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Research and Development

At Jazz, we use patient-centric innovation to drive our research and development (R&D) strategy, investment and growth. This starts with identifying unmet needs across our therapeutic areas of focus—neuroscience and oncology—and take the best discovery and development approach to solutions, whether that leverages our proprietary, industry-defining research platforms or our collaborations with leading investigators and bioscience companies.

We believe all people, including those who live with complex conditions, deserve new and better therapeutic options. Our teams use their expertise to lead clinical programs across sleep, epilepsy, movement disorders, psychiatry and neurodegenerative diseases, as well as blood and solid tumor cancers. We are committed to innovation, leveraging both our market-defining cannabinoid science capabilities and other proprietary platforms. As part of our commitment to patients with unmet needs, we continually work to expand the science behind our therapies, acquire rights to develop new medicines, and partner with researchers, academia and other industry leaders to advance molecules with great potential to create new or better standards of care.

Pipeline

PRE-CLINICAL
Undisclosed targets
Neuroscience
CombiPlex
Exploratory activities
Undisclosed target
Ras/Raf/MAP kinase pathway1
Exosome targets
(Up to 3 targets)1
Hematological malignancies/solid tumors
JZP898 (WTX-613)2
Conditionally-activated IFNα
Undisclosed targets
Oncology
Undisclosed targets
Cannabinoids
PHASE 1
JZP3243
Oxybate extended-release formulation
JZP441 (DSP-0187)2
Orexin-2 receptor agonist
JZP815
Pan-Raf Inhibitor program
Raf & Ras mutant tumors
Zanidatamab2
HER2-targeted bispecific antibody
Breast cancer
JZP341
(Long-acting Erwinia asparaginase)
Solid tumors
JZP351 (Vyxeos)
Low Intensity Dosing for higher risk MDS4
JZP351 + other approved therapies
  • R/R AML or HMA Failure MDS4
  • First-line, fit AML (Phase 1b)
  • Low Intensity Therapy for first-line, unfit AML (Phase 1b)
Additional Cannabinoids
Neonatal hypoxic-ischemic encephalopathy
Additional Cannabinoids
Neuropsychiatry targets
PHASE 2
Suvecaltamide (JZP385)
Essential tremor
Suvecaltamide (JZP385)
Parkinson’s Disease Tremor
JZP1505
PTSD
Zanidatamab2
HER2-targeted bispecific antibody
  • 2L zani monotherapy for BTC6 (Pivotal trial)
  • Additional trials ongoing in BTC, GEA and CRC
  • Multiple trials ongoing in breast cancer
Lurbinectedin (Zepzelca)
Basket trial: advanced urothelial cancer, large cell neuroendocrine tumor of the lung, HRD+ cancers
JZP351
  • HR-MDS (EMSCO)7
  • Newly diagnosed older adults with HR-AML7
JZP351 + venetoclax
de novo or R/R AML4
JZP541

Irritability associated with autism spectrum disorders
Additional Cannabinoids

Autism spectrum disorders
PHASE 3
Zanidatamab2
HER2-targeted bispecific antibody
1L zani + chemo ± tislelizumab for GEA8
(Pivotal trial)

Lurbinectedin1
1L treatment SCLC in combination with Tecentriq® (atezolizumab)
JZP351
  • AML or HR-MDS >60 yrs (AML18)7
  • Newly diagnosed adults with standard- and HR-AML (AMLSG)7
  • Newly diagnosed <22 yrs with AML (COG)7
Cannabidiol (Epidiolex)
EMAS

Cannabidiol (Epidyolex)
LGS, DS, TSC in Japan

REGULATORY
JZP458 (Rylaze)9
(recombinant Erwinia asparaginase)
ALL/LBL
Neuroscience
Oncology
Cannabinoids
1Partnered collaboration;
2Recently acquired;
3Planned;
4Jazz & MD Anderson Cancer Center collaboration study;
5JZP150 is a fatty acid amide hydrolase inhibitor which modulates the endocannabinoid anandamide;
6HERIZON-BTC-01;
7Cooperative group study;
8HERIZON-GEA-01;
9FDA approval on June 30, 2021 and FDA approval of sBLA for MWF IM dosing on November 18, 2022. In February 2023, received a complete response letter from FDA requesting additional clinical data on the IV administration sBLA; currently evaluating complete response letter to determine next steps. Submitted Marketing Authorization Application to EMA.

1L = first line, ALL/LBL = acute lymphoblastic leukemia / lymphoblastic lymphoma, AML = acute myeloid leukemia, COG = Children’s Oncology Group, BTC = biliary tract cancer, CRC = colorectal cancer, DS = Dravet syndrome, EMAS = epilepsy with myoclonic-atonic seizures, GEA = gastroesophageal adenocarcinoma, HMA = hypomethylating agents, HR = high-risk, HRD+ = homologous recombinant deficient positive, LGS = Lennox-Gastaut syndrome, MDS = myelodysplastic syndromes, PTSD = post-traumatic stress disorder, R/R = relapsing/refractory, SCLC = small cell lung cancer, SG = study group, TSC = Tuberous sclerosis complex.

Through Jazz’s patented CombiPlex® platform, Jazz is evaluating oncology combinations to support identification of clinical product candidate(s) and is leading a collaborative approach with partners to research and develop new therapies.