Novartis personalized cell therapy CTL019 receives FDA Breakthrough Therapy designation

EAST HANOVER, N.J., July 7, 2014 /PRNewswire/ -- 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 need1,2,3,4. Novartis' Zykadia(™) (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 FDA5.

"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 met6.

"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 years7. 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 treatment8.

About Zykadia

Zykadia (ceritinib) is indicated in the US for the treatment of patients with ALK+ metastatic NSCLC who have progressed on or are intolerant to crizotinib. This indication is approved under accelerated approval based on tumor response rate and duration of response. An improvement in survival or disease-related symptoms has not been established. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

Zykadia is an FDA-approved prescription medicine that is currently available through a number of specialty pharmacies in the US. Outside of the US, Zykadia (LDK378) is an investigational agent and has not been approved by regulatory authorities.

Disclaimer

The foregoing release contains forward-looking statements that can be identified by words such as "Breakthrough Therapy," "investigational," "trajectory," "potential," "potentially," "hopefully," "promising," "can," "look forward," "continue," or similar terms, or by express or implied discussions regarding potential marketing approvals for CTL019, or regarding potential future revenues from CTL019 and Zykadia. You should not place undue reliance on these statements. Such forward-looking statements are based on the current beliefs and expectations of management regarding future events, and are subject to significant known and unknown risks and uncertainties. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those set forth in the forward-looking statements. There can be no guarantee that CTL019 will be submitted or approved for sale in any market, or at any particular time. Nor can there be any guarantee that CTL019 or Zykadia will be commercially successful in the future. In particular, management's expectations regarding CTL019 and Zykadia could be affected by, among other things, the uncertainties inherent in research and development, including unexpected clinical trial results and additional analysis of existing clinical data; unexpected regulatory actions or delays or government regulation generally; the company's ability to obtain or maintain proprietary intellectual property protection; general economic and industry conditions; global trends toward health care cost containment, including ongoing pricing pressures; unexpected manufacturing issues, and other risks and factors referred to in Novartis AG's current Form 20-F on file with the US Securities and Exchange Commission. Novartis is providing the information in this press release as of this date and does not undertake any obligation to update any forward-looking statements contained in this press release as a result of new information, future events or otherwise.

About Novartis

Novartis Pharmaceuticals Corporation researches, develops, manufactures and markets innovative prescription drugs used to treat a number of diseases and conditions, including cardiovascular, dermatological, central nervous system, bone disease, cancer, organ transplantation, psychiatry, infectious disease and respiratory. The company's mission is to improve people's lives by pioneering novel healthcare solutions.

Located in East Hanover, New Jersey, Novartis Pharmaceuticals Corporation is an affiliate of Novartis AG, which provides innovative healthcare solutions that address the evolving needs of patients and societies. Headquartered in Basel, Switzerland, Novartis offers a diversified portfolio to best meet these needs: innovative medicines, eye care, cost-saving generic pharmaceuticals, preventive vaccines, over-the-counter and animal health products. Novartis is the only global company with leading positions in these areas. In 2013, the Group achieved net sales of USD 57.9 billion, while R&D throughout the Group amounted to approximately USD 9.9 billion (USD 9.6 billion excluding impairment and amortization charges). Novartis Group companies employ approximately 135,000 full-time-equivalent associates and sell products in more than 150 countries around the world. For more information, please visit http://www.novartis.com.

References

  1. Novartis Press Release. "Novartis meningitis B vaccine Bexsero receives FDA Breakthrough Therapy designation in the US." Available at: http://www.novartis.com/newsroom/media-releases/en/2014/1774805.shtml. Accessed June 2014.
  2. Novartis Press Release. "Novartis compound LDK378 receives FDA Breakthrough Therapy designation for ALK+ non-small cell lung cancer." Available at: http://www.novartis.com/newsroom/media-releases/en/2013/1685517.shtml. Accessed June 2014.
  3. Novartis Press Release. "FDA grants Breakthrough Therapy designation to Novartis' serelaxin (RLX030) for acute heart failure." Available at: http://www.novartis.com/newsroom/media-releases/en/2013/1711047.shtml. Accessed June 2014.
  4. Novartis Press Release. "Novartis receives FDA breakthrough therapy designation for BYM338 (bimagrumab) for sporadic inclusion body myositis (sIBM)." Available at: http://www.novartis.com/newsroom/media-releases/en/2013/1723765.shtml. Accessed June 2014.
  5. Novartis Press Release. "FDA Approves Zykadia (LDK378) in ALK+ Non-Small Cell Lung Cancer." Available at: http://www.nibr.com/newsroom/stories/2014Apr30_FDA-Approves-Zykadia.shtml. Accessed June 2014.
  6. US Food and Drug Administration. Frequently Asked Questions: Breakthrough Therapies. Available at: http://www.fda.gov/regulatoryinformation/legislation/federalfooddrugandcosmeticactfdcact/ significantamendmentstothefdcact/fdasia/ucm341027.htm Accessed June 2014.
  7. Howlader, N., Noone, A. M., Krapcho, M, et al. SEER Cancer Statistics Review, 1975–2010. National Cancer Institute, April 2013; Section 28.9 (12). http://www.seer.cancer.gov/csr/1975_2010/results_merged/sect_28_childhood_cancer.pdf. Accessed June 2014.
  8. Apostolidou, Effrosyni, et al. Treatment of Acute Lymphoblastic Leukaemia. Drugs 2007; 67 (15): 2153-2171.

SOURCE Novartis

Posted: July 2014

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