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Azetukalner Efficacy and Safety - Azetukalner demonstrated a 90.9% median percent change (MPC) reduction in monthly focal onset seizure (FOS) frequency after 48 months in the open-label extension (OLE) [92] - A subset of patients receiving 1-2 anti-seizure medications (ASMs) at double-blind period (DBP) baseline experienced a 100% monthly reduction in FOS frequency [92] - 38% of patients treated with azetukalner for at least 48 months achieved seizure freedom for one year or longer [92] - Azetukalner's safety and tolerability profile in the OLE remained consistent with the DBP [92, 73] - In the Phase 2b X-TOLE study, azetukalner showed statistically significant and dose-dependent seizure reduction, with up to a 52.8% median percent change from baseline [34] Commercial Opportunity and Market Insights - Xenon Pharmaceuticals has $555.3 million in cash, cash equivalents, and marketable securities as of September 30, 2025, with anticipated cash runway into 2027 [18] - An estimated 2.6 million adults in EU4 + UK, 3.0 million in Japan, and 0.8 million in the U.S have diagnosed epilepsy [95] - Focal onset seizures (FOS) represent the largest segment of the epilepsy population [95] - Up to 50% of epilepsy patients may require additional treatment options, presenting a significant commercial opportunity [102, 97] Clinical and Patient Burden - Depression is a significant burden for patients with FOS, highlighting the need for therapeutics that do not exacerbate mood disorders [92, 79] - Titration of anti-seizure medications (ASMs) poses a significant burden for both patients and healthcare professionals (HCPs), underscoring the need for simplified or no-titration ASMs [92, 87]

"The ARGUS trial is one of the most advanced development programs in the 5-HT2 (serotonin) agonist class and the effectiveness, safety and tolerability data of EPX-100 dosed BID from the open-label extension study are very encouraging,†said Kumar Budur, MD, MS, Chief Medical and Scientific Officer at Harmony Biosciences. "These initial results support the advancement of our epilepsy franchise as we progress toward the topline data readout from the ARGUS trial in 2026.†EPX-100 is being evaluated as an inve ...

Positive Results in the EMBOLD study of relutrigine showed a 53% placebo-adjusted reduction in seizures (p < 0.0002), 66% increase in motor seizure-free days (p = 0.0340), broad and clinically meaningful functional improvements (p ≤ 0.002) Patients in the RADIANT study of vormatrigine showed rapid, consistent and durable improvement in seizure reduction, reaching 100% for patients continuing through 16 weeks Expansion of the Praxis Analysis of Concordance Framework to include DEEs Showcase of the most compr ...

Core Insights - Xenon Pharmaceuticals Inc. is set to present multiple research findings at the American Epilepsy Society Annual Meeting (AES 2025) from December 5-9, 2025, focusing on their lead molecule azetukalner for epilepsy treatment [1][5][9] - The presentations will include long-term data from the X-TOLE open-label extension study, which supports the potential for extended seizure freedom with azetukalner [1][5][6] - Additional findings will address the impact of depression on epilepsy patients and new pre-clinical data from the NaV1.1 program targeting Dravet syndrome [1][5][9] Presentation Details - Seven posters will be presented, including: - Long-term safety and efficacy data of azetukalner in adults with focal epilepsy, with a ≥48-month interim analysis from the ongoing 7-year X-TOLE study [6] - Characterization of long-term seizure freedom in the ongoing open-label extension of X-TOLE [6] - Studies on depression symptoms among epilepsy patients and their impact on treatment outcomes [6][5] - A symposium titled "Exploring Depression and Anxiety in Epilepsy" will be held in partnership with the Epilepsy Foundation of America [5] Company Overview - Xenon Pharmaceuticals is a neuroscience-focused biopharmaceutical company dedicated to drug discovery and development of therapeutics for epilepsy and other neurological disorders [9] - The company's lead molecule, azetukalner, is a selective KV7 potassium channel opener currently in Phase 3 clinical trials for epilepsy, major depressive disorder, and bipolar depression [9] - Xenon is also advancing early-stage programs targeting potassium and sodium channels for potential pain treatment [9]

Core Viewpoint - Avenue Therapeutics has announced the acquisition of its subsidiary Baergic Bio by Axsome Therapeutics, which includes the global rights to BAER-101, a novel therapy for epilepsy, potentially enhancing treatment options in a field with high unmet needs [1][2][7]. Company Overview - Avenue Therapeutics, Inc. is a specialty pharmaceutical company focused on developing therapies for neurologic diseases, headquartered in Miami, FL, and founded by Fortress Biotech, Inc. [5]. Transaction Details - The acquisition involves Axsome obtaining worldwide commercial, development, and manufacturing rights to BAER-101, with Baergic shareholders eligible for up to approximately $82 million in potential payments, including a $0.3 million upfront payment and milestone payments based on development and sales achievements [1][7]. - Avenue expects to receive approximately 74% of all future payments and royalties under the agreement [7]. Product Information - BAER-101, now referred to as AXS-17 by Axsome, has shown a promising safety and tolerability profile in clinical studies involving over 700 patients and is being evaluated for its anti-convulsant effects in epilepsy [3][2].

Core Insights - JAZZ Pharmaceuticals has signed an agreement with Saniona to acquire worldwide rights to the investigational epilepsy drug SAN2355 [1][8] - SAN2355 is designed to selectively activate Kv7.2/Kv7.3 potassium channels, aiming to avoid safety issues associated with previous therapies like GSK's Potiga [2][3] - The deal allows JAZZ to enhance its neuroscience pipeline, complementing its existing epilepsy treatment, Epidiolex, which is expected to achieve blockbuster status [7][9] Financial Terms of the Deal - JAZZ will pay Saniona an upfront cash payment of $42.5 million, with potential milestone payments up to $192.5 million for development and regulatory achievements, and up to $800 million for commercial milestones [4] - Saniona will also receive royalties on future sales of SAN2355 [4] Market Context - JAZZ's stock has seen a decline of 3% year-to-date, contrasting with a 3% growth in the industry [5] - The acquisition of SAN2355 positions JAZZ for future innovation in the epilepsy market, potentially serving as a successor to Epidiolex once it loses exclusivity [9]

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 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]

Company Overview - NeuroPace, Inc. is a medical device company based in Mountain View, California, focused on transforming the lives of people living with epilepsy by reducing or eliminating debilitating seizures [3] - The company has developed the RNS System, which is the first and only commercially available brain-responsive platform that delivers personalized, real-time treatment at the seizure source [3] - The RNS System aims to improve the standard of care for patients with drug-resistant epilepsy and has potential applications for other brain disorders [3] Financial Results Announcement - NeuroPace will report its financial results for the second quarter of 2025 after market close on August 12, 2025 [1] - A conference call will be held at 1:30 p.m. Pacific Time (4:30 p.m. Eastern Time) to discuss the results [1] Conference Call Participation - Investors can access the live webcast of the conference call at the provided link [2] - For those wishing to participate via telephone, the call can be accessed by dialing +1-877-407-3982 and referencing Conference ID 13754370 [2] - The webcast will be archived on the company's investor relations website and available for replay for at least 90 days after the event [2]