MedPacto Applies to US FDA for IND of Bactosertib Monotherapy in Osteosarcoma

[Asia Economy Reporter Lee Gwan-joo] MedPacto announced on the 11th that it has submitted an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) for a clinical trial of Bactosertib monotherapy in patients with recurrent and refractory osteosarcoma.

This clinical trial is a Phase 1/2 study of Bactosertib monotherapy in osteosarcoma patients and will be conducted at institutions including Case Western Reserve University School of Medicine and UH Rainbow Babies and Children’s Hospital.

The trial will evaluate the safety, tolerability, pharmacokinetics, and anticancer efficacy of Bactosertib monotherapy in 48 adolescent and adult patients aged 14 years and older with recurrent, refractory, or progressive osteosarcoma.

In preclinical studies on osteosarcoma, Bactosertib inhibited the growth of osteosarcoma cancer cells and lung metastasis, improving survival rates in mice by 100%. It was also confirmed to completely suppress lung metastasis of osteosarcoma cancer cells. In fact, at Case Western Reserve University in the U.S., Bactosertib was administered as a monotherapy to pediatric osteosarcoma patients under FDA-approved Compassionate Use, resulting in the complete disappearance of cancer cells metastasized to the lungs and brain.

Osteosarcoma is a malignant tumor arising from bone or bone-related cartilage and other osseous tissues, accounting for 5.6% of sarcomas and classified as a rare cancer. It is one of the representative pediatric cancers with a high incidence in children and adolescents. Notably, 25-50% of osteosarcoma patients experience lung metastasis, which often progresses during chemotherapy and leads to death.

Currently, treatments for osteosarcoma include surgery, chemotherapy, and radiation therapy. Combined surgery and chemotherapy account for approximately 45.5%, while treatment with drugs alone accounts for only 15.9%. Chemotherapy is still conducted using conventional methods, and immune checkpoint inhibitors have shown no efficacy, leading to a steadily increasing demand for new treatment options.

A MedPacto official stated, “While immune checkpoint inhibitors are ineffective against osteosarcoma, efficacy has been confirmed with Bactosertib monotherapy, prompting us to proceed with clinical trials.” They added, “Depending on the interim analysis results, we plan to redesign the trial for regulatory approval purposes.” Furthermore, they mentioned, “We also plan to apply for support from U.S. nonprofit organizations that assist clinical trials.”

This content was produced with the assistance of AI translation services.