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Phase 1/2a Study of NK Cell Therapy SNK01 for Parkinson’s Disease Approved by FDA

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 green light from the US Food and Drug Administration (FDA) for an investigational new drug application to initiate a Phase 1/2a clinical trial, which is expected to start this year.

The trial will test how safe and well-tolerated SNK01 is when administered 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 approval marks an important milestone in the advancement of our pipeline of NK cell therapies in neurodegenerative diseases,” said Paul Y. Song, MD, chairman and CEO of NKGen, in a company press release.

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

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

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

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A person with a microphone speaks, while another in a lab coat shines a light on an oversized brain.

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

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

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

A 47-year-old individual with advanced Parkinson’s received SNK01 under a single compassionate use protocol, an FDA pathway that makes an unapproved drug available to treat someone who has 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 was shown to be safe, well tolerated, and slow cognitive decline in people with advanced Alzheimer’s disease. It is now being tested at a higher dose against a placebo in a larger phase 1/2 trial (NCT06189963).

In addition to reducing neuroinflammation, SNK01 also reduced levels of toxic proteins, including alpha-synuclein, in the cerebrospinal fluid, which surrounds the brain and spinal cord, suggesting that this may also be beneficial for Parkinson’s disease.

“Given the encouraging results regarding the reduction of neuroinflammation observed in our Phase 1 trials in Alzheimer’s disease, we are optimistic about the potential benefits that could result from our research in Parkinson’s,” said Song. “While directly inhibiting neuroinflammation, either as an independent approach or in combination with other interventions, may not address the etiology, it could potentially reduce the production of factors that contribute to neurotoxicity, hopefully leading to clinical improvements.”

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