BOSTON, Nov. 24, 2025 (GLOBE NEWSWIRE) -- Praxis Precision Medicines, Inc. (NASDAQ: PRAX), a clinical-stage biopharmaceutical company translating genetic insights into therapies for central nervous system (CNS) disorders driven by neuronal excitation-inhibition imbalance, today announced they will present the latest preclinical and clinical data and progress on trials across its precision epilepsy pipeline at the American Epilepsy Society Annual (AES) Meeting, December 5-9, 2025, in Atlanta, Georgia. “The A ...

Core Insights - The company is experiencing significant developments, particularly following a successful readout for essential tremor studies, with a filing anticipated early in the year [1] - Progress is being made on several epilepsy assets, including the DEE program with relutrigine, targeting rare indications SCN2A and 8A [1] - An interim analysis for the ongoing study is currently being conducted in Q4, with expectations for positive results leading to an NDA filing for the indication [1] Company Developments - The company is pleased with its progress but maintains a humble outlook despite recent successes [1] - Recruitment for the larger study, referred to as EMERALD, is ongoing and is expected to yield results next year [1] - The company is optimistic about the interim analysis and its implications for future regulatory filings [1]

Praxis Precision Medicines (NasdaqGS:PRAX) 2025 Conference November 18, 2025 08:30 AM ET Company ParticipantsMarcio Souza - President and CEOConference Call ParticipantsAndrew Tsai - Senior Biotech AnalystAndrew TsaiAll right, we're going to get started with our next session. I'm Andrew Tsai, Senior Biotech Analyst at Jefferies, and it's my pleasure to have Marzso Souza joining me today, CEO of Praxis. Welcome, Marzio.Marcio SouzaThank you. Thanks, Andrew. Appreciate it.Andrew TsaiMaybe really briefly, beca ...

Summary of Praxis Precision Medicines FY Conference Call Company Overview - **Company**: Praxis Precision Medicines (NasdaqGS:PRAX) - **Date of Conference**: November 10, 2025 - **Key Executives Present**: CEO Marcio Souza, SVP Finance Lauren, Matt Krogo Key Points Discussed Pipeline and Upcoming Developments - The company is preparing for a potential New Drug Application (NDA) filing in the next few months, particularly for essential tremors (ET) and ulixacaltamide [6][10] - There is a focus on the Essential 3 program, which has shown positive results, and the company is looking to leverage these results for future drug applications [7][8] - The company is moving forward with the SCN2A EMBOLD program, with plans for an NDA submission early next year [9][10] FDA Interactions - The upcoming FDA meeting is crucial for discussing the NDA submission and the adequacy of the evidence from the Essential 3 program [12][15] - The company has had multiple interactions with the FDA, which have been positive, indicating good continuity in the review process [11][20] - The key question for the FDA meeting is whether the current data package constitutes substantial evidence of effectiveness for approval [15][16] Market Opportunity - There are approximately 7 million Americans living with essential tremor, with at least 2 million actively seeking treatment, indicating a significant market potential [37][38] - The company estimates that with conservative market penetration, potential sales could reach $10 billion in the U.S. alone, and $13 billion to $15 billion when including ex-U.S. markets [39][40] - The pricing strategy is expected to be competitive, with a potential 25%-50% discount compared to existing treatments [38][39] Commercial Strategy - The company is building infrastructure to better understand and recruit patients, aiming to expand its database from 200,000 to about 1 million patients before launch [41][42] - The commercial organization is being strengthened, leveraging the experience of executives who have previously launched multiple drugs [42][43] Other Assets and Future Plans - The company is also focusing on relutrigine, which has received breakthrough designation, and is preparing for an interim analysis that could lead to an NDA submission [44][46] - The market for SCN2A and 8A is projected to be significant, with estimates of over $1 billion at launch [50][51] - Upcoming data from the RADIANT study is expected to provide insights into the effectiveness of relutrigine in generalized patients [54][55] Additional Insights - The company emphasizes the importance of FDA's role in ensuring drug safety and efficacy, and it aims to provide substantial evidence to support its applications [20][21] - There is a recognition of the need to overcome biases in market assumptions to accurately reflect the potential of their treatments [37][39] This summary captures the essential discussions and strategic directions of Praxis Precision Medicines as presented in the conference call.

