Press release

Investor Relations

Joshua Murray

Orca Bio Presents Data Demonstrating its Lead Investigational High-Precision Cell Therapy Orca-T Significantly Improved Relapse-Free Survival at 64th Annual Meeting

Orca Bio Presents Data Demonstrating its Lead Investigational High-Precision Cell Therapy Orca-T Significantly Improved Relapse-Free Survival at 64th ASH Annual Meeting

Updated data from Phase 1b/2 trials include 151 patients with acute myeloid leukemia, acute lymphocytic leukemia, myelodysplastic syndromes and other hematologic malignancies

Relapse-free survival was 87% at one year in a group of 71 patients who received the preferred conditioning regimen being used in the Precision-T Phase 3 Study of Orca-T

Data also showed improved graft-versus-host disease-free, relapse-free survival rates and overall survival rates at one year with Orca-T compared to standard of care

Precision-T is ongoing and enrolling patients at transplant centers across the U.S.

MENLO PARK, CA, Dec. 10, 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 presented updated positive clinical data on its lead investigational cell therapy, Orca-T, during an oral presentation at the 64th American Society of Hematology (ASH) Annual Meeting.

The data demonstrated a significant improvement in relapse-free survival (RFS) at one year with 87% in 71 patients who received Orca-T and busulfan, fludarabine, and thiotepa (BFT) conditioning. BFT is the preferred conditioning regimen used in Precision-T, the ongoing Phase 3 pivotal study of Orca-T. Additionally, across all 151 patients, there was an increase in graft-versus-host disease, relapse-free survival (GRFS) rates and overall survival rates at one year with Orca-T compared to an independent cohort.

“Today, providers must balance treatment decisions with the goal of fighting cancer while minimizing toxicities. With a standard allogeneic transplant, highly myeloablative conditioning regimens can lead to reduced rates of patient relapse, but can also increase rates of non-relapse mortality,” said Everett Meyer, M.D., Ph.D., primary investigator. “It is encouraging to see that the use of Orca-T across multiple hematological malignancies resulted in a remarkable improvement in relapse-free and overall survival while lowering the rate of serious toxicities compared to standard of care allogeneic hematopoietic stem cell transplants.”

The results of the patient subgroup conditioned with BFT were presented today along with pooled results from the single-center Phase 2 and multi-center Phase 1b trials from 151 patients treated with Orca-T with acute myeloid leukemia (AML), acute lymphocytic leukemia (ALL), myelodysplastic syndromes (MDS) and other hematological malignancies. For comparison purposes, an independent Center for International Blood and Marrow Transplant Research (CIBMTR)-based cohort was identified consisting of patients with AML, ALL or MDS who received a standard of care allogeneic hematopoietic stem cell transplant.

Results demonstrated that patients conditioned with BFT and treated with Orca-T experienced:

87% RFS at one year among all groups, including minimal residual disease positive acute leukemia patients.
81% GRFS at one year.
0% non-relapse mortality and only 5% moderate-to-severe chronic GvHD at one year.
94% overall survival (OS) at one year.

In pooled results from all patients treated with Orca-T, patients experienced:

70% GRFS at one year compared to 21% in the CIBMTR-based cohort.
4% non-relapse mortality at one year compared to 10% in the CIBMTR-based cohort.

88% OS at one year compared to 68% in the CIBMTR-based cohort.

Importantly, Orca-T was manufactured reliably and delivered with vein-to-vein times of 72 hours or less across the U.S.

“These relapse-free and overall survival rates from more than 150 patients treated with Orca-T are very encouraging, and we are extremely pleased to see the strong results of patients who were given the preferred conditioning regimen used in our pivotal Phase 3 trial,” said Ivan Dimov, Ph.D., co-founder and chief executive officer of Orca Bio. “These new data continue to reinforce the potential of this high-precision cell therapy to become a better treatment option for patients and transplant physicians. We remain laser-focused on advancing Orca-T through Precision-T and ultimately on delivering the therapy to more patients in need.”

The full presentation is available on www.orcabio.com.

About Orca-T

Orca-T is an investigational high-precision allogeneic cellular therapy consisting of infusions containing regulatory T-cells, conventional T-cells and CD34+ stem cells derived from peripheral blood from either related or unrelated matched donors. Orca-T has received Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. Food and Drug Administration and is being studied to treat multiple hematologic malignancies.

