Eradicating Measurable Residual Disease in Patients With Acute Myeloid Leukemia (AML) Prior to StEm Cell Transplantation (ERASE): A MyeloMATCH Treatment Trial

This phase II MyeloMATCH treatment trial compares cytarabine versus (vs.) cytarabine and venetoclax vs. liposome-encapsulated daunorubicin-cytarabine and venetoclax vs. azacitidine and venetoclax for treating patients who have residual disease after treatment for acute myeloid leukemia (AML). Cytarabine is in a class of medications called antimetabolites. It works by slowing or stopping the growth of cancer cells in the body. Venetoclax is in a class of medications called B-cell lymphoma-2 (BCL-2) inhibitors. It may stop the growth of cancer cells by blocking Bcl-2, a protein needed for cancer cell survival. Liposome-encapsulated daunorubicin-cytarabine is a drug formulation that delivers daunorubicin and cytarabine in small spheres called liposomes, which may make the drugs safer or more effective. Azacitidine is a drug that interacts with DNA and leads to the activation of tumor suppressor genes, which are genes that help control cell growth. This study may help the study doctors find out if the different drug combinations are equally effective to the usual approach of cytarabine alone while requiring a shorter duration of treatment. To decide if they are better, the study doctors will be looking to see if the study drugs lead to a higher percentage of patients achieving a deeper remission compared to cytarabine alone.

Start Date01 Jul 2026

Sponsor / Collaborator

National Cancer Institute

NCT06770257

/ WithdrawnPhase 2IIT

A Randomized Phase ll Study Evaluating the Efficacy and Safety of Combination Therapy of Daunorubicin, Cytarabine Liposomes and Venetoclax Versus Combination Therapy of Azacitidine and Venetoclax in Newly Diagnosed AML Patients Not Eligible for Intensive Chemotherapy

Daunorubicin, Cytarabine Liposomes(CSPC Zhongnuo Pharmaceutical (Shijiazhuang) Co., Ltd.) is a dual-drug liposomal encapsulation of cytarabine and daunorubicin at a fixed 5:1 synergistic molar ratio.
This is a phase 2, randomized, controlled study in patients who are >= 18 or more years old and have not been treated before. Participants who take part in this study should not be suitable for intensive induction therapy.
In this study, patients will be randomized by 1:1:1 to receive Daunorubicin, Cytarabine Liposomes + Venetoclax(14d) or Daunorubicin, Cytarabine Liposomes + Venetoclax(21d) or Venetoclax + Azacitidine.
Participants will continue to have study visits and receive treatment for as long as they are having a clinical benefit.

Start Date10 Dec 2025

Sponsor / Collaborator

CSPC ZhongNuo Pharmaceutical (Shijiazhuang) Co. Ltd.

NCT07008638

/ RecruitingPhase 1/2IIT

HM2024-29: Phase I/II Clinical Trial of Proteasome Inhibitor in Combination With CPX-351 for the Treatment of Newly-Diagnosed TP53-mutated Acute Myeloid Leukemia (AML)

This is a Phase I/II study evaluating safety and efficacy of proteasome inhibitor (bortezomib) in combination with CPX-351 (liposomal daunorubicin and cytarabine) for the treatment of newly-diagnosed TP53-mutated acute myeloid leukemia (TP53m AML).
The primary endpoint of the study is to define safety/tolerability (phase I) and preliminary efficacy profile (phase II) of the treatment. The secondary endpoints of interest are complete remission (CR) rate, detectable minimal residual disease (MRD) status, overall response rate (ORR), rate of allogeneic hematopoietic cell transplantation (allo-HCT), treatment-related mortality (TRM), overall survival (OS), achievement of complete remission anytime in 1 year, and disease-free survival (DFS) at 1 year and 2 years. All the patient outcomes assessments will be performed as part of standard-of-care AML management.
The hypothesis is the combination of bortezomib and CPX-351 will have an acceptable safety profile in this patient population based on the data from previous studies. The treatment will attenuate Nuclear Factor kB pathway activation in these cells and eradicate TP53m leukemia stem cells (LSC) leading to increased response rate and survival in these patients.

Start Date07 Jul 2025

Sponsor / Collaborator

University of Minnesota Masonic Cancer Center

100 Clinical Results associated with Cytarabine/Daunorubicin Liposomal

100 Translational Medicine associated with Cytarabine/Daunorubicin Liposomal

100 Patents (Medical) associated with Cytarabine/Daunorubicin Liposomal

261

Literatures (Medical) associated with Cytarabine/Daunorubicin Liposomal

31 Dec 2026·Hematology

Adverse karyotype amplifies risk in secondary acute myeloid leukemia (AML) and AML with myelodysplasia-related changes

Article

Author: Hayashi, Toshiaki ; Nagai, Jun ; Kakinoki, Yasutaka ; Igarashi, Tetsuya ; Teshima, Takanori ; Yoshida, Shota ; Onozawa, Masahiro ; Tsutsumi, Yutaka ; Ibata, Makoto ; Takahashi, Tomoki ; Hashimoto, Daigo ; Mori, Akio ; Adachi, Masaaki ; Oyake, Tatsuo ; Saga, Tomoyuki ; Yoshida, Masahiro ; Ota, Shuichi ; Matsukawa, Toshihiro ; Hashiguchi, Junichi ; Iyama, Satoshi ; Wakasa, Kentaro ; Fujimoto, Katsuya ; Yamamoto, Satoshi ; Fujii, Fumiaki ; Miyagishima, Takuto

BACKGROUND:

Secondary acute myeloid leukemia (sAML) and AML with myelodysplasia-related changes (AML-MRC) are associated with poor prognosis, but the impact relative to de novo AML remains controversial. We investigated clinical and genetic features in a multicenter Japanese cohort before CPX-351 approval.

METHODS:

We retrospectively analyzed 294 patients with newly diagnosed AML registered in the Hokkaido Leukemia Net between 2022 and 2023. Propensity score matching was used to adjust baseline variables. Genetic profiles were assessed in 160 matched patients.

