Tarextumab Gets FDA Orphan Drug Designation for Lung, Pancreatic Cancer

The US Food and Drug Administration (FDA) has granted orphan drug designation to the OncoMed agent tarextumab for the treatment of pancreatic cancer and lung cancer. Tarextumab (formerly OMP-59R5) is a fully human monoclonal antibody targeting the Notch 2/3 receptors.

“We are excited to receive two separate orphan drug designations for tarextumab for the treatment of pancreatic and small-cell lung cancer,” said Paul J. Hastings, OncoMed’s chairman and chief executive officer, in a statement.

Earlier this month the drug company announced final phase 1b clinical and biomarker data from the ALPINE (Antibody Therapy in First-Line Pancreatic Cancer Investigating Anti-Notch Efficacy and Safety) study, which tested tarextumab in combination with gemcitabine plus nab-paclitaxel in pancreatic cancer.

Of the 29 patients in the trial, 21 (73%) achieved either a partial response or stable disease with the combination therapy.

The drug was well tolerated with a manageable toxicity profile. The most common adverse events (> 50%) included anemia, fatigue, diarrhea, and thrombocytopenia. Other common adverse events (≥ 25%) included nausea, alopecia, vomiting, edema, decreased appetite, pyrexia, neuropathy, and decreased weight.

Biomarker analyses found that tumors with high levels of Notch 3 expression may correlate with higher response rates and better survival. Median progression-free survival was 6.6 months in patients with high Notch 3 expression, and overall survival was 14.6 months.

In PINNACLE (A Phase Ib/II Study of OMP-59R5 in Combination With Etoposide and Platinum Therapy in Subjects With Untreated Extensive-Stage Small-Cell Lung Cancer), researchers tested tarextumab in combination with platinum chemotherapy and etoposide. Of 16 evaluable patients, 13 achieved partial response (81.3%) and 3 (18.8%) achieved stable disease.

Final data from the phase Ib portion of the study, including safety and survival data, will be presented later this year at a medical meeting.

OncoMed is currently enrolling patients for two randomized phase II clinical trials of tarextumab in pancreatic cancer and extensive-stage small-cell lung cancer.

The FDA orphan drug designation program provides incentives for drug companies to develop agents that treat rare diseases that might otherwise be overlooked.

Approximately 46,000 new cases of pancreatic cancer are diagnosed each year in the United States, according to the American Cancer Society. It is the fourth-leading cause of cancer deaths. The majority of cases are diagnosed after the disease has spread locally or metastasized, and the median overall survival is less than 12 months.

Small-cell lung cancer makes up about 10% to 15% of the over 220,000 newly diagnosed lung cancer cases in the United States each year. The disease tends to grow and spread quickly, and is typically not discovered until it has metastasized. For these patients, with extensive-stage disease, the median overall survival is less than 12 months.