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 Mar 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

255

Literatures (Medical) associated with Cytarabine/Daunorubicin Liposomal

01 Apr 2026·CURRENT OPINION IN PHARMACOLOGY

CPX-351: From preclinical studies to future directions

Review

Author: Ciotti, G ; Gottardi, M ; Basso, M ; Sperotto, A

For decades, the standard induction treatment for patients with acute myeloid leukemia (AML) has been the 7 + 3 regimen (cytarabine plus daunorubicin). The US-Food and Drug Administration (FDA) approved, in August 2017, the use CPX-351 for adults with newly diagnosed secondary AML. CPX-351 (also known as VYXEOS) is a dual-drug liposomal encapsulation of cytarabine and daunorubicin in a synergistic 5:1 M ratio, and CombiPlex was utilized, a combination drug technology platform. In this review, we analyze the path from preclinical studies to drug registration. Recent research highlights intestinal and heart toxicity of the drug, as well as current and potential future uses for CPX-351.

01 Mar 2026·LEUKEMIA RESEARCH

CPX-351 versus venetoclax plus hypomethylating agents for newly diagnosed acute myeloid leukemia: A systematic review and meta-analysis

Article

Author: Alabbas, Fahad ; Fero, Henri ; Miyashita, Akihiro ; Basendwah, Abdulrahim Mohammed

Acute myeloid leukemia (AML) predominantly affects older adults, with median age at diagnosis being 68-70 years. Selecting an appropriate regimen is important for older patients. CPX-351 demonstrated superior overall survival (OS) compared to 7 + 3 chemotherapy, while venetoclax and azacitidine significantly improved OS and composite remission rate compared to azacitidine alone. However, the optimal regimen in real-world practice remains uncertain. We performed a systematic review and meta-analysis comparing CPX-351 and the combination of venetoclax and hypomethylating agents (Ven/HMA) in newly diagnosed AML. We systematically searched PubMed, Scopus, and Cochrane Central Register of Controlled Trials from inception to January 2026. Eleven retrospective studies comprising 1852 patients comparing CPX-351 and Ven/HMA were included. The weighted mean of median age was 63.5 years in the CPX-351 cohort and 73 years in the Ven/HMA cohort. CPX-351 did not significantly improve OS (hazard ratio [HR] 0.89; 95 % CI 0.76-1.04; p = 0.1486) with median OS being 13.1 months versus 11.6 months in Ven/HMA cohort. There were no differences in the rates of composite remission rate (48.3 % vs 48.4 %; odds ratio [OR] 0.83; 95 % CI 0.58-1.18; p = 0.295), minimal residual disease negative remission (13.5 % vs 33.9 %; OR 0.61; 95 % CI 0.25-1.49; p = 0.281), 30-day mortality, and 60-day mortality. In conclusion, CPX-351 did not demonstrate superiority over Ven/HMA in composite remission rate and OS. Both regimens remain reasonable therapeutic options for older, newly diagnosed AML patients, and prospective comparative studies are needed to better guide treatment selection.

15 Jan 2026·BLOOD

CPX-351 in Down syndrome–associated myeloid leukemia: results and prognostic factors from the phase 3 ML-DS 2018 trial

Article

Author: Schuschel, Konstantin ; Gonçalves-Dias, José ; Klusmann, Jan-Henning ; Diederichs, Antonia ; Thol, Felicitas ; Laszig, Stephanie ; Kreyenberg, Hermann ; Kerp, Helena ; Pawińska-Wąsikowska, Katarzyna ; Issa, Hasan ; Miladinovic, Milica ; Goemans, Bianca F. ; Waack-Buchholz, Katharina ; Reinhardt, Dirk ; Boztug, Heidrun ; Salzmann-Manrique, Emilia ; Scheidegger, Nastassja ; Bremm, Melanie ; Rettinger, Eva ; De Moerloose, Barbara ; Wehner, Sibylle ; Hünecke, Sabine

Abstract:

