Designation supports the advancement of CTL019 to help address the unmet need of patients with relapsed/refractory acute lymphoblastic leukemia (r/r ALL) The filing was submitted by the University of Pennsylvania's Perelman School of Medicine which is conducting the CTL019 Phase I/II clinical trials Novartis and Penn have exclusive global collaboration to research, develop and commercialize CAR T cell therapies for the investigational treatment of cancers

Basel, July 7, 2014 - Novartis announced today that the United States Food and Drug Administration (FDA) has granted Breakthrough Therapy status to CTL019, an investigational chimeric antigen receptor (CAR) therapy for the treatment of pediatric and adult patients with relapsed/refractory acute lymphoblastic leukemia (r/r ALL). The Breakthrough Therapy filing was submitted by the University of Pennsylvania's Perelman School of Medicine (Penn) which has an exclusive global agreement with Novartis to research, develop and commercialize personalized CAR T cell therapies for the treatment of cancers.

This is the fifth Breakthrough Therapy designation for Novartis, continuing the company's trajectory as a leader in developing innovative therapies to help treat diseases in which there remains significant unmet medical need[1],[2],[3],[4]. Novartis' Zykadia(TM) (ceritinib, previously known as LDK378), for the treatment of anaplastic lymphoma kinase positive (ALK+) metastatic non-small cell lung cancer (NSCLC), is one of the first medicines to receive an FDA approval following earlier receipt of Breakthrough Therapy designation by the FDA[5].

"This Breakthrough Therapy designation underscores the potential of CTL019 as a life-saving therapy for patients with relapsed/refractory ALL, who are in desperate need of new treatment options," said David Epstein, Division Head, Novartis Pharmaceuticals. "Novartis welcomes increased dialogue with the FDA and a potentially expedited review to streamline the development of CTL019 and hopefully bring this promising therapy to patients as quickly as possible."

According to the FDA, Breakthrough Therapy designation is intended to expedite the development and review of new medicines that treat serious or life-threatening conditions if the therapy has demonstrated substantial improvement over an available therapy on at least one clinically significant endpoint. The designation includes all of the fast track program features, as well as more intensive FDA guidance. It is a distinct status from both accelerated approval and priority review, which can also be granted to the same drug if relevant criteria are met[6].

"This is a major milestone as we are now one step closer in helping address the high unmet needs of this patient population," said Carl H. June, M.D., Richard W. Vague Professor in Immunotherapy in the department of Pathology and Laboratory Medicine in the Perelman School of Medicine and director of Translational Research in the Abramson Cancer Center of the University of Pennsylvania. "We are excited about the strength of the positive early data seen in pediatric and adult patients with relapsed/refractory acute lymphoblastic leukemia and look forward to building upon these findings as we continue advancing the CTL019 clinical program in Phase II trials."

Novartis recently established the Cell and Gene Therapies Unit under the leadership of Usman Azam, Global Head, to bring an intense focus on advancing innovative cell-based therapies, including the development of CARs. Novartis holds the worldwide rights to CARs developed through the collaboration with Penn for all cancer indications, including the lead program, CTL019.

About CTL019 CTL019 uses CAR technology to reprogram a patient's own T cells to "hunt" cancer cells that express specific proteins, called CD19. After they have been reprogrammed, the T cells (now called CTL019) are re-introduced into the patient's blood; they proliferate and bind to the targeted CD19+ cancer cells and destroy them.

Because CTL019 is an investigational therapy, the safety and efficacy profile has not yet been established. Access to investigational therapies is available only through carefully controlled and monitored clinical trials. These trials are designed to better understand the potential benefits and risks of the therapy. Because of uncertainty of clinical trials, there is no guarantee that CTL019 will ever be commercially available anywhere in the world.

About Acute Lymphoblastic Leukemia Acute lymphoblastic leukemia (ALL) is the most common cancer diagnosed in children, representing approximately 25% of cancer diagnoses among children younger than 15 years[7]. It can also occur in adults. ALL is a type of cancer in which the bone marrow makes too many abnormal white blood cells (lymphocytes). ALL usually gets worse quickly if it is not treated and can be fatal within a few months; therefore it is critical for patients to start treatment soon after diagnosis. Patients with relapsed ALL experience ALL cells returning in the marrow and a decrease in normal blood cells following their remission. Patients with refractory ALL still have leukemia cells in their bone marrow following treatment[8].

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