FDA Grants Fast Track Designation to CYNK-001 for AML Treatment

A novel natural killer cell therapy is now on the fast track to FDA approval and being assessed in a phase 1 clinical trial.

The FDA has granted fast track designation to, CYNK-001, a novel on-genetically modified cryopreserved human placental hematopoietic stem cell-derived natural killer (NK) cell therapy for the treatment of patients with acute myeloid leukemia, according to a press release issued by Celularity Inc.1

CYNK-001 contains NK cells derived from human placental CD34+ cells and culture-expanded. In a new phase 1 multi-dose study (NCT04310592) of approximately 56 adult patients with AML, CYNK-001 cells will be given after lymphodepleting chemotherapy to determine its safety and whether NK cells be used to treat AML.1,2

“The majority of patients with AML continue to have poor long-term outcomes, particularly those who suffer relapse or have measurable residual disease, necessitating development of novel therapies, including CYNK-001,” said Andrew Pecora, MD, president of Celularity, in a press release.1

“We believe that the unique properties of our cell source, including the ability to proliferate and maintain activity, could be the key to improving response rates and durability for patients. We are pleased to receive this fast-track designation from the FDA for AML supporting continued development of our placental-derived NK cell platform. CYNK-001 previously received orphan drug designation for malignant gliomas and fast track designation for glioblastoma multiforme,” said Robert Hariri, MD, PhD, founder, chairperson, and chief executive officer of Celularity, in the press release.

In the phase 1 study of CYNK-001 the coprimary end points include dose-limiting toxicity (DLT) in minimal residual disease (MRD) positive patients with AML, DLT in relapsed or refractory patients with AML, the maximum-tolerated dose (MTD) in the MED -positive population, MTD in the relapsed/refractory population, and the frequency and severity of adverse events (AEs). Secondary end points that will be evaluated in the study include the number of patients who experienced MRD response, time to MRD response, duration of MRD response, progression-free survival, time to progression, duration of morphologic complete remission, overall survival, overall response rate, and duration of response.2

Patients aged 18 to 80 years with either primary/secondary AML or relapsed/refractory AML are eligible to enroll. Patients are required to have an ECOG performance status of ≤ 2 and must be able off immunosuppressive drugs for at least 3 days before CYNK-001 infusion. Both male and female patients are required to contraception during the study and females cannot be pregnant when enrolling. The study also permits the inclusion of patients with central nervous system involvement that has been treated and who have clear cerebral spinal fluid for at least 2 weeks before joining the study.

The study is actively recruiting adults with AML to at 8 centers across the United States, including in Colorado, Illinois, New Jersey, New York, Tennessee, Washington.


1. Celularity receives fast track designation from U.S. FDA for its NK cell therapy CYNK-001 in development for the treatment of AML. News release. December 27, 2021. Accessed December 29, 2021. https://bit.ly/3sE9g1O

2. Natural killer cell (CYNK-001) infusions in adults with AML (CYNK001AML01). Clinicaltrials.gov. Accessed December 29, 2021.