Data support further evaluation of investigational antisense OT‑101 (trabedersen) as a TGFB2‑targeted approach in pancreatic ductal adenocarcinoma (PDAC).
AGOURA HILLS, Calif., July 17, 2025 (GLOBE NEWSWIRE) — 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, today highlighted the publication of new translational research evaluating TGFB2 expression and promoter methylation as potential prognostic markers in pancreatic ductal adenocarcinoma (PDAC). 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.†
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.†
About Oncotelic Therapeutics, Inc.
Originally founded as OXiGENE, Inc. in 1988 (New York) and reincorporated in Delaware (1992), the Company subsequently operated as Mateon Therapeutics, Inc. (2016) before adopting the name Oncotelic Therapeutics, Inc. in November 2020. Oncotelic leverages deep oncology drug‑development experience with a focus on high‑need and rare cancer indications. Programs within the Oncotelic/GMP Bio/Sapu ecosystem include: OT‑101 (TGFB2 antisense; DIPG and other indications), CA4P (melanoma), and OXi 4503 (acute myeloid leukemia), among others.† Additional assets include AL‑101, an intranasal apomorphine program in-licensed in Q4 2021 and being assessed for several neurologic and sexual‑health indications.† For a fuller description of our pipeline and risk factors, please see our Form 10‑K for the year ended December 31, 2024, filed April 15, 2025.
Additional Resources
- IJMS Publication: DOI 10.3390/ijms26136357 (open access).
- PDAOAI Public Discord: Community access to data sources used in manuscript development (non‑controlled, research discussion environment; not intended for making investment or treatment decisions).†
Oncotelic Cautionary Note on Forward‑Looking Statements
This press release contains forward‑looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements in this release other than statements of historical fact are forward‑looking and are based on current expectations, estimates, and projections about our business and future plans. In some cases, you can identify forward‑looking statements by terms such as “may,” “will,” “could,” “would,” “should,” “expect,” “plan,” “anticipate,” “intend,” “believe,” “estimate,” “project,” “forecast,” “potential,” “continue,” and similar expressions (including the negative of such terms).
Forward‑looking statements in this release include, without limitation: our plans, timelines, and priorities for the OT‑101 program in PDAC and other indications; potential biomarker‑driven development strategies; the advancement, scope, timing, and results of current or future preclinical and clinical studies; regulatory interactions and potential approvals; development or commercialization of any product candidates within the Oncotelic/GMP Bio/Sapu ecosystem; the utility of our PDAOAI platform; future financings, strategic transactions, and/or public offerings involving our joint ventures or affiliates; and other statements that are not historical facts. Actual results may differ materially from those indicated by such forward‑looking statements as a result of various important factors, including, but not limited to: the inherent uncertainties of drug discovery and development; our ability to enroll patients and complete studies on expected timelines; whether preclinical or early clinical findings (including biomarker associations) will be replicated in larger, controlled trials; regulatory developments in the United States and other jurisdictions; competitive developments; our ability to obtain or maintain intellectual‑property protection; our liquidity and access to capital; the performance of collaborators, suppliers, and manufacturers; and other risks described in our filings with the Securities and Exchange Commission (SEC), including the “Risk Factors” section of our most recent Form 10‑K and subsequent periodic reports.
Forward‑looking statements speak only as of the date of this press release, and we undertake no obligation to update or revise such statements, whether as a result of new information, future events, or otherwise, except as required by law.
Investor & Media Contact
Oncotelic Therapeutics, Inc.
Investor Relations
ir@oncotelic.com
† Source: Company materials and the IJMS publication, TGFB2 Expression and Methylation Predict Overall Survival in Pancreatic Ductal Adenocarcinoma Patients (Saif et al. 2025).
