Orca Bio to Present New Clinical Data on Its High-Precision Cell Therapies at the 67th American Society of Hematology Annual Meeting

Press release


Menlo Park, CA

December 6, 2025

Orca Bio Presents New Clinical Data on Orca-T® in Older Patients Using Reduced Intensity Conditioning Plus New Analyses from the Precision-T Phase 3 Study at the 67th ASH Annual Meeting 

Patients aged 60-75 treated with Orca-T and a reduced intensity conditioning regimen (RIC) experienced low incidence of acute and chronic GvHD with low rates of disease relapse   

Results also highlight the encouraging early use of RIC Orca-T in the outpatient setting for the first time  

An observational analysis comparing Phase 3 outcomes to PTCy found Orca-T delivered favorable overall survival and lower non-relapse mortality  

New Precision-T analyses suggest the benefit of Orca-T extends to older patients and those with higher-risk disease  

Patient reported outcomes demonstrate favorable health-related quality of life metrics, fewer ICU stays and lower likelihood of rehospitalizations with Orca-T 

MENLO PARK, CA, December 6, 2025 – Orca Bio, a late-stage biotechnology company committed to transforming the lives of patients through high-precision cell therapy, today announced new data presented on its lead investigational allogeneic T-cell immunotherapy, Orca-T, at the 67th American Society of Hematology (ASH) Annual Meeting. 

 

Orca-T with Reduced Intensity Conditioning 

 

The new results of a single-center, open-label Phase 1 investigator-sponsored trial evaluating Orca-T in patients aged 60-75 (median 68 years) with a reduced intensity conditioning regimen (RIC) for the treatment of acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), myelodysplastic syndrome (MDS) and myeloproliferative neoplasm (MPN) showed a low incidence of both acute and chronic graft versus host disease (aGvHD, cGvHD) while maintaining a low rate of disease relapse. 

 

“Many older patients with hematological malignancies are not eligible for myeloablative allogeneic stem cell transplant due to the significant toxicities associated with it. A conventional reduced intensity alloHSCT can be safer and more tolerable, but it may also reduce the curative potential,” said Everett Meyer, M.D., Ph.D., hematologist and associate professor of medicine in Blood and Marrow Transplantation and Cellular Therapy at Stanford Health Care. “These early results suggest Orca-T following reduced intensity conditioning may preserve a meaningful graft-versus-leukemia effect while achieving low rates of toxicities, including acute and chronic GvHD. While additional research is needed, these findings support Orca-T as a potentially feasible option for older adults with blood cancer.” 

 

Highlighted in an oral session, patients (n=46) with 8/8 matched donors were conditioned with fludarabine/melphalan/total body irradiation (TBI) (n=11) or fludarabine/thiotepa/TBI (n=12). Conditioning was further reduced where 23 patients were enrolled in a cohort eligible for outpatient treatment. Also included in the analyses was a cohort of patients (n=7) with 7/8 matched donors receiving fludarabine/thiotepa/TBI.  

 

All patients (n=53) had successful neutrophil engraftment at a median of 15 days (range 9-39). At one year, there was no aGvHD grade 3-4 observed, and the rate of aGvHD grade 2 was 12.3% (95% CI, 5-23%). The rate of moderate-to-severe cGvHD was 9.6% (95% CI, 3-21%). At one year, relapse-free survival (RFS) and graft versus host disease relapse-free survival (GRFS) were 82% (95% CI, 72-94%) and 72% (95% CI, 60-87%), respectively. The overall survival (OS) was 88% (95% CI, 79-98%) and non-relapse mortality (NRM) was 10% (95% CI, 4-20%). The outpatient-eligible cohort experienced NRM of 0%, RFS of 80% (95% CI, 65-100%) and OS of 95% (95% CI, 87-100%). 

 

“We are energized by these new data, which reinforce our belief that Orca-T has the potential to expand curative treatment options to many more people living with serious blood cancers, including those who may not be eligible for a myeloablative transplant today, and even patients treated in the outpatient setting,” said Nate Fernhoff, Ph.D., co-founder and chief executive officer at Orca Bio. “Our Serene-T Phase 2 study evaluating Orca-T with RIC recently opened for enrollment and is the next step towards understanding if Orca-T may provide a path to treatment for more patients in need of therapeutic options.” 

