Core Insights - Praxis Precision Medicines announced topline results from the Phase 2 RADIANT study, showing that dosing with vormatrigine over 8 weeks led to a 56.3% median reduction in seizure frequency [1] - Approximately 22% of patients achieved a 100% reduction in seizure frequency during the last 28 days of treatment [1] - Over 54% of patients experienced a 50% response in the first week, indicating a rapid and sustained response [1] - Vormatrigine demonstrated a favorable safety profile and was generally well tolerated [1] Company Overview - Praxis Precision Medicines is a clinical-stage biopharmaceutical company focused on developing therapies for central nervous system disorders characterized by neuronal excitation-inhibition imbalance [7] - The company is applying genetic insights to discover and develop therapies for both rare and prevalent neurological disorders through its proprietary platforms [7] - Praxis has established a diversified CNS portfolio, including multiple programs across epilepsy and movement disorders, with four clinical-stage product candidates [7] Product Details - Vormatrigine is a next-generation, functionally selective small molecule targeting hyperexcitable sodium channels in the brain, developed as a once-daily oral treatment for adult focal onset seizures and generalized epilepsy [6] - Preclinical data suggests that vormatrigine has the potential to be best-in-class for focal epilepsy, demonstrating superior selectivity for disease-state NaV channel hyperexcitability [6] - Initial results from the RADIANT study indicate robust seizure reduction and a generally safe and well-tolerated profile for vormatrigine [6] Future Developments - Praxis plans to complete the pivotal 12-week POWER1 study in Q4 2025 and expects to initiate the POWER2 study shortly based on the results from RADIANT [2] - Additional data from the RADIANT study will be presented at the 36th International Epilepsy Congress on August 31, 2025, in Lisbon, Portugal [4] - A late-breaker abstract for the full study results will be submitted for presentation at the American Epilepsy Society Annual Meeting in December 2025 in Atlanta, Georgia [4]

BOSTON, July 29, 2025 (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 topline results from the RADIANT trial of vormatrigine and its financial results from the second quarter ended June 30, 2025, before the financial markets open on Monday, August 4, 2 ...

Core Insights - The U.S. FDA has granted Breakthrough Therapy Designation (BTD) for relutrigine, a sodium channel functional state modulator, aimed at treating pediatric patients with SCN2A and SCN8A developmental and epileptic encephalopathies (DEEs) [1][2] - The EMBOLD cohort 2 pivotal trial is expected to deliver topline results in the first half of 2026, with a New Drug Application (NDA) filing to follow [1][3] - Praxis has initiated the EMERALD study to evaluate relutrigine in a broader DEE patient population [1][3] Clinical Trial Results - The EMBOLD cohort 1 study included a severely affected DEE population, with patients averaging three failed treatments prior to enrollment [2] - Results from the open-label extension of the study showed approximately 90% reduction in seizures and an average of 67 days without seizures compared to 3 days in the baseline period [2][4] - During the double-blind period, a placebo-adjusted monthly motor seizure reduction of 46% was observed, with over 30% of patients achieving seizure freedom while on relutrigine [2][4] Drug Mechanism and Designation - Relutrigine is a first-in-class small molecule that preferentially inhibits persistent sodium current, a key driver of seizure symptoms in severe DEEs [4] - The drug has received Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation from the FDA for SCN2A-DEE and SCN8A-DEE, in addition to the BTD [4][5] Company Overview - Praxis Precision Medicines is focused on developing therapies for CNS disorders characterized by neuronal excitation-inhibition imbalance, utilizing genetic insights [6] - The company has a diversified CNS portfolio, including multiple programs across epilepsy and movement disorders, with four clinical-stage product candidates [6]

