Akt-1 inhibitors(Serine/threonine-protein kinase AKT1 inhibitors), CLPP agonists(caseinolytic mitochondrial matrix peptidase proteolytic subunit agonists), D2 receptor antagonists(Dopamine D2 receptor antagonists)

Therapeutic Areas

NeoplasmsNervous System DiseasesOther Diseases+ [5]

Active Indication

Diffuse midline glioma, point mutation K27M in histone H3Diffuse Intrinsic Pontine GliomaRecurrent Glioma+ [12]

Inactive Indication

Acute lymphoblastic leukemia recurrentAdvanced cancerAdvanced Colorectal Adenocarcinoma+ [32]

Originator Organization

Chimerix, Inc.

Active Organization

Jazz Pharmaceuticals Plc

University of Nebraska

Stephenson Cancer Center

+ [6]

Inactive Organization

Oncoceutics, Inc.Jonsson Comprehensive Cancer Center

University of California, Los Angeles

+ [5]

License Organization

China Resources Sanjiu Medical & Pharmaceutical Co., Ltd.

Chimerix, Inc.

Healthcare Royalty Management LLC

+ [3]

Drug Highest PhaseApproved

First Approval Date

United States (06 Aug 2025),

Diffuse midline glioma, point mutation K27M in histone H3

RegulationPriority Review (United States), Fast Track (United States), Accelerated Approval (United States), Orphan Drug (European Union), Orphan Drug (Japan), Orphan Drug (Australia), Rare Pediatric Disease (United States)

Structure/Sequence

Molecular FormulaC24H26N4O

InChIKeyVLULRUCCHYVXOH-UHFFFAOYSA-N

CAS Registry1616632-77-9

Clinical Trials associated with Dordaviprone

NCT06012929

/ WithdrawnPhase 2IIT

A Pilot Study, Open-Label Study of ONC201 for Refractory Meningioma

The goal of this clinical trial is to learn about treatment for a type of brain tumor called a meningioma. This study will enroll two groups of people. One group will be for people who will receive surgery to remove their brain tumor. The other group will be for people who have previously received treatment for their brain tumor but do not have any other available options for treatment.
The primary goals of this study are:
1. To measure how much of the study drug is present in tumor tissue taken from patients during surgery to remove their brain tumor
2. To measure the length of time between a study participant's first dose of study treatment until the time when their brain tumor gets worse or their death

Start Date01 Dec 2026

Sponsor / Collaborator

University of Nebraska

[+1]

NCT05630794

/ RecruitingPhase 1IIT

Phase I Trial of ONC201 for Chemoprevention of Colorectal Cancer

The purpose of this phase I trial is to test the safety and cancer preventive effects of different doses of ONC201 in people with familial adenomatous polyposis (FAP) or a history of multiple polyps. People with familial adenomatous polyposis (FAP) or a history of multiple polyps are at higher than average risk of developing colorectal cancer. ONC201, now known as dordaviprone, is a drug that may stop cancer cells from growing. This drug has been shown in previous studies to cause cancer cell death but not harm normal cells. If successful, this study may help us develop a new option for colorectal cancer prevention.

Start Date06 Oct 2025

Sponsor / Collaborator

National Cancer Institute

NCT04629209

/ WithdrawnPhase 2IIT

A Phase II, Open Label Study of ONC201 in Adults With EGFR-low Glioblastoma

This is an open-label, two arm study. The trial will enroll a total of up to 36 patients. Arm A will enroll up to a total of 6 evaluable patients and Arm B will enroll up to a total of 30 evaluable patients. Arm A will explore the intra-tumoral ONC201 concentrations and pharmacodynamic activity in adult EGFR-low glioblastoma patients. Arm B will determine the radiographic efficacy of ONC201 in adult recurrent EGFR-low glioblastoma patients. All patients will be treated with oral ONC201 (625 mg) twice weekly, 2 consecutive days on and 5 days off per week schedule.

Start Date28 Jun 2024

Sponsor / Collaborator

University of Minnesota Masonic Cancer Center

[+1]

100 Clinical Results associated with Dordaviprone

100 Translational Medicine associated with Dordaviprone

100 Patents (Medical) associated with Dordaviprone

259

Literatures (Medical) associated with Dordaviprone

01 Sep 2026·AMERICAN JOURNAL OF HEMATOLOGY

Dordaviprone Maintenance After Allogeneic HCT for High‐Risk Acute Myeloid Leukemia and Myelodysplastic Neoplasm

Article

Author: James E. Talmadge ; Alyssa Bouska ; Vijaya Raj Bhatt ; Krishna Gundabolu ; Michael Haddadin ; Javeed Iqbal ; Christopher S. Wichman ; Moataz Ellithi ; Lori J. Maness

ABSTRACT:

Dordaviprone is a first‐in‐class small‐molecule imipridone. In preclinical studies, it is active against leukemia cells harboring a TP53 mutation or complex karyotype and leukemia stem cells while sparing normal bone marrow cells. This study aimed to determine the safety of dordaviprone maintenance after allogeneic hematopoietic cell transplantation (HCT) for high‐risk acute myeloid leukemia (AML) and myelodysplastic neoplasm (MDS). In a Phase I trial ( n = 20), adults with high‐risk AML or MDS received escalating doses of oral dordaviprone administered weekly for up to 13 four‐weekly cycles ( ClinicalTrials.gov ID: NCT03932643). The primary objective was to determine the rate of dose‐limiting toxicities (DLTs) and grade ≥ 3 adverse events (AEs). Participants' characteristics included a median age of 68 years (range: 39–75 years) and high‐risk features such as adverse‐risk AML (50%) or very poor‐risk MDS (75%), TP53 mutations (36%), or measurable residual disease before HCT (41% of AML). A total of 10 patients (one unevaluable) received 250–500 mg weekly doses, whereas the remaining 10 patients received 625 mg weekly doses. Patients received a median of 8.5 cycles (range: 1–13) of dordaviprone. No DLTs or graft failures were noted. Grades 3–4 acute GVHD was noted in 5%. Grade ≥ 3 AEs occurred in 45% (15% related). Grade 3–4 cytopenias were infrequent: anemia (15%), neutropenia (10%), and thrombocytopenia (15%). At 2 years, relapse‐free survival and overall survival were 60% (95% confidence interval [CI]: 42%–86%) and 70% (95% CI: 53%–93%), respectively. Posttransplant dordaviprone maintenance was determined to be safe with low rates of cytopenias and promising efficacy in this high‐risk population.

Trial Registration:

ClinicalTrials.gov identifier: NCT03932643

01 Aug 2026·NEURO-ONCOLOGY

Point/Counterpoint: Dordaviprone for diffuse midline glioma - A landmark approval or a premature step?

