Core Insights - The FDA has granted Rare Pediatric Disease Designation (RPDD) for relutrigine in the treatment of Dravet syndrome, marking a significant milestone for Praxis Precision Medicines [1][2] - Relutrigine has shown promising clinical data, demonstrating a 46% reduction in monthly motor seizures and over 30% of patients achieving seizure freedom [4] - Praxis plans to initiate an all-DEE trial (EMERALD) in the first half of 2025, which will include Dravet syndrome patients [1] Company Overview - Praxis Precision Medicines is a clinical-stage biopharmaceutical company focused on developing therapies for central nervous system disorders, particularly those related to neuronal excitation-inhibition imbalance [8] - The company utilizes genetic insights to create therapies for both rare and prevalent neurological disorders through its proprietary platforms [8] Product Information - Relutrigine is a first-in-class small molecule designed to treat developmental and epileptic encephalopathy (DEE) by inhibiting persistent sodium currents, which are key drivers of seizure symptoms [5] - The drug has received Orphan Drug Designation (ODD) and RPDD from the FDA, as well as ODD from the European Medicines Agency for SCN2A and SCN8A DEEs [5] Clinical Data - In the EMBOLD study, relutrigine demonstrated a placebo-adjusted monthly motor seizure reduction of 46% and a 77% reduction in median seizure rate during long-term extension [4] - The company is currently enrolling patients in a second registrational cohort for SCN2A and SCN8A patients, with topline results expected in the first half of 2026 [4]

Core Insights - Praxis Precision Medicines, Inc. is a clinical-stage biopharmaceutical company focused on developing therapies for central nervous system disorders related to neuronal excitation-inhibition imbalance [1] - The company will participate in two investor conferences in December 2024, including a fireside chat at the Piper Sandler 36th Annual Healthcare Conference and a panel at the Oppenheimer Movers in Rare Disease Summit [2][3] Company Overview - Praxis is leveraging genetic insights from genetic epilepsies to create therapies for CNS disorders, utilizing its proprietary platforms, Cerebrum™ and Solidus™ [5] - The company has a diversified CNS portfolio with multiple programs targeting movement disorders and epilepsy, featuring four clinical-stage product candidates [5]

BOSTON, Nov. 06, 2024 (GLOBE NEWSWIRE) -- Praxis Precision Medicines, Inc. (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 that management will be participating in three upcoming investor conferences in November 2024. Praxis management will participate in a fireside chat at the Truist Securities BioPharma Symposium, ta ...

Financial Data and Key Metrics Changes - In Q3 2024, Praxis reported operating expenses of $57.1 million, with $41.9 million allocated to R&D and $15.3 million to G&A, reflecting increased clinical activity [30] - The company ended Q3 with $411.2 million in cash, cash equivalents, and marketable securities, a significant increase from $81.3 million at the end of 2023, primarily due to net proceeds from follow-on public offerings [31] Business Line Data and Key Metrics Changes - The Phase 3 study in Essential Tremor (Essential3) for ulixacaltamide is progressing well, with interim analysis results expected in Q1 2025 [6][17] - Positive top-line results were reported for relutrigine in the Phase 2 EMBOLD trial, showing a 46% reduction in motor seizures versus placebo, with one-third of patients achieving seizure-free status [8][24] - Vormatrigine is advancing in the ENERGY clinical program, with an observational study (EMPOWER) attracting over 1,000 patient registrations [9] Market Data and Key Metrics Changes - The U.S. market is expected to account for approximately 70% of the revenue potential in developmental and epileptic encephalopathies (DEEs), with around 200,000 patients in the U.S. [58] - The company is targeting a multibillion-dollar opportunity with four programs in registration [6] Company Strategy and Development Direction - Praxis aims to deliver precision therapies for patients with CNS disorders, with a focus on advancing its pipeline and preparing for NDA submissions in 2025 [10][29] - The company is exploring the potential of vormatrigine in pain management, leveraging its pharmacological profile [20][22] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the execution of the interim analysis for ulixacaltamide, emphasizing the importance of delivering a successful program for NDA filing [40] - The company is preparing to restart its Parkinson's disease program contingent on the success of the Essential Tremor study [44] Other Important Information - The Essential3 program includes two concurrent Phase 3 studies, with a focus on patient recruitment and managing treatment variability [14][82] - Elsunersen is being studied in Brazil, with ongoing assessments of safety and efficacy in a severe disease population [28][72] Q&A Session Summary Question: Clarification on relutrigine's expanded cohort enrollment criteria - Management confirmed that the expanded cohort will have a different starting dose but similar inclusion criteria, aiming for a more efficacious outcome [34] Question: Timing of interim analysis and its implications - Management explained that the interim analysis was moved to Q1 2025 to ensure no negative influence on Study 2 results, emphasizing the importance of a successful program [38][42] Question: Inclusion of Lennox-Gastaut in DEE study - Management confirmed that Lennox-Gastaut patients will be included, focusing on the sodium channel mechanism's relevance [46] Question: Enrollment status in Essential3 studies - Management indicated that there has been no slowdown in patient randomization, maintaining a steady enrollment rate [87] Question: Commercial potential in DEEs - Management projected that approximately 70% of the revenue potential will come from the U.S. market, with a significant patient population to target [58]

Praxis Precision Medicines, Inc. (PRAX) came out with a quarterly loss of $2.75 per share versus the Zacks Consensus Estimate of a loss of $2.01. This compares to loss of $2.70 per share a year ago. These figures are adjusted for non-recurring items.This quarterly report represents an earnings surprise of -36.82%. A quarter ago, it was expected that this company would post a loss of $2.38 per share when it actually produced a loss of $1.74, delivering a surprise of 26.89%.Over the last four quarters, the co ...

Interim analysis for Study 1 of Essential3 Phase 3 program for ulixacaltamide in essential tremor (ET) confirmed for Q1 2025; NDA filing anticipated in 2025 Registrational Cohort 2 of EMBOLD study recruiting following unprecedented seizure freedom seen in positive topline EMBOLD results for Cohort 1 in SCN2A and SCN8A developmental and epileptic encephalopathies (DEEs) Vormatrigine (PRAX-628) on track for topline from POWER1 study in focal epilepsy and RADIANT study in focal and generalized epilepsy in 2025 ...

BOSTON, Nov. 01, 2024 (GLOBE NEWSWIRE) -- Praxis Precision Medicines, Inc. (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 it will report its financial results from the third quarter ended September 30, 2024, before the financial markets open on Wednesday, November 6, 2024. The Company will host a live webcast the same ...

Praxis Precision Medicines, Inc. (PRAX) shares ended the last trading session 8.4% higher at $66.30. The jump came on an impressive volume with a higher-than-average number of shares changing hands in the session. This compares to the stock's 0.7% gain over the past four weeks. The recent surge in the stock price is driven by positive investor expectations regarding the company's pipeline. Praxis is currently assessing its lead candidate, ulixacaltamide, in two separate phase III studies under its Essential ...

Shares of Praxis Precision Medicines (PRAX) gained 4.2% on Sept. 4 after the company announced positive top-line data from its mid-stage EMBOLD proof of concept study evaluating relutrigine (PRAX-562) to treat SCN2A and SCN8A developmental and epileptic encephalopathy (DEE) patients. Praxis' EMBOLD Study Reduces Seizures in Epilepsy Patients Data from the phase II EMBOLD study of relutrigine showed that patients treated with relutrigine experienced a placebo-adjusted reduction of 46% in countable monthly mo ...

BOSTON, Sept. 04, 2024 (GLOBE NEWSWIRE) -- Praxis Precision Medicines, Inc. (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 that that it will present preclinical and clinical data from three of its epilepsy programs at the International League Against Epilepsy (ILAE) 15th European Epilepsy Congress (EEC), being held fr ...