On December 2, 2019 Oncotelic Inc. ("Oncotelic"), a wholly owned subsidiary of Mateon Therapeutics Inc. (OTCQB:MATN, "Mateon") dedicated to the development of innovative treatments for cancer, reported the publication of two peer-reviewed expert editorials authored by Fatih Uckun MD PhD, the Chief Medical Officer for Mateon, and Vuong Trieu, PhD, the Chief Executive Officer and President for Mateon, at Oncotelic in the oncology journals Cancer Clinics Journal and Annals of Hematology and Oncology Research (Press release, Mateon Therapeutics, DEC 2, 2019, View Source [SID1234551813]). Also published was a peer-reviewed research article in the prestigious oncology journal Cancers.

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The editorial titled "Monotherapy of High-Grade Gliomas with a TGF-beta2 Targeting RNA Therapeutic" provides an overview regarding the development status, mechanism of action, and clinical potential of Mateon’s first-in-class RNA therapeutic OT101 for the treatment of the most aggressive brain tumors in adults.

Citation Reference: Uckun FM, Trieu VN. Monotherapy of High-Grade Gliomas with a TGF-beta2 Targeting RNA Therapeutic. Cancer Clin J. 2019; 1(1): 1007 (View Source)

The editorial titled "Convection-enhanced delivery of an anti-TGFbeta RNA therapeutic as a new therapeutic concept for children with diffuse intrinsic pontine glioma ("DIPG")" explains a new concept for the treatment of pediatric DIPG patients with an RNA therapeutic targeting TGFbeta 2 and the scientific rationale and proof of concept data behind it.

Citation Reference: Uckun FM, Trieu VN. Convection-enhanced delivery of an anti-TGFbeta RNA therapeutic as a new therapeutic concept for children with diffuse intrinsic pontine glioma. Annals of Hematology and Oncology Research 2019; 1(1):1003 (View Source)

"These editorials emphasize our commitment to advance our investigational drug OT101 which shows high clinical impact potential to address unmet needs in neuro-oncology.", stated Dr. Trieu.

The research article "Recurrent or Refractory High-Grade Gliomas Treated by Convection-Enhanced Delivery of a TGFβ2-Targeting RNA Therapeutic: A Post-Hoc Analysis with Long-Term Follow-Up" was published in Cancers as part of the Special Issue Personalized Medicine: Recent Progress in Cancer Therapy and is available online:

Abstract: View Source

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PDF Version: View Source/pdf

Special Issue: View Source

Citation Reference: Uckun, F.M.; Qazi, S.; Hwang, L.; Trieu, V.N. Recurrent or Refractory High-Grade Gliomas Treated by Convection-Enhanced Delivery of a TGFβ2-Targeting RNA Therapeutic: A Post-Hoc Analysis with Long-Term Follow-Up. Cancers 2019, 11, 1892 (View Source/htm)

Dr. Uckun explained: "Our post-hoc analysis of the NCT00431561 Phase II clinical study outcome data demonstrates that the anti-TGFβ2 RNA therapeutic OT101 when intratumorally-administered via convection-enhanced delivery (CED) exhibits promising single-agent activity against recurrent or refractory (R/R) high-grade gliomas (HGG). Most importantly, OT101 induced durable complete response (CR), partial response (PR) and stable disease (SD) in more than one third of the efficacy population. In the objective responders, OT101 displayed a previously not reported pattern of single agent activity, which was characterized by (i) slow but robust tumor size reductions; (ii) very often, an early onset transient increase of tumor edema and/or pseudo-progression which preceded the tumor size reductions; and (iii) late-onset objective responses that were achieved at a median of 287 days. To our knowledge, this is the first demonstration that the intratumoral delivery of an RNA therapeutic via extended CED in the absence of other therapeutic agents or radiation results in >3 year median PFS and >3.5 year OS in greater than one third of the efficacy population in a R/R HGG trial. It will be important to evaluate OT101 in difficult-to-treat IDH-wildtype, MGMT-unmethylated R/R HGG patients who have failed a temozolomide-based first line adjuvant therapy, as they have a dismal prognosis and are in urgent need for new and effective salvage therapies. Intratumorally administered OT101 exhibited a promising safety profile, but some CED procedure and device-related potential complications were also identified. Therefore, risk mitigation strategies, especially preventive methods aimed at reducing the risk of infections, will be employed in future trials to further improve the potential patient benefit of intratumoral OT101 therapy"

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 not only for adult high-grade glioma patients but also for pediatric DIPG patients. 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.