Myeloid leukemia of Down syndrome (ML-DS) is associated with an excellent prognosis but high treatment-related toxicity and mortality. The Phase 3 Clinical Trial for CPX-351 in ML-DS 2018 aimed to maintain the excellent event-free survival (EFS) achieved in the previous ML-DS 2006 trial while reducing the treatment intensity. Intensity-reduced induction and reinduction therapy with cytarabine and idarubicin with or without etoposide was replaced with CPX-351 (66 U/m2 on 3 days in course 1 and on 2 days in course 2). Risk stratification was based on flow cytometric measurable residual disease (MRD) after first induction. High-risk patients received high-dose cytarabine (3 g/m2 per 12 hour) in consolidation; standard-risk patients received cytarabine at a dose of 1 g/m2 per 12 hour. A total of 35 patients were enrolled until the trial was halted because of an unexpectedly high relapse rate. A per-protocol interim analysis revealed a significantly lower 24-month EFS when compared with the ML-DS 2006 trial (69% vs 90%; P< .001). In contrast with previous studies, most patients who relapsed responded to salvage therapy, leading to a comparable 24-month overall survival of 88% (vs 92%; P = .612). CPX-351 demonstrated a favorable toxicity profile with no treatment-related mortality. Positive MRD by error-corrected GATA1 next-generation sequencing, the presence of trisomy 8 or a complex karyotype were associated with an increased risk for relapse. In conclusion, replacing intensity-reduced induction therapy with CPX-351 in ML-DS led to a significantly lower EFS, highlighting the need for dose optimization to balance the efficacy and toxicity in this sensitive patient population. This trial was registered at https://www.clinicaltrialsregister.eu as EudraCT #2018-002988-25.

News (Medical) associated with Cytarabine/Daunorubicin Liposomal

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

03 Dec 2024

·investor.jazzpharma.com

Jazz Pharmaceuticals to Present Advancements in Solid Tumors and Blood Cancer Research at San Antonio Breast Cancer Symposium and American Society of Hematology Annual Meeting