RESULTS:

In the matched cohort, sAML/AML-MRC did not show inferior overall survival compared with non-sAML/non-AML-MRC (P = 0.90). Adverse karyotypes were the predominant determinant among sAML/AML-MRC. Among sAML/AML-MRC patients, adverse karyotypes were associated with poorer survival than those without adverse karyotypes (P = 0.0075). In contrast, non-sAML/non-AML-MRC groups did not affect survival regardless of adverse karyotypes (P = 0.51). Among 65 patients with ELN 2017 adverse-risk, those with TP53 mutations had markedly shorter survival than those with TP53 wild-type (P = 0.018).

CONCLUSIONS:

sAML/AML-MRC with an adverse karyotype had a dismal outcome. These findings provide a benchmark for risk stratification in the pre- CPX-351 era.

01 Jun 2026·EUROPEAN JOURNAL OF HAEMATOLOGY

Νοvel Therapies in High‐Risk Myelodysplastic Syndromes

Review

Author: Galanopoulos, Athanasios G. ; Papi, Christina

ABSTRACT:

Myelodysplastic syndromes/neoplasms (MDS) constitute a very heterogeneous group of clonal myeloid neoplasms characterized by a variable clinical course and recurrent genetic abnormalities. Their treatment relies on risk classification as lower or higher‐risk categories by the original International Prognostic Scoring System (IPSS) or the current revised (IPSS‐R) or molecular IPSS‐M. Higher‐risk MDS (HR‐MDS) are clonal hematopoietic disorders characterized by significant cytopenias, dysplastic changes, and a high propensity for progression to acute myeloid leukemia (AML). Up to 40% of them usually progress to AML within two years of diagnosis. Allogeneic stem cell transplantation (HSCT) remains the only potential cure and standard of care for eligible patients. Despite standard treatments such as Allo‐HSCT and hypomethylating agents (HMAs), outcomes remain suboptimal. Recent advances have led to the development of novel therapeutic strategies, such as BCL‐2 inhibitors (venetoclax), IDH1/2 inhibitors (ivosidenib, enasidenib), CD47 inhibitors (magrolimab), TIM‐3 inhibitors (sabatolimab), XPO1 inhibitors (eltanexor), NEDD8‐activating enzyme inhibitors (pevonedistat), TP53‐targeted agents (eprenetapopt), liposomal chemotherapy (CPX‐351), and oral HMA formulations. Combinations of hypomethylating agents with these new drugs, as first‐line treatment, have to date not proven more efficacious than HMA monotherapy. This review summarizes the current therapeutic landscape on novel therapies for HR‐MDS, highlighting their mechanism of action, efficacy, and demonstrates the unmet clinical need for more effective therapies.

01 May 2026·LEUKEMIA RESEARCH

Clinical strategies for leukemia management: Recommendations from the Bridging the Gaps in Hematology Oncology Consensus Conference 2025

Article

Author: Park, Jae H ; Perl, Alexander E ; Carraway, Hetty E ; Brunner, Andrew ; Wang, Eunice S ; DeAngelo, Daniel J ; Zeidner, Joshua F ; Luskin, Marlise R ; Komrokji, Rami S ; Lai, Catherine ; Zeidan, Amer ; Chandhok, Namrata S ; Weeks, Lachelle ; Loghavi, Sanam

Rapid progress in leukemia management has increased treatment complexity, resulting in clinical scenarios not fully addressed by standard guidelines. To provide expert guidance, the Bridging the Gaps in Hematology Consensus Conference reunited U.S.-based experts in acute myeloid leukemia (AML), myelodysplastic syndromes (MDS), and acute lymphoblastic leukemia (ALL) on February 25-26, 2025. Key consensus recommendations were established across AML, MDS, and ALL. For AML, consensus favored CPX-351 for fit patients with secondary or therapy-related AML, without access to clinical trials, and prioritized clinical trial enrollment for other specific molecular subtypes, including those eligible for menin inhibitors. In MDS, consensus supported moving to a harmonized World Health Organization/International Consensus Classification, diagnosing AML based on genetic markers in conjunction with blast counts, and prioritizing stem cell transplant (HSCT) for high-risk patients regardless of response to hypomethylating therapy, particularly for multi-hit TP53-mutated MDS. For ALL, recommendations highlighted avoiding transplant in most Philadelphia chromosome-positive patients achieving minimal residual disease (MRD)-negativity by noting the high relapse risk with certain IKZF1, CDKN2A/B, and PAX5 aberrations, and administering blinatumomab regardless of MRD status in Philadelphia chromosome-positive and -negative ALL. Areas lacking consensus were also identified, highlighting the need for further research. Future directions include refining treatment sequencing, improving outcomes for high-risk subtypes, evaluating the role of HSCT in the era of novel therapies, and standardizing MRD assessment. This consensus report provides valuable expert insights to inform clinical practice and guide future research in leukemia.

News (Medical) associated with Cytarabine/Daunorubicin Liposomal

24 Feb 2026

·www.prnewswire.com

Jazz Pharmaceuticals Announces Full Year and Fourth Quarter 2025 Financial Results and Provides 2026 Financial Guidance

