NKGen Biotech Announces Administration of First Dose of Troculeucel to Frontotemporal Dementia 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 frontotemporal dementia (FTD) patient under FDA-cleared compassionate use program. The patient carries the C9orf72 gene mutation, representing one of the estimated 60,000 people living with FTD in the U.S.
This administration marks a significant step in exploring troculeucel's potential for treating neurodegenerative diseases, particularly in cases where existing therapies have proven ineffective. The company's previous experience with Alzheimer's and Parkinson's patients has shown that troculeucel can cross the blood brain barrier and improve various protein levels in CSF, including reducing neuroinflammation markers like GFAP.
The treatment will be conducted at the Sarcoma Oncology Center under Dr. Sant Chawla and Dr. Erlinda Gordon's guidance, with patient assessment by UCLA's Dr. Mario Mendez. This compassionate use program serves as an initial step towards a future full IND application.
NKGen Biotech (NKGN) ha somministrato la sua prima dose di troculeucel, una terapia cellulare NK autologa espansa, a un paziente affetto da demenza frontotemporale (FTD) nell'ambito di un programma di uso compassionevole approvato dalla FDA. Il paziente presenta la mutazione del gene C9orf72, che rappresenta uno dei circa 60.000 individui che vivono con la FTD negli Stati Uniti.
Questa somministrazione segna un passo significativo nell'esplorazione del potenziale del troculeucel per il trattamento delle malattie neurodegenerative, in particolare nei casi in cui le terapie esistenti si sono dimostrate inefficaci. L'esperienza precedente dell'azienda con pazienti affetti da Alzheimer e Parkinson ha dimostrato che il troculeucel può attraversare la barriera emato-encefalica e migliorare vari livelli di proteine nel liquido cerebrospinale, inclusa la riduzione di marcatori di neuroinfiammazione come il GFAP.
Il trattamento sarà condotto presso il Sarcoma Oncology Center sotto la guida del Dr. Sant Chawla e della Dr.ssa Erlinda Gordon, con la valutazione del paziente da parte del Dr. Mario Mendez dell'UCLA. Questo programma di uso compassionevole rappresenta un primo passo verso una futura domanda di IND completa.
NKGen Biotech (NKGN) ha administrado su primera dosis de troculeucel, una terapia celular NK autóloga expandida, a un paciente con demencia frontotemporal (FTD) en el marco de un programa de uso compasivo aprobado por la FDA. El paciente presenta la mutación del gen C9orf72, que representa a una de las aproximadamente 60,000 personas que viven con FTD en los EE. UU.
Esta administración marca un paso significativo en la exploración del potencial de troculeucel para tratar enfermedades neurodegenerativas, especialmente en casos donde las terapias existentes han demostrado ser ineficaces. La experiencia previa de la compañía con pacientes de Alzheimer y Parkinson ha mostrado que troculeucel puede cruzar la barrera hematoencefálica y mejorar varios niveles de proteínas en el líquido cefalorraquídeo, incluida la reducción de marcadores de neuroinflamación como el GFAP.
El tratamiento se llevará a cabo en el Sarcoma Oncology Center bajo la dirección del Dr. Sant Chawla y la Dra. Erlinda Gordon, con la evaluación del paciente a cargo del Dr. Mario Mendez de UCLA. Este programa de uso compasivo sirve como un primer paso hacia una futura solicitud completa de IND.
NKGen Biotech (NKGN)는 FDA의 승인된 동정적 사용 프로그램에 따라 전두측두엽 치매(FTD) 환자에게 확장된 자가 NK 세포 치료인 troculeucel의 첫 번째 용량을 투여했습니다. 이 환자는 C9orf72 유전자 변이를 가지고 있으며, 이는 미국에서 FTD로 삶을 살아가는 약 60,000명의 사람들 중 하나를 나타냅니다.
이번 투여는 특히 기존 치료가 효과가 없는 경우에 troculeucel의 신경퇴행성 질환 치료 가능성을 탐색하는 중요한 단계로 여겨집니다. 회사의 알츠하이머 및 파킨슨병 환자에 대한 이전 경험은 troculeucel이 혈액-뇌 장벽을 통과하고 CSF에서 다양한 단백질 수준을 개선할 수 있음을 보여주었으며, GFAP와 같은 신경염증 마커를 줄이는 효과도 있습니다.
