Bexicaserin

EPIDIOLEX has significant market potential due to the growing demand for alternative therapies and its effectiveness in treating rare seizure disorders like Lennox-Gastaut syndrome and Dravet syndrome. As awareness around the benefits of CBD in treating neurological conditions increases, EPIDIOLEX stands to benefit from expanding indications and market reach. LAS VEGAS, May 1, 2025 /PRNewswire/ -- DelveInsight's ' EPIDIOLEX Market Size, Forecast, and Market Insight Report' highlights the details around EPIDIOLEX, the first FDA-approved prescription cannabidiol (CBD) to treat seizures associated with Lennox-Gastaut syndrome (LGS), Dravet syndrome, or tuberous sclerosis complex (TSC) in patients 1 year of age or older. The report provides product descriptions, patent details, and competitor products (marketed and emerging therapies) of EPIDIOLEX. The report also highlights the historical and forecasted sales from 2020 to 2034 segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan]. Jazz Pharmaceuticals' EPIDIOLEX (cannabidiol) Overview EPIDIOLEX (formerly known as GWP42003-P and marketed as EPIDYOLEX in Europe) is the first plant-based, prescription cannabis-derived oral medication developed by GW Pharmaceuticals. It represents a new category of antiepileptic drugs with a unique mechanism of action. The active compound, cannabidiol (CBD), is a naturally occurring cannabinoid found in the Cannabis sativa L. plant. While the exact way EPIDIOLEX works to reduce seizures in humans remains unclear, it is distinct from traditional anticonvulsants. CBD does not act through CB1 receptors or by blocking voltage-gated sodium channels, which are typical pathways for other antiepileptic drugs. Instead, CBD is thought to work by influencing multiple internal pathways, potentially producing a combined anticonvulsant effect. These include enhancing neuronal inhibition (through synaptic and extrasynaptic GABA channels), affecting intracellular calcium levels (via TRPV channels, VDAC, and GPR55), and reducing inflammation, possibly via adenosine modulation. Importantly, CBD does not directly bind to or activate the CB1 or CB2 receptors at levels relevant to its seizure-reducing effects. Ongoing research is examining other possible mechanisms such as interactions with adenosine, glycine, GABAergic systems, and serotonin receptors. EPIDIOLEX has received regulatory approval in numerous countries, including the U.S. (FDA), Europe (European Commission), Great Britain (MHRA), Australia (Therapeutic Goods Administration), Switzerland (Swissmedic), Israel (Ministry of Health), and New Zealand (Medicines and Medical Devices Safety Authority). It is formulated as an oral solution containing highly purified CBD. In the U.S., it is approved to treat seizures linked to Lennox-Gastaut syndrome (LGS), Dravet syndrome (DS), and tuberous sclerosis complex (TSC) in patients aged one year and older. In the European Union, under the name Epidyolex, it is approved as an add-on treatment with clobazam for LGS and DS in patients two years and older, and for TSC-associated seizures as well. Both the FDA and EMA have granted Orphan Drug Designation (ODD) for EPIDIOLEX/EPIDYOLEX for these conditions. EPIDIOLEX/EPIDYOLEX net product sales rose by 15% to USD 972.4 million in 2024 and by 14% to USD 275.0 million in the fourth quarter of 2024, compared to the corresponding periods in 2023. Learn more about EPIDIOLEX/EPIDYOLEX projected market size for DEE @ EPIDIOLEX Market Potential Developmental and epileptic encephalopathies (DEE) encompass a group of severe neurological conditions marked by developmental delays and persistent epilepsy. These disorders typically present early in life and are linked to significant cognitive deficits. According to DelveInsight's analysis, there were approximately 287K diagnosed prevalent cases of DEE across the 7MM in 2024. This number is projected to increase due to the rise in neurological disorders, including epilepsy, as well as environmental and maternal health factors contributing to a higher DEE prevalence throughout the forecast period (2025−2034). The US FDA has approved a range of medications for conditions like LGS, Dravet Syndrome, and CDKL5 Deficiency Disorder, including various drug classes such as sodium channel modulators, GABA receptor