Candel Therapeutics Receives EMA Orphan Designation for CAN-2409 for the Treatment of Pancreatic Cancer
Candel Therapeutics (Nasdaq: CADL) has received European Medicines Agency (EMA) Orphan Designation for its CAN-2409 treatment for pancreatic cancer. This adds to existing FDA Orphan Drug and Fast Track Designations received in 2024 and 2023.
The company's phase 2a clinical trial demonstrated remarkable results, with median overall survival of 31.4 months in the CAN-2409 plus standard of care arm versus 12.5 months in the control arm. Notably, three of seven treated patients were still alive at data cut-off with survival of 66.0, 63.6, and 35.8 months.
CAN-2409 is an investigational immunotherapy that delivers the HSV-tk gene to tumor cells, potentially inducing systemic immune responses against multiple therapy-resistant solid tumors. The EMA designation provides benefits including reduced fees, protocol assistance, and up to 10 years of market exclusivity if approved.
Candel Therapeutics (Nasdaq: CADL) ha ottenuto la Designazione Orfana dall'Agenzia Europea per i Medicinali (EMA) per il suo trattamento CAN-2409 per il cancro al pancreas. Questo si aggiunge alle precedenti Designazioni Orfane e Fast Track ricevute dalla FDA nel 2024 e 2023.
Lo studio clinico di fase 2a dell'azienda ha mostrato risultati notevoli, con una sopravvivenza mediana complessiva di 31,4 mesi nel gruppo trattato con CAN-2409 più lo standard di cura, rispetto a 12,5 mesi nel gruppo di controllo. In particolare, tre dei sette pazienti trattati erano ancora vivi al momento del cut-off dei dati, con sopravvivenze di 66,0, 63,6 e 35,8 mesi.
CAN-2409 è una terapia immunitaria sperimentale che veicola il gene HSV-tk nelle cellule tumorali, potenzialmente inducendo risposte immunitarie sistemiche contro diversi tumori solidi resistenti alle terapie. La designazione EMA offre vantaggi come riduzioni delle tariffe, assistenza sul protocollo e fino a 10 anni di esclusiva di mercato in caso di approvazione.
Candel Therapeutics (Nasdaq: CADL) ha recibido la Designación de Medicamento Huérfano por parte de la Agencia Europea de Medicamentos (EMA) para su tratamiento CAN-2409 para el cáncer de páncreas. Esto se suma a las Designaciones de Medicamento Huérfano y Fast Track otorgadas por la FDA en 2024 y 2023.
El ensayo clínico de fase 2a de la compañía mostró resultados notables, con una supervivencia global mediana de 31,4 meses en el brazo de CAN-2409 más el estándar de cuidado, frente a 12,5 meses en el brazo de control. Cabe destacar que tres de los siete pacientes tratados seguían vivos al corte de datos, con supervivencias de 66,0, 63,6 y 35,8 meses.
CAN-2409 es una inmunoterapia en investigación que entrega el gen HSV-tk a las células tumorales, potencialmente induciendo respuestas inmunitarias sistémicas contra múltiples tumores sólidos resistentes a tratamientos. La designación EMA ofrece beneficios como reducción de tarifas, asistencia en el protocolo y hasta 10 años de exclusividad en el mercado si se aprueba.
Candel Therapeutics (나스닥: CADL)는 췌장암 치료제인 CAN-2409에 대해 유럽의약품청(EMA)으로부터 희귀의약품 지정(Orphan Designation)을 받았습니다. 이는 2023년과 2024년에 FDA로부터 받은 희귀의약품 및 패스트트랙 지정에 추가되는 것입니다.
회사의 2a상 임상시험 결과, CAN-2409와 표준 치료 병용군의 중앙 생존기간이 31.4개월로 대조군의 12.5개월에 비해 현저히 우수한 결과를 보였습니다. 특히, 7명 중 3명의 환자가 데이터 컷오프 시점에도 생존해 각각 66.0, 63.6, 35.8개월의 생존 기간을 기록했습니다.
CAN-2409는 종양 세포에 HSV-tk 유전자를 전달하는 실험적 면역치료제로, 여러 치료 저항성 고형암에 대해 전신 면역 반응을 유도할 가능성이 있습니다. EMA 지정은 수수료 감면, 프로토콜 지원, 승인 시 최대 10년간 시장 독점권 등의 혜택을 제공합니다.
