All Hematological Malignancies Targeted by KTE-C19 Development Now Covered by Orphan Drug Designations in U.S. and EU
SANTA MONICA, Calif., May 02, 2016 (GLOBE NEWSWIRE) -- Kite Pharma, Inc. (Nasdaq:KITE) (“Kite”), a clinical-stage biopharmaceutical company focused on developing engineered autologous T cell therapy (eACT™) products for the treatment of cancer, today announced that the U.S. Food and Drug Administration (FDA) granted orphan drug designations to Kite’s lead product candidate, KTE-C19, for the treatment of primary mediastinal B cell lymphoma (PMBCL), mantle cell lymphoma (MCL), follicular lymphoma (FL), acute lymphoblastic leukemia (ALL) and chronic lymphocytic leukemia (CLL). Kite previously received orphan drug designation for KTE-C19 for the treatment of diffuse large B cell lymphoma (DLBCL) in both the U.S. and the EU, as well as orphan drug designations in the EU for PMBCL, MCL, FL, ALL, and CLL.
“Kite has now secured Orphan Drug Designations in both the U.S. and the EU for the leading indications in hematological malignancies. This is an important regulatory milestone as we further our development of KTE-C19 in advanced hematological cancers, including the planned initiation of clinical studies in CLL and FL patients in 2017,” said Arie Belldegrun, M.D., FACS, Chairman, President, and Chief Executive Officer. "We are encouraged by the progress of our four ongoing ZUMA clinical trials and our ability to manufacture clinical supply of KTE-C19 in our own Santa Monica facilities. We look forward to working closely with the FDA to bringing this potentially transformative therapy to patients with a significant unmet need.”
Orphan drug designation is granted by the FDA’s Office of Orphan Products Development (OOPD) to drugs and biologics which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S. The OOPD provides incentives for sponsors to develop products for rare diseases which may include tax credits towards the cost of clinical trials and prescription drug user fee waivers. If a product that has orphan drug designation subsequently receives the first FDA approval for the disease for which it has such designation, the product is entitled to orphan product exclusivity, which means that the FDA may not approve any other applications to market the same biologic for the same indication for seven years, except in limited circumstances.
About Kite Pharma
Kite Pharma, Inc., is a clinical-stage biopharmaceutical company engaged in the development of novel cancer immunotherapy products, with a primary focus on engineered autologous cell therapy (eACT™) designed to restore the immune system's ability to recognize and eradicate tumors. Kite is based in Santa Monica, CA. For more information on Kite Pharma, please visit www.kitepharma.com. Sign up to follow @KitePharma on Twitter at www.twitter.com/kitepharma.
About Kite’s ZUMA Clinical Programs for KTE-C19
KTE-C19 is an investigational therapy in which a patient's T cells are genetically modified to express a CAR that is designed to target the antigen CD19, a protein expressed on the cell surface of B cell lymphomas and leukemias. Kite is currently enrolling four pivotal studies (also known as ZUMA studies) for KTE-C19 in patients with various B cell malignancies. The FDA has granted Breakthrough Therapy Designation status to KTE-C19 for the treatment of patients with refractory DLBCL, PMBCL, and transformed follicular lymphoma. KTE-C19 has also secured Orphan Drug Designations in the U.S. and the EU for DLBCL, PMBCL, MCL, FL, ALL and CLL.
| Study | Phase | Indication | Status |
| ZUMA-1 NCT02348216 | Phase 2 Pivotal (N=112) | Refractory DLBCL, PMBCL, TFL | Phase 2 enrolling |
| ZUMA-2 NCT02601313 | Phase 2 Pivotal (N=70) | Relapsed/refractory MCL | Phase 2 enrolling |
| ZUMA-3 NCT02614066 | Phase 1/2 Pivotal (N=75) | Relapsed/refractory Adult ALL | Phase 1/2 enrolling |
| ZUMA-4 NCT02625480 | Phase 1/2 Pivotal (N=75) | Relapsed/refractory Pediatric ALL | Phase 1/2 enrolling |
| DLBCL = diffuse large B cell lymphoma | |||
| PMBCL = primary mediastinal B cell lymphoma | |||
| TFL = transformed follicular lymphoma | |||
| MCL = mantle cell lymphoma | |||
| ALL = acute lymphoblastic leukemia | |||
Cautionary Note on Forward-Looking Statements
This press release contains forward-looking statements for purposes of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. We may, in some cases, use terms such as "predicts," "believes," "potential," "proposed," "continue," "estimates," "anticipates," "expects," "plans," "intends," "may," "could," "might," "will," "should" or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Forward-looking statements include statements regarding our intentions, beliefs, projections, outlook, analyses or current expectations concerning, among other things: the ability to advance multiple clinical trials of KTE-C19, the ability to manufacture clinical supply of KTE-C19, the timing and ability to initiate clinical trials of KTE-C19 for the treatment of CLL and FL, and to obtain regulatory approval of KTE-C19. Various factors may cause differences between Kite's expectations and actual results as discussed in greater detail in Kite's filings with the Securities and Exchange Commission, including without limitation in Kite's Annual Report on Form 10-K filed with the SEC on February 29, 2016. Any forward-looking statements that we make in this press release speak only as of the date of this press release. We assume no obligation to update our forward-looking statements whether as a result of new information, future events or otherwise, after the date of this press release.