Small molecule inhibitor of human epididymis protein 4 (HE4) for treating cancer


Epithelial ovarian cancer (EOC), is the fifth leading cause of cancer deaths in women, and the deadliest of all gynecologic cancers. The response of current chemotherapies has plateaued, creating a severe unmet need for superior therapies. Human epididymis protein 4 (HE4) is inordinately overexpressed in over 70% of EOC patients, acts as a prime driver of EOC growth, chemoresistance and tumor-immune evasion and is associated with highly reduced survival rates in EOC patients. Similarly, over 417,000 new cases of endometrial cancer (EC) are diagnosed with the majority presenting HE4 overexpression. Therefore, HE4 is an attractive therapeutic target and inhibition of HE4 could provide viable treatment options for patients in dire need of more effective therapies.

Technology Overview

Researchers at the University of Rochester have identified the first-in-field small molecule inhibitor of HE4 and have developed a biomarker (that is currently clinically used worldwide to diagnose HE4) which will serve as a companion marker to monitor new drug candidates’ response in EOC and EC patients. This molecule provides a method to reduce ascites and tumor deposition in the abdomen area and on the omentum, the key disease sites.


  • Can be used alone or combined with chemotherapy or used as a maintenance therapy post-surgery/chemotherapies to suppress ascites formation.
  • Potential uses for targeting a wide spectrum of cancers that go from reproductive organs to cancers of the airways and mouth. 
  • Inhibition of HE4 may restore patient’s antitumor immune response and may lead to eradication of established tumors via re‐invigorating the patient’s immune system.
  • Novel approach for determining both whether a patient would benefit from immunotherapy as well as for monitoring the course of treatment in a patient undergoing immunotherapy, based on the assessment of expression levels of HE4.


  • Ovarian cancer
  • Endometrial cancer
  • Pancreatic cancer and ductal breast cancers
  • Inflammatory disorders
  • Fibrosis of organs

As a first‑in‑field inhibitor of HE4, this molecule could potentially occupy a significant place in the growing market for both EOC and EC. The ovarian cancer market was valued at $1.8B in 2018 and it will continue to rise significantly. By 2028, the total value of the market is forecast to reach $6.7B. Similarly, the EC market will experience overall growth, with total sales increasing from $2.5B in 2020 to $5.3B in 2030.

URV Reference Number: 6-20042
Patent Information:
For Information, Contact:
Blaze Pharoah
Licensing Manager
University of Rochester
Richard Moore
Rakesh Singh
Kyu Kwang Kim