- Bexicaserin achieved a median seizure reduction of 53.3% in
countable motor seizures compared to 20.8% in the placebo group
across the DEE study population
- A median seizure reduction of 72.1% in Dravet Syndrome (DS),
48.1% in Lennox-Gastaut Syndrome (LGS) and 61.2% in DEE Other was
achieved
- Favorable safety and tolerability results
- Longboard is rapidly moving forward with preparations for its
global Phase 3 program
- Conference call and webcast to be held today at 8:30am ET
Longboard Pharmaceuticals, Inc. (Nasdaq: LBPH), a clinical-stage
biopharmaceutical company focused on developing novel,
transformative medicines for neurological diseases, today announced
positive topline data from the PACIFIC Study evaluating bexicaserin
(LP352), a potentially best-in-class and highly selective, oral,
novel 5-HT2C receptor superagonist for seizures associated with a
broad range of Developmental and Epileptic Encephalopathies
(DEEs).
The PACIFIC Study Topline Results:
In the innovative PACIFIC Study, 52 participants ages 12-65
years old with a DEE diagnosis were enrolled at 34 study sites
across the United States and Australia to evaluate the safety,
tolerability, efficacy and pharmacokinetics of oral bexicaserin (6
mg, 9 mg and 12 mg) three times daily (TID) versus placebo.
Participant DEE diagnoses included DS, LGS, and other qualifying
DEEs (DEE Other). Following a 5-week screening period and baseline
evaluations, study participants initiated a dose titration over a
15-day period and subsequently continued on the highest tolerated
dose throughout the maintenance period of 60 days. Of the 52
participants enrolled in the study, 43 participants were randomized
to bexicaserin (DS=4, LGS=24, DEE Other=15) and 9 to placebo (DS=0,
LGS=5, DEE Other=4). The median number of countable motor seizures
per 28-day period at baseline was 38.8 in the bexicaserin group
compared to 20.8 in the placebo group. Participants were able to
remain on a contemporary, stable polytherapy regimen of up to 4
anti-seizure medications (ASMs) throughout the study, with the most
common ASMs being clobazam, cannabidiol, lamotrigine and
levetiracetam.
Summary of Efficacy Data:
The median change in countable motor seizure frequency (primary
efficacy endpoint) from baseline for the evaluable participants
treated with bexicaserin (n=35) was a decrease of 53.3%, compared
to a 20.8% decrease for those receiving placebo (n=9). Overall,
this represents a placebo-adjusted reduction in seizure frequency
of 32.5%. The median change in countable motor seizure frequency
from baseline in the DS, LGS and DEE Other cohorts was a decrease
of 72.1%, 48.1% and 61.2%, respectively. This represents a
placebo-adjusted reduction in seizure frequency of 27.3% and 28.6%
in LGS and DEE Other, respectively.
Summary of Safety Data:
Bexicaserin exhibited favorable safety and tolerability results.
Most participants (85.7%) in the bexicaserin treated group (n=35)
that started the maintenance period tolerated the highest dose (12
mg). The most common adverse events (AEs) observed were somnolence,
decreased appetite, constipation, diarrhea and lethargy. There were
3 participants that reported a serious adverse event (SAE) (ankle
fracture, constipation, increased seizures) and no deaths were
reported in the study. Overall, 9 participants in the bexicaserin
group discontinued due to an AE. Of note, 2 of these participants
discontinued during the maintenance period (7 participants
discontinued during the titration period). No participants in the
placebo group discontinued or experienced an SAE.
100% of the participants who completed the PACIFIC Study elected
to enroll in the ongoing 52-week open-label extension study.
Additional data from the PACIFIC Study are intended to be
presented at future medical meetings.
“These exciting PACIFIC Study results underscore our belief that
bexicaserin’s differentiated profile will translate into a
clinically and commercially best-in-class product and has the
potential to redefine the standard of care in DEEs. We are pleased
to see such strong seizure reduction across a wide range of DEE
syndromes with varying etiologies coupled with favorable safety and
tolerability results,” stated Dr. Randall Kaye, Longboard’s Chief
Medical Officer. “We would like to thank the entire DEE community,
including study participants, their caregivers and advocacy groups,
as well as the investigators, sites and coordinators for their
participation and continued partnership as we advance into a Phase
3 program. This tremendous milestone brings us one step closer to
improving the lives of those living with these devastating diseases
and their families.”