Candel Therapeutics (Nasdaq : CADL) a obtenu la désignation médicament orphelin de l'Agence européenne des médicaments (EMA) pour son traitement CAN-2409 contre le cancer du pancréas. Cette reconnaissance s'ajoute aux désignations médicament orphelin et voie rapide accordées par la FDA en 2023 et 2024.
L'essai clinique de phase 2a de la société a révélé des résultats remarquables, avec une survie globale médiane de 31,4 mois dans le groupe CAN-2409 plus traitement standard, contre 12,5 mois dans le groupe contrôle. Notamment, trois des sept patients traités étaient encore en vie au moment de la coupure des données, avec des survies de 66,0, 63,6 et 35,8 mois.
CAN-2409 est une immunothérapie expérimentale qui délivre le gène HSV-tk aux cellules tumorales, pouvant induire des réponses immunitaires systémiques contre plusieurs tumeurs solides résistantes aux traitements. La désignation EMA offre des avantages tels que des réductions de frais, une assistance au protocole et jusqu'à 10 ans d'exclusivité commerciale en cas d'approbation.
Candel Therapeutics (Nasdaq: CADL) hat von der Europäischen Arzneimittel-Agentur (EMA) die Orphan-Drug-Zulassung für seine CAN-2409-Behandlung bei Bauchspeicheldrüsenkrebs erhalten. Dies ergänzt die bereits 2023 und 2024 von der FDA erhaltenen Orphan-Drug- und Fast-Track-Designationen.
Die Phase-2a-Studie des Unternehmens zeigte bemerkenswerte Ergebnisse mit einem medianen Gesamtüberleben von 31,4 Monaten in der CAN-2409- plus Standardbehandlungsgruppe gegenüber 12,5 Monaten in der Kontrollgruppe. Bemerkenswert ist, dass drei von sieben behandelten Patienten zum Zeitpunkt der Datenauswertung noch lebten, mit Überlebenszeiten von 66,0, 63,6 und 35,8 Monaten.
CAN-2409 ist eine experimentelle Immuntherapie, die das HSV-tk-Gen in Tumorzellen einschleust und möglicherweise systemische Immunantworten gegen mehrere therapieresistente solide Tumoren induziert. Die EMA-Zulassung bietet Vorteile wie reduzierte Gebühren, Protokollunterstützung und bis zu 10 Jahre Marktexklusivität bei Zulassung.
- Phase 2a trial showed significant survival benefit with median OS of 31.4 months vs 12.5 months in control arm
- Three of seven treated patients demonstrated long-term survival (35.8 to 66.0 months)
- EMA Orphan Designation provides potential 10-year market exclusivity in EU
- Multiple regulatory designations received across different jurisdictions and indications
- Small patient sample size in clinical trial (only seven patients)
- Treatment still in early clinical development phase
Insights
EMA Orphan Designation for CAN-2409 boosts Candel's pancreatic cancer therapy after impressive 31.4 vs 12.5 months survival data.
Candel Therapeutics has secured European Medicines Agency (EMA) Orphan Designation for CAN-2409 in pancreatic cancer, complementing its existing FDA Orphan Drug and Fast Track Designations for pancreatic ductal adenocarcinoma (PDAC). This triple regulatory recognition significantly strengthens the company's development pathway for this challenging indication.
The clinical data driving these designations is particularly noteworthy. In the phase 2a trial, CAN-2409 plus standard care demonstrated an estimated median overall survival of 31.4 months versus just 12.5 months in the control arm—a remarkable 151% improvement in this historically treatment-resistant cancer. Perhaps more striking is the emergence of a long survival tail, with three of seven treated patients still alive at 66.0, 63.6, and 35.8 months after enrollment at the February 2025 data cutoff.
CAN-2409's mechanism explains these results: it delivers the HSV-tk gene to tumor cells which, when combined with valacyclovir, triggers immunogenic cell death that releases tumor antigens. This effectively converts "cold" tumors (immunologically inactive) to "hot" tumors (immunologically active)—a critical transformation for pancreatic cancer, which typically evades immune detection through its dense stroma and immunosuppressive microenvironment.
The EMA designation provides tangible benefits including protocol assistance, reduced fees, and potentially 10 years of market exclusivity upon approval. For patients with pancreatic cancer, where five-year survival rates remain below 10%, therapies demonstrating such significant survival advantages represent a genuine breakthrough in a field where progress has been frustratingly slow.
