Praxis Precision Medicines Announces Positive Results from the EMBRAVE Part A Trial of Elsunersen in Patients with SCN2A Early-Onset Developmental and Epileptic Encephalopathy

Rhea-AI Summary

Praxis Precision Medicines (NASDAQ: PRAX) reported positive topline EMBRAVE Part A results for elsunersen in pediatric SCN2A early-onset developmental and epileptic encephalopathy (DEE). Elsunersen showed a 77% placebo-adjusted seizure reduction from baseline (p=0.015) and sustained benefit in an open-label extension for up to one year.

Additional findings: 57% of patients had a ≥28-day seizure-free period; 100% of treated patients had improvements in sleep, motor function, muscle tone or attention; no drug-related serious adverse events were reported. Nine patients were randomized 3:1; dosing started at 1 mg with escalation to 8 mg.

Positive

• 77% placebo-adjusted seizure reduction (p=0.015)
• 57% of patients achieved at least a 28-day seizure-free period
• Efficacy sustained up to one year in the open-label extension
• No drug-related serious adverse events and no neuroinflammation signals

Negative

• Very small trial size: 9 patients randomized (3:1), limiting statistical power
• Results are from a Phase 1/2 study; pivotal data pending from EMBRAVE3

News Market Reaction – PRAX

-0.09%

2 alerts

-0.09% Session close to close

$8.64B Market Cap

2.12K Volume

In the Apr 6 session, PRAX declined 0.09%, reflecting a mild negative market reaction. Our momentum scanner triggered 2 alerts that day, indicating moderate trading interest and price volatility.

Data tracked by StockTitan Argus on the day of publication.

Market Context

This announcement highlights robust early clinical signals for elsunersen, with a 77% placebo-adjust...

Analysis

This announcement highlights robust early clinical signals for elsunersen, with a 77% placebo-adjusted seizure reduction, 57% of patients achieving a 28-day seizure-free period, and no drug-related SAEs. It builds on a pipeline already supported by NDAs for ulixacaltamide and relutrigine. Investors may watch for EMBRAVE3 pivotal data, regulatory interactions, and how Praxis deploys its substantial cash balance disclosed in recent filings.

Key Figures

Seizure reduction: 77% placebo-adjusted reduction from baseline P-value: p=0.015 Responder rate: 71% with >50% seizure reduction by period 6 +5 more

8 metrics

Seizure reduction 77% placebo-adjusted reduction from baseline EMBRAVE Part A efficacy result

P-value p=0.015 Statistical significance for seizure reduction

Responder rate 71% with >50% seizure reduction by period 6 Elsunersen-treated patients in EMBRAVE Part A

Seizure freedom 57% had at least a 28-day seizure-free period EMBRAVE Part A efficacy outcome

Clinical improvements 100% of elsunersen patients with additional improvements; 0% placebo Sleep, motor function, muscle tone, attention, development

Patient count 9 patients aged 2–12 years Randomized 3:1 to elsunersen or sham

Dosing schedule 1 mg starting dose, up to 8 mg every 4 weeks for 24 weeks EMBRAVE Part A dosing regimen

Safety outcomes No drug-related SAEs; no neuroinflammation signals Safety profile at doses up to 8 mg

Historical Context

5 past events · Latest: Mar 30 (Positive)

Pattern 5 events

Date	Event	Sentiment	24h Move	Catalyst
Mar 30	NDA priority review	Positive	+16.6%	FDA accepted relutrigine NDA with priority review and set PDUFA date.
Mar 04	Inducement grants	Neutral	-4.5%	New employee RSU awards under 2024 Inducement Plan reported under Nasdaq rules.
Feb 19	Earnings and update	Positive	+0.8%	Reported 2025 results, strong cash position, and two NDA submissions highlighted.
Feb 09	Earnings date, conferences	Neutral	+0.3%	Announced Q4/FY 2025 earnings date and participation in investor conferences.
Feb 04	Inducement grants	Neutral	+0.8%	Reported RSU grants to new employees under 2024 Inducement Plan.

24h Move is the share-price change in the day after each event; other market factors may also have contributed.

Pattern Detected

PRAX has generally reacted positively to major clinical and regulatory milestones, with only administrative items showing occasional negative divergence.

Recent Company History

Over recent months, Praxis reported several key milestones. On Mar 30, 2026, FDA acceptance and priority review of the relutrigine NDA for SCN2A/SCN8A DEEs drove a 16.55% gain. A Q4 2025 update on Feb 19, 2026 highlighted $926.1M cash and two NDAs, with a modestly positive move. Routine inducement grant announcements in February and March saw mostly muted price changes, aside from a -4.47% drop on Mar 4, 2026.

