Epilepsies, Partial

Interim data presented by the company last July demonstrated a dose-dependent slowing of the progression of Huntington’s Disease in individuals treated with the therapy. Credit: bangoland/Shutterstock. The US Food and Drug Administration (FDA) has awarded breakthrough therapy designation to uniQure’s AMT-130 for treating Huntington’s disease, a rare neurodegenerative condition. This designation for the therapy adds to a list of the agency’s recognitions, including regenerative medicine advanced therapy (RMAT), fast track and orphan drug designations. Clinical data from the ongoing Phase I/II studies of the therapy support this latest designation. Interim data presented by the company last July demonstrated a dose-dependent slowing of progression of the disease in individuals treated with the therapy, against a propensity-weighted natural history. Till now, 45 subjects received the treatment. uniQure chief medical officer Walid Abi-Saab said: “Receiving breakthrough therapy designation underscores both the urgent need for effective treatments for Huntington’s disease and the encouraging interim data demonstrating that AMT-130 has the potential to slow disease progression. “It’s a powerful recognition of the promise of AMT-130 and the important progress we’ve made. We deeply value the FDA’s continued commitment to advancing innovative gene therapies for patients with critical unmet needs, and we look forward to working closely with the agency to bring AMT-130 to the Huntington’s disease patient community as quickly as possible.” The breakthrough therapy status is granted to accelerate investigational therapies development for serious conditions when preliminary clinical evidence suggests a substantial improvement over existing treatments. uniQure noted that Huntington’s disease, which results in chorea, cognitive decline, and behavioural abnormalities, affects approximately 70,000 people in Europe and the US, with many more at risk. The company’s gene therapy for haemophilia B has paved the way for new treatment approaches in genomic medicine. uniQure is currently progressing with its pipeline of gene therapies aimed at treating Huntington’s disease, refractory temporal lobe epilepsy, Fabry disease, amyotrophic lateral sclerosis (ALS), and other severe conditions.

–EFFICACY: 92% median reduction in disabling seizures during 7-12-month efficacy endpoint period, and 80% (4/5) of patients responding with >75% seizure reduction in Cohort 1. –DURABILITY: All responders (4/4) in Cohort 1 continue to have durable seizure control for 18-24 months after a single-dose of NRTX-1001. –COGNITION AND QUALITY OF LIFE: All patients (5/5) in Cohort 1 had significantly improved quality-of-life test scores, with no patient experiencing persistent decline in cognition. – SAFETY: NRTX-1001 continues to be well-tolerated at both high and low doses with no adverse events attributed to the cell therapy. –NEXT STEPS: Phase 3 EPIC trial planned to start enrolling in 2H 2025, as aligned with FDA under RMAT designation, to support submission of a Biologics License Application (BLA). April 08, 2025 -- Neurona Therapeutics, a clinical-stage biotherapeutics company developing regenerative cell therapies for epilepsy and other disorders of the nervous system, today presented updated data from its open-label Phase 1/2 clinical trial of NRTX-1001 in adults with drug-resistant, unilateral mesial temporal lobe epilepsy (MTLE) at the American Academy of Neurology (AAN) 2025 Annual Meeting in San Diego, CA. The ongoing multicenter Phase 1/2 trial for unilateral MTLE has enrolled 18 subjects in Cohorts 1 and 2 across 13 comprehensive epilepsy centers in the United States, and 10 subjects have been followed for 9 to 24 months after NRTX-1001 administration. Patients in the low-dose Cohort 1 (N=5) achieved median disabling seizure reduction of 92% from baseline during the primary efficacy evaluation period in months 7-12 post-treatment, and a 97% median reduction of disabling seizures after month 13. In the high dose Cohort 2 (N=5), patients achieved median disabling seizure reduction of 72% from baseline in months 4-9, the most recent 6-month epoch, and have not yet reached month 12. “We are pleased with the results to-date from the ongoing trial of NRTX-1001 cell therapy,” said Dr. Jerry Shih, M.D., Neurologist and Director of the University of California San Diego (UCSD) Comprehensive Epilepsy Center, who has treated 4 subjects in the trial alongside UCSD Neurosurgeon and Surgical Director of Epilepsy, Dr. Sharona Ben-Haim, M.D. “Our first subject in the study is now 19 months post-cell administration and has experienced a 98% reduction in his drug-resistant disabling seizures. He successfully stopped the temporary immunosuppression regimen 6 months ago, and continues to have a durable outcome, free from any study-related