About Orca Bio

Orca Bio is a late-stage biotechnology company developing purified, high-precision cell therapies for the treatment of cancer, autoimmune diseases and genetic blood disorders. Our investigational therapies are designed to deliver better survival rates with dramatically fewer risks than standard allogeneic stem cell transplants, like graft versus host disease and other debilitating transplant-related toxicities. At Orca Bio, we hope to not only replace patients' blood and immune systems with healthy ones, but restore their quality of life. For more information, visit www.orcabio.com and follow Orca Bio on Twitter: @orcabio.

Contact:

Corporate Communications

Investor Relations

Joshua Murray

Orca Bio to Present Positive Clinical Outcomes with Orca-T in Patients with AML at 2024 ASCO Annual Meeting

Relapse-free survival and overall survival were 82.5% and 100% at 12 months, respectively, in 37 patients with AML, an aggressive form of blood cancer

Non-relapse mortality at 12 months was 0%

Orca-T is currently being evaluated in a pivotal Phase 3 clinical trial at leading treatment centers across the U.S.

MENLO PARK, CA, May 23, 2024 – Orca Bio, a biotechnology company committed to transforming the lives of patients through high-precision cell therapy, today announced it will present new data at the 2024 American Society of Clinical Oncology (ASCO) Annual Meeting in Chicago, Illinois, from May 31–June 4. The presentation will highlight outcomes of its lead investigational allogeneic T-cell immunotherapy, Orca-T, in patients with acute myeloid leukemia (AML).

AML is an aggressive form of blood cancer and the most common acute leukemia in adults. There are an estimated 20,800 new diagnoses and nearly 11,200 deaths in the U.S. each year.

“Today, only a fraction of adults diagnosed with AML undergo curative treatment with standard of care allogeneic hematopoietic stem cell transplant due to the serious and life-threatening risks often associated with it,” said Rawan Faramand, M.D., assistant member of Blood and Marrow Transplant and Cellular Immunotherapy at Moffitt Cancer Center. “These new findings suggest Orca-T has the potential to offer a cure with low rates of treatment-related mortality, and could provide an important option for patients with AML. With its goal of alleviating the tradeoff between the risk of relapse and the risk of toxicities observed with standard of care stem cell transplant, this novel approach has the potential to expand curative treatment to many more patients.”

A new subanalysis from the ongoing multi-center Phase 1b single-arm trial evaluated outcomes with Orca-T among a subgroup of 37 patients with AML who had a median age of 51 years and median follow-up of 14 months. All patients received myeloablative conditioning (MAC) with busulfan, fludarabine and thiotepa (BFT), followed by Orca-T and single-agent graft versus host disease (GvHD) prophylaxis with tacrolimus. At 12 months, results found relapse-free survival was 82.5% (95% CI: 65.0, 91.7) and non-relapse mortality was 0%. Overall survival was 100% (95% CI: 100, 100) at 12 months. The safety profile of Orca-T in this subgroup was consistent with the larger Phase 1b population with no new safety signals identified. Across all patients, Orca-T was manufactured reliably and delivered with vein-to-vein times of 72 hours or less across the U.S.

“We’re pleased to present at ASCO for the first time and share the latest findings that support the potential for Orca-T to offer a curative solution and fulfill a significant unmet need for patients with AML,” said Scott McClellan, M.D., Ph.D., chief medical officer at Orca Bio. “These data continue to advance our mission of expanding potentially life-saving treatment to patients and their providers who face limited viable options today.”

Details of the Orca Bio presentation follow:

Title: Treatment of Acute Myeloid Leukemia with Orca-T

Session: Hematologic Malignancies—Leukemia, Myelodysplastic Syndromes, and Allotransplant

Abstract Number: 6552

Poster Code: 111

Date and Time: June 3, 2024, from 9AM - 12PM CDT

Location: Oncology Professionals Hall

The ASCO abstracts are available at https://conferences.asco.org/am/abstracts.

About Orca-T

Orca-T is an investigational allogeneic T-cell immunotherapy being evaluated in clinical trials for the treatment of multiple hematologic malignancies. Orca-T is comprised of highly purified regulatory T-cells, CD34+ stem cells and conventional T-cells derived from peripheral blood from either related or unrelated matched donors. Orca-T is currently being evaluated in a pivotal Phase 3 clinical trial at leading transplant centers across the U.S. and has received Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. Food and Drug Administration.

