NKGen Biotech Announces Administration of First Dose of Troculeucel to Stroke Patient Under FDA-Cleared Compassionate Use Program
NKGen Biotech (NKGN) has administered its first dose of troculeucel, an expanded autologous NK cell therapy, to a stroke patient under an FDA-cleared compassionate use program. The treatment, administered at George Washington University Medical Center, marks the company's initial exploration into stroke treatment applications.
The initiative, led in collaboration with Dr. Dimitri Sigounas and Dr. Amarendra K. Neppalli, aims to investigate troculeucel's potential in reducing chronic neuroinflammation in post-stroke patients. This is particularly significant as stroke remains the second leading cause of death and long-term disability, with 20% of survivors developing dementia.
The company's previous Alzheimer's trials demonstrated troculeucel's ability to cross the blood-brain barrier and reduce cerebrospinal fluid levels of brain injury markers GFAP and NfL. This compassionate use program represents a stepping stone toward a possible full IND application for stroke treatment.
NKGen Biotech (NKGN) ha somministrato la sua prima dose di troculeucel, una terapia cellulare NK autologa espansa, a un paziente colpito da ictus nell'ambito di un programma di uso compassionevole approvato dalla FDA. Il trattamento, somministrato presso il George Washington University Medical Center, segna l'inizio dell'esplorazione dell'azienda nelle applicazioni per il trattamento dell'ictus.
L'iniziativa, condotta in collaborazione con il Dr. Dimitri Sigounas e il Dr. Amarendra K. Neppalli, mira a investigare il potenziale di troculeucel nella riduzione della neuroinfiammazione cronica nei pazienti post-ictus. Questo è particolarmente significativo poiché l'ictus rimane la seconda causa principale di morte e disabilità a lungo termine, con il 20% dei sopravvissuti che sviluppano demenza.
I precedenti studi dell'azienda sull'Alzheimer hanno dimostrato la capacità di troculeucel di attraversare la barriera emato-encefalica e ridurre i livelli nel liquido cerebrospinale dei marcatori di danno cerebrale GFAP e NfL. Questo programma di uso compassionevole rappresenta un passo verso una possibile applicazione IND completa per il trattamento dell'ictus.
NKGen Biotech (NKGN) ha administrado su primera dosis de troculeucel, una terapia celular NK autóloga expandida, a un paciente que sufrió un accidente cerebrovascular bajo un programa de uso compasivo aprobado por la FDA. El tratamiento, administrado en el George Washington University Medical Center, marca la primera exploración de la compañía en aplicaciones de tratamiento para el accidente cerebrovascular.
La iniciativa, liderada en colaboración con el Dr. Dimitri Sigounas y el Dr. Amarendra K. Neppalli, tiene como objetivo investigar el potencial de troculeucel para reducir la neuroinflamación crónica en pacientes post-ictus. Esto es particularmente significativo, ya que el accidente cerebrovascular sigue siendo la segunda causa principal de muerte y discapacidad a largo plazo, con un 20% de los sobrevivientes desarrollando demencia.
Los ensayos previos de la compañía sobre el Alzheimer demostraron la capacidad de troculeucel para cruzar la barrera hematoencefálica y reducir los niveles en el líquido cefalorraquídeo de los marcadores de daño cerebral GFAP y NfL. Este programa de uso compasivo representa un paso hacia una posible aplicación IND completa para el tratamiento del accidente cerebrovascular.
NKGen Biotech (NKGN)는 FDA의 승인된 동정적 사용 프로그램에 따라 뇌졸중 환자에게 확장 자가 NK 세포 요법인 troculeucel의 첫 번째 용량을 투여했습니다. 조지 워싱턴 대학교 의료 센터에서 시행된 이 치료는 회사가 뇌졸중 치료 응용 프로그램을 탐색하는 첫 걸음을 내딛는 것을 의미합니다.
이 이니셔티브는 Dr. Dimitri Sigounas와 Dr. Amarendra K. Neppalli와 협력하여 진행되며, troculeucel이 뇌졸중 후 환자의 만성 신경 염증을 줄이는 데 잠재력이 있는지 조사하는 것을 목표로 합니다. 이는 뇌졸중이 여전히 사망 및 장기 장애의 두 번째 주요 원인으로 남아 있으며, 생존자의 20%가 치매를 개발하는 점에서 특히 중요합니다.
