TeraImmune, Inc., a biotechnology company focused on discovery and development of novel Treg-based cell therapies for autoimmune diseases, announced today that the U.S. Food and Drug Administration (FDA) has granted Investigational New Drug (IND) clearance to commence the Company’s Phase 1/2a clinical trial to evaluate its lead candidate, TI-168, in congenital Hemophilia A (HA) patients with refractory inhibitors. TI-168 is a next-generation, autologous FVIII TCR-Treg cell therapy candidate to eliminate FVIII inhibitors in HA patients.
The multi-center, open-label, Phase 1/2a study is designed to assess the safety and efficacy of TI-168 in up to eighteen congenital hemophilia A (HA) patients with refractory inhibitors. All subjects will continue to receive mandatory prophylactic emicizumab and standard of care treatment with their usual episodic agents to treat breakthrough bleeds, as needed. The primary objectives are to evaluate the safety and feasibility of TI-168 and determine the maximum tolerated dose. The secondary objectives are to evaluate the efficacy of TI-168 and to evaluate the characteristics of engrafted TI-168.
“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,” said Yong Chan Kim, PhD, Chief Executive Officer of TeraImmune. “Approximately 30% of HA 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.”
About Hemophilia A: Hemophilia A (HA) is a rare genetic bleeding disorder that is caused by a lack of clotting factor VIII (FVIII). FVIII replacement is a standard of care treatment of HA, but, the formation of anti-FVIII antibody (a.k.a., “FVIII inhibitor”, also known as FVIII anti-drug antibody (ADA)) is often problematic in the FVIII-replaced HA patients. FVIII Immune Tolerance Induction (ITI) is currently the only treatment option, which costs an average of $1 million annually per patient. Thirty percent (30%) of the patients with ITI still fail to control the FVIII ADA, rendering the treatment ineffective. Treg therapy has been highlighted as an ideal approach in autoimmune diseases and autoimmune disorders such as an anti-drug antibody formation. However, the technical hurdles to manufacture the FVIII-specific immune cell therapies have delayed the development of immune cell therapies for ADA in hemophilia A.
About TI-168: TeraImmune is dedicated to the development of Treg therapy specific to HA (pipeline candidate TI-168). TI-168 is a next-generation, FVIII specific Treg therapy designed to reliably and effectively address Hemophilia A patients with FVIII inhibitor. By combining the patented Treg culture method (TREGableTM) and TeraImmune designed FVIII-specific TCR, the team has successfully demonstrated the therapeutic concept of FVIII TCR-Treg therapy in controlling of FVIII ADA in a hemophilic animal model. The lead program TI-168 has shown highly encouraging pre-clinical data and the FDA has granted IND clearance to commence a Phase 1/2a clinical trial for the treatment of hemophilia A.