Juno Unveils Promising Early Data on Two CAR T Cell Product Candidates

Juno’s Investigational CAR T Cell Product Candidates JCAR018 and JTCR016 Demonstrate Encouraging Clinical Responses in Patients with B-Cell and Mesothelioma Cancers

SEATTLE--(BUSINESS WIRE)--

Juno Therapeutics, Inc. (JUNO), a biopharmaceutical company focused on re-engaging the body’s immune system to revolutionize the treatment of cancer, today announced, in partnership with its collaborators, early clinical data from two oral presentations for two product candidates at the American Association for Cancer Research (AACR) Annual Meeting 2016 in New Orleans, Louisiana.

JCAR018 is a chimeric antigen receptor (CAR) T cell product candidate targeting CD22, and had data from a Phase I trial in pediatric and young adult patients with relapsed or refractory B-cell acute lymphoblastic leukemia (r/r ALL). JTCR016, a T cell receptor (TCR) cell product candidate targeting Wilms tumor-1 (WT-1), had data from a Phase I trial in patients with acute myelogenous leukemia (AML) at high risk of relapse following an allogeneic hematopoietic stem cell transplant and patients with either mesothelioma or non-small cell lung cancer.

“As we advance our CD19-directed portfolio, we are encouraged by the early signs of clinical activity from product candidates against different targets,” said Mark J. Gilbert, M.D., Juno’s Chief Medical Officer. “The data from JCAR018 suggest two paths to improve outcomes – use in patients with CD19 negative disease and a combination of CD19 and CD22 to decrease the risk of resistant cells and increase the percentage of patients that demonstrate a long-term durable remission in B cell malignancies. Additionally, JTCR016 continues to show an encouraging safety profile and signals of clinical activity, including evidence of tumor reduction as well as significant T cell expansion and persistence in a patient with mesothelioma.”

In an oral presentation on Monday, April 18, 2016, Terry J. Fry, M.D., Investigator, Pediatric Oncology Branch and Head of Hematologic Malignancies Section, National Cancer Institute, National Institutes of Health, presented “CD22 CAR Update and Novel Mechanisms of Leukemic Resistance.” Dr. Fry reported on CD22-directed CAR T cell therapy (JCAR018) in pediatric and young adult patients with r/r B-cell ALL. Key takeaways include:

• Data from nine enrolled and treated patients were reported in this Phase I dose-escalation trial. (JCAR018; Clinical Trials Identifier: NCT02315612).
• As previously reported at the American Society of Hematology meeting in December 2015, six patients were treated at the lowest dose, with one patient achieving a complete remission (CR) and complete molecular remission as measured by flow cytometry (CmR). This patient relapsed after three months.
• Three patients have enrolled at dose level 2 (1 x 10⁶ cells/kg), which is in the dose range of CD19-directed CAR T programs. All three patients achieved a CR and CmR. These patients remain in complete remission with follow-up ranging from 3 to 6 months.
• The complete remissions have been seen in both patients naïve to CAR T therapies as well as those with CD19 negative relapse after prior CD19-directed CAR T therapy.
• Limited cytokine release syndrome (CRS) was seen at dose level 2, with two patients at Grade 1 and one patient at Grade 2. No severe neurotoxicity was observed in these treatment cohorts. Dose limiting toxicity was observed at higher doses, so dosing continues at dose level 2 (1 x 10⁶ cells/kg).

In an oral presentation on Wednesday, April 20, 2016, Phil Greenberg, M.D., Head of Program in Immunology at the Fred Hutchinson Cancer Research Center and Professor, Medicine/Oncology and Immunology, University of Washington, presented “Targeting Cancer with Engineered T Cells.” Dr. Greenberg reported on WT-1 TCR cell therapy (JTCR016) in refractory mesothelioma and AML. Key takeaways include:

• In a Phase I/II study designed to evaluate genetically modified T cells targeting WT-1 in WT-1-expressing non-small cell lung cancer (NSCLC) and mesothelioma using a WT-1-specific T-cell receptor, WT-1 TCR (JTCR016; Clinical Trials Identifier: NCT02408016), there have been five patients enrolled.
• Three patients have been treated to date. Preliminary data show one mesothelioma patient with an ongoing partial response to the WT-1 TCR and one with stable disease. The responses appear to correlate with the pharmacokinetics of the engineered T cells, as the patient with the partial response had the best T cell expansion and persistence. The patient had progressed after multiple therapies, including chemotherapy and radiation, prior to receiving JTCR016.
• JTCR016 was generally well-tolerated in these three patients, with no evidence of severe CRS or severe neurotoxicity.
• In a Phase I dose-escalation trial in patients with AML following allogeneic hematopoietic stem cell transplantation, 11 patients with no measurable disease but at high risk of relapse have been treated to date. JTCR016 continues to be relatively well-tolerated with prolonged persistence of the engineered T cells and no relapses to date.
• This AACR 2016 Major Symposium presentation also highlighted encouraging pre-clinical data from a mesothelin-directed TCR for the treatment of pancreatic cancer, demonstrating the potential of these therapies in treating solid tumors, and next-generation strategies to make these T cells more potent.

About Juno’s Chimeric Antigen Receptor (CAR) and T Cell Receptor (TCR) Technologies

Juno’s CAR and TCR technologies genetically engineer T cells to recognize and kill cancer cells. Juno’s CAR T cell technology inserts a gene for a particular CAR into the T cell, enabling it to recognize cancer cells based on the expression of a specific protein located on the cell surface. Juno’s TCR technology provides the T cells with a specific T cell receptor to recognize protein fragments derived from either the surface or inside the cell. When either type of engineered T cell engages the target protein on the cancer cell, it initiates a cell-killing response against the cancer cell. JCAR018 and JTCR016 are investigational product candidates and their safety and efficacy have not been established.

About Juno

Juno Therapeutics is building a fully integrated biopharmaceutical company focused on re-engaging the body’s immune system to revolutionize 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 solid tumors. 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, 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.

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, clinical benefits, clinical trial results and the implications thereof, presentations at AACR, clinical trial plans and emerging medical trends. 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 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 Biotechnology (Shanghai) Co., Ltd, over which Juno does not exercise complete control, 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 Annual Report on Form 10-K filed with the Securities and Exchange Commission on February 29, 2016 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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