Cue Biopharma Receives FDA Feedback on Pre-IND Briefing Document Reinforcing Company’s Intention to Advance IND Submission for CUE-401 to Address Unmet Need in the Treatment of Autoimmune Disease
Cue Biopharma (Nasdaq: CUE) has received positive Pre-IND feedback from the FDA regarding CUE-401, their lead autoimmune asset. CUE-401 is a first-in-class bispecific fusion protein designed to induce and expand regulatory T cells (Tregs) through the combined activity of TGF-β and modified IL-2.
The FDA reviewed the company's first-in-human trial design, including dose escalation plans, proposed populations, and safety monitoring protocols. Based on this feedback, Cue Biopharma plans to proceed with an IND filing once final enabling studies are completed.
The company believes CUE-401's novel mechanism, combining TGF-β and IL-2 activities, could provide durable immune rebalancing and tolerance across multiple disease indications, potentially disrupting the current Treg-directed therapy landscape.
Cue Biopharma (Nasdaq: CUE) ha ricevuto un riscontro positivo dalla FDA in merito al Pre-IND relativo a CUE-401, il loro principale farmaco per le malattie autoimmuni. CUE-401 è una proteina di fusione bispecifica innovativa, progettata per indurre ed espandere le cellule T regolatorie (Tregs) attraverso l'attività combinata di TGF-β e IL-2 modificata.
La FDA ha esaminato il disegno dello studio clinico di prima somministrazione nell’uomo, inclusi i piani di aumento della dose, le popolazioni proposte e i protocolli di monitoraggio della sicurezza. Sulla base di questo riscontro, Cue Biopharma intende procedere con la presentazione della domanda IND una volta completati gli studi abilitanti finali.
L’azienda ritiene che il meccanismo innovativo di CUE-401, che combina le attività di TGF-β e IL-2, possa offrire un riequilibrio immunitario duraturo e tolleranza in diverse indicazioni patologiche, con il potenziale di rivoluzionare l’attuale panorama delle terapie dirette alle Treg.
Cue Biopharma (Nasdaq: CUE) ha recibido comentarios positivos previos al IND de la FDA sobre CUE-401, su principal producto para enfermedades autoinmunes. CUE-401 es una proteína de fusión bispecífica innovadora diseñada para inducir y expandir las células T reguladoras (Tregs) mediante la actividad combinada de TGF-β e IL-2 modificada.
La FDA revisó el diseño del primer ensayo en humanos de la compañía, incluyendo los planes de escalado de dosis, las poblaciones propuestas y los protocolos de monitoreo de seguridad. Con base en estos comentarios, Cue Biopharma planea proceder con la presentación del IND una vez que se completen los estudios finales habilitantes.
La empresa considera que el novedoso mecanismo de CUE-401, que combina las actividades de TGF-β e IL-2, podría ofrecer un reequilibrio inmunológico duradero y tolerancia en múltiples indicaciones, con el potencial de transformar el panorama actual de terapias dirigidas a las Tregs.
Cue Biopharma (나스닥: CUE)는 주요 자가면역 치료제인 CUE-401에 대해 FDA로부터 긍정적인 Pre-IND 피드백을 받았습니다. CUE-401은 TGF-β와 변형된 IL-2의 결합 작용을 통해 조절 T세포(Tregs)를 유도하고 확장하도록 설계된 최초의 이중특이성 융합 단백질입니다.
FDA는 회사의 최초 인체 임상시험 설계, 용량 증량 계획, 대상 환자군 및 안전성 모니터링 프로토콜을 검토했습니다. 이 피드백을 바탕으로 Cue Biopharma는 최종 임상시험 준비가 완료되는 대로 IND 신청을 진행할 계획입니다.
회사는 TGF-β와 IL-2 활동을 결합한 CUE-401의 혁신적인 기전이 여러 질환에서 지속적인 면역 균형 회복과 내성을 제공할 수 있어, 현재 Treg 표적 치료 분야에 변화를 가져올 잠재력이 있다고 믿고 있습니다.
Cue Biopharma (Nasdaq : CUE) a reçu un retour positif pré-IND de la part de la FDA concernant CUE-401, leur principal candidat thérapeutique pour les maladies auto-immunes. CUE-401 est une protéine de fusion bispécifique innovante conçue pour induire et étendre les cellules T régulatrices (Tregs) grâce à l’activité combinée du TGF-β et de l’IL-2 modifiée.
La FDA a examiné le plan d’essai clinique chez l’humain, incluant les protocoles d’escalade des doses, les populations ciblées et les procédures de surveillance de la sécurité. Sur la base de ce retour, Cue Biopharma prévoit de déposer une demande IND une fois les études finales de validation achevées.
L’entreprise estime que le mécanisme novateur de CUE-401, combinant les activités du TGF-β et de l’IL-2, pourrait offrir un rééquilibrage immunitaire durable et une tolérance sur plusieurs indications, avec le potentiel de bouleverser le paysage actuel des thérapies ciblant les Tregs.
