Panbela Therapeutics announces presentation on results for CPP-1X

Panbela Therapeutics announces a poster presentation highlighting the results for CPP-1X in recent onset type 1 diabetes at the Endocrine Society meeting, which was held June 15-18, 2023. The work reflects the Company’s ongoing collaboration with Indiana University School of Medicine. The research is part of a multi-site clinical trial led by Indiana University School of Medicine, supported by funding from JDRF, the leading global type 1 diabetes research and advocacy organization. The preclinical data were generated by Raghavendra Mirmira’s laboratory at the University of Chicago. Panbela Therapeutics is providing the drug at no cost to researchers and was not involved in the design and analysis of these studies. The poster investigates the mechanism of polyamines and polyamine inhibition by CPP-1X on beta cell stress that plays a role in the onset of type 1 diabetes in in vitro and ex vivo models. Human islet cells were either treated with vehicle or CPP-1X and RNA sequencing and proteomics were performed. Results showed that CPP-1X treatment of stressed human islets cells resulted in altered pathways related to endoplasmic reticulum-related protein processing, antigen presentation, and reactive oxygen species. In vivo studies in mice with beta cell-specific deletion of ODC showed improved glucose tolerance, increased beta cell mass, and reduced incidence of diabetes following streptozotocin treatment. In the Phase 1 dose range finding study of in patients with recent onset T1D, CPP-1X was well tolerated and a dose dependent inhibition of ODC was observed. An exploratory secondary analysis showed that at the two highest dose levels, treatment with CPP-1X stabilized C-peptide areas under the curve compared to placebo. When assessing immune cell populations, there were no differences between the placebo and CPP-1X patients. Results from these studies suggest that CPP-1X is a safe, oral treatment option that may improve beta cell function and/or survival in recent onset T1D.