Article

Author: Verhaak, Roel G W ; Bouffet, Eric ; Hansford, Jordan R ; Dun, Matthew D ; Mueller, Sabine ; Wen, Patrick Y ; Arrillaga-Romany, Isabel ; Koschmann, Carl ; Khasraw, Mustafa ; Singh, Kirit

Abstract:

Diffuse midline glioma (DMG) remains one of the most aggressive and uniformly fatal brain tumors, with a median overall survival (OS) of only 11 months and no systemic therapy yet demonstrating a meaningful survival benefit. Primarily affecting children and young adults, DMG represents an area of profound unmet clinical need where families and oncologists have faced decades of therapeutic stagnation. The recent accelerated approval by the United States (US) Food and Drug Administration (FDA) of dordaviprone (ONC201, ModeysoTM) for patients with progressive H3 K27M–altered DMG marks the first regulatory recognition of a systemic therapy for this devastating disease.The decision has generated both optimism and debate within the neuro-oncology community. For many, the approval symbolizes long-overdue progress and affirms that systemic agents can demonstrate activity in DMG. For others, it raises concerns over whether the current evidence base is sufficient to justify widespread clinical adoption. The accelerated approval pathways allow for earlier access to therapy for serious life-threatening conditions based on surrogate endpoints, such as durable objective response rates (ORRs), while mandating post-approval studies to confirm benefit. However, while approvals using data from single-arm trials can make promising therapies available to patients expeditiously, such exceptional approvals present challenges that must be carefully considered. In the case of dordaviprone, this mechanism was applied following an integrated analysis of early-phase studies (ONC006 (NCT02525692), ONC013 (NCT03295396), ONC014 (NCT03416530),1 ONC016 (NCT05392374), and ONC018 (NCT03134131))2 that demonstrated measurable radiographic and biological responses in a subset of patients.Here, we examine the scientific, clinical, and regulatory context of the dordaviprone approval through contrasting perspectives. We consider both the landmark nature of this decision and the potential risks of overinterpreting preliminary evidence, aiming to clarify what this approval means for current practice and for the future development of therapies in DMG and other brain tumors.

01 May 2026·Neuro-oncology advances

A new hope: Clinical advances in targeted therapies for pediatric diffuse midline glioma

Review

Author: Ziegler, David S ; Zhu, Joshua ; Mueller, Sabine ; Tsoli, Maria ; Bertoldo, Jean

Abstract:

Diffuse midline glioma (DMG) is an aggressive pediatric brain tumor with a 1%–2% overall survival, largely due to the ineffectiveness of conventional treatments such as chemotherapy and radiotherapy, as well as the inoperability of the tumors because of their critical location and infiltrative diffuse growth. Recent advances in targeted therapies offer new hope, particularly those addressing key molecular characteristics and newly identified cancer dependencies. Among these are histone deacetylase inhibitors (HDACis), receptor tyrosine kinase inhibitors, and novel agents such as ONC201 and unesbulin that target metabolic and epigenetic pathways respectively. In addition, emerging therapies like FACT inhibitors and polyamine pathway inhibitors are showing promise by disrupting critical cancer cell processes. Immunotherapies, including CAR-T cells targeting surface antigens such as GD2 and B7-H3, cancer vaccines, monoclonal antibodies, and oncolytic viruses, are also gaining traction, offering a new approach by harnessing the immune system to attack tumor cells. Despite these advances, challenges such as drug delivery across the blood–brain barrier and therapeutic resistance persist, necessitating the development of combination therapies and innovative delivery methods. Ongoing research is focused on refining these strategies and exploring additional molecular and immunological targets to improve outcomes for children with DMG.

