Supernus Announces Promising Interim Data from Ongoing Open-Label Phase 2a Study of SPN-817 in Epilepsy
May 23 2024 - 4:05PM
Supernus Pharmaceuticals, Inc. (Nasdaq: SUPN), a biopharmaceutical
company focused on developing and commercializing products for the
treatment of central nervous system (CNS) diseases, today announced
data from the planned interim analysis of the exploratory
open-label Phase 2a clinical study of SPN-817 for
treatment-resistant seizures. The study is examining the safety and
tolerability of SPN-817 as adjunctive therapy in adult patients
with treatment-resistant seizures, as well as finding effective
doses in various treatment-resistant seizure types. The interim
analysis is as of May 1, 2024, and is based on 41 enrolled
subjects, of which 19 completed the maintenance period. Of these 19
subjects, 16 subjects had focal seizures.
Summary of the Interim Data
- 75% median focal
seizure reduction at the 3mg to 4mg twice daily doses in the
maintenance period.
- 86% median focal
seizure reduction at the 3mg to 4mg twice daily doses in the open
label extension period.
- Responder
analysis across all doses in maintenance period:
- 81% of subjects
with focal seizures had 30% or more seizure reduction.
- 63% of subjects
with focal seizures had 50% or more seizure reduction.
- 19% of subjects
with focal seizures had 75% or more seizure reduction.
- Median focal
seizure reduction with more severe subjects (greater than 11.3 mean
baseline number of seizures per 28-day period):
- 74% in the
maintenance period.
- 86% in the open
label extension period.
- Median seizure
reduction in subjects with three or more other anti-seizure
medications (ASMs):
- 70% in the
maintenance period.
- 60% in the
open-label extension period.
- Responder
analysis in subjects across all doses with three or more ASMs in
maintenance period:
- 100% of subjects
with focal seizures had 30% or more seizure reduction.
- 82% of subjects
with focal seizures had 50% or more seizure reduction.
- 27% of subjects
with focal seizures had 75% or more seizure reduction.
- Overall focal
seizure reduction (all doses, 1mg to 4mg twice daily):
- 58% median
seizure reduction in the maintenance period.
- 38% median
seizure reduction in the open label extension period.
- Assessment by
Epitrack®, a validated cognitive screening tool that is designed
for patients with epilepsy, indicated that 83% of twelve subjects
from whom data are available, was equally split into those who
showed improvement and those who had no change in cognitive
function.
- SPN-817 was safe
and had acceptable tolerability with a discontinuation rate due to
adverse events (AEs) of 22% in the titration period and 2.4% in the
maintenance period.
- Most common AEs
related to the drug were consistent with the known profile of
acetylcholinesterase inhibitors and included nausea, diarrhea,
headache, dizziness, and decreased appetite. Additional AEs such as
fatigue, insomnia, vomiting, blurred vision, somnolence, and
irritability were observed.
“This planned interim analysis of our Phase 2a
clinical study provides early, yet what seems to be strong
evidence, of the clinical utility of SPN-817 in epilepsy. In
addition, the data provide important insights for the design of the
upcoming Phase 2b clinical study that we plan on initiating before
year end 2024,” said Jack Khattar, President and CEO of Supernus.
“We will continue to analyze the valuable information provided on
safety and tolerability of SPN-817 and the effective dose range in
subjects. We will extend the current Phase 2a study to explore
potential approaches that we have identified to further improve the
tolerability during titration. Full topline results from the Phase
2a study excluding this new extension period are still on track to
report in the second half of 2024. We believe SPN-817 could provide
a novel, differentiated treatment option for this hard-to-treat and
underserved patient population by currently available
therapies.”
Webcast Details
A live webcast with presentation slides will be
available via this webcast link or in the Events &
Presentations section of the Company’s Investor Relations website
at www.supernus.com/investors. Following management’s prepared
remarks and discussion of the interim trial results, the call will
open for questions.
Participants may also pre-register any time
before the call here. Once registration is completed, participants
will be provided a dial-in number with a personalized conference
code to access the call. Please dial in 15 minutes prior to the
start time.
Following the live call, a replay will be
available on the Company's Investor Relations website at
www.supernus.com/investors. The webcast will be available on the
Company’s website for 60 days following the live call.
About SPN-817
(huperzine A)
SPN-817 represents a novel mechanism of action
(MOA) for an anticonvulsant. SPN-817 is a novel synthetic form of
huperzine A, whose MOA includes potent acetylcholinesterase
inhibition, with pharmacological activities in CNS conditions such
as epilepsy. The development will initially focus on the drug's
anticonvulsant activity, which has been shown in preclinical models
to be effective for the treatment of partial seizures and Dravet
Syndrome. SPN-817 has received Orphan Drug designation for both
Dravet Syndrome and Lennox-Gastaut Syndrome from the U.S. Food and
Drug Administration (FDA). We are focused on completing and
optimizing the synthesis process of the synthetic drug as well as
developing a novel dosage form. Given the potency of SPN-817, a
novel extended-release oral dosage form is critical to the success
of this program because initial studies with the immediate-release
formulations of non-synthetic SPN-817 have shown serious
dose-limiting, side effects. An open-label Phase 2a clinical study
of SPN-817 in adult patients with treatment-resistant seizures is
ongoing.
