Jazz Pharmaceuticals to Present Extensive New Data and Real-World Evidence Highlighting Epidiolex® (cannabidiol) Outcomes in Treatment-Resistant Epilepsies at the American Epilepsy Society 2025 Annual Meeting
Rhea-AI Summary
Jazz Pharmaceuticals (Nasdaq: JAZZ) will present eight abstracts, including four late-breaking posters, on Epidiolex (cannabidiol) at the American Epilepsy Society 2025 meeting (Dec 5–9, 2025) in Atlanta.
Key findings include a 6-month intermediate EpiCom analysis showing reductions in TAND-SQ and ABC subscale scores and caregiver/clinician‑reported behavioral severity after 26 weeks of adjunctive Epidiolex in tuberous sclerosis complex; a Phase 1 PK study finding no pharmacokinetic interaction between Epidiolex and cenobamate; preclinical synergistic PD effects with cenobamate; and multiple real-world analyses reporting reduced polypharmacy, antiseizure‑medication cycling, healthcare resource utilization, and seizure frequency versus external control.
Positive
- EpiCom 6‑month analysis: reductions in TAND‑SQ and ABC subscales
- Phase 1 PK study: no pharmacokinetic interaction between Epidiolex and cenobamate
- Real‑world data: reduced polypharmacy and healthcare resource utilization at 12 months
- Expanded Access post‑hoc: reduced convulsive and total seizure frequency versus external placebo control
Negative
- None.
Key Figures
Market Reality Check
Peers on Argus
Peers in Biotechnology show mixed, modest moves, with names like CORT up 0.95% and TECH down 0.64%, suggesting today’s action in JAZZ is more company-specific than sector-driven.
Historical Context
| Date | Event | Sentiment | Move | Catalyst |
|---|---|---|---|---|
| Dec 02 | Oncology trial data | Positive | +0.2% | Announced highly significant Phase 3 Ziihera results to be presented at ASCO GI. |
| Nov 18 | Conference appearance | Neutral | -2.5% | Planned participation in Citi’s 2025 Global Healthcare Conference and webcast. |
| Nov 17 | Pivotal trial win | Positive | +20.6% | Positive top-line Phase 3 HERIZON-GEA-01 results supporting Ziihera combinations. |
| Nov 11 | Clinical data update | Positive | +2.9% | Showcased Modeyso and JZP3507 clinical and translational data at SNO 2025. |
| Nov 05 | Earnings and guidance | Positive | -0.4% | Reported 3Q25 revenue growth and updated 2025 guidance with Modeyso launch contribution. |
Major positive Phase 3 oncology data drew a strong upside reaction, while conferences and earnings updates have shown mixed or even negative next-day moves.
Over the last month, Jazz reported 3Q25 results with revenue of $1.126B, driven by Xywav, Epidiolex and the Modeyso launch, but the stock dipped slightly after earnings. Subsequent clinical and data-focused releases, including positive Phase 3 HERIZON-GEA-01 results for Ziihera and Modeyso data at SNO 2025, were followed by reactions ranging from modest gains to a strong +20.57% move. Today’s AES 2025 Epidiolex data update fits the pattern of Jazz regularly highlighting new clinical and real‑world evidence across its portfolio.
Market Pulse Summary
This announcement highlights broad clinical and real‑world evidence for Epidiolex across rare epilepsies, including TSC, LGS and DEEs, with 8 abstracts and a 6‑month EpiCom analysis of behavioral outcomes. In context of earlier positive Ziihera Phase 3 results and growing revenues from Epidiolex, the update underscores Jazz’s epilepsy focus. Investors may watch for longer‑term outcomes, additional real‑world datasets, and how these data support label use and clinical adoption.
Key Terms
tuberous sclerosis complex medical
phase 3b/4 medical
pharmacokinetic medical
pharmacodynamic medical
expanded access program regulatory
developmental and epileptic encephalopathies medical
AI-generated analysis. Not financial advice.
New interim results from the EpiCom trial, a prospective evaluation of behavioral outcomes in patients with tuberous sclerosis complex, suggest improvements in non-seizure outcomes
Eight abstracts, including four late-breaking abstracts, underscore Jazz's continued commitment to the epilepsy community and advancing the comprehensive treatment for rare forms of epilepsy
For
Notable data include new results from the EpiCom trial, an ongoing Phase 3b/4 study evaluating behavioral and other non-seizure outcomes after initiation of adjunctive Epidiolex treatment in participants with tuberous sclerosis complex (TSC)-associated seizures. Notably, the prespecified 6-month intermediate analysis of EpiCom demonstrated promising reductions in the TSC–Associated Neuropsychiatric Disorders Self-Report Quantified Checklist (TAND-SQ) and Aberrant Behavior Checklist (ABC) subscale scores, as well as in the severity of behavioral problems reported by caregivers and clinicians after 26 weeks of Epidiolex treatment.
