Clinical Development and Trials - Praxis anticipates four pivotal readouts in 2025, with three late-stage assets having blockbuster potential and four commercial assets expected by 2028[1][2]. - The Essential3 program for ulixacaltamide in essential tremor is on track for an interim analysis in Q1 2025, with an NDA filing expected in 2025[1][4]. - Enrollment in the EMBOLD study for relutrigine cohort 2 is progressing, targeting an NDA filing in 2026[1][3]. - The RADIANT study for vormatrigine is expected to provide topline results in the first half of 2025, with the POWER1 study results anticipated by the end of 2025[1][3]. - Praxis plans to re-initiate a study of ulixacaltamide in Parkinson's disease following positive results from the Essential3 program[5]. - Praxis is currently focused on the expected timing of clinical trials and regulatory submissions for its programs[18]. Financial Position - Praxis has approximately $470 million in cash and investments at the end of 2024, supporting operations into 2028[1]. Study Results and Patient Engagement - The EMBOLD study for relutrigine showed a 46% placebo-adjusted reduction in monthly motor seizures over 16 weeks, with 77% reduction in ongoing open-label extension[7][11]. - Over 100,000 patients have expressed interest in participating in the Essential3 program since recruitment began in November 2023[4]. - The EMPOWER observational study has enrolled over 2,000 patients to characterize seizure burden, with early results shared at the 2024 American Epilepsy Society Annual Meeting[7]. Licensing and Collaborations - UCB has licensed the KCNT1 small molecule candidate for global development, with potential milestone payments totaling up to approximately $100 million[8]. Forward-Looking Statements - The company emphasizes that its forward-looking statements are based on information currently known and should not be solely relied upon[18]. - Praxis has no obligation to publicly update or revise any forward-looking statements made[18].

Core Insights - Praxis Precision Medicines, Inc. announced an interim analysis outcome for Study 1 of the Essential3 program, recommending the study be stopped for futility due to unlikely success in meeting primary efficacy endpoints [2] - Despite the interim analysis, the company will continue both Study 1 and Study 2, with topline results expected in Q3 2025, and will decide on NDA submission based on final results [3] - The company remains focused on other development programs, including upcoming topline results from studies on focal onset seizures and generalized epilepsy, and plans to initiate a registrational study for relutrigine by mid-2025 [4] Study Updates - The Independent Data Monitoring Committee (IDMC) advised Praxis to stop Study 1 for futility, suggesting alternative analysis methods for the final dataset [2] - Praxis will continue with both Study 1 and Study 2, expecting topline results in Q3 2025 [3] Financial Overview - As of December 31, 2024, Praxis reported $469.5 million in cash, cash equivalents, and marketable securities, a significant increase from $81.3 million in 2023, primarily due to public offerings [9] - The company recognized collaboration revenue of $7.5 million for Q4 2024 and $8.6 million for the full year, related to an agreement with UCB [10] - Research and development expenses rose to $56.3 million in Q4 2024 from $18.4 million in Q4 2023, totaling $152.4 million for the year, driven by increased costs associated with the Cerebrum™ platform [11] Operational Highlights - Praxis is advancing multiple programs, including the RADIANT and POWER1 studies for focal onset seizures and generalized epilepsy, with topline results expected in 2025 [4][6] - The company has received Rare Pediatric Disease Designation for relutrigine for Dravet Syndrome, marking its third such designation [6] - The EMBOLD study is currently enrolling patients, with topline results anticipated in the first half of 2026 [6] Shareholder Information - As of December 31, 2024, Praxis had 19.4 million shares of common stock outstanding [15]

Company Overview - Praxis Precision Medicines, Inc. (PRAX) is expected to report a quarterly loss of $2.91 per share, reflecting a year-over-year change of +2% [3] - Revenues are anticipated to be $0.48 million, which is a decrease of 7.7% from the same quarter last year [3] Earnings Estimates and Trends - The consensus EPS estimate has been revised 0.49% lower in the last 30 days, indicating a reassessment by analysts [4] - The Most Accurate Estimate for Praxis is higher than the Zacks Consensus Estimate, resulting in an Earnings ESP of +4.07% [10][11] - The company currently holds a Zacks Rank of 3, suggesting a likelihood of beating the consensus EPS estimate [11] Earnings Surprise History - In the last reported quarter, Praxis was expected to post a loss of $2.01 per share but actually reported a loss of $2.75, resulting in a surprise of -36.82% [12] - Over the past four quarters, the company has beaten consensus EPS estimates two times [13] Industry Context - Intellia Therapeutics, Inc. (NTLA), another player in the Zacks Medical - Biomedical and Genetics industry, is expected to report a loss of $1.32 per share, with revenues projected at $8.82 million, reflecting a significant year-over-year increase of 559.4% [17] - Intellia's consensus EPS estimate has been revised 2.5% higher in the last 30 days, and it has an Earnings ESP of 35.03%, indicating a strong likelihood of beating the consensus EPS estimate [18]

Company Overview - Praxis Precision Medicines, Inc. is a clinical-stage biopharmaceutical company focused on developing therapies for central nervous system (CNS) disorders characterized by neuronal excitation-inhibition imbalance [3] - The company utilizes genetic insights to create therapies for both rare and prevalent neurological disorders through its proprietary platforms, Cerebrum™ and Solidus™ [3] - Praxis has a diversified CNS portfolio that includes multiple programs across movement disorders and epilepsy, with four clinical-stage product candidates [3] Upcoming Events - The company will participate in the Guggenheim Securities SMID Cap Biotechnology Conference on February 6, 2025, at 2:30 p.m. EST, featuring a fireside chat [4] - Additionally, Praxis will take part in the Oppenheimer 35th Annual Healthcare Life Sciences Conference on February 11, 2025, at 11:20 a.m. EST, also featuring a fireside chat [4] - Both events will have live webcasts available, and replays will be accessible for 90 days on the company's website [2]

Core Insights - Praxis Precision Medicines, Inc. is a clinical-stage biopharmaceutical company focused on developing therapies for central nervous system disorders characterized by neuronal excitation-inhibition imbalance [1][3] - The company will present a corporate overview at the 43rd Annual J.P. Morgan Healthcare Conference on January 15, 2025 [1] - Praxis utilizes genetic insights to develop therapies for both rare and prevalent neurological disorders through its proprietary platforms, Cerebrum™ and Solidus™ [3] Company Overview - Praxis Precision Medicines specializes in translating genetic insights from genetic epilepsies into therapies for CNS disorders [3] - The company has established a diversified portfolio that includes multiple programs across movement disorders and epilepsy, featuring four clinical-stage product candidates [3] - The company’s approach leverages an understanding of shared biological targets and circuits in the brain [3] Event Information - A live webcast of the corporate overview will be available, with a replay accessible for 30 days on the company's website [2]

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 ...

Praxis Precision Medicines, Inc. (NASDAQ:PRAX) Q3 2024 Earnings Conference Call November 6, 2024 8:00 AM ET Company Participants Dan Ferry - IR, LifeSci Communications Marcio Souza - President & CEO Tim Kelly - CFO Conference Call Participants Ritu Baral - TD Cowen Yasmeen Rahimi - Piper Sandler Joon Lee - Truist Securities Francois Brisebois - Oppenheimer Yatin Suneja - Guggenheim Douglas Tsao - H.C. Wainwright Kambiz Yazdi - Jefferies Ami Fadia - Needham Joel Beatty - Baird Laura Chico - Wedbush Securitie ...

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 ...