Cue Biopharma (Nasdaq: CUE) hat von der FDA positives Pre-IND-Feedback zu CUE-401, ihrem führenden Autoimmunwirkstoff, erhalten. CUE-401 ist ein neuartiges bispezifisches Fusionsprotein, das darauf ausgelegt ist, regulatorische T-Zellen (Tregs) durch die kombinierte Wirkung von TGF-β und modifiziertem IL-2 zu induzieren und zu erweitern.
Die FDA prüfte das Studiendesign der ersten klinischen Prüfung am Menschen, einschließlich Dosiseskalationsplänen, vorgeschlagenen Patientengruppen und Sicherheitsüberwachungsprotokollen. Basierend auf diesem Feedback plant Cue Biopharma, nach Abschluss der abschließenden unterstützenden Studien einen IND-Antrag einzureichen.
Das Unternehmen ist der Ansicht, dass der neuartige Wirkmechanismus von CUE-401, der TGF-β- und IL-2-Aktivitäten kombiniert, eine dauerhafte Immun-Neujustierung und Toleranz bei verschiedenen Krankheitsbildern ermöglichen könnte und somit das aktuelle Therapiefeld für Treg-orientierte Behandlungen verändern kann.
- Received positive FDA feedback on Pre-IND submission for CUE-401
- Novel first-in-class bispecific molecule with potential for multiple disease indications
- Differentiated mechanism of action from other Treg-directed therapies
- Potential for durable, long-lasting immune rebalance and tolerance
- Still requires completion of final IND enabling studies before submission
- Early-stage development with no clinical data yet
- Effectiveness and safety in humans yet to be demonstrated
Insights
FDA's positive feedback on CUE-401's pre-IND package marks significant regulatory progress for Cue Biopharma's novel autoimmune treatment platform.
The FDA's feedback on Cue Biopharma's pre-IND briefing for CUE-401 represents a critical regulatory milestone that clears the path toward an IND submission following completion of final enabling studies. This development is particularly significant as CUE-401 employs a first-in-class mechanism combining TGF-β and modified IL-2 to induce and expand regulatory T cells (Tregs) in vivo.
The regulatory feedback specifically addressed key elements including the first-in-human trial design, dose escalation approach, proposed patient populations, and safety monitoring protocols. The positive nature of this interaction suggests the FDA sees no fundamental obstacles to the program's advancement, which significantly de-risks the upcoming IND submission process.
What's mechanistically noteworthy about CUE-401 is its potential to go beyond simply expanding natural Tregs - it aims to convert effector/autoreactive T cells into cells with regulatory phenotypes, potentially establishing immune tolerance. This "master switch" approach differentiates CUE-401 from other Treg-directed therapies currently in development.
For investors, this regulatory progress indicates Cue Biopharma is executing effectively on its clinical development strategy and suggests their novel platform technology is gaining validation. However, the timeline to market remains lengthy, as the company must still complete IND-enabling studies, file the formal IND, and progress through clinical trials before potential commercialization.
CUE-401, a first-in-class bispecific molecule designed to induce and expand Tregs in vivo through the co-activity of transforming TGF-β and a modified variant of IL-2
BOSTON, June 24, 2025 (GLOBE NEWSWIRE) -- Cue Biopharma, Inc. (Nasdaq: CUE), a clinical-stage biopharmaceutical company developing a novel class of therapeutic biologics to selectively engage and modulate disease-specific T cells for the treatment of autoimmune disease, today announced it has received Pre-Investigational New Drug (Pre-IND) feedback from the U.S. Food and Drug Administration (FDA). The FDA reviewed the first-in-human trial design, including the Company’s plan for dose escalation, proposed populations and safety monitoring plan. On the basis of the FDA feedback, the Company, intends to file an IND pending completion of final IND enabling studies. CUE-401 is the Company’s lead autoimmune asset, a first-in-class bispecific fusion protein/molecule designed to induce and expand regulatory T cells (Tregs) in vivo through the co-activity of transforming growth factor beta (TGF-β) and a modified variant of interleukin 2 (IL-2).
“We are highly encouraged by the FDA’s positive feedback on our proposed development plan for this important program. We believe CUE-401, with its first-in-class mechanism exploiting the combined activities of TGF-β and IL-2 is a potentially disruptive approach differentiated from other Treg-directed therapies, and has the potential to provide durable, long-lasting immune rebalance and tolerance addressing multiple, significant disease indications,” said Daniel Passeri, chief executive officer of Cue Biopharma.
Dr. Dan Baker, chief development officer of Cue Biopharma commented, “CUE-401’s mechanistic design extends beyond nTreg proliferation by transforming effector/autoreactive responses to an anti-inflammatory and/or suppressive response, with the prospects of establishing tolerance. The combination of interleukin 2 (IL-2) and transforming growth factor beta (TGF-ß) is considered the ‘master switch’ for conversion of activated T effector cells into T cells with a regulatory phenotype.”