"We are proud to share extensive research at AES 2025, including findings from the innovative EpiCom trial which utilized novel endpoints focused on individualized outcomes, including each participant's respective most problematic behavior as identified by caregivers and clinicians to ensure our research addresses the priorities of those we serve," said Jessa Alexander, Ph.D., neuroscience therapeutic area head, global medical and scientific affairs of Jazz Pharmaceuticals. "Data from EpiCom and real-world insights continue to expand our understanding of the complex realities faced by patients living with rare epilepsy disorders while further underscoring Epidiolex's ability to contribute to meaningful, holistic management."
Additional highlights at AES 2025 include:
- A late-breaking poster featuring results from a Phase 1 clinical study evaluating bidirectional pharmacokinetic (PK) drug-drug interactions between Epidiolex and cenobamate, which did not find PK interactions between cannabidiol and cenobamate parent molecules when concomitantly administered at clinically relevant dosages.
- A late-breaking poster showcasing preclinical data that demonstrates a novel synergistic pharmacodynamic interaction between Epidiolex and cenobamate in effectiveness in the mouse maximal electroshock seizure model of acute generalized seizures.
- Two presentations highlight real-world effectiveness of Epidiolex using data from the
U.S. Optum® Market Clarity Database (an integrated electronic health record and claims database). One late-breaking poster features data that shows Epidiolex initiation in CBD-naïve patients with Dravet Syndrome (DS), LGS or TSC is associated with reduced polypharmacy and healthcare resource utilization (HCRU) at 12 months, with further reductions among early-line Epidiolex initiators. The other poster highlights data that indicates Epidiolex initiation also reduces antiseizure medication cycling at 12 months, as well as decreased polypharmacy and HCRU in both pediatric and adult patients. - A poster presentation featuring post-hoc analysis from the Expanded Access Program demonstrates Epidiolex treatment is associated with reductions in convulsive and total seizure frequency in patients with developmental and epileptic encephalopathies (DEEs) and rare epilepsy syndromes. The comparative effectiveness analysis confirms the superiority of Epidiolex in reducing seizure frequency from baseline compared to an external placebo control arm.
- A poster presentation highlighting a subgroup analysis of Epidiolex pivotal clinical trial data showing participants with Lennox-Gastaut syndrome (LGS) diagnoses, which are secondary to a genetic DEE, experienced consistent efficacy and safety as compared to the overall LGS pivotal trial populations.
- A late-breaking poster presentation on data from the Epilepsy Learning Healthcare System (ELHS), an Epilepsy Foundation-sponsored initiative, highlights its potential to provide real-world treatment insights beyond clinical trials and claims data.
All AES 2025 abstracts are available online at the following link: aesnet.org/education/annual-meeting/aes-abstract-search.
A full list of Jazz Pharmaceuticals' presentations follows below:
Presentation Title | Presenting Author | Poster Details | Date & Time (ET) |
Cannabidiol Efficacy in Patients With Lennox-Gastaut Syndrome With Developmental | E Thiele | Poster Number: 1.382 Session Date/Time: Saturday |
Phase 1, Open-Label, Fixed-Sequence, Bidirectional, Pharmacokinetic Drug-Drug | A Vijan | Poster Number: 1.545 Session Date/Time: Saturday |
An Assessment of Acute Pharmacodynamic Drug-Drug Interactions Between | W Hind | Poster Number: 1.546 Session Date/Time: Saturday |
A Population Health Study of Patients Prescribed Cannabidiol in the Epilepsy | KM Farrell | Poster Number: 1.549 Session Date/Time: Saturday |
Effectiveness of Cannabidiol in Patients With Rare Epilepsies Compared to External | Y Park | Poster Number: 2.337 Session Date/Time: Sunday |
Real-World Polypharmacy and Healthcare Resource Utilization After Early-Line | M Navetta | Poster Number: 2.433 Session Date/Time: Sunday |
Tuberous Sclerosis Complex (TSC)–Associated Neuropsychiatric Disorders (TAND) | J Stevens | Poster Number: 3.351 Session Date/Time: Monday |
Real-World Effectiveness of Cannabidiol on Antiseizure Medication Cycling, | A Sillah | Poster Number: 3.368 Session Date/Time: Monday |
About Tuberous Sclerosis Complex
Tuberous sclerosis complex (TSC) is a rare genetic condition.1 The condition causes mostly benign tumors to grow in vital organs of the body including the brain, skin, heart, eyes, kidneys and lungs2 and is a leading cause of genetic epilepsy.3 People with TSC may experience a variety of seizure types. One of the most common is infantile spasms that typically present in the first year of life; focal (or partial) seizures are also very common.4 TSC is associated with an increased risk of autism and intellectual disability5 and the severity of the condition can vary widely. In some children the disease is very mild, while others may experience life-threatening complications.4 Epilepsy is present in about
About Dravet Syndrome
Dravet syndrome (DS) is a rare genetic condition that appears during the first year of life with frequent fever-related seizures (febrile seizures). Later, other types of seizures typically arise, including myoclonic seizures (involuntary muscle spasms).11 Additionally, status epilepticus, a potentially life-threatening state of continuous seizure activity requiring emergency medical care, may occur. Children with DS typically experience poor development of language and motor skills, hyperactivity and difficulty relating to others.