About Orca Bio

Contact:

Corporate Communications

Kelsey Grossman

Investor Relations

Joshua Murray

Orca Bio Presents Positive Data Demonstrating the Potential for Orca-T and Orca-Q to Expand Treatment to Additional Patient Groups at the 65th ASH Annual Meeting

Orca-T, Orca Bio’s lead investigational high-precision cell therapy, showed similar results in older patients undergoing myeloablative conditioning as seen in younger patients across disease control, non-relapse mortality and overall survival

Orca-T in older patients given a reduced intensity conditioning regimen demonstrated curative potential with no incidence of relapse and encouraging relapse-free survival

Orca-T showed positive results in patients with intermediate to high-risk myelodysplastic syndrome

Orca-Q, Orca Bio’s second investigational high-precision cell therapy, showed encouraging data in graft-versus-host-disease-free and relapse-free survival in patients with a haploidentical donor without the use of post-transplant cyclophosphamide

MENLO PARK, CA, Dec. 9, 2023 – Orca Bio, a late-stage biotechnology company developing high-precision cell therapies for the treatment of cancer, autoimmune diseases and genetic blood disorders, today announced positive new data that suggest its investigational high-precision cell therapies, Orca-T and Orca-Q, have the potential to deliver improved clinical outcomes across different age ranges, donor types and conditioning regimens relative to existing standard of care allogeneic hematopoietic stem cell transplants (alloHSCT). The results presented at the 65th American Society of Hematology (ASH) Annual Meeting showed promising data with the use of Orca Bio’s cell therapies across several patient groups where there is significant unmet medical need.

Orca-T in Older Patients with Myeloablative Conditioning

Results highlighted in an oral presentation showed Orca-T’s ability to deliver similar outcomes in older patients undergoing myeloablative conditioning (MAC) as younger patients. Notably, Orca-T delivered similar results across disease control, non-relapse mortality (NRM) and overall survival (OS).

“While the use of MAC offers the best chance of a cure, it also increases life-threatening complications in older patients receiving standard of care alloHSCT,” said presenting author Caspian Oliai, M.D., medical director of the UCLA Bone Marrow Transplantation Stem Cell Processing Center. “These latest findings suggest a cell therapy like Orca-T, which has the potential to offer a cure while reducing toxicity and lowering treatment-related mortality, may allow older patients to better tolerate MAC. This approach could expand curative treatment to older patients who might not be offered a MAC regimen today, and potentially provide physicians with an important new option in our ongoing efforts to balance the risk of relapse with transplant-related side effects.”

In a subgroup analysis, 64 patients from Orca Bio’s ongoing multi-center Phase 1b clinical trial who received Orca-T and busulfan, fludarabine and thiotepa (BFT) conditioning for the treatment of acute myeloid leukemia (AML), acute lymphocytic leukemia (ALL), mixed phenotype leukemia, chronic myeloid leukemia (CML) and myelodysplastic syndromes (MDS) were divided into two age groups: 18-54 years of age (n=39), and 55 years of age and older (n=25). With a median follow-up of 12 months, the following outcomes were reported:

Relapse-free survival (RFS) was 84.8% in younger patients and 82.3% in older patients.
NRM was 0% in both groups.
OS was 100% and 95.5% in the younger and older patient groups, respectively.
Events of grade 3-4 acute graft versus host disease (GvHD) and moderate-to-severe chronic GvHD were low across the younger group (0 and 3, respectively) and older group (1 and 3, respectively).

Across all patients, Orca-T continued to be manufactured reliably and delivered with vein-to-vein times of 72 hours or less across the U.S.

Orca-T in Older Patients with Reduced Intensity Conditioning

Additional data from a single-center open-label Phase 1 clinical trial presented at ASH looked at the outcomes of older patients treated with Orca-T and a reduced intensity conditioning (RIC). These patients were found to be unfit for a MAC regimen due to the significant risks associated with it, particularly in older age. At 12 months, patients treated with a RIC Orca-T (n=15) saw no observable compromise in curative outcomes, with no patients experiencing relapse (0%). Patients also saw encouraging RFS (79%) and a low incidence of both grade 3-4 acute and chronic GvHD (0% and 7%, respectively).

Collectively, these findings support the potential for Orca-T to treat older patients with hematological malignancies, whether they are given a MAC or RIC regimen, with no new safety signals reported.

Orca-T in Patients with MDS

New data was also presented on the performance of Orca-T in patients with intermediate to high-risk MDS. Current treatments for MDS aren’t often curative, and many patients relapse or become resistant to first-line treatment. There remains an unmet need for new, more effective but tolerable strategies to manage MDS.

In this subgroup from the ongoing multi-center Phase 1b clinical trial, Orca-T demonstrated promising results in this patient population (n=16). At one year, RFS with Orca-T was 94%. No patients experienced grade 3-4 acute GvHD, and moderate-to-severe chronic GvHD occurred in two patients. The rates of NRM and OS with Orca-T were 0% and 94%, respectively.

Orca-T is currently being evaluated in a pivotal Phase 3 clinical study for the treatment of AML, ALL and MDS at leading transplant centers across the U.S.