치료는 Dr. Sant Chawla와 Dr. Erlinda Gordon의 지도 하에 Sarcoma Oncology Center에서 진행되며, 환자 평가는 UCLA의 Dr. Mario Mendez가 담당합니다. 이 동정적 사용 프로그램은 향후 완전한 IND 신청을 위한 첫 번째 단계로 작용합니다.
NKGen Biotech (NKGN) a administré sa première dose de troculeucel, une thérapie cellulaire NK autologue élargie, à un patient atteint de démence frontotemporale (FTD) dans le cadre d'un programme d'utilisation compassionnelle approuvé par la FDA. Le patient présente la mutation du gène C9orf72, représentant l'une des 60 000 personnes vivant avec la FTD aux États-Unis.
Cette administration marque une étape significative dans l'exploration du potentiel de troculeucel pour traiter les maladies neurodégénératives, en particulier dans les cas où les thérapies existantes se sont révélées inefficaces. L'expérience antérieure de l'entreprise avec des patients atteints de la maladie d'Alzheimer et de Parkinson a montré que troculeucel peut traverser la barrière hémato-encéphalique et améliorer divers niveaux de protéines dans le LCR, y compris la réduction des marqueurs de neuroinflammation tels que le GFAP.
Le traitement sera effectué au Sarcoma Oncology Center sous la direction du Dr Sant Chawla et de la Dr Erlinda Gordon, avec l'évaluation du patient par le Dr Mario Mendez de l'UCLA. Ce programme d'utilisation compassionnelle constitue un premier pas vers une future demande complète d'IND.
NKGen Biotech (NKGN) hat die erste Dosis von troculeucel, einer erweiterten autologen NK-Zelltherapie, einem Patienten mit frontotemporaler Demenz (FTD) im Rahmen eines von der FDA genehmigten Programms zur mitfühlenden Verwendung verabreicht. Der Patient trägt die C9orf72-Genmutation, die eine der geschätzten 60.000 Personen darstellt, die in den USA mit FTD leben.
Diese Verabreichung stellt einen bedeutenden Schritt bei der Erforschung des Potenzials von troculeucel zur Behandlung neurodegenerativer Erkrankungen dar, insbesondere in Fällen, in denen bestehende Therapien sich als ineffektiv erwiesen haben. Die bisherigen Erfahrungen des Unternehmens mit Alzheimer- und Parkinson-Patienten haben gezeigt, dass troculeucel die Blut-Hirn-Schranke überwinden und verschiedene Proteinlevel im Liquor cerebrospinalis verbessern kann, einschließlich der Reduzierung von Neuroinflammationsmarkern wie GFAP.
Die Behandlung wird im Sarcoma Oncology Center unter der Leitung von Dr. Sant Chawla und Dr. Erlinda Gordon durchgeführt, wobei die Patientenbewertung von Dr. Mario Mendez von der UCLA erfolgt. Dieses Programm zur mitfühlenden Verwendung dient als erster Schritt in Richtung eines zukünftigen vollständigen IND-Antrags.
- First FDA-cleared compassionate use of troculeucel for FTD treatment
- Previous success in crossing blood brain barrier and improving protein levels in Alzheimer's patients
- Addresses an unmet medical need with 60,000 FTD patients in the U.S.
- Collaboration with prestigious UCLA medical team
- Single patient compassionate use only, not a full clinical trial
- No proven efficacy in FTD patients yet
- Early-stage development with uncertain outcomes
Insights
This compassionate use approval marks a strategic expansion of NKGen's NK cell therapy platform into frontotemporal dementia, a devastating condition affecting approximately 60,000 patients in the U.S. with no existing disease-modifying treatments. The selection of an FTD patient with the C9orf72 gene mutation is particularly noteworthy, as genetic cases often provide clearer efficacy signals in early clinical development.
The preliminary data from Alzheimer's trials showing troculeucel's ability to cross the blood-brain barrier and modulate key biomarkers (tau proteins and GFAP) provides a strong mechanistic rationale for FTD application. This approach represents a novel paradigm in neurodegeneration treatment - using engineered immune cells to target both inflammation and protein aggregation simultaneously.