About the Phase 2a Clinical
Study
The study is a Phase 2a multicenter,
three-phase, long-term open-label study assessing the safety and
tolerability of SPN-817 in adults 18-70 years of age with treatment
resistant seizures, as well as assessing efficacy. The screening
period is up to 8 weeks in duration. For eligible participants,
treatment period is 20 weeks in duration followed by an option
open-label extension period which is up to 52 weeks in duration.
The primary outcome measure is incidence of AEs and AEs leading to
discontinuation. Key secondary outcome measures include: 1) Percent
Change from Baseline (PCB) in quantifiable motor seizure frequency
per 28 days throughout SPN-817 dosing during maintenance period and
open-label extension, 2) Treatment response defined as ≥30%, ≥50%,
and ≥75% reduction in quantifiable motor seizure frequency per 28
days relative to the baseline period, 3) Change from baseline in
Clinical Global Impression-Severity (CGI-S) scores, and 4) Change
from baseline in cognitive profile as assessed by EpiTrack®.
About Supernus Pharmaceuticals,
Inc.
Supernus Pharmaceuticals is a biopharmaceutical
company focused on developing and commercializing products for the
treatment of central nervous system (CNS) diseases.
Our diverse neuroscience portfolio includes
approved treatments for epilepsy, migraine, ADHD, hypomobility in
Parkinson’s disease (PD), cervical dystonia, chronic sialorrhea,
dyskinesia in PD patients receiving levodopa-based therapy, and
drug-induced extrapyramidal reactions in adult patients. We are
developing a broad range of novel CNS product candidates including
new potential treatments for hypomobility in PD, epilepsy,
depression, and other CNS disorders.
For more information, please visit
www.supernus.com.
Forward Looking StatementsThis
press release includes forward-looking statements within the
meaning of the Private Securities Litigation Reform Act of 1995.
These statements do not convey historical information but relate to
predicted or potential future events that are based upon
management's current expectations. These statements are subject to
risks and uncertainties that could cause actual results to differ
materially from those expressed or implied by such statements. In
addition to the factors mentioned in this press release, such risks
and uncertainties include, but are not limited to, the Company’s
reporting on preliminary and partial interim data from an
exploratory open label clinical study on SPN-817, the Company’s
ability to sustain and increase its profitability; the Company’s
ability to raise sufficient capital to fully implement its
corporate strategy; the implementation of the Company’s corporate
strategy; the Company’s future financial performance and projected
expenditures; the Company’s ability to increase the number of
prescriptions written for each of its products and the products of
its subsidiaries; the Company’s ability to increase its net
revenue; the Company’s ability to commercialize its products and
the products of its subsidiaries; the Company’s ability to enter
into future collaborations with pharmaceutical companies and
academic institutions or to obtain funding from government
agencies; the Company’s ability to conduct and progress product
research and development activities, including the timing and
progress of the Company’s clinical trials, and projected
expenditures; the Company’s ability to receive, and the timing of
any receipt of, regulatory approvals to develop and commercialize
the Company’s product candidates including SPN-817; the Company’s
ability to protect its intellectual property and the intellectual
property of its subsidiaries and operate its business without
infringing upon the intellectual property rights of others; the
Company’s expectations regarding federal, state and foreign
regulatory requirements; the therapeutic benefits, effectiveness
and safety of the Company’s product candidates including SPN-817;
the accuracy of the Company’s estimates of the size and
characteristics of the markets that may be addressed by its product
candidates; the Company’s ability to increase its manufacturing
capabilities for its products and product candidates including
SPN-817; the Company’s projected markets and growth in markets; the
Company’s product formulations and patient needs and potential
funding sources; the Company’s staffing needs; the Company’s
ability to increase the number of prescriptions written for each of
its products and the products of its subsidiaries; the Company’s
ability to increase its net revenue from its products and the
products of its subsidiaries; and other risk factors set forth from
time to time in the Company’s filings with the Securities and
Exchange Commission made pursuant to Section 13 or 15(d) of the
Securities Exchange Act of 1934, as amended. The Company undertakes
no obligation to update the information in this press release to
reflect events or circumstances after the date hereof or to reflect
the occurrence of anticipated or unanticipated events.
CONTACTS:
Jack A. Khattar, President and CEOTimothy C. Dec, Senior Vice
President and CFOSupernus Pharmaceuticals, Inc.(301) 838-2591
or
INVESTOR CONTACT:Peter VozzoICR Westwicke(443)
213-0505peter.vozzo@westwicke.com
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