Ami Fadia

Douglas Tsao

Joon Lee

Ritu Baral

Yasmeen Rahimi

Yatin Suneja

François Brisebois

Joel Beatty

Kambiz Yazdi

Brian Skorney

David Hoang

Doug Tsao

Jay Olson

Laura Chico

Rudy Li

- Praxis's **elixacalamide hydrochloride** achieved positive results in its **Essential Three Phase III program**, marking the **first drug to demonstrate positive Phase III outcomes for essential tremor**.  **[0001689548_2188222_1]** **[0001689548_2188222_1]** 
- **Both studies met their primary endpoints**, and the drug was found to be generally **safe and well tolerated** with **no drug-related severe adverse events**.  **[0001689548_2188222_1]** **[0001689548_2188222_1]** **[0001689548_2188222_4]** 
- Praxis has **submitted a pre-NDA meeting request to the FDA** and has **completed 80% of the required documents for the NDA submission**.  **[0001689548_2188222_1]** **[0001689548_2188222_1]** **[0001689548_2188222_7]** 
- The company estimates potential sales to be at least in the **mid to high single-digit billions**, with reasonable estimates reaching the **mid-teens for billions**, addressing an unmet need for an estimated **seven million people in the United States** affected by essential tremor.  **[0001689548_2188222_1]** **[0001689548_2188222_1]** **[0001689548_2188222_7]**

PRAX Announces Positive Phase III Results for Essential Tremor Drug

- Praxis Precision Medicines, Inc. announced **positive topline results** from its **Phase 3 Essential3 program of ulixacaltamide** for essential tremor (ET) on October 16, 2025.  **[1689548_0001689548-25-000100_prax-20251016.htm:0]** **[1689548_0001689548-25-000100_prax-20251016.htm:1]** 
- In **Study 1**, ulixacaltamide demonstrated a **statistically significant and clinically meaningful 4.3 point mean improvement in the mADL11 score at Week 8** (p<0.0001) compared to placebo.  **[1689548_0001689548-25-000100_prax-20251016.htm:1]** **[1689548_0001689548-25-000100_e3_toplinexfinaloctober2.htm:4]** 
- **Study 2** showed that **55% of patients in the ulixacaltamide arm maintained response** versus 33% in the placebo group (p=0.037).  **[1689548_0001689548-25-000100_prax-20251016.htm:2]**  
- Ulixacaltamide was **generally well tolerated with no drug-related serious adverse events**.  **[1689548_0001689548-25-000100_prax-20251016.htm:2]**  
- The company has submitted a **pre-NDA meeting request to the FDA** and plans to submit the **NDA by early 2026**.  **[1689548_0001689548-25-000100_prax-20251016.htm:2]** **[1689548_0001689548-25-000100_e3_toplinexfinaloctober2.htm:2]**

Praxis Precision Medicines Announces Positive Topline Results for Essential3 Program

- Praxis Precision Medicines (PRAX) reported that its drug candidate, vormatrigine, achieved a **56% sustained overall seizure reduction rate** in the RADIAN study.  **[1689548_tx_381223_2]**  Additionally, **22% of patients saw 100% seizure reduction** in the last 28 days, with **14% being seizure-free over the entire eight-week treatment period**.  **[1689548_tx_381223_5]**  These results are considered "**best of disease**" and represent the "**largest effects ever seen**" at eight weeks compared to other drugs.  **[1689548_tx_381223_6]** 
- The company expects the ongoing Power One study to show **at or better than 56% seizure reduction**.  **[1689548_tx_381223_10]**  The larger Power Two study is starting in the last month of Q3 2025, with recruitment expected to take **10 to 12 months** and finalization late next year.  **[1689548_tx_381223_11]**  This study, using a higher 40mg dose, is anticipated to show an **even better response** and, combined with Power One, could enable regulatory submission (NDA) in the next **18 months or so**.  **[1689548_tx_381223_8]** **[1689548_tx_381223_11]** 
- While the RADIAN study had higher-than-expected discontinuation rates, these were attributed to patient counseling and site experience rather than treatment-emergent adverse events (TEAEs), which were significantly lower than other drugs.  **[1689548_tx_381223_8]** **[1689548_tx_381223_9]**  Power One is already showing much lower discontinuation rates.  **[1689548_tx_381223_9]** 
- Vormatrigine holds significant commercial potential, with a projected **$1.5 billion peak sale** in the refractory epilepsy market, and potentially over **$3 billion** with broader use, including replacing less effective current treatments like levetiracetam.  **[1689548_tx_381223_10]**

Praxis Precision Medicines Discusses Vormatrigine Clinical Data and Commercial Outlook