Arbele's ARB1002 Receives FDA Orphan Drug Designation for Pancreatic Cancer Treatment
Key Insights
The FDA has granted Orphan Drug Designation to ARB1002 (search), Arbele (search)'s investigational antibody-drug conjugate targeting CDH17 (search) for pancreatic cancer (search) treatment.
ARB1002 (search) combines an anti-CDH17 (search) monoclonal antibody with a cytotoxic agent to selectively target tumor cells while limiting off-target exposure.
The designation provides regulatory incentives including tax credits, fee exemptions, and seven years of market exclusivity following approval.
Arbele (search), a clinical-stage biotechnology company, announced that the U.S. Food and Drug Administration has granted Orphan Drug Designation to ARB1002 (search), its investigational antibody-drug conjugate for treating pancreatic cancer (search). The designation represents a significant regulatory milestone for the Seattle-based company's CDH17 (search)-targeted therapy program.
Novel ADC Design Targets Pancreatic Cancer
ARB1002 (search) is composed of an anti-CDH17 (search) monoclonal antibody chemically linked to a potent cytotoxic agent. The ADC is designed to selectively bind CDH17, a cell-surface adhesion molecule overexpressed in pancreatic cancer (search), enabling targeted delivery of the cytotoxic payload to tumor cells while limiting off-target exposure.
"Orphan Drug Designation for ARB1002 (search) marks a significant regulatory milestone for Arbele (search) and reflects the critical need for new treatment options for patients with pancreatic cancer (search)," said Dr. Linda Wu, Chief Development Officer of Arbele. "This designation reinforces our confidence in the therapeutic potential of ARB1002 and supports its continued advancement through clinical development."
Addressing Critical Unmet Medical Need
Pancreatic cancer (search) is among the deadliest solid tumors, with limited effective therapies and poor long-term survival outcomes. The FDA's Orphan Drug Designation program is intended to incentivize the development of therapies for rare diseases affecting fewer than 200,000 patients in the United States.
The designation offers several regulatory benefits including tax credits for qualified clinical development expenses, exemption from certain FDA user fees, and seven years of market exclusivity following regulatory approval.
Clinical Development Timeline
ARB1002 (search) is planned to enter a Phase 1 clinical trial this year, with Arbele (search) completing IND-enabling studies to support its clinical evaluation in pancreatic cancer (search). The company is positioning the therapy as part of its broader CDH17 (search)-targeted development strategy.
"This designation highlights CDH17 (search) as a promising target in pancreatic cancer (search) and validates Arbele (search)'s ADC development strategy," added Dr. Tony Wong, Chief Technology Officer. "We look forward to continued engagement with the FDA as we progress ARB1002 (search)."
Company Focus on Gastrointestinal Cancers
Arbele (search) is developing potential first-in-class and best-in-class immunotherapies for gastrointestinal cancers including colorectal, gastric and oesophageal cancers with high unmet medical needs. With a focus on CDH17 (search)-targeted T-cell engagers and antibody-drug conjugates, the company's mission is to improve survival outcomes for patients with limited treatment options.