Panbela announces publication of preclinical, clinical data of CPP-1X

Panbela Therapeutics announces the publication of preclinical and clinical data from studies of CPP-1X 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 beta cell stress that plays a role in the onset of type 1 diabetes in in vitro and ex vivo models. Results showed that DFMO treatment may preserve beta cell function, reflected by C-peptide levels in patients with T1D through the modulation of urinary polyamines, in particular putrescine. 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 beta cell function and/or survival in recent onset T1D. “By investigating beta cell-specific deletion of ornithine decarboxylase in preclinical models, our collaborators were able to demonstrate the protection against toxin-induced diabetes which suggests a role for polyamine dysregulation in T1D.” said Jennifer K. Simpson, PhD, MSN, CRNP, President & Chief Executive Officer of Panbela. “This observation was extended to the clinical setting where results from the multi-site randomized, placebo-controlled Phase I trial in patients with recent onset T1D showed that inhibition of ODC by CPP-1X may improve beta cell function.”