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]

Summary of the Conference Call Company and Industry Overview - The conference call is focused on Praxis, a company involved in CNS (Central Nervous System) drug development, particularly targeting epilepsy and autism spectrum disorders [1][4][6] - The discussion highlights the unmet medical needs in neurology, emphasizing the large therapeutic area and the potential for significant commercial opportunities [7][8] Core Points and Arguments Neurology Market Potential - Neurology is characterized by a significant "white space" where large unmet needs exist, particularly in conditions like epilepsy and autism spectrum disorders [7][8] - The company aims to address these needs through innovative drug development, focusing on both genetic and non-genetic factors [13][14] Rilutrogene Program - The rilutrogene program is highlighted as a key focus, with promising results from ongoing studies [22] - The potential market for rilutrogene is substantial, with estimates suggesting a significant growth in the patient population over the next decade [14][15] - The company anticipates a market opportunity of approximately $1 billion globally for rilutrogene, with half of that in the U.S. [17][18] EMERALD Program - The EMERALD program is introduced as a critical initiative aimed at addressing a broad patient population, with the potential to expand market reach significantly [15][23] - The program is expected to yield results in the first half of the next year [22] Praxis 100 Program - The Praxis 100 program represents the company's first venture into autism spectrum disorders, targeting a significant patient population with no current drugs in development [19][20] - The program aims to address genetic autism, which is a major cause of the condition [20] Important but Overlooked Content - The call emphasizes the importance of precision medicine in developing effective treatments for epilepsy, focusing on the pharmacological profile of rilutrogene [25][26] - The discussion includes insights into the unique binding properties of rilutrogene compared to existing treatments, highlighting its potential to selectively inhibit pathological firing without impairing normal neuronal function [30][31][32] - The company is leveraging advanced computational methods to understand drug interactions at a cellular level, which may provide a competitive edge in drug development [100][101] Conclusion - Praxis is positioned to capitalize on significant unmet needs in the neurology market through innovative drug development programs, particularly rilutrogene and the EMERALD and Praxis 100 initiatives [4][15][19] - The company is optimistic about the future growth of its patient population and the commercial opportunities that lie ahead, driven by advancements in precision medicine and a focus on addressing the underlying causes of neurological disorders [14][22][25]

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].

Financial Performance - Praxis reported a net loss of $69.3 million for Q1 2025, compared to a net loss of $39.6 million in Q1 2024[12]. - Net loss for Q1 2025 was $69,296,000, compared to a net loss of $39,553,000 in Q1 2024, representing an increase in loss of approximately 75.3%[26]. - Net loss per share attributable to common stockholders increased from $2.84 in Q1 2024 to $3.29 in Q1 2025, a rise of about 15.8%[26]. - Accumulated deficit grew from $836,740,000 on December 31, 2024, to $906,036,000 on March 31, 2025, an increase of about 8.3%[24]. Research and Development - Research and development expenses increased to $60.8 million for Q1 2025, up from $27.0 million in Q1 2024, primarily due to increased expenses related to the Cerebrum™ platform[10]. - Research and development expenses increased to $60,806,000 in Q1 2025, up from $26,984,000 in Q1 2024, reflecting a rise of approximately 125.5%[26]. - Total operating expenses rose to $74,728,000 in Q1 2025, compared to $42,317,000 in Q1 2024, an increase of about 76.4%[26]. - Praxis plans to initiate pivotal studies for relutrigine and elsunersen by mid-2025, targeting developmental and epileptic encephalopathy[1]. Cash and Investments - Praxis reported cash and investments of $472 million as of March 31, 2025, maintaining a runway into 2028[1]. - Cash and cash equivalents decreased by $49,805,000, from $215,372,000 to $165,567,000, representing a decrease of about 23.1%[24]. - Total assets decreased from $483,110,000 on December 31, 2024, to $478,736,000 on March 31, 2025, a decline of approximately 0.8%[24]. Collaboration Revenue - Praxis did not recognize any collaboration revenue in Q1 2025, a decrease from $0.4 million in Q1 2024, due to the completion of obligations under an agreement with UCB[9]. - Collaboration revenue dropped to $0 in Q1 2025, down from $431,000 in Q1 2024, indicating a significant decline[26]. Clinical Studies and Results - The registrational cohort of the EMBOLD study is on track for topline results no later than the first half of 2026[2]. - Topline results for vormatrigine from the RADIANT study are expected by mid-2025, with additional results from the POWER1 study anticipated in the second half of 2025[2]. - The EMPOWER registrational study has over 3,000 patients registered to date, supporting recruitment for the RADIANT and POWER1 studies[5]. - The Phase 1 food effect study for vormatrigine demonstrated that food intake does not significantly impact its absorption, enhancing dosing flexibility[3]. - The Essential3 study for ulixacaltamide is expected to complete topline readout in Q3 2025, despite an interim analysis recommending study stoppage for futility[5]. Other Income - Other income increased from $2,333,000 in Q1 2024 to $5,432,000 in Q1 2025, reflecting an increase of approximately 133.5%[26].

Corporate Update - Praxis Precision Medicines is on track for six major study readouts across four programs over the next 12 months, with pivotal studies for developmental and epileptic encephalopathy (DEE) programs set to begin in mid-2025 [1][2] - The company reported cash and investments of $472 million as of March 31, 2025, which is expected to fund operations into 2028 [1][6] Product Development - Vormatrigine is demonstrating a best-in-class safety profile, with new data showing no food effect and higher dosing tolerability [1][3] - The registrational cohort of the EMBOLD study is recruiting strongly, with topline results expected by the first half of 2026 [2][5] - The EMERALD study for relutrigine will be initiated in mid-2025, targeting a broader DEE patient population [2][5] - The EMBRAVE study with elsunersen is progressing well, with topline results expected in the first half of 2026 [2][5] Financial Performance - Research and development expenses increased to $60.8 million for Q1 2025, compared to $27.0 million in Q1 2024, primarily due to increased expenses related to the Cerebrum™ platform [8] - General and administrative expenses decreased to $13.9 million for Q1 2025, down from $15.3 million in Q1 2024 [9] - The net loss for Q1 2025 was $69.3 million, compared to a net loss of $39.6 million in Q1 2024 [10][21]

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 more 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, featuring four clinical-stage product candidates [3] Upcoming Event - Praxis will host a virtual investor event on May 2, 2025, from 10:00 a.m. to 11:30 a.m. EDT, focusing on its clinical programs in developmental and epileptic encephalopathies (DEEs) [1][2] - The event will be led by the Praxis management team, and a replay will be available for 90 days on the company's website [2] Clinical Studies - The company is conducting several studies, including: - The EMBOLD study evaluating relutrigine for SCN2A and SCN8A DEEs [4] - The EMERALD study evaluating relutrigine for broader DEEs [4] - The EMBRAVE3 study evaluating elsunersen for SCN2A gain-of-function DEE [4]

BOSTON, April 03, 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 that it will be participating in two upcoming investor conferences this April: Praxis management will be presenting a corporate overview at the 24th Annual Needham Virtual Healthcare Conference, ta ...

BOSTON, March 04, 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 that it will be participating in five upcoming investor conferences: Praxis management will be presenting a corporate overview at the 45th Annual TD Cowen Health Care Conference, taking place at t ...

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