NKGen Receives FDA Expanded Access Protocol Authorization for Landmark Treatment of Several Neurodegenerative Diseases
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.
NKGen Biotech (OTC: NKGN) ha ottenuto l'autorizzazione dalla FDA per un Programma di Accesso Esteso (EAP) per troculeucel, la sua terapia con cellule natural killer autologhe potenziate. L'autorizzazione consente il trattamento di fino a 20 pazienti affetti da diverse malattie neurodegenerative, ampliando l'ambito oltre l'attuale trial di Fase 2a sull'Alzheimer.
Il programma EAP includerà pazienti con Alzheimer in fase iniziale, Parkinson, SLA, Atrofia Multisistemica, Paralisi Sopranucleare Progressiva, Demenza Frontotemporale, Degenerazione Corticobasale, Sclerosi Multipla e Demenza a Corpi di Lewy. Troculeucel ha mostrato benefici clinici preliminari senza eventi avversi correlati al farmaco in due trial di Fase I sull'Alzheimer, dimostrando la capacità di attraversare la barriera emato-encefalica e ridurre i marcatori neuroinfiammatori.
NKGen Biotech (OTC: NKGN) ha recibido la autorización de la FDA para un Programa de Acceso Ampliado (EAP) para troculeucel, su terapia con células natural killer autólogas mejoradas. La autorización permite el tratamiento de hasta 20 pacientes con diversas enfermedades neurodegenerativas, ampliando más allá de su actual ensayo de Fase 2a para Alzheimer.
El EAP incluirá pacientes con Alzheimer en etapa temprana, Parkinson, ELA, Atrofia Multisistémica, Parálisis Supranuclear Progresiva, Demencia Frontotemporal, Degeneración Corticobasal, Esclerosis Múltiple y Demencia con Cuerpos de Lewy. Troculeucel ha mostrado beneficios clínicos preliminares sin eventos adversos relacionados con el medicamento en dos ensayos de Fase I para Alzheimer, demostrando su capacidad para cruzar la barrera hematoencefálica y reducir marcadores neuroinflamatorios.
NKGen Biotech (OTC: NKGN)는 자가 강화 자연 살해 세포 치료제인 troculeucel에 대해 FDA의 확대 접근 프로그램(EAP) 승인을 받았습니다. 이 승인은 현재 진행 중인 알츠하이머병 2a상 임상을 넘어 다양한 신경퇴행성 질환을 가진 최대 20명의 환자 치료를 허용합니다.
EAP에는 초기 알츠하이머병, 파킨슨병, 루게릭병(ALS), 다계통 위축증, 진행성 핵상 마비, 전두측두엽 치매, 피질기저 변성, 다발성 경화증, 루이체 치매 환자들이 포함됩니다. Troculeucel은 두 건의 1상 알츠하이머 임상에서 약물 관련 부작용 없이 초기 임상적 이점을 보였으며, 혈뇌장벽을 통과하고 신경염증 마커를 감소시키는 능력을 입증했습니다.
NKGen Biotech (OTC : NKGN) a reçu l'autorisation de la FDA pour un Programme d'Accès Élargi (EAP) pour troculeucel, sa thérapie par cellules tueuses naturelles autologues améliorées. Cette autorisation permet de traiter jusqu'à 20 patients atteints de diverses maladies neurodégénératives, élargissant ainsi le champ au-delà de leur essai de phase 2a sur la maladie d'Alzheimer.
L'EAP inclura des patients atteints de maladie d'Alzheimer à un stade précoce, Parkinson, SLA, atrophie multisystémique, paralysie supranucléaire progressive, démence frontotemporale, dégénérescence corticobasale, sclérose en plaques et démence à corps de Lewy. Troculeucel a montré des bénéfices cliniques préliminaires sans événements indésirables liés au médicament dans deux essais de phase I sur Alzheimer, démontrant sa capacité à traverser la barrière hémato-encéphalique et à réduire les marqueurs neuro-inflammatoires.
NKGen Biotech (OTC: NKGN) hat von der FDA eine Genehmigung für ein Expanded Access Program (EAP) für troculeucel, seine autologe verbesserte natürliche Killerzelltherapie, erhalten. Die Genehmigung erlaubt die Behandlung von bis zu 20 Patienten mit verschiedenen neurodegenerativen Erkrankungen und erweitert damit das aktuelle Phase-2a-Alzheimer-Studium.
Das EAP wird Patienten mit frühem Alzheimer, Parkinson, ALS, multipler Systematrophie, progressiver supranukleärer Blickparese, frontotemporaler Demenz, kortikobasaler Degeneration, Multipler Sklerose und Lewy-Körper-Demenz einschließen. Troculeucel zeigte in zwei Phase-I-Alzheimer-Studien vorläufige klinische Vorteile ohne medikamentenbedingte Nebenwirkungen und bewies die Fähigkeit, die Blut-Hirn-Schranke zu überwinden und neuroinflammatorische Marker zu reduzieren.
- 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
- Enrollment speed depends on funding availability
- Limited to only 20 patients in the expanded access program
Insights
FDA grants NKGen expanded access for troculeucel therapy in multiple neurodegenerative diseases, broadening beyond current Alzheimer's trials, promising potential treatment options.
NKGen has received a critical FDA authorization to expand their troculeucel therapy beyond Alzheimer's disease to several additional neurodegenerative conditions. This expanded access protocol represents a significant regulatory milestone, allowing the company to treat up to 20 patients with conditions that currently have limited or no effective treatments.
The therapy itself is particularly noteworthy - troculeucel is an autologous enhanced natural killer cell therapy that has already shown preliminary clinical benefits in two Phase I Alzheimer's trials without drug-related adverse events. What makes this approach scientifically compelling is its mechanism targeting neuroinflammation through IV administration, with evidence suggesting it can cross the blood-brain barrier to reduce inflammatory and protein biomarkers in cerebrospinal fluid.
The expansion to include Parkinson's Disease, ALS, Multiple System Atrophy, Progressive Supranuclear Palsy, Frontotemporal Dementia, Corticobasal degeneration, Multiple Sclerosis, and Lewy Body Dementia significantly broadens the potential applications. While their current Phase 2a study focuses only on moderate-stage Alzheimer's, this expanded access creates a parallel development pathway for exploring efficacy in these additional indications.
This regulatory achievement provides NKGen with valuable opportunities to gather preliminary data across multiple conditions while potentially helping patients with few options. However, the CEO's mention that "speed of enrollment will depend on funding" signals that financial constraints may impact the program's advancement timeline.
- 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?
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What is the mechanism of action for troculeucel in neurodegenerative diseases?
