Praxis Precision Medicines to Advance Phase 2 PRAX-562 Study in Pediatric Patients with Developmental Encephalopathies and Epilepsy | MarketScreener

BOSTON, November 28, 2022 (GLOBE NEWSWIRE) — Praxis Precision MedicinesInc. (NASDAQ: PRACTICE), a clinical-stage biopharmaceutical company applying genetic insights to develop therapies for central nervous system (CNS) disorders characterized by neuronal excitation-inhibition imbalance, today announced plans to launch the PRACTICE-562 EMBOLD Phase 2 study for the treatment of pediatric patients with developmental and epileptic encephalopathies (DEE), following US Food and Drug Administration (FDA) to continue the study as proposed by Praxis, up to the maximum planned dose of 1.0 mg/kg/day. The EMBOLD study should start in the WE in the first quarter of 2023, with two distinct cohorts of early-onset SCN2A-DEE and SCN8A-DEE patients. The first results for both cohorts are expected in the second half of 2023.

” We have made PRACTICE-562 as a significantly differentiated sodium channel blocker specifically for the treatment of DEE, with the goal of bringing the first precision treatment option to patients living with these devastating forms of epilepsy,” said Steven Petrou, co-founder and scientific director of Praxis. “Every day matters to these patients and their caregivers, and we look forward to initiating the EMBOLD study to assess the promise of PRACTICE-562 for processing 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 PRACTICE-562 in pediatric participants aged 2 to 18 years with EDE, followed by open-label extension. Approximately 20 participants will be enrolled in a total of 2 separate cohorts (n≈10 for SCN2A-DEE and n≈10 for SCN8A-DEE).

About PRACTICE-562
PRACTICE-562 is a first-in-class small molecule being developed for the treatment of DEEs as a preferential inhibitor of persistent sodium current, which has been shown to be a key contributor to epileptic symptoms in SCN2A-DEEs and SCN8A-DEEs early onset. In vitro, PRACTICE-562 has demonstrated superior selectivity for disease-state NaV channel hyperexcitability and a wider therapeutic window compared to other antiepileptic drugs, with potential for improved efficacy and tolerability. In-vivo studies of PRACTICE-562 demonstrated dose-dependent blockade of seizures up to complete inhibition of seizure activity in SCN2A, SCN8A, and other DEE mouse models. PRACTICE-562 was generally well tolerated in three phase 1 studies and demonstrated biomarker changes indicative of NaV channel blocking effects. PRACTICE-562 has received Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation from the FDA, and the ODD of the European Medicines Agency for processing SCN2A-DEE and SCN8A-DEE respectively.

About SCN2A-DEE
SCN2A-DEE is a monogenic seizure disorder caused by a variant of the SCN2A gene. The SCN2A gene is essential for 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 part of generating electrical signals called action potentials, the way cells communicate. SCN2A-DEE exhibits a wide range of phenotypes. Early-onset SCN2A-DEE presents before three months and can have a profound impact on patients, including drug-resistant seizures, significant cognitive impairment, movement disorders such as dystonia or ataxia, and in other body systems such as gastrointestinal or ocular. There is currently no approved treatment for SCN2A-DEE, and the standard of care usually involves a regimen of many concurrent antiepileptic drugs as well as drugs for comorbidities. 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-DEE
SCN8A-DEE is a rare developmental and epileptic encephalopathy caused by a variant of the SCN8A gene. The SCN8A gene is essential for the formation of sodium channel proteins in the brain, which control the tracking of sodium ions in neurons. This movement of sodium ions is a major part of generating electrical signals called action potentials, the way cells communicate. Patients suffer from recurrent, usually drug-resistant seizures that begin on the first day of life. Seizures can be of several different types, up to dozens per day, with poor response to current treatment options. Patients with SCN8A-DEE have significant cognitive impairment, ranging from moderate to severe; often movement disorders, such as dystonia or ataxia; and problems in other bodily systems such as gastrointestinal or ocular. SCN8A-DEE patients may also exhibit autonomic features such as increases or decreases in heart rate, abnormal breathing, and cyanosis.

About Praxis
Praxis Precision Medicines is a clinical-stage biopharmaceutical company that translates genetic insights into the development of therapies for CNS disorders characterized by neuronal excitation-inhibition imbalance. Praxis applies insights from genetic epilepsies to rare and more prevalent neurological disorders, using our understanding of shared biological targets and circuits in the brain. Praxis has established a broad pipeline with multiple programs, including product candidates in movement disorders, epilepsy and psychiatric disorders, with four clinical-stage product candidates. For more information, visit www.praxismedicines.com and follow us on LinkedIn and Twitter.

Forward-looking statements
This 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 Praxis’ forecast clinical trial schedule, our expectations, plans and timeline for Praxis clinical data and the development of Praxis’ product candidates, including clinical trial design and therapeutic potential of Praxis’ product candidates, and other statements containing the words “anticipate”, “believe”, “continue”, “could”, “endeavour”, “estimate”, “expect” , “anticipate”, “intend”, “may”, “could”, “plan”, “potential”, “predict”, “project”, “seek”, “should”, “target”, ” will be” or “would be” and expre statements 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: the 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, objectives and anticipated timelines, Praxis’ ability to initiate, register, conduct or complete ongoing and planned clinical trials and Praxis’ timelines for regulatory submissions; and other risks relating to Praxis’ programs and operations, as described in its Quarterly Report on Form 10-Q for the quarter ended June 30, 2022 and other deposits made with the Security and Exchange Commission. Although Praxis’ forward-looking statements reflect the good faith judgment of its management, such statements are based solely on information and factors currently known to Praxis. Accordingly, 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 

Source: Praxis Precision Medicines, Inc.

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