154

News (Medical) associated with Dordaviprone

03 Aug 2026

·www.prnewswire.com

Jazz Pharmaceuticals Announces Second Quarter 2026 Financial Results

– Continued commercial execution drives record total revenues of $1.2 billion (+16% YoY) – – Company raises 2026 revenue guidance – – Prepared to launch Ziihera® in HER2+ 1L GEA immediately following FDA approval – – Xywav® revenues grew 13% YoY with 525 net patient adds in 2Q26 – – Epidiolex® revenues grew 16% YoY – DUBLIN, Aug. 3, 2026 /PRNewswire/ -- Jazz Pharmaceuticals plc (Nasdaq: JAZZ) today announced financial results for the second quarter of 2026 (2Q26) and raised revenue guidance for 2026. "Our second quarter results highlight strong execution and momentum across the business, delivering 16% year-over-year total revenue growth and driving a considerable increase to our full-year revenue guidance," said Renee Gala, president and chief executive officer of Jazz Pharmaceuticals. "We remain focused on long-term growth as we prepare to launch Ziihera in HER2+ 1L GEA, advance the zanidatamab and Epidiolex clinical programs, and expand our pipeline through targeted corporate development and internal research and development. The combination of commercial execution, portfolio expansion and our strong financial foundation positions Jazz to deliver meaningful innovation for patients and substantial value for shareholders." Recent Key Highlights Highest ever total quarterly revenues of $1.2 billion with 16% year-over-year (YoY) growth. Generated GAAP / non-GAAP1 adjusted earnings per share (EPS) of $2.78 / $5.71 with $824 million in cash from operations in the first half of 2026. U.S. Food and Drug Administration (FDA) granted Priority Review and set Prescription Drug User Fee Act (PDUFA) target action date of August 25, 2026 for supplemental Biologics License Application (sBLA) for zanidatamab containing combinations in first-line (1L) gastroesophageal adenocarcinoma (GEA). Results from Phase 3 HERIZON-GEA-01 published in The New England Journal of Medicine; additional subgroup analyses presented in an oral presentation at the 2026 ASCO Annual Meeting showing improved clinical outcomes with zanidatamab-containing combinations regardless of PD-L1 expression, including in PD-L1-negative patients. FDA granted Breakthrough Therapy designation (BTD) for zanidatamab in adults with previously treated locally advanced, unresectable, or metastatic HER2-positive colorectal cancer (CRC). Following strong 1H26 commercial execution, the company raised its 2026 revenue guidance range to $4.60 - $4.75 billion, reflecting anticipated double-digit YoY revenue growth from both Xywav and the combined epilepsy and oncology franchises. Business Updates Xywav (calcium, magnesium, potassium, and sodium oxybates) oral solution: Xywav net product sales increased 13% YoY to $471 million in 2Q26. Robust new patient growth, with approximately 525 net patient adds in 2Q26. There were approximately 17,125 active patients exiting the quarter, comprised of approximately 11,275 narcolepsy patients and approximately 5,850 idiopathic hypersomnia (IH) patients. Continued physician and patient demand for the differentiated benefits of low-sodium Xywav. Epidiolex/ Epidyolex (cannabidiol): Epidiolex/Epidyolex net product sales increased 16% YoY to $292 million in 2Q26, driven by continued strong demand. Expanded Epidiolex development program to reach more patients with the following clinical trials: Phase 3 trial in developmental and epileptic encephalopathy (DEE), Phase 2/3 trial in juvenile myoclonic epilepsy (JME) and Phase 3b/4 trial in adult Lennox-Gastaut syndrome (LGS). Submitted New Drug Application (NDA) for cannabidiol capsule formulation to broaden utilization of cannabidiol in currently approved indications and increase flexibility for patients. Ziihera (zanidatamab-hrii): Ziihera net product sales in biliary tract cancer (BTC) were $15 million in 2Q26. Prepared to launch zanidatamab in HER2+ 1L GEA (PDUFA date of August 25, 2026). Top-line results from the second interim overall survival (OS) analysis for the HERIZON-GEA-01 trial doublet regimen are expected in 3Q26. Modeyso™ (dordaviprone): Modeyso net product sales were $48 million in 2Q26 with more than 600 patients having received Modeyso from product launch in August 2025 through the end of the second quarter of 2026. Anticipate the OS interim analysis for the event-driven Phase 3 ACTION trial in 1H27, based on current pace of event accrual. Zepzelca® (lurbinectedin): Zepzelca net product sales increased 42% YoY to $106 million in 2Q26, driven by continued uptake of the Zepzelca and atezolizumab combination in the 1L maintenance ES-SCLC setting, partially offset by a decline in second-line use. Based on the results from the LAGOON trial of Zepzelca in second-line metastatic SCLC, and in alignment with FDA, in 3Q26, we will submit for FDA's review and subsequent action a labeling supplement to remove the second-line indication. The first-line maintenance indication will not be affected. Corporate Development: Announced a preclinical research collaboration with AbCellera Biologics Inc. (AbCellera), to develop next-generation T-cell engaging multispecific antibodies for multiple gastrointestinal (GI) cancers and other solid tumors. The company continues to actively evaluate additional value-enhancing corporate development. Financial Highlights GAAP and non-GAAP adjusted net income in 2Q26 includes acquired in-process research and development (IPR&D) expense of $77.0 million, relating to upfront payments made in connection with our collaboration and license agreement with AbCellera and asset purchase agreement to acquire remaining rights for JZP898 from Werewolf Therapeutics, Inc. (Werewolf). This impacted our GAAP and non-GAAP adjusted results by $65.4 million (net of tax of $11.6 million) or $0.94 per share. GAAP and non-GAAP adjusted net loss in 2Q25 includes acquired IPR&D expense of $905.4 million representing the value allocated to Modeyso in the Chimerix Acquisition, which impacted our results by $14.78 per share and $14.75 per share on a GAAP and non-GAAP adjusted basis, respectively. Reconciliations of applicable GAAP reported to non-GAAP adjusted information are included at the end of this press release. Total Revenues Total revenues increased 16% in 2Q26 YoY primarily due to higher Xywav, Epidiolex/Epidyolex and Zepzelca net product sales and the inclusion of Modeyso net product sales, following FDA approval in August 2025. Operating Expenses and Income Tax Expense (Benefit) Changes in operating expenses and income tax expense (benefit) in 2Q26 over the prior year period are primarily due to the following: Cost of product sales, on a GAAP and non-GAAP adjusted basis, increased in 2Q26, primarily due to higher royalty expenses, driven by higher revenues of Modeyso and Zepzelca, offset on a GAAP basis, by lower acquisition accounting inventory fair value step up expense. Selling, general and administrative (SG&A) expenses, on a GAAP and non-GAAP adjusted basis, increased in 2Q26, primarily due to higher marketing investment and compensation-related expenses in support of our commercial portfolio. Research and development (R&D) expenses, on a GAAP and non-GAAP adjusted basis, increased in 2Q26, driven by higher clinical studies costs, primarily related to zanidatamab. Acquired IPR&D, on a GAAP and non-GAAP adjusted basis, in 2Q26 comprised the upfront payments to AbCellera and Werewolf. Income tax expense, on a GAAP and non-GAAP adjusted basis, in 2Q26 reflects changes in the geographic mix of income and expenses compared to 2Q25. Cash Flow and Balance Sheet As of June 30, 2026, cash, cash equivalents and investments were $2.2 billion, and the outstanding principal balance of the company's long-term debt was $4.4 billion. In addition, the company had undrawn borrowing capacity under a revolving credit facility of $885 million. In June 2026, we repaid the $1.0 billion aggregate principal amount of the 2.00% exchangeable senior notes due 2026. For the six months ended June 30, 2026, the company generated $824 million of cash from operations reflecting strong business performance and continued financial discipline. 2026 Financial Guidance Jazz Pharmaceuticals has updated its full-year 2026 financial guidance as shown in the table below. Conference Call Details Jazz Pharmaceuticals will host an investor conference call and live audio webcast today at 4:30 p.m. ET to provide a business and financial update and discuss its 2026 second quarter results. Interested parties may register for the call here or via the Investors section of the Jazz Pharmaceuticals website at . To ensure a timely connection, it is recommended that participants register at least 15 minutes prior to the scheduled webcast. A replay of the webcast will be available via the Investors section of the Jazz Pharmaceuticals website at . 