NEEDHAM, Mass., July 24, 2025 (GLOBE NEWSWIRE) -- Candel Therapeutics, Inc. (Candel or the Company) (Nasdaq: CADL), a clinical-stage biopharmaceutical company focused on developing multimodal biological immunotherapies to help patients fight cancer, today announced that the European Medicines Agency (EMA) has granted Orphan Designation for CAN-2409 (aglatimagene besadenovec) for the treatment of pancreatic cancer. This designation complements CAN-2409’s existing U.S. Food and Drug Administration (FDA) Orphan Drug Designation and FDA Fast Track Designation for the treatment of pancreatic ductal adenocarcinoma (PDAC) awarded to CAN-2409 in April 2024 and December 2023, respectively, and underscores the significant unmet medical need in this disease beyond the U.S.
CAN-2409 is an investigational, off-the-shelf, replication-defective adenovirus engineered to deliver the herpes simplex virus thymidine kinase (HSV-tk) gene to tumor cells. When administered with a prodrug (valacyclovir or acyclovir), the HSV-tk enzyme converts the prodrug into DNA-incorporating nucleotide analogs, causing immunogenic cell death and the release of tumor (neo)antigens within the tumor microenvironment (TME). This mechanism has the potential to induce an individualized, systemic immune response against multiple therapy-resistant solid tumors, including prostate cancer, non-small cell lung cancer (NSCLC), and PDAC.
The Company previously reported positive overall survival (OS) data from its randomized controlled phase 2a clinical trial of CAN-2409 plus valacyclovir in borderline resectable PDAC, demonstrating remarkable survival benefits with estimated median OS of 31.4 months in the CAN-2409 plus standard of care arm versus 12.5 months in the standard of care control arm, supported by immunological biomarker data. Notably, three of seven patients treated with CAN-2409 were still alive at the time of data cut-off (February 20, 2025) with survival of 66.0, 63.6 and 35.8 months after enrollment, respectively, suggesting a long tail of survival.
“Pancreatic cancer remains one of the most challenging malignancies to treat, particularly in patients whose disease is unresectable,” said Garrett Nichols, MD, MS, Chief Medical Officer at Candel. “We observed that CAN-2409 profoundly remodels the tumor microenvironment, transforming it from ‘cold’ (non-inflamed and immunosuppressive) to ‘hot’ (inflamed with active immune infiltration) and extends survival.”
The EMA grants Orphan Designation to drugs and biologics intended for the treatment, diagnosis or prevention of rare, life-threatening or chronically debilitating diseases or conditions that affect fewer than five in 10,000 people in the European Union. Orphan Designation allows pharmaceutical companies certain benefits, including reduced regulatory fees, clinical protocol assistance, research grants and up to 10 years of market exclusivity in the European Union, if approved.
“Receiving EMA Orphan Designation for CAN-2409 represents a significant regulatory milestone for CAN-2409 in this disease,” said Paul Peter Tak, MD, PhD, FMedSci, President and Chief Executive Officer of Candel. “This designation, alongside our existing FDA Orphan Drug and Fast Track Designations for CAN-2409 in PDAC, underscores the promise of our novel multimodal immunotherapy approach. The notable benefits we’ve observed with CAN-2409, including evidence of a long tail of survival, highlight the transformative potential of this immunotherapy. As we advance our regulatory strategy across global markets, we remain committed to bringing CAN-2409 to patients who face limited therapeutic options. We look forward to continuing our development efforts and working with regulatory authorities across the world to make this innovative therapy available to patients in need.”
CAN-2409 has received regulatory designations recognizing its potential across multiple solid tumor indications. In addition to the EMA Orphan Designation announced today and the FDA Orphan Drug Designation and FDA Fast Track Designation for PDAC mentioned above, CAN-2409 has also received FDA Fast Track Designation for NSCLC and localized prostate cancer, as well as FDA Regenerative Medicine Advanced Therapy designation (RMAT) for intermediate-to-high risk, localized prostate cancer.