“The remarkable results from the PACIFIC Study give hope to
patients and their loved ones who are in dire need of research and
novel therapies in these severe syndromes. A tremendous unmet need
remains not only for those living with LGS, but for the many other
DEE patients who have not received the attention they deserve, and
I applaud this innovative and inclusive approach that is designed
to get therapies quickly and safely to even more people,” said
Tracy Dixon-Salazar, PhD, Executive Director of the LGS
Foundation.
“As the principal investigator, I am delighted to see these
highly anticipated and, more importantly, clinically meaningful
results from the PACIFIC Study. Physicians are looking for options
with fewer side effects and less burden, and that are easy to add
onto existing medications in these patients with highly refractory,
treatment resistant seizures. This is an innovative and unique
approach to clinical development in broadening research across the
DEE population. I am looking forward to participating in the future
development of this compound,” stated Dennis Dlugos, MD, MSCE,
pediatric neurologist at Children’s Hospital of Philadelphia, Vice
President & Officer of the Epilepsy Study Consortium, and
Principal Investigator of the PACIFIC Study.
“Given the groundbreaking design of the PACIFIC Study and the
broad efficacy of bexicaserin observed across DEEs in this study,
we believe that bexicaserin provides us with the cornerstone to
build a world-class epilepsy franchise and to explore development
paths forward that may offer novel options to DEE patients that are
vastly underserved. We are continuing our Phase 3 preparations as
we evaluate the broader dataset,” stated Kevin R. Lind, Longboard’s
President and Chief Executive Officer.
About the PACIFIC Study
The PACIFIC Study is a Phase 1b/2a double-blind,
placebo-controlled clinical trial to assess the safety,
tolerability, efficacy and pharmacokinetics of bexicaserin (LP352)
in 52 participants between the ages of 12 and 65 years old at 34
sites across the United States and Australia. Following a 5-week
screening period and baseline evaluations, study participants
initiated a dose titration over a 15-day period and subsequently
continued on the highest tolerated dose throughout the maintenance
period of 60 days. Following the maintenance period, participants
were then titrated down, and eligible participants were given the
opportunity to enroll in a 52-week open-label extension program.
The primary efficacy measure was median percent change from
baseline in countable motor seizure frequency over the 75-day
treatment period.
Conference Call and Webcast Details
Longboard will host a conference call today at 8:30am ET.
Stockholders and other interested parties may participate in the
call by following the instructions below. The live webcast can be
accessed on the Events & Presentations portion of the investor
page of Longboard’s website at https://ir.longboardpharma.com. A
replay will be available on Longboard’s website shortly after
completion of the event and will be archived for up to 30 days.
Participant Webcast Link:
https://edge.media-server.com/mmc/p/sqg9yxpf
Participant Call Link:
https://register.vevent.com/register/BIb92b4a3dd66f44fdbc3fcd202fca9caf
About Longboard Pharmaceuticals
Longboard Pharmaceuticals, Inc. is a clinical-stage
biopharmaceutical company focused on developing novel,
transformative medicines for neurological diseases. Longboard is
working to advance a portfolio of centrally acting product
candidates designed to be highly selective for specific G
protein-coupled receptors (GPCRs). Longboard’s small molecule
product candidates are based on more than 20 years of GPCR
research. Longboard plans to advance bexicaserin (LP352), an oral,
centrally acting 5-hydroxytryptamine 2C (5-HT2C) receptor
superagonist, with no observed impact on 5-HT2B and 5-HT2A receptor
subtypes, into a global Phase 3 program. Longboard reported topline
data from a Phase 1b/2a clinical trial for bexicaserin, the PACIFIC
Study, evaluating participants ages 12 to 65 years old with a broad
range of Developmental and Epileptic Encephalopathies (DEEs),
including Lennox-Gastaut syndrome, Dravet syndrome and other DEEs.
Longboard is also evaluating LP659, an oral, centrally acting,
sphingosine-1-phosphate (S1P) receptor subtypes 1 and 5 modulator,
which is in development for the potential treatment of rare
neuroinflammatory conditions. Longboard has initiated a Phase 1
single-ascending dose (SAD) clinical trial for LP659 in healthy
volunteers, with topline data expected in the first half of
2024.
