The Food and Drug Administration (FDA) has granted Orphan Drug designation to CellProtect (XNK Therapeutics) for the treatment of multiple myeloma.

CellProtect is an autologous ex vivo expanded and activated NK (natural killer) cell-based therapy. The investigational drug product is produced by selective expansion and activation of NK cells from the peripheral blood of patients with cancer. It would be delivered to the clinic upon need, where the product is thawed and infused to patients without any further processing. 

The designation is supported by results from the first-in-human, phase 1/2 clinical trial of CellProtect following high dose stem cell transplantation in 12 patients newly diagnosed with multiple myeloma. According to the Company, findings showed that the treatment achieved its primary objective of safety and tolerability, with no severe adverse events reported.

Full trial results have not yet been posted but the Company indicated that there was a response to NK cell infusion in terms of M-component and/or minimal residual disease.

The Company plans to initiate a phase 2 clinical trial combining CellProtect with an anti-cancer antibody in the near future.

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“Obtaining an ODD by the FDA is a significant milestone for XNK and our goal of taking the present drug candidate to the next level,” said Johan Liwing, CEO of XNK Therapeutics. “This is the starting point for us to expand clinical development into the most important market globally for cancer treatment.”

The FDA’s Orphan Drug designation is granted to medicines intended to treat or prevent rare diseases or disorders that affect fewer than 200,000 individuals. 

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  1. XNK Therapeutics receives US Orphan Drug status for NK cell-based immunotherapy in multiple myeloma. [press release]. Stockholm, Sweden: XNK Therapeutics; November 4, 2020. 
  2. CellProtect, autologous ex vivo expanded and activated NK cells with increased tumor killing capacity, is the lead investigational drug candidate of XNK Therapeutics. XNK Therapeutics website. Accessed November 4, 2020.