Core Insights - Praxis Precision Medicines announced positive results from two pivotal Phase 3 Essential3 studies of ulixacaltamide HCl in essential tremor, with a pre-NDA meeting with the FDA scheduled for Q4 2025 [1][2] - The company plans to accelerate the development of relutrigine for SCN2A and SCN8A developmental and epileptic encephalopathies (DEE) after a comprehensive Type B meeting with the FDA [1][2] - Recruitment for the POWER1 study of vormatrigine in focal onset seizures has been completed, with topline results expected in the first half of 2026, while the POWER2 study has commenced [1][2] Clinical Development Updates - Ulixacaltamide demonstrated significant improvements in symptoms for essential tremor, with a mean improvement of 4.3 points in the modified Activities of Daily Living 11 (mADL11) at Week 8 [3][4] - The first study met all primary and secondary endpoints, while the second study showed 55% of patients on ulixacaltamide maintained their response compared to 33% on placebo [3][4] - Vormatrigine showed a 56.3% median reduction in seizure frequency in the RADIANT study, with 22% of patients achieving a 100% reduction in the last 28 days [4][15] Financial Overview - As of September 30, 2025, Praxis had $389.2 million in cash and marketable securities, down from $469.5 million at the end of 2024, primarily due to cash used in operating activities [6][7] - The company completed a public offering in October 2025, generating net proceeds of approximately $567 million, expected to fund operations into 2028 [7][8] - Research and development expenses increased to $65.8 million for Q3 2025, up from $41.9 million in Q3 2024, largely due to investments in the Cerebrum™ platform [9][10] Market Position and Future Prospects - Praxis is positioned to establish itself as a leader in innovative therapies for CNS disorders, with multiple clinical-stage product candidates across movement disorders and epilepsy [2][18] - The company is on track to nominate development candidates for early-stage antisense oligonucleotide (ASO) therapeutic initiatives in the first half of 2026 [13][18] - The EMBOLD study for relutrigine is expected to provide substantial evidence for NDA submission in early 2026, contingent on a successful interim analysis [4][16]

Core Insights - Praxis Precision Medicines has reached alignment with the FDA regarding the relutrigine program for SCN2A and SCN8A developmental and epileptic encephalopathies (DEEs) following a comprehensive Type B meeting [1][2] - The company plans to conduct an interim analysis of the EMBOLD cohort 2 pivotal trial in Q4 2025, which, if positive, could support a New Drug Application (NDA) submission in early 2026 [1][2] 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 [6] - The company utilizes genetic insights to create therapies for both rare and prevalent neurological disorders through its proprietary platforms [6] Product Development - Relutrigine (PRAX-562) is a first-in-class small molecule being developed for the treatment of DEEs, showing promise in preclinical studies and early clinical trials [5] - The EMBOLD study involves a randomized trial design to evaluate the efficacy of relutrigine in reducing seizure frequency among patients [3][4] - The interim analysis will assess approximately 70% of patients enrolled in the study, with a focus on achieving statistically significant results [4] Market Context - SCN2A and SCN8A-DEEs are ultra-rare, life-threatening pediatric epilepsies with no FDA-approved therapies currently available, affecting around 5,000 patients in the US [2] - The only existing treatment option is off-label polytherapy with antiseizure medications (ASMs), which is largely ineffective and carries significant risks [2]

Core Insights - Praxis Precision Medicines experienced a significant stock surge of 251% following positive phase 3 trial results for ulixacaltamide, a treatment for essential tremor patients [2][3] - The essential tremor condition affects approximately 10 million Americans, with only one approved treatment currently available, indicating a substantial market opportunity for new therapies [5] - Ulixacaltamide has shown promising results in clinical trials, but the company is still in the pre-commercial stage, which may pose risks for investors [3][15] Company Overview - Praxis Precision Medicines is focused on developing treatments for neurological disorders, with ulixacaltamide targeting essential tremor and vormatrigine aimed at epilepsy [13][14] - The company has a market capitalization of approximately $4 billion, reflecting investor interest in its late-stage drug candidates [12][16] - Praxis recently raised $525 million in a secondary offering, bolstering its cash reserves to support ongoing development efforts [15] Clinical Trial Results - In the first phase 3 study, patients treated with ulixacaltamide showed an average improvement of 4.3 points on a modified Activities of Daily Living test, compared to a 1.7-point improvement in the placebo group [7] - The second study indicated that patients who continued treatment with ulixacaltamide had a significantly higher likelihood of maintaining a response and reported improved disease outcomes [8] - Despite the positive results, a notable discontinuation rate of 35.6% in the first study and 38.1% in the second study raises concerns about treatment tolerability [10][11] Market Potential - The potential annual sales for ulixacaltamide could reach billions, given the large population of underserved essential tremor patients [3] - Up to 77% of essential tremor patients feel inadequately treated, and nearly 50% are not receiving any treatment, highlighting a significant unmet medical need [6] Future Prospects - Praxis is also advancing other drug candidates, including vormatrigine for epilepsy, which has shown promising phase 2 results with a 60% reduction in seizure rates for treated patients [13] - The company is preparing to enter phase 3 trials for vormatrigine, indicating a robust pipeline of potential therapies [14] - Investors with a high-risk tolerance may find Praxis an attractive opportunity due to its focus on underserved populations and multiple late-stage candidates [16]

