Panbela Therapeutics, Inc. announced the publication of preclinical and clinical data from studies of CPP-1X (also known as a-Difluoromethylornithine (DFMO) or Eflornithine) in recent onset type 1 diabetes (T1D). According to Sims et al, although therapy of T1D has improved, the morbidity, mortality and cost continue to impact the quality of life for those affected highlighting the need for safe and effective therapies that address the underlying pathology. Data published in the journal Cell Reports Medicine investigated the mechanism of polyamines and polyamine inhibition by CPP-1X on ß cell stress that plays a role in the onset of type 1 diabetes in vitro and ex vivo models.

Results showed that DFMO treatment may preserve ß cell function, reflected by C-peptide levels in patients with T1D through the modulation of urinary polyamines, in particular putrescine. 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 (IU) School of Medicine, supported by funding from JDRF, the leading global T1D 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. From the Phase 1 dose range finding study of CPP-1X 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 ß cell function and/or survival in recent onset T1D.