Phase 1/2a trial of NK cell therapy SNK01 wins FDA clearance

Study will include 30 people with Parkinson's disease; 20 will receive treatment

Margarida Maia, PhD avatar

by Margarida Maia, PhD |

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NKGen Biotech plans to begin clinical testing of SNK01, a natural killer (NK) cell therapy being developed to treat neurodegenerative diseases, in people with Parkinson’s disease, the company announced.

This follows a go-ahead by the U.S. Food and Drug Administration (FDA) for an investigational new drug (IND) application to start a Phase 1/2a clinical trial, which is expected to launch this year.

The trial will test how safe and well tolerated SNK01 is when given directly into the bloodstream and how well it works in up to 30 people with Parkinson’s. Twenty will be assigned to receive SNK01, and 10 will receive a placebo.

“The IND clearance marks a significant milestone as we advance our pipeline of NK cell therapy in neurodegenerative diseases,” Paul Y. Song, MD, NKGen’s chairman and CEO, said in a company press release.

Like other neurodegenerative diseases, Parkinson’s occurs when certain proteins fold into an incorrect shape and begin to deposit as clumps that are toxic to nerve cells in the brain. As nerve cells become damaged and die, symptoms develop that worsen over time.

Immune NK cells can recognize a wide range of other cells in distress and clear protein deposits of alpha-synuclein, which forms clumps in Parkinson’s. They also inhibit overactive immune cells, which are thought to drive neuroinflammation in the disease.

NKGen is working toward “developing safe and effective treatments that target both protein deposition and neuroinflammation for patients with neurodegenerative diseases,” Song said. “We are excited to start our first clinical trial in [Parkinson’s] as there currently is a high unmet medical need in this indication.”

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What is SNK01 and how does it work in Parkinson’s?

SNK01 contains highly pure NK cells harvested from a patient’s own blood. Using its technology, NKGen can grow and activate enough NK cells from a single blood sample to fulfill up to six months of treatment, according to the company.

NK cells can be frozen and stored before being infused back into a patient’s bloodstream. This should boost the immune response against what may be causing Parkinson’s. Unlike other cell therapies, NK cells are less likely to drive an unwanted immune response.

A 47-year-old person with advanced Parkinson’s has received SNK01 under a single compassionate use protocol, an FDA pathway that makes an unapproved medication available to treat someone who’s exhausted all available treatment options.

The patient received six doses of SNK01 at the University of California, Los Angeles Medical Center. Over nearly four months of therapy, the patient showed improvements in measures of cognitive and motor function.

In a small Phase 1 trial (NCT04678453), SNK01 appeared to be safe and well tolerated and to slow cognitive decline in people with advanced Alzheimer’s. It’s now being tested at a higher dose against a placebo in a larger Phase 1/2 trial (NCT06189963).

Along with reducing neuroinflammation, SNK01 also lowered levels of toxic proteins, including alpha-synuclein, in the cerebrospinal fluid, which surrounds the brain and spinal cord, suggesting it may also benefit Parkinson’s.

“Given the encouraging outcomes regarding the reduction in neuroinflammation observed in our Phase 1 Alzheimer’s trials, we are optimistic about the potential benefits that may emerge in our Parkinson’s trial,” Song said. “While directly inhibiting neuroinflammation, whether as an independent approach or in conjunction with other interventions, may not address the etiology, it can potentially decrease the production of factors that contribute to neurotoxicity, thereby hopefully leading to clinical improvements.”

In late 2022, NKGen inked a partnership with the Parkinson’s Foundation to help accelerate clinical testing of SNK01 in Parkinson’s.