Orca-Q for Patients with Haploidentical Donors

In an oral presentation, Orca Bio shared updated data from a multi-center Phase 1 clinical trial of its second high-precision cell therapy, Orca-Q, in patients with a haploidentical donor without the use of post-transplant cyclophosphamide (PTCy).

“Orca-Q is a first-in-class therapy that has the potential to improve patient outcomes and reduce the risk of graft versus host disease without the use of PTCy,” said presenting author Samer Srour, M.D., M.S., Department of Stem Cell Transplantation and Cellular Therapy at The University of Texas MD Anderson Cancer Center. “While PTCy has increased the use of haploidentical HSCTs, it can bring a myriad of risks and toxicities that we didn’t experience with Orca-Q. These updated results in an expanded group of patients continue to support Orca-Q’s promise to overcome the challenges of haploidentical stem cell transplant by providing a solution where patients and physicians may no longer have to compromise between the risk of relapse and the risk of GvHD.”

The positive outcomes from an expanded group of 33 patients with AML, ALL and CML support Orca-Q as a potential treatment option for patients with a haploidentical donor. With a median of 375 days follow-up, updated safety and efficacy data include:

No patients experienced moderate-to-severe chronic GvHD (0%).
There was one event of grade 3 acute GvHD and no grade 4 acute GvHD.
NRM was 9%.
RFS, GRFS and OS at one year were 82%.

Additionally, no new safety signals were identified. The estimated incidence of CTCAE grade 2 and greater than grade 3 infections at one year were 9% and 15%, respectively.

“At Orca Bio, we understand the challenges providers face in achieving the right balance in offering blood cancer patients the best option for a cure with optimal quality of life,” said Ivan Dimov, Ph.D., co-founder and chief executive officer of Orca Bio. “We are pleased to present these latest findings at ASH that add to our growing body of clinical evidence which underscore the potential of our novel high-precision platform to expand potentially life-saving treatment to more patient groups who could benefit.”

The full ASH presentations will be made available on www.orcabio.com.

About Orca-T

Orca-T is an investigational high-precision allogeneic cell therapy being evaluated in clinical trials for the treatment of multiple hematologic malignancies. Orca-T includes infusions containing regulatory T-cells, CD34+ stem cells and conventional T-cells derived from peripheral blood from either related or unrelated matched donors. Orca-T is currently being evaluated in a pivotal Phase 3 clinical trial at leading transplant centers across the U.S. and has received Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. Food and Drug Administration.

About Orca-Q

Orca-Q is an investigational high-precision allogeneic cell therapy being evaluated in clinical trials for the treatment of hematologic malignancies in patients with haploidentical donors. Orca-Q is a proprietary composition of stem cells combined with specific T-cell subsets derived from healthy donors and engineered by Orca Bio’s high-precision platform. Orca-Q has the potential to improve patient outcomes and reduce the risks of toxicities without the use of post-transplant cyclophosphamide (PTCy) in patients unable to identify a full human leukocyte antigen (HLA) match.

About Orca Bio

Orca Bio is a late-stage biotechnology company developing high-precision cell therapies for the treatment of cancer, autoimmune diseases and genetic blood disorders. Our investigational products are designed to safely replace a patient’s diseased blood and immune system with a healthy one, delivering significantly better outcomes with dramatically fewer risks than the standard of care. Our manufacturing platform uses single-cell precision to create proprietary, uniquely-defined products that have the potential to transform allogeneic cell therapy. At Orca Bio, our mission is to make curative cell therapies both more effective and safer, and in doing so, push past the field’s current boundaries and redefine its future. For more information, visit www.orcabio.com and follow Orca Bio on Twitter: @OrcaBio.

Contact:

Corporate Communications

Kelsey Grossman

Investor Relations

Joshua Murray

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

New data evaluating Orca-T® with reduced intensity conditioning for the treatment of hematological malignancies in patients 60 to 75 years of age

Clinical findings of Orca-Q® with and without the use of GvHD prophylaxis in patients with hematological malignancies including AML, ALL and MDS

Expanded feasibility data on the combination of Orca-T and an allogeneic CAR-T (OrCAR-T™) in patients with high-risk B-ALL

Additional presentations will highlight overall survival and non-relapse mortality with Orca-T versus PTCy, and additional subsets from the pivotal Phase 3 study including relapse-free survival and patient-reported outcomes

MENLO PARK, CA, November 3, 2025 – Orca Bio, a late-stage biotechnology company committed to transforming the lives of patients through high-precision cell therapy, today announced that new clinical data will be presented in three oral and three poster sessions at the American Society of Hematology (ASH) Annual Meeting from December 6-9 in Orlando, FL. The presentations will span its pipeline of investigational allogeneic T-cell immunotherapies for the treatment of multiple hematological malignancies including Orca-T, Orca-Q and the Orca-T and allogeneic CAR-T combination therapy, OrCAR-T.