회사의 이전 알츠하이머 임상 시험에서는 troculeucel이 혈액-뇌 장벽을 통과하고 뇌 손상 지표인 GFAP 및 NfL의 뇌척수액 수준을 감소시키는 능력을 입증했습니다. 이 동정적 사용 프로그램은 뇌졸중 치료를 위한 가능한 전체 IND 신청을 향한 이정표가 됩니다.
NKGen Biotech (NKGN) a administré sa première dose de troculeucel, une thérapie cellulaire NK autologue étendue, à un patient victime d'un AVC dans le cadre d'un programme d'utilisation compassionnelle approuvé par la FDA. Le traitement, administré au George Washington University Medical Center, marque le début de l'exploration de l'entreprise dans les applications de traitement des AVC.
L'initiative, dirigée en collaboration avec le Dr Dimitri Sigounas et le Dr Amarendra K. Neppalli, vise à étudier le potentiel de troculeucel pour réduire la neuroinflammation chronique chez les patients post-AVC. Cela est particulièrement significatif car l'AVC reste la deuxième cause de décès et de handicap à long terme, 20 % des survivants développant une démence.
Les essais précédents de l'entreprise sur Alzheimer ont montré que troculeucel peut franchir la barrière hémato-encéphalique et réduire les niveaux des marqueurs de lésion cérébrale GFAP et NfL dans le liquide céphalorachidien. Ce programme d'utilisation compassionnelle représente une étape vers une éventuelle demande IND complète pour le traitement des AVC.
NKGen Biotech (NKGN) hat die erste Dosis von troculeucel, einer erweiterten autologen NK-Zelltherapie, einem Schlaganfallpatienten im Rahmen eines von der FDA genehmigten Programms für mitfühlende Nutzung verabreicht. Die Behandlung, die im George Washington University Medical Center durchgeführt wurde, markiert die erste Erkundung des Unternehmens in den Anwendungen zur Schlaganfallbehandlung.
Die Initiative, die in Zusammenarbeit mit Dr. Dimitri Sigounas und Dr. Amarendra K. Neppalli geleitet wird, zielt darauf ab, das Potenzial von troculeucel zur Reduzierung chronischer Neuroinflammation bei Schlaganfallpatienten zu untersuchen. Dies ist besonders bedeutend, da Schlaganfälle die zweithäufigste Todesursache und Ursache für langfristige Behinderungen bleiben, wobei 20 % der Überlebenden an Demenz erkranken.
Die vorherigen Alzheimer-Studien des Unternehmens haben gezeigt, dass troculeucel in der Lage ist, die Blut-Hirn-Schranke zu überwinden und die Werte der Gehirnverletzungsmarker GFAP und NfL in der Gehirn-Rückenmarksflüssigkeit zu senken. Dieses Programm für mitfühlende Nutzung stellt einen Schritt in Richtung einer möglichen vollständigen IND-Anwendung zur Behandlung von Schlaganfällen dar.
- FDA cleared compassionate use IND for troculeucel in stroke treatment
- Demonstrated ability to cross blood-brain barrier in Alzheimer's trials
- Potential expansion into large stroke treatment market
- Early-stage development with only single patient approval
- No efficacy data available yet for stroke treatment
- Uncertain timeline for full IND application
Insights
NKGen Biotech's administration of troculeucel to a stroke patient marks a significant regulatory milestone as the company secured FDA clearance for this compassionate use IND. This represents NKGen's first exploration of its NK cell therapy beyond neurodegenerative diseases like Alzheimer's into the substantial stroke treatment space.
The scientific approach leverages NKGen's prior findings that troculeucel can cross the blood-brain barrier and reduce cerebrospinal fluid levels of brain injury markers NfL and GFAP. These biomarkers are particularly relevant in stroke assessment, providing a mechanistic rationale for this therapeutic exploration.
