NKT Therapeutics Presents Data on its Therapeutic Program Targeting Sickle Cell Disease at the American Society of Hematology

WALTHAM, Mass., Dec. 8, 2014 /PRNewswire/ -- NKT Therapeutics (NKTT), a clinical stage biotechnology company, presented this week positive Phase 1 clinical data for its lead compound NKTT120 in patients with sickle cell disease (SCD), as well as supportive data in a preclinical model of that condition. The new findings were presented by NKTT scientists and collaborators at the American Society of Hematology Annual Meeting, held from December 5 – 9, 2014 in San Francisco, CA.

The presented data included:

• Demonstration by our collaborator Dr. Joel Linden (La Jolla Institute of Allergy and Immunology) that specific antibody-mediated iNKT cell depletion reduces pulmonary inflammation and improves lung function in mice genetically modified to have human sickle cell hemoglobin (Presentation entitled "Antibody Mediated Depletion of iNKT Cells Protects Against Hypoxia-Induced Pulmonary Injury in a Murine Model of Sickle Cell Disease").
• Demonstration that iNKT cells are activated in patients with stable sickle cell disease, consistent with a role for these cells in mediating the inflammation that underlies the pain and organ damage associated with this disease (Presentation entitled "Adult Patients with Stable Sickle Cell Disease Prior to Treatment with the iNKT Cell Depleting Antibody NKTT120 Have Consistent and Elevated Numbers of Activated (CD69+) iNKT Cells Relative to Healthy Adult Volunteers").
• Demonstration that a single dose of NKTT120 results in prolonged reduction of iNKT cells in the peripheral blood without dose limiting toxicities or serious adverse events related to the study drug (Presentation entitled "NKTT120 Reduces iNKT Cells without Dose Limiting Toxicity in Stable Adult Sickle Cell Patients in a Phase 1 Trial"). The Phase 1b trial, which met all its primary endpoints, was designed to determine the safety, maximum tolerated dose (MTD), pharmacokinetics, and pharmacodynamics of NKTT120 in adults with stable SCD. 

"Sickle cell disease is a devastating orphan disease associated with early mortality and patients and physicians need improved treatment options," stated Dr. Martin Steinberg, Professor of Medicine and Director of the Center for Excellence in Sickle Cell Disease at Boston University, commenting independently on the presented data.  "The preclinical and clinical data support moving this novel treatment paradigm forward into clinical efficacy studies."

About NKT Therapeutics

NKT Therapeutics, Inc. is a privately held biotechnology company focused on developing therapeutics based on unique immune cells called natural killer T (NKT) cells. The company's mission is to use its expertise to develop a pipeline of first-in-class NKT-based therapeutics to treat sickle cell disease, autoimmune and inflammatory diseases, and cancer. For more information on the company, please visit http://www.nktrx.com.

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SOURCE NKT Therapeutics