– Record total revenues of $4.3 billion in 2025 (+5% YoY) and $1.2 billion (+10% YoY) in 4Q25 – – Expect to complete sBLA submission in 1Q26 under RTOR for zanidatamab in HER2+ 1L GEA – – Xywav® achieved $1.7 billion in revenue and 12% YoY growth in 2025 – – Epidiolex® achieved $1.1 billion in revenue and 9% YoY growth in 2025 – – Strong Modeyso™ launch with $37 million in revenue in first full quarter on market – – Expect 2026 total revenues of $4.25 to $4.50 billion – DUBLIN, Feb. 24, 2026 /PRNewswire/ -- Jazz Pharmaceuticals plc (Nasdaq: JAZZ) today announced financial results for the full year and fourth quarter of 2025 and provided financial guidance for 2026. "2025 was an exceptional year for Jazz, representing our 21st consecutive year of top-line growth and underscoring our commitment to operational excellence as we deliver meaningful innovation for patients," said Renee Gala, president and chief executive officer of Jazz Pharmaceuticals. "In the fourth quarter, our disciplined execution resulted in $1.2 billion in revenue, reflecting 10% year-over-year growth and our highest revenue quarter ever. This performance provides us with strong momentum into 2026, as we prepare for the potential launch of zanidatamab in GEA, sustain launch execution for Modeyso and Zepzelca®, and reinforce the differentiated profiles of Epidiolex and Xywav as the leading branded treatments for epilepsy and narcolepsy, respectively. In parallel, we continue to advance our pipeline and pursue a corporate development strategy aligned with our rare disease focus that supports durable growth and long-term value creation for patients and shareholders." "Jazz had a transformative year across our R&D pipeline, led by the HERIZON-GEA-01 data, which we believe firmly positions zanidatamab as the HER2-targeted agent of choice, with the potential to reshape first-line treatment for HER2+ metastatic GEA patients," said Rob Iannone, M.D., M.S.C.E., executive vice president, global head of research and development, and chief medical officer of Jazz Pharmaceuticals. "We expect to build on this progress in 2026, as these results not only highlight zanidatamab's potential to help patients with GEA, but also de-risk our clinical trials in additional indications, including HER2+ metastatic breast cancer." Key 2025 Highlights Total revenues in 2025 grew to $4.3 billion (+5% year-over-year (YoY)), generating $1.4 billion in cash from operations. Research & Development: Practice-changing Phase 3 HERIZON-GEA-01 results support zanidatamab as the HER2-targeted agent of choice in HER2+ 1L gastroesophageal adenocarcinoma (GEA), regardless of PD-L1 status. 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. Commercial: Continued leadership in rare sleep with Xywav net product sales increasing to $1.7 billion (+12% YoY) and total sleep franchise1 revenues exceeding $2 billion in 2025. Epidiolex/Epidyolex® generated more than $1 billion in 2025 net product sales (+9% YoY). Completed acquisition of Chimerix Inc., secured FDA approval for and successfully launched Modeyso (dordaviprone) in H3 K27M-mutant diffuse midline glioma, achieving $48 million in sales since launch in August 2025. Received FDA approval and launched Zepzelca, in combination with atezolizumab, for first-line maintenance treatment of extensive-stage small cell lung cancer. Company expects 2026 total revenue of between $4.25 and $4.50 billion, with double-digit growth across the combined epilepsy and oncology franchises, and Xywav revenue flat to up mid-single digits. Tom Riga was named chief business officer to accelerate corporate development efforts across rare disease. Business Updates Xywav (calcium, magnesium, potassium, and sodium oxybates) oral solution: Xywav net product sales increased 12% to $1.7 billion in 2025 and increased 16% to $465 million in 4Q25 compared to the same periods in 2024. Strong new patient growth continued, with approximately 500 net patient adds in 4Q25. There were approximately 16,175 active patients exiting the quarter, comprised of approximately 10,950 narcolepsy patients and approximately 5,225 idiopathic hypersomnia (IH) patients. Epidiolex/ Epidyolex (cannabidiol): Epidiolex/Epidyolex achieved blockbuster status in 2025 with net product sales increasing 9% to $1.1 billion in 2025 and increasing 4% to $287 million in 4Q25 compared to the same periods in 2024. Ziihera® (zanidatamab-hrii): Ziihera net product sales in biliary tract cancer (BTC) were $25 million in 2025 and $9 million in 4Q25 following product launch in December 2024. Expect to complete supplemental biologics license application (sBLA) submission under Real Time Oncology Review (RTOR) in 1Q26 with potential launch in 1L HER2+ GEA in 2H26. FDA granted Breakthrough Therapy designation (BTD) for zanidatamab's development for patients with HER2+ unresectable locally advanced or metastatic GEA. Submitted HERIZON-GEA-01 data for potential inclusion in National Comprehensive Cancer Network (NCCN) guidelines. EmpowHER-BC-303 trial in mBC patients previously treated with, or intolerant to, trastuzumab deruxtecan on track to complete enrollment in 1H27, with top-line results expected in late 2027 or early 2028. Modeyso (dordaviprone): Following product launch in August 2025, Modeyso net product sales were $48 million in 2025 and $37 million in 4Q25. The Company sold its Rare Pediatric Disease Priority Review Voucher for gross proceeds of $200 million (50% to Jazz). Zepzelca (lurbinectedin): Zepzelca net product sales decreased 4% to $307 million in 2025 and increased 15% to $90 million in 4Q25 compared