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Jazz Pharmaceuticals & Codiak BioSciences Announce Strategic Collaboration to Research, Develop, and Commercialize Engineered Exosomes to Create Therapies for Hard-to-Treat Cancers; Codiak to Receive $56 Million Upfront & Up to $1 Billion in Milestones

On January 3, 2018, Jazz Pharmaceuticals plc (Nasdaq: JAZZ) and Codiak BioSciences, Inc., announced that the companies have entered into a strategic collaboration agreement focused on the research, development, and commercialization of exosome therapeutics to treat cancer. Codiak granted Jazz an exclusive, worldwide, royalty-bearing license to develop, manufacture, and commercialize therapeutic candidates directed at five targets to be developed using Codiak’s engEx™ precision engineering platform for exosome therapeutics. The targets focus on oncogenes that have been well validated in hematological malignancies and solid tumors, but have been undruggable with current modalities, including NRAS and STAT3. Under the terms of the agreement, Codiak is responsible for the execution of pre-clinical and early clinical development of therapeutic candidates directed at all five targets through Phase 1/2 proof-of-concept studies. Following the conclusion of the applicable Phase 1/2 study, Jazz will be responsible for future development, potential regulatory submissions, and commercialization for each product. Codiak has the option to participate in co-commercialization and cost/profit-sharing in the U.S. and Canada on up to two products. As part of the agreement, Jazz will pay Codiak an upfront payment of $56 million. Codiak is eligible to receive up to $20 million in preclinical development milestone payments across all five programs. Codiak is also eligible to receive milestone payments totaling up to $200 million per target based on Investigational New Drug application acceptance, clinical and regulatory milestones, including approvals in the U.S., European Union and Japan, and sales milestones. Codiak is also eligible to receive tiered royalties on net sales of each approved product, with percentages ranging from mid-single digits in the lowest tier to high-teens in the highest tier.

“We are looking forward to partnering with Codiak and their world-class management and R&D team. Their innovative exosome platform offers the opportunity to address targets that have been frequently implicated in hematological malignancies and solid tumors, including targets recognized as undruggable with current approaches,” said Bruce Cozadd, Chairman and CEO of Jazz Pharmaceuticals.

“The exosome approach is differentiated and represents the ideal complement to our CombiPlex® platform and other collaboration programs. The ability to develop multiple new therapies through these technologies is an important advance as we seek to add long-term value for patients and shareholders.”

“We are excited to be working with the team at Jazz to bring a new class of medicines into clinical testing to treat patients with cancer,” said Douglas E. Williams, PhD, President and CEO of Codiak. “We believe that the Codiak engEx™ platform, along with Jazz’s insights into relevant, but intractable, targets, creates a unique opportunity to improve patient outcomes in various cancers. Codiak’s proprietary engEx™ platform allows us to deliver potent therapeutics in a precise, targeted, and tunable way. This collaboration allows Codiak to accelerate the potential of our engEx™ platform, as we advance our lead candidates and core pipeline.”

Exosomes are naturally occurring, nanometer-sized vesicles that are released and received by nearly all cells in the body. They serve as an efficient system of intercellular communication. In the tumor microenvironment, exosomes have been shown to carry molecular messages between tumor and immune cells. Codiak has co-opted this process and, through its engEx™ platform, engineers potent drug molecules into the exosome to create bespoke therapeutic candidates which have shown potent preclinical antitumor activity.

With the engEx™ platform, Codiak has the ability to incorporate a range of therapeutic drug classes—including small molecules, proteins, peptides, cytokines, and nucleic acids—onto the surface or in the lumen of its therapeutic exosomes. engEx™ also enables precise modifications of the engineered exosomes to optimize potency and to alter or enhance tropism for directed delivery to desired cell types. These flexible design features allow Codiak to create an optimized exosome therapeutic for the drug target and desired site of action.


Jazz Pharmaceuticals and Codiak will host an investor conference call and live audio webcast on Friday, January 4, 2019 at 8:30 a.m. EST (1:30 p.m. GMT) to discuss the collaboration. The live webcast may be accessed from the Investors section of the Jazz Pharmaceuticals website at Please connect to the website prior to the start of the conference call to ensure adequate time for any software downloads that may be necessary. Investors may participate in the conference call by dialing +1 855 353 7924 in the US, or +1 503 343 6056 outside the US, and entering passcode 9698918.

A replay of the conference call will be available through January 11, 2019 by dialing +1 855 859 2056 in the US, or +1 404 537 3406 outside the US, and entering passcode 9698918. An archived version of the webcast will be available for at least one week in the Investors section of the Jazz Pharmaceuticals website at


Jazz Pharmaceuticals plc (Nasdaq: JAZZ) is an international biopharmaceutical company focused on improving patients’ lives by identifying, developing, and commercializing meaningful products that address unmet medical needs. The company has a diverse portfolio of products and product candidates with a focus in the areas of sleep and hematology/oncology. In these therapeutic areas, Jazz Pharmaceuticals markets Xyrem® (sodium oxybate) oral solution, Erwinaze® (asparaginase Erwinia chrysanthemi), Defitelio® (defibrotide sodium), and Vyxeos® (daunorubicin and cytarabine) liposome for injection in the U.S., and markets Erwinase®, Defitelio® (defibrotide) and Vyxeos® 44 mg/100 mg powder for concentrate for solution for infusion in countries outside the U.S. For more information, please visit and follow us on Twitter at @JazzPharma.


Codiak BioSciences is a leader in the emerging field of exosome therapeutics. Founded in 2015 and headquartered in Cambridge, Massachustts, Codiak has built a proprietary technology platform for exosome engineering and manufacturing that allows for precise therapeutic targeting — opening the door to the development of therapies to treat a diverse array of diseases that are currently difficult or impossible to treat using existing methodologies. Codiak BioSciences is taking advantage of the natural propensity of exosomes to deliver macromolecule payloads either on or in the exosome, to cells, to treat a variety of human diseases. The company was formed on the basis of research from the VentureLabs innovation unit of Flagship Pioneering; the research of Jan Lotvall, MD, PhD, Chairman of the Krefting Research Centre at University of Gothenburg, Sweden; and Raghu Kalluri, MD, PhD, at The University of Texas MD Anderson Cancer Center in Houston. For more information, visit


Exosomes are nanometer-sized membrane sacs, or vesicles, that are released and received by nearly all cells. Exosomes are an ancient system of intercellular communication, through which they can deliver various molecules (DNA, RNA, proteins, lipids) from cell to cell. Upon delivery, exosomal cargo can change biological functions in recipient cells according to the instructions encoded by the payload. Exosomes traffic to specific cellular targets, thereby offering the potential to deliver diverse therapies to specific cells and modulate cellular function to achieve therapeutic benefit.

[Press release] [Jazz Pharmaceuticals] [Codiak BioSciences]