Forward-Looking Statements
Certain statements in this press release are forward-looking
statements that involve a number of risks and uncertainties. In
some cases, you can identify forward-looking statements by words
such as “moving forward”, “to be held”, “focused on”, “potential”,
“intended”, “belief”, “will”, “would”, “advance into”, “closer to”,
“hope”, “designed to”, looking forward to”, “future”,
“opportunity”, “may”, “working to”, “plans”, “expect” or the
negative, plural or other tenses of these words or other comparable
language, and they may include, without limitation, statements
about the following: the potential of bexicaserin (including to be
best-in-class, to change the DEE landscape and to serve as the
cornerstone of a world-class epilepsy franchise); Longboard’s
planned global Phase 3 program for bexicaserin; Longboard’s
clinical and preclinical product candidates and programs, including
their advancement, timing of initiating dosing in clinical trials,
timing of topline data from clinical trials, characteristics of
clinical trial participants, their potential (including to be
highly selective and the numbers and types of conditions they may
address), and their design and characteristics; Longboard’s ability
to develop product candidates and deliver medicines; Longboard’s
focus and work; and Longboard’s plans to present additional data
from the PACIFIC Study at future medical meetings. For such
statements, Longboard claims the protection of the Private
Securities Litigation Reform Act of 1995. Actual events or results
may differ materially from Longboard’s expectations. Factors that
could cause actual results to differ materially from those stated
or implied by Longboard’s forward-looking statements include, but
are not limited to, the following: topline data may not accurately
reflect the complete results of a particular study or trial and
remain subject to audit, and final data may differ materially from
topline data; PACIFIC Study participants’ diagnoses are as of time
of screening and are subject to change; risks related to
Longboard’s limited operating history, financial position and need
for additional capital; Longboard will need additional managerial
and financial resources to advance all of its programs, and you and
others may not agree with the manner Longboard allocates its
resources; risks related to the development and commercialization
of Longboard’s product candidates; Longboard’s product candidates
are in the early to middle phases of a lengthy research and
development process, the timing, manner and outcome of research,
development and regulatory review is uncertain, and Longboard’s
product candidates may not advance in research or development or be
approved for marketing; enrolling participants in Longboard’s
ongoing and intended clinical trials is competitive and
challenging; nonclinical and clinical data is voluminous and
detailed, and regulatory agencies may interpret or weigh the
importance of data differently and reach different conclusions than
Longboard or others, request additional information, have
additional recommendations or change their guidance or requirements
before or after approval; results of clinical trials and other
studies are subject to different interpretations and may not be
predictive of future results; macroeconomic events stemming from
the COVID-19 pandemic or evolving geopolitical developments such as
the conflicts in Ukraine and the Middle East, including but not
limited to the impact on Longboard’s clinical trials and
operations, the operations of Longboard’s suppliers, partners,
collaborators, and licensees, and capital markets, which in each
case remains uncertain; risks related to unexpected or unfavorable
new data; risks related to principal stockholders or management
selling some or all of their stock; risks related to relying on
licenses or collaborative arrangements; other risks related to
Longboard’s dependence on third parties; competition; product
liability or other litigation or disagreements with others;
government and third-party payor actions, including relating to
reimbursement and pricing; risks related to regulatory compliance;
and risks related to Longboard’s and third parties’ intellectual
property rights. Additional factors that could cause actual results
to differ materially from those stated or implied by Longboard’s
forward-looking statements are disclosed in Longboard’s filings
with the Securities and Exchange Commission (SEC). These
forward-looking statements represent Longboard’s judgment as of the
time of this release. Longboard disclaims any intent or obligation
to update these forward-looking statements, other than as may be
required under applicable law.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20240102733902/en/
Corporate Contact: Megan E. Knight Head of Investor
Relations IR@longboardpharma.com 858.789.9283
Longboard Pharmaceuticals (NASDAQ:LBPH)
Historical Stock Chart
From Apr 2024 to May 2024
Longboard Pharmaceuticals (NASDAQ:LBPH)
Historical Stock Chart
From May 2023 to May 2024