Pre-clinical studies of EC2629, a highly potent folate- receptor-targeted DNA crosslinking agent

Sci Rep. 2020 Jul 29;10(1):12772. doi: 10.1038/s41598-020-69682-9.

Abstract

Folate receptor (FR)-targeted small molecule drug conjugates (SMDCs) have shown promising results in early stage clinical trials with microtubule destabilizing agents, such as vintafolide and EC1456. In our effort to develop FR-targeted SMDCs with varying mechanisms of action, we synthesized EC2629, a folate conjugate of a DNA crosslinking agent based on a novel DNA-alkylating moiety. This agent was found to be extremely potent with an in vitro IC50 ~ 100× lower than folate SMDCs constructed with various microtubule inhibitors. EC2629 treatment of nude mice bearing FR-positive KB human xenografts led to cures in 100% of the test animals with very low dose levels (300 nmol/kg) following a convenient once a week schedule. The observed activity was not accompanied by any noticeable weight loss (up to 20 weeks post end of dosing). Complete responses were also observed against FR-positive paclitaxel (KB-PR) and cisplatin (KB-CR) resistant models. When evaluated against FR-positive patient derived xenograft (PDX) models of ovarian (ST070), endometrial (ST040) and triple negative breast cancers (ST502, ST738), EC2629 showed significantly greater anti-tumor activity compared to their corresponding standard of care treatments. Taken together, these studies thus demonstrated that EC2629, with its distinct DNA reacting mechanism, may be useful in treating FR-positive tumors, including those that are classified as drug resistant.

MeSH terms

  • Alkylating Agents / chemistry
  • Animals
  • Antineoplastic Agents / pharmacology*
  • Cattle
  • Cisplatin / administration & dosage
  • Cross-Linking Reagents / pharmacology*
  • DNA / chemistry*
  • Dogs
  • Drug Delivery Systems
  • Drug Design
  • Drug Evaluation, Preclinical
  • Endometrial Neoplasms / drug therapy*
  • Female
  • Folate Receptors, GPI-Anchored / chemistry*
  • Folic Acid / analogs & derivatives
  • Folic Acid / pharmacology
  • Humans
  • Inhibitory Concentration 50
  • KB Cells
  • Ligands
  • Mice
  • Mice, Inbred C57BL
  • Mice, Nude
  • Ovarian Neoplasms / drug therapy*
  • Paclitaxel / administration & dosage
  • Rats
  • Triple Negative Breast Neoplasms / drug therapy*
  • Vinca Alkaloids / pharmacology
  • Xenograft Model Antitumor Assays

Substances

  • Alkylating Agents
  • Antineoplastic Agents
  • Cross-Linking Reagents
  • EC145
  • Folate Receptors, GPI-Anchored
  • Ligands
  • Vinca Alkaloids
  • DNA
  • Folic Acid
  • Paclitaxel
  • Cisplatin