FDA Grants Orphan Drug Designation to GTS Biotech’s CTB-1 as a Treatment for Osteosarcoma

February 05, 2024

GTS Biotech LLC, a preclinical-stage biotechnology company developing innovative less toxic immunotherapies for cancer announced today that the U.S. Food and Drug Administration (“FDA”) has granted orphan drug designation to its lead asset CTB-1, for the treatment of osteosarcoma. 

“We want to applaud the FDA’s recognition of CTB-1’s potential in the treatment of rare cancer indications, such as osteosarcoma,” said Gus Romero, Chief Executive Officer, This designation is an important milestone for patients who could benefit from a novel, less toxic therapy.”

“We are incredibly optimistic about CTB-1 and its ability to provide clinical benefit in patients with osteosarcoma and we look forward to further studying CTB-1 in a future trial,” said Tim Romero, Chief Development Officer. 

“CTB-1 has successfully expressed significant anti-cancer activity on several difficult to treat cell lines as well as xenograft tumor mouse models,” added Sachin A Shah, Pharm D, FACC, FAHA, GTS Biotech’s Chief Scientific Officer. “These results stem from CTB-1’s miraculous mechanism of action that allows for direct targeting of tumors in vivo and potentially in osteosarcoma patients. Osteosarcoma is the most common primary pediatric bone malignancy, derived from primitive bone-forming mesenchymal cells. It occurs in primary and secondary forms, accounting for approximately 20% of all primary bone tumors.” 

About Orphan Drug Designation

The FDA’s Orphan Drug Act is a law that was passed by Congress in 1983 as a way to incentivize the development of therapies for the treatment of rare (orphan) diseases or conditions. A disease is classified as “rare” if it affects fewer than 200,000 people total in the U.S., or if the cost of developing a drug and making it available in the U.S. for such diseases will exceed any potential profits from its sale due to the small target population size. The FDA’s ODD program provides multiple incentives to make orphan drug development more financially possible for companies to pursue, such as up to seven years of market exclusivity for the approved orphan drug, up to 20 years of federal tax credit for expenses incurred in conducting qualified clinical research within the U.S. and waiver of Prescription Drug User Fee Act (PDUFA) fees for orphan drugs. 

About CTB-1 

Cinnamtannin B-1 (CTB-1) is a natural trimeric proanthocyanidin typically derived from plants such as Cinnamomum zeyalanicum and Laurus nobilis. CTB-1 has shown anti-proliferative effects against OS cells while having minimal effects on growth of normal osteoblast cells required for bone homeostasis. CTB-1 inhibits the proliferation, migration, and invasion of osteosarcoma by working on the miR-1281/PPIF pathway via upregulation of miR-1281 and downregulates PPIF expression leading to increased levels of apoptosis. 1 CTB-1 has also demonstrated capability for PD-L1 inhibition and enhanced PD-1 expression through p53 stabilization. Due to these mechanisms, OS cell growth and invasion can be inhibited

About GTS Biotech, LLC 

We are a forward-thinking biotech company dedicated to revolutionizing cancer and immunology treatment through the discovery and development of compounds derived from natural sources.We firmly believe that the answers to many of today's medical challenges lie in the world around us. Harnessing the potential of natural compounds, we strive to unlock new frontiers in oncology, immunology and other areas of research to offer hope to patients fighting against cancer. 

For more information, please visit www.gtsbiotech.com