For a micro-cap biotech (
The collaboration with George Washington University Medical Center adds clinical credibility to this investigation. This development, while promising, represents an early exploratory phase rather than advanced clinical development, with outcomes from this single patient case serving as a critical initial data point.
The application of NK cell therapy to stroke treatment represents an innovative mechanistic approach targeting post-stroke neuroinflammation, which is a significant contributor to long-term neurological damage. The article highlights a concerning statistic: one-fifth of stroke survivors develop dementia with an
Troculeucel's ability to cross the blood-brain barrier is particularly noteworthy, as this represents a significant challenge for many potential stroke therapeutics. The focus on biomarkers NfL and GFAP is mechanistically sound, as these are established indicators of neuronal damage and astrocytic activation, respectively. Their reduction could potentially correlate with decreased neuroinflammation and improved functional outcomes.
This approach differs from traditional stroke therapies that typically focus on the acute phase through thrombolytics or mechanical thrombectomy. Instead, NKGen targets the secondary neuroinflammatory cascade that contributes to ongoing damage. The company's experience with these mechanisms in Alzheimer's disease provides some biological rationale for this therapeutic crossover, though stroke pathophysiology presents unique challenges requiring dedicated investigation.
While this single compassionate use case cannot establish efficacy, it represents an important translational step from preclinical findings to human application in the stroke setting.
NKGen explores the use of troculeucel as a potential treatment option for post-stroke patients.
SANTA ANA, Calif., March 03, 2025 (GLOBE NEWSWIRE) -- NKGen Biotech, Inc. (Nasdaq: 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 the administration of troculeucel, an expanded autologous NK cell therapy, in a stroke patient under a single compassionate use, Investigational New Drug (“IND”) cleared by the U.S. Food and Drug Administration (“FDA”).
Stroke is the second leading cause of death and long-term disability. It is accompanied by an increase in chronic neuroinflammation that can contribute to further subsequent neurological damage. Of note is that one fifth of stroke survivors go on to develop dementia after a stroke, with an
In collaboration with Dimitri Sigounas, M.D., Associate Professor of Neurological Surgery and Amarendra K. Neppalli, M.D., Director of Transplant and Cellular Therapy at George Washington University (“GWU”) Medical Center, Washington, D.C., NKGen has begun to explore the potential therapeutic role of troculeucel in the post-stroke setting. This FDA-cleared single compassionate use IND will be the initial step towards a possible full IND application. Dosing the first stroke patient is part of NKGen’s continued efforts to explore the potential of its NK cell therapy for individuals suffering post-stroke and traumatic brain injury, especially as a means to reduce or prevent chronic neuroinflammation and damage, alongside NKGen’s positive ongoing work in Alzheimer’s and other neurodegenerative diseases.
The patient will receive troculeucel infusions and undergo regular independent assessments by Dr. Sigounas at GWU Medical Center.
“Neurofilament light chain (“NfL”) and glial fibrillary acidic protein (“GFAP”) are markers of brain injury which have been used to assess functional outcome in stroke patients. In our Alzheimer’s trials, we have found that troculeucel can cross the blood brain barrier to reduce cerebrospinal fluid levels of GFAP and NfL” said Paul Y. Song, M.D., Chairman and Chief Executive Officer of NKGen. “We believe that troculeucel could potentially be a novel approach to reduce chronic neuroinflammation and the associated long-term sequelae in the post-stroke setting.”
“I am excited to explore whether enhanced NK cells can help reduce neuroinflammation in the post-stroke setting to help improve overall outcomes. If so, I believe this may be a very important first step in finding new ways to help stroke patients,” commented Dr. Sigounas.
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.
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.
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
External Contacts:
Chris Calabrese
Managing Director
LifeSci Advisors, LLC
ccalabrese@lifesciadvisors.com
Kevin Gardner
Managing Director
LifeSci Advisors, LLC
FAQ
What is the significance of NKGN's troculeucel treatment for stroke patients?
How does NKGN's troculeucel perform in crossing the blood-brain barrier?
What is the current development stage of NKGN's troculeucel for stroke treatment?
What percentage of stroke survivors develop dementia according to NKGN's research?
