Benzinga | Nov 4, 2021 11:07AM EDT

Jazz Pharmaceuticals To Present Data Showcasing Clinical Advances Across Hematology/Oncology At ASH 2021 Annual Meeting

Jazz Pharmaceuticals plc (NASDAQ:JAZZ) today announced that 16 new data abstracts from across its hematology/oncology development program will be presented at the 63rd American Society of Hematology (ASH) Annual Meeting, which will be held December 11-14, 2021. This includes five presentations from investigator-sponsored trials and three presentations from collaboration studies with The University of Texas MD Anderson Cancer Center (MD Anderson).

"The data at ASH demonstrates Jazz's focus on making a difference for people living with rare forms of leukemias and blood cancers, both through the development of new treatment options as well as further evaluating our currently approved medicines," said Robert Iannone, M.D., M.S.C.E., executive vice president, research and development and chief medical officer of Jazz Pharmaceuticals. "Our support of several investigator-sponsored and collaboration trials exemplifies our commitment to working with experts to enable studies beyond our own company-sponsored trials, and to identifying new treatment options for patients through a variety of means."

Highlights at ASH include:

* A poster presentation sharing, for the first time, data from the Phase 2/3 study of Rylaze in patients with acute lymphoblastic leukemia (ALL)/lymphoblastic lymphoma (LBL) who developed hypersensitivity or silent inactivation to a long-acting E. coli--derived asparaginase.

* Results for Vyxeos(r) (daunorubicin and cytarabine) in acute myeloid leukemia (AML) including an oral presentation from a real-world evidence study of Vyxeos use in newly diagnosed patients and a poster presentation from a Phase 1b study of lower-dose Vyxeos in combination with venetoclax in patients with AML who are unfit for intensive chemotherapy.

* Data for Vyxeos use in new patient populations, including oral presentations of two studies of Vyxeos as treatment in higher risk Myelodysplastic Syndrome (MDS).

* A poster presentation with final results from a real-world evidence study, DEFIFrance, of Defitelio(r) (defibrotide sodium) treatment in adults with severe or very severe veno-occlusive disease/sinusoidal obstruction syndrome after hematopoietic cell transplantation.

The ASH abstracts are available online starting today, November 4 at https://ash.confex.com/ash/2021/webprogram/start.html.

ASH will be held as a hybrid conference virtually and in-person in Atlanta, GA at the Georgia World Congress Center. A full list of Jazz and investigator-sponsored presentations follows below:

Rylaze Presentations

Presentation Topic AuthorDate / Time (EST) / Session Title / Presentation Number

* Type: Poster * Number: 2307 Initial Results from a Phase 2/3 Study * Session: 614. Acute of Recombinant Erwinia Asparaginase Lymphoblastic Leukemias: (JZP458) in Patients with Acute Luke Therapies, Excluding Lymphoblastic Leukemia (ALL)/ Maese Transplantation and Lymphoblastic Lymphoma (LBL) Who are et al. Cellular Immunotherapies:Allergic/Hypersensitive to E. Poster II Coli?Derived Asparaginases * Date/Time: Sunday, December 12, 2021: 6:00 PM-8:00 PM * Location: Hall B5

Vyxeos Presentations

Presentation Topic Author Date / Time (EST) / Session Title / Presentation Number

* Type: Oral Presentation * Number: 540 * Session: 637. Myelodysplastic A Pilot Study of CPX-351 (Vyxeos Syndromes ? Clinical and(R)) for Transplant Eligible, Meagan A. Epidemiological: Higher Risk Patients with Jacoby et al. Treatment of HIgh Risk Myelodysplastic Syndrome and Relapsed/Refractory Myelodysplastic Syndrome * Date/Time: Sunday, December 12, 2021: 4:30 PM-6:00 PM * Location: B211-B212