The collaboration with UCLA's neurology department, particularly with experts in behavioral neurology and molecular mechanisms, significantly enhances the trial's scientific rigor. Their involvement in biomarker analysis and clinical assessment will be important for generating the robust data needed to support a full IND application.
From a development perspective, this compassionate use program serves multiple strategic objectives: 1) It provides early proof-of-concept data in FTD while minimizing initial regulatory hurdles, 2) It allows for detailed biomarker analysis in a new indication, and 3) It potentially accelerates the pathway to a broader FTD development program. The company's approach of leveraging existing safety and biomarker data from their Alzheimer's program demonstrates efficient resource utilization in expanding their neurodegenerative disease portfolio.
NKGen takes a meaningful step towards potential treatment for frontotemporal dementia (“FTD”) patients with limited treatment options.
SANTA ANA, Calif., Feb. 21, 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 an FTD patient under a single compassionate use, Investigational New Drug (“IND”) cleared by the U.S. Food and Drug Administration (“FDA”).
It is estimated that about 60,000 people in the U.S. are currently living with FTD. Up to
NKGen has begun to explore the potential therapeutic role of troculeucel for the treatment of FTD in collaboration with Mario Mendez, M.D., Ph.D., Director of the Behavioral Neurology Program at the David Geffen School of Medicine at UCLA and Jessica Rexach, M.D., Ph.D., Director of the Rexach Lab at UCLA Department of Neurology. This single, FDA-cleared, IND compassionate use will be the initial step towards a future full IND application as the dosing of the first FTD patient is part of NKGen’s ongoing effort to explore the potential of its NK cell therapy for those suffering from neurodegenerative diseases, especially when existing therapies have not been effective.
“While most of our experience has been in treating patients with Alzheimer’s and Parkinson’s, we know that there is a common theme of neuroinflammation and damage in numerous neurodegenerative diseases,” said Paul Y. Song, M.D., Executive Chairman and Chief Executive Officer of NKGen. “We have previously shown that troculeucel can cross the blood brain barrier to improve levels of amyloid, α-synuclein, and tau proteins in CSF in our treated Alzheimer’s patients as well as reduce neuroinflammation as shown by a reduction in glial fibrillary acidic protein (“GFAP”). Of note is that GFAP and tau are both consistently elevated in patients with FTD and we believe this supports the rationale of trying troculeucel in such patients. We are excited with the potential outcomes using our NK cell therapy in a difficult-to-treat patient population.”
“The potential to influence brain disease by modulating peripheral immune components represents a truly pivotal frontier in our field,” said Dr. Rexach, M.D., Ph.D., Director of the Rexach Lab at UCLA Department of Neurology. “It’s imperative that we bring these opportunities forward to address additional devastating, currently incurable neurodegenerative disorders, like frontotemporal dementia, in addition to Alzheimer’s disease. By incorporating key biomarkers of both neuroinflammation and neurodegeneration, this work is designed to yield impact and achieve critical progress. This effort will ultimately inform effective strategies for immune cell modulation in FTD, paving the way for clinically effective strategies. This work is not only incredibly important, but also exceptionally timely.”
“FTD and its related disorders are devastating for patients and families. As a clinician caring for them, it is difficult to tell them that there are no disease-modifying treatments,” said Dr. Mendez, M.D., Ph.D., Director of the Behavioral Neurology Program at the David Geffen School of Medicine at UCLA. “I’m impressed with the preliminary troculeucel clinical and biomarker data in Alzheimer’s patients and believe its ability to reduce both tau proteins and neuroinflammation in the cerebral spinal fluid (“CSF”) might be beneficial in FTD and related disorders. I look forward to exploring this further in hopes of making a real difference in these disorders.”
The patient will be routinely assessed independently by Dr. Mendez at UCLA while receiving troculeucel infusions at the Sarcoma Oncology Center under the guidance of Dr. Sant Chawla and Dr. Erlinda Gordon.
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
dchua@nkgenbiotech.com
External Contacts:
Chris Calabrese
Managing Director
LifeSci Advisors, LLC
ccalabrese@lifesciadvisors.com
Kevin Gardner
Managing Director
LifeSci Advisors, LLC
kgardner@lifesciadvisors.com
FAQ
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