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

Phase 1/2a clinical trial of Treg therapy planned for early 2024

Andrea Lobo, PhD avatar

by Andrea Lobo, PhD |

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The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Baudax Bio’s investigational regulatory T-cell therapy TI-168 for the treatment of hemophilia A with inhibitors.

Orphan drug status is given to therapies intended to treat rare conditions, defined as those affecting less than 200,000 people in the U.S. The designation grants several benefits to support the therapy’s clinical development, including tax credits for clinical trials, clinical protocol assistance, waiver of application fees, and seven years of market exclusivity if the therapy is ultimately approved.

The company is also planning to initiate a Phase 1/2a clinical trial in early 2024 to investigate TI-168 in people with hemophilia A with inhibitors. The trial, which is expected to enroll up to 18 patients, was cleared last year by the FDA.

“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,” Gerri Henwood, president and CEO at Baudax Bio, said in a company press release.

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One-third of hemophilia A patients on replacement therapies develop inhibitors

Hemophilia A is caused by mutations that result in a reduced production or function of a blood clotting protein called factor VIII (FVIII). Standard treatment generally involves replacement therapies, which consist of supplying the missing clotting factor to the patient.

These therapies are generally effective at reducing bleeding events, the main symptom of the disease, but about one-third of hemophilia A patients develop antibodies against the supplied FVIII. Because they can significantly reduce the treatment’s efficacy, rendering it ineffective in some patients, these antibodies are also known as inhibitors.

TI-168 is designed to train the immune system to see FVIII replacement therapies as no threat, thereby reducing the production of inhibitors against the administered FVIII.

It does so by using regulatory T-cells (Tregs), a type of immune cell that helps suppress immune responses from other cells and prevent the immune system from mistakenly attacking healthy tissues. Tregs are derived from the patient using a patented Treg culture method, which produces Tregs with high affinity for FVIII.

The therapy was originally developed by TeraImmune, a company developing Treg-based therapies for autoimmune diseases, which was acquired by Baudax earlier this year.

“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,” Henwood said.

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Phase 1/2a trial to test TI-168 in patients with congenital hemophilia A

The upcoming Phase 1/2a trial will investigate TI-168 in patients with congenital (genetic) hemophilia A who have inhibitors that are resistant to treatment. All will receive the experimental treatment.

Participants will also be given preventive treatment with Hemlibra (emicizumab), an approved replacement therapy for people with or without inhibitors, as well as standard-of-care on-demand treatment for any bleeding events that might occur.

The trial’s main goal is to evaluate the safety and efficacy of TI-168. Researchers will measure the treatment’s efficacy and determine the highest dose that can be given without intolerable side effects.

“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,” Henwood said.