* Type: Oral Presentation * Number: 33 * Session: 615. Acute Myeloid Leukemias: Commercially Available Therapies, Excluding Real-World Experience of CPX-351 Transplantation and As First-Line Treatment in 188 Christina Cellular Patients with Acute Myeloid Rautenberg et Immunotherapies: Leukemia al. Innovative induction regimens in AML: data from real life and clinical trials * Date/Time: Saturday, December 11, 2021: 9:30 AM-11:00 AM * Location: B405-B407

* Type: Oral Presentation * Number: 243 * Session: 637. Myelodysplastic CPX 351 As First Line Treatment Syndromes ? Clinical andin Higher Risk MDS. a Phase II Pierre Peterlin Epidemiological: Trial By the GFM et al. Treatment of High Risk Myelodysplastic Syndrome * Date/Time: Saturday, December 11, 2021: 2:00 PM-3:30 PM * Location: B207-B208

* Type: Poster * Number: 1268 * Session: 615. Acute Myeloid Leukemias: Preliminary Results by Age Group Commercially Available of Treatment with CPX-351 Plus Therapies, Excluding Venetoclax in Adults with Newly Vinod Pullarkat Transplantation and Diagnosed AML: Subgroup Analysiset al. Cellular of the V-FAST Phase 1b Master Immunotherapies: Poster Trial I * Date/Time: Saturday, December 11, 2021: 5:30 PM-7:30 PM * Location: Hall B5

* Type: Poster * Number: 2316 * Session: 615. Acute Phase 1b Study of Lower-dose Myeloid Leukemias: CPX-351 Plus Venetoclax As Commercially Available First-line Treatment for Therapies, Excluding Patients with AML Who Are Unfit Geoffrey L. Uy Transplantation and for Intensive Chemotherapy: et al. Cellular Preliminary Safety and Efficacy Immunotherapies: Poster Results II * Date/Time: Sunday, December 12, 2021: 6:00 PM-8:00 PM * Location: Hall B5

* Type: Poster * Number: 1248 * Session: 615. Acute Myeloid Leukemias: Real-World Study of the Commercially Available Treatment Patterns of Patients Therapies, Excluding Diagnosed with Therapy-Related Alex Legg et Transplantation and AML or AML-MRC in England al. Cellular between 2013 and 2020 Using the Immunotherapies: Poster Cancer Analysis System Database I * Date/Time: Saturday, December 11, 2021: 5:30 PM-7:30 PM * Location: Hall B5

* Type: Poster * Number: 2310 * Session: 615. Acute Myeloid Leukemias: Commercially Available Real-World Study of CPX-351 Therapies, Excluding Treatment Outcomes for Acute Alex Legg et Transplantation and Myeloid Leukemia (AML) in al. Cellular England Immunotherapies: Poster II * Date/Time: Sunday, December 12, 2021: 6:00 PM-8:00 PM * Location: Hall B5

* Type: Poster * Number: 3674 Updated Results of a Phase 1/2 * Session: 637. Study of Lower Dose CPX-351 for Myelodysplastic Patients with Int-2 or High RiskGuillermo Syndromes ? Clinical andIPSS Myelodysplastic Syndromes Montalban-Bravo Epidemiological: Poster and Chronic Myelomonocytic et al. III Leukemia after Failure to * Date/Time: Monday, Hypomethylating Agents December 13, 2021: 6:00 PM-8:00 PM * Location: Hall B5

* Type: Poster * Number: 2323 Liposomal Cytarabine and * Session: 615. Acute Daunorubicin (CPX-351) in Myeloid Leukemias: Combination with Gemtuzumab Commercially Available Ozogamicin (GO) in Relapsed Therapies, Excluding Refractory (R/R) Patients with Daniel Rivera Transplantation and Acute Myeloid Leukemia (AML) andet al. Cellular Post-Hypomethylating Agent Immunotherapies: Poster (Post-HMA) Failure High-Risk II Myelodysplastic Syndrome * Date/Time: Sunday, (HR-MDS) December 12, 2021: 6:00 PM-8:00 PM * Location: Hall B5

