Results from Single Classroom-Style Trial Met
Pre-Specified Primary and Secondary Endpoints
KemPharm, Inc. (NASDAQ:KMPH), a specialty pharmaceutical company
focused on the discovery and development of proprietary prodrugs,
today announced top line results from a pivotal efficacy and safety
clinical trial of KP415, its investigational
attention-deficit/hyperactivity disorder (ADHD) product candidate
that contains serdexmethylphenidate (a prodrug of
d-methylphenidate) and d-methylphenidate. Results from the trial
(KP415.E01) indicated that KP415 successfully met the primary
efficacy endpoint in patients with ADHD between the ages of 6 and
12 years.
The trial was a multicenter, randomized,
parallel, double-blind, placebo-controlled analog laboratory
classroom clinical trial in 150 children aged 6-12 years old with a
diagnosis of ADHD to assess the efficacy and safety of KP415.
Subjects who received KP415 met the trial’s primary and secondary
efficacy endpoint, showing statistically significant improvement on
both the Swanson, Kotkin, Agler, M-Flynn, and Pelham Scale (SKAMP)
and the Permanent Product Measure of Performance (PERMP) scale.
“We are pleased with these top line results from
our pivotal trial of KP415. The trial met its pre-specified
primary endpoint, which is the mean difference in the
SKAMP-Combined score change from baseline across all post-dose time
points,” said Travis Mickle, Ph.D., KemPharm President and Chief
Executive Officer. “Pre-specified secondary endpoints of
SKAMP-C change at each time point from the pre-specified,
pre-randomization baseline indicated a drug effect from 1 to
10 hours post-dose, and data from the PERMP, PERMP-Attempted
and PERMP-Correct all exhibited improvement over placebo from 0.5
hours to 13 hours post-dose. Lastly, KP415 was generally
well-tolerated with adverse events (AEs) typical of stimulant
therapy.”
“The SKAMP-C change from pre-randomization
baseline was selected to mitigate a potential carryover effect
related to the pharmacokinetics of KP415 and an assumption that
placebo should have been more predictable, neither of which
actually occurred,” added Mickle. “As a result, the endpoint
analysis led to an inability to correct for a change of SKAMP-C
scores in the placebo group across the analog classroom day.
When this bias is accounted for, a similar statistical analysis
(post-hoc) of SKAMP-C scores also supports efficacy of KP415 from
0.5 to 13 hours post-dose.”
“We anticipate developing additional clinical
data for KP415 throughout 2018, including the completion of our
ongoing oral and intranasal Human Abuse Potential studies,” Mickle
concluded. “We believe that completing the analysis of the
full data set from KP415.E01 trial and these other studies will
allow us to submit our New Drug Application for KP415 with the FDA
in the first quarter of 2019.”
Conference Call
InformationKemPharm will host a conference call and live
audio webcast with slide presentation today, July 9, 2018, at 8:00
a.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: 4594415
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 the KP415.E01 Trial
TREATMENT
The KP415 capsules contain two active
pharmaceutical ingredients: d-MPH hydrochloride as the immediate
release (IR) d-MPH component, and serdexmethylphenidate (SDX), a
prodrug of d-MPH that provides the extended duration component. In
terms of d-MPH equivalent amounts, all capsule strengths contain
30% of d-MPH (IR component) and 70% of d-MPH from SDX. Subjects in
this trial received daily oral doses (1 capsule/day) of either 28/6
mg SDX/d-MPH, 42/9 mg SDX/d-MPH, or 56/12 mg SDX/d-MPH,
corresponding to equivalent d-MPH amounts of 20, 30 and 40 mg,
respectively. The dose of KP415 was optimized during a 3-week dose
optimization phase with open-label KP415 capsules. After completion
of the dose optimization period, subjects were randomized to 7 days
of double-blind treatment with their optimized dose of KP415 or
matching placebo.