About CUE-401
CUE-401 is a preclinical, bispecific fusion protein designed to induce and expand regulatory T cells (Tregs) through the co-activity of modified variants of transforming growth factor beta (TGF-β) and interleukin 2 (IL-2) with therapeutic potential across a range of T-cell mediated autoimmune and inflammatory diseases.
CUE-401 has been engineered to harness the Treg induction capacity of TGF-β combined with IL-2 signaling to provide what Cue Biopharma believes to be superior quality and stability of Tregs. The design and specifications of CUE-401 have been guided by leading scientific publications demonstrating that both IL-2 and TGF-β are required for stable and efficient production of active and durable Tregs.
CUE-401 is designed to overcome multiple hurdles required to exploit the therapeutic potential of a master switch with a first-in-class, bispecific molecule integrating a masked TGF-ß, with our clinically validated, attenuated IL-2 with an antibody Fc fragment. This novel design provides for “conditional binding” and avoids off target activity, simplifies manufacturing and has highly differentiated findings in multiple pre-clinical models.
In these models, CUE-401 behaves as a master switch to convert autoreactive effector T cells (inflammatory cells) into stable, induced T-regulatory cells (iTregs). These findings suggest that CUE-401 acts by establishing a ‘tolerance positive feedback loop’ that not only increases nonspecific Treg populations, but critically, reduces and converts specific autoreactive T cells into transdifferentiated iTregs that are specific for the disease-causing autoantigens.
About Cue Biopharma
Cue Biopharma, a clinical-stage biopharmaceutical company, is developing a novel class of injectable biologics to selectively engage and modulate disease-specific T cells directly within the patient’s body. The company’s proprietary platform, Immuno-STAT™ (Selective Targeting and Alteration of T cells), and biologics are designed to harness the curative potential of the body’s intrinsic immune system without the adverse effects of broad systemic immune modulation.
Headquartered in Boston, Massachusetts, we are led by an experienced management team with deep expertise in immunology and immuno-oncology as well as the design and clinical development of protein biologics.
For more information please visit www.cuebiopharma.com and follow us on X and LinkedIn.
Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include, but are not limited to, those regarding: the company’s expectations regarding the planned IND filing for CUE-401; the Company’ expectations regarding the potential characteristics and benefit of CUE-401; the company’s belief that the Immuno-STAT platform stimulates targeted immune modulation through the selective modulation of disease-relevant T cell and the applicability of the company’s platform across many cancers and autoimmune diseases; and the company’s business strategies, plans and prospects. Forward-looking statements, which are based on certain assumptions and describe the company’s future plans, strategies and expectations, can generally be identified by the use of forward-looking terms such as “believe,” “expect,” “may,” “will,” “should,” “would,” “could,” “seek,” “intend,” “plan,” “goal,” “project,” “estimate,” “anticipate,” “strategy,” “future,” “likely” or other comparable terms, although not all forward-looking statements contain these identifying words. All statements other than statements of historical facts included in this press release regarding the company’s pipeline of product candidates and platforms, and its strategies, prospects, plans and objectives are forward-looking statements. Important factors that could cause the company’s actual results and financial condition to differ materially from those indicated in the forward-looking statements include, among others, the Company’s ability to successfully advance is development plan for CUE-401; potential setbacks in the company’s research and development efforts including negative or inconclusive results from its preclinical studies or clinical trials or the company’s ability to replicate in later clinical trials positive results found in preclinical studies and early-stage clinical trials of its product candidates; serious and unexpected drug-related side effects or other safety issues experienced by participants in clinical trials; its ability to secure required U.S. Food and Drug Administration (“FDA”) or other governmental approvals for its product candidates, including FDA clearance of any future IND submission for CUE-401, and the breadth of any approved indication; adverse effects caused by public health pandemics, including possible effects on the company’s trials; delays and changes in regulatory requirements, policy and guidelines including potential delays in submitting required regulatory applications to the FDA; the company’s reliance on licensors, collaborators, contract research organizations, suppliers and other business partners; the company’s ability to obtain adequate financing to fund its business operations in the near term; the company’s ability to maintain and enforce necessary patent and other intellectual property protection; competitive factors; general economic and market conditions and the other risks and uncertainties described in the Risk Factors and in Management's Discussion and Analysis of Financial Condition and Results of Operations sections of the company’s most recently filed Annual Report on Form 10-K and any subsequently filed Quarterly Report(s) on Form 10-Q. Any forward-looking statement made by the company in this press release is based only on information currently available to the company and speaks only as of the date on which it is made. The company undertakes no obligation to publicly update any forward-looking statement, whether written or oral, that may be made from time to time, whether as a result of new information, future developments or otherwise.
Investor Contact
Marie Campinell
Senior Director, Corporate Communications
Cue Biopharma, Inc.
mcampinell@cuebio.com
Media Contact
Jonathan Pappas
LifeSci Communications
jpappas@lifescicomms.com
FAQ
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