Core Viewpoint - Praxis Precision Medicines, Inc. is under investigation for possible securities fraud following disappointing clinical trial results and a significant drop in stock price [1][3][4] Financial Results - On August 4, 2025, Praxis reported its financial results for Q2 2025, including mid-stage clinical trial results for its anti-seizure medication, vormatrigine [3] - The trial revealed that 36 out of 61 patients experienced treatment-emergent adverse events, with nearly 25% of participants discontinuing the study [3] Stock Performance - Following the announcement of the financial results and clinical trial outcomes, Praxis's stock price fell by $3.00 per share, approximately 5.55%, closing at $51.09 per share on August 4, 2025 [4] Legal Actions - The Portnoy Law Firm has initiated an investigation into Praxis and may file a class action on behalf of investors who suffered losses [1][2] - Investors are encouraged to contact the law firm for a complimentary case evaluation regarding their legal rights [2]

Summary of Praxis Precision Medicine Conference Call Company and Industry Overview - **Company**: Praxis Precision Medicine - **Industry**: Epilepsy treatment and pharmaceutical development Key Points and Arguments 1. **Market Size and Opportunity**: The epilepsy market in the U.S. is estimated to be around 3 to 3.5 million patients, with many existing drugs failing to adequately address the needs of these patients, indicating significant opportunities for new treatments [11][12][19] 2. **Efficacy of Vormetrigine**: The drug has shown promising results in treating focal onset seizures, with a median seizure reduction of 56.3% and 50% of patients experiencing a reduction in seizures [62][64] 3. **Patient Demographics**: 81% of patients in the study were on sodium channel blockers, which is typical for this patient population. Many patients were on multiple medications, indicating a complex treatment landscape [43][46][55] 4. **Recruitment Success**: The company successfully dosed 61 patients in the trial, demonstrating that patient recruitment for focal epilepsy studies is feasible, contrary to common beliefs [31][32] 5. **Background Therapy Impact**: The drug's efficacy was observed even in patients already on sodium channel blockers, suggesting that it can provide additional benefits in a heavily medicated population [20][62] 6. **Discontinuation Rates**: 23% of patients discontinued the study, which is consistent with industry norms. The reasons for discontinuation were primarily patient choice rather than adverse events [78][90] 7. **Adverse Events (AEs)**: The treatment emerged with a 59% rate of treatment-emerging AEs, which is considered manageable and comparable to other drugs in the market [100][101] 8. **Future Studies**: The company plans to conduct further studies (POWER 1, 2, and 3) to explore the drug's efficacy as a monotherapy and its potential to replace existing treatments [104][116] Additional Important Insights 1. **Sodium Channel Modulation**: Modulating sodium channels is identified as a key mechanism for treating focal seizures, with the drug positioned to potentially replace existing therapies [16][50] 2. **Market Dynamics**: The market is not winner-takes-all; multiple drugs can coexist, and the company aims to capture a significant share by demonstrating the efficacy of its drug [108][110] 3. **Patient-Centric Approach**: The company emphasizes the importance of understanding patient needs and the limitations of current treatments, aiming to provide a better therapeutic option [59][92] 4. **Data Analysis Methodology**: The company uses a robust methodology for analyzing patient data, including handling discontinuations in a way that reflects true treatment efficacy [81][88] This summary encapsulates the key discussions and insights from the conference call, highlighting the company's strategic positioning within the epilepsy treatment landscape and the promising data surrounding its lead drug, vormetrigine.

Core Insights - Praxis Precision Medicines, Inc. is a clinical-stage biopharmaceutical company focused on developing therapies for central nervous system disorders through genetic insights [1][2] - The company will have its CEO Marcio Souza participate in a virtual fireside chat hosted by Truist Securities on August 5, 2025 [1] Company Overview - Praxis specializes in translating insights from genetic epilepsies into therapies for CNS disorders characterized by neuronal excitation-inhibition imbalance [2] - The company utilizes its proprietary small molecule platform, Cerebrum™, and antisense oligonucleotide (ASO) platform, Solidus™, to develop therapies for both rare and prevalent neurological disorders [2] - Praxis has a diversified CNS portfolio that includes multiple programs across epilepsy and movement disorders, featuring four clinical-stage product candidates [2]

Praxis Precision Medicines (PRAX) Q2 2025 Earnings Call August 04, 2025 08:30 AM ET Company ParticipantsDan Ferry - Managing DirectorMarcio Souza - President & CEOSteven Petrou - Co-Founder & Chief Scientific OfficerConference Call ParticipantsYasmeen Rahimi - Senior Research AnalystRitu Baral - MD & Senior Biotechnology AnalystJoon Lee - MD & Senior Biotech AnalystDoug Tsao - MD & Senior Healthcare AnalystYatin Suneja - Biotechnology Research AnalystDavid Hoang - Director & Senior Analyst - BiotechnologyAm ...

