Juno Therapeutics Highlights Key Translational Insights With JCAR017 in Patients With DLBCL

SEATTLE--(BUSINESS WIRE)-- Juno Therapeutics (NASDAQ:JUNO), a biopharmaceutical company developing innovative cellular immunotherapies for the treatment of cancer, today presented new translational insights on clinical outcomes with its investigational CAR T product candidate JCAR017 at the 59^th American Society of Hematology (ASH) Annual Meeting and Exposition.

Juno also announced that JCAR017, a defined composition CD19-directed CAR T cell product candidate using a 4-1BB costimulatory domain, had received the United States Adopted Name lisocabtagene maraleucel (liso-cel).

“Today’s presentations suggest that the defined composition of JCAR017 supports a potential best-in-class clinical profile and helps to unlock key translational insights,” said Sunil Agarwal, M.D., Juno’s President of Research and Development. “These insights will help guide our next generation strategies to further improve durable responses.”

Tanya Siddiqi, M.D., of City of Hope National Medical Center presented insights from ASH Abstract #193, which analyzed patients enrolled in the diffuse large B-cell lymphoma (DLBCL) cohort of the TRANSCEND trial of JCAR017.

Results from these exploratory analyses suggest that patient factors may impact safety and efficacy for DLBCL patients. These factors included:

• Baseline patient characteristics, including high tumor burden and markers of inflammation, were associated with high CAR T cell expansion and increased rates of cytokine release syndrome (CRS) and neurotoxicity (NT).
• Data showed an approximately 8-fold increased risk for CRS and NT in patients with high baseline tumor burden.
• Baseline markers of inflammation were associated with more durable responses; with respect to tumor burden the association was less pronounced.

In a separate analysis, Howard Stern, M.D., Ph.D. of Juno Therapeutics, presented findings from ASH Abstract #194, examining JCAR017 infiltration into tumor tissue and exploring potential mechanisms of resistance and relapse. Results showed that JCAR017 CD4 and CD8 CAR T cells infiltrated tumors post-treatment. CAR T cell infiltration trended higher in patients who achieved a response. At disease progression, CAR T cells were rare or absent in tumor tissue despite the presence of CD19 and persistence of peripheral blood CAR T cells in most patients. There does not yet appear to be a singular resistance pathway upregulated in the tumor at the time of progression, but well-known pathways such as PD-L1 and IDO were upregulated in different patients. These data suggest that combinations with other immunotherapies may be beneficial to further improve outcomes with JCAR017 therapy. Juno and Celgene are conducting an ongoing combination trial with JCAR017 and durvalumab, an anti-PD-L1 antibody.

About Juno

Juno Therapeutics is building a fully integrated biopharmaceutical company focused on developing innovative cellular immunotherapies for the treatment of cancer. Founded on the vision that the use of human cells as therapeutic entities will drive one of the next important phases in medicine, Juno is developing cell-based cancer immunotherapies based on chimeric antigen receptor and high-affinity T cell receptor technologies to genetically engineer T cells to recognize and kill cancer. Juno is developing multiple cell-based product candidates to treat a variety of B-cell malignancies as well as multiple solid tumors and multiple myeloma. Several product candidates have shown compelling clinical responses in clinical trials in refractory leukemia and lymphoma conducted to date. Juno’s long-term aim is to leverage its cell-based platform to develop new product candidates that address a broader range of cancers and human diseases. Juno brings together innovative technologies from some of the world’s leading research institutions, including the Fred Hutchinson Cancer Research Center, Memorial Sloan Kettering Cancer Center, Seattle Children’s Research Institute (SCRI), the University of California, San Francisco, and The National Cancer Institute. Juno Therapeutics has an exclusive license to the St. Jude Children’s Research Hospital patented technology for CD19-directed product candidates that use 4-1BB, which was developed by Dario Campana, Chihaya Imai, and St. Jude Children’s Research Hospital. Juno’s product candidate JCAR017 (lisocabtagene maraleucel; liso-cel) was developed in collaboration with SCRI and others. JCAR017 is an investigational CAR T therapy and is not approved for use in any country.

About The Juno-Celgene Collaboration

Celgene Corporation and Juno Therapeutics formed a collaboration in June 2015 under which the two companies will leverage T cell therapeutic strategies to develop treatments for patients with cancer and autoimmune diseases with an initial focus on chimeric antigen receptor (CAR) and T cell receptor (TCR) technologies. In April 2016, Celgene exercised its option to develop and commercialize the Juno CD19 program outside North America and China.

Forward-Looking Statements

This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, Section 27A of the Securities Act of 1933, and Section 21E of the Securities Exchange Act of 1934, including statements regarding Juno’s mission, progress, and business plans; clinical or translational data and the implications thereof; the potential best-in-class profile for JCAR017 (liso-cel); the importance of controlling product composition; and the potential of Juno to apply these learnings to further improve durable responses. Forward-looking statements are subject to risks and uncertainties that could cause actual results to differ materially from such forward-looking statements, and reported results should not be considered as an indication of future performance. These risks and uncertainties include, but are not limited to, risks associated with: the success, cost, and timing of Juno’s product development activities and clinical trials; Juno’s ability to obtain regulatory approval for and to commercialize its product candidates; Juno’s ability to establish a commercially-viable manufacturing process and manufacturing infrastructure; regulatory requirements and regulatory developments; success of Juno’s competitors with respect to competing treatments and technologies; Juno’s dependence on third-party collaborators and other contractors in Juno’s research and development activities, including for the conduct of clinical trials and the manufacture of Juno’s product candidates; Juno’s ability to attract and retain key scientific, quality control/assurance, manufacturing or management personnel; Juno’s dependence on Celgene for the development and commercialization outside of North America and China of Juno’s CD19 product candidates and any other product candidates for which Celgene exercises an option; Juno’s dependence on JW Therapeutics (Shanghai) Co., Ltd and its affiliates for the development and commercialization of product candidates in China; Juno’s ability to obtain, maintain, or protect intellectual property rights related to its product candidates; amongst others. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to Juno’s business in general, see Juno’s Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on November 1, 2017 and Juno’s other periodic reports filed with the Securities and Exchange Commission. These forward-looking statements speak only as of the date hereof. Juno disclaims any obligation to update these forward-looking statements.

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