Praxis Precision Medicines Announces Granting of Rare Pediatric Disease Designation for Relutrigin in Dravet Syndrome

Dravet syndrome is a genetic developmental epileptic encephalopathy (DEE) often caused by a mutation in SCN1A.

This is the third rare pediatric disease designation for relutrigin, adding to those granted to EEDs SCN2A and SCN8A.

Praxis plans to launch a fully DEE trial (EMERALD), including Dravet syndrome, in 1H2025

BOSTON, December 18, 2024 (GLOBE NEWSWIRE) — Praxis Precision MedicinesInc. (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 Food and Drug Administration (FDA) of the United States. granted rare pediatric disease designation (RPDD) for relutrigin in Dravet syndrome.

“We are pleased to have received rare pediatric disease designation for relutrigin in Dravet syndrome. This important milestone reflects our commitment to addressing critical unmet needs in rare neurological disorders and highlights the potential of relutrigin as a meaningful new option for patients and families affected by this disease. debilitating developmental and epileptic encephalopathy. This designation is based on encouraging clinical data demonstrating relutrigin’s ability to reduce seizure frequency and improve overall seizure control, underscoring our commitment to progress. innovative therapies that can significantly impact the quality of life of people with limited treatment options,” said Marcio Souza, President and CEO.

Rare pediatric diseases are defined by the FDA as serious or life-threatening conditions primarily affecting children under the age of 18, with fewer than 200,000 cases in the United States. One of the main benefits of obtaining an RPDD is the ability to receive a priority review voucher after FDA approval of a product with RPDD if the marketing application filed for the product meets certain conditions. These supporting documents can significantly expedite the review process for future applications for new drugs or biologics licenses.

In September, and updated at the recent American Epilepsy Society 2024 Annual Meeting, Praxis shared results of relutrigin in SCN2A and SCN8A patients in Cohort 1 of the EMBOLD study which showed:

• Monthly reduction in motor seizures of 46% during the double-blind period, adjusted by placebo
• More than 30% of patients achieved seizure-free status on relutrigin
• Significant gains observed in terms of vigilance, communication and severity of crises
• 77% reduction in median seizure rate seen in patients in long-term extension

Based on the results of this study, Praxis has initiated and is currently recruiting patients in a second registration cohort for SCN2A and SCN8A patients, with initial results expected in the first half of 2026.

About Relutrigin (PRAX-562)

Relutrigine is a first-in-class small molecule in development for the treatment of developmental epileptic encephalopathy (DEE) as a preferential inhibitor of persistent sodium current, which has been shown to be a key contributor to seizure symptoms in SCN2A-DEE and SCN8A-DEE. The sodium channel modulation mechanism of relutrigin is consistent with higher selectivity for sodium channel (NaV) hyperexcitability in a disease state. In vivo studies of relutrigin demonstrated dose-dependent seizure inhibition up to complete control of seizure activity in SCN2A, SCN8A, and other DEE mouse models. Relutrigin was generally well tolerated in three phase 1 studies and demonstrated biomarker changes indicative of NaV channel blocking effects. Relutrigin has received Orphan Drug Designation (ODD) and RPDD from the FDA, as well as ODD from the European Medicines Agency for the treatment of SCN2A-DEE and SCN8A-DEE. To learn more about the EMBOLD study, please visit https://www.emboldstudy.org/.

About Dravet syndrome

Dravet syndrome is a severe and progressive genetic epilepsy that generally begins during the first year of life, marked by frequent, prolonged and treatment-resistant seizures. Beyond seizures, the disease often causes intellectual disability, developmental delays, movement and balance difficulties, speech and language disorders, growth abnormalities, sleep disorders, dysfunction of the autonomic nervous system and mood disorders. Classified among developmental and epileptic encephalopathies, Dravet syndrome is associated with significant cognitive and developmental disorders. Patients with this disease also face a high risk of early mortality, particularly due to sudden unexpected death due to epilepsy (SUDEP).

About Praxis 

Praxis Precision Medicines is a clinical-stage biopharmaceutical company translating knowledge of genetic epilepsies into the development of therapies for CNS disorders characterized by neuronal excitation-inhibition imbalance. Praxis applies its genetic knowledge to the discovery and development of therapies for rare and more prevalent neurological disorders through its proprietary small molecule platform, Cerebrum, and its antisense oligonucleotide (ASO) platform, Solidus, using our understanding shared biological targets and circuits in the brain. Praxis has established a diverse, multi-modal CNS portfolio including multiple programs spanning movement disorders and epilepsy, with four clinical-stage product candidates. For more information, please visitwww.praxismedicines.comand follow usFacebook,LinkedInAndTwitter/X.

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’s future expectations, plans and prospects, including, without limitation, statements regarding the timing of our clinical trials and development of our product candidates, as well as other statements containing the words “anticipate”, “believe”, “continue”, “may », “endeavour”, “ estimate,” “expect,” “anticipate,” “intend,” “could,” “could,” “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.

Any express or implied forward-looking statements included in this press release are predictions only and are subject to a number of risks, uncertainties and assumptions, including, without limitation: uncertainties inherent in clinical trials ; preliminary analyzes of ongoing studies differing materially from final data from completed preclinical studies and clinical trials; the anticipated schedule of clinical trials, data readings and their results, as well as submissions for regulatory approval or review by governmental authorities; regulatory approvals to conduct trials; and other risks relating to Praxis’ programs and operations as described in its Annual Report on Form 10-K for the fiscal year ended December 31, 2023, its Quarterly Reports on Form 10-Q and other documents filed with the Securities and Exchange Commission. Although Praxis’ forward-looking statements reflect the good faith judgment of its management, such statements are based only on information and factors currently known to Praxis. Accordingly, you are cautioned not to rely on these forward-looking statements. Any forward-looking statements made in this press release speak only as of the date on which they are made. Praxis undertakes no obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future developments or otherwise.

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