 

New Analyses from the Precision-T Phase 3 Study 

 

Retrospective Comparison of Orca-T versus PTCy-Based GvHD Prophylaxis 

 

An observational analysis compared a dataset derived from patients who received Orca-T in the Precision-T Phase 3 study (n=45) to a historical PTCy patient cohort (n=475) derived from the Center for International Blood and Marrow Transplant Research (CIBMTR).  

 

At one year, OS was 94% with Orca-T compared to 81% with PTCy. RFS was 86% and 70% for Orca-T and PTCy, respectively. There was 0% NRM with Orca-T and 9.7% with PTCy, which achieved statistical significance. There was 13.8% relapse in the Orca-T cohort and 21% in the PTCy cohort. The rate of cGvHD was 14.7% with Orca-T versus 8.2% with PTCy. Of note, the OS in patients over the age of 50 was 100% with Orca-T compared to 75% with PTCy. 

 

These outcomes were achieved using only single-agent tacrolimus for pharmacological GvHD prophylaxis for Orca-T recipients, in contrast to the triple-agent regimen used with PTCy, implicating the potential role of immune reconstitution in both disease control and mitigating posttransplant complications. 

 

Orca-T Improved GvHD-free Survival Across Patient Demographics 

 

In subset analyses from the Precision-T Phase 3 study comparing Orca-T to a conventional alloHSCT plus tacrolimus and methotrexate (Tac/MTX), Orca-T demonstrated improved clinical outcomes overall and across subgroups with varied demographic and clinical features. For all patients, the rate of survival free from cGvHD (cGFS) was 78% and 38% for Orca-T and Tac/MTX, respectively. For patients over the age of 50, the rate of cGFS was 74% and 35% for Orca-T and Tac/MTX, respectively. For all patients, the rate of GRFS was 63% and 31% for Orca-T and Tac/MTX respectively, and 59% and 23% for patients over the age of 50 with Orca-T and Tac/MTX, respectively. 

 

Notably, OS and NRM were similar for patients aged 51-65 as in the entire safety population. For patients over the age of 50 at one year, OS was 94% (77%, 98%) for Orca-T patients (n=31) versus 80% (61%, 91%) for Tac/MTX patients (n=32) (HR=0.48 [0.12, 1.89]). Rates of NRM were 6.5% (1.1%, 19%) with Orca-T vs 16% with Tac/MTX (5.7%, 31%) (HR=0.49 [0.11, 2.06]). Together, these data suggest that the results of Orca-T extend to older patients and those with high-risk disease.  

 

Health-Related Quality of Life: Patient Reported Outcomes 

 

An exploratory endpoint from the Precision-T Phase 3 study evaluating health-related quality of life (HRQoL) and hospitalization patterns showed that Orca-T delivered marked improvements over conventional alloHSCT. Patients receiving Orca-T experienced faster recovery to, and higher improvement above, baseline HRQoL, fewer ICU stays, lower likelihood of rehospitalization and higher rehospitalization-free survival, suggesting better early post-treatment recovery and a lower burden of GvHD symptoms.  

 

FACT-BMT total scores were consistently higher for Orca-T recipients across all time points, with Orca-T recipients exceeding baseline scores across physical well-being, functional well-being and transplant-specific subscales by day 100, while the alloHSCT arm did not surpass baseline until day 365. By day 365, Orca-T scores across these domains were higher by a magnitude of two or more compared to the control arm. 

 

Rehospitalizations due to adverse events occurred less frequently among Orca-T recipients (27.3% [24] vs. 45.7% [43]), with fewer total hospitalization days per patient (30.6 vs. 40.8). Rehospitalization-free survival at 18 months was also significantly improved with Orca-T, reaching 66.4% (95% CI: 54.0, 76.2) compared to 33.8% (95% CI: 18.5, 49.9) for conventional alloHSCT (p=0.0096; HR 0.53 [0.32, 0.86]). 

 

“The additional analyses from our Phase 3 study further highlight Orca-T’s potential superiority across critical measures, from lower rehospitalization rates to improved outcomes in older patients,” said Scott McClellan, M.D., chief medical officer at Orca Bio. “These results continue to strengthen our conviction that Orca-T has the potential to transform the transplant experience and meaningfully raise the standard of care for patients and providers.” 

