A Phase 3, Open-Label Study to Investigate the Long-Term Safety and Efficacy of LP352 in the Treatment of Seizures in Children and Adults With Developmental and Epileptic Encephalopathy

This (DEEp OLE Study) is a multicentre, open-label study to investigate the long-term safety, efficacy, tolerability, and pharmacokinetics (PK) of LP352 in the treatment of seizures in children and adults with DEE who completed Study LP352-301 or LP352-302. The study consists of 3 main phases: Screening, Titration period and Maintenance period, followed by a Taper period and Follow-Up. The total duration of the study will be approximately 14 months.

Start Date12 Feb 2025

Sponsor / Collaborator

Longboard Pharmaceuticals, Inc.

NCT06719141

/ RecruitingPhase 3

A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Investigate the Efficacy, Safety, and Tolerability of LP352 in the Treatment of Seizures in Children and Adults With Developmental and Epileptic Encephalopathies

This (DEEp OCEAN Study) is a double-blind, randomized, placebo-controlled, multicenter study to investigate the efficacy, safety, and tolerability of LP352 in the treatment of seizures in children and adults with DEE. The study consists of 3 main phases: Screening, Titration period, Maintenance period, followed by a Taper period and Follow-Up. The total duration of the study will be approximately 24 months.

Start Date11 Nov 2024

Sponsor / Collaborator

Longboard Pharmaceuticals, Inc.

NCT06660394

/ RecruitingPhase 3

This (DEEp SEA Study) is a double-blind, randomized, placebo-controlled, multicenter study to investigate the efficacy, safety, and tolerability of LP352 in the treatment of seizures in children and adults with DS. The study consists of 3 main phases: Screening, Titration period, and Maintenance period, followed by a Taper period and Follow-Up. Participants will be randomized to LP352 or placebo. The total duration of the study will be approximately 24 months.

Start Date25 Sep 2024

Sponsor / Collaborator

Longboard Pharmaceuticals, Inc.

100 Clinical Results associated with Bexicaserin

100 Translational Medicine associated with Bexicaserin

100 Patents (Medical) associated with Bexicaserin

Literatures (Medical) associated with Bexicaserin

12 Jun 2025·JOURNAL OF MEDICINAL CHEMISTRY

Diazepine Agonists of the 5-HT_2C Receptor with Unprecedented Selectivity: Discovery of Bexicaserin (LP352)

Article

Author: Unett, David J. ; Srinivas, Nuggehally R. ; Schrader, Thomas O. ; Grottick, Andrew J. ; Dang, Huong ; Zhu, Xiuwen ; Morgan, Michael E. ; Whelan, Kevin T. ; Le, Minh ; Frazer, John ; Semple, Graeme ; Sage, Carleton R. ; Lehmann, Juerg ; Kasem, Michelle ; Ren, Albert ; McLin, Dewey

The clinical development of novel 5-HT_2C receptor (5-HT_2CR) therapies has been limited due to concerns over lack of selectivity and potential for off-target effects. Here, we report that the introduction of a secondary amide substituent into a 6,5,7-tricyclic benzodiazepine scaffold provided compounds with unprecedented selectivity for the 5-HT_2CR in both functional and binding assays. An early lead compound, 7b, had an in vivo half-life that was shorter than desired, which was improved by a targeted reduction in renal clearance. This provided the clinical candidate compound (+)-19m (later named bexicaserin), which displayed excellent oral bioavailability, good central nervous system partitioning, and decreased renal clearance in vivo. (+)-19m was also a potent inhibitor of acute refeeding in the fasted rat, suggesting on-target effects. (+)-19m demonstrated excellent selectivity for 5-HT_2CR over 5-HT_2AR and 5-HT_2BR and maximal activity exceeding that induced by the endogenous ligand 5-HT. (+)-19m has since progressed into clinical development.

