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Jazz Starts CAR T-cell Linked Neurotoxicity Study Enrollment

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Jazz Pharmaceuticals plc (JAZZ - Free Report) announced that it has started enrollment in an exploratory phase II study to evaluate defibrotide for the prevention of CAR T-cell associated neurotoxicity in patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL). The patients are enrolled in the study are being treated with Gilead’s GILD CAR-T therapy, Yescarta (axicabtagene ciloleucel), which is approved for treating relapsed/refractory DLBCL. Please note taht, Yescarta comes with a boxed warning for neurologic toxicities, which can be fatal.

Jazz commercializes defibrotide under the trade name of Defitelio for treating hepatic veno-occlusive disease with renal or pulmonary dysfunction following hematopoietic stem-cell transplantation under the tradename of Defitelio.

The two-part exploratory phase II study will first evaluate the safety of a 2.5 mg/kg and a 6.25 mg/kg dose of defibrotide in patients. The second part of the study will continue with the recommended dose based on data from the first part.

Jazz’s stock has lost 4.8% in the year so far compared with 1.8% decline of the industry.


CAR-T therapy is an emerging immunotherapy approach for treating cancer, especially hematologic malignancies. However, long-term impact of the treatment is yet to be evaluated. The therapy uses patients’ cells to identify and destroy cancer cells, thereby making it different from other small molecule or biologic therapies.

During the treatment, T cells are drawn from a patient's blood. These cells are then reprogramed in the manufacturing facility to create genetically coded cells. This leads to the creation of a chimeric antigen receptor, which recognizes and fights cancer cells and other B cells expressing a specific antigen. However, CAR-T therapies can damage certain cells which compromise the body’s ability to protect the central nervous system, causing neurotoxicity.

Apart from Yescarta, another marketed CAR-T therapy is Novartis’ Kymriah. Meanwhile, a few pharma companies are developing CAR-T therapies targeting multiple oncology indications, which include Gilead, bluebird bio BLUE and ZIOPHARM Oncology ZIOP.


Zacks Rank

Jazz currently sports a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

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