Jazz Pharmaceuticals to Highlight Neuro-Oncology Clinical Research at SNO 2026

JZP3507 has entered Phase 2 testing in recurrent meningioma, while Jazz expects an ACTION interim analysis in the first half of 2027.

Rhea-AI Summary

Jazz Pharmaceuticals (Nasdaq: JAZZ) will present neuro-oncology research at the Society for Neuro-Oncology Annual Meeting, including progress on its Phase 3 ACTION trial.

The meeting runs November 12–15, 2026, in Philadelphia. ACTION evaluates Modeyso (dordaviprone) in newly diagnosed H3 K27M-mutant diffuse glioma after frontline radiotherapy, with overall survival as its primary efficacy endpoint. Jazz expects an interim analysis in the first half of 2027. Full approval of Modeyso's accelerated-approval indication depends on post-marketing requirements, including verification of clinical benefit in ACTION.

A Phase 2 trial of JZP3507 has begun in recurrent meningioma. Separate preclinical findings in H3 G34-mutant glioma showed nanomolar potency in cell lines and patient-derived cells, with anti-cancer effects dependent on the mitochondrial protein ClpP.

3 points · 0 major

MixediHow this balance works

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Rhea-AI gives every point it takes from this document a weight. Minor counts 1, Moderate 3 and Major 9, so one Major point outweighs several Minor ones. The bar adds up the weights on each side, and when neither side holds more than 65% of the total the balance reads Mixed.

It reads the document as published, with the same rules for every company, and it does not look at what the market expected or at how the stock traded, so a point can be objectively good on a day the stock falls.

Rhea-AI Sentiment measures something else, the tone of the wording.

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0 major · 1 point

Hollow bars mark forward-looking points. How the balance works

Positive

• Minor pointJZP3507 has initiated Phase 2 testing in adults with recurrent grade 2 or 3 meningioma.
• Minor point. Forward-looking: it has not happened yet and may not happen.Phase 3 ACTION continues advancing; Jazz expects an interim analysis in the first half of 2027.
• Minor pointJZP3507 showed nanomolar potency in preclinical H3 G34-mutant glioma models, with ClpP-dependent anti-cancer effects.

Negative

• Moderate pointModeyso's full approval remains contingent on post-marketing requirements, including verification of clinical benefit in ACTION.

Key Figures

ACTION trial allocation: 1:1:1 Interim analysis timing: First half of 2027

ACTION trial allocation

1:1:1

Randomization to placebo, once-weekly dordaviprone, or twice-weekly dordaviprone

Interim analysis timing

First half of 2027

Phase 3 ACTION trial

Key Terms

overall survival, accelerated approval, qtc interval prolongation, cyp3a4 inhibitors

4 terms

overall survival medical

"The primary efficacy endpoint is overall survival."

Overall survival is the average or median length of time patients remain alive after starting a treatment or entering a clinical study, measured regardless of cause of death. Investors care because it is a clear, hard measure of a therapy’s real-world benefit — like timing how long a new battery actually runs — and strong improvements in overall survival can drive regulatory approval, market adoption and revenue potential.

accelerated approval regulatory

"Modeyso received accelerated approval based on overall response rate"

Accelerated approval is a process that allows new medical treatments to be approved more quickly than usual if they address serious or life-threatening conditions and show promising early results. For investors, it signals that a treatment may reach the market sooner, potentially boosting a company's prospects, but it also involves some uncertainty since full evidence of effectiveness is still being gathered.

qtc interval prolongation medical

"MODEYSO causes concentration-dependent QTc interval prolongation"

QTc interval prolongation is a lengthening of the heart’s electrical “reset” time measured on an electrocardiogram after adjusting for heart rate; think of it like a loading bar that takes longer than normal to return to zero. It matters to investors because pronounced prolongation can signal a risk of dangerous irregular heartbeats, trigger regulatory review, clinical-trial changes, safety warnings or market setbacks for drugs and medical devices, and therefore can affect a company’s valuation and timelines.

cyp3a4 inhibitors medical

"Avoid concomitant use of MODEYSO with strong and moderate CYP3A4 inhibitors."

