• In patients with relapsed refractory acute myeloid leukemia (R/R AML) treated with a
  four-dose regimen consisting of 1.8 billion CYNK-001 cells per dose, two of four patients achieved an objective response at Day 28

• In patients with measurable residual disease-positive (MRD positive) AML treated with a
  three-dose regimen consisting of 1.8 billion CYNK-001 cells per dose, one of three patients achieved MRD negativity

• Treatment with CYNK-001 was generally well-tolerated, with no observed dose-limiting toxicities, even at the highest dose

• These results provide a strong basis to conclude this study and optimize the treatment protocol in the design of the next trial employing Celularity’s next-generation genetically modified natural killer (NK) cell therapy candidate in AML, CYNK-301

 Celularity Inc. (Nasdaq: CELU), a biotechnology company developing placental-derived allogeneic cell therapies and biomaterial products, today announced preliminary results from its Phase 1 trial of CYNK-001, its investigational unmodified natural killer (NK) cell therapy in development for the treatment of R/R AML and MRD positive AML.

Cohort 6B of the Phase 1 trial was comprised of patients with heavily pre-treated R/R AML who received a four-dose regimen consisting of 1.8 billion CYNK-001 cells per dose following enhanced lymphodepletion. Of these, two of four patients achieved an objective response, defined as a morphological leukemia-free state, or MLFS.

Cohort 4A of the Phase 1 trial was comprised of patients with MRD positive AML. Of these, one of three patients achieved MRD negativity after treatment with a three-dose regimen consisting of 1.8 billion CYNK-001 cells per dose following enhanced lymphodepletion.

Based on preliminary analysis of the Phase 1 trial data, this trial will now be closed to further enrollment as Celularity refines the trial design for subsequent studies with a next-generation NK product candidate. Celularity will continue to follow up with the trial participants.

Treatment with CYNK-001 showed biological activity and was generally well-tolerated, with no dose-limiting toxicities observed with even the highest dose of CYNK-001. This trial also yielded important insights into the optimal approach to lymphodepletion, the role of IL-15 and IL-2 in NK-cell persistence, and the importance of baseline lymphoblast count in achieving better responses. A trend toward better outcomes was observed in patients with lower lymphoblast counts in R/R AML patients in the trial.

“In a very challenging clinical environment of treatment-resistant disease, where the optimized cellular immunotherapy regimen has yet to be determined, this trial has given us valuable insight into our unmodified NK-cell therapy, CYNK-001, as well as how we should identify those AML patients who might benefit most from therapy and the best approach to modifying NK cells in order to optimize their efficacy in treating AML,” said Adrian Kilcoyne, M.D., M.P.H., M.B.A., Celularity’s Chief Medical Officer. “As we look to the future, these results, as well as recent important learnings from the broader scientific community, will support the development of CYNK-301, Celularity’s next-generation genetically modified NK cell therapy candidate in AML.”

“Given the rapidly emerging landscape in NK-cell science overall, we felt it important to evaluate our unmodified CYNK-001 trial now to identify trends and opportunities and guide our next steps. This update from our CYNK-001 study, as well as relevant, recently published data from other trials, provides valuable insight into the optimal companion lymphodepletion and the role of IL-15 in the treatment of AML. It also will guide development of our next-generation NK cell candidate, CYNK-301,” said Robert J. Hariri, M.D., Ph.D., Celularity’s Chairman, Founder and CEO. “Our goal is to develop a fully allogeneic NK cell therapy for AML optimized to fit into combination therapies.”

Data from the CYNK-001 Phase 1 trial will be submitted for inclusion at an upcoming scientific conference.