 

The safety and efficacy of Orca-T have not been determined by any regulatory authority. Orca-T is currently being evaluated under Priority Review by the U.S. Food and Drug Administration (FDA) with a Prescription Drug User Fee Act (PDUFA) target action date of April 6, 2026. 

About Precision-T

Precision-T (NCT05316701) is a randomized, open-label multi-center study that evaluated the safety, efficacy and tolerability of Orca Bio’s lead investigational allogeneic T-cell immunotherapy, Orca-T, compared to conventional allogeneic hematopoietic stem cell transplant (alloHSCT). Orca Bio received guidance from the U.S. Food and Drug Administration on the design of Precision-T, which evaluated Orca-T in patients with acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), high-risk myelodysplastic syndrome (MDS) and mixed-phenotype acute leukemia (MPAL). There are 19 leading treatment centers participating in the trial, which enrolled 187 patients across the U.S. 

About Orca-T

Orca-T is an investigational allogeneic T-cell immunotherapy under evaluation for the treatment of multiple hematologic malignancies including acute leukemias and myelodysplastic syndromes. Orca-T is composed of highly purified regulatory T-cells, hematopoietic stem cells and conventional T-cells derived from either related or unrelated matched donors. Orca-T has received Regenerative Medicine Advanced Therapy (RMAT) and Orphan Drug Designation for the prevention of graft versus host disease or death in patients eligible for hematopoietic stem cell transplant from the U.S. Food and Drug Administration (FDA). The Biologics License Application (BLA) for Orca-T is currently under Priority Review with the FDA with a Prescription Drug User Fee Act (PDUFA) target action date of April 6, 2026.  

About Orca Bio

Orca Bio is a late-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 personalized cell therapy products intended 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. 

Trademarks or registered trademarks used in this press release are the property of their respective owners. 

Contact:

Corporate Communications

Kelsey Grossman

media@orcabio.com

Investor Relations

Joshua Murray

ir@orcabio.com


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Orca Bio’s TREGZI™ Receives U.S. FDA Approval as First and Only Precision-Engineered Cell Therapy for Allogeneic Transplant in Adults with Hematological Malignancies

Press release

Menlo Park, CA

June 30, 2026

Orca Bio’s TREGZI™ Receives U.S. FDA Approval as First and Only Precision-Engineered Cell Therapy for Allogeneic Transplant in Adults with Hematological Malignancies


The approval of TREGZI, clinically known as Orca-T®, marks a significant milestone for patients as a novel, personalized cell therapy for use in a stem cell transplant to improve survival free of chronic graft versus host disease (GVHD) in adults with hematological malignancies
Approval is based on the pivotal Precision-T study which demonstrated patients receiving TREGZI were twice as likely to be alive without chronic GVHD at one year compared with conventional allogeneic transplant
TREGZI is Orca Bio’s first approved therapy, validating the company’s high-precision platform and the potential of its pipeline

MENLO PARK, CA, June 30, 2026 – Orca Bio, a commercial-stage biotechnology company committed to transforming the lives of patients through high-precision cell therapy, today announced the U.S. Food and Drug Administration (FDA) has approved TREGZI™ (allogeneic regulatory T cell immunotherapy with HSPC and T cells-vldq), clinically known as Orca-T®, a precision-engineered cell therapy 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 (GVHD)-free survival (cGFS), in the treatment of adults with hematological malignancies. 

This press release features multimedia assets, available here.


"For transplant physicians, one of our greatest challenges has long been preserving the vital graft-versus-leukemia effect while minimizing the risk of GVHD and infection,” said Miguel-Angel Perales, M.D., medical oncologist and chief of the Adult Bone Marrow Transplant Service at Memorial Sloan Kettering Cancer Center. “The FDA approval of TREGZI signals a new era in transplant medicine. This precision-engineered cell therapy is built on the foundational principles established by our early CD34 cell selection work and can now be delivered at scale, equipping providers with a new option to reduce serious toxicities and improve treatment outcomes."


"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."


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.


“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."