CYP3A4 inhibitors are substances that slow or block the activity of CYP3A4, a key liver enzyme that acts like a conveyor belt breaking down many medicines. When that conveyor slows, drugs can stay in the body longer and reach higher levels, which can change a treatment’s safety, dose, or how it’s prescribed; investors watch for these interactions because they affect a drug’s regulatory approval, marketability, and potential liability.

AI-generated analysis. How Rhea-AI works. Not financial advice.

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Presentations showcase Phase 3 ACTION trial progress and continued development of novel therapies for rare brain cancers

For U.S. media and investors only

DUBLIN, Nov. 2, 2026 /PRNewswire/ -- Jazz Pharmaceuticals plc (Nasdaq: JAZZ) today announced that clinical and preclinical research from its neuro-oncology portfolio will be presented at the upcoming 2026 Society for Neuro-Oncology (SNO) Annual Meeting, taking place November 12-15 in Philadelphia.

Key highlights include:

• The confirmatory Phase 3 ACTION trial evaluating Modeyso^® (dordaviprone), formerly ONC201, continues to advance. In the trial, patients with newly diagnosed H3 K27M-mutant diffuse glioma who have completed standard frontline radiotherapy are randomized 1:1:1 to receive placebo, once-weekly dordaviprone, or twice-weekly dordaviprone on two consecutive days. The primary efficacy endpoint is overall survival.
• A Phase 2 clinical trial for JZP3507 (formerly ONC206) has been initiated in recurrent meningioma, the most common form of primary central nervous system cancer in adults. JZP3507 is a dual targeted investigational therapy that is an agonist of the mitochondrial protease ClpP and an antagonist of the G protein-coupled receptor DRD2. 
• An oral presentation for JZP3507 will show new preclinical findings characterizing the anti-cancer activity in H3 G34-mutant glioma. JZP3507 demonstrated nanomolar potency in cell lines and patient-derived cells, as well as a ClpP-dependent mechanism underlying its anti-cancer effects.

"The data being presented at SNO demonstrates Jazz's expanding commitment to scientific innovation and our progress to develop new therapies for patients facing rare and highly aggressive brain cancers," said Josh Allen, Ph.D., chief scientific officer, oncology, Jazz Pharmaceuticals. "Building on the landmark approval of Modeyso, we are advancing the Phase 3 ACTION trial, with an interim analysis in the first half of 2027, to potentially bring this therapy to patients earlier in the disease course." 

Presentations at SNO 2026 include:

