OT101, a first-in-class RNA therapeutic designed to abrogate the immunosuppressive actions of TGF-b2, is Oncotelic’s lead anti-brain tumor drug candidate. OT101 has been granted orphan designation by the
The Oncotelic CED platform appears to be the only reported platform that allows for the delivery of a brain tumor drug directly into the patients’ tumors via an extended continuous infusion for repeated treatment cycles for up to 6 months. Other CED systems in clinical development allow only short-term drug delivery for days to a few weeks.
Dr. Uckun explained: “Our enabling CED platform uniquely allows the personalized intratumoral delivery of immuno-oncology drugs designed to overcome the profound immunologic exhaustion and suppression in brain tumors. The insights and lessons learned from our clinical use of this CED platform in 90 high-grade glioma patients will not only inform our Phase III clinical study, but they may also provide the foundation for strategic alliances with other immune-oncology companies who are interested in using CED to explore the clinical potential of their lead therapeutic drug candidates for brain tumors.”
Dr. Uckun will also present a plenary talk at the 2nd
The safety and clinical potential of the lead anti-leukemia drug candidate OXi4503 was evaluated as part of a 2-drug combination regimen in a multi-institutional Phase IB clinical study at 4 academic centers in the USA. 29 relapsed/refractory acute myeloid leukemia (AML) and myeloid dysplastic syndrome (MDS) patients were evaluated and objective responses - including 4 complete responses - as well as extended survival were observed in some of the patients. These results will be presented by Dr. Uckun during the 3rd International Hematologists Summit in
High-grade gliomas are characterized by a T-cell exhaustion signature and pronounced T-cell hyporesponsiveness of their tumor microenvironment (TME). Transforming growth factor beta 2 (TGFb2) has been implicated as a key contributor to the immunosuppressive landscape of the TME in high-grade gliomas. OT-101, a TGFb2-specific first-in-class RNA therapeutic designed to abrogate the immunosuppressive actions of TGFb2. In a completed Phase 2 clinical study, OT-101 exhibited clinically meaningful single-agent activity and induces durable complete and partial responses in recurrent and refractory adult high-grade glioma patients, including adults with GBM.
OXi4503 (combretastatin A1-diphosphate or CA1P) is a dual-mechanism vascular disrupting agent. In preclinical and clinical studies, it has been observed to compromise the tumor vasculature, resulting in extensive tumor cell death. OXi4503 is being developed as a drug candidate for relapsed/refractory AML. In addition to Fast Track status, OXi4503 has been granted orphan drug designation for the treatment of AML in both the USA and Europe.
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This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements, other than statements of historical facts, included in this communication regarding strategy, future operations, future financial position, prospects, plans and objectives of management are forward-looking statements. Words such as “may”, “on-track”, “pivotal”, “expect”, “anticipate”, “hope”, “vision”, “optimism”, “design”, “exciting”, “promising”, “will”, “conviction”, "estimate", "intend", "believe" and similar expressions are intended to identify forward-looking statements. Forward-looking statements contained in this press release include, but are not limited to, statements about future plans, the progress, timing, clinical development, scope and success of future clinical trials, the reporting of clinical data for the company’s product candidates and the potential use of the company’s product candidates to treat various cancer indications. Each of these forward-looking statements involves risks and uncertainties and actual results may differ materially from these forward-looking statements. Many factors may cause differences between current expectations and actual results, including unexpected safety or efficacy data observed during preclinical or clinical studies, clinical trial site activation or enrollment rates that are lower than expected, changes in expected or existing competition, changes in the regulatory environment, failure of collaborators to support or advance collaborations or product candidates and unexpected litigation or other disputes. These risks are not exhaustive, the company faces known and unknown risks, including the risk factors described in the company’s annual report on Form 10-K filed with the
Source: Mateon Therapeutics