NKGen Receives FDA Expanded Access Protocol Authorization for Landmark Treatment of Several Neurodegenerative Diseases
Rhea-AI Summary
NKGen Biotech (OTC: NKGN) has received FDA authorization for an Expanded Access Program (EAP) for troculeucel, its autologous enhanced natural killer cell therapy. The authorization allows treatment of up to 20 patients with various neurodegenerative diseases, expanding beyond their current Phase 2a Alzheimer's trial.
The EAP will include patients with early-stage Alzheimer's, Parkinson's, ALS, Multiple System Atrophy, Progressive Supranuclear Palsy, Frontotemporal Dementia, Corticobasal degeneration, Multiple Sclerosis, and Lewy Body Dementia. Troculeucel has shown preliminary clinical benefits without drug-related adverse events in two Phase I Alzheimer's trials, demonstrating ability to cross the blood-brain barrier and reduce neuroinflammatory markers.
Positive
- FDA granted Expanded Access Program authorization for troculeucel treatment
- Previous Phase I trials showed clinical benefits with no drug-related adverse events
- Treatment potential for multiple neurodegenerative diseases with no current effective therapies
- Demonstrated ability to cross blood-brain barrier and reduce neuroinflammatory markers
Negative
- Enrollment speed depends on funding availability
- Limited to only 20 patients in the expanded access program
News Market Reaction
On the day this news was published, NKGN declined NaN%, reflecting a moderate negative market reaction.
Data tracked by StockTitan Argus on the day of publication.
- Troculeucel, a cryopreserved, autologous enhanced natural killer cell therapy, has demonstrated preliminary clinical benefit without any drug-related adverse events in two Phase I clinical trials in Alzheimer’s Disease.
- Company receives expanded access protocol authorization from the U.S. Food and Drug Administration (FDA) for troculeucel granted to treat patients with known diagnoses of multiple neurodegenerative diseases.
- IND approval allows for up to 20 patients to be enrolled; first patient enrollment is anticipated in Q3 2025.
SANTA ANA, Calif., July 21, 2025 (GLOBE NEWSWIRE) -- NKGen Biotech, Inc. (OTC: NKGN) (“NKGen” or the “Company”), a clinical-stage biotechnology company focused on the development and commercialization of innovative autologous and allogeneic natural killer (“NK”) cell therapeutics, today announced that the U.S. Food and Drug Administration (FDA) has granted Expanded Access Program (EAP) authorization for its IND for an open-label, non-randomized, multi-center intermediate size expanded access protocol for use of troculeucel for neurodegenerative diseases. This includes its use for the treatment of several neurodegenerative diseases, many of which currently have no effective therapy.
While NKGen’s ongoing double-blind randomized Phase 2a trial is focused on moderate-stage Alzheimer’s disease (NCT06189963), this EAP IND marks the company’s exploration into the treatment of other less common neurodegenerative diseases. In addition to earlier stage Alzheimer’s Disease (AD), this IND includes the treatment of Parkinson’s Disease (PD), Amyotrophic Lateral Sclerosis (ALS), Multiple System Atrophy (MSA), Progressive Supranuclear Palsy (PSP), Frontotemporal Dementia (FTD), Corticobasal degeneration (CBD), Multiple Sclerosis (MS) and Lewy Body Dementia (LBD).
This IND approval will allow NKGen to expand and explore the use of troculeucel in up to 20 patients.
“There is a common element of autoimmune neuroinflammation in all of these neurodegenerative diseases due to autoreactive T cells, many in response to either an amyloid, alpha-synuclein, or tau protein deposition,” said Paul Y. Song, MD, Chairman and Chief Executive Officer of NKGen Biotech. “As we have demonstrated in two Phase I trials in Alzheimer’s Disease, troculeucel, given via a simple IV, appears to cross the blood brain barrier to reduce neuroinflammatory and protein biomarkers in CSF. We believe there is ample scientific and clinical rationale to offer this to patients with other neurodegenerative diseases for which there is no effective therapy and little hope. We will begin enrolling patients as soon as possible, but the speed of enrollment will depend on funding.”
About NKGen Biotech
NKGen is a clinical-stage biotechnology company focused on the development and commercialization of innovative autologous and allogeneic NK cell therapeutics. NKGen is headquartered in Santa Ana, California, USA. For more information, please visit www.nkgenbiotech.com.
About Troculeucel
Troculeucel is a novel cell-based, patient specific, ex vivo expanded autologous NK cell immunotherapeutic drug candidate. NKGen is developing troculeucel for the treatment of neurodegenerative disorders and a broad range of cancers. Troculeucel is the International Nonproprietary Name (“INN”) for SNK01 assigned by the World Health Organization (“WHO”). The WHO INN approval of troculeucel establishes a universally recognized nonproprietary drug name for SNK01 and marks a significant step on NKGen’s journey toward bringing this therapy to market.
Forward-Looking Statements
Statements contained in this press release may contain “forward-looking statements” within the meaning of Section 27A of the Securities Act and Section 21E of the Securities Exchange Act of 1934, as amended. Forward-looking statements may be identified by the use of words such as “anticipate”, “believe”, “could”, “continue”, “expect”, “estimate”, “may”, “plan”, “outlook”, “future” and “project” and other similar expressions that predict or indicate future events or trends or that are not statements of historical matters. Because such statements are subject to risks and uncertainties, many of which are outside of the Company’s control, actual results may differ materially from those expressed or implied by such forward-looking statements. Such statements include, but are not limited to, statements regarding the Company’s plans and expected timing for developing troculeucel and SNK02, including the expected timing of completing and announcing further results from its ongoing clinical studies; and the Company’s expected timing for developing its product candidates and potential benefits of its product candidates. Risks that contribute to the uncertain nature of the forward-looking statements include: the Company’s ability to execute its plans and strategies; risks related to performing clinical studies; the risk that initial and interim results of a clinical study do not necessarily predict final results and that one or more of the clinical outcomes may materially change as patient enrollment continues, following more comprehensive reviews of the data, and as more patient data become available; potential delays in the commencement, enrollment and completion of clinical studies and the reporting of data therefrom; the risk that studies will not be completed as planned; the risk that the abstract will not be published as planned including delays in timing, format, or accessibility; and NKGen’s ability to raise additional funding to complete the development of its product candidates. These and other risks and uncertainties are described more fully under the caption “Risk Factors” and elsewhere in the Company’s filings and reports, which may be accessed for free by visiting the Securities and Exchange Commission’s website at www.sec.gov and on the Company’s website under the subheading “Investors—Financial and Filings”. Investors should take such risks into account and should not rely on forward-looking statements when making investment decisions. All forward-looking statements contained in this press release speak only as of the date on which they were made. The Company undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.
Internal Contact:
Denise Chua, MBA, CLS, MLS (ASCP)
SVP, Corporate Affairs
949-396-6830
dchua@nkgenbiotech.com
External Contact:
Kevin Gardner
Managing Director
LifeSci Advisors, LLC
kgardner@lifesciadvisors.com
FAQ
What FDA authorization did NKGen Biotech (NKGN) receive for troculeucel?
What diseases can be treated under NKGen's new expanded access protocol?
What results has troculeucel shown in previous clinical trials?
When will NKGN begin enrolling patients under the expanded access protocol?
What is the mechanism of action for troculeucel in neurodegenerative diseases?