Presentation Title	Authors	Presentation Details
ACTION trial update: A randomized phase 3 study of dordaviprone (ONC201) in patients with newly diagnosed H3 K27M-mutant diffuse glioma	Arrillaga-Romany I*, Chang JH, Pellerino A, Brazil L, Cappellano A, McBain C, Lombardi G, Cardoso A, Dahlrot RH, Carceller F, Gironés Sarrió R, Pavon-Mengual M, Bhatia A, Allen J, Melemed A, Mayo C, Tarapore R, Wen P, Cloughesy T	Type: Poster presentation Location: Exhibit Hall D & E, Second Floor, Pennsylvania Convention Center Date: Friday, November 13, 7:30-9:30 p.m. ET Number: TIP-11
A phase 2 study to investigate the efficacy and safety of JZP3507 (formerly ONC206) in adults with recurrent grade 2 or 3 meningioma	Ramage S*, Arrillaga-Romany I, Barbaro M, Gatson N, Johnson M, Kurz S, Mendez J, Odia Y, Sharma A, Umemura Y, Wen P, Mayo C, Shonka N	Type: Poster presentation Location: Exhibit Hall D & E, Second Floor, Pennsylvania Convention Center Date: Friday, November 13, 7:30-9:30 p.m. ET Number: TIP-12
Allosteric ClpP agonist ONC206 alters mitochondrial metabolism to elicit anti-cancer efficacy in H3 G34-mutant glioma	Prabhu V*, Lee A, Maranto C, Foster S, Allen J	Type: Oral presentation Date: Saturday, November 14, 2:30-3:30pm ET Location: 120 ABC, First Floor, Pennsylvania Convention Center Number: EXTH-15
EGFR signaling drives mesenchymal cell-state transition and therapeutic vulnerability in H3K27M-mutant Diffuse Midline Glioma	Ji S, Doherty R, Pearson A*, Wink K, Lau B, Murthy B, Mota M, Natarajan S, de la Nava D, Sajjakulnukit P, Adam T, Niculcea M, Vo K, Bavani S, Yadavalli S, Cruz A, Li F, Knowles T, Phillips J, Vo T, Liu I, Kumar P, McThenia S, Mazewski C, Pytel N, Bowers D, Green A, Lyssiotis C, Qin T, Solomon D, Beck A, Nuechterlein N, Agnihotri S, Toll S, Dun M, Venneti S, Mueller S, Nazarian J, Waszak S, Koschmann C	Type: Pediatric Oral Abstracts Session I Date: Saturday, November 14, 3:45-4:45pm ET Location: 120 ABC, First Floor, Pennsylvania Convention Center Number: CSIG-09
Spatial Architecture of H3 K27M-mutant Diffuse Midline Glioma Reveals Prognostic Niches in Patients from the PNOC022 Clinical Trial	Savary C*, Vo T, Jackson E, Persson M, Dubois O, Douglas A, McEwen H, McLachlan T, Kline C, Franson A, Valvi S, van der Lugt J, Nazarian J, Waszak S, Koschmann C, Gupta N, Mueller S, Dun M	Type: CNS Rare Tumor Oral Abstracts Session I Date: Friday, November 13, 2:30-3:30pm ET Location: 119 AB, First Floor, Pennsylvania Convention Center Number: BIOM-72
Leveraging the immunomodulatory effects of dordaviprone to induce adaptive immune surveillance in diffuse midline glioma	Persson M*, Savary C, Jackson E, Duchatel R, Vo T, Dubois O, Loughnan O, Findlay I, Douglas A, Bramberger L, de la Nava D, McEwen H, Beitaki T, Mannan A, Jayaraman R, Thomas B, Rodriguez de la Fuente L, Colino-Sanguino Y, Valdes Mora F, Faridi P, Holst J, Mayall J, Kline C, Franson A, Venneti V, Hondermarck H, Horvat J, Pearson A, Ji S, Nazarian J, Hulleman E, Mueller S, Vitanza N, Koschmann C, Alonso M, Carvalheiro T, van der Lugt J, Dun M	Type: Rapid Orals Session VI A Poster Session Date: Friday, November 13, 11:30am-12:30pm ET Friday, November 13, 7:30-9:30pm ET Location: Exhibit Hall D & E, Second Floor, Pennsylvania Convention Center Number: TMIC-98
Exploiting Tumor Stress and Innate Immunity: ONC201 Enhances NK Cell Therapy for Pediatric Diffuse Midline Gliomas	Cheng D, Zhang P, Basar R, Shanley M, Singh A, Swaminathan J, Shrestha R, Siddaway R, Vadivel AA, Sharma A, Yang Y, Anjier A, Maegawa S, Feng S, Gordon N, Daw N, Gorlick R, Tarapore R, Prabhu V, Allen J, Koul D, Hawkins C, Zaky W, Wintermark M, Rezvani K, Gopalakrishnan V*	Type: Poster Session Date: Friday, November 13, 7:30-9:30pm ET Location: Exhibit Hall D & E, Second Floor, Pennsylvania Convention Center Number: EXTH-26
In vitro and in vivo evaluation of CT-179 in combination with ONC201 and ONC206 in patient-derived models of diffuse midline glioma	Mizoguchi S, Yu J, Kritzer B, Laternser S, Stein G, Kesari S, Packer P, Nazarian J, Yadavilli S*	Type: Poster Session Date: Friday, November 13, 7:30-9:30pm ET Location: Exhibit Hall D & E, Second Floor, Philadelphia Convention Center Number: EXTH-33
* Presenting author