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 for more information. Non-GAAP Financial Measures To supplement Jazz Pharmaceuticals' financial results and guidance presented in accordance with U.S. generally accepted accounting principles (GAAP), the company uses certain non-GAAP (also referred to as adjusted or non-GAAP adjusted) financial measures in this press release and the accompanying tables. In particular, the company presents non-GAAP adjusted net income (loss) (and the related per share measure) and its line-item components, as well as certain non-GAAP adjusted financial measures derived therefrom, including non-GAAP adjusted gross margin percentage and non-GAAP adjusted effective tax rate. Non-GAAP adjusted net income (loss) (and the related per share measure) and its line-item components exclude from GAAP reported net income (loss) (and the related per share measure) and its line-item components certain items, as detailed in the reconciliation tables that follow, and in the case of non-GAAP adjusted net income (loss) (and the related per share measure), adjust for the income tax effect of the non-GAAP adjustments. In this regard, the components of non-GAAP adjusted net income (loss), including non-GAAP adjusted cost of product sales, SG&A expenses and R&D expenses, are income statement line items prepared on the same basis as, and therefore components of, the overall non-GAAP adjusted net income (loss) measure. The company believes that each of these non-GAAP financial measures provides useful supplementary information to, and facilitates additional analysis by, investors and analysts and that each of these non-GAAP financial measures, when considered together with the company's financial information prepared in accordance with GAAP, can enhance investors' and analysts' ability to meaningfully compare the company's results from period to period, to its forward-looking guidance, and to identify operating trends in the company's business. In addition, these non-GAAP financial measures are regularly used by investors and analysts to model and track the company's financial performance. Jazz Pharmaceuticals' management also regularly uses these non-GAAP financial measures internally to understand, manage and evaluate the company's business and to make operating decisions, and compensation of executives is based in part on certain of these non-GAAP financial measures. Because these non-GAAP financial measures are important internal measurements for Jazz Pharmaceuticals' management, the company also believes that these non-GAAP financial measures are useful to investors and analysts since these measures allow for greater transparency with respect to key financial metrics the company uses in assessing its own operating performance and making operating decisions. These non-GAAP financial measures are not meant to be considered in isolation or as a substitute for comparable GAAP measures; should be read in conjunction with the company's consolidated financial statements prepared in accordance with GAAP; have no standardized meaning prescribed by GAAP; and are not prepared under any comprehensive set of accounting rules or principles in the reconciliation tables that follow. In addition, from time to time in the future there may be other items that the company may exclude for purposes of its non-GAAP financial measures; and the company has ceased, and may in the future cease, to exclude items that it has historically excluded for purposes of its non-GAAP financial measures. Likewise, the company may determine to modify the nature of its adjustments to arrive at its non-GAAP financial measures. Because of the non-standardized definitions of non-GAAP financial measures, the non-GAAP financial measures as used by Jazz Pharmaceuticals in this press release and the accompanying tables have limits in their usefulness to investors and may be calculated differently from, and therefore may not be directly comparable to, similarly titled measures used by other companies. Cautionary Note Concerning Forward-Looking Statements This press release contains forward-looking statements, including, but not limited to, statements related to: the company's growth prospects and future financial and operating results, including the company's 2026 financial guidance and the company's expectations related thereto, including with respect to anticipated catalysts; the company's advancement of pipeline programs and the timing of development activities, regulatory activities, approvals, and submissions related thereto; the company's preparation for a near-term commercial launch of zanidatamab in 1L HER2+ GEA in the U.S. following potential approval on or before the PDUFA date; the company's expectations with respect to its regulatory submissions, including its NDA for a cannabidiol oral capsule formulation for potential use in existing approved Epidiolex/Epidyolex formulations; planned or anticipated clinical trial events, including with respect to initiations, enrollment and data read-outs, and the anticipated timing thereof, including: the second interim OS data from the Phase 3 HERIZON trial of zanidatamab in 1L GEA and top-line data from the Phase 3 ACTION trial of Modeyso in recurrent H3 K27M-mutant diffuse glioma; the company's expectations with respect to its preclinical research collaboration, option and license agreement with AbCellera; and the company's development, regulatory and commercialization strategy, including the company's expectation of portfolio expansion through business development and its research and development engine; the company's expectations with respect to its products and product candidates and the potential of the company's products and product candidates and the potential regulatory path related thereto, including zanidatamab's potential to become the HER2-targeted therapy of choice in 1L HER2+ GEA; the company's capital allocation and corporate development strategy; the potential successful future development, manufacturing, regulatory and commercialization activities; the company's ability to realize the commercial potential of its products; the company's net product sales and goals for net product sales from new and acquired products; the company's views and expectations relating to its patent portfolio, including with respect to expected patent protection, as well as expectations with respect to exclusivity; the company's clinical trials confirming clinical benefit or enabling regulatory submissions, including the potential of the ongoing Phase 3 ACTION trial to confirm clinical benefit of Modeyso in recurrent H3 K27M-mutant diffuse glioma and extend to use in 1L patients; and other statements that are not historical facts. These forward-looking statements are based on the company's current plans, objectives, estimates, expectations and intentions and inherently involve significant risks and uncertainties. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of these risks and uncertainties, which include, without limitation, risks and uncertainties associated with: maintaining or increasing sales of, and revenue from, Xywav, Epidiolex/Epidyolex, Ziihera, Modeyso, Zepzelca and other lead marketed products; effectively launching and commercializing the company's other products and product candidates; the successful completion of development and regulatory activities with respect to the company's product candidates; obtaining and maintaining adequate coverage and reimbursement for the company's products; the time-consuming and uncertain regulatory approval process, including the risk that the company's current and/or planned regulatory submissions may not be submitted, accepted or approved by applicable regulatory authorities in a timely manner or at all, including the risk that zanidatamab in 1L HER2+ GEA may not be approved in a timely manner or at all; the costly and time-consuming pharmaceutical product development process and the uncertainty of clinical success, including risks related to failure or delays in successfully initiating or completing clinical trials and assessing patients; global economic, financial, and healthcare system disruptions and the current and potential future negative impacts to the company's business operations and financial results; protecting and enhancing the company's intellectual property rights and the company's commercial success being dependent upon the company obtaining, maintaining and defending intellectual property protection and exclusivity for its products and product candidates; delays or problems in the supply or manufacture of the company's products and product candidates, including due to geopolitical tensions and military conflicts; complying with applicable U.S. and non-U.S. regulatory requirements, including those governing the research, development, manufacturing and distribution of controlled substances; government investigations, legal proceedings and other actions; identifying and consummating corporate development transactions, financing these transactions and successfully integrating acquired products, product candidates and businesses; the company's ability to realize the anticipated benefits of its collaborations and license agreements with third parties; the sufficiency of the company's cash flows and capital resources; the company's ability to achieve targeted or expected future financial performance and results and the uncertainty of future tax, accounting and other provisions and estimates; the company's ability to meet its projected long-term goals and objectives, in the time periods that the company anticipates, or at all, and the inherent uncertainty and significant judgments and assumptions underlying the company's long-term goals and objectives; fluctuations in the market price and trading volume of the company's ordinary shares; and other risks and uncertainties affecting the company, including those described from time to time under the caption "Risk Factors" and elsewhere in the company's Securities and Exchange Commission filings and reports, including the company's Annual Report on Form 10-K for the year ended December 31, 2025 and future filings and reports by the company. Other risks and uncertainties of which the company is not currently aware may also affect the company's forward-looking statements and may cause actual results and the timing of events to differ materially from those anticipated. Contacts: Investors: [email protected] Ireland +353 1 634 7800 U.S. +1 650 496 2717 Media: [email protected] Ireland +353 1 637 2141 U.S. +1 215 867 4948 21% more press release views with Request a Demo