Key Terms

phase 1/2 trial, placebo-controlled, developmental and epileptic encephalopathy, open-label extension, +3 more

7 terms

phase 1/2 trial medical

"EMBRAVE Part A is a randomized, placebo-controlled Phase 1/2 trial evaluating..."

A phase 1/2 trial combines the earliest human safety testing with an initial look at whether a treatment works, typically starting by checking tolerability and side effects and then expanding to measure early signs of benefit and the best dose. For investors, results from these trials are an early indicator of a drug’s clinical promise and regulatory path: positive data can materially increase a company’s value and reduce development risk, while negative data can sharply lower expectations.

placebo-controlled medical

"EMBRAVE Part A is a randomized, placebo-controlled Phase 1/2 trial..."

"Placebo-controlled" describes a testing method where one group receives the actual treatment or intervention, while another group receives a harmless, inactive version called a placebo. This approach helps determine whether the real treatment has genuine effects beyond psychological expectations. For investors, understanding this ensures confidence that reported benefits are real and not influenced by bias or false perceptions.

developmental and epileptic encephalopathy medical

"pediatric patients with early-seizure onset SCN2A developmental and epileptic encephalopathy (DEE)."

A severe neurological condition that begins in infancy or early childhood, marked by frequent, hard-to-control seizures and slowing or loss of normal development such as movement, learning and communication. Think of it as a critical control system in a child’s brain that malfunctions and interferes with growth and daily function. For investors it signals a high unmet medical need, focused regulatory attention and the potential for significant commercial value — but also greater clinical and development risk for therapies.

open-label extension medical

"24 weeks, followed by an open-label extension (OLE); all 9 patients continued..."

An open-label extension is a continuation of a clinical trial where all participants and researchers know which treatment is being given, often after an initial blinded phase. It allows further study of a drug's long-term safety and effectiveness. For investors, it can indicate ongoing interest and confidence in a product's potential, influencing perceptions of its future value.

treatment-emergent adverse events medical

"Most treatment-emergent adverse events (TEAEs) were mild to moderate."

Events or symptoms that either appear for the first time or get worse after a patient starts a treatment; think of new or intensified side effects that show up once medicine or a medical device is used. Investors watch these closely because they affect whether a therapy can gain regulatory approval, be prescribed widely, or face legal and commercial setbacks—similar to how early customer complaints can sink a new product’s prospects.

serious adverse events medical

"No treatment-emergent or serious adverse events related to study drug reported"

Serious adverse events are significant problems or negative outcomes that occur during a medical treatment or clinical trial, such as severe side effects, hospitalizations, or life-threatening conditions. They matter to investors because such events can impact a company's reputation, lead to regulatory scrutiny, or delay the development of new products, ultimately affecting the company’s financial performance.

neuroinflammation medical

"no discontinuations and no neuroinflammation signals at doses up to 8 mg"

Neuroinflammation is the brain or spinal cord’s immune reaction to injury, infection, or abnormalities, where cells and molecules become active to protect or repair nervous tissue. It matters to investors because it underlies many neurological diseases and is a common target for drugs and diagnostic tools; positive or negative trial results, safety signals, or new therapies can change a company’s value much like a major repair plan or recall would affect a carmaker’s prospects.

AI-generated analysis. How Rhea-AI works. Not financial advice.

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Elsunersen demonstrated placebo-adjusted seizure reduction from baseline of 77% (p=0.015)

71% of elsunersen-treated patients achieved >50% seizure reduction by period 6, with sustained benefit observed in the open-label extension for up to one year

100% of elsunersen patients - and none on placebo - had additional improvements, including sleep, motor function, muscle tone and attention

No treatment-emergent or serious adverse events related to study drug reported

BOSTON, April 06, 2026 (GLOBE NEWSWIRE) -- Praxis Precision Medicines, Inc. (NASDAQ: PRAX), a fully integrated, leading central nervous system (CNS) precision neuroscience biopharmaceutical company, today announced positive topline results from the EMBRAVE Part A trial of elsunersen in pediatric patients with early-seizure onset SCN2A developmental and epileptic encephalopathy (DEE). 

“We are thrilled to see the remarkable, consistent results from EMBRAVE Part A, showing 77% reduction in monthly seizures and disease modifying improvements in children with SCN2A early-seizure onset DEE. We are well underway with our pivotal EMBRAVE3 study and look forward to sharing this placebo-controlled data from the EMBRAVE Part A study with all key stakeholders,” said Marcio Souza, president and chief executive officer.

EMBRAVE Part A is a randomized, placebo-controlled Phase 1/2 trial evaluating the safety and efficacy of ascending doses of elsunersen in patients with SCN2A DEE. Nine patients, aged 2-12 years old, were randomized 3:1 to receive either elsunersen or sham procedure every 4 weeks for 24 weeks, followed by an open-label extension (OLE); all 9 patients continued to the OLE. Patients received a starting dose of 1 mg with optional dose escalation based on observed seizure reduction and individual tolerability.

