Xenon's Azetukalner Shows Sustained Efficacy and Safety in Long-Term Focal Onset Seizure Study
• Xenon Pharmaceuticals' azetukalner demonstrates sustained reduction in seizure frequency and impressive seizure freedom rates in patients with focal onset seizures.
• Approximately one-third of patients treated with azetukalner for at least 36 months achieved seizure freedom for a year or longer, showcasing long-term efficacy.
• The X-TOLE open-label extension study highlights a consistent safety profile, suggesting azetukalner is generally well-tolerated over extended periods.
• Pre-clinical data indicates that Nav1.1 potentiators may offer protection against spontaneous seizures and SUDEP in a Dravet syndrome model.
Antisense Oligonucleotide Therapies Show Promise in Treating Genetic Epilepsies
• Early trials of antisense oligonucleotide (ASO) therapies for rare genetic epilepsies demonstrate potential in reducing seizure frequency and increasing seizure-free days.
• Stoke Therapeutics' Phase 1/2a trial of an ASO in Dravet syndrome showed significant reductions in convulsive seizure frequency in clinically evaluable patients.
• Praxis Precision Medicine's PRAX-222 for SCN2A-related epilepsy led to a 43% median reduction in seizures and a 48% increase in seizure-free days in early-onset patients.
• Despite promising results, the high cost of ASO treatments poses a significant challenge to accessibility, necessitating strategies like early genetic screening.
Clinical Trials Offer Hope for Children and Teens with Uncontrolled Seizures
• One-third of children with newly diagnosed epilepsy do not respond to the first antiseizure medication, leading to ongoing seizures.
• Uncontrolled seizures can significantly impair a child's academic, social, and emotional development, causing anxiety and distress.
• SK Life Science is conducting clinical trials for teens with primary generalized tonic-clonic seizures and young children with partial-onset seizures.
• These trials aim to develop new treatments for pediatric patients whose seizures are not adequately managed by traditional medications.
FDA Halts Rapport Therapeutics' RAP-219 Trial for Diabetic Neuropathic Pain
• The FDA has placed a clinical hold on Rapport Therapeutics' IND application for RAP-219, an investigational therapy for diabetic peripheral neuropathic pain (DPNP).
• The agency is requesting additional information on the trial's protocol design, which Rapport Therapeutics believes it can address promptly.
• This hold does not affect Rapport's other RAP-219 studies, including the ongoing Phase 2a trial for focal epilepsy and planned trial for bipolar disorder.
• Rapport Therapeutics anticipates providing an update on the Phase 2a trial initiation timeline once the FDA's requests are resolved.
Rapport Therapeutics Provides Update on RAP-219 Clinical Programs and Financial Status
• Rapport Therapeutics is advancing RAP-219 in Phase 2a trials for focal epilepsy, with topline data expected in mid-2025, showing commitment to CNS disorder treatments.
• The FDA placed a clinical hold on Rapport's RAP-219 trial for diabetic peripheral neuropathic pain, prompting the company to address protocol feedback.
• The company appointed biotech leaders to its Board of Directors and reported $320.7 million in cash, expected to fund operations through 2026.
• Rapport is set to release topline data from the RAP-219 MAD-2 and PET trials in Q1 2025, informing the bipolar disorder trial and target receptor occupancy.
Rapport Therapeutics Advances RAP-219 Clinical Trials for CNS Disorders
• Rapport Therapeutics is progressing with Phase 2a trial of RAP-219 in focal epilepsy, with topline data expected in mid-2025.
• Multiple Ascending Dose (MAD-2) and PET trials of RAP-219 are ongoing, with topline results anticipated in Q1 2025.
• The FDA placed a clinical hold on the IND for RAP-219 in diabetic peripheral neuropathic pain, pending protocol amendments.
• New biotech industry leaders have been appointed to Rapport's Board of Directors, bringing expertise in drug discovery and clinical development.
New Clinical Trial Investigates Novel Treatment for Focal Epilepsy
• A nationwide clinical trial, named Rise, is evaluating a potential new treatment for focal (partial) onset epilepsy in patients aged 18-75.
• The study focuses on individuals who have experienced resistance to existing anti-seizure medications and continue to have frequent seizures.
• The trial aims to address the significant unmet need for effective epilepsy treatments, as approximately 40% of patients do not achieve seizure control with current options.
• Enrollment is open across the United States for those meeting specific criteria, offering a chance to contribute to research and potentially benefit from a new therapy.
Clinical Trials Offer Hope for Children and Teens with Uncontrolled Seizures
• One-third of children with newly diagnosed epilepsy do not respond to the first antiseizure medication, highlighting the need for new treatments.
• Pediatric patients with uncontrolled seizures may face lifelong challenges affecting academic performance, social interactions, and emotional well-being.
• SK Life Science is enrolling patients in Phase 1 and Phase 3 clinical trials to evaluate a potential treatment for primary generalized tonic-clonic and partial-onset seizures in children and teens.
• Participation in clinical trials may provide access to innovative therapies and improve the quality of life for young patients with drug-resistant epilepsy.
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