to the same periods in 2024. Financial Highlights Reconciliations of applicable GAAP reported to non-GAAP adjusted information are included at the end of this press release. Total Revenues Total revenues increased 5% in 2025 and 10% in 4Q25 compared to the same periods in 2024. Total neuroscience revenue, including high-sodium oxybate AG royalty revenue, was $3.1 billion in 2025, an increase of 6% compared to $2.9 billion in 2024, and $848 million in 4Q25, an increase of 8% compared to $786 million in 4Q24. The increase in both periods was primarily due to higher Xywav and Epidiolex/Epidyolex net product sales, partially offset by decreased Xyrem net product sales. Oncology net product sales were $1.1 billion in 2025, an increase of 2% compared to 2024, and $337 million in 4Q25, an increase of 16% compared to $292 million in 4Q24, primarily due to the inclusion of Modeyso and Ziihera net product sales in both periods. The increase in 4Q25 also included higher Zepzelca net product sales, partially offset by decreased Vyxeos® net product sales. Operating Expenses and Income Tax (Benefit) Expense Changes in operating expenses and income tax (benefit) expense in 2025 and 4Q25 over the prior year periods are primarily due to the following: Cost of product sales, on a GAAP and non-GAAP adjusted basis, increased in 2025 compared to 2024, primarily due to changes in product mix. Cost of product sales, on a GAAP basis, in 2025 included higher acquisition accounting inventory fair value step up expense compared to 2024. Cost of product sales, on a GAAP and non-GAAP adjusted basis, increased in 4Q25 compared to 4Q24, primarily due to changes in product mix, partially offset by lower inventory provisions. Selling, general and administrative (SG&A) expenses, on a GAAP and non-GAAP adjusted basis, increased in 2025 compared to 2024, primarily due to Xyrem antitrust litigation settlements of $234 million, the Avadel litigation settlement of $90 million and higher compensation-related expenses. SG&A expenses, on a GAAP and non-GAAP adjusted basis, increased in 4Q25 compared to 4Q24, primarily due to higher compensation-related expenses. Research and development (R&D) expenses, on a GAAP and non-GAAP adjusted basis, decreased in 2025 and 4Q25, compared to the same periods in 2024, primarily due to lower clinical study costs primarily related to zanidatamab as a result of timing of clinical trial activities, JZP385 (essential tremor) following discontinuation of this program, and JZP258 (XYLO/DUET) due to the completion of these trials in the first half of 2025, partially offset by the addition of costs relating to Modeyso and increased personnel costs following the acquisition of Chimerix. Acquired in-process research and development (IPR&D) in 2025, on a GAAP and non-GAAP adjusted basis, represents the value allocated to Modeyso in the Chimerix Acquisition of $905 million and the upfront payment made in connection with our global license agreement with Saniona of $43 million. Income tax benefit in 2025, on a GAAP and non-GAAP adjusted basis, included a benefit of $213 million on recognition of certain U.S. federal and state deferred tax assets acquired through the Chimerix acquisition. Income tax benefit, on a GAAP and non-GAAP adjusted basis, in 4Q24 was primarily due to patent box benefits recognized. Cash Flow and Balance Sheet As of December 31, 2025, cash, cash equivalents and investments were $2.4 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 year ended December 31, 2025, the Company generated $1.4 billion of cash from operations reflecting strong business performance and continued financial discipline. 2026 Financial Guidance Jazz Pharmaceutical's full year 2026 financial guidance is as follows: Conference Call Details Jazz Pharmaceuticals will host an investor conference call and live audio webcast today at 4:30 p.m. ET (9:30 p.m. GMT) to provide a business and financial update and discuss its 2025 full year and 4Q25 results and 2026 guidance. 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. In this regard, commencing with the first quarter of 2025, the Company is no longer including an adjustment for non-cash interest expense in the Company's non-GAAP adjusted financial measures. For purposes of comparability, non-GAAP adjusted financial measures for the 2024 periods have been updated to reflect this change. 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; anticipated multiple near-term pipeline catalysts that each represent significant opportunities to drive greater revenue and create long-term value; the Company's advancement of pipeline programs and the timing of development activities, regulatory activities, approvals, and submissions related thereto, including the timing of the completion of the submission of the sBLA for, and launch and approval of, zanidatamab in 1L GEA; planned or anticipated clinical trial events, including with respect to initiations, enrollment and data read-outs, and the anticipated timing thereof; 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 be the HER2-targeted agent of choice in HER2+ 1L GEA, regardless of PD-L1 status, and to reshape first-line treatment for HER2+ metastatic GEA patients; 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 first-line 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 the Company's sBLA submission for zanidatamab in 1L GEA may not be completed or, if completed, 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; 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 Logo - 21% more press release views with Request a Demo