adverse effects. If these results continue to be observed in the upcoming Phase 3 EPIC trial, cell therapy has the potential to become a paradigm-shifting treatment option for patients with drug-refractory focal epilepsy.” Said Dr. John Hixson, M.D., VP of Clinical Development at Neurona, “The results of the trial thus far point to the promise of NRTX-1001 being a cell therapy that can address a major unmet need for patients with drug-resistant focal seizures, providing durable seizure control and avoiding the significant risk of permanent neurocognitive impairment from current tissue-destructive surgeries like laser ablation and brain resection. To that end, NRTX-1001 could represent a new treatment option that preserves brain tissue and function.” Dr. Hixson continued, “We are pleased with the continued durability signal from the ongoing NRTX-1001 study, supporting a single ‘one-and-done’ dose. We are also very encouraged by the lack of cell-related adverse effects and absence of neurocognitive impairment thus far. In summary, the data give us confidence to proceed with our planned Phase 3 EPIC trial of NRTX-1001, as we seek to bring this novel therapy to patients as safely and expeditiously as possible.” Results: Low Dose Effect on Disabling Seizures (N=5): Months 7-12: 92% median seizure reduction Months 13+: 97% median seizure reduction Durability: All (4/4) patients who exhibited marked seizure reduction in first year continue to have durable seizure control in second year after completing the 12-month immunosuppression regimen, which is intended to facilitate long-term persistence of the allogeneic cell therapy Quality-of-Life: All patients have significantly improved Quality-Of-Life In Epilepsy (QOLIE) overall test scores from baseline levels High Dose Effect on Disabling Seizures (N=5): Months 4-9 (latest 6-month epoch to date): 72% median seizure reduction 80% (4/5) subjects with >50% seizure reduction from baseline Safety – Low and High Dose Cohorts (N=10): No adverse events determined to be related to NRTX-1001 Neurocognition – Low and High Dose Cohorts (N=10): No persistent decline in cognition based on word retrieval, verbal memory, and visuospatial memory batteries The open-label Phase 1/2 clinical trial was designed to evaluate the safety and preliminary efficacy of a single-dose administration of NRTX-1001 inhibitory neuron cell therapy for drug-resistant unilateral MTLE. This ongoing trial has been expanded to include 8 additional subjects for a total of 18 subjects across both cohorts: low-dose cohort 1 (N=9) and high-dose cohort 2 (N=9). Study subjects are being monitored for safety, tolerability, and effects on their epilepsy disease symptoms. In addition, a second multicenter, open-label Phase 1/2 clinical trial has been initiated to evaluate NRTX-1001 in adults with drug-resistant bilateral MTLE. For more information, please visit www.clinicaltrials.gov (NCT05135091 and NCT06422923). In February, the company announced plans to initiate its Phase 3 EPIC (EPIlepsy Cell therapy) trial, which is expected to be a single pivotal study and primary basis of a future BLA submission for NRTX-1001 in drug-resistant MTLE, based on alignment gained with FDA under the RMAT designation. The EPIC trial is anticipated to begin in the second half of 2025 and is designed as a randomized, sham-controlled, double-blind study of NRTX-1001 in adults with drug-resistant MTLE, the most common type of epilepsy in adults. Earlier this month, Neurona closed an upsized and oversubscribed $102 million private financing with support from new and existing investors. AAN Presentation Details: Title: First-in-Human Trial of NRTX-1001 GABAergic Interneuron Cell Therapy for Focal Epilepsy - Updated Clinical Trial Results Presenting Author: John D. Hixson, M.D., VP of Clinical Development, Neurona Presentation: Tuesday April 8, 2025; Poster Session 8 (8:00 AM-9:00 AM); Number: 9-003 Neurona is developing allogeneic, off-the-shelf, regenerative neural cell therapies with the potential to provide long-term targeted repair of the nervous system following a single administration. The Company’s lead product candidate, NRTX-1001, comprising GABAergic interneurons, is currently being studied for safety and efficacy in two ongoing open label multicenter Phase 1/2 trials: NCT05135091 for drug-resistant unilateral mesial temporal lobe epilepsy (MTLE), and NCT06422923 for drug-resistant bilateral MTLE, with expansion to neocortical focal epilepsy and other indications planned in the future. The Phase 1/2 MTLE clinical trials are supported by grants from the California Institute for Regenerative Medicine (CIRM; CLIN2-13355 and CLIN2-17135). The FDA granted the Regenerative Medicine Advanced Therapy (RMAT) designation to NRTX-1001 in June 2024. The content above comes from the network. if any infringement, please contact us to modify.