05 May 2026

·www.prnewswire.com

Jazz Pharmaceuticals Announces First Quarter 2026 Financial Results

– Strong commercial execution across franchises with total revenues of $1.1 billion (+19% YoY) – – Zanidatamab HER2+ 1L GEA sBLA granted Priority Review; PDUFA date of August 25, 2026 – – Xywav® revenues grew 18% YoY with 425 net patient adds – – Epidiolex® revenues grew 15% YoY– – Company reaffirms 2026 revenue and expense guidance – DUBLIN, May 5, 2026 /PRNewswire/ -- Jazz Pharmaceuticals plc (Nasdaq: JAZZ) today announced financial results for the first quarter of 2026 (1Q26). "Our first-quarter results reflect disciplined execution across the business, delivering 19% year-over-year growth alongside key pipeline advancements and positioning the company for an outstanding 2026," said Renee Gala, president and chief executive officer of Jazz Pharmaceuticals. "Demand for Xywav remained strong, our rare oncology launches with Modeyso™ and Zepzelca® in 1LM ES-SCLC gained significant momentum, and Epidiolex continued to provide consistent growth. Looking ahead, we are excited about the potential launch of zanidatamab in 1L GEA later this year, as we progress our pipeline and business development efforts to bring more life-changing therapies to patients and fuel durable long-term growth." Key First Quarter 2026 Highlights Total revenues in 1Q26 grew to $1.1 billion (+19% year-over-year (YoY)) Generated GAAP / non-GAAP1 adjusted earnings per share (EPS) of $4.43 / $6.34 with $408 million in cash from operations. Practice-changing Phase 3 HERIZON-GEA-01 results, presented as a late-breaker at ASCO GI, support zanidatamab as the HER2-targeted agent of choice in HER2+ 1L advanced gastroesophageal adenocarcinoma (GEA); additional benefit from tislelizumab irrespective of PD-L1 status. Supplemental Biologics License Application (sBLA) accepted by FDA under Real-Time Oncology Review (RTOR) program with potential approval and launch in 1L HER2+ GEA on or before PDUFA date. Business Updates Xywav (calcium, magnesium, potassium, and sodium oxybates) oral solution: Xywav net product sales increased 18% to $408 million in 1Q26, compared to 1Q25. Continued physician and patient demand for the differentiated benefits of low-sodium Xywav. Strong new patient growth, with approximately 425 net patient adds in 1Q26. There were approximately 16,600 active patients exiting the quarter, comprised of approximately 11,075 narcolepsy patients and approximately 5,525 idiopathic hypersomnia (IH) patients. Epidiolex/ Epidyolex (cannabidiol): Epidiolex/Epidyolex net product sales increased 15% YoY to $250 million in 1Q26, driven by continued strong demand. Announced agreement with Nippon Zoki to commercialize Epidyolex in Japan, following completion of ongoing clinical trials and potential regulatory approval. Ziihera ® (zanidatamab-hrii): Ziihera net product sales in biliary tract cancer (BTC) were $13 million in 1Q26. In April 2026, FDA accepted the zanidatamab sBLA in GEA for a Priority Review, with a PDUFA date of August 25, 2026. Submitted HERIZON-GEA-01 data for potential National Comprehensive Cancer Network (NCCN) guideline inclusion. HERIZON-GEA-01 data accepted for publication by a top-tier medical journal. The second interim overall survival (OS) analysis for the HERIZON-GEA-01 trial doublet regimen is expected in mid-2026. Multiple registrational trials of zanidatamab are underway, including in metastatic breast cancer (mBC), supporting a broad development program designed to maximize patient impact and long-term shareholder value. Modeyso (dordaviprone): Modeyso net product sales were $41 million in 1Q26 with ~500 patients having received Modeyso from product launch in August 2025 through the end of the first quarter. The company completed the sale of its Rare Pediatric Disease Priority Review Voucher (PRV) for gross proceeds of $200 million (50% to Jazz). Phase 3 ACTION trial remains on track with top-line readout expected late 2026 / early 2027. Zepzelca (lurbinectedin): Zepzelca net product sales increased 60% YoY to $101 million in 1Q26, driven by continued uptake of the Zepzelca and atezolizumab combination in the 1LM ES-SCLC setting, partially offset by a decline in second line use. The company expects second line use to decline throughout the year. Financial Highlights The GAAP net loss and non-GAAP adjusted net income for 1Q25 included an expense of $172 million related to Xyrem antitrust litigation settlements, which impacted our GAAP and non-GAAP results by $146 million (net of tax of $26 million), or $2.38 per share on a GAAP basis and $2.34 per share on a non-GAAP adjusted basis. Reconciliations of applicable GAAP reported to non-GAAP adjusted information are included at the end of this press release. Total Revenues Total revenues increased 19% YoY primarily due to higher Xywav, Zepzelca and Epidiolex/Epidyolex net product sales and the inclusion of Modeyso net product sales, following FDA approval in August 2025. Operating Expenses and Income Tax Expense (Benefit) Changes in operating expenses and income tax expense (benefit) in 1Q26 over the prior year period are primarily due to the following: Cost of product sales, on a GAAP and non-GAAP adjusted basis, increased in 1Q26, primarily due to higher royalty expenses, driven by higher revenues, and increased inventory provisions. The cost of product sales, on a GAAP basis, in 1Q26 included higher acquisition accounting inventory fair value step up expense compared to 1Q25. Selling, general and administrative (SG&A) expenses, on a GAAP and non-GAAP adjusted basis, decreased in 1Q26, primarily due to certain Xyrem antitrust litigation settlements of $172 million incurred in 1Q25, partially offset by higher compensation-related expenses in 1Q26 including costs relating to Modeyso. Research and development (R&D) expenses, on a GAAP and non-GAAP adjusted basis, increased in 1Q26, primarily due to the addition of costs relating to Modeyso including personnel costs. Income tax expense in 1Q26, on a GAAP basis, was primarily attributable to the gain recognized on the sale of the PRV, partially offset by excess tax benefits from share-based compensation. Income tax benefit in 1Q25, on a GAAP basis, was primarily attributable to the Xyrem antitrust litigation settlements. Cash Flow and Balance Sheet As of March 31, 2026, cash, cash equivalents and investments were $2.9 billion, and the outstanding principal balance of the company's long-term debt was $5.4 billion. In addition, the company had undrawn borrowing capacity under a revolving credit facility of $885 million. For the three months ended March 31, 2026, the company generated $408 million of cash from operations reflecting strong business performance and continued financial discipline. In 1Q26, the company received gross proceeds of $200 million (50% to Jazz) from the sale of the PRV. 2026 Financial Guidance Conference Call Details Jazz Pharmaceuticals will host an investor conference call and live audio webcast today at 4:30 p.m. ET to provide a business and financial update and discuss its 2026 first quarter results. Interested parties may register for the call here or via the Investors section of the Jazz Pharmaceuticals website at . To ensure a timely connection, it is recommended that participants register at least 15 minutes prior to the scheduled webcast. A replay of the webcast will be available via the Investors section of the Jazz Pharmaceuticals website at . 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 for more information. Non-GAAP Financial Measures To supplement Jazz Pharmaceuticals' financial results and guidance presented in accordance with U.S. generally accepted accounting principles (GAAP), the company uses certain non-GAAP (also referred to as adjusted or non-GAAP adjusted) financial measures in this press release and the accompanying tables. In particular, the company presents non-GAAP adjusted net income (and the related per share measure) and its line-item components, as well as certain non-GAAP adjusted financial measures derived therefrom, including non-GAAP adjusted gross margin percentage and non-GAAP adjusted effective tax rate. Non-GAAP adjusted net income (and the related per share measure) and its line-item components exclude from GAAP reported net income (loss) (and the related per share measure) and its line-item components certain items, as detailed in the reconciliation tables that follow, and in the case of non-GAAP adjusted net income (and the related per share measure), adjust for the income tax effect of the non-GAAP adjustments. In this regard, the components of non-GAAP adjusted net income, including non-GAAP adjusted cost of product sales, SG&A expenses and R&D expenses, are income statement line items prepared on the same basis as, and therefore components of, the overall non-GAAP