Drug ApprovalBreakthrough TherapyPhase 3AcquisitionFinancial Statement

08 Jan 2026

·www.biospace.com

AB Science reports fourth consecutive case of response from Phase 1 data for the combination of AB8939 with venetoclax for the treatment of refractory or relapsed acute myeloid leukemia

PRESS RELEASE AB SCIENCE REPORTS FOURTH CONSECUTIVE CASE OF RESPONSE FROM PHASE 1 DATA FOR THE COMBINATION OF AB8939 WITH VENETOCLAX FOR THE TREATMENT OF REFRACTORY OR RELAPSED ACUTE MYELOID LEUKEMIA The combination treatment was well-tolerated, with no hematological toxicity and no dose limiting toxicity The fourth patient had a complex karyotype including a monosomy of chromosome 5 and TP53 mutation, and was in the third-line of treatment. He had a near complete response after 14 days of treatment with AB8939 at 21 mg/m 2 plus venetoclax This is the fourth patient responding to the combination from a total of 4 patients treated The partial response rate is 100% (4/4), including one patient in complete remission, one near complete response and 2 partial responses The results were obtained after the first cycle of treatment (14 days) in patients receiving third- or fourth-line treatment, two of whom had previously progressed on venetoclax in combination with other chemotherapies These four patients all have very difficult to treat cytogenetic pro including complex karyotype, TP53 mutation, NRAS mutation, monosomy 5 and MECOM-rearrangement, that typically have a poor prognosis due to their aggressive disease course and treatment resistance This diversity of responsive patients appears to corroborate the mechanism of action of AB8939, which is capable of destabilizing microtubules while evading multi-drug resistance and also targeting cancer stem cells without eliminating non tumoral stem cells These results corroborate the positioning of AB8939 in patients with adverse genetics, complex karyotypes, TP53 mutations, NRAS and KRAS mutations, monosomy 5 and 7, and MECOM-rearrangement, which represents the highest unmet medical need. Paris, 07 January 2026, 6pm CET AB Science SA (Euronext - FR0010557264 - AB) today provides an update on the Phase 1 study of the molecule AB8939 and the fourth consecutive response with the combination of AB8939 + venetoclax in patients with acute myeloid leukemia (AML) associated with a very unfavorable genetic profile. The fourth patient received AB8939 (21.3 mg/m²) plus venetoclax for 14 days. The patient had AML with a very negative risk profile, complex karyotype including a monosomy of chromosome 5 and also an identified TP53 mutation in third-line of treatment, having progressed after CPX-351 treatment and Citarine + Idarrubincine + Fludarabine (3+7) regimen. Under the AB8939 + venetoclax combination, the patient achieved a partial response. This fourth result is consistent with the responses previously reported on October 14, 2025, in the first three patients who received AB8939 + venetoclax. Nicholas J. Short, MD, Associate Professor and Co-Lead of the Section of Developmental Therapeutics, Department of Leukemia, MD Anderson Cancer Center, said, " This new data is very encouraging, particularly considering the very adverse risk pro this patient’s leukemia. These early efficacy and safety data suggest that AB8939 can be combined with venetoclax and could have significant activity in the highest-risk subtypes of AML. There is a strong interest in continuing the development of this combination in patients whose AML has high-risk features that are expected to lead to resistance to venetoclax + azacitidine. " Professor Olivier Hermine, MD, President of the Scientific Committee of AB Science and member of the Académie des Sciences in France, said, " There is a strong rationale to combine AB8939 and venetoclax as both molecules have low hematologic toxicity and complementary mode of actions. These first results are supportive of this rationale." About AB8939 AB8939 is a drug candidate that targets (i) cancer cells by destabilizing microtubules (essential for cell division) and (ii) cancer stem cells by inhibiting ALDH1A1 and ALDH2 (enzymes essential for maintaining their physiological state and survival). AB8939 has shown in vitro activity in Ara-C (cytarabine, which is one of the standards of care) resistant patient cell lines, including adverse genetic MECOM and TP53 mutations. Analysis of cell lines responsive to AB8939 showed that AB8939 is effective in cell lines with TP53 mutations, MECOM, and complex karyotypes, whereas ARAC and azacitidine are not effective. AB8939 increased survival and had an additive effect in combination with venetoclax (another standard of care) in vivo in a MECOM-grafted PDX mouse model. AB8939 increased survival and had an additive effect in combination with Vidaza (azacitidine, another standard of care) in vivo in the MECOM PDX#C1005 mouse model of leukemia. AB8939 eradicated Leukemia Cancer Stem Cells in vivo in a human PDX AML mouse model, which is compatible with targeting stem cells via ALDH. AB8939 is currently being evaluated in a Phase 1 clinical trial (study AB18001, NCT05211570) in patients with refractory and relapsed AML. The Phase 1 clinical trial of AB8939 has completed its first two stages, which consisted of determining the maximum tolerated dose (MTD) after 3 and 14 consecutive days of monotherapy. In both cases, the MTD was 21.3 mg/m². The third stage, currently underway, involves evaluating the combination of AB8939 and venetoclax. Three patients were evaluated at the first dose level (AB8939 14 days at a dose of 16 mg/m² + venetoclax 14 days). The current dose level evaluates (AB8939 14 days at a dose of 21.3 mg/m² + venetoclax 14 days) Medical need in AML and AB8939 mechanism of action Although several drugs have been registered for AML, 70% of patients still relapse and die, creating a persistent unmet medical need for effective treatments. Acute myeloid leukemia remains the most lethal form of leukemia in humans. AML is a heterogeneous disease, and its outcome is highly dependent on genetic factors. TP53 mutation has a very poor prognosis, with a median overall survival (OS) of 5.5 months. NRAS and KRAS mutants have a poor prognosis, with a median OS of 12.1 months. MECOM also has a very poor prognosis in AML, with a median OS of 5.5 months in relapsed or refractory settings. The challenge in AML is the recurrence of tumors due to a combination of two factors: the resistance of cancer cells to chemotherapy and relapse due to the persistence of cancer stem cells. This challenge may be overcome by AB8939’s dual mechanism of action. First, AB8939 blocks the proliferation of leukemia cells through microtubule disruption. It is not subject to multi-drug resistance as it does not bind to PgP, which is responsible for efflux outside the cells, and is not degraded by myeloperoxidase. Second, AB8939 targets leukemia cancer stem cells by inhibiting ALDH and promotes bone marrow repopulation of normal