Data showcased at SABCS and ASH reflect progress and growth of Jazz's research and development in oncology and demonstrate ongoing efforts to redefine what is possible for cancer treatment DUBLIN , Dec. 3, 2024 /PRNewswire/ -- Jazz Pharmaceuticals plc (Nasdaq: JAZZ) today announced that the Company and its partners will present two abstracts at the 2024 San Antonio Breast Cancer Symposium (SABCS) from December 10-13 and 13 abstracts at the 66th Annual American Society of Hematology (ASH) Annual Meeting from December 7-10. A trial-in-progress poster presentation at SABCS outlines the trial design of the ongoing Phase 3 EmpowHER-303 trial (NCT06435429), which is evaluating the efficacy and safety of Ziihera® (zanidatamab-hrii) vs trastuzumab with chemotherapy in patients with metastatic HER2-positive breast cancer who have progressed on, or are intolerant to, trastuzumab deruxtecan. New data from a Phase 1b/2 study of zanidatamab plus evorpacept (NCT05027139) in patients with pre-treated HER2-positive and HER2-low metastatic breast cancer will be featured as a poster spotlight. "Following the recent FDA approval of Ziihera for the treatment of adults with previously treated, unresectable or metastatic HER2-positive (IHC 3+) biliary tract cancer, we are pleased to present meaningful data at SABCS on the positive impact zanidatamab can have for patients with HER2-expressing cancers. We continue to advance our clinical program for zanidatamab with the goal to improve outcomes for patients with difficult-to-treat HER2-positive cancers. Our development program includes multiple ongoing Phase 3 trials evaluating 1L BTC, 1L GEA, with top-line PFS results expected in the second quarter of 2025, and metastatic breast cancer after T-DXd treatment, supporting the use of zanidatamab after other HER2-targeted therapies," 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 . "Additionally, we look forward to highlighting 13 abstracts featuring oncology research at ASH 2024, which underscores our commitment to improving standards of care in blood cancer and other hematologic diseases." The full SABCS abstracts are available here. The Jazz and partner-supported presentations at SABCS 2024 are: Ziihera® (zanidatamab-hrii) Presentations Topic Author Presentation Details EmpowHER 303: A phase 3 study to evaluate the efficacy and safety of zanidatamab vs trastuzumab withchemotherapy in patients (pts) with metastatic HER2-positive breast cancer who have progressed on, or are intolerant to, trastuzumab deruxtecan Sara M Tolaney, et al. Presentation Type: Poster Session Abstract Number: SESS-1922 Date: Friday December 13, 2024 Time: 12:00-2:00 PM (PST) Zanidatamab in combination with evorpacept in HER2-positive and HER2-low metastatic breast cancer: Results from a phase 1b/2 study Alberto J Montero, et al. Presentation Type: Poster Spotlight Presentation Abstract Number: SESS-2007 Date: Thursday December 12, 2024 Time: 7:00-8:30 AM (PST) The full ASH abstracts are available here. The Jazz and partner-supported presentations at ASH 2024 are: Vyxeos® (daunorubicin and cytarabine) Presentations Topic Author Presentation Details A Randomized Comparison of CPX-351 and FLAG-Ida in Patients WithHigh-Risk Acute Myeloid Leukemia(AML)/Myelodysplastic Syndrome (MDS) And MDS-Related GeneMutations: A Subgroup Analysis of the UK NCRI AML19 Trial Priyanka Mehta , et al. Presentation Type: Oral Presentation Abstract Number: #55 Date: Saturday December 7, 2024 Time: 9:30 AM (PST) AML-MR Mutations Drive the Benefit of CPX-351 over 7+3 in the Pivotal Phase 3 AML Trial Shai O Shimony, et al. Presentation Type: Oral Presentation Abstract Number: #60 Date: Saturday December 7, 2024 Time: 10:45 AM (PST) Phase Ib/II Study of CPX-351 in Combination with Venetoclax in Patients with Newly Diagnosed, HighRisk Acute Myeloid Leukemia Emmanuel Almanza , et al. Presentation Type: Poster Presentation Abstract Number: #1511 Date: Saturday December 7, 2024 Time: 5:30-7:30 PM (PST) A Randomised Comparison of CPX-351 versus Standard Daunorubicin and Cytarabine plus FractionatedGemtuzumab Ozogamicin in Older AML Adults Without Known AdverseRisk Cytogenetics: Results of the NCRIAML18 Trial Steven Knapper , et al. Presentation Type: Oral Presentation Abstract Number: #59 Date: Saturday December 7, 2024 Time: 10:30 AM (PST) Phase 1/1b Dose Escalation and Expansion