Group 1 - Praxis Precision Medicines reported a quarterly loss of $2.94 per share, slightly better than the Zacks Consensus Estimate of a loss of $2.96, and an improvement from a loss of $2.97 per share a year ago, indicating an earnings surprise of 0.68% [1] - The company achieved revenues of $7.46 million for the quarter ended December 2024, significantly surpassing the Zacks Consensus Estimate by 1,467.86%, compared to revenues of $0.52 million in the same quarter last year [2] - The stock has underperformed, losing about 15.5% since the beginning of the year, while the S&P 500 has declined by only 0.3% [3] Group 2 - The earnings outlook for Praxis Precision Medicines is mixed, with the current consensus EPS estimate for the coming quarter at -$2.80 on $0.5 million in revenues, and -$12.22 on $1.91 million in revenues for the current fiscal year [7] - The Zacks Industry Rank for Medical - Biomedical and Genetics is in the top 29% of over 250 Zacks industries, indicating that the industry outlook can significantly impact stock performance [8] - Atara Biotherapeutics, a peer in the same industry, is expected to report a quarterly loss of $3.82 per share, reflecting a year-over-year change of +72.7%, with revenues projected to be $13.1 million, up 208.2% from the previous year [9]

Financial Performance - The company reported net losses of $182.8 million and $123.3 million for the years ended December 31, 2024 and 2023, respectively, with an accumulated deficit of $836.7 million as of December 31, 2024[487]. - The net loss for 2024 was $182.819 million, compared to a net loss of $123.277 million in 2023, indicating a year-over-year increase in losses of approximately 48%[577]. - The net loss for the quarter ending September 30, 2024, was $58,679, compared to a net loss of $51,910 for the quarter ending June 30, 2024, indicating a sequential increase in losses of about 13.5%[584]. - The company reported a net loss of $32,677 for the quarter ending June 30, 2024, which was an improvement compared to a net loss of $39,553 for the quarter ending March 31, 2024[584]. - The balance of accumulated deficit increased to $(836,740) as of December 31, 2024, from $(778,061) at September 30, 2024, reflecting a worsening of approximately 7.5%[584]. Revenue Generation - The company has not generated any revenue from product sales since inception and does not expect to do so for several years, but recognized $8.6 million and $2.4 million of collaboration revenue from a partnership with UCB Biopharma SRL during the years ended December 31, 2024 and 2023, respectively[502]. - Collaboration revenue rose by $6.1 million to $8.6 million in 2024, primarily due to an option exercise fee from UCB[519]. - The Company recognized $8.6 million in collaboration revenue for the year ended December 31, 2024, compared to $2.4 million for the year ended December 31, 2023[680]. - Upon UCB exercising its option in December 2024, the Company recognized a $6.0 million option exercise fee as collaboration revenue[679]. Cash and Funding - The company has cash, cash equivalents, and marketable securities of $469.5 million as of December 31, 2024, which are expected to fund operations into 2028[498]. - The company raised $1.1 billion in aggregate cash proceeds from various stock offerings since inception[523]. - A public offering in April 2024 generated approximately $216.0 million in net proceeds from the sale of common stock[495]. - The company completed a public offering in January 2024, generating approximately $161.6 million in net proceeds after expenses[527]. - The company expects its cash and marketable securities will be sufficient to fund operations for at least one year from the issuance of the financial statements[598]. Research and Development - Total research and development expenses increased by $65.6 million to $152.4 million in 2024, up from $86.8 million in 2023[507]. - The company plans to continue advancing its product candidates and expanding its research and development activities, requiring substantial additional funding[488]. - The company anticipates that research and development expenses will be maintained or increase as product candidates advance through development[507]. - The company is focused on developing therapies for central nervous system disorders through its two proprietary platforms, Cerebrum™ and Solidus™, with multiple clinical-stage product candidates[592]. Stock and Equity - A reverse stock split of 1-for-15 was executed on November 28, 2023, affecting the company's common stock[499]. - The company's additional paid-in capital rose to $1.282 billion in 2024, up from $723.577 million in 2023, reflecting an increase of about 77%[575]. - The weighted average common shares outstanding increased to 17,906,794 in 2024 from 6,594,316 in 2023, indicating a growth of approximately 172%[577]. - The total number of shares outstanding increased from 17,755,506 on June 30, 2024, to 19,422,358 by December 31, 2024, representing an increase of approximately 9.4%[584]. Operating Expenses - Total operating expenses rose to $208.718 million in 2024, compared to $128.820 million in 2023, reflecting an increase of about 62%[577]. - General and administrative expenses increased by $14.3 million to $56.3 million in 2024, up from $42.1 million in 2023[518]. - The Cerebrum™ platform saw a $61.9 million increase in expenses, mainly driven by a $49.8 million rise in spending for the ulixacaltamide program[520]. - The company incurred stock-based compensation expenses of $41.4 million in 2024, up from $24.9 million in 2023, reflecting a 66% increase[590]. Clinical Trials and Studies - The Phase 3 Essential3 clinical trials for ulixacaltamide are ongoing, with topline results expected in the third quarter of 2025[483]. - The company announced positive results from the Photo-Paroxysmal Response study for vormatrigine in Q1 2024 and has initiated multiple studies to gather further data[484]. - The EMBOLD study for relutrigine has shown positive topline results from the first cohort, with the second cohort currently enrolling and results expected in the first half of 2026[484]. Tax and Deferred Assets - The company has U.S. federal and state net operating loss carryforwards that may offset future taxable income, beginning to expire in 2035[516]. - The company has not recorded any U.S. federal or state income tax benefits for net losses incurred since inception[516]. - The Company assesses the likelihood of recovering deferred tax assets based on future taxable income and establishes a valuation allowance if necessary[647]. Collaboration Agreements - As of December 31, 2024, the Company had an option and license agreement with UCB Biopharma SRL, entered into in December 2022, and a collaboration and license agreement with Tenacia Biotechnology, entered into in January 2024[635]. - The Company entered into a Collaboration Agreement with UCB in December 2022, receiving a $5.0 million upfront payment for research services[677]. - The Collaboration Agreement allows UCB to in-license global development and commercialization rights, with potential milestone payments totaling up to $98.5 million and tiered royalties on net sales[677].

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 ...