Biomarker-based new drug developer MedPacto has applied to the U.S. Food and Drug Administration for the investigational new drug (IND) clinical trial of Vactosertib as a solo therapy for recurrent and intractable osteosarcoma patients.

This upcoming clinical trial, a phase 1/2 trial of Vactosertib as a single agent, examines its safety and tolerability on 48 patients aged 14 and older suffering from recurrent or progressive osteosarcoma at Case Western Reserve University School of Medicine and UH Rainbow Babies and Children's Hospital.

Osteosarcoma, a malignant tumor that occurs on the bone or the osteoid tissue, such as the cartilage around the bone, is rare cancer accounting for 5.6 percent of sarcoma cancer. It is also one of the typical pediatric tumors showing a high incidence rate among children and teenagers.

Treatment for osteosarcoma involves surgery, chemotherapy, and radiotherapy. Among them, surgery combined with chemotherapy accounts for 45.5 percent, while drug therapy takes up only 15.9 percent. There is a growing demand for a new treatment as chemotherapy proceeds in a time-old fashion, and immuno-oncology remains ineffective.

Against this backdrop, a preclinical trial of Vactosertib on osteosarcoma improved the mouse survival rate by 100 percent, significantly restraining tumor growth and pulmonary metastasis. It also suppressed osteosarcoma cancer cells’ pulmonary metastasis by 100 percent, according to MedPacto.

“We are pushing ahead with the trial as Vactosertib has proved its efficacy on osteosarcoma,” a MedPacto official said. “We will also apply for support from the U.S. nonprofit clinical trial foundations.”