EFFICACY ENDPOINTS:
The efficacy evaluation was based on SKAMP and
PERMP scores pre-dose, and at 0.5, 1, 2, 4, 8, 10, 12 and 13 hours
post-dose during a full laboratory classroom day at Visit 6 (at the
end of the seven-day treatment period). The baseline SKAMP score
was measured pre-dose at Visit 5 (immediately prior to
randomization and the first dose in the seven-day treatment period)
due to potential concerns of carryover of methylphenidate into the
Visit 6 classroom day which would have disadvantaged KP415, as well
as an assumption that placebo would not significantly change from
Visit 5 to Visit 6. A post-hoc analysis was conducted using the
baseline SKAMP scores measured at pre-dose Visit 6. The SKAMP is a
validated rating of classroom behaviors in children with ADHD; the
PERMP is an adjusted math test designed to assess attention in
children with ADHD through a subject’s ability to initiate,
self-monitor, and complete the test.
The study met the primary endpoint of mean
SKAMP-C change from baseline score (pre-dose Visit 5 as baseline)
across all post-dose time points (Visit 6): The least-squares mean
post-dose SKAMP-C difference from baseline (150 subjects) was
higher for placebo than for KP415 (0.54 vs. -4.87, respectively;
p<0.001), indicating fewer ADHD symptoms with KP415 therapy than
with placebo.
Using pre-dose Visit 5 as baseline
(pre-specified), the SKAMP-C change from baseline was statistically
significantly better (p<0.001) with KP415 versus placebo from
1–10 hours post-dose; using pre-dose Visit 6 as baseline
(post-hoc), the SKAMP-C change from baseline was statistically
significantly better (p<0.01) with KP415 versus placebo from
0.5–13 hours post-dose.
The PERMP-A and PERMP-C scores (secondary
endpoints) support the primary endpoint conclusion overall (mean
changes; p<0.01) and showed statistically significantly better
performance with KP415 versus placebo at each time point from
0.5-13 hours (p<0.05).
SAFETY ENDPOINTS:
The observed AEs were mild to moderate in
severity and were typical of those seen in other stimulant trials.
No serious adverse events were reported.
KP415 – Prodrug Composition of d-MPH for
ADHDKP415 is KemPharm’s d-MPH prodrug composition product
candidate designed for the broad treatment needs of the ADHD
population. KemPharm believes the prodrug may also
demonstrate a lower abuse potential as well as less variability in
the delivery of d-MPH compared to current methylphenidate
products.
About KemPharmKemPharm is a
specialty pharmaceutical company focused on the discovery and
development of proprietary prodrugs to treat serious medical
conditions through its proprietary LATTM (Ligand Activated Therapy)
platform technology. KemPharm utilizes its proprietary LATTM
platform technology to generate improved prodrug versions of
FDA-approved drugs in the high need areas of ADHD, pain 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/effect
profiles for the treatment of ADHD. In addition, the company
has received FDA approval for Apadaz™, an immediate-release
combination product candidate of benzhydrocodone, a prodrug of
hydrocodone, and acetaminophen. The company is also advancing
KP201/IR, an acetaminophen-free immediate-release formulation of
the company’s benzhydrocodone prodrug candidate. Both Apadaz™ and
KP201/IR are intended for the treatment of acute pain severe enough
to require an opioid analgesic and for which alternative treatments
are inadequate. 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
StatementsThis 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
KemPharm’s product candidates, including KP415, as well as the
expected timing of the completion of any clinical trials or studies
related to KP415 and the expected timing of the NDA submission for
KP415. 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 quarter
ended March 31, 2018, filed with the Securities and Exchange
Commission on May 11, 2018, 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: |
Media Contact: |
Jason
Rando / Joshua Drumm, Ph.D.Tiberend Strategic Advisors,
Inc.212-375-2665 / 2664jrando@tiberend.comjdrumm@tiberend.com |
Daniel
L. CohenExecutive VP, Government and Public RelationsKemPharm,
Inc.202-329-1825dcohen@kempharm.com |
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