Partner Therapeutics has announced the submission of a supplemental Biologics License Application (sBLA) to the US Food and Drug Administration (FDA) for BIZENGRI (zenocutuzumab-zbco), seeking approval for the treatment of adults with advanced unresectable or metastatic cholangiocarcinoma harboring a neuregulin 1 (NRG1) gene fusion. Cholangiocarcinoma is a rare, aggressive malignancy of the bile ducts with few effective treatment options. There are no approved therapies specifically targeting cholangiocarcinoma harboring NRG1 gene fusions.

"This submission marks an important step in advancing BIZENGRI for patients with NRG1 fusion positive cholangiocarcinoma, a population with limited treatment options, and historically poor outcomes. Cholangiocarcinoma remains a challenging and aggressive disease, and we believe these data support the potential of BIZENGRI to address a critical unmet need for patients whose tumors are driven by NRG1 gene fusions. Tissue?based RNA testing is essential to identify rare oncogenic fusions such as NRG1 and ensure patients with these actionable alterations are not overlooked," said Pritesh J. Gandhi, Chief Development Officer, Partner Therapeutics.

The sBLA is supported by data from the eNRGy study evaluating zenocutuzumab-zbco in patients with NRG1 fusion-positive cancers, including cholangiocarcinoma. In the cholangiocarcinoma cohort, BIZENGRI demonstrated an Overall Response Rate (ORR) of 36.8 percent (95 percent CI: 16.3, 61.6 percent) and a median Duration of Response (DOR) of 12.9 months, as assessed by Blinded Independent Central Review (BICR). Treatment was generally well tolerated, and no patients discontinued therapy due to adverse events.

Based on these data, National Comprehensive Cancer Network (NCCN) added zenocutuzumab?zbco to the Oncology Clinical Practice Guidelines for biliary tract cancers as a Category 2A subsequent?line therapy and as a Category 2B recommendation for front?line treatment of NRG1 fusion-positive cholangiocarcinoma.

"Cholangiocarcinoma remains a devastating disease, particularly in the advanced setting. The identification of NRG1 gene fusions has highlighted an actionable biomarker, and the eNRGy study data suggest that targeted inhibition with zenocutuzumab may represent a meaningful treatment approach for these patients," said Dr. James Cleary, Dana-Farber Cancer Institute.

BIZENGRI received US Food and Drug Administration (FDA) accelerated approval for the treatment of adults with advanced unresectable or metastatic Non-Small Cell Lung Cancer (NSCLC) and pancreatic adenocarcinoma harboring NRG1 gene fusions with disease progression on or after prior systemic therapy.