modulators, calcium channel blockers, receptor blockers, and others. Some of the available anti-epileptic drugs (AEDs) include FINTEPLA, EPIDIOLEX, TOPAMAX, BANZEL, LAMICTAL, FELBATOL, ONFI, KLONOPIN, DIACOMIT, SABRIL, and more. In 2024, the total market size for DEE in the 7MM was around USD 2.3 billion, and this figure is expected to grow by 2034, driven by wider adoption of approved therapies due to label expansions and the introduction of emerging treatments. Discover more about the DEE market in detail @ Developmental and Epileptic Encephalopathy Market Report Emerging Competitors of EPIDIOLEX Some of the drugs in the pipeline include EPX-100 (Harmony Biosciences), Bexicaserin (Lundbeck), Zorevunersen (Stoke Therapeutics/Biogen), Relutrigine (Praxis Precision Medicines), and others. In August 2024, the FDA removed a partial clinical hold as the company advances toward a Phase III registrational study of zorevunersen (STK-001) in children and adolescents with Dravet syndrome. In July 2024, Longboard Pharmaceuticals announced that the FDA had granted Breakthrough Therapy designation for its investigational drug bexicaserin for the treatment of seizures associated with DEEs for patients 2 years of age or older. To know more about the number of competing drugs in development, visit @ EPIDIOLEX Alternatives Key Milestones of EPIDIOLEX In August 2024, Jazz Pharmaceuticals reported top-line results from a Phase 3 open-label, single-arm study conducted in Japan, assessing the safety and effectiveness of its cannabidiol oral solution (marketed globally as EPIDIOLEX/EPIDYOLEX) as an add-on therapy for seizures linked to Lennox-Gastaut syndrome (LGS), Dravet syndrome (DS), or tuberous sclerosis complex (TSC). While the trial did not achieve its primary efficacy goal—defined as a pre-specified percentage reduction in seizure frequency during the up-to-16-week treatment period compared to baseline in Japanese pediatric patients—numerical improvements were noted in both the primary and several secondary measures. The study did not identify any new safety concerns. In May 2021, Jazz Pharmaceuticals acquired EPIDIOLEX as part of the acquisition of GW Pharmaceuticals plc, or GW, which expanded its growing neuroscience business with a global, high-growth childhood-onset epilepsy franchise. In April 2021, EPIDYOLEX was approved for adjunctive therapy of seizures associated with TSC for patients 2 years of age and older in the EU. In July 2020, EPIDYOLEX was approved for the treatment of seizures associated with TSC in patients one year of age and older. FDA also approved the expansion of all existing indications, LGS and DS, to patients one year of age and older. In September 2019, the European Commission approved EPIDYOLEX for use as adjunctive therapy of seizures associated with LGS or DS, in conjunction with clobazam, for patients two years of age and older. In June 2018, EPIDIOLEX was approved in the US for the treatment of seizures associated with two rare and severe forms of epilepsy, LGS and DS, in patients two years of age and older. EPIDIOLEX Launch Status EPIDIOLEX was launched on November 1, 2018, in the US market after FDA approval for the treatment of seizures associated with LGS or Dravet syndrome in patients two years of age and older. EPIDYOLEX was launched in 2019 in Germany and the UK market for use as adjunctive therapy for seizures associated with LGS or Dravet syndrome, in conjunction with clobazam, for patients two years of age and older (GW Pharmaceuticals, 2019b). In 2021, EPIDYOLEX was launched in Spain. In the first quarter of 2022, the company launched EPIDYOLEX for LGS, Dravet syndrome, and TSC in Ireland, for TSC in Scotland and Wales. In the third quarter of 2022, the company launched EPIDYOLEX for TSC in Italy and Switzerland. In the fourth quarter of 2022, the company successfully completed the pricing and reimbursement process and commercial launch of EPIDYOLEX in France. Discover how EPIDIOLEX/EPIDYOLEX is shaping the DEE treatment landscape @ EPIDIOLEX CBD EPIDIOLEX Market Dynamics EPIDIOLEX, the first FDA-approved cannabidiol (CBD)-based medication for the treatment of rare forms of epilepsy, has had a significant impact on the pharmaceutical market since its approval in 2018. The drug is primarily used to treat seizures associated