Oncotelic Therapeutics, Inc. is a publicly traded, clinical-stage biotechnology company developing RNA therapeutics and small-molecule drugs for immuno-oncology and virology. The company's primary focus is on advancing its lead candidate, OT-101, which targets the TGF-β pathway to treat solid tumors and severe viral diseases. Oncotelic also leverages a proprietary AI platform to support its research and development efforts.
OT-101 is a lead clinical-stage antisense RNA therapeutic that targets and inhibits the production of TGF-β2, a cytokine that suppresses the immune system within the tumor microenvironment. Initially developed for solid tumors like high-grade glioma, it is also being investigated as a host-directed therapy for severe viral infections, including COVID-19, by modulating a patient's immune response to the pathogen.
OT-101 aims to improve treatment outcomes and survival for patients with difficult-to-treat solid tumors by overcoming immune suppression. It also addresses the need for effective treatments for severe viral infections by mitigating dysregulated host immune responses.
For cancer patients, this addresses tumors that are resistant to or do not respond to existing immunotherapies. For potential partners, it provides a novel agent to use in combination therapies to enhance the efficacy of their own drugs, such as checkpoint inhibitors.