of CPX-351 in Combinationwith Gemtuzumab Ozogamicin in Newly Diagnosed Acute MyeloidLeukemia Onyee Chan , et al. Presentation Type: Poster Presentation Abstract Number: #4270 Date: Monday December 9, 2024 Time: 6:00-8:00 PM (PST) A Phase 3 Randomized Trial for Patients with de novo AML Comparing Standard Therapy IncludingGemtuzumab Ozogamicin (GO) to CPX-351 with GO – A report from the Children's Oncology Group Jessica A Pollard, et al. Presentation Type: Oral Presentation Abstract Number: #967 Date: Monday December 9, 2024 Time: 4:30 PM (PST) Combination of CPX-351 andGemtuzumab Ozogamicin (GO) inRelapsed Refractory (R/R) AcuteMyeloid Leukemia and PostHypomethylating Agent (HMA) FailureHigh-Risk Myelodysplastic Syndrome (HR-MDS) Jayastu Senapati , et al. Presentation Type: Poster Presentation Abstract Number: #2903 Date: Sunday December 8, 2024 Time: 6:00-8:00 PM (PST) Phase Ib/II Study of CPX-351 plusVenetoclax in Patients with Relapsed/Refractory Acute MyeloidLeukemia (AML) Vanthana Bharathi , et al. Presentation Type: Poster Presentation Abstract Number: #4272 Date: Monday December 9, 2024 Time: 6:00-8:00 PM (PST) Rapid, Reliable, and ComprehensiveIdentification of MDS-DefiningCytogenetic Changes By a FISH-Panel Containing Six Probes in a Real-World Population of Patients with Suspected AML Katayoon Shirneshan, et al. Presentation Type: Poster Presentation Abstract Number: #4308 Date: Monday December 9, 2024 Time: 6:00-8:00 PM (PST) Prospective Evaluation of the Impact ofMeasurable Residual Disease (MRD) by Error Corrected Next-GenerationSequencing (NGS) with CPX-351 inAcute Myeloid Leukemia (AML) David Sallman , et al. Presentation Type: Poster Presentation Abstract Number: #4332 Date: Monday December 9, 2024 Time: 6:00-8:00 PM (PST) A Phase 1 Study of CPX-351 Plus Gilteritinib in Relapsed or Refractory, FLT3-Mutated Acute Myeloid Leukemia Onyee Chan , et al. Presentation Type: Poster Presentation Abstract Number: #1520 Date: Saturday December 7, 2024 Time: 5:30-7:30 PM (PST) Defitelio® (defibrotide sodium) Presentations Topic Author Presentation Details Genetic Susceptibility in Sinusoidal Obstruction Syndrome/Veno-OcclusiveDisease Ioulia Mavrikou, et al. Presentation Type: Poster presentation Abstract Number: #4778 Date: Monday December 9, 2024 Time: 6:00-8:00 PM (PST) Defibrotide reduces hypercoagulablestate in patients with Sickle Cell Disease-Related Acute ChestSyndrome Edo Schaefer , et al. Presentation Type: Poster presentation Abstract Number: #2515 Date: Sunday December 8, 2024 Time: 6:00-8:00 PM (PST) About Ziihera® (zanidatamab-hrii) Ziihera (zanidatamab-hrii) is a bispecific HER2-directed antibody that binds to two extracellular sites on HER2. Binding of zanidatamab-hrii with HER2 results in internalization leading to a reduction of the receptor on the tumor cell surface. Zanidatamab-hrii induces complement-dependent cytotoxicity ( CDC ), antibody-dependent cellular cytotoxicity (ADCC) and antibody-dependent cellular phagocytosis (ADCP). These mechanisms result in tumor growth inhibition and cell death in vitro and in vivo.1 In the United States , Ziihera is indicated for the treatment of adults with previously treated, unresectable or metastatic HER2-positive (IHC 3+) biliary tract cancer (BTC), as detected by an FDA-approved test.1 The U.S. Food and Drug Administration (FDA) granted accelerated approval for this indication based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).1 Zanidatamab is not approved anywhere else in the world. Zanidatamab is being developed in multiple clinical trials as a targeted treatment option for patients with solid tumors that express HER2. Zanidatamab is being developed by Jazz and BeiGene, Ltd. (BeiGene) under license agreements from Zymeworks, which first developed the molecule. The FDA granted Breakthrough Therapy designation for zanidatamab development in patients with previously treated HER2 gene-amplified BTC, and two Fast Track designations for zanidatamab: one as a single agent for refractory BTC and one in combination with standard-of-care chemotherapy for 1L gastroesophageal adenocarcinoma (GEA). Additionally, zanidatamab has received Orphan Drug designations from FDA for the treatment of BTC and GEA, as well as Orphan Drug designation from the European Medicines Agency for the treatment of BTC and gastric cancer. Important Safety