Last Stop Biotech

OVERVIEW

A clinical-stage biotechnology company NKGen, developing innovative autologous and allogeneic natural killer (NK) cell therapies announced today that its Chairman and CEO, Paul Y. Song, M.D., has invested $2.65 million in cash. These proceeds aim to accelerate the Company’s Phase 2 clinical trial for Alzheimer’s disease, reduce outstanding payables, and support general working capital and public company compliance costs. Dr. Song acquired this influx of cash from the recent sale of another biotech venture he co-founded.

SNK01 is composed of natural killer (NK) cells derived from a patient’s blood. According to the company, NKGen can expand and activate a sufficient number of these cells from a single collection to support up to six months of treatment. The NK cells can be cryopreserved and later infused into the patient’s bloodstream to enhance the immune system’s response to potential underlying causes of Alzheimer’s disease. Unlike other cell therapies, NK cells carry a lower risk of triggering adverse immune reactions.

the clinical outcome

In the Phase 1 study conducted in Hospital Angeles in Tijuana, Mexico, researchers first examined whether patients tolerated the treatment. They also looked at the highest dose tolerated by patients in a dose escalation study to inform the Phase 2 study design. SNK01 is administered intravenously and appears to cross the blood-brain barrier into the cerebrospinal fluid (CSF), where it has been associated with reductions in beta-amyloid, tau, and alpha-synuclein— a protein commonly linked to Parkinson’s disease that can also build up in Alzheimer’s patients. Researchers at the clinical site had this report:

“There were no treatment-related side effects. “SNK01 was safe and well tolerated” and “appears to have clinical activity in [Alzheimer’s],” the researchers wrote in an abstract to the presentation. “A larger trial with a higher dosing/duration has been initiated.”

NKGen’s SNK01 recently got approved by its internal safety committee to proceed to Phase 2 clinical studies. NKGen’s initial proof-of-concept Phase 1 study was an open-label, 3+3 dose-escalation trial that evaluated the safety and biological activity of SNK01. Administered via simple IV infusion at doses of 1, 2, or 4 billion cells over four treatments, SNK01 was well-tolerated and demonstrated the ability to cross the blood-brain barrier. The therapy showed a dose-dependent reduction in amyloid, tau, and alpha-synuclein proteins and neuroinflammation. Notably, 90% of participants—with a median MMSE score of 14—either improved or maintained stable cognitive function, as measured by the Alzheimer’s Disease Composite Score (ADCOMS). This ADCOM indicates that SNK01 is one of those rare therapies that do more than slow down Alzheimer’s disease.

SNK01 is also being studied in clinical trials as a standalone therapy and in combination with other treatments—such as checkpoint inhibitors and cell engagers—for patients with advanced, treatment-resistant solid tumors. In February 2025, the FDA granted approval for the compassionate use of SNK01 in a patient with Parkinson’s disease.

NKgen’s CEO stands out

SNK01 is riding a large momentum wave and has showcased excellent efficacy compared to its competitors in Alzheimer’s disease. CEO Dr. Young’s reinvesting of 2.65 million dollars shows his confidence and faith in SNK01 as a breakthrough treatment for neurodegenerative diseases and valuable for patients suffering from various cancers. He had this to comment on his view toward recent developments for SNK01:

“We are excited to announce another important clinical milestone in our mission to advance SNK01 as the first potential disease modifying treatment of its kind for patients with more advanced Alzheimer’s disease,

The Phase 2 trial will utilize our cryopreserved SNK01 product given at the highest dose we have ever delivered, and for a full year duration.

To date, SNK01 has been found to be well-tolerated with no reported drug-related adverse events. We are also very encouraged with the clinical progress we have made to date in moderate Alzheimer’s disease research and look forward to generating additional significant clinical and biomarker data from our randomized placebo-controlled Phase 2 trial.”

Amidst the graveyard of failed therapies for Alzheimer’s disease, SNK01 appears to be one of those rare shining stars that might have what it takes to clear Phase 3. Treating neurodegenerative diseases comes with many serious challenges that are not seen in other disease designations; one can get your drug payload across the blood-brain barrier (BBB) in enough amounts to alter disease progression. SNK01’s ability to infiltrate the BBB without causing adverse patient outcomes and showing efficacy is a great sign.