Praxis Precision Q3 2025 slides: RADIANT study reveals promising epilepsy results

Introduction & Market Context

Praxis Precision Medicines Inc (NASDAQ:PRAX) shares jumped 4.83% in premarket trading to $56.70 after the company presented positive topline results from its RADIANT study for Vormatrigine on August 4, 2025. The clinical-stage biopharmaceutical company, focused on developing treatments for central nervous system (CNS) disorders, revealed encouraging efficacy data for its lead epilepsy candidate.

Epilepsy remains a significant unmet medical need affecting approximately 3 million patients with focal epilepsy in the United States. According to the company’s presentation, 63% of these patients require multiple anti-seizure medications (ASMs), highlighting the need for more effective treatment options.

Executive Summary

Praxis highlighted its strong position in the CNS space with four assets in late-stage development, five clinical readouts expected in the next four quarters, and a cash runway extending into 2028. The company’s presentation emphasized its dual platform approach to address both common epilepsy conditions affecting millions and rare developmental epileptic encephalopathies (DEEs).

The focal point of the presentation was Vormatrigine’s impressive performance in the RADIANT Phase 2 study, showing a 56.3% overall median seizure reduction in patients with focal epilepsy. The drug demonstrated rapid onset of action and maintained efficacy even when used alongside existing treatments, positioning it as a potential best-in-class therapy.

As shown in the following comprehensive pipeline overview, Praxis has multiple clinical programs advancing toward key milestones:

Detailed Clinical Results

The RADIANT study evaluated Vormatrigine in patients with focal epilepsy, with 61 patients dosed as of July 25, 2025, and 37 patients included in the topline analysis. The study population had a median baseline seizure rate of 12 seizures and was taking an average of 2.2 background ASMs, representing a challenging patient population with significant disease burden.

Vormatrigine demonstrated compelling efficacy with a 56.3% overall median seizure reduction. More impressively, 60% of patients achieved at least a 50% reduction in seizures, and approximately 22% of patients were seizure-free during the last 28 days of treatment.

The following chart illustrates the significant seizure reduction achieved with Vormatrigine:

The response to treatment was both rapid and durable, with 54% of patients achieving at least 50% seizure reduction by week 1, increasing to 67% by week 8. This suggests that patients may experience meaningful benefits shortly after initiating treatment.

As shown in the following chart detailing the response pattern over time:

Importantly, Vormatrigine maintained its efficacy regardless of baseline seizure burden or concomitant medications. The drug showed similar response rates in patients with higher (>12) or lower (≤12) baseline seizure frequency. Even when used alongside sodium channel blockers (57% response), SV2A modulators (63% response), or cenobamate (55% response), Vormatrigine demonstrated consistent efficacy.

On the safety front, Vormatrigine showed a favorable tolerability profile with 59% of patients experiencing at least one treatment-emergent adverse event (TEAE), most of which were mild to moderate. The most common adverse events were dizziness (29.5%), somnolence (16.4%), and headache (13.1%).

The following safety data highlights Vormatrigine’s tolerability profile:

When compared to other epilepsy treatments in development or on the market, Vormatrigine appears to offer advantages in terms of safety profile, dosing convenience, and lack of significant drug-drug interactions:

Strategic Initiatives

Based on the positive RADIANT results, Praxis is advancing its comprehensive development program for Vormatrigine. The company outlined plans for POWER2, a pivotal study that will enroll approximately 400 patients randomized to receive 20mg, 30mg, 40mg of Vormatrigine or placebo once daily for 12 weeks.

The company is also planning POWER3, a study evaluating Vormatrigine as a single agent in patients with refractory epilepsy currently on 1-2 ASMs. This study is expected to initiate in the first half of 2026 and represents an important strategic move to position Vormatrigine as a standalone treatment option.

The following slide illustrates how Praxis is targeting the full spectrum of epilepsy conditions:

Competitive Industry Position

Praxis is positioning Vormatrigine as a potentially best-in-class ASM with several key advantages:

1. Superior efficacy with 56.3% median seizure reduction

2. Favorable safety and tolerability profile compared to existing options

3. Convenient once-daily dosing without titration requirements

4. No food effect or significant drug-drug interactions

5. Efficacy maintained even when used with other ASMs

The company emphasized that the majority of focal epilepsy patients quickly progress through multiple ASMs through trial and error, with decreasing duration of therapy for each subsequent treatment. This creates an opportunity for a more effective and better-tolerated option like Vormatrigine to potentially capture significant market share.

Forward-Looking Statements

Praxis outlined an ambitious timeline for Vormatrigine’s development, with multiple catalysts expected in the coming quarters:

1. POWER1 Phase 2/3 study to be finalized in Q4 2025

2. POWER2 Phase 2/3 study to initiate in Q3 2025 with enrollment completion expected in 2H 2026

3. POWER3 monotherapy study to initiate in 1H 2026

Beyond Vormatrigine, the company has several other programs advancing toward key milestones, including Relutrigine for DEEs with topline results from the EMBOLD study expected in 1H 2026, and Ulixacaltamide for essential tremor with topline results from the ESSENTIAL3 studies expected in early fall 2025.

With a cash runway extending into 2028, Praxis appears well-positioned to execute its clinical development strategy across multiple CNS indications. The stock’s premarket surge of 4.83% to $56.70 reflects investor optimism about the RADIANT results and the company’s broader pipeline potential.

Full presentation:

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