Information for ZIIHERA® WARNING: EMBRYO-FETAL TOXICITY Exposure to ZIIHERA during pregnancy can cause embryo-fetal harm. Advise patientsof the risk and need for effective contraception. WARNINGS AND PRECAUTIONS Embryo-Fetal ToxicityZIIHERA can cause fetal harm when administered to a pregnant woman. In literature reports, use of a HER2-directed antibody during pregnancy resulted in cases of oligohydramnios and oligohydramnios sequence manifesting as pulmonary hypoplasia, skeletal abnormalities, and neonatal death. Verify the pregnancy status of females of reproductive potential prior to the initiation of ZIIHERA. Advise pregnant women and females of reproductive potential that exposure to ZIIHERA during pregnancy or within 4 months prior to conception can result in fetal harm. Advise females of reproductive potential to use effective contraception during treatment with ZIIHERA and for 4 months following the last dose of ZIIHERA. Left Ventricular DysfunctionZIIHERA can cause decreases in left ventricular ejection fraction (LVEF). LVEF declined by >10% and decreased to <50% in 4.3% of 233 patients. Left ventricular dysfunction (LVD) leading to permanent discontinuation of ZIIHERA was reported in 0.9% of patients. The median time to first occurrence of LVD was 5.6 months (range: 1.6 to 18.7). LVD resolved in 70% of patients. Assess LVEF prior to initiation of ZIIHERA and at regular intervals during treatment. Withhold dose or permanently discontinue ZIIHERA based on severity of adverse reactions. The safety of ZIIHERA has not been established in patients with a baseline ejection fraction that is below 50%. Infusion-Related ReactionsZIIHERA can cause infusion-related reactions (IRRs). An IRR was reported in 31% of 233 patients treated with ZIIHERA as a single agent in clinical studies, including Grade 3 (0.4%), and Grade 2 (25%). IRRs leading to permanent discontinuation of ZIIHERA were reported in 0.4% of patients. IRRs occurred on the first day of dosing in 28% of patients; 97% of IRRs resolved within one day. Prior to each dose of ZIIHERA, administer premedications to prevent potential IRRs. Monitor patients for signs and symptoms of IRR during ZIIHERA administration and as clinically indicated after completion of infusion. Have medications and emergency equipment to treat IRRs available for immediate use. If an IRR occurs, slow, or stop the infusion, and administer appropriate medical management. Monitor patients until complete resolution of signs and symptoms before resuming. Permanently discontinue ZIIHERA in patients with recurrent severe or life-threatening IRRs. DiarrheaZIIHERA can cause severe diarrhea. Diarrhea was reported in 48% of 233 patients treated in clinical studies, including Grade 3 (6%) and Grade 2 (17%). If diarrhea occurs, administer antidiarrheal treatment as clinically indicated. Perform diagnostic tests as clinically indicated to exclude other causes of diarrhea. Withhold or permanently discontinue ZIIHERA based on severity. ADVERSE REACTIONS Serious adverse reactions occurred in 53% of 80 patients with unresectable or metastatic HER2-positive BTC who received ZIIHERA. Serious adverse reactions in >2% of patients included biliary obstruction (15%), biliary tract infection (8%), sepsis (8%), pneumonia (5%), diarrhea (3.8%), gastric obstruction (3.8%), and fatigue (2.5%). A fatal adverse reaction of hepatic failure occurred in one patient who received ZIIHERA. The most common adverse reactions in 80 patients with unresectable or metastatic HER2-positive BTC who received ZIIHERA (≥20%) were diarrhea (50%), infusion-related reaction (35%), abdominal pain (29%), and fatigue (24%). USE IN SPECIFIC POPULATIONS Pediatric UseSafety and efficacy of ZIIHERA have not been established in pediatric patients. Geriatric UseOf the 80 patients who received ZIIHERA for unresectable or metastatic HER2-positive BTC, there were 39 (49%) patients 65 years of age and older. Thirty-seven (46%) were aged 65-74 years old and 2 (3%) were aged 75 years or older. No overall differences in safety or efficacy were observed between these patients and younger adult patients. About Vyxeos® (daunorubicin and cytarabine) liposome for injectionVyxeos is a liposomal combination of daunorubicin, an anthracycline topoisomerase inhibitor, and cytarabine, a nucleoside metabolic inhibitor. In the U.S., Vyxeos (daunorubicin and