This report provides a comprehensive list of events and catalysts that were added or updated at the 33rd Annual J.P. Morgan Healthcare Conference in San Francisco. A free copy can be downloaded via the form on the right. Key Events Included in this Report:

• MM-121 for Breast Cancer (MACK). Merrimack announced plans to initiate heregulin (HRG) biomarker-selected enrollment for a Phase II trial for MM-121. This trial will enroll HRG+/HER2- metastatic breast cancer patients for second-line treatment. The company also guided that its two ongoing Phase II trials of MM-121 are potentially registrational.
• MM-398 for Pancreatic Cancer (MACK). Merrimack highlighted its lead pipeline asset, MM-398 including an announcement that a rolling NDA had been initiated in pancreatic cancer with a completed filing anticipated in late Q1 or early Q2 2015. The presentation emphasized that approval is being sought for all post-gemcitabine use, not just second-line or later since gemcitabine can be used as adjuvant therapy. A partnership with Baxter is also specifically evaluating MM-398 in the front-line pancreatic cancer setting. Finally, evaluation of MM-398 as a front-line gastric cancer therapy has also restarted after an apparent halt to development, and a Phase II study in gastric cancer is anticipated in to initiate in 2015.
• Non-viral CAR-T Program (XON). Intrexon, Ziopharm (ZIOP), and MD Anderson announced a collaboration to develop a new CAR-T therapy. The program licenses technology from MD Anderson to generate CAR-T cells through a non-viral DNA/transposon-based genetic engineering approach as opposed to the more common viral gene transfer techniques. The non-viral approach could lead to differentiation from the increasingly crowded CAR-T landscape by reducing or eliminating the T-cell expansion time with directly infused gene-tranferred cells expanding in the patient. This could potentially reduced the treatment delay to hours instead of weeks. The JPM presenter also suggested it could lead to off-the-shelf (allogeneic) products, but it was unclear how those would be differentiated from any generated by traditional viral gene transfer. The collaboration is expected to generate up to five CARs entering the clinic in 2015 with off-the-shelf programs starting in 2016.
• CAR-T Therapies for Cancer (JUNO). Juno provided updates for its early-stage development pipeline, including two preclinical CAR-T products targeting solid tumors. One CAR-T product for ovarian cancer targets MUC-16 (which is cleaved to generate CA-125) and utilizes the company’s “armored CAR” technology to express the immunostimulatory cytokine IL-12; a Phase I trial will begin in 2015. A second CAR-T program set to start a Phase I trial in 2015 targets ROR-1, an embryonic antigen expressed on some lymphomas and epithelial cancers.
• LentiGlobin for Sickle Cell Anemia (BLUE). The bluebird presentation was fairly uneventful, especially following on the excitement surrounding the company’s LentiGlobin results for beta-thalassemia at the recent ASH meeting. Nevertheless, CEO Nick Leschly did reiterate how the same efficacy in beta-thalassemia should translate to success in treating sickle cell disease, and a Phase I SCD study has already initiated. Along with all the other CAR-T therapy news at JPM, bluebird indicated that its own CAR-T program should enter the clinic by early 2016.

For the full report, please download the PDF version at the top of this page.

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