Key results from EMBRAVE Part A

Efficacy

• Elsunersen treatment led to a 77% placebo-adjusted seizure reduction from baseline (p=0.015, 95 CI [33,92]
• 57% of patients had at least a 28-day period of seizure freedom
• Efficacy was sustained in the OLE for up to one year
• 100% of elsunersen patients improved across sleep, motor function, muscle tone, attention or neuropsychomotor development compared to no improvements in placebo group

Safety

• Elsunersen was well-tolerated, with no drug-related SAEs, no discontinuations and no neuroinflammation signals at doses up to 8 mg
• Most treatment-emergent adverse events (TEAEs) were mild to moderate. TEAEs were consistent with the EMBRAVE Part 1 study

Additional results will be presented at upcoming scientific meetings.

About Elsunersen (PRAX-222)
Elsunersen is an antisense oligonucleotide (ASO) designed to selectively decrease SCN2A gene expression, directly targeting the underlying cause of early-seizure-onset SCN2A-DEE to treat seizures and other symptoms in patients with gain-of-function SCN2A mutations. In vitro studies of elsunersen have demonstrated reduction in both SCN2A gene expression and protein levels. In vivo, elsunersen has demonstrated significant, dose-dependent reduction in seizures, improvement in behavioral and locomotor activity and increased survival in SCN2A mouse models, with potential to be the first disease-modifying treatment for SCN2A-DEE. Elsunersen has received ODD and RPDD from the FDA, and ODD and PRIME designations from the European Medicines Agency for the treatment of SCN2A-DEE. The elsunersen program is ongoing under a collaboration with Ionis Pharmaceuticals, Inc., and RogCon, Inc. To learn about previous studies and publications about elsunersen please visit the https://praxismedicines.com/resources. To learn more about the EMBRAVE3 study, please visit https://www.embravestudy.com/.

About Praxis
Praxis Precision Medicines is a fully integrated, leading central nervous system (CNS) precision neuroscience biopharmaceutical company, translating insights from genetic epilepsies into the development of therapies for CNS disorders characterized by neuronal excitation-inhibition imbalance. Praxis is applying genetic insights to the discovery and development of therapies for rare and more prevalent neurological disorders through our proprietary small molecule platform, Cerebrum™, and antisense oligonucleotide (ASO) platform, Solidus™, using our understanding of shared biological targets and circuits in the brain. Praxis has established a diversified, multimodal CNS portfolio including multiple programs across movement disorders and epilepsy, with four late-stage product candidates. For more information, please visit www.praxismedicines.com and follow us on Facebook, LinkedIn and X/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 the anticipated timing of clinical trials and the development of Praxis’ product candidates, the anticipated timing of regulatory submissions and interactions, as well as other 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: uncertainties inherent in clinical trials; the expected timing of clinical trials, data readouts and the results thereof, and submissions for regulatory approval or review by governmental authorities; regulatory approvals to conduct trials; and other risks concerning Praxis’ programs and operations as described in its Annual Report on Form 10-K for the year ended December 31, 2025 and in subsequent 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: 
Praxis Precision Medicines 
investors@praxismedicines.com 
857-702-9452 
 
Media Contact:
Dan Ferry
LifeSci Advisors
Daniel@lifesciadvisors.com
617-430-7576

FAQ

What did Praxis announce about elsunersen efficacy in SCN2A DEE (PRAX) on April 6, 2026?

Elsunersen showed a 77% placebo-adjusted seizure reduction from baseline (p=0.015). According to the company, results included 57% of patients with a ≥28-day seizure-free period and sustained benefit through a one-year open-label extension.

How many patients were enrolled in the EMBRAVE Part A trial of elsunersen (PRAX)?

Nine pediatric patients aged 2–12 years were randomized 3:1 to elsunersen or sham. According to the company, all nine continued into the open-label extension after the 24-week blinded period.

What safety findings did Praxis report for elsunersen in EMBRAVE Part A (PRAX)?

No treatment-related serious adverse events or neuroinflammation signals were reported at doses up to 8 mg. According to the company, most treatment-emergent adverse events were mild to moderate and consistent with prior study data.

Did elsunersen show benefits beyond seizure reduction in the EMBRAVE Part A trial (PRAX)?

Yes. According to the company, 100% of elsunersen-treated patients had additional improvements in sleep, motor function, muscle tone, attention, or neuropsychomotor development versus no improvements in placebo.

What are the next clinical steps for elsunersen after EMBRAVE Part A (PRAX)?

Praxis is progressing a pivotal EMBRAVE3 study to generate confirmatory placebo-controlled data. According to the company, EMBRAVE3 aims to build on the Phase 1/2 topline results reported April 6, 2026.