Press release CROSSJECT advances on the development of ZEPIZURE® Junior, its solution for epilepsy crises in children Announces the effective calibration of ZENEO® for the use of ZEPIZURE® in the pediatric population.CROSSJECT completed an echography clinical study allowing to determine and validate the targeted depth of injection in children as compared to adults. Dijon, France 11 June 2025 (07h30 CET) -- CROSSJECT (ISIN: FR0011716265; Euronext: ALCJ), the specialty pharma company in advanced phases of dev ...

NeuroPace (NPCE) Update Summary Company Overview - **Company**: NeuroPace (NPCE) - **Industry**: Medical Devices, specifically focused on epilepsy treatment Key Points and Arguments 1. **NAUGHTLIST Study Results**: Preliminary one-year results from the NAUGHTLIST study were released, supporting an expanded clinical indication for idiopathic generalized epilepsy (IGE) [2][5] 2. **Safety and Effectiveness**: The trial met its primary safety endpoint but did not reach statistical significance for its primary effectiveness endpoint. However, there was observed efficacy in a clinically meaningful subgroup of patients [5][11] 3. **Patient Improvement**: Patients experienced improvements over time, with a median percent reduction in seizures after one year of RNS treatment higher than that observed in focal epilepsy trials leading to FDA approval [6][13] 4. **Clinical Need**: The data provides new hope for the IGE community, which has historically had limited treatment options [6][15] 5. **Study Design**: The NAUGHTLIST study was a prospective, multicenter, randomized, single-blind, sham-controlled trial involving 100 participants, with a focus on patients with drug-resistant IGE [8][9] 6. **Long-term Efficacy**: Data on longer-term seizure outcomes is encouraging, with median percent seizure reductions showing progressive improvement over time [12][14] 7. **FDA Engagement**: NeuroPace plans to submit the full dataset to the FDA in the second half of 2025 and is committed to engaging with the FDA regarding the potential for labeling aligned with the substantial subpopulation showing therapeutic benefit [15][18] 8. **Market Opportunity**: The total market for generalized epilepsy is estimated at around $800 million, with a significant portion likely represented by the subgroup that showed the highest clinical benefit [22][23] 9. **Growth Strategy**: The company’s base business is growing over 20% year over year, and the long-term strategy remains focused on expanding access to personalized therapy for epilepsy patients [19][70] Additional Important Content 1. **Forward-Looking Statements**: The call included forward-looking statements regarding projections, business opportunities, and market conditions, which involve risks and uncertainties [3][4] 2. **Patient Population Insights**: The trial included patients with severe IGE, and the majority had a lower frequency of generalized tonic-clonic seizures, which is reflective of the real-world patient population [25][62] 3. **Ongoing Analysis**: Further analyses are being conducted to understand patient characteristics that differentiate responses to treatment [12][38] 4. **Regulatory Designation**: The FDA granted breakthrough designation for the trial, recognizing it as a novel therapy addressing an unmet clinical need [64] 5. **Long-term Guidance**: The company’s long-term guidance remains unchanged, with ongoing discussions with the FDA and a commitment to advancing treatment options for IGE patients [70][71]

