Praxis Precision Medicines (NasdaqGS:PRAX) Update / Briefing October 16, 2025 08:00 AM ET Speaker0Good day. Thank you for standing by. Welcome to the Practice Precision Medicine's Essential Treat Topline Results Conference Call. After the speaker's presentation, there will be a question and answer session. Please note that today's conference may be recorded.I will now hand the conference over to your speaker host, Dan Ferry. Please go ahead.Speaker1Good morning, and welcome to the Praxis Precision Medicine' ...

Essential3 Program Topline Results October 16, 2025 Forward Looking Statements This presentation may contain "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 relating to our business, operations, and financial conditions, including but not limited to express or implied statements regarding the current beliefs, expectations and assumptions regarding the future of our business, future plans and strategies, , including statements regarding the estimated ma ...

Core Insights - Praxis Precision Medicines experienced a significant stock increase of approximately 134% in premarket trading following the announcement of successful results from two late-stage trials for its experimental therapy ulixacaltamide, aimed at treating essential tremor, the most common type of movement disorder [2] Company Summary - The Boston-based biotech company, Praxis Precision Medicines, is focused on developing therapies for neurological disorders [2] - The successful trials for ulixacaltamide mark a critical milestone in the company's research and development efforts [2] Industry Context - Essential tremor is identified as the most prevalent movement disorder, indicating a substantial market opportunity for effective treatments [2] - The positive trial results could position Praxis Precision Medicines favorably within the competitive landscape of biotech firms targeting neurological conditions [2]

Core Insights - Praxis Precision Medicines announced that its experimental drug for movement disorders has shown positive results in improving daily functioning for patients in two late-stage trials [1] Company Summary - The company is focused on developing treatments for movement disorders and has reached significant milestones in its clinical trials [1]

Core Insights - Praxis Precision Medicines announced positive topline results for the Phase 3 Essential3 program of ulixacaltamide in essential tremor (ET) [1][2][19] - The studies demonstrated statistically significant improvements in key efficacy endpoints, indicating ulixacaltamide's potential as a treatment for ET [11][13] Study Results - In Study 1, patients treated with ulixacaltamide showed a mean improvement of 4.3 points in the Modified Activities of Daily Living (mADL11) at Week 8, with p<0.0001 [1][11] - All key secondary endpoints in Study 1 were statistically significant, including the rate of disease improvement and Patient Global Impression of change (PGI-C) [1][10] - Study 2 confirmed the maintenance of effect for ulixacaltamide, with 55% of patients maintaining response compared to 33% in the placebo group (p=0.0369) [13][14] Safety Profile - Ulixacaltamide was generally well tolerated, with no drug-related serious adverse events reported [1][16] - The most common treatment-emergent adverse events included constipation, dizziness, and headache, with discontinuations primarily due to dizziness and brain fog [16][17] Corporate Developments - Praxis has submitted a pre-NDA meeting request to the FDA and plans to submit the NDA by early 2026 [19][20] - The company intends to share additional data from these studies at upcoming medical conferences and peer-reviewed publications [19] Market Context - Essential tremor affects approximately seven million people in the U.S., representing a significant unmet medical need for effective therapies [22] - Current treatment options are limited, with propranolol being the only approved pharmacotherapy, highlighting the potential market opportunity for ulixacaltamide [22][23]

Summary of Praxis Precision Medicines FY Conference Call Company Overview - **Company**: Praxis Precision Medicines (NasdaqGS:PRAX) - **Key Assets**: Matrigene for the treatment of epilepsy, specifically focusing on FOS (focal onset seizures) Industry Insights - **Market Context**: There is a significant unmet need in the epilepsy treatment market, with approximately 1 to 2 million patients in the U.S. switching therapies annually due to inadequate responses to current medications [4][20] - **Physician Feedback**: Over 4,000 epilepsy physicians expressed excitement about new treatments, including Matrigene, which shows promise in improving patient outcomes after decades of stagnation in treatment efficacy [3][4] Core Points and Arguments - **Efficacy of Matrigene**: The data presented at the International Epilepsy Congress highlighted the potential of Matrigene to significantly reduce seizures, with a notable onset of effects and a favorable tolerability profile compared to existing treatments [10][11] - **Patient Population**: The studies involve a challenging patient population, including those who have failed multiple treatments, which adds to the significance of the positive results [10][19] - **Market Opportunity**: The refractory epilepsy market is vast, with estimates suggesting that the current market caps of leading players do not reflect the true potential of the market [20][22] - **Commercialization Strategy**: The company is shifting focus towards commercialization, emphasizing the need to discuss how their drugs can help patients in a commercial setting [20][21] Upcoming Studies and Data - **Power One and Power Two Studies**: Power One is nearing completion, with results expected soon, while Power Two is set to start in Q3 2025. The company anticipates rapid enrollment and potential NDA submission shortly thereafter [30][32] - **Power Three Study**: This monotherapy study is generating excitement among physicians, with plans to gradually reduce background medications to assess the efficacy of Matrigene as a standalone treatment [18][19] Additional Assets - **Religiogene**: This asset is in a registration cohort and has shown a 90% reduction in seizures in a severe patient population. The company has received breakthrough designation from the FDA, which may accelerate the development process [38][39] - **Emeralds Study**: This study is also progressing well, with a potential market value of around $1 billion, indicating significant growth opportunities for the company [39] Conclusion - **Strategic Focus**: Praxis Precision Medicines is positioned to address significant unmet needs in the epilepsy market with its innovative treatments. The company is actively working towards commercialization and is optimistic about the upcoming data from its clinical trials, which could lead to substantial market opportunities [20][32][39]

