The US Food and Drug Administration (FDA) has granted investigational new drug clearance for TeraImmune’s lead candidate, TI-168, allowing for a phase 1/2a trial that will examine the safety and efficacy of the therapy in patients with congenital hemophilia A with refractory inhibitors.

Yong Chan Kim, PhD, chief executive officer of TeraImmune, Inc., expressed his gratitude for the FDA’s decision. “We are thankful to the FDA for their engagement and are pleased with their decision to issue IND clearance for our Phase 1/2a trial to evaluate TI-168 in congenital HA patients with refractory inhibitors of TI-168,” he said in a press release.

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


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The multicenter, open-label, phase 1/2a trial is expected to enroll up to 18 patients with congenital hemophilia A with refractory inhibitors. The primary goals of the trial are to evaluate the safety and feasibility of TI-168, as well as determine the maximum tolerated dose. In addition, the trial aims to evaluate the efficacy of TI-168 and investigate the characteristics of engrafted TI-168.

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In addition to TI-168 therapy, all participants will receive mandatory prophylactic emicizumab (Hemlibra®) and standard-of-care treatment to manage episodic, breakthrough bleeds.

TI-168 is a next-generation FVIII-specific Treg therapy that combines the patented Treg culture method and an FVIII-specific T-cell receptor developed by TeraImmune. Preclinical studies were encouraging, showing that the therapeutic approach was able to control FVIII antidrug antibodies in a hemophilic animal model.

Reference

TeraImmune receives FDA IND clearance to initiate phase 1/2a clinical trial of TI-168 for treatment of hemophilia A with refractory inhibitors. News release. TeraImmune, Inc.; October 11, 2022.