* Type: Poster * Number: 1275 * Session: 616. Acute Myeloid Leukemias: A Phase II Study of CPX-351 plus Investigational Venetoclax in Patients with Therapies, Excluding Relapsed/Refractory (R/R) or Kunhwa Kim et Transplantation and Newly Diagnosed Acute Myeloid al. Cellular Leukemia (AML) Immunotherapies: Poster I * Date/Time: Saturday, December 11, 2021: 5:30 PM-7:30 PM * Location: Hall B5

Defitelio Presentations

Presentation Topic Author Date / Time (EST) / Session Title / Presentation Number

* Type: Oral Presentation * Number: 749 * Session: 721. Allogeneic Transplantation: A Phase 3, Randomized, Adaptive Study Conditioning Regimens, of Defibrotide (DF) Vs Best Supportive Engraftment and Acute Care (BSC) for the Prevention of Stephan Toxicities; Prevention Hepatic Veno-occlusive Disease/ A. Grupp and Management of Sinusoidal Obstruction Syndrome (VOD/ et al. Complications SOS) in Patients (pts) Undergoing * Date/Time: Monday, Hematopoietic Cell Transplantation December 13, 2021; 2:45 (HCT): Preliminary Results PM ? 4:15 PM EST * Presentation Time: 3:45 PM EST * Location: Thomas Murphy Ballroom 3-4

* Type: Poster * Number: 1789 Final Long-term Results from the * Session: 721. AllogeneicDEFIFrance Registry Study: Efficacy and Transplantation: Safety of Defibrotide for the TreatmentMohamad Conditioning Regimens, of Severe/Very Severe Veno-Occlusive Mohty et Engraftment and Acute Disease/Sinusoidal Obstruction Syndromeal. Toxicities: Poster I after Hematopoietic Cell * Date/Time: Saturday, Transplantation December 11, 2021; 5:30 PM ? 7:30 PM EST * Location: Hall B5

* Type: Poster Veno-Occlusive Disease/Sinusoidal * Number: 1946 Obstruction Syndrome Without * Session: 904. Outcomes Hematopoietic Cell Transplantation in aXue Wang Research?Non-Malignant Real-World Population in the United et al. Conditions: Poster I States: Patient Characteristics, Prior * Date/Time: Saturday, Treatment Patterns, and Time to December 11, 2021; 5:30 Diagnosis PM ? 7:30 PM EST * Location: Hall B5

* Type: Poster * Number: 3237 * Session: 332. Gregory Anticoagulation and Defibrotide Therapy for Sars CoV2 AcuteYanik et Antithrombotic Respiratory Distress Syndrome al. Therapies: Poster III * Date/Time: Monday, December 13, 2021: 6:00 PM-8:00 PM * Location: Hall B5

* Type: Oral Presentation * Number: 672 Use of Defibrotide in Patients with * Session: 332. COVID-19 Pneumonia; Results of the Annalisa Anticoagulation and Ruggeri Antithrombotic TherapiesDefi-VID19 Phase 2 Trial et al. * Date/Time: Monday, December 13, 2021: 2:45 PM-4:15 PM * Location: B401-B402

About Rylaze(tm) (asparaginase erwinia chrysanthemi (recombinant)-rywn)

Rylaze, also known as JZP458, is approved in the U.S. for use as a component of a multi-agent chemotherapeutic regimen for the treatment of acute lymphoblastic leukemia (ALL) or lymphoblastic lymphoma (LBL) in pediatric and adult patients one month and older who have developed hypersensitivity to E. coli-derived asparaginase. Rylaze has orphan drug designation for the treatment of ALL/LBL in the United States. Rylaze is a recombinant erwinia asparaginase that uses a novel Pseudomonas fluorescens expression platform. JZP458 was granted Fast Track designation by the U.S. Food and Drug Administration (FDA) in October 2019 for the treatment of this patient population. Rylaze was approved as part of the Real-Time Oncology Review program, an initiative of the FDA's Oncology Center of Excellence designed for efficient delivery of safe and effective cancer treatments to patients.