adjusted net income measure. The company believes that each of these non-GAAP financial measures provides useful supplementary information to, and facilitates additional analysis by, investors and analysts and that each of these non-GAAP financial measures, when considered together with the company's financial information prepared in accordance with GAAP, can enhance investors' and analysts' ability to meaningfully compare the company's results from period to period, to its forward-looking guidance, and to identify operating trends in the company's business. In addition, these non-GAAP financial measures are regularly used by investors and analysts to model and track the company's financial performance. Jazz Pharmaceuticals' management also regularly uses these non-GAAP financial measures internally to understand, manage and evaluate the company's business and to make operating decisions, and compensation of executives is based in part on certain of these non-GAAP financial measures. Because these non-GAAP financial measures are important internal measurements for Jazz Pharmaceuticals' management, the company also believes that these non-GAAP financial measures are useful to investors and analysts since these measures allow for greater transparency with respect to key financial metrics the company uses in assessing its own operating performance and making operating decisions. These non-GAAP financial measures are not meant to be considered in isolation or as a substitute for comparable GAAP measures; should be read in conjunction with the company's consolidated financial statements prepared in accordance with GAAP; have no standardized meaning prescribed by GAAP; and are not prepared under any comprehensive set of accounting rules or principles in the reconciliation tables that follow. In addition, from time to time in the future there may be other items that the company may exclude for purposes of its non-GAAP financial measures; and the company has ceased, and may in the future cease, to exclude items that it has historically excluded for purposes of its non-GAAP financial measures. Likewise, the company may determine to modify the nature of its adjustments to arrive at its non-GAAP financial measures. Because of the non-standardized definitions of non-GAAP financial measures, the non-GAAP financial measures as used by Jazz Pharmaceuticals in this press release and the accompanying tables have limits in their usefulness to investors and may be calculated differently from, and therefore may not be directly comparable to, similarly titled measures used by other companies. Cautionary Note Concerning Forward-Looking Statements This press release contains forward-looking statements, including, but not limited to, statements related to: the company's growth prospects and future financial and operating results, including the company's 2026 financial guidance and the company's expectations related thereto, including with respect to anticipated catalysts; the company's advancement of pipeline programs and the timing of development activities, regulatory activities, approvals, and submissions related thereto; the potential for a near-term commercial launch of zanidatamab in 1L HER2+ GEA in the U.S., if approved; planned or anticipated clinical trial events, including with respect to initiations, enrollment and data read-outs, and the anticipated timing thereof, including: the second interim OS data from the Phase 3 HERIZON trial of zanidatamab in 1L GEA and top-line data from the Phase 3 ACTION trial of Modeyso in recurrent H3 K27M-mutant diffuse glioma; and the company's development, regulatory and commercialization strategy; the company's expectations with respect to its products and product candidates and the potential of the company's products and product candidates and the potential regulatory path related thereto, including zanidatamab's potential to become the HER2-targeted therapy of choice in 1L HER2+ GEA, regardless of PD-L1 status; the company's capital allocation and corporate development strategy; the potential successful future development, manufacturing, regulatory and commercialization activities; the company's ability to realize the commercial potential of its products; the company's net product sales and goals for net product sales from new and acquired products; the company's views and expectations relating to its patent portfolio, including with respect to expected patent protection, as well as expectations with respect to exclusivity; the company's clinical trials confirming clinical benefit or enabling regulatory submissions, including the potential of the ongoing Phase 3 ACTION trial to confirm clinical benefit of Modeyso in recurrent H3 K27M-mutant diffuse glioma and extend to use in 1L patients; and other statements that are not historical facts. These forward-looking statements are based on the company's current plans, objectives, estimates, expectations and intentions and inherently involve significant risks and uncertainties. Actual results and the timing of events could differ materially from those anticipated in such forward- looking statements as a result of these risks and uncertainties, which include, without limitation, risks and uncertainties associated with: maintaining or increasing sales of, and revenue from, Xywav, Epidiolex/Epidyolex, Ziihera, Modeyso, Zepzelca and other lead marketed products; effectively launching and commercializing the company's other products and product candidates; the successful completion of development and regulatory activities with respect to the company's product candidates; obtaining and maintaining adequate coverage and reimbursement for the company's products; the time-consuming and uncertain regulatory approval process, including the risk that the company's current and/or planned regulatory submissions may not be submitted, accepted or approved by applicable regulatory authorities in a timely manner or at all, including the risk that zanidatamab in 1L HER2+ GEA may not be approved in a timely manner or at all; the costly and time-consuming pharmaceutical product development process and the uncertainty of clinical success, including risks related to failure or delays in successfully initiating or completing clinical trials and assessing patients; global economic, financial, and healthcare system disruptions and the current and potential future negative impacts to the company's business operations and financial results; protecting and enhancing the company's intellectual property rights and the company's commercial success being dependent upon the company obtaining, maintaining and defending intellectual property protection and exclusivity for its products and product candidates; delays or problems in the supply or manufacture of the company's products and product candidates, including due to geopolitical tensions and military conflicts; complying with applicable U.S. and non-U.S. regulatory requirements, including those governing the research, development, manufacturing and distribution of controlled substances; government investigations, legal proceedings and other actions; identifying and consummating corporate development transactions, financing these transactions and successfully integrating acquired products, product candidates and businesses; the company's ability to realize the anticipated benefits of its collaborations and license agreements with third parties; the sufficiency of the company's cash flows and capital resources; the company's ability to achieve targeted or expected future financial performance and results and the uncertainty of future tax, accounting and other provisions and estimates; the company's ability to meet its projected long-term goals and objectives, in the time periods that the company anticipates, or at all, and the inherent uncertainty and significant judgments and assumptions underlying the company's long-term goals and objectives; fluctuations in the market price and trading volume of the company's ordinary shares; and other risks and uncertainties affecting the company, including those described from time to time under the caption "Risk Factors" and elsewhere in the company's Securities and Exchange Commission filings and reports, including the company's Annual Report on Form 10-K for the year ended December 31, 2025 and future filings and reports by the company. Other risks and uncertainties of which the company is not currently aware may also affect the company's forward-looking statements and may cause actual results and the timing of events to differ materially from those anticipated. Contacts: Investors: [email protected] Ireland +353 1 634 3211 U.S. +1 650 496 2717 Media: [email protected] Ireland +353 1 637 2141 U.S. +1 215 867 4948 SOURCE Jazz Pharmaceuticals plc 21% more press release views with Request a Demo

