On July 17, 2025 Oncotelic Therapeutics, Inc. (OTCQB: OTLC) ("Oncotelic" or the "Company"), a clinical stage biopharmaceutical company developing RNA targeted and small molecule therapeutics for cancer and rare diseases, reported the publication of new translational research evaluating TGFB2 expression and promoter methylation as potential prognostic markers in pancreatic ductal adenocarcinoma (PDAC) (Press release, Oncotelic, JUL 17, 2025, View Source [SID1234654427]). The article, "TGFB2 Expression and Methylation Predict Overall Survival in Pancreatic Ductal Adenocarcinoma Patients," appears in the International Journal of Molecular Sciences (IJMS). The work involved investigators affiliated with Sapu Biosciences, LLC ("Sapu"), a wholly owned subsidiary of GMP Biotechnology Limited ("GMP Bio"), of which Oncotelic holds a 45% ownership interest.

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Access the publication: DOI 10.3390/ijms26136357 (open access via IJMS).

Study Highlights

Clinical data from the OT 101 P001 PDAC study suggest that targeting TGFB2 merits additional evaluation in younger patients; in a treated subset characterized by low IL 6, median OS was 12.7 months.
Investigator Commentary

"These findings give clinicians a practical way to stratify patients and design more precise, age focused trials, an urgently needed step toward improving outcomes in this notoriously lethal disease. OT-101, TGFB2 targeted approach deserves testing in randomized clinical trials," said Professor Wasif Saif, MD, co author of the study and Director of Eisenberg Center for Translational Therapeutics and Co-director of gastro-enterology Oncology Program at Karmanos Cancer Institute. He further added. "Our analysis shows that high TGFB2 expression was significantly associated with reduced overall survival ("OS") in patients under 65 (TGFB2 high median vs. low median OS: 17.9 vs. 66.9 months) but not in older cohorts. Moreover, elevated TGFB2 methylation showed improved survival in younger patients (high methylation vs. low methylation median OS: 66.9 vs. 17.9 months). In addition, our clinical data from a Pancreatic Ductal Adenocarcinoma trial using OT-101, an antisense oligonucleotide targeting TGFB2, further supported these findings that young patients treated with OT-101 showed improved OS compared to untreated controls. TGFB2 is not just another biomarker; its expression clearly delineates a younger subset of pancreatic cancer patients who experience far poorer survival, and a patient population we are challenged more commonly over the last few years."

Additional Company Perspective

"Younger adults now represent the fastest-growing slice of PDAC incidence ( 4 % per year in the 15-34 bracket). Once diagnosed, their absolute outcomes remain grim-five-year survival for all PDAC is only ~12 %. We see OT-101 playing an important therapeutic role in combating early onset PDAC," said Wen Han Chang, PhD, the lead bioinformatic scientist and Sr. Manager of Nanomedicine.

"Our PDAOAI knowledge platform helped our team efficiently assemble and interrogate the multi omic and clinical datasets underlying this manuscript-an example of how we aim to accelerate therapeutic insight generation," added Scott Myers, Product Manager.

About OT 101 (trabedersen)

OT 101 is an investigational phosphorothioate antisense oligonucleotide designed to down regulate transforming growth factor beta 2 (TGFB2), a cytokine implicated in tumor immune evasion, fibrosis, and resistance mechanisms across multiple solid tumors. OT 101 has received Rare Pediatric Disease Designation for diffuse intrinsic pontine glioma (DIPG) via the Company’s 45% joint venture, GMP Bio. Additional development work is ongoing across multiple tumor settings.