July 12, 2017
Today is a milestone day in the development of CAR T cell therapy. The Oncologic Drugs Advisory Committee (ODAC) is convening a hearing for the consideration of Novartis’s tisagenlecleucel in pediatric and young adults with relapsed/refractory (r/r) acute lymphoblastic leukemia (ALL). This new class of therapies has the potential to become a foundational therapeutic in the fight against hematological malignancies, including adult ALL, aggressive r/r Non Hodgkin Lymphoma, chronic lymphocytic leukemia and multiple myeloma.
Data recently announced from several companies in the field, including Juno, show substantial improvements over existing therapies with unprecedented response rates and apparent long-term benefit. The discussion at this ODAC may shape the development and innovation of this nascent treatment paradigm.
The rapidly evolving science of CAR T cells and advances in the methods by which these therapies are designed, manufactured, and administered should significantly improve their clinical profiles. As with other targeted and immuno-oncology agents, CAR T cell products showing high response rates in early trials are likely good candidates for alternative endpoints and trial designs to support rapid approval. The personalized nature of CAR T cell products also presents an attractive opportunity to use Real World Evidence to support ongoing clinical development and label expansion. We strongly encourage the FDA to work with sponsors to enhance their understanding of these products and explore novel trial designs and endpoints to advance these breakthrough therapies showing such promise to address significant unmet medical needs.
Today is the beginning of a new era of medicine and brings new hope to the children and their families battling this terrible disease. We congratulate Novartis, researchers, physicians, and most importantly, patients, who have contributed to this important moment in CAR T cell development.
The above message 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 the potential of CAR T cell therapies, including Juno’s, and future improvements in these therapies. 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 product development activities and clinical trials; ability to obtain regulatory approval for and to commercialize its product candidates; ability to establish a commercially-viable manufacturing process and manufacturing infrastructure; regulatory requirements and regulatory developments; dependence on third-party collaborators and other contractors in research and development activities, including for the conduct of clinical trials and the manufacture of product candidates; and ability to obtain, maintain, or protect intellectual property rights related to 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 May 4, 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.