On March 25, 2025 Oncotelic Therapeutics, Inc (OTCQB:OTLC) ("Oncotelic", the "Company" or "We" or "Our"), "), a leader in RNA-based therapeutics, reported the successful completion of a Phase 1 clinical trial evaluating OT-101, in combination with IL-2 for advanced or metastatic solid tumors (Press release, Oncotelic, MAR 25, 2025, View Source [SID1234651421]). These results set the stage for new studies that combine OT-101,an antisense therapeutic targeting Transforming Growth Factor Beta 2 (TGFβ2), with checkpoint inhibitors ("CKIs") and recombinant IL-2 (aldesleukin) ("IL-2").

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The announcement coincides with a presentation delivered by Vuong Trieu, Ph.D., Chairman & CEO of Oncotelic, at the 5th Symposium on World Cancer Research (SWCR) 2025. In his talk, titled "Transforming Growth Factor Beta 2 in Aging, Cancer, Lupus, and Immuno-Oncology: A Convergence of Disease Pathways and Therapeutic Potential," Dr. Trieu described the central role of TGFβ2 in immune suppression across multiple diseases, and outlined the ongoing development of OT-101 in pancreatic cancer (Phase 3 STOP-PC study), gliomas, and combination regimens with immunotherapies.

Phase 1 Trial of OT-101 (TASO) and IL-2 Successfully Completed

The Phase 1 trial (ClinicalTrials.gov ID: NCT04862767) investigated the safety and tolerability of OT-101 in combination with recombinant IL-2 in patients with advanced or metastatic solid tumors.
The combination showed a tolerable safety profile at the planned dosing schedule, with no unexpected safety signals identified.
Based on the favorable safety data, Oncotelic plans to advance OT-101 plus IL-2 into further clinical studies, exploring synergies with CKIs such as PD-1 blockers.
Key Highlights From the Kyoto 2025 Presentation

TGFβ2’s Central Role in Cancer and Beyond:
Dr. Trieu presented evidence that TGFβ2 is a critical driver of immunosuppression, fueling tumor progression by promoting an M2-like macrophage phenotype and blunting antitumor immunity.
OT-101’s Clinical Progress and Versatility:
In pancreatic ductal adenocarcinoma (PDAC), OT-101 is currently in a Phase 3 clinical trial (the STOP-PC study) combined with mFOLFIRINOX.
OT-101 has shown encouraging activity in gliomas, where high intratumoral TGFβ2 expression correlates with poor prognosis.
Combination regimens with CKIs and IL-2 address multiple pathways of immune evasion, potentially amplifying therapeutic benefits.
Next Studies Targeting TGF β2 and Beyond:
With the newly completed OT-101 and IL-2 Phase 1 trial, Oncotelic is poised to begin further combination trials to determine the added efficacy of OT-101, IL-2, and CKIs in solid tumors such as lung cancer, melanoma, and colorectal cancer.
Next wave of clinical trials aiming to knock down TGF β 2 (e.g., with OT-101) paired with intervention with a complementary therapy, checkpoint blockade, IL-2, interferon-based regimens, or standard-of-care chemo, depending on the tumor indication
"We are thrilled to announce the completion of our Phase 1 study evaluating OT-101 with IL-2, a key milestone that sets the stage for next-generation immunotherapy combinations," said Dr. Vuong Trieu, Chairman & CEO of Oncotelic. Our findings reinforce that OT-101’s specific inhibition of TGFβ2 can significantly enhance the immune response, and we are eager to test these synergies with checkpoint inhibitors and IL-2 to maximize therapeutic potential for patients with hard-to-treat cancers."

The presentation, along with a chatbot powered by PDAOAI, can be accessed on our new PDAOAI public Discord: View Source

Our PDAOAI Discord server allows members of the public to view and download the presentation. In addition, by using "/query", they can utilize our proprietary chatbot technology to ask questions related to the presentation. We highly recommend joining Discord. If you need any help, members of the PDAOAI team can assist. In addition, you can use "/help". We intend to use this server for our PDAOAI platform. For those who are interested in joining our investor Discord, can do so here: View Source

"We are excited to bring PDAOAI to the public and are excited for the user feedback. This presentation is a beginning to bringing our AI technology to the masses and not just our internal team," said Scott Myers, Product Manager.