CytoSen Therapeutics Receives Favorable Response to Pre-IND Meeting Package for Phase 2 Study in Acute Myeloid Leukemia

CytoSen Therapeutics, a private biopharmaceutical company accelerating innovation in Natural Killer (NK) cell therapy, announced on 9/27/18 it has received a favorable response from the U.S. Food and Drug Administration (“FDA”) to its pre-Investigational New Drug (“pre-IND”) meeting package for the proposed Phase 2 trial of CytoSen’s adoptive NK cell therapy, CSTD002-NK, for the reduction of relapse in high risk acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) in patients undergoing haploidentical hematopoietic stem cell transplantation (HSCT). The FDA agreed that the Company’s pre-clinical studies, in combination with results from the clinical trial conducted at The University of Texas MD Anderson Cancer Center adequately support the Company’s proposed Phase 2 clinical trial. The FDA also deemed the proposed dose regimen, safety assessment and study endpoints as appropriate, and confirmed that interim results from the clinical study could be considered for a Regenerative Medicine Advanced Therapy (RMAT) designation.

"We are pleased with the feedback and clarification we received from the FDA, which reinforces our clinical development strategy and serves as a critical next step to initiate our planned AML-transplant study,” said Trent Carrier, Ph.D., CEO of CytoSen. “We look forward to commencing the trial in the first half of 2019.”

CSTD002-NK is a high dose, adoptive haploidentical NK cell therapy expanded ex vivo using CytoSen’s patented nanoparticle platform. The proposed Phase 2 trial is CytoSen’s first clinical study utilizing the nanoparticle technology, which produces highly activated NK cells within a scalable manufacturing platform.

Natural Killer cells play a significant role in the body’s innate immune response, circulating through the body to rapidly recognize and kill cancerous cells. CytoSen’s nanoparticle processing technology enables improved and sustained growth of high-dose NK cells that have potent anti-cancer cytotoxicity as well as increased expansion and activation capabilities. CytoSen’s lead clinical product, CSTD002-NK, is poised to initiate Phase 2 trial enrollment in the U.S. in the first half of 2019 as a best-in-class NK cell therapy for the treatment of acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) in HSCT patients.

On September 19 of 2018, Cytosen announced a partnership with KBI Biopharma to manufacture NK Cells and nanoparticles. KBI will manufacture the NK cells in its The Woodlands, Texas facility for CytoSen’s Phase 2 trial of CSTD002-NK.