100) increased with dose with maximum effects in
the early to late afternoon, with the most pronounced effects measured
at the two highest doses. Similarly, mean VAS scores of feeling
Energized (unipolar scale: 0 to 100) increased from mean baseline scores
of less than 20 and up to a peak range of 61 to 82, across all dose
levels, with the largest effects measured at the two highest doses. Both
Alertness and feeling Energized are relevant pharmacodynamic metrics
for treating patients with sleep disorders like IH.
The following table summarizes the trial results for the relevant pharmacodynamic metrics:
Drowsiness/Alertness VAS1
(bipolar scale: 0 to 100)
Baseline Score Range:
37 to 50
Peak Score Range:
67 to 89
Energized VAS2
(unipolar scale: 0 to 100)
Baseline Score Range:
15 to 20
Peak Score Range:
61 to 82
1The Drowsiness/Alertness VAS is an at-the-moment bipolar scale where a score of 50 is neither drowsy nor alert, a score below 50 is drowsy, and a score above 50 is alert. 2The Energized VAS is an at-the-moment unipolar scale measuring the feeling of excess energy where a score of 0 is “definitely no” and a score of 100 is “definitely so”
The
full data and detailed results are expected to be submitted for
presentation at future scientific conferences and publication in
peer-reviewed journals.
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) platform technology. KemPharm utilizes its proprietary LAT®
platform 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 idiopathic hypersomnia (IH) and other CNS/rare diseases.
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), which is being commercialized by Corium,
Inc. in the U.S., and APADAZ®, an immediate-release
combination product containing benzhydrocodone, KemPharm’s prodrug of
hydrocodone, and acetaminophen, which is being commercialized by
KVK-Tech, Inc. in the U.S. 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 within the meaning
of the Private Securities Litigation Reform Act of 1995.
Forward-looking statements include all statements that do not relate
solely to historical or current facts, including without limitation and
which can be identified by the use of words such as “may,” “will,”
“expect,” “project,” “estimate,” “anticipate,” “plan,” “believe,”
“potential,” “should,” “continue,” “could,” “intend,” “target,”
“predict,” or the negative versions of those words or other comparable
words or expressions, although not all forward-looking statements
contain these identifying words or expressions. Forward-looking
statements are not guarantees of future actions or performance. These
forward-looking statements include statements regarding the potential
benefits of KP1077, the promise and potential impact of our preclinical
or clinical trial data, including without limitation the timing and
results of any clinical trials or readouts, the timing or results of any
IND applications, the potential benefits of KP1077, SDX or any other
product candidates for any specific disease indication, or the potential
benefits of any of KemPharm’s product candidates, the submission of
data for publication at scientific conferences or publication in
journals, and our strategic and product development objectives. 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 known and unknown uncertainties, risks and other important
factors that may cause our actual results, performance or achievements
to be materially different from any future results, performance or
achievements expressed or implied by the forward-looking statements.
These and other important factors are described in detail in the “Risk
Factors” section of KemPharm’s Annual Report on Form 10-K for the year
ended December 31, 2020, KemPharm’s Quarterly Report for the quarter
ended September 30, 2021, and KemPharm’s other filings with the
Securities and Exchange Commission.
While we may
elect to update such forward-looking statements at some point in the
future, except as required by law, we disclaim any obligation to do so,
even if subsequent events cause our views to change. Although we believe
the expectations reflected in such forward-looking statements are
reasonable, we can give no assurance that such expectations will prove
to be correct. These forward-looking statements should not be relied
upon as representing our views as of any date subsequent to the date of
this press release.
This press release also may
contain estimates and other statistical data made by independent parties
and by us relating to market size and other data about our industry.
This data involves a number of assumptions and limitations, and you are
cautioned not to give undue weight to such estimates. In addition,
projections, assumptions and estimates of our future performance and the
future performance of the markets in which we operate are necessarily
subject to a high degree of uncertainty and risk.
BioFlorida is the voice of Florida's life sciences industry, representing 8,600 establishments and research organizations in the BioPharma, MedTech, Digital Health and Health Systems that collectively employ nearly 107,000 Floridians. Source: TEConomy/BIO (released 2022)
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