Nanotherapy Targeting Metastatic Factor RHAMM Positive Tumors

Background & Unmet Need

• Pancreatic neuroendocrine tumors (PNETs) often lead to incurable, metastatic cancer, with only a 15% five-year survival rate
• Approved therapies such as sunitinib (a multi-targeted receptor tyrosine kinase inhibitor) and everolimus (an mTOR inhibitor) have advanced the standard of care for PNETs
• However, many patients eventually develop drug resistance and relapse, resulting in poor long-term survival
• Unmet Need: Novel targets and therapies for PNET treatment, particularly for patients who relapsed or refractory disease

Technology Overview

• The Technology: Nanoparticle-based methods and compositions for the treatment of RHAMM-positive cancers
• The Discovery: Identification of an isoform of Receptor for Hyaluronic Acid Mediated Motility (RHAMMB) as being consistently upregulated in various high-grade tumors and metastases including PNETs
• The inventors designed gold nanoparticles (AuNP) that carry the pro-apoptotic peptide KLA and silencing RNA for Bcl-xL (siBcl-xL) to specifically target RHAMMB+ PNETs
• PoC Data: The nanoparticles successfully targeted RHAMMB+ PNETs and led to a significant reduction in tumor weight and volume during in vivo studies
• A synergistic killing effect was achieved with co-delivery of siBcl-xL and KLA peptide compared to either agent alone

Technology Applications

• RHAMMB-specific targeting and treatment of PNETs
• Treatment of other solid tumors with demonstrated RHAMMBoverexpression (e.g., breast, pancreatic, ovarian, endometrial, lung, prostate, colorectal)

Technology Advantages

• AuNPs have tunable size, are biocompatible, and have low cytotoxicity
• RHAMMB-specific delivery targets tumors with minimal adverse effects to healthy cells
• Combinational therapy produces synergistic pro-apoptotic effects