progenitors. AB8939 + venetoclax combination There is a strong rationale to combine AB8939 with venetoclax Both molecules exhibit low hematologic toxicity. This combination is expected to be less toxic than azacitidine + venetoclax as first-line treatment for AML These molecules have different and complementary targets in cancer cells. There is an additive, even synergistic, efficacy potential for the combination, with three mechanisms of action in a single treatment. Venetoclax’s mechanism of action inhibits the BCL2 pathway, a protein that prevents apoptosis (programmed cell death) in cancer cells. BCL2 is a key factor in AML resistance, as it allows cancer cells to survive despite treatment AB8939 is pro-apoptotic, destabilizing microtubules, and would benefit from BCL2 inhibition to optimize apoptosis In addition, AB8939 specifically targets cancer stem cells by inhibiting ALDH, reducing resistance to treatment and limiting the risk of relapse Next steps The next step is to complete phase 1 in combination and launch an expansion study in approximately 15 AML patients eligible for AB8939 + venetoclax at the appropriate dose. The expansion phase is expected to generate robust preliminary evidence of efficacy in the AML label, sufficient to support the clinical development plan and a beneficial partnership agreement. AB Science has started to discuss three possibilities for registration studies, which are not mutually exclusive, with the European Medicines Agency (EMA) and US Food and Drug Administration (FDA): AB8939 + venetoclax as first-line treatment, with aged patients and/or patients with adverse genetics (complex karyotypes, TP53 mutations, NRAS and KRAS mutations, monosomy 5 and 7, and MECOM-rearrangement) AB8939 + venetoclax as a second- or third-line treatment, in all patients or patients with adverse genetics AB8939 as a single agent in MECOM as a second or third-line treatment. Addressable market with AB8939 in relapsed/refractory AML Treatments for relapsed or refractory AML represent an estimated market size potential of greater than EUR 2 billion per annum. Region Incidence Case (1) % Relapse or Refractory (2,3) % Insured Patients (4) Drug Price (€) Market Size (per in Mio EUR) USA / CANADA 23,700 50% 90% 100,000 (5) 1 000 000 EUROPE 27,600 90% 60,000 770 000 APAC 27,800 30% 60,000 250 000 INDIA 11,000 30% 60,000 100,000 LATAM 7,200 30% 60,000 65 000 MENA 3,900 30% 60,000 35 000 TOTAL 90,200 2 200 000 EUROPE = EU27 + Norway + United Kingdom + Switzerland ; APAC = Australia, People’s Republic of China , Japan, New Zealand, Singapore, Taiwan ; LATAM = Argentina, Brazil, Chile, Colombia, Costa Rica, Mexico ; MENA = Algeria, Bahrain, Egypt, Israel, Kuwait, Morocco, Oman, Qatar, Saudi Arabia, Tunisia, United Arab Emirates (1) Zhou, Y et al. Global, regional, and national burden of acute myeloid leukemia, 1990–2021: a systematic analysis for the global burden of disease study 2021. Biomark Res 12, 101 (2024). (2) Ravandi F. Relapsed acute myeloid leukemia: Why is there no standard of care Best Pract Res Clin Haematol. 2013;26(3):253-9 (3) Walter RB et al. Resistance prediction in AML: analysis of 4601 patients from MRC/NCRI, HOVON/SAKK, SWOG and MD Anderson Cancer Center. Leukemia (2015) 29:312–20. . (4) Estimated (5) Choi M. et al. Costs per patient achieving remission with venetoclax-based combinations in newly diagnosed patients with acute myeloid leukemia ineligible for intensive induction chemotherapy. Journal of Managed Care & Specialty Pharmacy Volume 28, Number 9. Intellectual property AB8939 intellectual property rights in AML are secured until 2036 through a ‘composition of matter’ patent and potentially until 2041 with a 5 years extension. Two additional ‘second medical use’ patent applications have been filed to protect the use of AB8939 in the treatment of AML with specific chromosomal abnormalities. If these applications are accepted, the protection for AB8939 will be extended until 2044 and 2046 for these AML subpopulations. AB8939 has also received orphan drug designation for AML by both the EMA and FDA. This orphan drug designation confers 10 and 7 years of marketing exclusivity in Europe and the US, respectively, from the date of product registration. AB Science is the sole proprietary holder of AB8939 and its family of compounds. About AB Science Founded in 2001, AB Science is a pharmaceutical company specializing in the research, development and commercialization of protein kinase inhibitors (PKIs), a class of targeted proteins whose action are key in signaling pathways within cells. Our programs target only diseases with high unmet medical needs, often lethal with short term survival or rare or refractory to previous line of treatment. AB Science has developed a proprietary portfolio of molecules and the Company’s lead compound, masitinib, has already been registered for veterinary medicine and is developed in human medicine in oncology, neurological diseases, inflammatory diseases and viral diseases. The company is headquartered in Paris, France, and listed on Euronext Paris (ticker: AB). Further information is available on AB Science’s website: . Forward-looking Statements - AB Science This press release contains forward-looking statements. These statements are not historical facts. These statements include projections and estimates as well as the assumptions on which they are based, statements based on projects, objectives, intentions and expectations regarding financial results, events, operations, future services, product development and their potential or future performance. These forward-looking statements can often be identified by the words "expect", "anticipate", "believe", "intend", "estimate" or "plan" as well as other similar terms. While AB Science believes these forward-looking statements are reasonable, investors are cautioned that these forward-looking statements are subject to numerous risks and uncertainties that are difficult to predict and generally beyond the control of AB Science and which may imply that results and actual events significantly differ from those expressed, induced or anticipated in the forward-looking information and statements. These risks and uncertainties include the uncertainties related to product development of the Company which may not be successful or to the marketing authorizations granted by competent authorities or, more generally, any factors that may affect marketing capacity of the products developed by AB Science, as well as those developed or identified in the public documents published by AB Science. AB Science disclaims any obligation or undertaking to update the forward-looking information and statements, subject to the applicable regulations, in particular articles 223-1 et seq. of the AMF General Regulations. For additional information, please contact: AB Science Financial Communication & Media Relations investors@ab-science.com Attachment CP AB8939 JAN 26 VENG VF