The FDA approval of TREGZI is based on results from the randomized, multi-center Precision-T Phase 3 study of patients (n=187) with a median age of 43.6 years (range 19-65 years) with acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), myelodysplastic syndrome (MDS) and mixed-phenotype acute leukemia (MPAL). Results with TREGZI plus single-agent tacrolimus (TAC) compared with a conventional allogeneic hematopoietic stem cell transplant (alloHSCT) plus TAC/methotrexate (TAC/MTX) found the following at 12 months:


  • The primary endpoint of chronic cGFS was 78% with TREGZI compared to 38% with alloHSCT (HR 0.26; p<0.00001), an improvement driven by a reduction in chronic GVHD and fewer patient deaths.  
  • The rate of chronic GVHD was 13% and 44% with TREGZI and alloHSCT, respectively (HR 0.19; p<0.00002).
  • Overall survival (OS) was 94% with TREGZI and 83% with alloHSCT. 
  • GVHD-free and relapse-free survival (GRFS) was 63% and 31% with TREGZI and alloHSCT, respectively. 
  • Non-relapse mortality (NRM) was 3% for TREGZI compared with 13% for alloHSCT. 

Additional safety findings were consistent with previous studies. The cumulative incidence for Grade 3 or 4 acute GVHD at day +180 with TREGZI was 6% versus 10% with alloHSCT (HR 0.37; p=0.044). Grade ³3 infections were less common with TREGZI, with a one year estimated incidence of 44% for TREGZI and 51% for alloHSCT. 


"Historically, surviving a blood cancer has often meant navigating serious, long-term effects that can shape patients’ lives well beyond treatment," said Gwen Nichols, M.D., executive vice president and chief medical officer at Blood Cancer United. “As a researcher, physician and a patient advocate, it’s exciting that patients will have a new option that may change what life after transplant can look like, including the potential to support recovery and quality of life.”


Hematological malignancies such as AML, ALL and MDS, commonly referred to as blood cancers, are cancers that can originate in the bone marrow and disrupt normal cell production. Despite therapeutic advances, these diseases, particularly in adult and high-risk populations, remain associated with poor outcomes, high relapse rates and significant treatment-related toxicities. 


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 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-disease-free survival, in the treatment of adults with hematological malignancies.

 

Please see accompanying full Prescribing Information.


About Precision-T

Precision-T (NCT05316701) is 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). Orca Bio received guidance from the U.S. Food and Drug Administration on the design of Precision-T, which evaluated TREGZI in patients with acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), high-risk myelodysplastic syndrome (MDS) and mixed-phenotype acute leukemia (MPAL). There were 19 leading treatment centers participating in the trial, which enrolled 187 patients across the U.S. Results were published in Blood in December 2025.

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. Orca Bio is a privately held company that completed Series F financing led by Lightspeed Venture Partners earlier this year. 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. 

Trademarks or registered trademarks used in this press release are the property of their respective owners. 

Dr. Perales has financial interests related to Orca Bio. 

Contact:

Corporate Communications

Kelsey Grossman

media@orcabio.com

Investor Relations

Joshua Murray

ir@orcabio.com

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Orca Bio Announces the Precision-T Phase 3 Study is Open and Enrolling Patients at Transplant Centers Across the U.S.

– The pivotal Precision-T Study is evaluating the safety and efficacy of Orca Bio’s investigational Orca-T compared to standard of care allogeneic hematopoietic stem cell transplant –

– Precision-T is expected to enroll approximately 174 patients across more than 20 clinical trial sites –

MENLO PARK, CA, August 2, 2022 – Orca Bio, a late-stage biotechnology company developing high-precision cell therapies for the treatment of cancer, genetic blood disorders and autoimmune diseases, today announced Precision-T, its pivotal Phase 3 study, is open, enrolling and treating patients at clinical trial sites including Stanford Health Care, City of Hope, Winship Cancer Institute of Emory University, Sarah Cannon Research Institute, Ronald Reagan UCLA Medical Center and Oregon Health & Science University. Precision-T is expected to enroll approximately 174 patients at more than 20 transplant centers across the U.S.

Precision-T (NCT05316701) is a randomized, open-label multi-center study that is evaluating the safety and efficacy of Orca Bio’s lead investigational high-precision cell therapy, Orca-T, compared to standard of care allogeneic hematopoietic stem cell transplant (standard allo-HSCT). Orca Bio received guidance from the Food and Drug Administration on the design of Precision-T, which will evaluate Orca-T in patients with acute myeloid leukemia (AML), acute lymphocytic leukemia (ALL) and high-risk myelodysplastic syndromes (MDS).

