Jazz Pharmaceuticals Announces First Patient Enrolled in Pivotal Phase 2/3 Study Evaluating JZP-458 for the Treatment of Acute Lymphoblastic Leukemia or Lymphoblastic Lymphoma

Pivotal Phase 2/3 study is being conducted in collaboration with Children's Oncology Group

DUBLIN, Ireland I December 30, 2019 I Jazz Pharmaceuticals plc (Nasdaq: JAZZ) today announced that the first patient has been enrolled in the pivotal Phase 2/3 clinical study for JZP-458, a recombinant Erwinia asparaginase molecule that uses a novel Pseudomonas fluorescens expression platform. The study, conducted in collaboration with the Children's Oncology Group (COG), is evaluating JZP-458 as a potential treatment for pediatric and adult patients with acute lymphoblastic leukemia (ALL) or lymphoblastic lymphoma (LBL) who are hypersensitive to E. coli-derived asparaginases. Hypersensitivity reactions affect up to 30 percent of patients with ALL and LBL who are treated with E. coli-derived asparaginase.1

"We're pleased to collaborate with Jazz on this important study," said Dr. Mignon Loh, professor of pediatrics at the University of California San Francisco (UCSF), Deborah and Arthur Ablin Endowed Chair in Pediatric Molecular Oncology and COG's Acute Lymphoblastic Leukemia Disease Committee Chair.

"This clinical trial represents a tremendously important effort as it is investigating a novel asparaginase, JZP-458, which can be critically important for the treatment of some children with ALL, the most common type of childhood malignancy," stated Dr. Luke Maese, assistant professor at the University of Utah, Primary Children's Hospital and Huntsman Cancer Institute.

The single-arm, open-label, multicenter, dose confirmation and confirmatory study of JZP-458 will evaluate pediatric and adult patients with ALL or LBL who have silent inactivation or an allergic reaction to E. coli-derived asparaginases and have not previously received asparaginase Erwinia chrysanthemi. This study is designed to assess the safety, tolerability and efficacy of JZP-458 and is expected to enroll patients in approximately 60 COG institutions in the U.S. and Canada. The primary objective of the study is to determine the efficacy of JZP-458 measured by asparaginase activity.

"When undergoing treatment for ALL with asparaginase, it is critically important for patients to receive all of the necessary doses to maintain therapeutic levels throughout their regimen, something not always possible for patients who have an allergy to E. coli-derived asparaginase," said Robert Iannone, M.D., M.S.C.E., executive vice president, research and development of Jazz Pharmaceuticals. "Our ongoing collaboration with COG for this JZP-458 study, and the receipt of Fast Track designation from the U.S. Food and Drug Administration in October, are significant because they could potentially allow us to more quickly address this need with a new asparaginase option. Jazz is committed to addressing unmet needs for patients with hematologic cancers and the continued expansion of our asparaginase franchise is an important component of our development programs."

Additional information about the trial, including eligibility criteria and a list of clinical trial sites, can be found at: https://clinicaltrials.gov (ClinicalTrials.gov Identifier: NCT04145531).

About JZP-458
JZP-458 is a recombinant Erwinia asparaginase 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. JZP-458 was granted Fast Track designation by the U.S. Food and Drug Administration in October 2019 for the treatment of this patient population.

About Acute Lymphoblastic Leukemia
Acute lymphoblastic leukemia (ALL) is a cancer of the blood and bone marrow that can progress quickly if not treated.2 Leukemia is the most common cancer in children, and about three out of four of these cases are ALL.3 Although it is one of the most common cancers in children, ALL is among the most curable of the pediatric malignancies due to recent advancements in treatment.4,5 Adults can also develop ALL, and about four of every 10 cases of ALL diagnosed are in adults.6 The American Cancer Society estimates that almost 6,000 new cases of ALL will be diagnosed in the United States in 2019.6 Asparaginase is a core component of multi-agent chemotherapeutic regimens in ALL.7 However, asparaginase treatments derived from E. coli are associated with the potential for development of hypersensitivity reactions.8

About the Children's Oncology Group
The Children's Oncology Group (www.childrensoncologygroup.org) is the world's largest organization devoted exclusively to childhood and adolescent cancer research. The Children's Oncology Group (COG) unites almost 10,000 experts in childhood cancer at more than 200 leading children's hospitals, universities, and cancer centers across United States, Canada, Australia, New Zealand, and parts of world in the fight against childhood cancer. Today, more than 90% of the 14,000 children and adolescents diagnosed with cancer each year in the United States are cared for at COG member institutions. Research performed by the COG institutions over the past fifty years has transformed childhood cancer from a virtually incurable disease to one with a combined 5-year survival rate of 80%. COG's mission is to improve the cure rate and outcome for all children with cancer.

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® liposomal 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.

References:

1. Vrooman et al. Erwinia Asparaginase after Allergy to E. coli Asparaginase in Children with Acute Lymphoblastic Leukemia. Pediatr Blood Cancer. 2010 February; 54(2): 199–205. doi:10.1002/pbc.22225.
2. National Cancer Institute. Adult Acute Lymphoblastic Leukemia Treatment (PDQ®)–Patient Version. Available at www.cancer.gov/types/leukemia/patient/adult-all-treatment-pdq. Accessed December 27, 2019. 
3. American Cancer Society. Key Statistics for Childhood Leukemia. Available at www.cancer.org/cancer/leukemia-in-children/about/key-statistics.html. Accessed December 27, 2019.
4. American Cancer Society. Cancer Facts & Figures 2019. www.cancer.org/research/cancer-facts-statistics/all-cancer-facts-figures/cancer-facts-figures-2019.html. Accessed December 27, 2019.
5. Pui C, Evans W. A 50-Year Journey to Cure Childhood Acute Lymphoblastic Leukemia. Seminars in Hematology. 2013;50(3), 185-196. 
6. American Cancer Society. Key Statistics for Acute Lymphocytic Leukemia (ALL). Available at www.cancer.org/cancer/acute-lymphocytic-leukemia/about/key-statistics.html. Accessed December 27, 2019.
7. 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.
8. 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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