Clinical ResultPhase 1Orphan Drug

18 Dec 2024

·www.globenewswire.com

Akari Therapeutics Announces Key Leadership Appointments

Samir R. Patel, M.D., appointed as Chief Executive Officer Abizer Gaslightwala appointed to Board of Directors BOSTON and LONDON, Dec. 18, 2024 (GLOBE NEWSWIRE) -- Akari Therapeutics, Plc (Nasdaq: AKTX) today announced the appointment of Samir R. Patel, M.D., as Chief Executive Officer, effective December 16, 2024. Dr. Patel has served as interim Chief Executive Officer since May 2024. Additionally, the Company announced it has appointed Abizer Gaslightwala to its Board of Directors, effective December 16, 2024. Michael Grissinger, a member of the Board of Directors, provided notice of his resignation on December 16, 2024. “Since beginning my role as interim CEO in May 2024, we have worked diligently to close the merger with Peak Bio, which we accomplished in November, and position Akari for continued growth and value creation. Looking ahead, we are focusing our efforts and resources to streamline operations and execute on our portfolio prioritization – our potentially best-in-class ADC platform. I am pleased to solidify my role with the Akari team and look forward to building momentum and driving shareholder value in the near and long term,” commented Dr. Patel, Chief Executive Officer of Akari. About Samir R. Patel, M.D. Dr. Patel currently serves as founder and principal of PranaBio Investments, LLC, a firm providing consulting, strategic advisory, and investment services for small cap biotechnology companies. He is also a consultant to GE Global Research, GE’s innovation engine that is creating novel products and solutions across several sectors including biomanufacturing and biotechnology. He has more than 20 years of experience in life sciences including founding SPEC Pharma, LLC, a company that develops and manufactures injectables used in human and veterinary applications. Previously, Dr. Patel held multiple roles in Medical Affairs with Centocor, Inc. (now Johnson & Johnson Innovative Medicine, part of Johnson & Johnson). He holds multiple patents, has been an author on several publications and has been an investigator in numerous clinical research studies. He has served on the boards of several public companies, including Rezolute Bio. Dr. Patel received his medical degree from the Medical College of Ohio and completed his internal medicine internship, residency, and rheumatology fellowship at The University of New Mexico School of Medicine Affiliated Hospitals. About Abizer Gaslightwala Mr. Gaslightwala is a well-established biotechnology / pharmaceutical industry leader with a demonstrated track record of success spanning over 25 years in the development and commercialization of novel medicines across a range of companies and therapeutic areas. Mr. Gaslightwala currently serves as the Senior Vice President and Franchise Head for Oncology at Jazz Pharmaceuticals, where he has full responsibility for a portfolio of products spanning both solid and hematological malignancies that have total annual sales of $1B. These brands include ZIIHERA® for HER2+ cancers, ZEPZELCA® for small cell lung cancer, and RYLAZE®, DEFITELIO®, and VYXEOS® for a range of hematological malignancies. “We are pleased to welcome Abizer to our Board of Directors. We believe the depth and breadth of his development and commercialization leadership and expertise amassed over the course of his career will provide valuable insight as we work to propel Akari to its next phase of growth. Additionally, we would like to thank Michael for his years of service on our board. His perspective and guidance have played a key role in getting the Company to where we are today,” added Dr. Patel. Mr. Gaslightwala added, “I'm thrilled to join the Akari board and have been extremely compelled by the Company’s unique ADC platform targeting the spliceosome, that has the potential to set a new standard of care for cancer patients. I look forward to working closely with the management team and other members of the board to advance this platform forward and potentially address areas of significant unmet need for cancer patients.” Prior to his current position at Jazz Pharmaceuticals, Mr. Gaslightwala has led and driven growth in several leadership roles at Amgen, Pfizer, and Johnson & Johnson across multiple brands including Kyprolis®, Vectibix®, Neulasta®, XGEVAf®, Repatha®, and ELIQUIS®. His experience spans business unit leadership, brand marketing, sales leadership, commercial pipeline planning, advanced analytics and insights, and business development. Mr. Gaslightwala also helped lead R&D strategic planning within the autoimmune/inflammation portfolio at Johnson & Johnson, as well as lead commercial planning for Remicade® and several novel pipeline molecules focused on rheumatoid arthritis, inflammatory bowel disease, psoriasis, and atopic dermatitis. Additionally, Mr. Gaslightwala advised several life science companies through his time at the Boston Consulting Group (BCG). Mr. Gaslightwala holds a BS in Chemical Engineering from Cornell University, and an MBA from the Sloan School of Management, and a MS in Chemical Engineering from MIT. About Akari Therapeutics Akari Therapeutics, Plc (Nasdaq: AKTX) is a biotechnology company developing advanced therapies for autoimmune, oncology and inflammatory diseases. Akari has two lead assets, investigational nomacopan and an antibody drug conjugate (ADC) platform. Nomacopan is a bispecific recombinant inhibitor of complement C5 activation and leukotriene B4 (LTB4) activity. The Company’s ADC platform includes novel toxins and linkers coupled with important cancer antibody targets. For more information about Akari, please visit akaritx.com. Cautionary Note Regarding Forward-Looking Statements This press release includes express or implied forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended (the Exchange Act), about the Company that involve risks and uncertainties relating to future events and the future performance of the Company. Actual events or results may differ materially from these forward-looking statements. Words such as “will,” “could,” “would,” “should,” “expect,” “plan,” “anticipate,” “intend,” “believe,” “estimate,” “predict,” “project,” “potential,” “continue,” “future,” “opportunity” “will likely result,” “target,” variations of such words, and similar expressions or negatives of these words are intended to identify such forward-looking statements, although not all forward-looking statements contain these identifying words. Examples of such forward-looking statements include, but are not limited to, express or implied statements regarding: the business combination and related matters, including, but not limited to, post-closing operations and the outlook for the Company’s business; the Company’s targets, plans, objectives or goals for future operations, including those related to its product candidates; financial projections; future economic performance,; and the assumptions underlying or relating to such statements. These statements are based on the Company’s current plans, estimates and projections. By their very nature, forward-looking statements involve inherent risks and uncertainties, both general and specific. A number of important factors, including those described in this communication, could cause actual results to differ materially from those contemplated in any forward-looking statements. Factors that may affect future results and may cause these forward-looking statements to be inaccurate include, without limitation: the risk that Akari and Peak Bio may not realize the anticipated benefits of the Merger in the time frame expected, or at all; the ability to retain and hire key personnel; potential adverse reactions or changes to business relationships resulting from the Merger; the potential impact of unforeseen liabilities, future capital expenditures, revenues, costs, expenses, earnings, synergies, economic performance, indebtedness, financial condition and losses on the future prospects, business and management strategies for the management, expansion and growth of the combined business; uncertainties as to the long-term value of Akari’s American Depositary Shares (and the ordinary shares represented thereby), including the dilution caused by Akari’s issuance of additional American Depositary Shares (and the ordinary shares represented thereby) in connection with the Merger; risks related to global as well as local political and economic conditions, including interest rate and currency exchange rate fluctuations; potential delays or failures related to research and/or development of the Company’s programs or product candidates; risks related to any loss of the Company’s patents or other intellectual property rights; any interruptions of the supply chain for raw materials or manufacturing for the Company’s product candidates, the nature, timing, cost and possible success and therapeutic applications of product candidates being developed by the Company and/or its collaborators or licensees; the extent to which the results from the research and development programs conducted by the Company, and/or its collaborators or licensees may be replicated in other studies and/or lead to advancement of product candidates to clinical trials, therapeutic applications, or regulatory approval; uncertainty of the utilization, market acceptance, and commercial success of the Company’s product candidates; unexpected breaches or terminations with respect to the Company’s material contracts or arrangements; risks related to competition for the Company’s product candidates; the Company’s ability to successfully develop or commercialize its product candidates; potential exposure to legal proceedings and investigations; risks related to changes in governmental laws and related interpretation thereof, including on reimbursement, intellectual property protection and regulatory controls on testing, approval, manufacturing, development or commercialization of any of the Company’s product candidates; the Company’s ability to maintain listing of its ADSs on the Nasdaq Capital Market. While the foregoing list of factors presented here is considered representative, no list should be considered to be a complete statement of all potential risks and uncertainties. More detailed information about the Company and the risk factors that may affect the realization of forward-looking statements is set forth in the Company's filings with the SEC, copies of which may be obtained from the SEC's website at www.sec.gov. The Company assumes no, and hereby disclaims any, obligation to update the forward-looking statements contained in this press release. Investor Contact: JTC Team, LLCJenene Thomas908.824.0775AKTX@jtcir.com A photo accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/110aebf9-2683-41c9-ba9d-bf06f7e42054