“Our ongoing clinical evaluation of our pipeline across diverse patient populations, conditioning regimens and donor types underscores our commitment to advancing care for people with blood cancer,” said Nate Fernhoff, Ph.D., co-founder and chief executive officer of Orca Bio. “We look forward to presenting new and expanded evidence from the pivotal Phase 3 study of our lead T-cell immunotherapy, Orca-T, along with data highlighting the potential of our next-generation candidate, Orca-Q, to enable treatment without GvHD prophylaxis. Overall, these findings mark meaningful progress toward our goal of delivering our high-precision approach to more patients who may benefit.”

The ASH abstracts are now available at www.hematology.org. Details of the Orca Bio oral and poster presentations follow:

Oral Session: 721. Allogeneic Transplantation: Conditioning Regimens, Engraftment, and Acute Toxicities: Improving Outcomes by Decreasing Transplant-Related Complications

Title: Allogeneic HSC and Regulatory T cell (Orca-T) Engineered Cell Therapy Following Reduced Intensity Conditioning: Results of a Single Center Phase 1 Study

Abstract Number: 111

Date and Time: December 6, 2025 at 10:00 AM EST

Location: OCCC - Chapin Theater (320)

Oral Session: 732. Allogeneic Transplantation: Disease Response and Comparative Treatment Studies: Biologic Predictors and Novel Platforms Integrating CAR T with Transplant

Title: Superior Efficacy and Persistence of Orca-T-Allogeneic CAR19/22 Versus Autologous CAR19/22 in High-Risk Adult B-ALL

Abstract Number: 514

Date and Time: December 7, 2025 at 10:15 AM EST

Location: OCCC - Chapin Theater (320)

Oral Session: 722. Allogeneic Transplantation: Acute and Chronic GVHD and Immune Reconstitution: GVHD Prevention: Post-transplant cyclophosphamide and beyond

Title: Preliminary Safety and Efficacy of Myeloablative Orca-Q with Tacrolimus or without GVHD Prophylaxis for Treatment of Advanced Hematologic Malignancies

Abstract Number: 932

Date and Time: December 8, 2025 at 3:00 PM EST

Location: OCCC - W331

Poster Session: 722. Allogeneic Transplantation: Acute and Chronic GVHD and Immune Reconstitution: Poster I

Title: Orca-T Improves cGVHD-free Survival in Patients with a Broad Range of Demographic and Clinical Variables: Results of Randomized, Phase 3 Trial

Abstract Number: 2477

Date and Time: December 6, 2025 at 5:30 PM - 7:30 PM EST

Location: OCCC - West Halls B3-B4

Poster Session: 722. Allogeneic Transplantation: Acute and Chronic GVHD and Immune Reconstitution: Poster I

Title: Orca-T Demonstrates Favorable Quality of Life and Healthcare Resource Use Compared to Standard AlloHSCT plus Tac/MTX for GVHD Prevention in a Randomized Phase 3 Clinical Trial (Precision-T)

Abstract Number: 2473

Date and Time: December 6, 2025 at 5:30 PM - 7:30 PM EST

Location: OCCC - West Halls B3-B4

Poster Session: 722. Allogeneic Transplantation: Acute and Chronic GVHD

and Immune Reconstitution: Poster III

Title: Observational Comparison of Orca-T to Registry-Based Post-Transplant Cyclophosphamide Patients using Matched Unrelated Donor

Abstract Number: 6029

Date and Time: December 8, 2025 at 6:00 PM - 8:00 PM EST

Location: OCCC - West Halls B3-B4

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 PDUFA target action date of April 6, 2026.

About Orca-Q

Orca-Q is Orca Bio’s second-generation investigational allogeneic T-cell immunotherapy under evaluation in clinical trials for the treatment of multiple hematologic malignancies, including in patients with haploidentical and mismatched donors. Orca-Q is a proprietary composition of stem cells combined with specific T-cell subsets derived from healthy donors and engineered by Orca Bio’s high-precision platform.