PARAMUS, N.J., April 4, 2025 /PRNewswire/ -- SK Life Science, Inc., a global leader in developing treatments for central nervous system (CNS) disorders and a subsidiary of SK Biopharmaceuticals Co., Ltd., will present meaningful encore data on XCOPRI® (cenobamate tablets) CV, an anti-seizure medication, at the 77th American Academy of Neurology (AAN) Annual Meeting, taking place April 5-9, 2025, in San Diego, California. This presentation reinforces the growing body of clinical and real-world evidence supporting cenobamate's role in reducing seizure activity and optimizing epilepsy management. "Our goal at SK Life Science is to redefine seizure management by advancing research and education that translates into meaningful improvements for people living with epilepsy," said Louis Ferrari, BS, RPh, MBA, vice president, Medical Affairs at SK Life Science, Inc. "Seizure freedom remains an elusive, yet critical, objective for many patients, and this research offers valuable insights into how RNS data can be used as an objective measure to assess the efficacy of XCOPRI, and possibly other add-on anti-seizure medications." At AAN 2025, SK Life Science will highlight the impact of cenobamate on RNS epileptiform events. A retrospective, multicenter, observational, 24-week study among 37 patients with uncontrolled seizures showed a meaningful reduction in epileptiform events and clinically reported seizures during adjunctive cenobamate treatment. Results from this analysis support, through electrocorticographic data and observation of clinical seizures, the effectiveness of cenobamate in the treatment of focal seizures. Additional clinical and real-world data exploring cenobamate's efficacy, safety, and therapeutic potential in diverse patient populations will also be presented. Select SK Life Science posters presented at 2025 AAN include: Title: The Effect of Cenobamate on Responsive Neurostimulation Epileptiform Events (Aboumatar et al) Session: P5: Epilepsy/Clinical Neurophysiology (EEG): Neuromodulation Devices and Seizure Tracking Date/Time: Monday, April 7, from 8:00 a.m. – 9:00 a.m. PT Abstract Number: 2991 Title: A Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of Adjunctive Cenobamate in Asian Patients With Focal Seizures, With Optional Open-Label Extension (Misra et al) Session: P8: Epilepsy/Clinical Neurophysiology (EEG): Anti-seizure Medications: Clinical Trials Date/Time: Tuesday, April 8, from 8:00 a.m. – 9:00 a.m. PT Abstract Number: 1406 Title: Early Response Rates With Adjunctive Cenobamate in Uncontrolled Focal Seizures: A Randomized, Double-Blind, Placebo-Controlled, Multicenter Study in a Multinational Asian Population (Pradhan et al) Session: P8: Epilepsy/Clinical Neurophysiology (EEG): Anti-seizure Medications: Clinical Trials Date/Time: Tuesday, April 8, from 8:00 a.m. – 9:00 a.m. PT Abstract Number: 1404 Title: Efficacy of Adjunctive Cenobamate by Focal Seizure Subtypes: A Randomized, Double-Blind, Placebo-Controlled, Multicenter Study in a Multinational Asian Population (Ferrari et al) Session: P8: Epilepsy/Clinical Neurophysiology (EEG): Anti-seizure Medications: Clinical Trials Date/Time: Tuesday, April 8, from 8:00 a.m. – 9:00 a.m. PT Abstract Number: 1408 Title: Clarification of the Mechanism of Action of Cenobamate (Sankar et al) Session: P9: Epilepsy/Clinical Neurophysiology (EEG): Anti-seizure Medications: Mechanisms and Pharmacology Date/Time: Tuesday, April 8, from 11:45 a.m. – 12:45 p.m. PT Abstract Number: 1381 Title: Wide Range of Cenobamate Doses Associated With Initial Seizure Freedom in Patients With Uncontrolled Focal Seizures: Post-hoc Analysis of a Phase 3, Multicenter, Open-Label Study (Rosenfeld et al) Session: P12: Epilepsy/Clinical Neurophysiology (EEG): Retrospective Studies, Reviews, and Meta-analyses in Epilepsy Date/Time: Wednesday, April 9, from 11:45 a.m. – 12:45 p.m. PT Abstract Number: 158 A complete list of SK Life Science abstracts presented at AAN 2025 can be found here. In addition to these scientific presentations, SK Life Science, Inc. will host a booth (#223) in the Exhibit Hall, open from 11:30 a.m. PT on Sunday, April 6, 2025, until 4 p.m. PT on Wednesday, April 9, 2025. Attendees are encouraged to visit the booth to learn more about SK Life Science's commitment to epilepsy research and patient care. About SK Life Science, Inc. and SK Biopharmaceuticals Co., Ltd. SK Life Science, Inc., with headquarters in Paramus, New Jersey, is a U.S. subsidiary of SK Biopharmaceuticals Co., Ltd., a pioneering South Korean company in drug development and commercialization. Together, they are advancing innovative treatments for central nervous system (CNS) disorders and oncology, with eight compounds currently in development. Utilizing target-based drug