Executive Change

100 Deals associated with Cytarabine/Daunorubicin Liposomal

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R&D Status

Approved

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Indication	Country/Location	Organization	Date
Treatment related acute myeloid leukaemia	European Union	Jazz Pharmaceuticals Ireland Ltd.	23 Aug 2018
Treatment related acute myeloid leukaemia	Iceland	Jazz Pharmaceuticals Ireland Ltd.	23 Aug 2018
Treatment related acute myeloid leukaemia	Liechtenstein	Jazz Pharmaceuticals Ireland Ltd.	23 Aug 2018
Treatment related acute myeloid leukaemia	Norway	Jazz Pharmaceuticals Ireland Ltd.	23 Aug 2018
Acute Myeloid Leukemia	United States	Jazz Pharmaceuticals, Inc.	03 Aug 2017
Acute Myeloid Leukemia with Myelodysplasia-Related Changes	United States	Jazz Pharmaceuticals, Inc.	03 Aug 2017

Developing

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Indication	Highest Phase	Country/Location	Organization	Date
High Risk Myelodysplastic Syndrome	Phase 3	United States	Jazz Pharmaceuticals Plc	13 Dec 2012
High Risk Myelodysplastic Syndrome	Phase 3	Canada	Jazz Pharmaceuticals Plc	13 Dec 2012
Acute Myeloid Leukemia with FLT3/ITD Mutation	Phase 2	United States	Jazz Pharmaceuticals Plc	05 Jul 2022
Acute Myelomonocytic Leukemia	Phase 2	United States	Jazz Pharmaceuticals, Inc.	30 Jan 2022
Acute Myelomonocytic Leukemia	Phase 2	United States	The University of Texas MD Anderson Cancer Center	30 Jan 2022
Residual Neoplasm	Phase 2	United States	Jazz Pharmaceuticals Plc	09 Aug 2021
Acute myeloid leukaemia with 11q23 abnormality	Phase 2	United States	The University of Texas MD Anderson Cancer Center	30 Dec 2020
IDH1 Mutation Acute Myeloid Leukemia	Phase 2	United States	The University of Texas MD Anderson Cancer Center	30 Dec 2020
Myeloproliferative Disorders	Phase 2	United States	The University of Texas MD Anderson Cancer Center	30 Dec 2020
Myeloid Tumor	Phase 2	United States	Jazz Pharmaceuticals Plc	27 May 2020

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04195945 (CTgov)	Phase 2	Myeloid Tumor \| Acute Myeloid Leukemia	60	CPX-351 (CPX-351 Induction Therapy)	yknbncmsch = wzbtvvsbsm khcimnvgqw (sydqphnjbo, feigvtekdu - pfevvffgmh) View more	-	14 May 2026
				CLAG-M+cladribine (CLAG-M Induction Therapy)	yknbncmsch = sdnafyerlo khcimnvgqw (sydqphnjbo, iiqvrwlxcl - xzllvlqmbz) View more
EHA2026	Not Applicable	Acute myeloid leukemia with mutated NPM1 NPM1 mutation	59	CPX-351 (NPM1-mutated AML)	rsmaoqynep(kqhsidfkls) = mfcsjgtwqi rcwpxeqzkn (gfojoazeqp ) View more	Positive	12 May 2026
EHA2026	Not Applicable	Treatment related acute myeloid leukaemia \| Acute Myeloid Leukemia with Myelodysplasia-Related Changes	75	CPX-351	civzqtfxdt(onshuqtivz) = oazlvvxxnh eccgkyxrwt (dttkxhlene, 47.1 - 85.7) View more	Negative	12 May 2026
				standard 7+3	civzqtfxdt(onshuqtivz) = wkzklpknbq eccgkyxrwt (dttkxhlene, 51.5 - 78.6) View more
EHA2026	Not Applicable	Acute Myeloid Leukemia	39,649	CPX-351	lcvyofbnfr(nsanmzyndd) = irvfakxmht knhakrrund (ofhjjbkvor ) View more	Positive	12 May 2026
				CYTARABINE	ppcjnoeoak(mdtidahupu) = wezvkbuiez yhzirjnvtb (scgfdeybcu ) View more
EHA2026	Not Applicable	Myeloid Leukemia \| Myelodysplastic Syndromes TP53 mutated	104	CPX-351	wlfzxvqscv(oazhueyubs) = mlrnkbukre wkddritshc (grrflgwpfi )	Positive	12 May 2026
EHA2026	Not Applicable	Acute Myeloid Leukemia	22	CPX-351	knkxvrkosw(zgsxyptysx) = liydyiqnju zosyejkipp (vqutochips ) View more	Negative	12 May 2026
EHA2026	Not Applicable	Acute Myeloid Leukemia	22	CPX-351	dwuehteufn(dteqbocgfi) = jnifrfrwje qhqgdvlrak (gdarafpubh ) View more	Positive	12 May 2026
				CPX-351 (CR MRD-)	llhgxnwray(jxfrppuidh) = vdfgdueqtz ownbveybfw (fesjzxxsnm ) View more
NCT03878199 (CTgov)	Phase 1/2	Polycythemia Vera \| Myelofibrosis \| Thrombocythemia, Essential	12	Ruxolitinib (Phase 1 Dose Level 1 (10mg Ruxolitinib))	dpcabdbzzs = xbhyafytso hbswbylndl (sleqqqzjro, cbiiodhgij - aohytcnlmt) View more	-	24 Apr 2026
				Ruxolitinib (Phase 1 Dose Level 2 (20mg Ruxolitinib))	dpcabdbzzs = qhrstghgql hbswbylndl (sleqqqzjro, hhkqwtnbpf - dvotrydgoz) View more
NCT04231851 (UCHMC 1913, CTgov)	Phase 2	Myelodysplastic Syndromes \| Acute Myeloid Leukemia with Myelodysplasia-Related Changes	30	Glasdegib+CPX-351	iktkjpexhh = yagrlpgybi otklssutqb (bzsivknidn, zgsgguukef - jgojdzbtcx) View more	-	06 Mar 2026
NCT02272478 (NCRI AML18, Pubmed \| Blood)	Phase 3	Acute Myeloid Leukemia	439	CPX-351	selunzutbm(evwkqxokhc) = ymozeutumg ttgtekxlkb (fxfkifxcun ) View more	Positive	05 Mar 2026
				Daunorubicin+cytarabine+gemtuzumab ozogamicin	selunzutbm(evwkqxokhc) = eknacaqoqz ttgtekxlkb (fxfkifxcun ) View more

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