OT-101

OT-101 is a first-in-class antisense oligodeoxynucleotide (ODN) therapeutic designed to target and inhibit Transforming Growth Factor Beta 2 (TGF-β2). By blocking the expression of TGF-β2 mRNA, OT-101 disrupts a key driver of immunosuppression in the tumor microenvironment, lifting TGF-β-mediated immune inhibition and allowing enhanced innate and therapeutic anti-tumor immune responses. OT-101 is under development primarily for the treatment of various solid tumors that overexpress TGF-β2, including pancreatic ductal adenocarcinoma, high-grade gliomas (including glioblastoma multiforme and diffuse intrinsic pontine glioma), malignant melanoma, colorectal carcinoma, and other TGF-β2 overexpressing malignancies. It has demonstrated single-agent activity in clinical trials for relapsed/refractory cancer patients, particularly in high-grade glioma and pancreatic cancer, and is also being investigated in combination with immune checkpoint inhibitors (such as pembrolizumab and atezolizumab) and interleukin-2 (IL-2) to further boost anti-tumor immune responses[1][3][5][7][9]. The drug is administered intravenously and has completed Phase 2 clinical studies for certain indications. OT-101 has received orphan drug designation from the FDA for gliomas and rare pediatric disease designation for diffuse intrinsic pontine glioma[1].

Other names

TrabedersenAP12009AP-12009AP 12009