cytarabine) liposome for injection is indicated for the treatment of newly-diagnosed therapy-related acute myeloid leukemia (t-AML) or AML with myelodysplasia-related changes (AML-MRC) in adults and pediatric patients 1 year and older.3 More information about Vyxeos in the United States, including Full Prescribing Information and BOXED Warning, is available here. Important Safety Information for VYXEOS® WARNING: VYXEOS has different dosage recommendations from other medications that contain daunorubicin and/or cytarabine. Do not substitute VYXEOS for other daunorubicin and/or cytarabine-containing products. VYXEOS should not be given to patients who have a history of serious allergic reaction to daunorubicin, cytarabine, or any of its ingredients. VYXEOS can cause a severe decrease in blood cells (red and white blood cells and cells that prevent bleeding, called platelets) which can result in serious infection or bleeding and possibly lead to death. Your doctor will monitor your blood counts during treatment with VYXEOS. Patients should tell the doctor about new onset fever or symptoms of infection or if they notice signs of bruising or bleeding. VYXEOS can cause heart-related side effects. Tell your doctor about any history of heart disease, radiation to the chest, or previous chemotherapy. Inform your doctor if you develop symptoms of heart failure such as: shortness of breath or trouble breathing swelling or fluid retention, especially in the feet, ankles, or legs unusual tiredness VYXEOS may cause allergic reactions including anaphylaxis. Seek immediate medical attention if you develop signs and symptoms of anaphylaxis such as: trouble breathing severe itching skin rash or hives swelling of the face, lips, mouth, or tongue VYXEOS contains copper and may cause copper overload in patients with Wilson's disease or other copper-processing disorders. VYXEOS can damage the skin if it leaks out of the vein. Tell your doctor right away if you experience symptoms of burning, stinging, or blisters and skin sores at the injection site. VYXEOS can harm your unborn baby. Inform your doctor if you are pregnant, planning to become pregnant, or nursing. Do not breastfeed while receiving VYXEOS. Females and males of reproductive potential should use effective contraception during treatment and for 6 months following the last dose of VYXEOS. The most common side effects are bleeding events, fever, rash, swelling, nausea, sores in the mouth or throat, diarrhea, constipation, muscle pain, tiredness, stomach pain, difficulty breathing, headache, cough, decreased appetite, irregular heartbeat, pneumonia, blood infection, chills, sleep disorders, and vomiting. Call your doctor for medical advice about side effects. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088. You may also report side effects to Jazz Pharmaceuticals at 1-800-520-5568. About Defitelio® (defibrotide sodium)In the U.S., Defitelio® (defibrotide sodium) injection 80mg/mL received U.S. Food and Drug Administration (FDA) marketing approval on March 30, 2016, and it is indicated for the treatment of adult and pediatric patients with hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS), with renal or pulmonary dysfunction following hematopoietic stem-cell transplantation (HSCT) and is the first and only FDA-approved therapy for patients with this rare, potentially fatal complication.6 Please see full Prescribing Information for Defitelio in the United States . In Europe, defibrotide is marketed under the name Defitelio® ▼ (defibrotide). In October 2013, the European Commission granted marketing authorization to Defitelio under exceptional circumstances for the treatment of severe VOD in patients after HSCT therapy. In Europe, Defitelio is indicated in patients over one month of age. ▼This medicinal product is subject to additional monitoring. This will allow quick identification of new safety information. Healthcare professionals are asked to report any suspected adverse reactions via the national reporting system found under section 4.8 of the SmPC (https://www.ema.europa.eu/en/documents/product-information/defitelio-epar-product-information_en.pdf) The full Summary of Product Characteristics of Defitelio in Europe is available here. Important Safety Information for Defitelio® Defitelio should not be given to patients who are: Currently taking