Phase 3Financial StatementDrug ApprovalPriority ReviewNDA

04 May 2026

·www.biospace.com

Artios Further Expands Leadership Team to Advance Oncology Pipeline and Accelerate Regulatory Execution

Jeremy B. Fitch, MBA, appointed as Chief Business Officer Michael A. Alrutz, J.D., Ph.D., appointed as General Counsel Guy C. Ruble, PharmD, appointed as VP, Regulatory Affairs CAMBRIDGE, United Kingdom and NEW YORK, May 04, 2026 – Artios Pharma Limited (“Artios” or “the Company”), a clinical-stage biopharmaceutical company pioneering the development of new classes of DNA Damage Response (DDR) medicines to deliver meaningful survival benefits for patients with cancer, today announced the appointment of three senior executives to the leadership team as the Company accelerates its potential first-in-class oncology pipeline. Jeremy B. Fitch, MBA, joins as Chief Business Officer (CBO) and will shape Artios’ corporate business development and strategic partnerships. Michael A. Alrutz, J.D., Ph.D., joins as General Counsel and will oversee Artios’ legal, corporate governance, regulatory compliance, risk management, and intellectual property management, while serving as a strategic partner to the Board of Directors and executive leadership team. Guy C. Ruble, PharmD, has been appointed as Vice President of Regulatory Affairs to lead the Company’s regulatory strategy and initiatives. These appointments come as Artios advances alnodesertib into late-stage development and prepares for potential U.S. commercialization, executes a randomized Phase 2 study for ART6043, and rapidly advances its DNA Damage Response antibody drug conjugate platform toward clinical development. “Jeremy brings a proven track record of executing high‑impact transactions and shaping portfolio and corporate strategy,” said Mike Andriole, Chief Executive Officer of Artios. “His appointment as CBO, together with Michael’s deep legal and governance expertise and Guy’s leadership in global regulatory strategy, comes at a pivotal inflection point as we progress alnodesertib toward potential registration and commercialization. These leadership additions further strengthen our ability to execute with discipline, accelerate paths to registration, and urgently deliver first‑in‑class therapies to patients with late‑stage cancers who need more treatment options.” “With three potentially first-in-class DDR programs under rapid development, Artios is uniquely positioned with a differentiated pipeline and clear opportunities to create long-term value,” said Jeremy B. Fitch, MBA, Chief Business Officer of Artios. “I look forward to working with the rest of the management team to develop and execute value-creating transactions which leverage our substantial DDR scientific and clinical expertise.” Jeremy B. Fitch, MBA, Chief Business Officer Jeremy joins Artios with more than 30 years of experience in the biopharmaceutical industry with Eli Lilly and Company (“Lilly”), across operational, financial, and corporate business development roles. Jeremy spent the past 15 years in Lilly’s Corporate Business Development group, most recently as Vice President of Transactions, where he led strategic deal-making spanning M&A, asset acquisitions/divestitures, and licensing agreements. Having led more than $5 billion in transactions during his career, Jeremy has been central to developing and implementing strategies that have driven substantial value creation across Lilly’s pipeline. Jeremy is a Certified Licensing Professional and holds an MBA from Duke University’s Fuqua School of Business. Michael A. Alrutz, J.D., Ph.D., General Counsel Dr. Alrutz is a seasoned General Counsel and Corporate Secretary, bringing 25 years of biotechnology legal affairs experience to Artios across both private and public biopharmaceutical companies. Dr. Alrutz brings a strong track record advising leadership teams through complex corporate inflection points, including IPOs, follow-on financings, intellectual property matters, pre-commercialization matters, and high-value corporate transactions. Most recently, he was SVP, General Counsel, Secretary, and Chief Compliance Officer at Chimerix, where he led SEC compliance, board governance, intellectual property management, and the legal facets of corporate transactions, including the company’s $935 million all-cash merger with Jazz Pharmaceuticals as the company prepared for commercialization of Modeyso® (dordaviprone). Previously, while General Counsel at Trimeris, Inc., he provided legal counsel for the company’s commercial launch of its antiretroviral, Fuzeon® (enfuvirtide). Dr. Alrutz is registered to practice before the USPTO, and holds a J.D. from Duke University School of Law, and a Ph.D. in Microbiology and Molecular Biology from Tufts University. Guy C. Ruble, PharmD, VP of Regulatory Affairs Guy brings more than 20 years of regulatory affairs leadership and nearly three decades of biopharmaceutical industry experience to Artios. He most recently served as Vice President of Regulatory Affairs at Iovance Biotherapeutics, where he led the U.S. FDA accelerated approval of AMTAGVI® (li ), the first tumor-infiltrating lymphocyte (TIL) therapy approved in the United States and Canada, and also led global submissions across Europe and Asia. Prior to Iovance, Guy spent nearly 23 years at Eli Lilly and Company, leading global regulatory strategy for multiple oncology programs, including the U.S. FDA approval of Verzenio® (abemaciclib). He earned recognition as a Top 100 Lilly Innovator. Guy holds a Doctor of Pharmacy (PharmD) from Purdue University and is Regulatory Affairs Certified (RAC-US). He is a member of the Regulatory Affairs Professional Society and the American Society of Clinical Oncology. About Artios Pharma Ltd. Artios’ mission is to develop new classes of medicines that harness DNA Damage Response (DDR) pathways, targeting DNA replication stress and synthetic lethality, to deliver meaningful survival benefits for patients with cancer. Its three potentially first-in-class programs, each with a novel mechanism of action, include ATR inhibitor alnodesertib, the DNA polymerase theta (Polθ) inhibitor ART6043, and a preclinical portfolio of DDRi-ADC candidates with novel payloads. Together, these programs are designed to eliminate cancer cells’ survival mechanisms, driving cancer cell death and improving clinical outcomes. Visit our website at to learn more about Artios. For more information, please contact: Trophic Communications Jacob Verghese or Verena Schossmann Tel: +49 151 7441 6179 Email: artios@trophic.eu Attachment Artios Leadership PR

Drug ApprovalPhase 2Executive Change

100 Deals associated with Dordaviprone

External Link

KEGG	Wiki	ATC	Drug Bank
D12733 D12734	-	-	DB14844

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Diffuse midline glioma, point mutation K27M in histone H3	United States	Chimerix, Inc.	06 Aug 2025