with Dravet syndrome and Lennox-Gastaut syndrome, two rare and severe forms of epilepsy. The market dynamic for EPIDIOLEX is shaped by several factors, including increasing awareness of its therapeutic potential, the expanding medical cannabis industry, and the growing demand for alternative treatments to traditional anti-epileptic drugs. One of the main drivers of the EPIDIOLEX market is the rising acceptance of CBD-based therapies. As medical cannabis becomes more mainstream and research into its medicinal properties continues to grow, patients and healthcare providers are increasingly turning to EPIDIOLEX as a treatment option. The growing body of clinical evidence supporting its effectiveness and safety has reinforced this trend. Additionally, as the stigma around cannabis-derived medications continues to fade, it opens up more opportunities for EPIDIOLEX in both pediatric and adult patient populations. However, the market for EPIDIOLEX is not without its challenges. High treatment costs and insurance coverage issues can limit accessibility for some patients, especially in markets outside the United States. Despite its clinical efficacy, the price point for EPIDIOLEX remains a point of contention, as it may not be affordable for all families, especially when compared to other conventional anti-epileptic drugs. Furthermore, competition from other cannabinoid-based therapies, as well as new developments in the broader epilepsy drug market, could impact the drug's market share over time. Looking ahead, the market dynamics for EPIDIOLEX will likely continue to evolve as the global regulatory landscape changes and new clinical trials further establish its benefits in other neurological conditions. With increasing research into CBD's potential applications, there may be opportunities for EPIDIOLEX to expand its indications, providing a solid foundation for its continued growth. However, market growth will also depend on the ability to address pricing concerns, improve insurance coverage, and navigate potential competitive pressures from both traditional and emerging therapies. Dive deeper to get more insight into EPIDIOLEX/EPIDYOLEX's strengths & weaknesses relative to competitors @ EPIDIOLEX Market Drug Report Table of Contents Related Reports Developmental and Epileptic Encephalopathies Market Developmental and Epileptic Encephalopathies Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key DEE companies, including vid Therapeutics, Stoke Therapeutics, Biogen, Harmony Biosciences, Lundbeck (Longboard Pharmaceuticals), Praxis Precision Medicines, SK Life Science, Cerecin Neurosciences, Encoded Therapeutics, Praxis Precision Medicines, among others. Developmental and Epileptic Encephalopathies Pipeline Developmental and Epileptic Encephalopathies Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key DEE companies, including Denovo Biopharma, Cantex Pharmaceuticals, CNS Pharmaceuticals, CANbridge Pharmaceuticals, Vaximm, Inovio Pharmaceuticals, Mustang Bio, Bullfrog AI Holdings, Cantex, Chimeric Therapeutics, Philogen, Boehringer Ingelheim, Photonamic GmbH, Berg Pharma, Beyond Bio, Genenta Science, Polaris Pharmaceuticals, Telix Pharmaceuticals, Shanghai Simnova Biotechnology, NEONC Technologies, among others. Dravet Syndrome Market Dravet Syndrome Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key Dravet syndrome companies including Biocodex, GW Pharmaceuticals, Zogenix, Ovid Therapeutics, PTC Therapeutics, among others. Lennox-Gastaut Syndrome Market Lennox-Gastaut Syndrome Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key Lennox-Gastaut syndrome companies, including GlaxoSmithKline, Meda Pharmaceuticals, Roche, Lundbeck, Greenwich Biosciences, Janssen Pharmaceuticals, Eisai, Zogenix, Takeda, Ovid Therapeutics, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

