A significant fraction of patients with solid tumor cancers in metastatic and advanced settings do not respond to immunotherapies due to a lack of T-cell-inflamed tumor microenvironment. This botanical-derived non-toxic biomolecule BG34-200 developed in Dr. Zhang’s laboratory can be intravenously injected to modulate macrophages and create a tumor microenvironment that is vital for the generation of antitumor T-cell responses (Figure 3). The project has received multiple translational grants to complete de-risking milestones. The team is launching a clinical trial targeting canine metastatic osteosarcoma (OS) to collect key and gap data in preparation for a First-In-Human clinical trial targeting pediatric and adolescent and young adult (AYA) OS.

Figure 3. IV Administration of BG34-200 Induces macrophage re-programming
and T-cell responses in multiple solid tumor cancers.

Very recently, BG34-200 was approved for Rare Pediatric Disease Designation by the FDA on September 22nd, 2020. The U.S. Food and Drug Administration (FDA) approved our application for rare pediatric disease designation of a glucan-type immunotherapeutic for “treatment of pediatric osteosarcoma” on September 22nd,.  The FDA grant designation for this immunotherapeutic for treatment of osteosarcoma as a drug for a “rare pediatric disease,” as defined in section 529(a)(3) of the Federal Food, Drug, and Cosmetic Act (FD&C Act) (21 U.S.C. 360ff(a)(3)). The approval enables our potential to apply for the Priority Review Voucher once the drug gets to marketing stage in the future. Most importantly, the approval has allowed us to secure this part of the drug development program with the FDA.

Most importantly, BG34-200 was recently approved for Orphan-Drug Designation by the FDA on Oct 21st, 2020 The U.S. Food and Drug Administration (FDA) granted our application for orphan-drug designation of a glucan-type immunotherapeutic for “treatment of osteosarcoma”. The approval is pursuant to section 526 of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360bb). Note that this designation is broader than the indication granted for rare pediatric disease designation. Orphan-drug designation has great benefit to new drug development program when the drug gets to the marketing stage. The majors are: 1) Tax credits of 50% off the clinical drug testing cost award upon approval; 2) Eligibility for market exclusivity for 7 years post approval; 3) Waiver of new drug application (NDA) and Biologics license application (BLA) application fee. Most importantly, the approval has allowed us to secure this part of the drug development program with the FDA.