Orca Bio, a commercial-stage biotechnology company committed to transforming the lives of patients through high-precision cell therapy, announced the publication of data from a retrospective analysis which suggests a three-year overall survival (OS) benefit with TREGZITM, in patients with hematologic malignancies. This retrospective analysis evaluated data from the Precision-T phase 1b multicenter study comparing TREGZI + tacrolimus (Tac) to a CIBMTR-NMDP registry cohort of patients receiving conventional allo-HSCT + PTCy based GVHD prophylaxis. The findings, featured in the journal Transplantation and Cellular Therapy, showed improved survival outcomes for patients treated with TREGZI compared to similar patients treated with PTCy in the myeloablative setting. The data published in Transplantation and Cellular Therapy reinforce the potential role of high-precision graft engineering platforms and therapeutic interventions like TREGZI, in improving long-term outcomes in patients with blood cancer.
“Allogeneic stem cell transplant has long been considered the only curative option for advanced hematologic malignancies, but the risks associated with them – including graft versus host disease (GVHD) – are potentially life threatening,” said Caspian Oliai, MD, medical director of the UCLA Bone Marrow Transplantation Stem Cell Processing Center. “While there are treatment options available to help prevent GVHD, data on the impact of them on overall survival have been limited. The findings from this retrospective analysis on the recently approved therapy, TREGZI, point to the value of cell therapy in potentially improving transplant outcomes for patients without sacrificing long-term survival.”
The TREGZI arm was associated with higher OS over a three-year follow-up period compared to PTCy arm (HR=0.41; log-rank p=0.003). Overall survival at one, two, and three years for patients receiving TREGZI (n=76) was 96% (95% CI: 88%-99%), 86% (76%-92%), and 83% (73%-90%), respectively.
For patients included in the PTCy cohort (n=360), OS at one, two, and three years was 81% (95% CI:77%-85%), 72% (67%-77%), and 66% (60%-71%), respectively.
Historically, a drug cocktail of Tacrolimus/methotrexate (tac/MTX) was the predominant standard of care to prevent GVHD for patients following alloHSCT. However, there has been increased adoption of different approaches to GVHD prevention such as, PTCy-based regimens and the recently approved high-precision cell therapy, TREGZI.
In clinical studies, both TREGZI + Tac and allo-HSCT + PTCy have demonstrated the reduced cumulative incidence of GVHD relative to Tac/MTX.
“We welcome the publication of these data in Transplantation and Cellular Therapy as it contributes to the body of evidence that supports the therapeutic value of graft engineering approaches in GVHD,” said Nate Fernhoff, co-founder and CEO, Orca Bio. “We are a company committed to redefining what’s possible for patients in allogeneic cell therapy, and these data reinforce the value of Orca Bio’s technology in the durable restoration of a healthy immune system and reducing treatment-related toxicities in patients with blood cancer.”
About the Phase 1b Study and Comparative Analysis
The data published in Transplantation and Cellular Therapy, were based on a retrospective analysis of long-term survival follow up from a Phase 1b multicenter trial evaluating TREGZI for the treatment of hematologic malignancies, data from which have been presented at multiple scientific congresses including the American Society of Hematology (ASH) and the Tandem meetings of ASTCTTM and CIBMTRTM. The three-year OS outcomes observed with TREGZI were compared to a historical cohort of patients receiving an allogeneic stem cell transplant with PTCy-based GVHD prophylaxis. The cohort included was from registry data collected by CIBMTR-NMDP.
The inclusion criteria for this comparative analysis were similar to those of the TREGZI Precision-T Phase 3 trial including: patients who were ≤65 years of age with confirmed diagnoses of either intermediate- or high-risk acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL) in complete remission or myelodysplastic syndrome (MDS), who were treated with a myeloablative conditioning (MAC) regimen, and an 8/8 HLA-matched donor.
IMPORTANT SAFETY INFORMATION AND INDICATION
WARNINGS AND PRECAUTIONS
Graft Failure: Graft failure has occurred after TREGZI administration. Screen TREGZI recipients for antidonor antibodies that may prevent engraftment. Monitor patients closely for laboratory evidence of hematopoietic recovery.
Graft-Versus-Host Disease: Acute and chronic Graft-Versus-Host disease (GVHD), including life-threatening and fatal cases, occurred following treatment with TREGZI. Acute GVHD manifests as maculopapular rash, gastrointestinal symptoms, and elevated bilirubin. Chronic GVHD may include skin rash, mouth sores, dry eyes, liver inflammation, and development of scar tissue in the skin and joints and damage to the lungs. Treat patients with a single agent calcineurin inhibitor as prophylaxis to decrease the risk of GVHD. Monitor for signs and symptoms of GVHD, and treat if GVHD develops.