anticoagulants or fibrinolytics Allergic to Defitelio or any of its ingredients Defitelio may increase the risk of bleeding in patients with VOD and should not be given to patients with active bleeding. During treatment with Defitelio, patients should be monitored for signs of bleeding. In the event that bleeding occurs during treatment with Defitelio, treatment should be temporarily or permanently stopped. Patients should tell the doctor right away about any signs or symptoms of hemorrhage such as unusual bleeding, easy bruising, blood in urine or stool, headache, confusion, slurred speech, or altered vision. Defitelio may cause allergic reactions including anaphylaxis. Patients who develop signs and symptoms of anaphylaxis such as trouble breathing, severe itching, skin rash or hives, or swelling of the face, lips, mouth or tongue should seek medical attention immediately. The most common side effects of Defitelio are decreased blood pressure, diarrhea, vomiting, nausea and nose bleeds. Defitelio is not approved for the prevention of VOD. It is not indicated in patients with hypersensitivity to defibrotide or any of its excipients or with concomitant use of thrombolytic therapy. 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 serious diseases—often with limited or no therapeutic options. We have a diverse portfolio of marketed medicines, including leading therapies for sleep disorders and epilepsy, and a growing portfolio of cancer treatments. Our patient-focused and science-driven approach powers pioneering research and development advancements across our robust pipeline of innovative therapeutics in oncology and neuroscience. Jazz is headquartered in Dublin, Ireland with research and development laboratories, manufacturing facilities and employees in multiple countries committed to serving patients worldwide. Please visit www.jazzpharmaceuticals.com for more information. Jazz Pharmaceuticals plc Caution Concerning Forward-Looking Statements This press release contains forward-looking statements, including, but not limited to, statements related to expectations of top-line PFS results in the second quarter of 2025 and other statements that are not historical facts. These forward-looking statements are based on Jazz Pharmaceuticals' 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 pharmaceutical product development, and other risks and uncertainties affecting Jazz Pharmaceuticals and its development programs, including those described from time to time under the caption "Risk Factors" and elsewhere in Jazz Pharmaceuticals plc's Securities and Exchange Commission filings and reports (Commission File No. 001-33500), including Jazz Pharmaceuticals' Annual Report on Form 10-K for the year ended December 31, 2023 , as supplemented by our Quarterly Report on Form 10-Q for the quarter ended September 30, 2024 , and future filings and reports by Jazz Pharmaceuticals . Other risks and uncertainties of which Jazz Pharmaceuticals is not currently aware may also affect Jazz Pharmaceuticals' forward-looking statements and may cause actual results and the timing of events to differ materially from those anticipated. The forward-looking statements herein are made only as of the date hereof or as of the dates indicated in the forward-looking statements, even if they are subsequently made available by Jazz Pharmaceuticals on its website or otherwise. Jazz Pharmaceuticals undertakes no obligation to update or supplement any forward-looking statements to reflect actual results, new information, future events, changes in its expectations or other circumstances that exist after the date as of which the forward-looking statements were made. Contacts: Jazz Media Contact: Kristin Bhavnani Head of Global Corporate Communications Jazz Pharmaceuticals plc CorporateAffairsMediaInfo@jazzpharma.com Ireland +353 1 637 2141 U.S. +1 215 867 4948 Jazz Investor Contact: Jeff Macdonald Executive Director, Investor Relations Jazz Pharmaceuticals plc investorinfo@jazzpharma.com Ireland +353 1 634 3211 U.S. +1 650 496 2717 View original content to download multimedia:https://www.prnewswire.com/news-releases/jazz-pharmaceuticals-to-present-advancements-in-solid-tumors-and-blood-cancer-research-at-san-antonio-breast-cancer-symposium-and-american-society-of-hematology-annual-meeting-302320929.html SOURCE Jazz Pharmaceuticals plc

