Imfinzi (durvalumab)

The TOPAZ-1 study results represented significant advancement in the treatment of advanced biliary tract cancer (BTC) by combining durvalumab with gemcitabine-cisplatin (DGC). Further real-world investigations are still warranted to determine if the DGC regimen has a broader therapeutic indication and to identify predictive markers for survival benefit. Efforts are required to improve the cost-effectiveness of the DGC regimen to facilitate its wider and standardized use.

Our findings directly inform clinical guidelines, address gaps in current therapeutic decision-making. Durvalumab or pembrolizumab combined with GC are optimal first-line regimens for advanced BTC, balancing survival benefits and safety. Sintilimab plus anlotinib combined with GC demonstrates superior PFS but requires further validation. While EGFR inhibitors plus chemotherapy demonstrate potential in KRAS wild-type patients, confirmation in large-scale RCTs is required. PD-L1 expression may represent a promising predictive biomarker for response to PD-1 inhibitor therapy.

Gemcitabine-cisplatin remains effective and tolerable in metastatic GBC. Despite the emergence of Gemcitabine-Cisplatin with durvalumab as new standard, this prospective dataset provides valuable real-world outcomes from a high-incidence region with limited access to immunotherapy.

P=N/A, N=200, Recruiting, AstraZeneca | Not yet recruiting --> Recruiting | Trial completion date: Apr 2028 --> Sep 2028 | Trial primary completion date: Apr 2028 --> Sep 2028

Neoadjuvant cisplatin-based combination chemotherapy or perioperative durvalumab with neoadjuvant gemcitabine-cisplatin has been the primary treatment for localized muscle-invasive bladder cancer (MIBC)...Twenty-five patients (cT2-4aN0-2M0) received at least four cycles of RC48 (2.0 mg/kg, Q2W or Q3W) with toripalimab, tislelizumab, or pembrolizumab, followed by radical cystectomy...Treatment-related adverse events were manageable. These findings suggest that RC48 combined with PD-1 inhibitors is a promising neoadjuvant strategy for localized MIBC and warrants further validation in biomarker-selected populations.

P2, N=176, Recruiting, Fondazione Ricerca Traslazionale | Trial primary completion date: May 2024 --> May 2026 | Trial completion date: Apr 2025 --> May 2027

Prior radiotherapy improves PFS in patients treated with tremelimumab-durvalumab for advanced HCC. The abscopal effect and up-regulation of immune mechanisms may contribute to improved outcomes.

P2, N=172, Recruiting, Nanjing Leads Biolabs Co.,Ltd | Not yet recruiting --> Recruiting

P1, N=120, Active, not recruiting, NeoTX Therapeutics Ltd. | Recruiting --> Active, not recruiting

P3, N=111, Active, not recruiting, AstraZeneca | Trial primary completion date: Sep 2025 --> Dec 2025