“By precision engineering the donor graft, we aim to create a cell therapy that retains the benefits of transplant without serious complications like graft versus host disease and disease relapse,” said Robert Negrin, M.D., professor of medicine at the Stanford School of Medicine. “This has been demonstrated by the recent results of the Phase 1b/2 single-arm trials with Orca-T, and we are pleased to be evaluating this novel cell therapy in a randomized Phase 3 clinical trial.”

The primary endpoint of the Precision-T study is the rate of moderate-to-severe chronic GvHD-free survival. The secondary endpoints are graft-versus-host-disease and relapse-free survival (GRFS), moderate-to-severe chronic GvHD and relapse-free survival. The randomized Phase 3 trial is being conducted concurrently with Orca Bio’s ongoing single-arm Phase 1b trial of Orca-T.

“The Precision-T study is an important step forward for patients battling deadly blood cancers like AML and ALL, which are often aggressive and for which standard allo-HSCT treatment carries significant risks,” said Ivan Dimov, Ph.D., co-founder and chief executive officer of Orca Bio. “We are pleased to collaborate with these clinical trial centers and the broader blood cancer community to advance this study with the ultimate goal of delivering a safe and effective therapy to the patients who need it.”

Orca-T is a first-in-class high-precision cell therapy that combines purified cells from a matched donor. It is designed to replace a patient’s diseased blood and immune system with a healthy one while lowering the risk of developing GvHD and other life-threatening transplant-related side effects. Almost 200 patients have been treated to date with Orca-T in single-arm trials with extremely encouraging results, which were recently presented at the European Hematology Association (EHA) 2022 Congress, the 2022 Transplantation & Cellular Therapy ASTCT and CIBMTR Tandem Meetings and the American Society of Hematology (ASH) Annual Meeting.

More information about the Precision-T study can be found at www.precisiontstudy.com or www.clinicaltrials.gov. 

Orca Bio Presents Positive Data Reinforcing Clinical Profile and Manufacturing Reliability of Orca-T at 2022 Transplantation & Cellular Therapy ASTCT and CIBMTR Tandem Meetings

– Pooled data from 137 patients show Orca-T high-precision cell therapy improved overall survival and reduced chronic graft versus host disease compared to standard of care –

– Orca-T was well-tolerated and resulted in engraftment with regression of marrow fibrosis in patients with myelofibrosis –

– Manufacturing reliability analysis found vein-to-vein times for all grafts were under 72 hours regardless of donor collection site and transplant center locations –

– The Precision-T Phase 3 randomized registrational study of Orca-T versus standard of care is now open –

MENLO PARK, CA, Apr. 25, 2022 – Orca Bio, a clinical-stage biotechnology company developing purified, high-precision cell therapies for the treatment of cancer, genetic blood disorders and autoimmune diseases, today announced that positive new data were presented at the 2022 Transplantation & Cellular Therapy ASTCT and CIBMTR Tandem Meetings in Salt Lake City, Utah.

The new data on Orca Bio’s lead investigational high-precision cell therapy, Orca-T, include updated results from 137 patients with hematologic malignancies, which continued to show an increase in overall survival rates and a reduction of acute and chronic graft versus host disease (GvHD) compared to standard of care; positive outcomes in a subset of patients with myelofibrosis who received Orca-T; and new analyses demonstrating Orca Bio’s manufacturing platform is reliable, robust and scalable.

“These data from an expanded group of patients are very encouraging and demonstrate that Orca-T appears to improve survival while reducing GvHD. This could be a compelling option for patients battling serious hematological malignancies who currently face devastating transplant-related risks,” said Everett Meyer, M.D., Ph.D., primary investigator. “These results, combined with the reliable and centralized Orca-T manufacturing, could potentially transform treatment options for patients and physicians.’