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][3] - The company will participate in three upcoming investor conferences, providing opportunities for one-on-one meetings with interested investors [1][4] Company Overview - Praxis utilizes genetic insights from epilepsy to develop therapies for CNS disorders through its proprietary platforms, Cerebrum™ and Solidus™ [3] - The company has a diversified portfolio that includes multiple programs targeting epilepsy and movement disorders, with four clinical-stage product candidates [3] Upcoming Events - Praxis will present at the Baird 2025 Global Healthcare Conference on September 9th from 9:00-9:35am EDT [4] - A fireside chat is scheduled for September 10th at the H.C. Wainwright 27th Annual Global Investment Conference at 8:30am EDT [4] - A virtual corporate presentation will take place on September 18th at the TD Cowen 5th Annual Novel Mechanisms in Neuropsychiatry & Epilepsy Summit from 3:00-3:30pm EDT [4]

Summary of Praxis Precision Medicines (PRAX) Conference Call Company Overview - **Company**: Praxis Precision Medicines (PRAX) - **Event**: Conference Call on September 02, 2025 - **Key Speaker**: Marcio Souza, CEO Core Industry Insights - **Industry Focus**: Epilepsy drug development - **Market Size**: Estimated at approximately 3 million patients in the US [62] - **Unmet Need**: Significant unmet medical need in epilepsy treatment, indicating a larger market opportunity than previously anticipated [11] Key Points Discussed RADIANCE Study Highlights - **Study Objective**: To evaluate the efficacy of vormatrogene in reducing seizures, focusing on the reduction in seizure frequency and understanding pharmacokinetics (PK) and pharmacodynamics (PD) in patients with focal onset seizures [17][18] - **Results**: - Achieved a **56% sustained overall seizure reduction rate** [20] - **22% of patients** experienced a **100% seizure reduction** in the last 28 days, with **14% being seizure-free** over the entire 8-week treatment period [45] - **Rapid Response**: Seizure reduction was observed as early as one week into treatment, indicating a rapid therapeutic effect [20][22] Comparative Efficacy - **Placebo Effect**: Expected placebo seizure reduction rate estimated at **10-15%**, making the observed efficacy of 56% particularly robust [44] - **Comparison with Other Drugs**: Vormatrogene's efficacy is positioned as superior to many existing treatments, with the potential to replace less effective drugs like levetiracetam [106][110] Safety and Tolerability - **Adverse Events**: Treatment-emergent adverse events (TEAEs) were reported to be lower than those of other drugs, although dropout rates were higher than expected [86][101] - **Patient Experience**: The experience of principal investigators (PIs) and patient counseling were identified as critical factors influencing dropout rates [90][91] Future Development Plans - **Upcoming Studies**: - **Power One**: Expected to provide further data on efficacy and safety, with recruitment ongoing and anticipated completion in late 2026 [119][122] - **Power Two**: Set to begin shortly, with expectations of higher response rates due to increased dosing [116][121] - **Regulatory Submission**: Anticipated NDA submission within the next 18 months based on the outcomes of ongoing studies [122] Market Positioning - **Differentiation**: Vormatrogene is positioned as a highly effective and convenient treatment option for refractory epilepsy, with a compelling profile that could dominate the market [105][110] - **Physician Feedback**: Positive reception from physicians regarding the potential to replace existing treatments, highlighting the drug's efficacy and tolerability [106][107] Additional Insights - **Patient Compliance**: High compliance rates observed with electronic diaries for tracking seizures, enhancing data accuracy for ongoing studies [128][129] - **Long-term Expectations**: Anticipation of a significant number of patients achieving seizure freedom as treatment progresses and background medications are optimized [54][55] This summary encapsulates the key discussions and insights from the Praxis Precision Medicines conference call, focusing on the RADIANCE study results, market positioning, and future development plans.

Company Overview - Praxis Precision Medicines, Inc. is a clinical-stage biopharmaceutical company focused on developing therapies for central nervous system (CNS) disorders, particularly epilepsy [1][13] - The company utilizes proprietary small molecule and antisense oligonucleotide (ASO) platforms to address neurological disorders characterized by neuronal excitation-inhibition imbalance [13] Upcoming Event Participation - Praxis will participate in the International Epilepsy Congress (IEC) from August 30 to September 3, 2025, in Lisbon, Portugal, showcasing its advancements in epilepsy treatment [1][2] - The company plans to present topline results from the RADIANT study in focal onset seizures (FOS) and share updates on its precision epilepsy pipeline through twelve presentations [2][4] Research and Development Highlights - Vormatrigine, a small molecule targeting sodium channels, is being developed for adult focal onset seizures and generalized epilepsy, showing robust seizure reduction in clinical studies [4][8] - Relutrigine, a first-in-class small molecule for developmental and epileptic encephalopathies (DEEs), has demonstrated significant seizure control in preclinical models and received multiple designations from regulatory agencies [11][12] - Elsunersen, an ASO targeting SCN2A gene expression, has shown promise in reducing seizures and improving outcomes in SCN2A-DEE models, with ongoing studies under collaboration with Ionis Pharmaceuticals [12][13] Presentation Schedule - Praxis will conduct various presentations during the IEC, including platform and poster presentations, detailing the predictive validity of preclinical seizure models and updates from ongoing studies [3][6][10]

Core Insights - Bragar Eagel & Squire, P.C. is investigating potential claims against Praxis Precision Medicines, Inc. for possible violations of federal securities laws and unlawful business practices [1][2]. Financial Performance - On August 4, 2025, Praxis reported its second-quarter financial results, including mid-stage study results for its anti-seizure medication vormatrigine, where 36 out of 61 patients experienced treatment-emergent adverse events, and nearly 25% of participants discontinued the study [2]. Stock Market Reaction - Following the announcement of the adverse study results, Praxis's stock price decreased by $3.00 per share, or 5.55%, closing at $51.09 per share on August 4, 2025 [3].