FDA Clears Trial of TI-168 Therapy to Eliminate Hemophilia A Inhibitors

TeraImmune planning launch of Phase 1/2a clinical study of Treg therapy

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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The U.S. Food and Drug Administration (FDA) has cleared the biotechnology company TeraImmune to launch a Phase 1/2a clinical trial testing TI-168, its regulatory T-cell therapy designed to eliminate inhibitors in people with hemophilia A.

The upcoming trial is expected to enroll up to 18 people with congenital (genetic) hemophilia A with resistant-to-treatment (refractory) inhibitors. All participants will receive treatment with TI-168.

The patients also will receive prophylactic or preventive treatment with Hemlibra (emicizumab), an approved hemophilia A therapy, and standard of care treatment for any bleeds that occur.

The study’s main goal will be to evaluate the safety profile of TI-168 and determine the highest dose that can be given without intolerable side effects. Efficacy measures also will be assessed.

“We are thankful to the FDA for their engagement and are pleased with their decision to issue IND [investigational new drug] clearance for our Phase 1/2a trial to evaluate TI-168 in congenital [hemophilia A] patients with refractory inhibitors of TI-168,” Yong Chan Kim, PhD, CEO of TeraImmune, said in a press release.

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In congenital hemophilia A, genetic mutations interfere with the production or function of a blood clotting protein called factor VIII (FVIII). Standard treatment generally consists of replacement therapies, which basically involve administering a version of this clotting protein to patients.

Although replacement therapies can effectively reduce bleeding, in about one in three hemophilia A patients, the body’s immune system registers the delivered FVIII as a threat and makes antibodies against it. These antibodies are called inhibitors, as they can substantially lower the effectiveness of treatment.

“Approximately 30% of [hemophilia A] patients develop anti-FVIII antibodies against FVIII treatment and there is no cure for this complication,” Kim said.

TI-168 is designed to eliminate inhibitors using regulatory T-cells, or Tregs, a type of immune cell that normally helps “put the brakes” on immune attacks.

Within the body, Tregs help prevent the immune system from accidentally attacking healthy tissue. The basic idea behind TI-168 is to train the immune system not to register FVIII replacement therapies as a threat, thereby reducing inhibitor production.

According to TeraImmune, the investigational treatment, which utilizes Tregs derived from the person being treated, has shown promise in controlling inhibitor production in an animal model of hemophilia.

“Given the strength of our existing data, paired with FDA’s clearance to proceed with the trial of TI-168, we believe that we will be able to work expeditiously towards developing a promising treatment for [hemophilia A] patients with refractory inhibitors,” Kim said.