Phase 3Clinical ResultDrug ApprovalFast TrackBreakthrough Therapy

100 Deals associated with Cytarabine/Daunorubicin Liposomal

External Link

KEGG	Wiki	ATC	Drug Bank
-	-	L01XY01	-

R&D Status

Approved

10 top approved records.

to view more data

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

10 top R&D records.

to view more data

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
Anemia, Refractory, With Excess of Blasts	Phase 2	United States	Jazz Pharmaceuticals Plc	09 Aug 2021
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

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04802161 (not available, CTgov)	Phase 2	Treatment related acute myeloid leukaemia \| Acute myeloid leukemia with multilineage dysplasia \| Chronic Myelomonocytic Leukemia ... View more	50	Bone Marrow Aspiration and Biopsy+Pomalidomide+Liposome-encapsulated Daunorubicin-Cytarabine (Arm A (Daunorubicin and Cytarabine Liposome, Pomalidomide))	hpjwduqqpt = poedskmkxd boilcjwmin (aqrajuuhpu, hxooargkzz - avfsdlccry) View more	-	19 Dec 2025
NCT04802161 (not available, CTgov)	Phase 2		50	Bone Marrow Aspiration and Biopsy+Liposome-encapsulated Daunorubicin-Cytarabine (Arm B (Daunorubicin and Cytarabine Liposome))	hpjwduqqpt = bgkpscprsd boilcjwmin (aqrajuuhpu, ltsnucdiea - rqgwaqdtxv) View more	-	19 Dec 2025
NCT05656248 (ASH2025)	Phase 1	Myeloid Tumor	25	CPX-351 100 units/m2	gtrebscxtt(ymhdnytymh) = sqihwhxuhj kqruyccylf (jbmoijfynq )	Positive	06 Dec 2025
ASH2025	Not Applicable	Acute Myeloid Leukemia	219	CPX-351	efquaervwg(whyexxllyg) = kihhapyxvi firleqdpwz (lvxjcuywhc ) View more	Positive	06 Dec 2025
NCT04195945 (ASH2025)	Phase 2	Acute Myeloid Leukemia \| Myeloid Tumor	60	CPX-351	lfmfkwnspr(wrnjgngwsd) = ndlogticsj iawyxfywus (tfaejtwyvz ) View more	Positive	06 Dec 2025
NCT04195945 (ASH2025)	Phase 2	Acute Myeloid Leukemia \| Myeloid Tumor	60	CLAG-M (Cladribine, High-Dose Cytarabine, G-CSF, and Mitoxantrone)	lfmfkwnspr(wrnjgngwsd) = pzzipdolsp iawyxfywus (tfaejtwyvz ) View more	Positive	06 Dec 2025
ASH2025	Not Applicable	Acute Myeloid Leukemia	85	CPX-351	wmwcbuuecw(btaeminkgb) = kdxqbsoapt ezkeqkqeeb (vlrwdekyuw ) View more	Positive	06 Dec 2025
ASH2025	Not Applicable	Acute Myeloid Leukemia	134	CPX-351	nklivgxdah(rplqfrtgxt) = qwbsyywkvb jxdhwkshmc (tciiqqhcbl ) View more	Positive	06 Dec 2025
ASH2025	Not Applicable	Acute Myeloid Leukemia FLT3-ITD-negative	25	CPX-351 ± Gemtuzumab Ozogamicin	avpnflvhmd(iaurgtlkwf) = adtnaoqrdx nszzryulyl (akvhubwaen ) View more	Positive	06 Dec 2025
NCT03896269 (ASH2025)	Phase 1/2	High Risk Myelodysplastic Syndrome \| Chronic Myelomonocytic Leukemia	6	CPX-351 + Venetoclax	nnoonnbbyq(holffbkisl) = unsumhhfqh aowolyukit (oerylfctgf ) View more	Positive	06 Dec 2025
ASH2025	Not Applicable	Chromosome 17, Trisomy 17p \| Acute Myeloid Leukemia with Myelodysplasia-Related Changes	335	CPX-351 (TP53 mutated)	fyzojpjvav(ongzbntszd) = rbsczkaffm hgeusaqyzr (lvxfpajuks )	Positive	06 Dec 2025
ASH2025	Not Applicable		335	CPX-351 (TP53 wild-type)	fyzojpjvav(ongzbntszd) = dablftibqe hgeusaqyzr (lvxfpajuks ) View more	Positive	06 Dec 2025
ASH2025	Not Applicable	Acute Myeloid Leukemia First line	870	CPX-351	zqijxtpfgz(qkhuumaxms) = a consistently higher % time at home was observed in patients treated with CPX-351 (range: 80.4% to 97.6%) vs VEN-AZA (range: 74.7% to 90.2%), typically ~5-10% higher per month (monthly difference range: +3.5 to +11.9%). zylmhvhurg (kzxxrhtpaw ) View more	Positive	06 Dec 2025
ASH2025	Not Applicable	Acute Myeloid Leukemia First line	870	VEN-AZA		Positive	06 Dec 2025

Translational Medicine

Boost your research with our translational medicine data.

view full example data

Deal

Boost your decision using our deal data.

view full example data

Core Patent

Boost your research with our Core Patent data.

view full example data

Clinical Trial

Identify the latest clinical trials across global registries.

view full example data

Approval

Accelerate your research with the latest regulatory approval information.

view full example data

Regulation

Understand key drug designations in just a few clicks with Synapse.

view full example data

AI Agents Built for Biopharma Breakthroughs

Accelerate discovery. Empower decisions. Transform outcomes.

Get started for free today!

Accelerate Strategic R&D decision making with Synapse, PatSnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.

Start your data trial now!

Synapse data is also accessible to external entities via APIs or data packages. Empower better decisions with the latest in pharmaceutical intelligence.

Bio

Bio Sequences Search & Analysis

Chemical

Chemical Structures Search & Analysis