discovery, high-throughput organic screening/high content screening, computer-aided drug design, and combinatorial chemistry, the companies drive R&D efforts in biology/discovery, medicinal chemistry, pharmacology, and clinical development. For more information, visit . SK Biopharmaceuticals Co., Ltd. is part of SK Group, South Korea's second-largest conglomerate. SK Group is a collection of global industry-leading companies driving innovations in energy, advanced materials, biopharmaceuticals and digital business. Based in Seoul, SK invests in building sustainable businesses around the world with a shared commitment to reducing global greenhouse gas emissions. SK companies combined have $151 billion in global annual revenue and employ more than 100,000 people worldwide. SK Group is one of TIME's 100 Most Influential Companies of 2023. SK Inc., the parent company of SK Biopharmaceuticals, continues to enhance its portfolio value by executing long-term investments with a number of competitive subsidiaries in various business areas, including pharmaceuticals and life science, energy and chemicals, information and telecommunication, and semiconductors. In addition, SK Inc. is focused on reinforcing its growth foundations through profitable and practical management based on financial stability, while raising its enterprise value by investing in new future growth businesses. For more information about SK Inc., visit . For more information about SK Biopharmaceuticals, visit . About XCOPRI® (cenobamate tablets) CV Cenobamate is an antiseizure medication (ASM) discovered and developed by SK Biopharmaceuticals and SK Life Science. Cenobamate reduces neuronal excitability through a unique dual mechanism of action, preferentially inhibiting the persistent sodium current and enhancing GABAergic inhibition at the type A γ-aminobutyric acid (GABAA) ion channel. The precise mechanism by which cenobamate exerts its therapeutic effect is unknown. Cenobamate is marketed under the brand name XCOPRI® in the U.S. by SK Life Science, Inc. Additionally, XCOPRI is commercialized in Canada and Israel by SK Biopharmaceuticals' partners, Paladin Labs Inc. and Dexcel Ltd. Cenobamate is marketed as ONTOZRY® by Angelini Pharma S.p.A. in Europe, the UK, and Switzerland. Cenobamate is also being developed for commercialization by SK Biopharmaceuticals' partners in many other countries to meet the needs of patients living with epilepsy, including Dong-A ST Co., Ltd., Eurofarma Laboratórios S.A., Hikma MENA FZE, Ignis Therapeutics, Inc. and ONO Pharmaceutical Co., Ltd. IMPORTANT SAFETY INFORMATION AND INDICATION FOR XCOPRI® (cenobamate tablets) CV DO NOT TAKE XCOPRI IF YOU: Are allergic to cenobamate or any of the other ingredients in XCOPRI. Have a genetic problem (called Familial Short QT syndrome) that affects the electrical system of the heart. XCOPRI CAN CAUSE SERIOUS SIDE EFFECTS, INCLUDING: Allergic reactions: XCOPRI can cause serious skin rash or other serious allergic reactions which may affect organs and other parts of your body like the liver or blood cells. You may or may not have a rash with these types of reactions. Call your healthcare provider right away and go to the nearest emergency room if you have any of the following: swelling of your face, eyes, lips, or tongue, trouble swallowing or breathing, a skin rash, hives, fever, swollen glands, or sore throat that does not go away or comes and goes, painful sores in the mouth or around your eyes, yellowing of your skin or eyes, unusual bruising or bleeding, severe fatigue or weakness, severe muscle pain, frequent infections, or infections that do not go away. Take XCOPRI exactly as your healthcare provider tells you to take it. It is very important to increase your dose of XCOPRI slowly, as instructed by your healthcare provider. QT shortening: XCOPRI may cause problems with the electrical system of the heart (QT shortening). Call your healthcare provider if you have symptoms of QT shortening including fast heartbeat (heart palpitations) that last a long time or fainting. Suicidal behavior and ideation: Antiepileptic drugs, including XCOPRI, may cause suicidal thoughts or actions in a very small number of people, about 1 in 500. Call your health care provider right away if you have any of the following symptoms, especially if they are new, worse, or worry you: thoughts about suicide or dying; attempting to commit suicide; new or worse depression, anxiety, or irritability; feeling agitated or restless; panic attacks; trouble sleeping (insomnia); acting aggressive; being angry or violent; acting on dangerous impulses; an extreme increase in activity and talking (mania); or other unusual changes in behavior or mood. Nervous system problems: XCOPRI may cause problems that