Orca Bio presented pooled data from 137 patients in the single-center Phase 2 and multi-center Phase 1b trials of Orca-T with acute myeloid leukemia (AML), acute lymphocytic leukemia (ALL) and myelodysplastic syndromes (MDS), with at least 100 days of follow-up and a median of 341 days. For comparison purposes, an independent CIBMTR-based control arm was identified, consisting of similar patients with hematologic malignancies who received a standard of care allogeneic hematopoietic stem cell transplant (“alloHSCT”) over a similar timeframe. Results demonstrated that:

Additionally, Orca Bio presented findings from a manufacturing reliability analysis of 100 Orca-T products in the Phase 1b trial. All investigational cell therapies were manufactured at Orca Bio’s cGMP facility in Sacramento, CA, and transplant centers and donor sites were located across the U.S. All products were successfully delivered and infused to patients within 72 hours or less.

“We produce a unique batch of drug for each patient who receives an Orca-T product, and this drug is made up of fresh, living cells that need to be infused on an extremely rapid timeline,” said Nate Fernhoff, Ph.D., co-founder and chief scientific officer at Orca Bio. “Reliable and scalable manufacturing have been integral to the development of our therapies since day one. These results not only show that Orca-T continues to reduce GvHD and improve survival rates over time, but that we have maintained the ability to reliably manufacture, deliver and infuse all patients in a timely manner regardless of donor and patient location. This is an important indication of our ability to potentially deliver this therapy urgently and seamlessly to patients in need.”

In a separate poster, Orca Bio presented updated data on the treatment impact of Orca-T in 12 patients with myelofibrosis compared to nine patients who received a standard of care alloHSCT. Orca-T recipients had lower incidence of acute and chronic GvHD (33% with Orca-T versus 100% with alloHSCT) and the data suggest Orca-T was well-tolerated. Treatment with Orca-T resulted in engraftment with regression of marrow fibrosis, indicating potential efficacy for the treatment of myelofibrosis.

Based on the favorable results of the Phase 1b and 2 clinical trials, a randomized registrational Phase 3 trial evaluating Orca-T against standard of care alloHSCT, named Precision-T, is now open. More details will be announced in the coming weeks.

Links to the abstracts follow:

Orca Bio Receives Regenerative Medicine Advanced Therapy (RMAT) Designation for Orca-T

FDA grants RMAT and Orphan Drug Designations (ODD) to Orca-T for treating patients eligible for allogeneic hematopoietic stem cell transplant

MENLO PARK, CA, October 14, 2020 – Orca Bio, a clinical-stage biotechnology company, today announced that the US Food and Drug Administration (FDA) has granted RMAT designation to Orca-T (NCT04013685). Orca-T is an investigational allogeneic, high-precision cell therapy for patients with blood cancers who are eligible for a hematopoietic (blood) stem cell transplant. In addition, Orca-T has received ODD from the FDA for enhancing cell engraftment in patients who qualify for a hematopoietic stem cell transplant.

RMAT is a dedicated program designed to expedite the development of new regenerative therapies, including cell therapies that have the potential to treat life-threatening medical conditions. Cell therapies granted the RMAT designation also receive the benefits of fast track and breakthrough therapy designations. These advantages include early guidance from the FDA to steer manufacturing and clinical development, such as determination of intermediate endpoints to potentially support accelerated approval. ODD status is granted for therapies that address rare diseases affecting fewer than 200,000 people in the US. If a therapy with ODD receives the first FDA approval for the designated condition, it is eligible for 7-year market exclusivity.

For many patients with blood cancers, blood stem cell transplants from donors are the only hope for a cure. However, conventional transplants are often associated with cancer relapse and complications such as graft-versus-host disease (GvHD)—a condition in which immune cells from the donor attack the patient’s healthy cells, leading to organ failure and even death. Orca-T is a precisely controlled formulation of hematopoietic stem cells and T cells that aims to improve outcomes for these patients.

“The RMAT and ODD for Orca-T is uplifting news for patients with various blood cancers, including acute myeloid leukemia, acute lymphoid leukemia, myelodysplastic syndrome, and myelofibrosis,” said Ivan Dimov, PhD, Chief Executive Officer and Cofounder of Orca Bio. “We now have the opportunity to work closely with the FDA to open potential ways to support accelerated approval.” Clinical data from the ongoing Orca-T trial will be presented at the 62nd American Society of Hematology Annual Meeting later this year.

