Longboard Pharmaceuticals Receives Breakthrough Therapy Designation for Bexicaserin (LP352)
July 01 2024 - 8:00AM
Business Wire
- U.S. Food and Drug Administration (FDA) has granted
Breakthrough Therapy designation for bexicaserin for the treatment
of seizures associated with Developmental and Epileptic
Encephalopathies (DEEs)
Longboard Pharmaceuticals, Inc. (Nasdaq: LBPH), a clinical-stage
biopharmaceutical company focused on developing novel,
transformative medicines for neurological diseases, today announced
that the FDA has granted Breakthrough Therapy designation for its
investigational drug bexicaserin for the treatment of seizures
associated with Developmental and Epileptic Encephalopathies (DEEs)
for patients two years of age or older.
“We are thrilled to receive Breakthrough Therapy designation for
bexicaserin and believe this important milestone underscores our
innovative approach to potentially treating a broad range of DEE
patients,” stated Dr. Randall Kaye, Longboard’s Chief Medical
Officer. “The FDA will work closely with us to provide guidance on
subsequent development of bexicaserin to help us design and conduct
a development program as efficiently as possible. We are looking
forward to initiating our global Phase 3 program later this
year.”
“I am excited about what this designation means for the DEE
community. Most of those living with DEEs do not have access to
novel medications, nor have they had the opportunity to participate
in trials designed to collect data specific to their condition. I
am thrilled that we are making strides towards advancing DEE
research for the broader population and pleased that there is a
move towards increased equity and access for underserved patients
and families to clinical trials and potential novel treatments,”
stated Gabrielle Conecker, MPH, Executive Director & Co-Founder
of Decoding Developmental Epilepsies, home of the International
SCN8A Alliance, DEE-P Connections, and The Inchstone Project.
Breakthrough Therapy designation is a process designed to
expedite the development and regulatory review of drugs that are
intended to treat serious or life-threatening conditions and
preliminary clinical evidence indicates that the drug may
demonstrate a substantial improvement over available therapy on at
least one clinically significant endpoint. A drug that receives
Breakthrough Therapy designation is eligible for more intensive
guidance on an efficient drug development program and
organizational commitment involving senior managers from the
FDA.
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. Earlier this year,
Longboard reported positive topline data from a Phase 1b/2a
clinical trial (the PACIFIC Study) evaluating bexicaserin in
participants ages 12 to 65 years old with 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 recently completed a Phase
1 single-ascending dose (SAD) clinical trial for LP659 in healthy
volunteers.
Bexicaserin and LP659 are investigational compounds that are not
approved for marketing by the FDA or any other regulatory
authority.
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 “focus”, “believe”, “potential”, “will”, “optimistic”,
“designed to”, “intended to”, “eligible”, “may”, “working to”,
“plans”, or the negative, plural or other tenses of these words,
references to future dates or time periods, or other comparable
language, and they may include, without limitation, statements
about the following: bexicaserin, including its potential to treat
a broad range of DEEs, its development and advancement into a
global Phase 3 program, the regulatory process and Breakthrough
Therapy designation, and its selectivity and novel chemistry; the
DEE community; Longboard’s ability to develop product candidates
and deliver medicines; Longboard’s focus and work; and LP659,
including its potential and Longboard’s Phase 1 SAD clinical trial
for LP659. 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: the standard for
Breakthrough Therapy designation is not the same as the standard
for drug approval, the clinical evidence supporting Breakthrough
Therapy designation is preliminary, and not all drugs designated as
Breakthrough Therapies ultimately will be shown to have substantial
improvement over available therapies; the FDA may later decide to
rescind a Breakthrough Therapy designation if it determines the
designation is no longer supported by subsequent data; Longboard’s
product candidates are in a lengthy research and development
process, the timing, manner and outcome of research, development
and regulatory review is uncertain, and Longboard’s product
candidates, including bexicaserin and LP659, may not advance in
research or development or be approved for marketing; results of
clinical trials and other studies are subject to different
interpretations and may not be predictive of future results;
topline or interim 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 or interim data;
enrolling participants in clinical trials is competitive and
challenging; risks related to unexpected or unfavorable new data;
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; 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; 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.
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Megan E. Knight VP, Head of Investor Relations
IR@longboardpharma.com 858.789.9283
Longboard Pharmaceuticals (NASDAQ:LBPH)
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