01 Apr 2025·BIOMEDICAL CHROMATOGRAPHY

A Sensitive and Selective LC–MS/MS‐ESI Method for the Quantitation of Metabolites M9, M12, and M20 of Bexicaserin in Human Plasma and Urine Matrices

Article

Author: Neal, Katie ; Yeager, Maisy ; Srinivas, Nuggehally R. ; Fletcher, Katharine ; Chan, Rosa ; Kallem, Raja Reddy

ABSTRACT:

Bexicaserin is a highly selective 5HT2c receptor agonist being developed for the treatment of seizures associated with developmental and epileptic encephalopathies (DEEs). We report an LC–MS/MS method for the quantitative estimation of three pharmacologically inactive metabolites (M9, M12, and M20) of bexicaserin in human plasma/urine. Sample preparation involves the extraction of M9, M12, M20, and internal standards (ISs) from 25‐μL plasma and 50‐μL urine following protein precipitation. The chromatographic separation of analytes was achieved on a HSS T3‐C18 column. The calibration curves ranged from 0.1 to 100 ng/mL for M9, 0.5–500 ng/mL for M12, and 1.0–1000 ng/mL for M20 in plasma and 2.0–2000 ng/mL for M9 and M12 and 10–10,000 ng/mL for M20 in urine. Intraday/interday precision and accuracy, linearity, matrix effect, extraction recovery, carry‐over, dilution integrity, stability studies, and incurred sample reanalysis were performed in both plasma and urine. The intraday and interday accuracy and precision for metabolites met the stipulated regulatory guidelines. Stability studies in plasma and urine showed that analytes were stable at bench‐top for > 23.5 h and in autosampler for > 69 h. Analytes were stable after five freeze–thaw cycles and > 552 days of long‐term storage at −20°C and −80°C.

01 Feb 2025·BIOMEDICAL CHROMATOGRAPHY

A Highly Sensitive Triple Quad LC–MS/MS Method Development and Validation for the Determination of Bexicaserin (LP352) in Human Plasma and Urine Matrices

Article

Author: Neal, Katie ; Yeager, Maisy ; Chan, Rosa ; Fletcher, Katharine ; Srinivas, Nuggehally R. ; Kallem, Raja Reddy

ABSTRACT:

Bexicaserin is a highly selective agonist at the 5‐HT2c receptor in clinical development for the treatment of seizures associated with developmental and epileptic encephalopathies (DEEs). We report an LC–MS/MS method for the quantitative estimation of bexicaserin in human plasma and urine. The sample preparation involves the extraction of bexicaserin and internal standard (13CD2‐bexicaserin; IS) from 150 μL plasma and 50 μL urine using a solid phase extraction method. The chromatographic separation of bexicaserin and IS was achieved on a Poroshell EC‐C18 column using 5 min gradient program. The calibration curve ranged from 0.1 to 100 ng/mL in plasma and 1.0 to 1000 ng/mL in urine. Intraday and interday precision and accuracy, linearity, matrix effect, extraction recovery, carry‐over, dilution integrity, a battery of stability studies, and incurred sample reanalysis were performed for bexicaserin in both plasma and urine. The intraday and interday accuracy and precision were well within the acceptable limits in both plasma and urine matrices. Stability studies in plasma and urine showed that bexicaserin was stable on bench for 24 h, in autosampler over 54 h, five freeze–thaw cycles, and long‐term storage at −20°C and −80°C for > 368 days. The validated methods were successfully applied in clinical study.

News (Medical) associated with Bexicaserin

01 May 2025

·www.prnewswire.com

EPIDIOLEX Continues to Lead the Way in Cannabinoid-Based Epilepsy Treatment | DelveInsight

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

Drug ApprovalPhase 3AcquisitionBreakthrough TherapyOrphan Drug

04 Apr 2025

·www.prnewswire.com

Lundbeck to present positive pipeline data at American Academy of Neurology (AAN) Annual Meeting

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

Clinical ResultPhase 3Drug ApprovalBreakthrough Therapy

11 Mar 2025

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Apnimed Appoints Kevin R. Lind as Non-Executive Chairman of the Board of Directors

