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FDA accepts Cellenkos’ IND to begin clinical trial of CK0801 for bone marrow failure syndromes

Published 11 June 2018

The US Food and Drug Administration (FDA) has cleared Cellenkos’ investigational new drug (IND) application to proceed with a phase I clinical trial of CK0801, third party cord blood derived regulatory T cells, in patients with bone marrow failure syndrome including aplastic anemia, myelodysplastic syndrome and myelofibrosis.

Cellenkos submitted its IND application on May 11, 2018.  The Phase I clinical trial of CK0801 will commence in the third quarter of 2018 at The University of Texas MD Anderson Cancer Center, Houston, Texas.

"The clearance of this IND is a significant milestone for Cellenkos™ and for its' platform technology, CK0801, as part of the overall clinical development plan of demonstrating adoptive cellular therapy with CK0801 to treat autoimmune diseases and inflammatory disorders", said Founder and Chief Medical Officer, Simrit Parmar.

"We are excited to launch the development of CK0801 as a novel cellular therapy approach to treat bone marrow dysfunction arising due to irregularities in the patient's immune system.  CK0801 works to replace and replenish the defective regulatory T cells and blocks the uncontrolled inflammatory signal generated by the cytotoxic T cells against patients' own tissues.

“Such an approach may lead to calming of the bone marrow inflammatory microenvironment as a result of the infused CK0801 which may then lead to the reversal of the hematopoietic arrest that will potentially translate into clinical improvement."

Tara Sadeghi, Senior Clinical Director, added, "The US FDA's clearance of this IND validates our commitment towards creating an 'off-the-shelf' cord blood derived regulatory T cell therapeutic platform for treating autoimmune diseases.  

“We are confident and eager that this Phase I study will confirm the safety of CK0801.  We remain grateful to all our colleagues and partners in academia, whose constant support and guidance allows us to take this step forward."

Bone marrow failure syndrome refers to the decreased production of one or more major hematopoietic lineages (white cells, red cells and platelets), which leads to diminished or absent hematopoietic precursors in the bone marrow.  Bone marrow failure remains an incurable disease except for those patients who are eligible to receive an allogeneic stem cell transplantation.

CK0801, is a first-in-class, cord blood-derived T-regulatory cellular product, developed using Cellenkos's proprietary manufacturing platform, that overcomes immune dysfunction by inhibiting key regulators of inflammation.  

CK0801 contains healthy and robust regulatory T cells derived from donor cord blood units and adoptive therapy with CK0801 has the potential to replenish the immune system and impact patient care by providing novel, non-pharmacologic options.  CK0801 will be manufactured at the Cellenkos FDA registered manufacturing facility located in Houston, Texas.

Source: Company Press Release

Source: Company Press Release