affect your nervous system. Symptoms of nervous system problems include: dizziness, trouble walking or with coordination, feeling sleepy and tired, trouble concentrating, remembering, and thinking clearly, and vision problems. Do not drive, operate heavy machinery, or do other dangerous activities until you know how XCOPRI affects you. Do not drink alcohol or take other medicines that can make you sleepy or dizzy while taking XCOPRI without first talking to your healthcare provider. DISCONTINUATION: Do not stop taking XCOPRI without first talking to your healthcare provider. Stopping XCOPRI suddenly can cause serious problems. Stopping seizure medicine suddenly in a patient who has epilepsy can cause seizures that will not stop (status epilepticus). DRUG INTERACTIONS: XCOPRI may affect the way other medicines work, and other medicines may affect how XCOPRI works. Do not start or stop other medicines without talking to your healthcare provider. Tell healthcare providers about all the medicines you take, including prescription and over-the-counter medicines, vitamins and herbal supplements. PREGNANCY AND LACTATION: XCOPRI may cause your birth control medicine to be less effective. Talk to your health care provider about the best birth control method to use. Talk to your health care provider if you are pregnant or plan to become pregnant. It is not known if XCOPRI will harm your unborn baby. Tell your healthcare provider right away if you become pregnant while taking XCOPRI. You and your healthcare provider will decide if you should take XCOPRI while you are pregnant. If you become pregnant while taking XCOPRI, talk to your healthcare provider about registering with the North American Antiepileptic Drug (NAAED) Pregnancy Registry. The purpose of this registry is to collect information about the safety of antiepileptic medicine during pregnancy. You can enroll in this registry by calling 1-888233-2334 or go to . Talk to your health care provider if you are breastfeeding or plan to breastfeed. It is not known if XCOPRI passes into breastmilk. Talk to your healthcare provider about the best way to feed your baby while taking XCOPRI. COMMON SIDE EFFECTS: The most common side effects in patients taking XCOPRI include dizziness, sleepiness, headache, double vision, and feeling tired. These are not all the possible side effects of XCOPRI. Tell your healthcare provider if you have any side effect that bothers you or that does not go away. For more information, ask your healthcare provider or pharmacist. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088 or at . DRUG ABUSE: XCOPRI is a federally controlled substance (CV) because it can be abused or lead to dependence. Keep XCOPRI in a safe place to prevent misuse and abuse. Selling or giving away XCOPRI may harm others and is against the law. INDICATION: XCOPRI is a prescription medicine used to treat partial-onset seizures in adults 18 years of age and older. It is not known if XCOPRI is safe and effective in children under 18 years of age. Please see additional patient information in the Medication Guide. This information does not take the place of talking with your healthcare provider about your condition or your treatment. Please see full Prescribing Information. About Epilepsy Epilepsy is the fourth most common neurological disorder. There are approximately 3.4 million people living with epilepsy in the United States, with 150,000 new cases each year in the country.1,2 Epilepsy is characterized by recurrent, unprovoked seizures. The seizures in epilepsy may be related to a brain injury or a family tendency, but often the cause is completely unknown. Having seizures and epilepsy can affect one's safety, relationships, work, driving, and much more.3,4 People with epilepsy are at risk for accidents and other health complications, including falling, drowning, depression and sudden unexplained death in epilepsy (SUDEP).3,4 Despite the availability of many antiepileptic therapies, almost 40 percent of people with epilepsy are not able to achieve seizure freedom, meaning they have epilepsy that remains uncontrolled.5 XCOPRI® and ONTOZRY® are registered trademarks of SK Biopharmaceuticals Co., Ltd. References Cleveland Clinic. Epilepsy. . Accessed April 2025. Epilepsy Foundation. Facts & Statistics About Epilepsy. . Accessed April 2025. Epilepsy Foundation. Staying Safe. . Accessed April 2025. Epilepsy Foundation. Complications and Risks. . Accessed April 2025. Chen Z, Brodie MJ, Liew D, Kwan P. Treatment outcomes in patients with newly diagnosed epilepsy treated with established and new antiepileptic drugs: a 30-year longitudinal cohort study. . Published online December 26, 2017. SOURCE SK Life Science, Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED