Jazz Pharmaceuticals Advances Recombinant Crisantaspase Development Program

Positive Phase 1 study of JZP-458 completed

Pivotal Phase 2/3 study expected to initiate later this year in collaboration with Children's Oncology Group

DUBLIN, Ireland I August 6, 2019 I Jazz Pharmaceuticals plc (Nasdaq: JAZZ) today announced that the Phase 1 study of its recombinant crisantaspase molecule, JZP-458, met its efficacy and safety objectives. The company plans to initiate a single-arm, pivotal Phase 2/3 study evaluating JZP-458 as a potential treatment option for patients with acute lymphoblastic leukemia (ALL) or lymphoblastic lymphoma (LBL) who are hypersensitive to E. coli-derived asparaginase products.

"Jazz is committed to the ALL patient community, and we are pleased to advance this development program with the goal of bringing a new treatment option to ALL and LBL patients who are hypersensitive to E. coli-derived asparaginase products as soon as possible," said Robert Iannone, M.D., M.S.C.E., executive vice president, research and development of Jazz Pharmaceuticals. "Following a meeting with the U.S. Food and Drug Administration, we are finalizing the Phase 2/3 study protocol in collaboration with the Children's Oncology Group and plan to initiate the study later this year."

A recombinant crisantaspase Phase 1 study in healthy volunteers in the U.S. met safety and efficacy objectives with efficacy based on measurement of serum asparaginase activity levels. Results of this Phase 1 study will be submitted for presentation at an upcoming medical meeting.

About JZP-458
JZP-458 is a recombinant crisantaspase that uses a novel Pseudomonas fluorescens expression platform. It is being developed for use as a component of a multi-agent chemotherapeutic regimen in the treatment of pediatric and adult patients with acute lymphoblastic leukemia (ALL) or lymphoblastic lymphoma (LBL) who are hypersensitive to E. coli-derived asparaginase products. A Phase 1 study of healthy volunteers was recently completed, and a single-arm, pivotal Phase 2/3 study is planned for initiation later in 2019.

About Acute Lymphoblastic Leukemia (ALL)
Acute Lymphoblastic Leukemia (ALL) is a cancer of the blood and bone marrow that can progress quickly if not treated.1 Leukemia is the most common cancer in children, and about three out of four of these cases are ALL.2 Adults can also develop ALL, and about four of every 10 cases of ALL diagnosed are in adults.3 The American Cancer Society estimates that almost 6,000 new cases of ALL will be diagnosed in the U.S. in 2019.4 Asparaginase is a core component of multi-agent chemotherapeutic regimens in ALL.5  However, asparaginase treatments derived from E. coli are associated with the development of hypersensitivity reactions.6

About Jazz Pharmaceuticals plc
Jazz Pharmaceuticals plc (Nasdaq: JAZZ), a global biopharmaceutical company, is dedicated to developing life-changing medicines for people with limited or no options. As a leader in sleep medicine and with a growing hematology/oncology portfolio, Jazz has a diverse portfolio of products and product candidates in development, and is focused on transforming biopharmaceutical discoveries into novel medicines. Jazz Pharmaceuticals markets Sunosi™ (solriamfetol), Xyrem® (sodium oxybate) oral solution, Defitelio® (defibrotide sodium), Erwinaze® (asparaginase Erwinia chrysanthemi) and Vyxeos® (daunorubicin and cytarabine) liposome for injection in the U.S. and markets Defitelio® (defibrotide), Erwinase® and Vyxeos® 44 mg/100 mg powder for concentrate for solution for infusion in countries outside the U.S. For country-specific product information, please visit www.jazzpharmaceuticals.com/medicines. For more information, please visit www.jazzpharmaceuticals.com and follow us on Twitter at @JazzPharma.

1 National Cancer Institute. Adult Acute Lymphoblastic Leukemia Treatment (PDQ®)–Patient Version. Available at www.cancer.gov/types/leukemia/patient/adult-all-treatment-pdq. Accessed August 6, 2019.
2 American Cancer Society. Key Statistics for Childhood Leukemia. Available at www.cancer.org/cancer/leukemia-in-children/about/key-statistics.html. Accessed August 6, 2019.
3 American Cancer Society. Key Statistics for Acute Lymphocytic Leukemia (ALL). Available at www.cancer.org/cancer/acute-lymphocytic-leukemia/about/key-statistics.html. Accessed August 6, 2019.
4 American Cancer Society. Key Statistics for Acute Lymphocytic Leukemia (ALL). Available at: www.cancer.org/cancer/acute-lymphocytic-leukemia/about/key-statistics.html. Accessed August 6, 2019.
5 Salzer W, Bostrom B, Messinger Y et al. 2018. Asparaginase activity levels and monitoring in patients with acute lymphoblastic leukemia. Leukemia & Lymphoma. 59:8, 1797-1806, DOI: 10.1080/10428194.2017.1386305.
6 Hijiya N, van der Sluis IM. Asparaginase-associated toxicity in children with acute lymphoblastic leukemia. Leuk Lymphoma. 2016;57(4):748–757. doi:10.3109/10428194.2015.1101098

SOURCE: Jazz Pharmaceuticals

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