About Lennox-Gastaut Syndrome
Lennox-Gastaut syndrome (LGS) begins in childhood. It is characterized by multiple types of seizures. People with LGS begin having frequent seizures in early childhood, usually between ages 3 and 5.12 More than three-quarters of affected individuals have tonic seizures, which cause the muscles to contract uncontrollably. Almost all children with LGS develop learning problems and intellectual disability. Many also have delayed development of motor skills such as sitting and crawling. Most people with LGS require help with usual activities of daily living.
About Epidiolex®/Epidyolex® (cannabidiol)
Epidiolex/Epidyolex is a prescription, plant-derived cannabis-based medicine administered as an oral solution which contains highly purified cannabidiol (CBD). Cannabidiol, the active ingredient in Epidiolex, is a cannabinoid that naturally occurs in the Cannabis sativa L. plant. The precise mechanisms by which Epidiolex exerts its anticonvulsant effect in humans are unknown. Epidiolex was approved by the
Important Safety Information & Indications
CONTRAINDICATION: HYPERSENSITIVITY
EPIDIOLEX (cannabidiol) oral solution is contraindicated in patients with a history of hypersensitivity to cannabidiol or any ingredients in the product.
WARNINGS & PRECAUTIONS
Hepatic Injury:
EPIDIOLEX can cause dose-related transaminase elevations. Concomitant use of valproate and elevated transaminase levels at baseline increase this risk. Obtain transaminase and bilirubin levels prior to starting treatment, at 1, 3, and 6 months after initiation of treatment, and periodically thereafter, or as clinically indicated. Resolution of transaminase elevations occurred with discontinuation of EPIDIOLEX, reduction of EPIDIOLEX and/or concomitant valproate, or without dose reduction. For patients with elevated transaminase levels, consider dose reduction or discontinuation of EPIDIOLEX or concomitant medications known to affect the liver (e.g., valproate or clobazam). Dose adjustment and slower dose titration is recommended in patients with moderate or severe hepatic impairment. Consider not initiating EPIDIOLEX in patients with evidence of significant liver injury. There have been postmarketing reports of cholestatic or mixed patterns of liver injury. Elevated ammonia levels were reported in some patients with transaminase elevations; most taking concomitant valproate, clobazam, or both. Consider discontinuation or dose adjustment of valproate or clobazam if ammonia is elevated.
Somnolence and Sedation:
EPIDIOLEX can cause somnolence and sedation that generally occurs early in treatment and may diminish over time; these effects occur more commonly in patients using clobazam and may be potentiated by other CNS depressants.
Suicidal Behavior and Ideation:
Antiepileptic drugs (AEDs), including EPIDIOLEX, increase the risk of suicidal thoughts or behavior. Inform patients, caregivers, and families of the risk and advise them to monitor and report any signs of depression, suicidal thoughts or behavior, or unusual changes in mood or behavior. If these symptoms occur, consider if they are related to the AED or the underlying illness.
Withdrawal of Antiepileptic Drugs:
As with most AEDs, EPIDIOLEX should generally be withdrawn gradually because of the risk of increased seizure frequency and status epilepticus.
ADVERSE REACTIONS:
The most common adverse reactions in patients receiving EPIDIOLEX (≥
PREGNANCY:
EPIDIOLEX should be used during pregnancy only if the potential benefit justifies the potential risk to the fetus. Encourage women who are taking EPIDIOLEX during pregnancy to enroll in the EPIDIOLEX Pregnancy Surveillance Program and the North American Antiepileptic Drug (NAAED) Pregnancy Registry.