VALBY, Denmark, April 4, 2025 /PRNewswire/ -- H. Lundbeck A/S (Lundbeck) will present positive interim results from the open-label extension of the PACIFIC trial investigating bexicaserin, in addition to new analyses from clinical trials and real-world data with eptinezumab. H. Lundbeck A/S (Lundbeck) today announced that recent pipeline data will be presented at the 2025 AAN Annual Meeting in San Diego, U.S. The data includes an oral presentation of the six-month results from the open-label extension (OLE) of the Phase 1b/2a PACIFIC trial of bexicaserin, a novel treatment under development for seizures associated with Developmental and Epileptic Encephalopathies (DEEs).1 DEEs are the most severe group of epilepsies characterized by drug-resistant seizures, and developmental slowing or regression.2 "DEEs can be caused by a range of acquired, syndromal, and genetic etiologies, with more than 900 genes implicated. However, only a few subtypes currently have approved therapies, leaving many patients in need. The DEE-inclusive PACIFIC trial and six-month OLE data indicate that bexicaserin may be able to help address this unmet need across multiple DEE types, and highlights Lundbeck's expanding commitment to the neuro-rare space. We look forward to engaging with the global neuroscience community at AAN to discuss the data and potential of bexicaserin to support patients, caregivers and healthcare professionals in the management of DEEs," said Johan Luthman, EVP and Head of Research & Development at Lundbeck. The OLE included patients who successfully completed the PACIFIC trial3 and was designed to evaluate the long-term safety (up to 52 weeks), tolerability, and efficacy of bexicaserin in a cohort of participants with DEEs who were newly exposed to bexicaserin for at least 6 months.1 The OLE interim analysis showed that bexicaserin continues to exhibit a favourable safety and tolerability profile at six months, consistent with the safety profile observed during the PACIFIC trial. All bexicaserin treatment-naive patients (n=9) successfully transitioned from placebo to bexicaserin, reinforcing the tolerability of bexicaserin in an inclusive DEE population. In this placebo-bexicaserin switch population, a 57.3% reduction in countable motor seizures and 61.2% reduction in total seizures was observed, consistent with the response in non-naive participants at 6 months (n=32).* Moreover, more than half of patients newly exposed to bexicaserin (n=5/9) experienced a ≥50% reduction from baseline in countable motor seizures.1* In addition, Lundbeck will present recent post-hoc analyses and real-world data for eptinezumab investigating meaningful endpoints, such as good days per month†, and sustained treatment response. Lundbeck remains focused on raising the bar around preventive treatment expectations in migraine and increasing the understanding of the holistic impact of migraine on quality of life. *As compared to baseline at entry into PACIFIC study. †As reported by trial patients based on individual migraine experience Details of Lundbeck presentations at AAN 2025: Bexicaserin Oral Presentation Monday, April 7, Session S20, 4:18 – 4:30pm PT Title: Safety, Tolerability, and Efficacy of Bexicaserin in a Cohort of Participants with Developmental and Epileptic Encephalopathies: Interim Results of a Phase 1b/2a PACIFIC Study Open-Label Extension Eptinezumab Poster Presentations: Title: Long-term Maintenance of ≥50% and ≥75% Migraine Response With Eptinezumab in Patients With High-frequency Episodic Migraine and Chronic Migraine and 2-4 Prior Migraine Preventive Treatment Failures4 I Poster number: P12.005 Title: Patient-reported Impact of ≥75% Increase in Good Days/Month on Migraine Symptoms, Quality of Life, and Brain Fog: Real-world Study of Adults With Chronic Migraine Treated With Eptinezumab5 I Poster number: P12.010 Title: Long-term Reductions in Monthly Headache Days With Eptinezumab Treatment in Adults With Chronic Migraine: Results From the PREVAIL Study6 I Poster number: P12.003 About Bexicaserin Bexicaserin (LP352) is an oral, centrally acting 5-hydroxytryptamine 2C (5-HT2C) receptor superagonist with no observed impact on 5-HT2B and 5-HT2A receptor subtypes. It is being evaluated in a global Phase III clinical program (the DEEp Program). The FDA has granted Breakthrough Therapy designation for bexicaserin for the treatment of seizures associated with Developmental and Epileptic Encephalopathies (DEEs) for patients two years of age and older. Bexicaserin is an investigational compound that is not approved for marketing by any regulatory authority worldwide. The PACIFIC trial was a randomised, double-blind, placebo-controlled, DEE-inclusive trial (NCT05364021) in 52 participants with Dravet syndrome, Lennox-Gastaut syndrome, or DEE Other, who