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

Investor Relations

Joshua Murray

Orca Bio Presents Positive Phase 1 and 1b Data on Orca-T and Orca-Q at the 2025 Tandem Meetings of ASTCT® and CIBMTR®

Orca Bio Presents Positive Phase 1 and Phase 1b Data on Orca-T and Orca-Q at the 2025 Tandem Meetings of ASTCT® and CIBMTR®

MENLO PARK, CA, February 12, 2025 – Orca Bio, a late-stage biotechnology company committed to transforming the lives of patients through high-precision cell therapy, today announced positive data on its investigational allogeneic T-cell immunotherapies will be presented across four sessions at the 2025 Tandem Meetings of ASTCT® and CIBMTR®, February 12-15, 2025, in Honolulu, HI.

"We are pleased to present positive findings across four presentations at the 2025 Tandem Meetings, underscoring our commitment to engaging global experts in a collective effort to advance the field for patients and the physicians who treat them,” said Scott McClellan, MD, chief medical officer at Orca Bio. “These data add to our growing body of clinical evidence which reinforces the potential of our high-precision approach to offer new and potentially transformative treatment options for patients."

Highlights include:

New research that sheds light on early T-cell activation following treatment with Orca Bio's lead investigational allogeneic T-cell immunotherapy, Orca-T, compared with unmanipulated peripheral blood stem cell grafts, in addition to the identification of a novel T-cell subset which may be predictive of long-term immune activation after Orca-T administration.
Three-year follow-up data from the Phase 1b trial of Orca-T demonstrating improved overall survival in AML, ALL and high-risk MDS patients compared to a conventional allogeneic stem cell transplant (alloHSCT) with post-transplant cyclophosphamide (PTCy)-based GvHD prophylaxis. Results previously presented at the 2024 American Society of Hematology (ASH) Annual Meeting.
Encouraging results from a single-center open-label Phase 1 study of an expanded group of older patients treated with Orca Bio Orca-T and a reduced-intensity conditioning (RIC) regimen. Cohorts of fewer patients were previously presented at the 2023 ASH Annual Meeting and the 50th Annual Meeting of the EBMT.
Results from the Phase 1 trial of Orca Bio’s second investigational allogeneic T-cell immunotherapy, Orca-Q, demonstrating promising outcomes without the use of pharmacological GvHD prophylaxis in patients with fully matched donors. Data previously presented at the 2024 ASH Annual Meeting.

The abstracts are available at www.tandemmeetings.com. Details of the presentations follow:

Oral Session: Session L - Autoimmune Disease and Immune Reconstitution

Title: FOXP3 and Helios Expressing CD4+ T Conventional Cells Correlate with T Cell Activation after Orca-T Allogeneic T Cell Immunotherapy

Date and Time: February 15, 2025 at 10:30 AM HST

Location: Ballroom C (HCC)

Poster Session: Acute and Chronic Leukemia (AML, MDS, MPD ALL, CML) - Clinical

Title: Observational Comparison of Overall Survival between Phase 1b Orca-T and Registry-Based Post-Transplant Cyclophosphamide Patients

Date and Time: February 13, 2025 at 6:45 PM HST

Location: Exhibit Hall 3 (HCC)

Poster Session: Graft-versus-Host and Graft-versus-Tumor - Clinical: Prevention, Treatment and Biomarkers

Title: Phase 1 Trial Results for Patients with Advanced Hematologic Malignancies Treated with Allogeneic T Cell Immunotherapy and Reduced Intensity Conditioning

Date and Time: February 13, 2025 at 6:45 PM HST

Location: Exhibit Hall 3 (HCC)

Poster Session: Acute and Chronic Leukemia (AML, MDS, MPD ALL, CML) - Clinical

Title: Preliminary Safety and Efficacy of Myeloablative Orca-Q with No GvHD Prophylaxis for Treatment of Advanced Hematologic Malignancies

Date and Time: February 13, 2025 at 6:45 PM HST

Location: Exhibit Hall 3 (HCC)

About Orca-T

Orca-T is an investigational allogeneic T-cell immunotherapy being evaluated in clinical trials for the treatment of multiple hematologic malignancies. Orca-T is comprised of highly purified regulatory T-cells, CD34+ stem cells and conventional T-cells derived from peripheral blood from either related or unrelated matched donors. Orca-T is currently being evaluated in Precision-T, a pivotal Phase 3 clinical trial, which has completed enrollment at leading transplant centers across the U.S. Orca-T has received Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. Food and Drug Administration.

About Orca Bio

Orca Bio is a late-stage biotechnology company developing high-precision cell therapies for the treatment of cancer and autoimmune diseases. Our 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.

