Praxis Precision Medicines (NasdaqGS:PRAX) FY Conference September 10, 2025 08:30 AM ET Company ParticipantsMarcio Souza - CEO[Unknown Speaker]Okay, we'll kick it off. Good morning, everybody. We're in the home stretch, the final day of the conference. I appreciate everybody making it out for these morning sessions. With that, we're thrilled to have Praxis Precision Medicines with us, represented by the company's CEO, Marcio Souza. We also acknowledge Tim Kelly, the company's CFO, is in the audience. Marcio ...

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].

Core Viewpoint - The Schall Law Firm is investigating Praxis Precision Medicines, Inc. for potential violations of securities laws related to misleading statements and undisclosed information affecting investors [1][2]. Group 1: Investigation Details - The investigation centers on whether Praxis issued false or misleading statements and failed to disclose critical information to investors [2]. - Praxis reported its Q2 2025 financial results on August 4, 2025, revealing concerning mid-state clinical results for its anti-seizure medication, vormatrigine [2]. - More than half of the patients in the trial experienced treatment-emergent adverse events, with nearly 25% of participants discontinuing the medication [2]. Group 2: Market Reaction - Following the announcement of the adverse clinical results, shares of Praxis fell by more than 5% on the same day [2].

Core Viewpoint - Pomerantz LLP is investigating potential securities fraud or unlawful business practices involving Praxis Precision Medicines, Inc. and its officers or directors [1]. Financial Results - On August 4, 2025, Praxis reported its second-quarter financial results, including mid-stage study results for its anti-seizure medication vormatrigine [3]. - The study revealed that 36 out of 61 patients experienced treatment-emergent adverse events, with nearly 25% of participants discontinuing the study [3]. - Following the announcement, Praxis's stock price decreased by $3.00 per share, or 5.55%, closing at $51.09 per share on the same day [3].

Core Viewpoint - Pomerantz LLP is investigating potential securities fraud or unlawful business practices involving Praxis Precision Medicines, Inc. and its officers or directors [1] Financial Results - On August 4, 2025, Praxis reported its financial results for Q2 2025, including mid-stage study results for its anti-seizure medication vormatrigine [3] - The study revealed that 36 out of 61 patients experienced treatment-emergent adverse events, with nearly 25% of participants discontinuing the study [3] - Following the announcement, Praxis's stock price decreased by $3.00 per share, or 5.55%, closing at $51.09 per share on the same day [3]

Summary of Praxis Precision Medicines (PRAX) Fireside Chat - August 07, 2025 Company Overview - **Company**: Praxis Precision Medicines (PRAX) - **Focus**: Development of therapies for epilepsy, specifically the drug rometragene Key Industry Insights - **Market Size**: Approximately 3 million patients with epilepsy, with over 60% unable to tolerate or respond to current treatments, indicating a significant unmet need in the epilepsy market [10][11] - **Drug Efficacy**: The drug has shown promising results in treating refractory epilepsy, with a notable response rate in difficult-to-treat patients [9][12] Core Points Discussed 1. **Phase One Data**: Initial phase one data did not provide clear differentiation for rometragene compared to other therapies, but subsequent data has shown significant promise [3][6] 2. **RADIANT Study**: The RADIANT study involved 61 patients as of July 25, with a target of 75 patients. The study aims to provide data on both generalized and focal epilepsy patients [13][15] 3. **Patient Response**: The drug has demonstrated a high response rate, with 100% of patients responding in earlier studies, although translating this to broader patient populations remains complex [8][9] 4. **Discontinuation Rates**: The discontinuation rate reported was 23%, primarily occurring within the first three weeks of treatment. This is considered acceptable given the severity of the patient population [36][37] 5. **Safety and Efficacy**: The pharmacokinetics (PK) of rometragene in patients were comparable to healthy volunteers, and the drug did not adversely affect safety or discontinuation rates [22][34] 6. **Seizure Reduction**: The total seizure reduction was reported at 56%, with potential for up to 70% reduction by week eight. The drug's efficacy appears to improve over time [66][72] Additional Considerations - **Patient Recruitment**: The study has faced challenges in patient recruitment due to the complexity of screening and the nature of the patient population [80][81] - **Open Label Data**: Concerns regarding the validity of open-label data were addressed, emphasizing that treatment effects are consistent regardless of whether patients are aware of their treatment status [83][89] - **Future Expectations**: The company anticipates that longer treatment durations will lead to improved outcomes, with ongoing studies expected to confirm these trends [68][72] Conclusion Praxis Precision Medicines is positioned in a significant market with a promising drug candidate for epilepsy. The insights from the RADIANT study and ongoing trials suggest a strong potential for rometragene to address unmet needs in the epilepsy treatment landscape. The company is focused on refining its approach to patient recruitment and data collection to ensure robust outcomes in future studies.

Core Viewpoint - Pomerantz LLP is investigating potential securities fraud or unlawful business practices involving Praxis Precision Medicines, Inc. and its officers or directors [1] Financial Results - On August 4, 2025, Praxis reported its financial results for Q2 2025, including mid-stage study results for its anti-seizure medication vormatrigine [2] - In the study, 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 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]