GPR55

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

Funding of $1.49 million USD to support the development of KLS-13019 targeting neuroinflammation and mitochondrial dysfunction Kannalife has developed a new class of orally bioavailable drugs to address neurodegenerative and inflammatory diseases by targeting mNCX-1, GPR55 and the NLRP3 inflammasome June 25, 2024 -- Kannalife Sciences, Inc., a pioneering biotech company dedicated to developing small molecule therapeutics targeting inflammation, is pleased to announce it has been awarded funding of $1,493,409 million from The Michael J. Fox Foundation for Parkinson’s Research (MJFF). This grant will support preclinical research of Kannalife’s lead therapeutic KLS-13019 by investigating its effects on neuroinflammation and mitochondrial dysfunction, which are implicated in Parkinson’s disease (PD). Parkinson’s disease is the fastest growing neurodegenerative movement disorder affecting up to 2% of those aged 60 years or older. Douglas Brenneman, PhD, Chief Pharmacologist at Kannalife Sciences and Principal Investigator of the study, expressed gratitude for the Foundation's support, stating, "We are honored to receive this grant support from The Michael J. Fox Foundation, a leading organization dedicated to accelerating the development of improved therapies for Parkinson's disease. We are confident that we can make meaningful progress toward this goal and, ultimately, improve the lives of patients living with Parkinson's." Kannalife Sciences, located at the Pennsylvania Biotechnology Center (PABC) in Doylestown, PA, is leveraging its globally patented technology into neuroinflammatory disorders targeting the novel G protein-coupled receptor (GPCR), GPR55 and the NLRP3 inflammasome. Advanced preclinical research and IND enabling studies to date have validated KLS-13019’s effects on biological targets in the mitochondria including mNCX-1, with the aim of addressing unmet needs in inflammatory diseases. KLS-13019’s promising preclinical results in models of neuroinflammation and oxidative stress, portend to be key factors of neurodegeneration in Parkinson's disease. Jessica Tome Garcia, PhD, associate director of research programs at MJFF, stated, "We look forward to seeing the results of Kannalife Sciences’ innovative work on KLS-13019. This funding highlights the Foundation’s commitment to advancing Parkinson’s therapeutic research by addressing neuroinflammation and mitochondrial dysfunction, two key factors in the disease." Dr. Brenneman further stated, “This grant represents a significant milestone in our research efforts. Special thanks to the whole team at Kannalife, especially Dean Petkanas for his persistence and dedication in mobilizing KLS-13019 into a potential therapeutic for the treatment of Parkinson’s Disease. I also want to thank our research partners Prof. Dr. Med. Chi Wang Ip of Universitätsklinikum Würzburg, Tom H. Johnston, PhD of Atuka, Inc. and James P. Koprich, MA, PhD, for taking the time and effort to put an outstanding study protocol together for our research program in PD.” Through research efforts with MJFF and other partners, the company aims to accelerate the development of KLS-13019 and pave the way for novel therapeutic interventions in Parkinson’s disease and beyond. KLS-13019 is currently undergoing small pre-clinical model efficacy, chemical scale-up, and toxicology studies supported by an STTR Phase 2 study grant from the National Institute of Neurological Disorders and Stroke (NINDS) of the National Institutes of Health (NIH) under the HEAL Initiative. Successful completion of these IND enabling studies will allow initiation of human clinical studies for the treatment of chemotherapy-induced peripheral neuropathy (CIPN). This financial support underscores the broad scientific validation of this promising therapeutic agent. The PABC is a nonprofit life sciences incubator-accelerator providing state-of-the-art laboratory and office space to early-stage biotech companies, the Hepatitis B Foundation, and the Blumberg Institute. Managed by the Institute and overseen by a Foundation-appointed board, PABC supports nearly 80 member companies fostering success through biotech advancements and collaborations. Located near Doylestown in the Philadelphia-New Jersey pharma belt, the PABC website offers further information, visit www.pabiotechbc.org. Kannalife Sciences, Inc. is a pioneering biotech company specializing in the development of small molecule synthetic cannabinoid therapeutics targeting inflammation. Headquartered at the Pennsylvania Biotechnology Center of Bucks County in Doylestown, PA, Kannalife Sciences is a wholly owned subsidiary of Neuropathix, Inc. For more information about Kannalife, visit www.kannalife.com and the Company’s Twitter page at @kannalife. The content above comes from the network. if any infringement, please contact us to modify.