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Diffuse Intrinsic Pontine Glioma	Phase 3	Denmark	Jazz Pharmaceuticals Plc	29 Sep 2022
Diffuse Intrinsic Pontine Glioma	Phase 3	Finland	Jazz Pharmaceuticals Plc	29 Sep 2022
Diffuse Intrinsic Pontine Glioma	Phase 3	France	Jazz Pharmaceuticals Plc	29 Sep 2022
Diffuse Intrinsic Pontine Glioma	Phase 3	Norway	Jazz Pharmaceuticals Plc	29 Sep 2022
Diffuse Intrinsic Pontine Glioma	Phase 3	Spain	Jazz Pharmaceuticals Plc	29 Sep 2022
Diffuse Intrinsic Pontine Glioma	Phase 3	Sweden	Jazz Pharmaceuticals Plc	29 Sep 2022
Meningioma	Phase 2	-	Jazz Pharmaceuticals Plc	01 Dec 2026
Recurrent Glioma	Phase 2	United States	Jazz Pharmaceuticals Plc	20 Oct 2021
Recurrent Glioma	Phase 2	Australia	Jazz Pharmaceuticals Plc	20 Oct 2021
Recurrent Glioma	Phase 2	Israel	Jazz Pharmaceuticals Plc	20 Oct 2021

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT03416530 \| NCT03295396 \| NCT05392374 \| NCT03134131 \| NCT02525692 (ONC018, FDA) Manual	Phase 1	Diffuse midline glioma, point mutation K27M in histone H3 H3 K27M-mutant	50	Dordaviprone	mqynwtalnu(jmahnbhpko) = oeommcrqwm pwyzdwrywr (rfvunkzwyb, 12 - 36) View more	Positive	06 Aug 2025
NCT03295396 (ACTR-34 \| ONC013, CTgov)	Phase 2	Diffuse midline glioma, point mutation K27M in histone H3 \| Recurrent Malignant Glioma	73	Dordaviprone (Arm A)	bwixxsqtqe = znrhrelwsp mfkkgmwozl (hcaweqvrod, hkfdrvltrc - jxmxgkxkxm) View more	-	28 Jul 2025
				Dordaviprone (Arm B)	bwixxsqtqe = atvvqykqmw mfkkgmwozl (hcaweqvrod, omtpjqscca - tdypuryhel) View more
NCT03416530 \| NCT03295396 (ONC013 (Arm B) and ONC014 (Arm F), ASCO2025)	Phase 1/2	Diffuse midline glioma, point mutation K27M in histone H3 H3 K27M-mutant	41	Dordaviprone (ONC201) 625 mg	kekrrxtsbf(xwpmfaxepo) = vwjtmvqwmu hbqrezcwuh (kyxywyihpv, 5.6 - 34.7) View more	Positive	30 May 2025
				Dordaviprone (ONC201) dose scaled by body weight	kekrrxtsbf(xwpmfaxepo) = mhjwpexsaz hbqrezcwuh (kyxywyihpv ) View more
NCT02525692 (PATH-25 \| ACTR-51 \| ONC006, CTgov)	Phase 2	Diffuse midline glioma, point mutation K27M in histone H3 \| Basal Ganglia Diseases \| Recurrent Glioblastoma	84	Dordaviprone (ONC201) (Arm A)	xbwlmvwoil = jlrlpubdop berywdoizi (oqkbvltbeo, ifzfqkgpsm - eiirvpupmy) View more	-	24 Dec 2024
				Dordaviprone (Arm B)	xbwlmvwoil = tgrjdrsrln berywdoizi (oqkbvltbeo, lkzwdnrctg - olczkfvvtz) View more
NCT03485729 (CTgov)	Phase 2	Recurrent Endometrial Cancer	27	Dordaviprone (Arm B)	obcvhiaogs = sxnaybioha oncvdqgpan (ptumcrbxay, bommmhdwek - ujjyrigcfl) View more	-	24 Dec 2024
				Dordaviprone (Arm A)	efzfpyzdkh(pxnfhhihfk) = cnlpmklhnq bjaqoqnksa (xewyfmdfcu, ewcmjrgqgv - fmcglsqmdj) View more
NCT03034200 (CTgov)	Phase 2	Neuroendocrine Tumors \| Pheochromocytoma	30	ONC201 (Arm A: Metastatic PC-PG)	pqzgdcvgmx(gdlrydiqjb) = hjljmuxyki srvgfzrdoq (dazakajhfy, xjmtkmvedc - mllbzmftdz) View more	-	26 Nov 2024
				ONC201 (Arm B: Other NETs)	pqzgdcvgmx(gdlrydiqjb) = ycdxlxzdsb srvgfzrdoq (dazakajhfy, ibotaltezh - vdieqhvevp) View more
CTNI-54 (SNO2024)	Not Applicable	Diffuse midline glioma, point mutation K27M in histone H3 H3K27M-mutant	-	ONC201 (DORDAVIPRONE)	yicrzxmlff(wgrhtdjqxd) = zouhgituqm wgxmzndcnk (kutyfeckoi, 15 - 41) View more	Positive	11 Nov 2024
NCT05580562 (ACTION, ASTRO2024)	Phase 3	Diffuse midline glioma, point mutation K27M in histone H3	50	ONC201	efyauyvgbw(houvkelldm) = rfxjlwyrvl iqaooflufy (lcjccdaerx )	Positive	01 Oct 2024
				Standard Radiotherapy Dosing Regimens	efyauyvgbw(houvkelldm) = uvfkqfecmg iqaooflufy (lcjccdaerx )
NCT02863991 (CTgov)	Phase 1/2	Multiple Myeloma	17	Dexamethasone+ONC201 (Phase 1: 375 mg ONC201 + 20 mg Dexamethasone)	jodqestaxe = fxczuqyrko vatehqyjql (ouaxyibiqu, yxgzzvmpki - pnpwydboda) View more	-	03 Jul 2024
				Dexamethasone+ONC201 (Phase 2: 625 mg ONC201 + 20 mg Dexamethasone)	jodqestaxe = tkjsavmpby vatehqyjql (ouaxyibiqu, xjmumdyhtt - eldsxrxwef) View more
NCT05009992 (Biospace) Manual	Phase 2	Diffuse Midline Glioma First line	132	paxalisib in combination with ONC201 (Cohort 1)	ulsejgzfjt(jmtxkaafno) = ohxzwxrbib eyreqkajku (izrsqkzdij )	Positive	27 Jun 2024
				paxalisib in combination with ONC201 (Cohort 2)	ulsejgzfjt(jmtxkaafno) = whsxblzawa eyreqkajku (izrsqkzdij )

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