Research Program

To date the following research endpoints have been completed.

1. Pharmacological characterization of OST-01.
OST-01 has been evaluated in models of AML given the broad availability of the animal models and that of primary human sample that allow for
creation of patient xenograft models (PDXs), that are highly relevant to the biology and treatment response of the counterpart human disease. Meanwhile, additional studies have been pursued in models of other types of cancers.

The anticancer mechanism of action and evidence of activity and safety have been pursued through the following completed studies:

  • In vitro cell proliferation, differentiation, and apoptosis studies:
  • RNA-sequencing experiments have been performed; gene enrichment sequencing analyses have also been completed to identify key components of cellular pathways that are modulated by the OST-1 (or other derivatives) and that lead to cancer cell death.

2. Proteomics and metabolomics studies have been carried out using state of the art mass- spectrometry techniques to evaluate the ability of OST-01 and derivatives to modulate protein synthesis post-translational modification, activation, and metabolism in AML and other cancer cells.

3. Efficacy Studies. Dose-searching and efficacy studies have been successfully conducted in AML murine and patient- derived xenograft (PDXs) models and breast cancer.

4. Preliminary Toxicology. In addition to cage side observations and weights, major organs and tissues from mice treated with OST-01 or derivatives
have been surveyed for toxicity using a range of doses in mice. A licensed veterinary pathologist assists in the evaluation of the results.

To date the following research endpoints are underway:

1. Mass-spectrometry studies have been initiated to identify the active principle(s) contained OST-01 and those of other OST derivatives.

2. Validation studies utilizing individual basic components of OST-01 OST- derivatives.