had ≥4 countable motor seizures during the 28-day baseline period, and who were on a stable regimen of 1 to 4 concomitant antiseizure medications.3 About eptinezumab (Vyepti®) Eptinezumab is a humanized monoclonal antibody that binds to calcitonin gene-related peptide (CGRP) which was purposefully developed for IV administration. The efficacy and safety of eptinezumab 100 mg and 300 mg was investigated in two phase III clinical trials (PROMISE-1 in episodic migraine and PROMISE-2 in chronic migraine), where eptinezumab met its primary endpoint of decrease in mean monthly migraine days (MMD) over weeks 1-12 in both episodic and chronic migraine. Furthermore, the clinical trial program observed a treatment benefit over placebo that was observed for both doses of eptinezumab as early as day 1 post-infusion. The safety of eptinezumab was evaluated in more than 2,000 adult patients with migraine who received at least one dose of eptinezumab. The most common adverse reactions (≥2% and at least 2% or greater than placebo) in the clinical trials for the preventive treatment of migraine were nasopharyngitis and hypersensitivity. Approximately 8% of patients on 300 mg, 6% of patients on 100 mg and 6% of patients on placebo in PROMISE-1 and PROMISE-2 experienced nasopharyngitis. In PROMISE-1 and PROMISE-2, 1.9% of patients treated with eptinezumab discontinued treatment due to adverse reactions. Eptinezumab was approved by the U.S. Food and Drug Administration (FDA) for the preventive treatment of migraine in adults in February 2020, and in January 2022, eptinezumab was granted marketing authorization by the European Medicines Agency (EMA) for the prophylaxis of migraine in adults who have at least four migraine days per month. Today, eptinezumab is launched in the U.S. market, as well as in more than 30 markets worldwide. About H. Lundbeck A/S Lundbeck is a biopharmaceutical company focusing exclusively on brain health. With more than 70 years of experience in neuroscience, we are committed to improving the lives of people with neurological and psychiatric diseases. Brain disorders affect a large part of the world's population, and the effects are felt throughout society. With the rapidly improving understanding of the biology of the brain, we hold ourselves accountable for advancing brain health by curiously exploring new opportunities for treatments. As a focused innovator, we strive for our research and development programs to tackle some of the most complex neurological challenges. We develop transformative medicines targeting people for whom there are few or no treatments available, expanding into neuro-specialty and neuro-rare from our strong legacy within psychiatry and neurology. We are committed to fighting stigma and we act to improve health equity. We strive to create long term value for our shareholders by making a positive contribution to patients, their families and society as a whole. Lundbeck has approximately 5,500 employees in more than 50 countries and our products are available in more than 80 countries. For additional information, we encourage you to visit our corporate site and connect with us via LinkedIn. References: 1. American Academy of Neurology Annual Meeting 2025. Safety, Tolerability, and Efficacy of Bexicaserin in a Cohort of Participants with Developmental and Epileptic Encephalopathies: Interim Results of a Phase 1b/2a PACIFIC Study Open-Label Extension. 2. Sheffer I, et al. Epilepsia. 2025 Feb 27.doi: 10.1111/epi.18265. 3. Kaye R, et al. Efficacy and safety of bexicaserin (LP352) in adolescent and adult participants with developmental and epileptic encephalopathies: Results of the phase 1b/2a PACIFIC study. Presented at the Annual Meeting of the American Academy of Neurology; April 13-18, 2024 4. Ailani J, et al. Poster Presentation. American Academy of Neurology Annual Meeting 2025. 5. Argoff C, et al. Poster presentation. American Academy of Neurology Annual Meeting 2025. 6. Starling A, et al. Poster Presentation. American Academy of Neurology Annual Meeting 2025. CONTACT: H. Lundbeck A/S Ottiliavej 9, 2500 Valby, Denmark +45 3630 1311 [email protected] This information was brought to you by Cision The following files are available for download: SOURCE H. Lundbeck A/S WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED