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Oncotelic Therapeutics Completes Phase 1 Trial of OT-101/IL-2 Combination for Advanced Solid Tumors

• Oncotelic Therapeutics has successfully completed a Phase 1 trial evaluating OT-101, an antisense therapeutic targeting TGFβ2, in combination with IL-2 for advanced or metastatic solid tumors.

• The combination demonstrated a tolerable safety profile with no unexpected safety signals, positioning the company to advance into further clinical studies exploring synergies with checkpoint inhibitors.

• CEO Dr. Vuong Trieu presented findings at the SWCR 2025 Conference, highlighting TGFβ2's central role in immune suppression and OT-101's ongoing development in pancreatic cancer, gliomas, and combination immunotherapy regimens.

Oncotelic Therapeutics has successfully completed a Phase 1 clinical trial evaluating the combination of OT-101 and IL-2 (aldesleukin) in patients with advanced or metastatic solid tumors. The RNA therapeutics company announced that the combination demonstrated a tolerable safety profile at the planned dosing schedule, with no unexpected safety signals identified.
The trial results (ClinicalTrials.gov ID: NCT04862767) were highlighted at the 5th Symposium on World Cancer Research (SWCR) 2025, where Oncotelic's Chairman and CEO, Dr. Vuong Trieu, presented findings on the therapeutic potential of targeting Transforming Growth Factor Beta 2 (TGFβ2).

Mechanism of Action and Therapeutic Strategy

OT-101 is an antisense therapeutic specifically targeting TGFβ2, a critical driver of immunosuppression that fuels tumor progression by promoting an M2-like macrophage phenotype and blunting antitumor immunity. By inhibiting TGFβ2, OT-101 aims to enhance immune responses against cancer cells.
The combination with IL-2, a cytokine that stimulates T-cell proliferation and function, represents a strategic approach to potentially overcome immune evasion mechanisms in solid tumors. Based on the favorable safety data from this Phase 1 trial, Oncotelic plans to advance the OT-101/IL-2 combination into further clinical studies that will also explore synergies with checkpoint inhibitors (CKIs) such as PD-1 blockers.
"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. Trieu. "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."

Broader Development Program for OT-101

Dr. Trieu's presentation at SWCR 2025, titled "Transforming Growth Factor Beta 2 in Aging, Cancer, Lupus, and Immuno-Oncology: A Convergence of Disease Pathways and Therapeutic Potential," outlined the ongoing development of OT-101 across multiple indications:
  • In pancreatic ductal adenocarcinoma (PDAC), OT-101 is currently in a Phase 3 clinical trial (the STOP-PC study) combined with mFOLFIRINOX
  • The drug has shown encouraging activity in gliomas, where high intratumoral TGFβ2 expression correlates with poor prognosis
  • Combination regimens with checkpoint inhibitors and IL-2 are being developed to address multiple pathways of immune evasion

Future Clinical Development

With the newly completed Phase 1 trial, Oncotelic is positioned to begin further combination trials to determine the added efficacy of OT-101, IL-2, and checkpoint inhibitors in solid tumors such as lung cancer, melanoma, and colorectal cancer.
The company's next wave of clinical trials aims to knock down TGFβ2 with OT-101 paired with complementary therapies, including checkpoint blockade, IL-2, interferon-based regimens, or standard-of-care chemotherapy, depending on the tumor indication.

Company Background

Oncotelic Therapeutics, Inc. (OTCQB:OTLC), formerly known as Mateon Therapeutics, is focused on leveraging its expertise in oncology drug development to improve treatment outcomes and survival of cancer patients, with a special emphasis on rare pediatric cancers.
The company has rare pediatric designations for several indications, including Diffuse Intrinsic Pontine Glioma (DIPG) through OT-101, melanoma (through CA4P), and Acute Myeloid Leukemia (AML) through OXi 4503. In recent years, Oncotelic has expanded its portfolio through acquisitions, including PointR Data Inc. in 2019 to build an AI-driven biotechnology capability, and AL-101 in 2021 for the intranasal delivery of apomorphine for various indications.
The presentation from the SWCR 2025 conference, along with a proprietary chatbot powered by PDAOAI, can be accessed on Oncotelic's public Discord server, reflecting the company's efforts to integrate AI technology into its communications and research platforms.
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