Poster presentation details screening approach allowing selection of candidate molecules applicable to dry eye disease and chronic ocular pain, with potential to identify therapeutic candidates with novel mechanisms of action for other ocular pathologiesSAN DIEGO, May 10, 2024 (GLOBE NEWSWIRE) -- Skye Bioscience, Inc. (Nasdaq: SKYE) (“Skye”), a clinical-stage biotechnology company focused on the discovery, development and commercialization of novel classes of therapeutic drugs that modulate the endocannabinoid system (ECS), today announced that it presented new data in a poster titled “Development and screening of a novel library of synthetic endocannabinoid agonists and inhibitors to advance a potential therapy to treat dry eye disease and chronic ocular pain” on May 9th at the ARVO (Association of Research in Vision and Ophthalmology) 2024 Annual Meeting. “The endocannabinoid system offers unique opportunities to address unmet medical needs with new mechanisms of action. At ARVO we showcased Skye’s development and screening of a novel library of synthetic agonists and inhibitors that could potentially fulfill some of these opportunities,” said Punit Dhillon, Chief Executive Officer and Chairman of Skye. Key highlights Presented biological ECS screening program focused on pathways relevant to dry eye disease (DED) and chronic ocular painThree synthetic cannabinoid-based compounds relevant to DED and chronic ocular pain have been identified for further pharmaceutical developmentConclusion: a unique screening approach has the potential to provide a new class of therapeutics with novel mechanisms of action for the treatment of diverse ocular pathologies. Chris Twitty, PhD, Chief Scientific Officer of Skye, added: “Skye’s strategy is to develop advanced pharmaceutical drugs capable of modulating the endocannabinoid system to positively impact ocular pathologies. The study’s evaluation of a novel synthetic cannabinoid-based library with biologically relevant cell types, targets, and pathways enabled the selection of candidate molecules applicable to DED and chronic ocular pain. Ultimately, we selected cannabinerol and two non-electrophilic indolic adducts of cannabidiolquinone and cannabigeroquinone that warrant further interrogation and pharmaceutical development.” Study details A rigorous in vitro screening platform based on modulation of pathways relevant to cannabinoid biology in the context of ocular pathologies was designed to interrogate a library of 98 synthetic cannabinoid-based molecules for their potential to treat ocular diseases. Immortalized human ocular cell lines and stable modified HEK cells were used to measure activation/inhibition of specific cannabinoid receptors, including CB1, CB2, GPR55, and TRPV1. The library was initially interrogated using a threshold of relevant biological activity. The resulting 38 compounds were scored based on EC50/IC50 of signaling (CB1/2/TRPV1/PPARg/NF-kB) and inhibition of ROS and HIF-1a in human epithelial and endothelial (corneal/conjunctival) cells. These compounds were also interrogated for chemical/developability attributes, which helped provide the rationale for ultimately selecting three lead candidate molecules. About Skye Bioscience Skye is focused on unlocking the pharmaceutical potential of the endocannabinoid system to treat diseases with metabolic, inflammatory, and fibrotic processes. Backed by leading life science venture investors, Skye's strategy leverages biologic targets with substantial human proof of mechanism for the development of first-in-class therapeutics with significant clinical and commercial differentiation. Nimacimab, a negative allosteric modulating antibody that inhibits peripheral CB1, showed a favorable safety and tolerability profile in a Phase 1 study. Skye plans to start a Phase 2 clinical trial in obesity comparing monotherapy and combination arms of Nimacimab and a GLP-1R agonist in mid-2024. Enrollment has been completed for a Phase 2 clinical trial of SBI-100 Ophthalmic Emulsion, a CB1 agonist currently being studied in patients with glaucoma and ocular hypertension. Topline data for this study is expected in Q2 2024. For more information, please visit: https://www.skyebioscience.com. CONTACTInvestor Relationsir@skyebioscience.com(858) 410-0266 LifeSci Advisors, Mike Moyermmoyer@lifesciadvisors.com617-308-4306 Media InquiriesLifeSci Communications, Michael Fitzhughmfitzhugh@lifescicomms.com(628) 234-3889 FORWARD LOOKING STATEMENTSThis letter contains forward-looking statements, including statements regarding our product development, business strategy, the timing of clinical trials, and commercialization of cannabinoid-derived therapeutics. Such statements and other statements in this press release that are not descriptions of historical facts are forward-looking statements that are based on management’s current expectations and assumptions and are subject to risks and uncertainties. If such risks or uncertainties materialize or such assumptions prove incorrect, our business, operating results, financial condition, and stock price could be materially negatively affected. In some cases, forward-looking statements can be identified by terminology including “anticipated,” “plans,” “goal,” “focus,” “aims,” “intends,” “believes,” “can,” “could,” “challenge,” “predictable,” “will,” “would,” “may” or the negative of these terms or other comparable terminology. We operate in a rapidly changing environment, and new risks emerge from time to time. As a result, it is not possible for our management to predict all risks, nor can we assess the impact of all factors on our business or the extent to which any factor, or combination of factors, may cause actual results to differ materially from those contained in any forward-looking statements the Company may make. Risks and uncertainties that may cause actual results to differ materially include, among others, our capital resources, uncertainty regarding the results of future testing and development efforts and other risks that are described in the Risk Factors section of Skye’s most recent annual or quarterly report filed with the Securities and Exchange Commission. Except as expressly required by law, Skye disclaims any intent or obligation to update these forward-looking statements.