About Modeyso^® (dordaviprone)
Modeyso (dordaviprone) is approved by FDA for the treatment of adult and pediatric patients 1 year of age and older with diffuse midline glioma harboring an H3 K27M mutation with progressive disease following prior therapy.¹ Modeyso is an orally administered small molecule given once weekly. Modeyso is a protease activator of the mitochondrial ClpP and also inhibits DRD2. In vitro, dordaviprone activates the integrated stress response, induces apoptosis, and alters mitochondrial metabolism, leading to restored histone H3 K27 trimethylation in H3 K27M-mutant diffuse glioma.¹ 

Modeyso received accelerated approval based on overall response rate and duration of response derived from a pre-specified integrated efficacy analysis of 50 adult and pediatric patients with recurrent H3 K27M-mutant diffuse midline glioma enrolled across five open-label clinical studies (ONC006, ONC013, ONC014, ONC016, and ONC018). Full approval for this indication is contingent upon us satisfying post-marketing requirements, including the verification and description of clinical benefit in the Phase 3 ACTION confirmatory trial and other applicable post-marketing commitments. The FDA granted dordaviprone Rare Pediatric Disease designation and Fast-Track designation, and dordaviprone received Orphan Drug Designation in the United States, Europe and Australia. Modeyso was developed by Chimerix prior to its acquisition by Jazz Pharmaceuticals in April 2025.

Modeyso (dordaviprone) is approved in the United States and Japan. It is not approved anywhere else in the world.

IMPORTANT SAFETY INFORMATION

WARNINGS AND PRECAUTIONS 
Hypersensitivity 
MODEYSO can cause severe hypersensitivity reactions.

In the pooled safety population, Grade 3 hypersensitivity reactions occurred in 0.3% of patients receiving MODEYSO. Signs and symptoms of hypersensitivity may include rash, hives, fever, low blood pressure, wheezing, or swelling of the face or throat.

Inform patients about the signs and symptoms of hypersensitivity reactions and instruct them to seek immediate medical attention if symptoms occur.

If clinically significant hypersensitivity or anaphylaxis occur, immediately interrupt MODEYSO and initiate appropriate medical treatment and supportive care. Based on the severity of the adverse reaction, temporarily interrupt or permanently discontinue MODEYSO.

QTc Interval Prolongation 
MODEYSO causes concentration-dependent QTc interval prolongation, which can increase the risk for ventricular tachyarrhythmias (e.g. torsades de pointes) or sudden death.

In patients who received MODEYSO and underwent at least one post baseline ECG, QTcF increase of >60 msec compared to baseline and QTcF >500 msec occurred in 6% and 1.2% of patients, respectively.

Monitor ECGs and electrolytes prior to initiation and periodically during treatment, as clinically indicated. Increase the frequency of monitoring in patients with congenital long QT syndrome, existing QTc prolongation, a history of ventricular arrhythmias, electrolyte abnormalities, heart failure, or who are taking strong or moderate CYP3A4 inhibitors.

Avoid concomitant use with other agents known to prolong the QT interval. If concomitant use cannot be avoided, increase the frequency of monitoring and separate administration of MODEYSO and QT-prolonging product.

Interrupt or reduce the dose of MODEYSO in patients who develop QT prolongation; permanently discontinue in patients with signs of life-threatening arrhythmias.

Embryo-Fetal Toxicity 
MODEYSO can cause fetal harm when administered to a pregnant woman.