Contact:

Corporate Communications

Kelsey Grossman

Investor Relations

Joshua Murray

Orca Bio Expands Manufacturing Capabilities to Develop and Commercialize its Precision Cell Therapies

New state-of-the art manufacturing facility is designed to enable the reliable production of Orca Bio's investigational cell therapies from late-stage clinical development through potential commercialization

The 100,000-square-foot facility is located in Metro Air Park, Sacramento

MENLO PARK, CA, September 8, 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 the expansion of its current manufacturing capabilities with the construction of a new 100,000-square-foot commercial facility in Sacramento, CA. This facility will support the late-stage clinical development and commercialization of Orca Bio’s precision cell therapies, including Orca-T, which is currently being evaluated in a Phase 3 registrational trial.

“The new state-of-the-art facility represents critical infrastructure to enable the advancement of our pipeline and the future commercial availability of our potentially life-saving products,” said Jeroen Bekaert, Ph.D., co-founder and chief operating officer of Orca Bio. “With the capacity to manufacture approximately 3,000 cell therapy products per year, the facility will allow us to scale to meet future demand. Investing in our manufacturing capabilities is essential to our mission of delivering a cure without compromise while ensuring timely access to the patients who need it, when they need it.”

The new facility is strategically located in Sacramento's newly developed Metro Air Park next to the Sacramento International Airport, enabling direct shipping access to transplant centers across the U.S. It is also in close proximity to Orca Bio's existing clinical manufacturing building. The facility is uniquely tailored to manufacture precision cell therapies designed to replace cancerous blood and immune systems with healthy ones. It touts modular production suites, which are adjustable for future growth, quality control laboratories, warehouse space and offices. The construction began in August of 2021 and is scheduled to be completed later this year. The facility is expected to be fully validated and operational in the first half of 2023.

“Throughout the clinical development of our products, we have demonstrated the ability to reliably manufacture and deliver high-precision cell therapies with rapid turnaround times, regardless of donor and patient location,” said Ivan Dimov, Ph.D., co-founder and chief executive officer of Orca Bio. “This new facility will further enhance our capabilities to deliver our therapies urgently and seamlessly to patients at scale, while meeting the highest quality and regulatory standards.”

Orca Bio’s unique approach uses precision cell selection technology to identify the less than 1% of the 100 billion donor cells that potentially contain therapeutic benefits for patients. These cells are then manufactured into potentially curative cell therapies designed to maximize efficacy of treatment and significantly limit treatment-related risks. Orca Bio recently announced Precision-T, its pivotal Phase 3 study evaluating Orca-T for the treatment of acute myeloid leukemia (AML), acute lymphocytic leukemia (ALL) and high-risk myelodysplastic syndromes (MDS), is open for enrollment at leading transplant centers across the U.S.

About Orca-T

About Orca Bio

Orca Bio is a clinical-stage biotechnology company developing purified, high-precision cell therapies for the treatment of cancer, autoimmune diseases and genetic blood disorders. Our investigational therapies are designed to deliver better survival rates with dramatically fewer risks than standard allogeneic stem cell transplants, like graft versus host disease and other debilitating transplant-related toxicities. At Orca Bio, we hope to not only replace patients' blood and immune systems with healthy ones, but restore their quality of life. For more information, visit www.orcabio.com and follow Orca Bio on Twitter: @OrcaBio.

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Kelsey Grossman

Investor Relations

Joshua Murray

Orca Bio Announces Positive Results from the Pivotal Phase 3 Study of Investigational Orca-T® Compared to Allogeneic Stem Cell Transplant for the Treatment of Hematologic Malignancies

Precision-T study met the primary endpoint of a statistically significant improvement in survival free of moderate-to-severe chronic graft versus host disease (cGvHD), showing 78% with Orca-T versus 38% with conventional allogeneic stem cell transplant (alloHSCT) at one year (HR 0.26, p<0.00001)

Overall survival with Orca-T was 94% compared to 83% with alloHSCT at one year, and the cumulative incidence of moderate-to-severe cGvHD was 13% versus 44%, respectively

Orca Bio is preparing to submit these findings in a BLA to the U.S. FDA in 2025

Results will be presented at the 51st Annual Meeting of the EBMT

MENLO PARK, CA, March 17, 2025 – Orca Bio, a late-stage biotechnology company committed to transforming the lives of patients through high-precision cell therapy, today announced positive results from the pivotal Phase 3 Precision-T study of Orca-T, its lead investigational allogeneic T-cell immunotherapy, in patients with acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), high-risk myelodysplastic syndrome (MDS) and mixed-phenotype acute leukemia (MPAL). Orca-T is manufactured using highly purified regulatory T-cells, hematopoietic stem cells and conventional T-cells derived from peripheral blood from either related or unrelated matched donors.

