Orca Bio Nabs FDA Approval of TREGZI, its Cell Therapy for Allogeneic Transplant in Adults with Blood Disorders

Key Points

• Orca Bio has received FDA approval for TREGZI, also known clinically as Orca-T, for use in matched-donor allogeneic (donor derived) stem cell transplantation in adults with certain blood cancers.
• The approval was based on the Phase 3 Precision-T study, where patients receiving TREGZI had higher chronic graft-versus-host disease-free survival at one year than those receiving conventional allogeneic transplant.
• This is Orca Bio’s first approved product.

Orca Bio has received FDA approval for TREGZI, known as Orca-T, for use in matched-donor allogeneic (donor derived) hematopoietic stem cell transplantation with a myeloablative preparative regimen in adults with hematological malignancies. The therapy is approved for hematopoietic and immunologic reconstitution and to improve chronic graft-versus-host disease-free survival.

TREGZI is a personalized treatment manufactured for each patient using living cells from a matched donor; the therapy itself combines:

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• Hematopoietic stem and progenitor cells to help rebuild the immune system
• “Highly purified” regulatory T cells to suppress graft-versus-host disease
• Conventional T cells to support immune reconstitution and graft-versus-leukemia activity

This appears to be Orca’s first FDA-approved therapy, with several more in the pipeline.

“We founded Orca Bio on the audacious goal to engineer living cells into curative medicines, rooted in the belief that single-cell precision could fundamentally rewrite patient outcomes,” said Nate Fernhoff, Ph.D., co-founder and chief executive officer of Orca Bio. “The FDA approval of TREGZI is a significant milestone that stands on the shoulders of decades of pioneering science. As we enter this next chapter, our focus turns to the immense responsibility of delivering TREGZI reliably, precisely and safely to the patients and families counting on us.”

Phase 3 study results

The approval was based on the randomized, multi-center Phase 3 Precision-T study, which enrolled 187 patients in the U.S. with a median age of 43.6 years. The study included adults with acute myeloid leukemia, acute lymphoblastic leukemia, myelodysplastic syndrome, and mixed-phenotype acute leukemia.

TREGZI plus single-agent tacrolimus (an immunosuppressant) was compared with conventional allogeneic hematopoietic stem cell transplant plus tacrolimus and methotrexate (an immunosuppressant/chemotherapy).

At 12 months, the study reported:

• Chronic GVHD-free survival of 78% with TREGZI versus 38% with conventional transplant
• Chronic GVHD rates of 13% with TREGZI versus 44% with conventional transplant
• Overall survival of 94% with TREGZI versus 83% with conventional transplant
• GVHD-free and relapse-free survival of 63% with TREGZI versus 31% with conventional transplant
• Non-relapse mortality of 3% with TREGZI versus 13% with conventional transplant
• Grade 3 or 4 acute GVHD at day +180 of 6% with TREGZI versus 10% with conventional transplant
• Estimated one-year incidence of grade 3 or higher infections of 44% with TREGZI versus 51% with conventional transplant

Orca Bio also reported that TREGZI improved GVHD-free survival along with lower toxicity, fewer serious infections, and lower non-relapse mortality.

“Developing this concept from early foundational research in our labs based upon the fundamental biology of regulatory T cells, to it now receiving the first FDA approval for a therapy that utilizes highly purified Tregs, is a defining moment for the transplant community,” said Robert Negrin, M.D., professor of medicine, blood and marrow transplantation at Stanford Medicine. “The peer-reviewed findings demonstrated this precision-engineered cell therapy delivered improved GVHD-free survival alongside less toxicity, including fewer serious infections and lower non-relapse mortality.”

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