Panbela Therapeutics, a clinical stage biopharmaceutical company at the forefront of developing disruptive therapeutics for patients facing urgent unmet medical needs, has unveiled groundbreaking preclinical and clinical data on CPP-1X, also known as α-Difluoromethylornithine (DFMO) or Eflornithine, in the context of recent onset type 1 diabetes (T1D). The announcement comes as a beacon of hope for patients and researchers alike, shedding light on potential treatments that address the root causes of the condition.
Recent studies by Sims et al. have underlined that, while therapies for T1D have advanced, the persistent morbidity, mortality, and economic burden continue to erode the quality of life for affected individuals. This underscores the critical need for safe and efficacious interventions targeting the underlying pathology.
Following this significant revelation, shares of Panbela Therapeutics experienced a notable surge in the market today.
At the time of this publication, Panbela Therapeutics Inc stock (PBLA) has witnessed a surge.
Panbela Therapeutics Inc
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The data released by Panbela Therapeutics, which has been published in the esteemed journal Cell Reports Medicine, delves into the mechanism of polyamines and their inhibition by CPP-1X in relation to β cell stress, a pivotal factor in the onset of T1D, as demonstrated in in vitro and ex vivo models. Results showcase that DFMO treatment may preserve β cell function, as indicated by C-peptide levels in T1D patients, through the regulation of urinary polyamines, specifically putrescine. The study stands as a testament to the ongoing collaboration between Panbela Therapeutics and the Indiana University School of Medicine.
A comprehensive link to the publication can be accessed [here](https://www.cell.com/cell-reports-medicine/fulltext/S2666-3791(23)00438-X).
This research is an integral component of a multi-site clinical trial spearheaded by the Indiana University (IU) School of Medicine, generously supported by funding from JDRF, the foremost global organization devoted to T1D research and advocacy. The preclinical data were generated through the efforts of Raghavendra Mirmira’s laboratory at the University of Chicago. It is noteworthy that Panbela Therapeutics is providing the drug at no cost to researchers and played no part 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, it was observed that CPP-1X was well tolerated, and a dose-dependent inhibition of ornithine decarboxylase (ODC) was noted. An exploratory secondary analysis revealed that, at the two highest dose levels, treatment with CPP-1X stabilized C-peptide areas under the curve compared to placebo. Notably, when evaluating immune cell populations, no significant differences were observed between the placebo and CPP-1X groups.
These findings strongly suggest that CPP-1X represents a secure, orally-administered treatment option that holds the potential to enhance β cell function and/or prolong survival in recent onset T1D cases.
Jennifer K. Simpson, PhD, MSN, CRNP, President & Chief Executive Officer of Panbela, commented, “Through the investigation of β cell-specific deletion of ornithine decarboxylase (ODC) in preclinical models, our collaborators have provided compelling evidence for the protection against toxin-induced diabetes, suggesting a crucial role for polyamine dysregulation in T1D. This observation has been extended to the clinical setting, where results from the multi-site randomized, placebo-controlled Phase I trial in patients with recent onset T1D have indicated that inhibition of ODC by CPP-1X may enhance β cell function.”
Dr. Simpson added, “Overall, we are buoyed by these results, which indicate that CPP-1X may play a significant role in the clinical management of recent onset type 1 diabetes. These studies serve as the foundation for the ongoing IU and JDRF Phase II trial in recent onset T1D, aligning with the objective of developing effective, innovative therapies for patients with unmet medical needs.”
Emily K. Sims, MD, the first author and an associate professor of pediatrics at IU School of Medicine, as well as a pediatric endocrinologist at Riley Children’s Health, expressed optimism, stating, “With our promising early findings, we hold hope that DFMO, possibly as part of a combination therapy, could offer potential benefits not only to individuals with recent-onset type 1 diabetes but could ultimately also be tested for potential benefit to delay disease onset in those who are at risk of developing the condition.”
In conclusion, the data unveiled by Panbela Therapeutics stands as a beacon of hope, offering promising insights into the potential treatment of recent onset type 1 diabetes, and marking a significant milestone in the pursuit of innovative therapies for patients with urgent unmet medical needs.
