On October 23, 2019 Oncotelic Inc. ("Oncotelic"), a wholly owned subsidiary of Mateon Therapeutics Inc. (OTCQB:MATN) dedicated to the development of innovative treatments for cancer, reported the publication of a peer-reviewed research article authored by Fatih Uckun MD PhD and his colleagues at Oncotelic in the Journal of Clinical Research in Pediatrics (View Source) (Press release, Oncotelic, OCT 23, 2019, View Source [SID1234542449]). The article titled "In silico Molecular Target Validation Demonstrates Transforming Growth Factor Beta 2 is Strongly Expressed in Pediatric Diffuse Intrinsic Pontine Glioma and Glioblastoma Multiforme" demonstrates the therapeutic potential of Mateon’s first-in-class RNA therapeutic OT101 for the treatment of diffuse intrinsic pontine glioma (DIPG) and pediatric glioblastoma multiforme (GBM).
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Citation Reference: Uckun FM, Trieu V, Hwang L, Qazi S. In silico Molecular Target Validation Demonstrates Transforming Growth Factor Beta 2 is Strongly Expressed in Pediatric Diffuse Intrinsic Pontine Glioma and Glioblastoma Multiforme. Clin Res Pediatr 2019;2(1):1-10.
"Our research has revealed that the TGF beta2 gene product, which is the molecular target for OT101/Trabedersen, may serve as a target for immunotherapy in pediatric high-grade gliomas, especially DIPG. These in silico target validation data extend the promising clinical data on the therapeutic activity of OT101 in adults and young adults and further demonstrate the potential of OT101 as a promising drug candidate in the treatment of pediatric DIPG, an orphan disease with a low survival rate and no established or effective standard of care," explained Fatih Uckun, MD, PhD, the Chief Medical Officer of Mateon. "The durable objective responses achieved in adult patients with recurrent/refractory high-grade gliomas after treatment with our lead anti-TGF beta2 compound OT-101 contribute to our optimism that new treatment strategies leveraging OT101 may favorably change the therapeutic landscape for difficult-to-treat brain tumors with a very poor prognosis, including pediatric GBM and DIPG," Dr. Uckun explained. Last month, US Food and Drug Administration (FDA) granted Rare Pediatric Disease Designation for OT101/Trabedersen for the treatment of diffuse intrinsic pontine glioma (DIPG) as a drug for a "rare pediatric disease," as defined in section 529(a)(3) of the Federal Food, Drug, and Cosmetic Act.
OT101, a first-in-class RNA therapeutic designed to abrogate the immunosuppressive actions of TGF-beta 2, is Oncotelic’s lead anti-brain tumor drug candidate. In a completed Phase 2 clinical study, OT-101 exhibited clinically meaningful single-agent activity and induced durable complete and partial responses in recurrent and refractory adult high-grade glioma patients, including adults with GBM. DIPG, an orphan disease with a low survival rate and no established or effective standard of care. Despite numerous clinical trials of chemotherapeutic agents, immuno-oncology drugs and specific targeted therapies, no significant progress has been made in the treatment of DIPG and the prognosis remains dismal, with a mean OS of 9–12 months from the time of diagnosis, a median survival time of approximately 10 months, and a two-year OS rate of less than 10 percent. Five-year survival is less than 3 percent, and many long-term survivors have evidence of moderate or severe cognitive impairment, likely as a consequence of radiation therapy. Chemotherapy does not have an established role in the management of patients with DIPG. Furthermore, there is no standard treatment for progressive DIPG after the failure of radiation therapy and no salvage regimen has been shown to extend survival. Therefore, there is an urgent need for therapeutic innovations for treatment of DIPG, as reflected by multiple treatment modalities being evaluated in early neuro-oncology clinical trials. Further development of OT-101 may offer renewed hope for salvage therapy of pediatric DIPG patients who have this rare and fatal disease.
"As we move Trabedersen through its clinical development for Pediatric Diffuse Intrinsic Pontine Glioma and Glioblastoma Multiforme, we are moving CA4P forward along the same path as potential treatment for Pediatric Unresectable or Metastatic Melanoma- a rare and difficult to treat pediatric skin cancer, " said Dr. Vuong Trieu, Chief Executive Officer of Mateon "From the corporate side we are moving forward with activities to uplist Mateon. Looking forward to updating our shareholders on the coming quarter."