In the randomized Precision-T study, Orca-T met the primary endpoint of a statistically significant improvement in survival free of moderate-to-severe chronic graft versus host disease (cGvHD) with Orca-T. At one year, the rate for patients who received Orca-T was 78% compared to 38% for patients who received a conventional allogeneic hematopoietic stem cell transplant (alloHSCT). Patients in the Orca-T group achieved an estimated overall survival (OS) of 94% compared to 83% in the alloHSCT arm at one year.

"Today, treating patients with serious blood cancers using allogeneic stem cell transplants requires a difficult risk-benefit trade-off as clinicians aim to cure the disease while avoiding potentially deadly treatment-related toxicities, like GvHD," said presenting author Everett Meyer, M.D., Ph.D., hematologist and associate professor of medicine in Blood and Marrow Transplantation and Cellular Therapy at Stanford Health Care. "The Precision-T study showed double the rate of survival free from GvHD with Orca-T versus a conventional transplant, a relapse-free survival rate of 76% and no new safety concerns. These findings are highly encouraging and provide compelling new evidence as we work to solve for the critical factors contributing to the needs of this patient population."

Precision-T Study Results

In the study, all patients (n=187) with a median age of 43.5 years (range 19-65 years) were randomized 1:1 to Orca-T plus single-agent tacrolimus (TAC) or alloHSCT plus TAC methotrexate (TAC/MTX). Patients across both groups received myeloablative conditioning (MAC) and used a related or unrelated matched donor. Patients had a median follow-up time of 11.4 months (range 0.2-24.3 months) across both arms. Key results from the Precision-T study at one year are summarized below:

The primary endpoint of survival free of cGvHD was 78% (95% CI: 65%, 87%) in the Orca-T arm (n=93) and 38% (95% CI: 26%, 51%) in the alloHSCT arm (n=94) (HR 0.26; p<0.00001).
An interim analysis of the secondary endpoint of OS was 94% (95% CI: 86%, 97%) in the Orca-T arm and 83% (95% CI: 73%, 90%) in the alloHSCT arm (HR 0.49; p=0.11823).
An additional secondary endpoint of cumulative incidence of moderate-to-severe cGvHD was 13% (95% CI: 5%, 23%) and 44% (95% CI: 31%, 56%) in the Orca-T and alloHSCT arms, respectively (HR 0.19; p<0.00002).

Exploratory endpoints at one year include the rate of relapse-free survival which was 76% and 74% in the Orca-T and alloHSCT arms, respectively (HR 0.80, p=0.49). The cumulative incidence of non-relapse mortality was 3% in the Orca-T arm and 13% in the alloHSCT arm. Additionally, the cumulative incidence of Grade 3 or 4 acute GvHD was 6% and 17% in the Orca-T and alloHSCT arms, respectively.

No new safety issues were identified with Orca-T. Grade ≥ 4 infections per CTCAE scoring were noted in 6% and 10% of patients in the Orca-T and alloHSCT arms, respectively.

Orca-T was manufactured in Orca Bio’s centralized GMP facility and delivered to patients at 19 treatment centers across the U.S., with all infusions occurring within a vein-to-vein time of 72 hours or less.

“Approximately 46,000 people are diagnosed with AML, ALL and MDS in the U.S. each year, but only a fraction of them receive an allogeneic stem cell transplant within the current paradigm,” said Rawan Faramand, M.D., Blood and Marrow Transplant and Cellular Immunotherapy, Moffitt Cancer Center. “Additional treatment options are needed, and the introduction of a cell therapy like Orca-T that leverages a precision-based approach could pave the way for a new standard of care for patients with various hematologic malignancies.”

“These exciting results underscore Orca Bio’s vision of transforming the treatment landscape for patients living with serious blood cancers, potentially standardizing curative treatment for diseases like AML, ALL and MDS,” said Ivan Dimov, Ph.D., co-founder and chief executive officer at Orca Bio. “We are working closely with the FDA and expect to submit a Biologics License Application this year. These results support the validity of our high-precision platform as we continue to advance our robust pipeline of allogeneic cell therapies for the treatment of hematologic malignancies, autoimmune diseases and beyond.”

Orca Bio is grateful to the patients and families, donors and trial site investigators who participated in the Precision-T study.

The complete results will be presented on April 2, 2025, at the 51st Annual Meeting of The EBMT in Florence, Italy.

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 being evaluated for the treatment of multiple hematologic malignancies. Orca-T is composed of highly purified regulatory T-cells, CD34+ stem cells and conventional T-cells derived from peripheral blood 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.

About Orca Bio

Contact:

Corporate Communications

Kelsey Grossman

Investor Relations

Joshua Murray