FDA grants orphan drug status to TI-168 for hemophilia A inhibitors

Baudax Bio‘s investigational regulatory T-cell therapy, TI-168, has received orphan drug designation from the U.S. Food and Drug Administration (FDA) for the treatment of hemophilia A with inhibitors. Orphan drug status is granted to therapies addressing rare conditions affecting fewer than 200,000 people in the U.S. This designation offers various benefits, including tax credits, clinical protocol assistance, application fee waivers, and seven years of market exclusivity if the therapy gains approval.

Baudax Bio plans to launch a Phase 1/2a clinical trial for TI-168 in early 2024, focusing on individuals with hemophilia A and inhibitors. The FDA approved the trial, expected to enroll up to 18 patients, last year.

Expressing satisfaction with the FDA’s decision, Gerri Henwood, Baudax Bio’s president and CEO, emphasized the pressing need for innovative therapeutic options for hemophilia A patients.

Hemophilia A, resulting from mutations affecting the clotting protein factor VIII (FVIII), is commonly treated with replacement therapies. However, approximately one-third of patients develop inhibitors against FVIII, reducing treatment effectiveness. TI-168 aims to address this issue by training the immune system to view FVIII replacement therapies as non-threatening, thereby diminishing inhibitor production.

TI-168 utilizes regulatory T-cells (Tregs), immune cells suppressing responses from other cells to prevent the immune system from attacking healthy tissues. The therapy, initially developed by TeraImmune, and acquired by Baudax Bio, has shown promise in preclinical studies.

The upcoming Phase 1/2a trial will assess TI-168’s safety and efficacy in congenital hemophilia A patients with inhibitors resistant to treatment. Participants will receive TI-168 along with preventive Hemlibra (emicizumab) and standard on-demand bleeding event treatment. The trial aims to determine efficacy, dosage limits, and overall safety.

Henwood expressed confidence in initiating the Phase 1/2a trial with a modest budget, aiming to advance clinical investigations in early 2024.