– Larry Miller, CEO, to Continue Serving as Non-Independent Board Director – Phase 3 SynAIRgy and LunAIRo Clinical Trials on Track for Topline Results in 2Q2025 and 3Q2025, Respectively CAMBRIDGE, Mass., March 11, 2025 /PRNewswire/ -- Apnimed, Inc., a pharmaceutical company focused on discovering, developing, and commercializing first-in-class oral therapies for the neuromuscular dysfunction associated with obstructive sleep apnea (OSA) and other sleep-related breathing diseases, today announced the expansion of its Board of Directors with the appointment of Kevin R. Lind as non-executive Chairman of the Board. Mr. Lind brings decades of experience from the biotech and financial sectors. "We are thrilled to welcome Kevin to Apnimed's Board," said Larry Miller, MD, Chief Executive Officer of Apnimed. "His proven leadership in biotechnology and corporate strategy will provide invaluable insights as we approach Phase 3 clinical trial results for AD109 and prepare for its potential commercial launch as a once-nightly oral therapy for OSA. Our team has been executing to our stated milestones and, most importantly, our ongoing Phase 3 trials are on track to report top-line results in 2Q2025 and 3Q2025, respectively." "Apnimed is well-positioned for growth with both its promising late-stage asset, AD109, and its synergistic partnership with Shionogi to drive long-term value," said Mr. Lind. "I am excited to join Apnimed at this pivotal time and look forward to contributing to the company's continued clinical and future commercial successes." About Kevin Lind Kevin Lind recently served as Longboard Pharmaceuticals' President and Chief Executive Officer and as a member of its board from the company's inception in 2020 until Longboard was acquired by H. Lundbeck A/S in December 2024. Prior to co-founding Longboard Pharmaceuticals, Mr. Lind served as EVP and Chief Financial Officer during the turnaround of Arena Pharmaceuticals (acquired by Pfizer) from 2016 to 2020. As an executive leader at these organizations, he successfully raised over $1.1B in equity capital (including Longboard's IPO), secured valuable business development partnerships (including ~$1B in upfront payments), spun out two organizations, and was instrumental in activities resulting in the successful acquisitions of both companies for a cumulative value of ~$9.3B. At Longboard, Mr. Lind also led the initiation of a global Phase 3 program for a neurological drug candidate, bexicaserin, and innovative drug development strategies resulting in the conceptualization of a novel medical indication and securing of Breakthrough Therapy designation from the U.S. FDA. Prior to Longboard, Mr. Lind focused on healthcare investing at TPG Special Situations Partners from 2009 to 2016 and at TPG-Axon from 2006 to 2008. He served in various capacities as a healthcare investment banker at Lehman Brothers, Inc., a former global financial services firm, from 1998 to 2002 and 2004 to 2006. Mr. Lind received a B.S. from Stanford University in Biological Sciences and an MBA from UCLA Anderson School of Management. About AD109 AD109 is a once-nightly oral pill that could be the first pharmacological treatment to improve oxygenation during sleep by directly targeting the underlying neuromuscular dysfunction that causes the upper airway to collapse in people with obstructive sleep apnea (OSA). It is a first-in-class combination of aroxybutynin, a novel antimuscarinic, and atomoxetine, a selective norepinephrine reuptake inhibitor (NRI). Their combined pharmacological synergy targets the root neuromuscular cause of OSA. In a disease characterized by complex and invasive treatment options, AD109 may offer a simple solution to help improve oxygenation and overall health for people living with OSA. AD109 has successfully completed multiple Phase 1 and Phase 2 trials in OSA. The Phase 3 program is fully enrolled with topline results expected in mid-2025. About Obstructive Sleep Apnea Obstructive sleep apnea (OSA) is a serious, chronic sleep-related breathing disease in which the upper airway repeatedly collapses during sleep, leading to intermittent oxygen deprivation. It is caused by two overlapping mechanisms: neuromuscular dysfunction during sleep and predisposing anatomic abnormalities. It has been demonstrated that OSA affects people of all body types, ages and genders —there is no single "face" of the disease. An estimated 80 million people in the United States and 1 billion people worldwide suffer from OSA. An individual with OSA can experience hundreds of sleep apnea events in a single night, each one reducing the blood oxygen levels and negatively impacting cellular functions vital to normal health and function. Failure to effectively treat OSA increases the risk of serious long-term health consequences, such as high blood pressure, cardiovascular disease and type 2 diabetes, along with impacts on quality of life, including daytime fatigue and impaired judgement. Yet, the majority of those diagnosed with OSA refuse, abandon, or underutilize treatment. Currently, no available pharmacological treatments directly address the underlying neuromuscular dysfunction that is present in all people with OSA. About Apnimed Apnimed is a privately held clinical-stage pharmaceutical company dedicated to transforming the treatment landscape for sleep-related breathing diseases. Apnimed envisions a new era where novel oral therapies simplify intervention, expand the reach of diagnosis and treatment, and help more people get the oxygen and restorative sleep needed to thrive. Based in Cambridge, Mass., Apnimed is advancing a robust pipeline of oral pharmaceutical product candidates designed to improve oxygenation in individuals living with OSA and other sleep-related breathing disorders. This includes AD109, which is currently in Phase 3 clinical trials for the treatment of mild, moderate and severe OSA, as well as several therapies being developed as part of our joint venture with Shionogi & Co., Ltd, through Shionogi Apnimed Sleep Science (SASS). Learn more at apnimed.com or follow us on X and LinkedIn. Media Contact: [email protected] Investor Contact: Ramzi Benamar [email protected] SOURCE Apnimed, Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Phase 2Executive ChangePhase 3IPO

