Breakthrough Oncology Drug Shows Promising Results

Panavance Therapeutics

BERWYN, PAPanavance Therapeutics Inc. recently announced that positive data from the Company’s tumor cell selective and broadly active small molecule with a unique mechanism of action, GP-2250 (misetionamide), was presented at the American Association for Cancer Research (AACR) Annual Meeting 2023, taking place April 14-19, 2023, at the Orange County Convention Center in Orlando, FL.

The poster titled, Mechanisms and rational combinations with GP-2250, a novel oxathiazine derivative, in ovarian cancer was presented by Mark S. Kim, Ph.D., Assistant Professor of Gynecologic Oncology and Reproductive Medicine, The University of Texas MD Anderson Cancer Center and Anil Sood, M.D., Vice Chair for Translational Research in the Departments of Gynecologic Oncology and Cancer Biology and co-director of the Center for RNA Interference and Non-Coding RNA at MD Anderson as part of the Novel Antitumor Agents 2 session. The poster outlined the mechanism of action and the biological effects of GP-2250 using in vitro and in vivo models.

“The data generated by GP-2250 to date has continued to demonstrate promising treatment potential for cancers. These findings are encouraging and provide valuable insight as we advance the development of GP-2250 for the treatment of ovarian cancer. Of particular note, these new data indicate that GP-2250 monotherapy exerts profound effects on tumor metabolism and, when taken in combination with PARP inhibitors or bevacizumab, showed enhanced anti-tumor efficacy compared with any agent alone. With this growing body of data, we look forward to further evaluating GP-2250’s potential in the treatment of ovarian cancer,” commented Greg Bosch, Chairman and CEO of Panavance Therapeutics.

Dr. Sood commented, “There remains a significant unmet need in treatment options for ovarian cancer. The findings from these preclinical studies evaluating GP-2250 in combination with the current standards of care are incredibly encouraging as we continue to explore GP-2250 as a potential treatment option. With these promising preclinical data in hand and the validation of these findings for continued clinical development, we look forward to further evaluating the potential of GP-2250 to provide a much needed solution for patients in need.”

For the study, researchers from MD Anderson conducted in vitro experiments including MTT assay, Annexin V/PI assay, colony formation assay, reverse-phase protein array (RPPA), and HRLC/IC analysis to determine the biological activity of GP-2250 and investigate the mechanism of action. In vivo experiments were carried out to determine the therapeutic efficacy of GP-2250 alone and in combination with standard-of-care drugs (e.g., paclitaxel, cisplatin topotecan, PARP inhibitors and the antiangiogenic drug, bevacizumab (Avastin®). Additionally, researchers conducted an in vivo pharmacodynamic experiment using the OVCAR8 mouse model, which demonstrated that a dose of 500 mg/kg GP-2250 was the most effective in downregulating AKT and mTOR expression.

Data Highlights:

  • GP-2250 montherapy preclinical results demonstrated significant reductions in ovarian cancer tumor weight and number of tumor nodules, which were comparable to the results of PARP inhibitors and bevacizumab.
  • In vivo therapy experiment demonstrated that the combination of GP-2250 and each of the PARP inhibitors tested (olaparib, niraparib, and rucaparib) as well as bevacizumab showed a significant reduction of tumor weights and tumor nodules compared to a control group or the monotherapy groups.
  • GP-2250’s mechanism of action inhibited hypoxia-inducible factor-1α, AKT, and mTOR activation and expression level as revealed in RPPA analyses.
  • Ultra-high resolution mass spectrometry (HRMS) analysis also revealed that hexokinase-2 activity and expression was significantly reduced by GP-2250 treatment.
  • GP-2250 reduced GAPDH and HK2 impacting glycolysis and ATP synthesis in ovarian cancer cells.

The presented poster is now available on the Publications page of the Resource section of the Company’s website (

Panavance is currently advancing the development of GP-2250 towards multiple registration directed clinical studies including a Phase 2/3 for the treatment of ovarian cancer in combination with leading anti-cancer agents. These registration studies are expected to start in 2024.

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