Financial Data and Key Metrics Changes - The company reported a median seizure reduction of over 56% in the RADIANT study, with 60% of patients achieving at least a 50% reduction in seizures, indicating strong efficacy of the drug [13][14][20] - The cash runway extends into 2028, supporting the company's ambitious clinical agenda [6] Business Line Data and Key Metrics Changes - The RADIANT study focused on the efficacy of vermatrogene, which is positioned as a best-in-disease treatment for focal epilepsy, showing significant potential in a complex patient population [8][20] - The company has four late-stage assets and expects five clinical readouts within the next year, highlighting a robust pipeline [6][7] Market Data and Key Metrics Changes - The focal epilepsy market is characterized by a high unmet need, with over 60% of patients requiring multiple anti-seizure medications, underscoring the inadequacy of existing therapies [7][23] - The company plans to launch POWER three, aiming to establish vermatrogene as a standalone therapy for refractory epilepsy patients transitioning off current anti-seizure medications [24] Company Strategy and Development Direction - The company aims to revolutionize epilepsy treatments, focusing on innovative therapies that combine fast-acting mechanisms with minimal restrictions [24] - The addition of a 40 mg dose arm in the POWER two study is intended to explore greater efficacy [21] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the potential of vermatrogene to make a significant difference for patients with refractory epilepsy, emphasizing the importance of effective treatment options [20][25] - The management highlighted the positive impact on patients' moods observed during the RADIANT study, which will be included as endpoints in future studies [21][66] Other Important Information - The company received breakthrough designation for raletirgene in the US, which will expedite its path to registration for patients with specific genetic conditions [25] - The discontinuation rate in the RADIANT study was 23%, primarily linked to background anti-seizure medication dose adjustments, rather than the drug itself [18][56] Q&A Session Summary Question: Background therapies and response rates - The company observed robust effects across various background therapies, with over 57% of patients responding positively, even those on multiple medications [29][31] Question: Execution of the RADIANT study - The strong execution was attributed to effective recruitment strategies and high-quality site management, leading to a representative sample of the refractory epilepsy population [33][34] Question: Discontinuation rates and efficacy - Discontinuation rates were higher than expected, but the company noted that reducing background medications improved patient outcomes without negatively impacting efficacy [56][88] Question: Mood benefits and endpoints - The management acknowledged anecdotal reports of mood improvements and plans to incorporate systematic assessments in future studies [66][79] Question: Safety profile and background therapy interactions - The company reported that the adverse event rates were lower than other drugs, and the safety profile was favorable, with most adverse events being mild to moderate [63][64] Question: Future studies and market opportunities - The company is optimistic about the potential market opportunity for vermatrogene, especially in refractory and hyper-refractory patient populations [80][81]

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]

Praxis Precision Medicines Provides Corporate Update and Reports Second Quarter 2025 Financial Results RADIANT study with vormatrigine in focal onset seizure (FOS) patients over eight weeks demonstrated 56.3% median reduction in seizure frequency from baseline, with 22% at 100% seizure reduction in last 28 days Initiated two registrational studies for Developmental and Epileptic Encephalopathies (DEEs) programs: EMERALD for broad DEEs with relutrigine and EMBRAVE3 for SCN2A Gain-of-Function (GoF) with elsun ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended June 30, 2025 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission File Number: 001-39620 PRAXIS PRECISION MEDICINES, INC. (Exact Name of Registrant as Specified in its Charter) Delaware 47-5195942 (State or other ...

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]

Vormatrigine Efficacy and Development - RADIANT study showed a 56.3% overall median seizure reduction with vormatrigine[21] - A significant proportion of patients achieved ≥50% seizure reduction, independent of baseline seizure burden[26] - Vormatrigine is being developed as a potential stand-alone agent, with POWER3 study expected to initiate in 1H 2026[40, 42] - POWER2 study is staged to initiate this quarter and complete enrollment in 2H 2026, with a sample size of approximately 400 patients[37] Safety and Tolerability - In the RADIANT study, 59% of patients experienced ≥1 treatment-emergent adverse event (TEAE) with vormatrigine 30 mg[33] - 4.9% of patients experienced severe adverse events (AEs) and serious AEs (SAEs) with vormatrigine 30 mg[33] - 1.6% of patients experienced a related SAE with vormatrigine 30 mg[33] Pipeline and Catalysts - The company has assets in late stage development, with clinical readouts expected in the next 4 quarters[7] - Topline results for the RADIANT Phase 2 study are expected by mid-2025[8] - Finalization of the POWER1 Phase 2/3 study is expected in Q4 2025[8] - Candidate declaration for PRAX-100 (SCN2A LoF) is expected by mid-2025, and for PRAX-080 (PCDH19) and PRAX-090 (SYNGAP1) by year-end 2025[8] Financial Position - The company has a cash runway into 2028[7]