PR Newswire | Nov 4, 2021 11:05AM EDT

Hematology/Oncology at ASH 2021 Annual Meeting

11/04 10:04 CDT

Jazz Pharmaceuticals to Present Data Showcasing Clinical Advances Across Hematology/Oncology at ASH 2021 Annual MeetingSixteen new abstracts to be presented, including the first presentation of data from the Rylaze(tm) (asparaginase erwinia chrysanthemi (recombinant)-rywn) Phase 2/3 study that supported U.S. FDA approval earlier this year DUBLIN, Nov. 4, 2021

DUBLIN, Nov. 4, 2021 /PRNewswire/ -- 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

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

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

Vyxeos Presentations

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

* Type: Oral Presentation * Number: 540 * Session: 637. Myelodysplastic Syndromes - A Pilot Study of CPX-351 Clinical and (Vyxeos(r)) for Epidemiological: Transplant Eligible, Meagan A. Jacoby et al. Treatment of HIgh Higher Risk Patients with Risk and Relapsed/ Myelodysplastic Syndrome 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, Real-World Experience of Excluding CPX-351 As First-Line Transplantation andTreatment in 188 PatientsChristina Rautenberg et al. Cellular with Acute Myeloid Immunotherapies: Leukemia 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 Syndromes - CPX 351 As First Line Clinical and Treatment in Higher Risk Epidemiological: MDS. a Phase II Trial By Pierre Peterlin et al. Treatment of High the GFM 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 Commercially Age Group of Treatment Available with CPX-351 Plus Therapies, Venetoclax in Adults with Excluding Newly Diagnosed AML: Vinod Pullarkat et al. Transplantation andSubgroup Analysis of the Cellular V-FAST Phase 1b Master Immunotherapies: Trial Poster I * Date/Time: Saturday, December 11, 2021: 5:30 PM-7:30 PM * Location: Hall B5

* Type: Poster * Number: 2316 * Session: 615. AcutePhase 1b Study of Myeloid Leukemias: Lower-dose CPX-351 Plus Commercially Venetoclax As First-line Available Treatment for Patients Therapies, with AML Who Are Unfit Geoffrey L. Uy et al. Excluding for Intensive Transplantation andChemotherapy: Preliminary Cellular Safety and Efficacy Immunotherapies: Results Poster 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 Treatment Patterns of Available Patients Diagnosed with Therapies, Therapy-Related AML or Excluding AML-MRC in England Alex Legg et al. Transplantation andbetween 2013 and 2020 Cellular Using the Cancer Analysis Immunotherapies: System Database Poster 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 Real-World Study of Available CPX-351 Treatment Therapies, Outcomes for Acute Alex Legg et al. Excluding Myeloid Leukemia (AML) in Transplantation andEngland Cellular Immunotherapies: Poster II * Date/Time: Sunday, December 12, 2021: 6:00 PM-8:00 PM * Location: Hall B5

* Type: Poster Updated Results of a * Number: 3674 Phase 1/2 Study of Lower * Session: 637. Dose CPX-351 for Patients Myelodysplastic with Int-2 or High Risk Syndromes - IPSS Myelodysplastic Guillermo Montalban-Bravo Clinical and Syndromes and Chronic et al. Epidemiological: Myelomonocytic Leukemia Poster III 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. AcuteDaunorubicin (CPX-351) in Myeloid Leukemias: Combination with Commercially Gemtuzumab Ozogamicin Available (GO) in Relapsed Therapies, Refractory (R/R) PatientsDaniel Rivera et al. Excluding with Acute Myeloid Transplantation andLeukemia (AML) and Cellular Post-Hypomethylating Immunotherapies: Agent (Post-HMA) Failure Poster II High-Risk Myelodysplastic * Date/Time: Sunday, Syndrome (HR-MDS) December 12, 2021: 6:00 PM-8:00 PM * Location: Hall B5

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

DefitelioPresentations

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

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

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

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

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

* Type: Oral Presentation * Number: 672 Use of Defibrotide in * Session: 332. Patients with COVID-19 Anticoagulation and Pneumonia; Results of the Annalisa Ruggeri et al. Antithrombotic Defi-VID19 Phase 2 Trial Therapies * 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.

Important Safety Information

RYLAZE should not be given to people who have had:

* Serious allergic reactions to RYLAZE * Serious swelling of the pancreas (stomach pain), serious blood clots, or serious bleeding during previous asparaginase treatment

RYLAZE may cause serious side effects, including:

* Allergic reactions (a feeling of tightness in your throat, unusual swelling/redness in your throat and/or tongue, or trouble breathing), some of which may be life-threatening * Swelling of the pancreas (stomach pain) * Blood clots (may have a headache or pain in leg, arm, or chest) * Bleeding * Liver problems

Contact your doctor immediately if any of these side effects occur.

Some of the most common side effects with RYLAZE include:liver problems, nausea, bone and muscle pain, tiredness, infection, headache, fever, allergic reactions, fever with low white blood cell count, decreased appetite, mouth swelling (sometimes with sores), bleeding, and too much sugar in the blood.

RYLAZE can harm your unborn baby. Inform your doctor if you are pregnant, planning to become pregnant, or nursing. Females of reproductive potential should use effective contraception (other than oral contraceptives) during treatment and for 3 months following the final dose. Do not breastfeed while receiving RYLAZE and for 1 week after the final dose.

Tell your healthcare provider if there are any side effects that are bothersome or that do not go away.

These are not all the possible side effects of RYLAZE. For more information, ask your healthcare provider.

The full U.S. Prescribing Information for Rylaze is available at: http://pp.jazzpharma.com/pi/rylaze.en.USPI.pdf

About Vyxeos(r) (daunorubicin and cytarabine)

Vyxeos is a liposomal combination of daunorubicin, an anthracycline topoisomerase inhibitor, and cytarabine, a nucleoside metabolic inhibitor, that 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. For more information about Vyxeos in the United States, please visit https://vyxeos.com.

In Europe, Vyxeos(r) Liposomal (daunorubicin/cytarabine) is indicated for the treatment of adults with newly diagnosed, therapy-related acute myeloid leukemia (t-AML) or AML with myelodysplasia-related changes (AML-MRC).

Important Safety Information for Vyxeos(r)

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 were 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(r) (defibrotide sodium)In the U.S., Defitelio(r) (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. Defitelio is not approved for the prevention of VOD.

Please see full Prescribing Information for Defitelio in the United States.

In Europe, defibrotide is marketed under the name Defitelio(r) ? (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. It is not indicated in patients with hypersensitivity to defibrotide or any of its excipients or with concomitant use of thrombolytic therapy.

? 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.

( http://www.ema.europa.eu/ema/index.jsp?curl=/pages/medicines/human/medicines/002393/human_med_001646.jsp)

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.

About Jazz PharmaceuticalsJazz Pharmaceuticals plc (NASDAQ: JAZZ) is a global biopharmaceutical 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 and novel product candidates, from early- to late-stage development, in neuroscience and oncology. Within these therapeutic areas, we are identifying new options for patients by actively exploring small molecules and biologics, and through innovative delivery technologies and cannabinoid science. Jazz is headquartered in Dublin, Ireland and has employees around the globe, serving patients in nearly 75 countries. For more information, please visit www.jazzpharmaceuticals.com and follow @JazzPharma on Twitter.

Jazz Media Contact:Kristin BhavnaniHead of Global Corporate CommunicationsJazz Pharmaceuticals plcCorporateAffairsMediaInfo@jazzpharma.comIreland, +353 1 697 2141U.S., +1 215 867 4948

Jazz Investor Contact:Andrea N. Flynn, Ph.D.Vice President, Head, Investor RelationsJazz Pharmaceuticals plcinvestorinfo@jazzpharma.comIreland, +353 1 634 3211

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