Can advanced, cellular-based immunotherapy investigational therapies take out COVID-19 in hospitalized patients? Seattle’s Infectious Disease Research Institute (IDRI) along with study sponsor Celularity are conducting what could be a path-breaking COVID-19 treatment. This investigational cell-based therapy known as CYNK-001 was previously targeting various cancers. Developed by New Jersey-based Celularity and in collaboration with IDRI, Lung Biotechnology PBC, and the California Institute for Regenerative medicine (CIRM), the study commenced last week at the University of California Irvine. The first part of the Phase 1/2 study involves up to 14 patients hospitalized with COVID-19 symptoms. These participants will receive up to two doses of the “Natural Killer” cells (NK cells) over a week period at a total of eight trial site locations. A second Phase 2 will involve a larger group of patients, reports Geoff Baker with the Seattle Times.
What is CYNK-001?
CYNK-001 is positioned as one of the only, if not the only cryopreserved allogeneic, off-the-shelf NK cell therapy under development from placental hematopoietic stem cells. The biotech developing this investigational treatment, New Jersey-based Celularity, suggests their approach can eliminate the risks associated with immune system reactions that other sorts of donor cells can trigger.
More specifically, CYNK-001 is positioned by Celularity as the only cryopreserved, allogeneic, off-the-shelf, natural killer (NK) cell therapy under development from placental hematopoietic stem cells as a prospective treatment option for various hematologic cancers, solid tumors and infectious disease. Biotech companies such as Celularity investigate the use of NK cells as they represent a unique class of immune cells, inherently capable of targeting cancer cells and interacting with adaptive immunity. For more information, follow the link.
Early Stage Tests
According to Mr. Baker reporting at Seattle Times, CYNK-001 apparently was approved by the U.S. Food and Drug Administration (FDA) for early testing by May. Four patients, critically ill with COVID-19, weren’t responding to any other treatments. Hence the FDA allowed the use of this investigational product. Alan Lew, IDRI’s senior clinical trial manager, told the Seattle Times’ Geoff Baker that two of the four patients had improvements in their respiratory status, which was promising; the other two didn’t but Lew described that as due to “their underlying conditions.” With no serious adverse effects, Lew reported the NK cell-based therapy was “well tolerated,” and hence “a great sign.”
‘Seven Figure’ Deal
Apparently, IDRI negotiated a deal to study CYNK-001 back in March. Purporting to have secured “a seven-figure funding deal for testing,” by the next month, the FDA approved the IND. The sponsors jointly recruited trial site operations, in partner hospitals in what Mr. Baker described as COVID-19 “hot spots,” including a couple in California, three in New Jersey and others in Arizona, Arkansas and the Tacoma, Washington-based MultiCare Health System.
Research sites and associated Principal Investigator (PI) include Banner University Medical Center (Marilyn Glassberg, MD); University of Arkansas (Mary Burgess, MD); UC Irvine (Leonid Groysman, MD); UC Davis (Stuart Cohen, MD); Hackensack University (Michelle Donato, MD); Atlantic Health Morristown and Summit (Eric D. Whitman, MD); MultiCare Health System (Vinay Malhotra, MD).
The FDA approved the Investigational New Drug (IND) application in April 2020. The Phase 1/2 trial was funded to determine the safety and efficacy of CYNK-001 in hospitalized patients with moderate COVID-19 disease. Formally titled “A Phase I/II Study of Human Placenta Hematopoietic Stem Cell Derived Natural Killer Cells (CYNK-001) for the Treatment of Adults with COVID-19,” the study team will ultimately target 86 participating patients. As mentioned, 14 patients in the Phase 1 stage: the sponsors will evaluate the safety and efficacy of multiple doses of CYNK-001 (Days 1, 4 and 7) in 14 patients. During Phase 2, the sponsors will utilize a random, open-label design; multiple doses of CYNK-001 will be compared to the control group. With an emphasis on “Best Supportive Care,” this phase includes up to 72 individuals in the study randomized on a 1:1 ration.
The study’s estimated primary completion date is November 30, 2020 but that date could be delayed given Phase 1 just started. More than likely, there will be at least some delay. The estimated study completion date, November 30, 2021.
IDRI’s Corey Casper, MD, MPH, leads the study as Principal Investigator. In the Seattle Times piece, Alan Lew, IDRI’s senior clinical trial manager reports on the potential value of NK cells: “They are natural to your body; they are innate.” Hence Lew continued on this fact “So, they play a very important role against the various pathogens. So, with the multiple additional NK-cells we’re giving into the body, that will hopefully help to cause that immune response to destroy those viruses.”
Lew commented to the Seattle Times that the first phases of the study should run about six months followed by FDA requests, if all goes according to plan, for further testing up until the end of next year. Hence why the study protocol calls for the end of the study on November 30, 2021. But preliminary results should be available far before then.
The Infectious Disease Research Institute, a partner organization affiliated with the University of Washington’s Department of Global Health, embraces a holistic approach to fighting infectious diseases. Founded in 1993, this non-profit global health organization combines high-quality science of a research-based organization with the product development capabilities of a biotech venture to develop new drugs, diagnostics and vaccines. IDRI emphasizes discovery and development of antigen adjuvant formulation and delivery, therapeutic molecule discovery and development, and biomarker discovery for diagnostics.
The organization employs about 125, is headquartered in beautiful Seattle, and counts nearly 100 partners and collaborators from all over the world.
A well-capitalized spinout of Celgene, Celularity founder and serial entrepreneur Robert Hariri seeks to revolutionize cell therapy. Founded in 2016, the company is apparently not alone in this endeavor as a number of investors contributing to this young firm have raised $290 million in two financing rounds.
Corey Casper, MD, MPH, IRDI
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