Oncotelic Therapeutics, Inc. announced that its COVID-19 trial (“C001”) for OT-101 has successfully met its safety and efficacy endpoints.
OT-101 is a first-in-class anti-TGF-β ribonucleic acid (“RNA”) therapeutic that has exhibited single agent activity in relapsed/refractory cancer patients in multiple clinical trials. OT-101 has also demonstrated activity against the SARS-CoV-2 virus, the virus that causes COVID-19, and is currently being evaluated in the Company’s C001 clinical trial against hospitalized severe COVID-19 patients.
Both tumor cells and SARS-Cov-2 induce TGF-β as part of their immune evasion mechanism. Consequently, inhibiting TGF-β by OT-101 is expected to impact both cancer and COVID. By targeting the host protein, OT-101 is expected to work against multiple respiratory viruses, including related emerging variants, unlike traditional antiviral drugs and vaccines.
On October 18, Data lock and Study Data and Analysis Data Models (SDTMs & ADaMS Databases) were generated for a Phase 2 C001 Covid Study: “A Double-Blind, Randomized, Placebo Controlled, Multi-Center Study of OT-101 in Hospitalized COVID-19 Subjects”. The trial compares OT-101 + Standard of Care (“SOC”) versus Placebo + SOC (N= 32 pts at 2:1 randomization ratio). SOC includes dexamethasone, the only drug known to improve outcomes in severe cases of COVID-19. The top line data is:
1) Safety endpoints met. OT-101 as a TGF-β inhibitor was safe to administer to COVID-19 patients including severe/critical COVID-19 patients.
2) Efficacy signals were obtained. End of treatment (Day 7) mortality for the entire study population was 4.5% OT-101 versus 20% for placebo.
3) Incidence of >96% viral load knockdown on End of Treatment (Day 7) was 89% for OT-101 versus 67% for placebo.
4) Overall survival improved 3X for critical COVID-19 pts (4 days for placebo versus 14 days for OT-101, p < 0.0166).
“It is gratifying that the TGF-β concept that we put forward has now been validated,” said Dr. Vuong Trieu, CEO and Chairman of Oncotelic. “The data form the basis for further development of OT-101 as a viable treatment for severe respiratory viral infections, including flu and COVID-19. We thank the patients and investigators involved, especially Dr. Carbajal of Calle Mariscal Sucre, Chancay, Huaral, Lima, Peru, who drove the study to its conclusion.”
“The resulting data clearly suggest a favorable clinical response to OT-101 in the treatment of patients with COVID-19,” noted Dr. Anthony Maida, Chief Clinical Officer – Translational Medicine. “Additionally, we were able to demonstrate that there is no evidence of delivering OT-101 and any effect relating to cytokine release syndrome.”