The U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to TI-168, a T-cell therapy developed by Baudax Bio to treat people with hemophilia A and an inhibitor. ODD, which is granted by the FDA’s Office of Orphan Products Development, is awarded to therapies designed to treat rare diseases affecting fewer than 200,000 people in the United States.

TI-168 utilizes human immune regulatory T cells (“Tregs”), along with other proprietary technologies, to suppress immune responses that would target administered factor VIII-based therapies. 

“We are very pleased with the FDA’s decision to grant orphan drug designation to TI-168, which we believe highlights the urgent need for innovation and new therapeutic options for hemophilia A patients,” said Gerri Henwood, President, and Chief Executive Officer of Baudax Bio. “We believe this is an important therapeutic area, with established preclinical proof of concept in TI-168 through successes observed in hemophilia A with inhibitors in animal models.”

According to a new press release, the phase 1/2a study will investigate the safety and efficacy of TI-168 in patients with congenital hemophilia A and an inhibitor. Participants will also be treated prophylactically with emicizumab - indicated for people with or without inhibitors - as well as standard-of-care on-demand treatment for any bleeding events that may arise.

“With an Investigational New Drug (IND) application already FDA-cleared, we believe we can activate the Phase 1/2a Clinical Trial of TI-168 for treatment of hemophilia A with inhibitors with a modest initial budget and advance this therapy to further clinical investigation in early 2024,”added Henwood.

Source: Hemophilia News Today, September 29, 2023