Advise pregnant women and females of reproductive potential of the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during treatment with MODEYSO and for 1 month after the last dose. Advise male patients with female partners of reproductive potential to use effective contraception during treatment with MODEYSO and for 1 month after the last dose.

ADVERSE REACTIONS 
Serious adverse reactions occurred in 33% of the 376 patients who received MODEYSO. Serious adverse reactions in >2% of patients included hydrocephalus (5%), vomiting (4.3%), headache (3.2%), seizure (2.4%), and muscular weakness (2.1%). Fatal adverse reactions occurred in 1% of patients who received MODEYSO, including cardiac arrest (0.5%), intracranial hemorrhage (0.3%), and encephalopathy (0.3%).

The most common adverse reactions (≥20%) reported in clinical trials with MODEYSO were fatigue (34%), headache (32%), vomiting (24%), nausea (24%), and musculoskeletal pain (20%). The most common (≥2%) Grade 3 or 4 laboratory abnormalities were decreased lymphocytes (7%), decreased calcium (2.7%), and increased alanine aminotransferase (2.4%).

DRUG INTERACTIONS 
Strong and Moderate CYP3A4 Inhibitors 
Avoid concomitant use of MODEYSO with strong and moderate CYP3A4 inhibitors. If concomitant use cannot be avoided, reduce the MODEYSO dose as recommended and monitor for toxicity.

Strong and Moderate CYP3A4 Inducers 
Avoid concomitant use of strong and moderate CYP3A4 inducers with MODEYSO.

USE IN SPECIFIC POPULATIONS 
Lactation 
There are no data on the presence of MODEYSO in human milk because of the potential for serious adverse reactions from MODEYSO in breastfed children, advise women not to breastfeed during treatment with MODEYSO and for 1 week after the last dose.

Pediatric Use
The safety and effectiveness of MODEYSO have not been established in patients less than 1 year of age. Dosing has not been established for patients weighing less than 22 pounds (10 kg).

Please refer to the full Prescribing Information, including both Patient Information and Instructions for Use, for complete safety and administration information.

The full U.S. Prescribing Information for MODEYSO is available at: https://pp.jazzpharma.com/pi/modeyso.en.USPI.pdf

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 life-changing medicines for people with rare disease – often with limited or no therapeutic options. We have a diverse portfolio of medicines, including leading therapies addressing epilepsies, cancers and sleep disorders. Our patient-focused and science-driven approach powers pioneering research and development advancements across our robust pipeline of innovative therapeutics. 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: 
CorporateAffairsMediaInfo@jazzpharma.com
Ireland +353 1 637 2141
U.S. +1 215 867 4948

Investor: 
InvestorInfo@jazzpharma.com
Ireland +353 1 634 7800
U.S. +1 650 496 2717

¹ MODEYSO (dordaviprone) Prescribing Information. Palo Alto, CA: Jazz Pharmaceuticals, Inc.

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SOURCE Jazz Pharmaceuticals plc

FAQ

AI-generated questions and answers. How Rhea-AI works. Not financial advice.

When does Jazz Pharmaceuticals expect the Phase 3 ACTION interim analysis?

Jazz expects the Phase 3 ACTION interim analysis in the first half of 2027. The confirmatory trial evaluates dordaviprone in newly diagnosed H3 K27M-mutant diffuse glioma after standard frontline radiotherapy, with overall survival as its primary efficacy endpoint.

What new JZP3507 clinical trial has Jazz Pharmaceuticals started?

A Phase 2 trial of JZP3507, formerly ONC206, has begun in adults with recurrent grade 2 or 3 meningioma. The study investigates the treatment's efficacy and safety.

How are patients assigned to treatment in Jazz Pharmaceuticals' ACTION trial?

ACTION randomizes patients 1:1:1 to placebo, once-weekly dordaviprone, or twice-weekly dordaviprone given on two consecutive days. Participants have newly diagnosed H3 K27M-mutant diffuse glioma and have completed standard frontline radiotherapy.