Core Insights - Praxis Precision Medicines reported widening operational losses in Q2 FY2025, with a GAAP loss per share of ($3.31), missing the consensus estimate of ($3.25) [1] - The company recognized no revenue, missing expectations of $0.21 million, and reported a net loss of $71.1 million compared to $32.7 million in Q2 2024 [1][2] - Despite financial setbacks, progress in key clinical trials and regulatory achievements indicate operational momentum [1] Financial Performance - GAAP EPS for Q2 2025 was ($3.31), a 90.2% decline from ($1.74) in Q2 2024 [2] - Research and Development expenses increased by 130.8% to $63.0 million from $27.3 million in Q2 2024 [2][5] - General and Administrative expenses rose by 23.6% to $13.1 million from $10.6 million in Q2 2024 [2] - Cash, cash equivalents, and marketable securities totaled $446.6 million as of June 30, 2025, a decrease of approximately $22.9 million from December 31, 2024 [2][7] Business Strategy - Praxis focuses on therapies for neurological conditions, utilizing two core platforms: Cerebrum for small molecule drugs and Solidus for RNA-based therapies [3] - The company is advancing clinical-stage development programs, particularly ulixacaltamide for essential tremor and vormatrigine for epilepsy [4] - Key success factors include rapid enrollment in trials, obtaining regulatory designations, and maintaining a robust pipeline [4] Clinical Developments - The RADIANT Phase 2 study for vormatrigine showed a median 56.3% reduction in seizures, with 22% of patients seizure-free during the last 28 days [6] - Relutrigine received Breakthrough Therapy Designation from the FDA, which may expedite its approval process [6] Operational Indicators - Accounts payable increased from $12.5 million at year-end 2024 to $28.8 million by June 30, 2025 [8] - The share count rose to 21.5 million from 18.8 million in the prior-year quarter, indicating some dilution [8] Future Outlook - Management did not provide explicit financial guidance but emphasized upcoming clinical milestones as critical events [9] - Top-line results for ulixacaltamide and vormatrigine are expected by late 2025 or early 2026 [9] - Cash use is projected to remain high as clinical operations expand, but the current cash position supports operations into 2028 [10]

Core Insights - Praxis Precision Medicines reported positive results from the RADIANT study, showing a 56.3% median reduction in seizure frequency over eight weeks in focal onset seizure patients, with 22% achieving 100% seizure reduction in the last 28 days [1][2][3] - The company has initiated two registrational studies for Developmental and Epileptic Encephalopathies (DEEs): EMERALD for broad DEEs with relutrigine and EMBRAVE3 for SCN2A Gain-of-Function with elsunersen [1][2] - Relutrigine received U.S. FDA Breakthrough Therapy Designation for treating seizures associated with SCN2A and SCN8A DEEs, facilitating expedited development [1][2] - As of June 30, 2025, Praxis had approximately $447 million in cash and investments, providing a financial runway into 2028 [1][5] Clinical Development - The RADIANT study demonstrated a rapid and sustained response, with around 60% of patients achieving a 50% response rate [3] - Enrollment for the POWER1 pivotal study for vormatrigine is progressing well, with plans to initiate POWER2 and POWER3 studies soon [2][3] - The EMERALD study for relutrigine is expected to enroll up to 160 patients, with completion anticipated in 2026 [6] - The EMBRAVE3 study for elsunersen has been initiated, targeting SCN2A patients aged 2 to 18 years [6] Financial Performance - For Q2 2025, Praxis reported a net loss of $71.1 million, compared to a net loss of $32.7 million in Q2 2024 [10][21] - Research and development expenses increased to $63.0 million in Q2 2025 from $27.3 million in Q2 2024, primarily due to investments in the Cerebrum™ platform [8][21] - General and administrative expenses rose to $13.1 million in Q2 2025 from $10.6 million in Q2 2024 [9][21] Future Outlook - Praxis plans to present additional data at the 36th International Epilepsy Congress on August 31, 2025, and at the American Epilepsy Society Annual Meeting in December 2025 [3] - The company is on track to nominate development candidates for its early-stage ASO therapeutic initiatives by the end of 2025 [6]

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]