About CAN-2409
CAN-2409 (aglatimagene besadenovec), Candel’s most advanced multimodal biological immunotherapy candidate, is an investigational, off-the-shelf, replication-defective adenovirus designed to deliver the herpes simplex virus thymidine kinase (HSV-tk) gene to a patient’s tumor. After intratumoral administration, HSV-tk enzyme activity results in conversion of prodrug (valacyclovir) into deoxyribonucleic acid (DNA)-incorporating nucleotide analogs, leading to immunogenic cell death in cells exhibiting DNA damage and proliferating cells, with subsequent release of a variety of tumor (neo)antigens in the tumor microenvironment. At the same time, the adenoviral serotype 5 capsid protein promotes inflammation through the induction of expression of pro-inflammatory cytokines, chemokines, and adhesion molecules. Together, this regimen is designed to induce an individualized and specific CD8+ T cell-mediated response against the injected tumor and uninjected distant metastases for broad anti-tumor activity, based on in situ immunization against a variety of tumor antigens. CAN-2409 has the potential to treat a broad range of solid tumors. Encouraging monotherapy activity as well as combination activity with standard of care radiotherapy, surgery, chemotherapy, and immune checkpoint inhibitors have previously been shown in several preclinical and clinical settings. More than 1,000 patients have been dosed with CAN-2409 with a favorable tolerability profile to date, supporting the potential for combination with standard of care, when indicated.
About Candel Therapeutics
Candel is a clinical-stage biopharmaceutical company focused on developing off-the-shelf multimodal biological immunotherapies that elicit an individualized, systemic anti-tumor immune response to help patients fight cancer. Candel has established two clinical-stage multimodal biological immunotherapy platforms based on novel, genetically modified adenovirus and herpes simplex virus (HSV) gene constructs, respectively. CAN-2409 is the lead product candidate from the adenovirus platform.
The Company recently completed successful phase 2a clinical trials of CAN-2409 in NSCLC and PDAC, and a pivotal, placebo-controlled phase 3 clinical trial of CAN-2409 in localized prostate cancer, conducted under a Special Protocol Assessment (SPA) agreed with the FDA. Additionally, the FDA very recently granted Regenerative Medicine Advanced Therapy (RMAT) Designation to CAN-2409 for the treatment of newly diagnosed localized prostate cancer in patients with intermediate-to-high-risk disease.
CAN-3110 is the lead product candidate from the HSV platform and is currently in an ongoing phase 1b clinical trial in recurrent high-grade glioma (rHGG). Initial results were published in Nature and CAN-3110 received Fast Track Designation and Orphan Drug Designation from the FDA. Finally, Candel’s enLIGHTEN™ Discovery Platform is a systematic, iterative HSV-based discovery platform leveraging human biology and advanced analytics to create new viral immunotherapies for solid tumors.
For more information about Candel, visit: www.candeltx.com
Forward-Looking Statements
This press release includes certain disclosures that contain “forward-looking statements,” within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, express or implied statements regarding the timing and advancement of current and future development programs; expectations regarding the therapeutic benefit of the Company’s platforms, including the ability of its platforms to improve overall survival and/or disease-free survival of patients living with difficult to treat, solid tumors; and expectations regarding the potential benefits conferred by Orphan Designation. The words “may,” “will,” “could,” “would,” “should,” “expect,” “plan,” “anticipate,” “intend,” “believe,” “estimate,” “predict,” “project,” “potential,” “continue,” “target” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management’s current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, those risks and uncertainties related to the timing and advancement of development programs; expectations regarding the therapeutic benefit of the Company’s programs; that final data from the Company’s preclinical studies and completed clinical trials may differ materially from reported interim data from ongoing studies and trials; the Company’s ability to efficiently discover and develop product candidates; the Company’s ability to obtain and maintain regulatory approval of product candidates; the Company’s ability to maintain its intellectual property; the implementation of the Company’s business model, including strategic plans for the Company’s business and product candidates; and other risks identified in the Company’s filings with the U.S. Securities and Exchange Commission (SEC), including the Company’s most recent Annual Report on Form 10-K and Quarterly Report on Form 10-Q for the quarter ended March 31, 2025, each as filed with the SEC and any subsequent filings with the SEC. The Company cautions you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. The Company disclaims any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions, or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. Any forward-looking statements contained in this press release represent the Company’s views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date.
Investor Contact:
Theodore Jenkins
VP, Investor Relations and Business Development
Candel Therapeutics, Inc.
tjenkins@candeltx.com
Media Contact:
Ben Shannon
ICR Healthcare
CandelPR@icrhealthcare.com
FAQ
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