Summary of Praxis Precision Medicines Conference Call Company Overview - **Company**: Praxis Precision Medicines (PRAX) - **Event**: Jefferies Global Healthcare Conference 2025 - **Date**: June 04, 2025 Key Points on Clinical Development Sodium Channel Modulators - Praxis is developing sodium channel modulators, specifically vermatrogene and rilutrogen, targeting epilepsy treatment [2][4] - Vermatrogene is focused on adult patients with focal onset seizures and generalized epilepsies, with two ongoing studies [4][5] - Rilutrogen is aimed at pediatric patients with SCN2A and SCN8A mutations, which are difficult to treat [37] Clinical Trials and Expectations - The first study, Radiance, is expected to report results mid-year, focusing on pharmacology and seizure reduction [5][8] - A significant cohort of patients is involved, with a target seizure reduction of 40-60% [6][7] - The Power One study will follow, with results anticipated by the end of the year [7][8] - A registrational package for vermatrogene is expected to be completed within the next 12-18 months [8] Patient Population and Inclusion Criteria - High screen failure rates in trials are viewed positively, indicating rigorous patient selection [6][31] - Inclusion of patients on background sodium channel blockers is justified by preclinical data showing no negative impact on efficacy [20][22] Market Potential - There are approximately 3 million adults in the U.S. with epilepsy, many of whom are undertreated [11] - Both vermatrogene and rilutrogen have significant market opportunities, with potential revenues reaching hundreds of millions to billions [45] Rilutrogen Development - Rilutrogen has shown over 40% seizure reduction in a 16-week study, with additional benefits in communication and behavior [37][38] - The company is adding registrational cohorts to expedite the process for patients with severe conditions [40] - A new study, EMERALD, is being initiated to treat seizures in a broader patient population [42] Future Catalysts - Upcoming milestones include results from the Radiance and Power One studies, as well as the nomination of ASO PRX100 for autism spectrum disorder [54] - The company is also working on other programs, including Syngap and essential tremor treatments, with potential NDA submissions in the future [56] Additional Insights - The company emphasizes the importance of understanding patient needs and drug interactions in developing effective treatments [10][12] - There is a focus on multimodal therapies, similar to approaches seen in spinal muscular atrophy (SMA) treatments [50] - The enrollment process for trials is robust, with a significant percentage of patients coming from a dedicated initiative to understand epilepsy better [32][35] This summary encapsulates the critical aspects of Praxis Precision Medicines' conference call, highlighting their ongoing clinical developments, market potential, and future catalysts.

Financial Data and Key Metrics Changes - The company is focused on executing across its entire portfolio with four programs in late-stage development expected to reach pre-commercial stages within the next twelve to eighteen months [4][5] - The upcoming catalyst for vormatrogene, a sodium channel modulator for common epilepsies, includes a phase two open-label study with data expected by midyear [5][6] Business Line Data and Key Metrics Changes - The company has multiple studies planned for its key assets, including vormatrogene and lexicaltimide, with significant data readouts anticipated in the coming months [5][7][8] - Lexicaltimide's studies are continuing despite an interim review board's recommendation to stop due to futility, with results expected in Q3 [8][9] Market Data and Key Metrics Changes - The focal epilepsy space is described as competitive, prompting the company to stagger the enrollment of its registrational studies to avoid internal competition for patients [6][7] - The company is also expanding its focus on ralutrogene, which targets developmental epilepsies, with a broader study planned to enroll patients based on seizure burden rather than genetic mutation [10][12] Company Strategy and Development Direction - The company aims to derisk its assets through comprehensive data collection and analysis before proceeding with regulatory filings [33][34] - There is a strategic emphasis on balancing the need for rapid drug delivery to patients with the necessity of thorough clinical characterization to ensure market acceptance [27][28] Management's Comments on Operating Environment and Future Outlook - Management acknowledges the challenges in the epilepsy treatment landscape but emphasizes the importance of their unique approaches and methodologies in clinical trials [20][21] - The company is committed to addressing the significant unmet needs in the epilepsy market, particularly for patients with severe conditions [34][35] Other Important Information - The company is initiating several broader studies and expects to provide updates on these initiatives throughout the year [15][16] - There is a focus on leveraging existing data to inform future studies and improve patient outcomes [10][12] Q&A Session Summary Question: How does the company manage to enroll quickly while maintaining data quality compared to competitors? - The company utilizes a prescreening initiative that accounts for about 30% of patient throughput, significantly speeding up recruitment [21][22] - Management emphasizes a micromanagement approach to identify and address enrollment impediments daily [23][24] Question: How does the company balance optimizing programs versus getting drugs to market quickly? - The company focuses on achieving proof of concept and derisking assets before applying more capital, ensuring a balance between patient benefit and shareholder return [33][34] Question: How does the company view the potential overlap between its various drug candidates? - Management believes that while there may be overlaps, each drug serves distinct patient needs, and the company will continue to develop both small molecules and ASOs as appropriate [40][41]

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 epilepsy and movement disorders, with four clinical-stage product candidates [3] Upcoming Events - The company will participate in three investor conferences, including a fireside chat at the H.C. Wainwright 3rd Annual BioConnect Investor Conference on May 20 at 9:30 am ET [4] - Praxis management will also attend the Mizuho Neuro & Ophthalmology Summit in New York City on May 21 [4] - A corporate overview will be presented at the Jefferies Global Healthcare Conference on June 4 at 8:10 am ET [4] Investor Engagement - The company will be available for one-on-one meetings during all events, encouraging interested investors to contact their representatives [1] - Webcasts of the events will be accessible for 90 days through the "Events & Presentations" page on the company's website [2]

BOSTON, May 05, 2025 (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, hosted a virtual investor event on its clinical programs in developmental and epileptic encephalopathies (DEEs) on Friday, May 2, 2025. "We were excited to highlight the significant opportunity in DEEs and progress ...