Infusion Reactions: Infusion reactions (IRs) may occur during or following treatment with TREGZI. Serious hypersensitivity reactions including anaphylaxis may occur to DMSO, human serum albumin (HSA), Dextran or murine protein present in TREGZI. IRs may begin within minutes of the start of TREGZI infusion, although symptoms may continue to intensify and not peak for several hours after the completion of the infusion. Monitor patients for signs and symptoms of IRs during and after TREGZI administration. When a reaction occurs, pause the infusion and institute supportive care as needed. Premedicate patients with antipyretics and histamine antagonists prior to infusion to reduce the incidence and intensity of infusion reactions.
Secondary Malignancies and Malignancies of Donor Origin: Secondary malignancies and malignancies of donor origin may occur following treatment with TREGZI. Development of secondary malignancies, including posttransplantation lymphoproliferative disorder (PTLD) may occur many years after transplantation. PTLD manifests as a lymphoma-like disease favoring non-nodal sites. PTLD is usually fatal if not treated. Serial monitoring of blood for EBV DNA may be warranted in patients with persistent cytopenias. No patient treated with TREGZI has developed PTLD. Monitor for malignancies of donor origin and secondary malignancies. Contact Orca Bio at 1-877-411-6722 if any patient is diagnosed with a secondary malignancy or a malignancy of donor origin.
Transmission of Infectious Agents: Transmission of serious infectious or communicable disease or agents may occur with TREGZI treatment as it is derived from human donor blood and manufactured using animal-derived reagents. Risks of transmission of infectious agents may occur despite screening or testing of donors. Risks of transmission of serious infections include, but are not limited to, human immunodeficiency virus, human T cell lymphotropic virus (HTLV)-1 and -2, hepatitis B virus (HBV), hepatitis C virus (HCV), Treponema pallidum, Trypanosoma cruzi, West Nile virus (WNV), cytomegalovirus, transmissible spongiform encephalopathy agents and vaccinia. Monitor patients for signs and symptoms of infections, perform tests for infectious agents and treat as clinically indicated.
ADVERSE REACTIONS
The most common adverse reactions (incidence ≥ 20%) were mucositis, diarrhea, rash, viral infections, infections pathogen unspecified, abdominal pain, vomiting, nausea, bacterial infections, hemorrhage, aGVHD, edema, and fungal infections.
The most common Grade 3-4 laboratory abnormalities (≥ 20%) are lymphocyte count decreased, platelet count decreased, leukocyte count decreased, neutrophil count decreased and hemoglobin decreased.
INDICATIONS AND USAGE
TREGZI is indicated for use in matched donor hematopoietic stem cell transplantation with myeloablative preparative regimen, for hematopoietic and immunologic reconstitution and to improve chronic graft-versus-host-free survival, in the treatment of adults with hematological malignancies.
Please see accompanying full Prescribing Information.
About TREGZI™
TREGZI™ is the first-and-only FDA approved precision-cell therapy for allogeneic transplant in adults with hematologic malignancies to improve survival free of cGVHD. It is indicated for use in matched donor hematopoietic stem cell transplantation with myeloablative preparative regimen, for hematopoietic and immunologic reconstitution and to improve chronic graft-versus-host-disease-free survival, in the treatment of adults with hematological malignancies.
TREGZI is a personalized treatment manufactured for each individual patient using living cells from a matched donor. TREGZI uses hematopoietic stem and progenitor cells (HSPCs) to reconstitute the immune system, highly purified regulatory T cells (Tregs) to suppress GVHD and conventional T cells (Tcons) to accelerate immune reconstitution and produce graft-versus-leukemia (GVL) activity.
The FDA approval of TREGZI was based on the results of Precision-T, a randomized, open-label, multi-center study that evaluated the safety, efficacy and tolerability of TREGZI compared with conventional allogeneic hematopoietic stem cell transplant (alloHSCT).
About Orca Bio
Orca Bio is a commercial-stage biotechnology company developing high-precision cell therapies for the treatment of blood cancer and autoimmune diseases. The company’s manufacturing platform uses single-cell precision to create proprietary, uniquely defined products designed to replace a patient’s diseased blood and immune system with a healthy one. At Orca Bio, we are on a mission to redefine what’s possible for patients by transforming the field of curative allogeneic cell therapy. For more information, visit www.orcabio.com.
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