New Study Highlights Potential of OT-101 Targeted TGFB2 Therapy in Pancreatic Cancer

A recently published study offers new evidence supporting further evaluation of the experimental antisense oligonucleotide OT-101 (trabedersen) as a TGFB2-targeted therapy for pancreatic ductal adenocarcinoma (PDAC). The findings suggest that TGFB2 gene expression and promoter methylation may serve as significant prognostic markers for patients with this type of cancer.

Study Collaboration and Publication

This research was a collaboration between GMP Biotechnology Limited’s wholly owned subsidiary Sapu Biosciences, LLC, and Oncotelic Therapeutics, Inc. (OTCQB: OTLC), a clinical-stage biopharmaceutical company focused on developing RNA-targeted and small molecule therapies for cancer and rare diseases. The study was published in the International Journal of Molecular Sciences (IJMS) under the title "TGFB2 Expression and Methylation Predict Overall Survival in Pancreatic Ductal Adenocarcinoma Patients" (DOI 10.3390/ijms26136357). Researchers from Sapu Biosciences were key contributors to this work.

Key Findings

Clinical data from the OT-101 P001 pancreatic ductal adenocarcinoma study demonstrated that TGFB2-targeted therapy deserves further investigation, particularly in younger patients. In a specific subgroup characterized by low interleukin-6 (IL-6) levels, the median overall survival was reported as 12.7 months.

Expert Commentary

Professor Wasif Saif, MD, a co-author of the study and Director of the Eisenberg Center for Translational Therapeutics at the Karmanos Cancer Institute, emphasized the practical implications for clinicians:

"These results provide a practical framework for patient stratification and designing age-specific trials, which are urgently needed to improve outcomes for this deadly disease. OT-101 and TGFB2-targeted therapy warrant validation in randomized clinical studies."

Further analysis revealed a significant association between high TGFB2 expression and reduced overall survival (OS) in patients under 65 years old. Specifically, patients with high TGFB2 expression had a median OS of 17.9 months compared to 66.9 months for those with low expression. This association was not observed in older patients. Furthermore, increased TGFB2 methylation correlated with improved survival in younger patients, with a median OS of 66.9 months for those with high methylation versus 17.9 months for those with low methylation.

The clinical data from the OT-101 trials support these findings, suggesting that younger patients receiving OT-101 treatment experienced improved survival compared to control groups. Dr. Saif concluded that TGFB2 is more than just another biomarker—it identifies a subgroup of younger pancreatic cancer patients with shorter survival, highlighting a critical clinical challenge.

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