Use of Acetalax for Treatment of Triple Negative Breast Cancer

Triple negative (progesterone receptor (PR)-, estrogen receptor (ER)-, human epidermal growth receptor 2 (HER2)-) breast cancer (TNBC) is an aggressive subtype that affects 15-20% of the 1.7 million cases of breast cancer occurring annually.  Currently, standard treatments of TNBC include cytotoxic chemotherapies, surgery, and radiation. However, TNBC readily becomes resistant to chemotherapy, and those with TNBC are more likely to have a recurrence or die within five years compared to those with other breast cancer types. Therefore, there is a need for safer and more effective TNBC treatments to improve patient outcomes.
Investigators from the National Cancer Institute (NCI) and collaborating institutions have identified the compound acetalax (oxyphenisatin acetate) as a promising potential therapy for TNBC. Strikingly, acetalax’s cytotoxic effect checked first against ~178 FDA-approved or clinical trial oncology drugs on the three TNBC against the NCI-60 panel of cancer cell lines – subsequently followed up using the 22 different TNBC cell lines checked against 15 oncology drugs – were significantly most cytotoxic.
Acetalax’s efficacy in vivo has been investigated using a TNBC patient-derived xenograft (PDX) mouse model. Untreated PDX mice exhibited a tumor volume doubling rate of approximately 7-8 days whereas acetalax-treated mice tumor volumes decreased to undetectable levels in approximately 13 days. 
NCI seeks research co-development and/or potential licensees for a potential novel treatment for triple-negative breast cancer (TNBC) with acetalax (oxyphenisatin acetate).

Potential Commercial Applications: Competitive Advantages:
  • A therapeutic for Triple Negative Breast Cancer
  • Potential therapeutic for recalcitrant or estrogen-dependent cancers
  • Potential applicability to breast (non-Triple Negative), ovarian, pancreatic, sarcoma, and uterine cancers
  • Repurposed, previously approved FDA drug for a novel use as a therapeutic for TNBC and other cancers
  • Repurposed, previously approved drugs can have an expedited approval process due in part to pre-existing safety data

Development Stage:
Pre-clinical (in vivo)


William Reinhold

Matthew Garnett

Vinodh Rajapakse

Yves Pommier ()  ➽ more inventions...

Augustin Luna ()  ➽ more inventions...

Intellectual Property:
Application No. 62/627,926
Application No. PCT/US2019/017239
Application No. 19707221.8
Application No. 16/967,472

Rajapakse VN, et al. CellMinerCDB for Integrative Cross-Database Genomics and Pharmacogenomics Analyses of Cancer Cell Lines.  PMID 30553813

Collaboration Opportunity:

Licensing and research collaboration

Licensing Contact:
John Hewes, Ph.D.
Phone: 240-276-5515

OTT Reference No: E-041-2018
Updated: Sep 25, 2020