Summary of Xenon Pharmaceuticals Conference Call Company Overview - **Company**: Xenon Pharmaceuticals - **CEO**: Ian Mortimer Industry Context - **Industry**: Epilepsy treatment - **Market Size**: Approximately 3 million Americans have epilepsy, with 60% experiencing focal onset seizures, translating to about 2 million patients. 30-50% of these patients do not achieve good seizure control, indicating a significant market opportunity for new treatments [30][31]. Key Points and Arguments Clinical Trials and Drug Development - **Phase III Studies**: Xenon is conducting two Phase III clinical trials named XTOL-2 and XTOL-3, designed to mirror the successful Phase II study in terms of size, inclusion criteria, and dosing [6][7][9]. - **Efficacy Data**: The Phase II data for Ezetucalner showed robust efficacy, with a placebo-adjusted efficacy that is the best seen in focal onset seizures. The drug has a high power of over 99% at the primary endpoint in Phase III [4][9]. - **Enrollment Challenges**: There has been a slight delay in enrollment for XTOL-2, but the company is confident in completing enrollment in the coming months, with data expected in early 2026 [15][17]. Safety and Efficacy Profile - **Long-term Data**: Over 150 patients have been on the drug for more than three years, with a one in three chance of being seizure-free for 12 months or more. This is significant given the baseline characteristics of patients who had previously failed multiple treatments [22][23]. - **Adverse Events**: The safety profile is consistent with other anti-seizure medications, with common CNS adverse events like dizziness and fatigue. No significant issues related to pigmentation or urinary retention have been observed, addressing concerns from previous drugs in the same class [25][27][28]. Market Opportunity and Competitive Landscape - **Polypharmacy**: The treatment landscape for epilepsy often involves polypharmacy, with many patients not achieving adequate seizure control. Ezetucalner is positioned as a second or third-line treatment option [32][33]. - **Comparison with Competitors**: Ezetucalner offers advantages over competitors like XCOPRI, including no titration required and early onset of efficacy. XCOPRI is projected to generate $400-$450 million in sales this year, with expectations of reaching $1 billion by the end of the decade [34][35][36]. Future Directions - **Major Depressive Disorder (MDD)**: Xenon is also exploring Ezetucalner for MDD, with a Phase III program initiated. Initial data from a small investigator-sponsored study showed some treatment effects, but the company is focusing on its larger Phase III studies for more definitive results [37][41]. - **Pipeline Development**: The company is excited about its drug development pipeline, including new targets for pain and other syndromes, with plans for investor webinars to discuss these developments [52][53]. Additional Important Insights - **Regulatory Interactions**: Ongoing interactions with the FDA have not indicated any changes in the regulatory landscape, and the company expects more engagement as it approaches the NDA submission [19]. - **Patient Feedback**: Positive anecdotal feedback from physicians indicates that patients are experiencing improved quality of life, including increased independence and social interactions due to better seizure control [24]. This summary encapsulates the key points discussed during the conference call, highlighting the company's strategic focus, clinical trial progress, market positioning, and future opportunities.

Praxis Precision Medicines (PRAX) FY 2025 Conference May 20, 2025 09:30 AM ET Speaker0 Okay. Welcome, everybody. I'm Doug Sal, senior analyst at H. C. Wainwright. We are thrilled to have with us Praxis Precision Medicines, represented by the company's CFO, Tim Kelly. And Marcio Sauso, the company's CEO, is supposedly en route, so he might join us midway through the presentation. So you don't have to worry if somebody barges in and sits down. Speaker1 So it's Marcio versus the trains this morning. So I I thi ...

- Key Opinion Leaders (KOLs) on Absence seizures include Dennis Dlugos, MD, Wendyl D'Souza, MBChB, MPH, FRACP, PhD, and Alexander Rotenberg, MD, PhD - VANCOUVER, British Columbia, May 20, 2025 (GLOBE NEWSWIRE) -- Bright Minds Biosciences Inc. (CSE: DRUG) (NASDAQ: DRUG) ("Bright Minds" or the "Company"), a company focused on developing highly selective 5-HT2 agonists for the treatment of drug-resistant epilepsy, depression, and other central nervous system (CNS) disorders, today announced the featured speake ...

- BMB-101 demonstrated a complete elimination of drop attacks in the DBA/2 mouse model - - The DBA/2 model is highly predictive of sudden unexpected death in epilepsy (SUDEP) - - Findings highlight BMB-101’s potential to address critical gaps in SUDEP prevention - NEW YORK and VANCOUVER, British Columbia, May 13, 2025 (GLOBE NEWSWIRE) -- Bright Minds Biosciences Inc. (CSE: DRUG) (NASDAQ: DRUG) (“Bright Minds” or the “Company”), a company focused on developing highly selective 5-HT2 agonists for the treatmen ...

Raanana, Israel, May 13, 2025 (GLOBE NEWSWIRE) -- Polyrizon Ltd. (Nasdaq: PLRZ) (the “Company” or “Polyrizon”), a development stage biotech company specializing in the development of innovative intranasal hydrogels, recently announced the initiation of preclinical studies for intranasal Benzodiazepines (BZDs), a first-line treatment for acute repetitive seizures (ARS) and status epilepticus, using its proprietary drug delivery platform. According to the World Health Organization (WHO), epilepsy is a neurolo ...

On track for six major study readouts across four programs over the next 12 months Ready to initiate pivotal studies in two developmental and epileptic encephalopathy (DEE) programs in mid-year 2025: EMERALD for broad DEEs with relutrigine and EMBRAVE3 for SCN2A GoF with elsunersen Vormatrigine continues to generate a best-in-class safety profile with new data demonstrating no food effect and higher dosing tolerability Praxis to host a virtual investor event on Friday, May 2, 2025 to discuss its DEE portfol ...