Orca Bio Emerges With Nearly $300 Million to Transform Allogeneic Cell Therapy

Company aims to safely and effectively regenerate a healthy blood and immune system for patients with hematological malignancies, genetic diseases and autoimmune disorders

High-precision cell therapies manufactured by Orca Bio have the potential to replace conventional bone marrow transplants and expand the eligible patient population

$192 million Series D financing strengthens the company with resources to propel lead product candidate to completion of clinical development

MENLO PARK, CA, June 17, 2020 – Orca Bio, a clinical-stage biotechnology company developing high-precision allogeneic cell therapies, today announced a Series D financing that brings its total capital raised since its 2016 launch to nearly $300 million. The company creates precisely controlled cell therapies by building each dose cell-by-cell from another person’s blood. Each therapy is constructed by formulating a proprietary mixture of cells that aims to cure the patient’s disease and eliminate dangerous side effects.

Orca Bio’s $192 million Series D financing was co-led by Lightspeed Venture Partners and an undisclosed investor. Other new and existing blue-chip investors also participated in the latest round, including 8VC, DCVC Bio, ND Capital, Mubadala Investment Company, Kaiser Foundation Hospitals, Kaiser Permanente Group Trust and IMRF.

The financing will support the continued advancement of Orca Bio’s cell therapy pipeline and its novel manufacturing platform, which sorts blood with single-cell precision and a high level of purity and speed to create optimal therapeutic mixtures of immune and stem cells. These proprietary mixtures have the potential to revolutionize allogeneic cell therapy for hematological and other cancers, as well as many other diseases and disorders.

A conventional bone marrow transplant relies on naturally occurring T cells. However, the uncontrolled cellular composition often results in life-threatening complications. The company’s most advanced program, TRGFT-201, is evaluating a highly controlled formulation of T cells that includes subsets of regulatory T cells, in a phase I/II clinical study in patients with certain blood cancers. The company’s second program, OGFT-001, is evaluating a fully controlled cell product candidate that contains a next-generation formulation of T cells, in a phase I study, also in patients with blood cancers. Orca Bio’s 2 ongoing clinical studies are among the largest phase I cell therapy trials ever conducted. Each product candidate has the potential to deliver curative outcomes for the initial indications Orca Bio is pursuing, as well as the promise to significantly expand the eligible patient population by substantially reducing the severe toxicities associated with conventional bone marrow transplants.

“The capital we have raised has formed the launchpad for a world-class, fully integrated allogeneic cell therapy company differentiated from all others,” said Ivan Dimov, PhD, Cofounder and Chief Executive Officer of Orca Bio. “Replacing bone marrow transplants is a logical first step in next-generation allogeneic cell therapy. While a conventional bone marrow transplant administers an uncontrolled cell product, Orca Bio has been the first to deliver a high-precision cell therapy. We are initially focused on two clinical programs in patients with blood cancers and have successfully treated the largest-ever number of patients with a high-precision cell therapy. We believe our approach has the potential to transform allogeneic cell therapy, and thus the treatment of not only blood cancer, but also many other diseases with significant unmet need, such as a variety of genetic diseases and autoimmune disorders.”

“With precise reconstitution using highly defined cell preps and a swift reboot of the patient’s immune system, Orca Bio’s product candidates have the potential to eliminate fatal side effects, such as graft-versus-host disease, and infections commonly associated with bone marrow transplants while maintaining or enhancing antitumor efficacy,” said Rick Klausner, MD, an investor and member of Orca Bio’s advisory board. “The possibility of improving cure rates and minimizing toxicity holds the promise of expanding the eligible patient population for successful bone marrow transplantation in cancer.”

“Orca Bio’s visionary leadership team, seasoned advisors, solid financial foundation, and novel technology make the company uniquely suited to develop truly differentiated, scalable allogeneic cell therapies,” said Jonathan MacQuitty, PhD, Venture Partner at Lightspeed Venture Partners. “I look forward to the Orca Bio team’s continued development and commercialization of revolutionary allogeneic cell therapies.”