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Indication	Highest Phase	Country/Location	Organization	Date
Seizures	Phase 3	China	Longboard Pharmaceuticals, Inc.	20 Jun 2025
Seizures	Phase 3	Japan	Longboard Pharmaceuticals, Inc.	20 Jun 2025
Seizures	Phase 3	Belgium	Longboard Pharmaceuticals, Inc.	20 Jun 2025
Seizures	Phase 3	Brazil	Longboard Pharmaceuticals, Inc.	20 Jun 2025
Seizures	Phase 3	Canada	Longboard Pharmaceuticals, Inc.	20 Jun 2025
Seizures	Phase 3	Czechia	Longboard Pharmaceuticals, Inc.	20 Jun 2025
Seizures	Phase 3	Denmark	Longboard Pharmaceuticals, Inc.	20 Jun 2025
Seizures	Phase 3	Finland	Longboard Pharmaceuticals, Inc.	20 Jun 2025
Seizures	Phase 3	Mexico	Longboard Pharmaceuticals, Inc.	20 Jun 2025
Seizures	Phase 3	Netherlands	Longboard Pharmaceuticals, Inc.	20 Jun 2025

Clinical Result

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Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


PACIFIC Study (AAN2025)	Phase 1/2	Developmental and epileptic encephalopathy	-	Bexicaserin	luviswpiza(edtlmrsesc) = bypjxfnhkl oyjhiwcdwq (hdzhlmwrfg ) View more	Positive	07 Apr 2025
NCT05364021 (PACIFIC, NEWS) Manual	Not Applicable	Seizures	40	Bexicaserin	mnkxfocfny(rstmhuxzuf) = crgcccrjlq vrhsnnnkow (xuetcadyqb ) View more	Positive	17 Jun 2024
NCT05626634 (NEWS)	Phase 2	Developmental and epileptic encephalopathy	41	Bexicaserin	yptckffbib(jududjtsme) = bmnzzgoyfb lupxvrxlmv (wntitwcdgl )	Positive	10 Jun 2024
				Bexicaserin+placebo	yptckffbib(jududjtsme) = ezijtkggyn lupxvrxlmv (wntitwcdgl )
NCT05364021 (PACIFIC, Corporate Publications) Manual	Phase 1/2	Infantile Epileptic-Dyskinetic Encephalopathy	52	LP352	chtcpbpylu(aynosbstcu) = ziiomqmmbu tmjsqpypou (khlriourbb ) View more	Positive	02 Jan 2024
				Placebo	chtcpbpylu(aynosbstcu) = krkofuxtij tmjsqpypou (khlriourbb )
102 (Corporate Publications) Manual	Phase 1	Epileptic encephalopathy	20	LP-352 (Cohort 1 = 6 mg)	whotejbeax(todjitsles) = Favorable safety and tolerability results were observed in this study, with adverse events (AEs) generally consistent with previous clinical studies njgmuhuhyo (bogiglannr )	Positive	05 Dec 2022
				LP-352 (Cohort 2 = 12 mg)

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