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 ...

Core Viewpoint - Praxis Precision Medicines announced positive results from its mid-stage EMBOLD study for relutrigine, leading to a 4.2% increase in share price on September 4 [1] Group 1: EMBOLD Study Results - The phase II EMBOLD study showed a placebo-adjusted reduction of 46% in monthly motor seizures for patients treated with relutrigine [2] - Over 30% of evaluable patients achieved a 28-day seizure-free status while on relutrigine, compared to none on placebo [2] - Patients also reported significant improvements in disruptive behavior, communication, seizure severity, and alertness after 16 weeks of treatment [3] Group 2: Safety and Efficacy - Relutrigine was found to be "generally safe and well tolerated," with no patients requiring dose reduction during the study [5] - Seven patients increased their daily dose to the maximum allowed without adverse effects leading to discontinuation [5] - Adverse events were mostly mild to moderate in severity [5] Group 3: Future Developments - Based on the positive outcomes, Praxis has initiated the registrational phase of the EMBOLD study for SCN2A and SCN8A DEE, with no current approved treatments for these conditions [6] - Relutrigine is a first-in-class investigational drug targeting persistent sodium current, a key driver of seizures in the specified conditions [7] Group 4: Pipeline and Competitors - Praxis is also developing ulixacaltamide for essential tremor, with top-line results expected in the second half of 2024 [8] - Another candidate, PRAX-628, is being evaluated for focal onset seizures, with plans for four studies to support global regulatory registrations [9] - Competitor Xenon Pharmaceuticals is developing therapies for epilepsy, including a selective sodium channel inhibitor currently in phase II studies [10]

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 ...

Placebo-adjusted monthly motor seizure reduction of 46% during double-blind period Over 30% of patients achieved seizure freedom status while on relutrigine Meaningful gains observed in alertness, communication and seizure severity 75% reduction in median seizure rate seen for patients in the long-term extension Registrational phase of the EMBOLD study for SCN2A and 8A initiated BOSTON, Mass., Sept. 03, 2024 (GLOBE NEWSWIRE) -- Praxis Precision Medicines, Inc. (NASDAQ: PRAX), a clinical-stage biopharmaceuti ...

BOSTON, Mass., Sept. 02, 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 it will share the topline results from the Phase 2 EMBOLD study of relutrigine (PRAX562) in SCN2A and SCN8A Developmental and Epileptic Encephalopathies (DEEs) on Tuesday, September 3, ...

Praxis Precision Medicines, Inc. (PRAX) could be a solid choice for investors given its recent upgrade to a Zacks Rank #2 (Buy). This rating change essentially reflects an upward trend in earnings estimates -- one of the most powerful forces impacting stock prices. A company's changing earnings picture is at the core of the Zacks rating. The system tracks the Zacks Consensus Estimate -- the consensus measure of EPS estimates from the sell-side analysts covering the stock -- for the current and following yea ...