Internationally Recognized Experts and Leaders

Orca Bio’s leadership, Ivan Dimov, PhD, Chief Executive Officer, Nate Fernhoff, PhD, Chief Scientific Officer, and Jeroen Bekaert, PhD, Chief Operating Officer, met at Stanford University and launched the company in 2016. Orca Bio’s board of directors and advisory board are comprised of renowned scientific leaders and seasoned biotech executives with extensive experience in drug discovery and cell-based therapeutics, including:

Orca Bio and Lyell Immunopharma Announce Research Partnership

The partnership promotes complementary scientific technologies to develop next-generation cell-based therapies for patients with solid cancers

MENLO PARK, CA, and SOUTH SAN FRANCISCO, CA, September 2, 2020 – Orca Bio and Lyell Immunopharma today announced a research partnership to jointly identify next-generation T cell therapies that will combine the high-precision purification T cell technologies from Orca Bio with the scientific expertise in T cell biology from Lyell to generate potentially synergistic therapeutic solutions for solid tumors.

Lyell Immunopharma has brought together an unrivaled scientific team focused on advancing the science of therapeutic T cells, including the role of T cell differentiation. Orca Bio has developed T cell therapies for patients with blood diseases while also streamlining the commercial manufacture of high-precision cell therapies using OrcaSort™, an ultra-fast, clinically compatible cell sorter.

The ability to produce optimal starting-cell populations is one of many continuing challenges in the development of curative T cell therapies. Evidence from current generation cell therapies suggest that the proportion of cell types and their stages of differentiation may impact the safety and efficacy of T cell therapies. Together, the companies aim to mitigate this challenge by utilizing their proprietary technologies to effectively treat solid tumor cancers.

“Lyell Immunopharma is focused on developing curative T cell therapies for solid tumor cancers by defining starting-cell preparations and modulating T cells, so they are functional in the immunosuppressive tumor microenvironment,” said Nick Restifo, MD, Executive Vice President of Research for Lyell Immunopharma. “This collaboration with Orca Bio provides the potential to more efficiently define starting-cell preparations, which I believe could lead to more effective T cell therapies.”

“Orca Bio has developed extremely pure doses of stem and immune cells that can be precisely formulated to treat cancers,” said Ivan Dimov, PhD, cofounder and CEO of Orca Bio. “By combining our scientific expertise with that of Lyell Immunopharma, and by applying our specialized manufacturing platform, we aim to create the scalable production of adoptive T cell therapies that can reach patients with unprecedented speed.”

Orca Bio and Lyell Immunopharma will each continue to pursue programs independently and through their other partnerships.

Orca Bio Announces Oral Presentation of Orca-T Clinical Data at the 62nd American Society of Hematology (ASH) Annual Meeting

Data provide insights into manufacturing efficiency, logistics, and early clinical outcomes of Orca-T for patients with blood cancers

MENLO PARK, CA, November 4, 2020 – Orca Bio, a clinical-stage biotechnology company, today announced that preliminary data from the phase I/II clinical trials of Orca-T (NCT04013685 and NCT01660607) will be presented at the 62nd American Society of Hematology (ASH) Annual Meeting, to be held virtually from December 5-8, 2020. Data will be presented on manufacturing, logistics, and early clinical outcomes. Patients in the Orca-T trial are compared with a retrospective contemporaneous control group from Stanford University. Everett Meyer, MD, PhD, Medical and Scientific Director of the Cellular Therapy Facility, Stanford Health Care and Assistant Professor of Medicine, Blood and Marrow Transplantation, Stanford University, will present the data.

Orca-T is a high-precision allogeneic cell therapy under investigation to replace a conventional mobilized blood product in hematopoietic stem cell transplant (HSCT). The therapy is designed with the goal of preventing graft-versus-host disease and other transplant related complications. Orca-T is manufactured via a proprietary GMP platform in a central lab. Patients with both related donors and unrelated donors are eligible and fresh, viable products are distributed to clinical sites around the continental United States. Data supporting reliability of manufacturing and transport will also be presented.

The US Food and Drug Administration has granted Orca-T regenerative medicine advanced therapy (RMAT) and orphan drug designation to treat patients eligible for a hematopoietic stem cell transplant. The RMAT designation provides Orca Bio the avenue to receive early guidance from the FDA to steer manufacturing and clinical development.

“This therapy was originally pioneered by Dr. Everett Meyer and Dr. Robert Negrin in a single-center trial at Stanford University, and Orca Bio has further developed the Orca-T drug in a multicenter trial. Leveraging the Orca Bio manufacturing platform, we aim to provide access to this potentially curative cell therapy to patients worldwide,” said Nate Fernhoff, PhD, Chief Scientific Officer and Cofounder of Orca Bio.

Details of the presentation