On February 15, 2022 Celularity Inc. (Nasdaq: CELU) (Celularity), a clinical-stage biotechnology company developing placental-derived off-the-shelf allogeneic cell therapies, reported the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for its investigational natural killer (NK) cell therapy, CYNK-101, for treatment of gastric/gastroesophageal junction cancer (Press release, Celularity, FEB 15, 2022, View Source [SID1234608167]). CYNK-101 is being developed as a first-line treatment in combination with standard chemotherapy, trastuzumab and pembrolizumab in patients with locally advanced unresectable or metastatic HER2/neu positive gastric or gastroesophageal junction (G/GEJ) adenocarcinoma. CYNK-101 is an investigational genetically modified NK cell therapy designed to synergize with approved antibody therapeutics through enhanced antibody-dependent cellular cytotoxicity (ADCC).
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"This designation underscores the significant unmet need for these patients and CYNK-101’s potential in a new first-line treatment strategy," said Robert Hariri, M.D., Ph.D., Founder, Chairman and Chief Executive Officer of Celularity. "We are grateful for the FDA’s recognition of this potential treatment paradigm through Orphan Drug Designation and Fast Track designation, which we received earlier this year. At Celularity, we are committed to forging new treatment strategies that leverage the unique properties of placental-derived cellular therapies to improve the lives of patients with this difficult-to-treat cancer."
Andrew Pecora, M.D., President of Celularity, added, "The addition of cleavage-resistant CD16 significantly augments the antibody-dependent activity of our natural killer cell therapy. To date, we have seen promising clinical data from our unmodified NK cellular therapies and believe this genetically modified construct has significant potential in a range of indications. In our Phase 1/2a clinical trial of CYNK-101, we are excited to explore a multipronged strategy of our NK cells and activated T cells through check point inhibition to potentially enhance overall outcomes achieved with traditional chemotherapy and Traztuzumab in HER2/neu positive G/GEJ adenocarcinoma."
This is the fifth designation received by Celularity from the FDA in the past 12 months and follows fast track designations for CYNK-101, in development for the first-line treatment of advanced HER2/neu positive G/GEJ cancers, and CYNK-001, an unmodified investigational NK cell therapy in development for the treatment of acute myeloid leukemia and in development for the treatment of recurrent glioblastoma multiforme, as well as orphan drug designation for CYNK-001 for the treatment of malignant gliomas.
About Orphan Drug Designation
The Orphan Drug Act (ODA) provides incentives to encourage biotechnology and pharmaceutical companies to develop drugs for rare diseases and conditions. To qualify for orphan designation, both the drug and the condition must meet criteria specified in the ODA and FDA’s implementing regulations. Orphan designation qualifies the drug sponsor for development incentives including tax credits for qualified expenses, exemption from the FDA user fee, and the potential for seven years of exclusivity for a drug that obtains approval.
About Gastric Cancer
Gastric cancer is the fifth most common cancer worldwide. Despite recent improvements in treatment quality and options, advanced gastric cancer remains one of the hardest to cure cancers, with a median overall survival (OS) of 10–12 months and a five-year OS of approximately 5–20%.
About CYNK-101
Celularity’s lead therapeutic candidate based on its placental-derived genetically modified NK cell type is CYNK-101, an allogeneic, off-the-shelf human placental CD34+-derived NK cell product genetically modified to express high-affinity and cleavage-resistant CD16 (FCGRIIIA) variant to drive antibody-dependent cell-mediated cytotoxicity. Currently CYNK-101 is being developed as a treatment in combination with standard chemotherapy, trastuzumab and pembrolizumab for HER2 positive overexpressing gastric or gastroesophageal junction adenocarcinoma. The safety and efficacy of CYNK-101 have not been established, and CYNK-101 has not been approved for any use by the FDA or any other analogous regulatory authority.