DRUG INTERACTIONS:
Strong inducers of CYP3A4 and CYP2C19 may affect EPIDIOLEX exposure. EPIDIOLEX may affect exposure to CYP2C19 substrates (e.g., clobazam, diazepam, stiripentol), orally administered P-gp substrates, or other substrates (see full Prescribing Information). Consider dose reduction of orally administered everolimus, with appropriate therapeutic drug monitoring, when everolimus is combined with EPIDIOLEX. A lower starting dose of everolimus is recommended when added to EPIDIOLEX therapy. Concomitant use of EPIDIOLEX and valproate increases the incidence of liver enzyme elevations. Pneumonia was observed more frequently with concomitant use of EPIDIOLEX and clobazam. Dosage adjustment of EPIDIOLEX or other concomitant medications may be necessary.
INDICATIONS:
EPIDIOLEX (cannabidiol) oral solution is indicated for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS), Dravet syndrome (DS), or tuberous sclerosis complex (TSC) in patients 1 year of age and older.
Please read the EPIDIOLEX full Prescribing Information for additional important information here.
About Jazz Pharmaceuticals
Jazz Pharmaceuticals plc (Nasdaq: JAZZ) is a global biopharma company whose purpose is to innovate to transform the lives of patients and their families. We are dedicated to developing potentially life-changing medicines for people with serious diseases — often with limited or no therapeutic options. We have a diverse portfolio of marketed medicines, including leading therapies for sleep disorders and epilepsy, and a growing portfolio of cancer treatments. Our patient-focused and science-driven approach powers pioneering research and development advancements across our robust pipeline of innovative therapeutics in oncology and neuroscience. Jazz is headquartered in Dublin, Ireland with research and development laboratories, manufacturing facilities and employees in multiple countries committed to serving patients worldwide. Please visit www.jazzpharmaceuticals.com for more information.
Contacts:
Media:
Kristin Bhavnani
Head of Global Corporate Communications
Jazz Pharmaceuticals plc
CorporateAffairsMediaInfo@jazzpharma.com
Investors:
Jack Spinks
Executive Director, Investor Relations
Jazz Pharmaceuticals plc
investorinfo@jazzpharma.com
Ireland +353 1 634 3211
U.S. +1 650 496 2717
References: |
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1 TSC Alliance. What is TSC? https://www.tscalliance.org/understanding-tsc/what-is-tsc/. Accessed December 2025. |
2 National Institute of Neurological Disorders and Stroke. Tuberous Sclerosis Complex. https://www.ninds.nih.gov/health-information/disorders/tuberous-sclerosis-complex. Accessed December 2025. |
3 TSC Alliance. An introduction to tuberous sclerosis complex. https://www.tscalliance.org/wp-content/uploads/2023/12/An-introduction-to-TSC-2021.pdf. Accessed December 2025. |
4 Kingswood JC, d'Augères GB, Belousova E, et al. TuberOus SClerosis registry to increase disease Awareness (TOSCA) - baseline data on 2093 patients. 2017;12(1):2. |
5 de Vries PJ, Belousova E, Benedik MP, et al. TSC-associated neuropsychiatric disorders (TAND): findings from the TOSCA natural history study. Orphanet J Rare Dis. 2018;13(1):157. |
6 Tuberous Sclerosis Alliance. Diagnostic criteria. https://www.tscalliance.org/understanding-tsc/diagnosis-criteria/. Accessed December 2025. |
7 Jeong A, Wong M. Systemic disease manifestations associated with epilepsy in tuberous sclerosis complex. Epilepsia. 2016;57(9):1443-1449. |
8 Chu-Shore CJ, Major P, Camposano S, Muzykewicz D, Thiele EA. The natural history of epilepsy in tuberous sclerosis complex. Epilepsia. 2010;51(7):1236-1241. |
9 Kwan P, Brodie MJ. Early identification of refractory epilepsy. N Engl J Med. 2000;342(5):314-319. |
10 French JA. Refractory epilepsy: clinical overview. Epilepsia. 2007;48 Suppl 1:3-7. |
11 Epilepsy Foundation. Dravet Syndrome. https://www.epilepsy.com/what-is-epilepsy/syndromes/dravet-syndrome. Accessed December 2025. |
12 National Organization for Rare Disorders, Inc. Lennox-Gastaut Syndrome: Signs & Symptoms. https://rarediseases.org/rare-diseases/lennox-gastaut-syndrome/. Accessed December 2025. |
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