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 that it will deliver presentations on its epilepsy
programs at the American Epilepsy Society (AES) 2022 Annual
Meeting, held December 2-6, 2022 in Nashville, Tennessee.
“It’s an incredibly exciting time for Praxis and our epilepsy
portfolio, with first-in-patient studies for PRAX-222 and PRAX-562
and a first-in-human study for PRAX-628 expected to start shortly,”
said Steven Petrou, co-founder and chief scientific officer of
Praxis. “We look forward to presenting data for PRAX-562 and
PRAX-628 at AES, along with novel insights into DEEs and the
patient populations we are seeking to serve. Our epilepsy programs
have the potential to go beyond seizure control and disrupt the
course of the diseases they target by addressing the underlying
causes, and we are grateful for the opportunity to discuss these
programs with the epilepsy community at this important
meeting.”
Presentation Details:
PRAX-562 is a Well-tolerated, Novel Persistent Sodium
Channel Blocker with Broad Anticonvulsant Activity in Multiple DEE
Mouse Models
- Session Date/Time: Saturday, December 3, 12:00 p.m. - 2:00 p.m.
ET
- Abstract number: 1.281
- Summary: In multiple preclinical models of non-NaV
developmental and epileptic encephalopathy (DEE), PRAX-562
exhibited robust anticonvulsant activity indicating broad
protection regardless of the underlying genetic cause. Moreover,
PRAX-562 exhibited markedly improved preclinical tolerability
compared to standard of care NaV blockers, which may translate into
well-tolerated efficacy in epilepsy as well as other indications
caused by neuronal hyperexcitability.
Disease Impact and Burden in Patients with SCN2A-Related
Developmental and Epileptic Encephalopathy
- Session Date/Time: Sunday, December 4, 12:00 p.m. - 2:00 p.m.
ET
- Abstract number: 2.092
- Summary: Using large, real-world clinical data sets and
functional variant characterization, the study provides
unprecedented insight into clinical phenotypes, disease burden and
treatment patterns in SCN2A. Findings demonstrate that symptoms are
diverse and extend beyond seizures, with patient burden compounded
by comorbidities, high treatment use, procedural interventions, as
well as profound developmental impairment extending through to
early adulthood. This work provides novel insights into the broad,
longitudinal impact of disease, with the potential to inform trial
endpoints beyond seizure symptomatology.
A Novel Approach to Assess the Impact of Disease in
Patients with SCN8A-Related Developmental and Epileptic
Encephalopathy
- Session Date/Time: Sunday, December 4, 12:00 p.m. - 2:00 p.m.
ET
- Abstract number: 2.096
- Summary: Using large, real-world clinical data sets, this
natural history study provides the most robust, longitudinal
real-world dataset to date on disease burden and progression in
SCN8A-DEE. Our findings highlight the severity and burden of
disease in SCN8A-DEE, particularly in the first year of life for
patients with seizures presenting earlier in life (≤6 months);
further compounded by multiple factors including high medication
usage, hospital duration and comorbidities. Together with ongoing
efforts to better understand underlying genotype-phenotype
relationships, our findings will guide development of targeted,
innovative therapies that can benefit patients and their
caregivers.
A Phase 1 Trial Evaluating the Safety, Tolerability,
Pharmacokinetics and Food Effect of PRAX-562 in Healthy
Volunteers
- Session Date/Time: Sunday, December 4, 12:00 p.m. - 2:00 p.m.
ET
- Abstract number: 2.24
- Summary: In a first-in-human study to evaluate safety,
tolerability and pharmacokinetics, PRAX-562 was well tolerated in
healthy participants at single doses up to 150 mg (fasted), at
multiple doses of up to 120 mg once-daily (QD) for 14 days
(fasted), and at a single dose of 90 mg in the fed and fasted
states. These findings across 112 participants further suggest that
PRAX-562 can be administered without regard for food and support
the program's advancement into Phase 2 clinical investigation.
A Phase 1 Trial Evaluating the Safety, Tolerability,
Pharmacokinetics and Pharmacodynamics of PRAX-562 in Healthy
Volunteers
- Session Date/Time: Sunday, December 4, 12:00 p.m. - 2:00
p.m. ET
- Abstract number: 2.478
- Summary: In a two-part randomized, placebo-controlled Phase 1
study, PRAX-562 was evaluated at 90 mg QD for 28 days (Part A), and
in combination with oxcarbazepine at 120 mg QD (Part B). PRAX-562
was well tolerated in Part A. In Part B, the majority of AEs
including SAEs were considered to be due to coadministration of
projected supratherapeutic doses of PRAX-562 with oxcarbazepine,
and likely additive NaV blocking effects. Together with
pharmacokinetic findings demonstrating a 13-fold increase in
concentrations compared to preclinical maximal electroshock seizure
effects, our results are consistent with earlier work suggesting a
wide therapeutic window for PRAX-562. Furthermore,
pharmacodynamic findings indicate CNS modulation and expected
target engagement for PRAX-562 across multiple EEG
measures.
PRAX-628: A Novel Sodium Channel Blocker with Greater
Potency and Activity Dependence Compared to Standard of
Care
- Session Date/Time: Monday, December 5, 12:00 p.m. - 1:45 p.m.
ET
- Abstract number: 3.311
- Summary: In a study evaluating the in vitro effects of PRAX-628
on sodium current (INa), the next generation sodium channel (NaV)
blocker showed increased potency and activity dependence for peak
INa as well as greater potency for persistent INa. The preferential
targeting of neuronal hyperexcitability by PRAX-628 may represent a
differentiated therapeutic option for diseases of
hyperexcitability, where standard of care NaV blockers have
demonstrated efficacy but poor tolerability.
PRAX-628 is a Novel, Well-tolerated, Activity Dependent
Sodium Channel Blocker with Potent Anticonvulsant
Activity
- Session Date/Time: Monday, December 5, 12:00 p.m. - 1:45 p.m.
ET
- Abstract number: 3.28
- Summary: In in vivo studies, PRAX-628 exhibited markedly
improved preclinical tolerability compared to standard of care NaV
blockers, potentially due to its improved activity dependent
inhibition of peak INa. The demonstrated profile of PRAX-628 may
translate into well-tolerated efficacy in epilepsy as well as other
indications caused by neuronal hyperexcitability.
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 PRAX-628PRAX-628 is a novel
activity-dependent inhibitor of peak sodium current (INa) and
persistent INa currently being developed as a once daily, oral
treatment for adult focal onset epilepsy. Preclinical data
demonstrates PRAX-628 is differentiated from standard of care
sodium channel (NaV) blockers, with the potential to be a
best-in-class NaV blocker for focal epilepsy. In vitro, PRAX-628
has demonstrated superior selectivity for disease-state NaV channel
hyperexcitability. In vivo studies of PRAX-628 have demonstrated an
unprecedented therapeutic window which may translate to superior
safety and efficacy.
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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