Pipeline and Catalysts - The company has 4 assets in late stage development[6] - Clinical readouts are expected in the next 4 quarters[6] - Topline results for the RADIANT open label study of Vormatrigine in focal onset or generalized epileptic seizures are expected by mid-2025[17, 37] - Topline results from the ESSENTIAL3 studies of Ulixacaltamide in Essential Tremor are expected in Q3 2025[17] - EMBOLD Cohort 2 topline results for Relutrigine in SCN2A GoF and SCN8A are expected in 1H 2026, with NDA filing in 2026[17] Epilepsy Portfolio - The US market opportunity for Vormatrigine in common epilepsy (3.5 million US prevalence) is estimated to be greater than $2.5 billion[20] - The US market opportunity for Relutrigine in developmental epilepsies (over 200,000 patients) is estimated to be greater than $3 billion[20] - The US market opportunity for Elsunersen in SCN2A genetically typified developmental epilepsies (~2,000 patients) is estimated to be greater than $500 million[20] - EMBRAVE Part 1 data showed a 39% mean and 43% median reduction in seizures from baseline in SCN2A GoF patients treated with Elsunersen[82] Essential Tremor - Up to 50% of Essential Tremor patients are not receiving treatment for their condition[105] - 85% of neurologist visits are for patients seeking Essential Tremor treatment[108]

Praxis Precision Medicines (PRAX) Update / Briefing May 02, 2025 10:00 AM ET Speaker0 Good morning, everyone, and thanks for being with us here today in the office in Boston. I really appreciate it. And everyone else in the webcast as well for our GE portfolio updates at Praxis. As you can see here at the beginning, the way we tend to think about everything at Praxis is daring for more. So we're there for more today as well. I think it's a great show here in a not so sunny day in Boston, and hopefully, gonn ...

Financial Performance - The company reported a net loss of $69.3 million for the three months ended March 31, 2025, with an accumulated deficit of $906.0 million[101]. - Net loss for the three months ended March 31, 2025, was $69.3 million, compared to a net loss of $39.6 million for the same period in 2024[120]. - Net cash used in operating activities for the three months ended March 31, 2025, was $53.0 million, compared to $20.9 million for the same period in 2024[135]. - Total operating expenses for the three months ended March 31, 2025, were $74.7 million, up from $42.3 million in 2024[120]. - Collaboration revenue decreased by $0.4 million to $0 for the three months ended March 31, 2025, due to the completion of research service obligations under the Collaboration Agreement with UCB[121]. Research and Development - Research and development expenses for the three months ended March 31, 2025, totaled $60.8 million, a significant increase from $27.0 million in the same period of 2024[112]. - The Cerebrum™ platform incurred $42.8 million in research and development expenses for the first quarter of 2025, compared to $12.9 million in the prior year[112]. - Research and development expenses increased by $33.8 million to $60.8 million for the three months ended March 31, 2025, primarily driven by increased spending on the Cerebrum™ platform[122]. - The company plans to initiate the Phase 3 registrational study for elsunersen (formerly PRAX-222) in mid-2025, with topline results expected in the first half of 2026[99]. - The EMPOWER study for vormatrigine in epilepsy patients was initiated in Q3 2024, with topline results expected by mid-2025[97]. - The company anticipates nominating a development candidate for PRAX-100 by mid-2025, with additional candidates expected by the end of 2025[99]. - The company relies on external consultants and organizations for its research and development activities, operating in a "virtual" model since inception[100]. - The Independent Data Monitoring Committee recommended stopping the ulixacaltamide study for futility, but the company will continue both studies to completion[97]. Financial Position and Resources - The company has cash, cash equivalents, and marketable securities of $472.0 million as of March 31, 2025, expected to fund operations into 2028[106]. - Cash, cash equivalents, and marketable securities as of March 31, 2025, totaled $472.0 million[126]. - The company expects substantial increases in expenses related to ongoing research and development activities, particularly for clinical trials[139]. - The company anticipates that its cash resources will be sufficient to fund operations into 2028, based on current estimates[140]. - The company currently lacks committed external sources of funds, which may impact its ability to access capital during market volatility[145]. - If additional funds are raised through collaborations or licensing, the company may have to relinquish valuable rights to its technologies or future revenue streams[146]. - The company raised $1.2 billion in aggregate cash proceeds from various transactions since inception through March 31, 2025[126]. Accounting and Risk - The management's financial analysis is based on condensed consolidated financial statements prepared under U.S. GAAP, requiring estimates and assumptions that may differ from actual results[147]. - There have been no changes to critical accounting policies from those described in the Annual Report on Form 10-K filed on February 28, 2025, except as disclosed in the quarterly report[148]. - Recently issued accounting standards are not expected to have a material impact on the company's financial statements or operations[149]. - The company is exposed to market risk related to interest rate changes, but an immediate change of 100 basis points would not materially impact its financial position[150].