Praxis Precision Medicines to Advance PRAX-562 Phase 2 Study in Pediatric Patients with Developmental and Epileptic Encephalopathies
November 28 2022 - 8:00AM
Praxis Precision Medicines, Inc. (NASDAQ: PRAX), a clinical-stage
biopharmaceutical company translating genetic insights into the
development of therapies for central nervous system (CNS) disorders
characterized by neuronal excitation-inhibition imbalance, today
announced plans to initiate the PRAX-562 Phase 2 EMBOLD study for
the treatment of pediatric patients with developmental and
epileptic encephalopathies (DEEs), following U.S. Food and Drug
Administration (FDA) authorization to proceed with the study as
proposed by Praxis, up to the planned maximum dose of 1.0
mg/kg/day. The EMBOLD Study is expected to initiate in the U.S in
the first quarter of 2023, with two distinct cohorts in early-onset
SCN2A-DEE and SCN8A-DEE patients. Topline results for both cohorts
are expected in the second half of 2023.
“We designed PRAX-562 as a significantly differentiated sodium
channel inhibitor specifically for the treatment of DEEs, with a
goal of bringing the first precision treatment option to patients
living with these devastating forms of epilepsy,” said Steven
Petrou, co-founder and chief scientific officer of Praxis. “Every
day matters for these patients and their caregivers, and we look
forward to initiating the EMBOLD study to evaluate the promise of
PRAX-562 for the treatment of SCN2A, SCN8A and other DEEs.”
The EMBOLD Study is a randomized, double-blind,
placebo-controlled Phase 2 clinical trial to evaluate the safety,
tolerability, efficacy (motor seizure frequency) and
pharmacokinetics (PK) of PRAX-562 in pediatric participants aged 2
to 18 years with DEEs, followed by an open-label extension.
Approximately 20 participants will be enrolled in a total of 2
distinct cohorts (n≈10 for SCN2A-DEE and n≈10 for SCN8A-DEE).
About PRAX-562PRAX-562 is a first-in-class
small molecule in development for the treatment of DEEs as a
preferential inhibitor of persistent sodium current, shown to be a
key driver of seizure symptoms in early onset SCN2A-DEE and
SCN8A-DEE. In-vitro, PRAX-562 has demonstrated superior selectivity
for disease-state NaV channel hyperexcitability and a wider
therapeutic window compared to other anti-seizure medicines, with
potential for enhanced efficacy and improved tolerability. In-vivo
studies of PRAX-562 have demonstrated dose-dependent block of
seizures up to complete inhibition of seizure activity in SCN2A,
SCN8A and other DEE mouse models. PRAX-562 has been generally
well-tolerated in three Phase 1 studies and has demonstrated
biomarker changes indicative of NaV channel blocking effects.
PRAX-562 has received Orphan Drug Designation (ODD) and Rare
Pediatric Disease Designation from the FDA, and ODD from the
European Medicines Agency for the treatment of SCN2A-DEE and
SCN8A-DEE respectively.
About SCN2A-DEESCN2A-DEE is a monogenic
epilepsy disorder caused by a variant in the SCN2A gene. The SCN2A
gene is critical in the formation of sodium channel proteins in the
brain, which control the flow of sodium ions into neurons. This
movement of sodium ions is a major component of generating
electrical signals called action potentials, the way in which the
cells communicate. SCN2A-DEE presents with a wide range of
phenotypes. Early-onset SCN2A-DEE presents before three months and
can lead to profound impact on patients, including drug-resistant
seizures, significant cognitive impairment, movement disorders such
as dystonia or ataxia and problems in other body systems such as
gastrointestinal or ocular. Currently there are no approved
treatments for SCN2A-DEE, and the standard-of-care typically
involves a regimen of many concurrent anti-seizure medications as
well as medications for co-morbidities. Despite these
interventions, more than 70% of early-onset SCN2A-DEE patients live
with uncontrolled seizures, and approximately 75% live with severe
intellectual disability.
About SCN8A-DEESCN8A-DEE is a rare
developmental and epileptic encephalopathy caused by a variant in
the SCN8A gene. The SCN8A gene is critical in the formation of
sodium channel proteins in the brain, which control the follow of
sodium ions into neurons. This movement of sodium ions is a major
component of generating electrical signals called action
potentials, the way in which the cells communicate. Patients suffer
from recurrent, typically drug-resistant seizures which start as
early as the first day of life. The seizures can be of multiple
different types, up to dozens per day, with poor response to
current treatment options. Patients with SCN8A-DEE have significant
cognitive disabilities, ranging from moderate to severe; often
movement disorders, such as dystonia or ataxia; and problems in
other body systems such as gastrointestinal or ocular. SCN8A-DEE
patients also may experience autonomic features such as increases
or decreases in heart rate, abnormal breathing and cyanosis.
About PraxisPraxis Precision Medicines is a
clinical-stage biopharmaceutical company translating genetic
insights into the development of therapies for CNS disorders
characterized by neuronal excitation-inhibition imbalance. Praxis
is applying insights from genetic epilepsies to both rare and more
prevalent neurological disorders, using our understanding of shared
biological targets and circuits in the brain. Praxis has
established a broad portfolio with multiple programs, including
product candidates across movement disorders, epilepsy and
psychiatric disorders, with four clinical-stage product candidates.
For more information, please visit www.praxismedicines.com and
follow us on LinkedIn and Twitter.
Forward-Looking StatementsThis press release
contains forward-looking statements within the meaning of The
Private Securities Litigation Reform Act of 1995 and other federal
securities laws, including express or implied statements regarding
Praxis’ future expectations, plans and prospects, including,
without limitation, statements regarding the anticipated timing of
Praxis’ clinical trials, our expectations, plans and timing for
Praxis’ clinical data and the development of Praxis’ product
candidates, including the design of clinical trials and the
treatment potential of Praxis’ product candidates, as well as other
statements containing the words “anticipate,” “believe,”
“continue,” “could,” “endeavor,” “estimate,” “expect,”
“anticipate,” “intend,” “may,” “might,” “plan,” “potential,”
“predict,” “project,” “seek,” “should,” “target,” “will” or “would”
and similar expressions that constitute forward-looking statements
under the Private Securities Litigation Reform Act of 1995.
The express or implied forward-looking statements included in
this press release are only predictions and are subject to a number
of risks, uncertainties and assumptions, including, without
limitation: uncertainties inherent in clinical trials; the expected
timing of submissions for regulatory approval or review by
governmental authorities; risks, uncertainties and assumptions
regarding the impact of the continuing COVID-19 pandemic on Praxis’
business, operations, strategy, goals and anticipated timelines,
Praxis’ ability to initiate, enroll, conduct or complete ongoing
and planned clinical trials and Praxis’ timelines for regulatory
submissions; and other risks concerning Praxis’ programs and
operations as described in its Quarterly Report on Form 10-Q for
the quarter ended June 30, 2022 and other filings made with the
Securities and Exchange Commission. Although Praxis’
forward-looking statements reflect the good faith judgment of its
management, these statements are based only on information and
factors currently known by Praxis. As a result, you are cautioned
not to rely on these forward-looking statements. Any
forward-looking statement made in this press release speaks only as
of the date on which it is made. Praxis undertakes no obligation to
publicly update or revise any forward-looking statement, whether as
a result of new information, future developments or otherwise.
Investor Contact:
Alex Kane
Praxis Precision Medicines
investors@praxismedicines.com
617-300-8481
Media Contact:
Ian Stone
Canale Communications
Ian.stone@canalecomm.com
619-849-5388
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