Orca Bio Announces Leadership Updates

Press release

May 3, 2025

Orca Bio Announces Leadership Updates

Co-founder and Chief Scientific Officer of Orca Bio, Nate Fernhoff, Ph.D., appointed Chief Executive Officer 
Co-founder and Chief Operating Officer of Orca Bio, Jeroen Bekaert, Ph.D., appointed President
Company is accelerating investment in manufacturing and commercial infrastructure in preparation for planned 2025 BLA to the U.S. FDA

MENLO PARK, CA, May 3, 2025  Orca Bio, a late-stage biotechnology company committed to transforming the lives of patients through high-precision cell therapy, today announced the appointment of co-founder and Chief Scientific Officer Nate Fernhoff, Ph.D., as Chief Executive Officer (CEO), succeeding Ivan Dimov, Ph.D. In addition, co-founder and Chief Operating Officer, Jeroen Bekaert, Ph.D., has assumed the role of President at Orca Bio.


“I am honored and humbled to serve as CEO as we progress toward our vision of transforming the treatment landscape for patients living with blood cancer,” said Dr. Fernhoff. “Following the positive Phase 3 results for Orca-T, we remain focused on a 2025 BLA submission and ensuring commercial readiness. In parallel, we are advancing our pipeline of groundbreaking cell therapies that leverage the same high-precision platform to potentially offer new and innovative treatments to more patients who could benefit.”


Dr. Fernhoff continued, “I’m fortunate to continue to partner with Jeroen in his new role as President where he will oversee operations across all areas of the organization. Since co-founding Orca Bio, Jeroen has played an integral part in guiding the company from its inception through multiple phases of clinical development and organizational growth.”


After reporting positive Phase 3 clinical data on its lead investigational allogeneic T-cell immunotherapy, Orca-T, Orca Bio continues to scale its U.S. manufacturing infrastructure and operations to ensure consistent and reliable delivery of its products. This includes activating new production lines in its state-of-the-art commercial manufacturing facility to increase capacity and support future demand. The company is also strengthening the commercial organization with the addition of leaders who have successful cell therapy launch expertise across commercial operations, market access and stakeholder engagement.


Nate Fernhoff, Ph.D., Co-founder and Chief Executive Officer


Dr. Fernhoff is the CEO and co-founder of Orca Bio. In his previous role as Chief Scientific Officer, Dr. Fernhoff led the advancement of Orca Bio’s robust pipeline of high-precision cell therapies from early development through clinical translation. He is one of the inventors of Orca-Q, Orca Bio’s second-generation investigational allogeneic T-cell immunotherapy for patients with hematologic malignancies. Prior to Orca Bio, Dr. Fernhoff was a Ruth L. Kirchstein National Research Service Award (NRSA) Postdoctoral Scholar in the Irving Weissman laboratory at Stanford University. He holds a Ph.D. in molecular and cell biology from the University of California, Berkeley, and bachelor’s degrees in biological sciences and mathematics from Stanford University. Dr. Fernhoff currently serves on the board of the ML4 Foundation, an organization dedicated to funding, promoting and supporting medical research for Mucolipidosis Type IV (ML4), a genetic disease.


Jeroen Bekaert, Ph.D., Co-founder, President and Chief Operating Officer


Dr. Bekaert is the President and co-founder of Orca Bio where he oversees the operational activities across all company functions. Before joining Orca Bio, Dr. Bekaert was the head of Merck KGaA’s innovation center in Silicon Valley, where he was responsible for shaping and onboarding science and technology opportunities from universities and emerging biotech companies across Merck KGaA’s pharma and life science sectors. Prior to this, Dr. Bekaert held a variety of positions of increasing responsibility in Johnson & Johnson’s Supply Chain group, most recently as Director, worldwide engineering and technical operations, overseeing the EMEA portfolio. Dr. Bekaert started his career as a researcher at the Los Alamos National Laboratory in New Mexico. He holds a master’s and Ph.D. in computer science engineering from Ghent University. He was a Gustave Boël-Sofina Fellow and also holds a master’s degree in management from Stanford Graduate School of Business.

About Orca Bio

Orca Bio is a late-stage biotechnology company developing high-precision cell therapies for the treatment of 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.

Contact:

Corporate Communications

Kelsey Grossman

media@orcabio.com

Investor Relations

Joshua Murray

ir@orcabio.com

Orca Bio to Present Clinical Data on Its High-Precision Cell Therapies at the 66th American Society of Hematology Annual Meeting

Press release

Menlo Park, CA

November 5, 2024

Orca Bio to Present Clinical Data on Its High-Precision Cell Therapies at the 66th American Society of Hematology Annual Meeting

Long-term survival data with Orca-T will be presented for the first time in patients with AML, ALL and MDS

Early feasibility data on the combination of Orca-T and an allogeneic CAR-T in patients with high-risk B-ALL will be shown

A third oral presentation will highlight patient outcomes with Orca-Q without the use of any graft versus host disease (GvHD) prophylaxis in patients with hematologic malignancies 

MENLO PARK, CA, November 5, 2024 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 sessions at the American Society of Hematology (ASH) Annual Meeting from December 7-10 in San Diego, CA. 

Three-year survival data with Orca Bio’s lead investigational allogeneic T-cell immunotherapy, Orca-T, will be presented from the multicenter Phase 1b clinical trial of patients with acute myeloid leukemia (AML), acute lymphocytic leukemia (ALL) and high-risk myelodysplastic syndrome (MDS). Additionally, initial feasibility, safety and efficacy data from an investigator-sponsored trial evaluating the combination of Orca-T with allogeneic CD19/CD22-CAR-T cells in patients with high-risk B-cell acute lymphoblastic leukemia (B-ALL) will be shared. In a third oral session, data from a multicenter Phase 1 clinical trial will be presented from patients treated with Orca Bio’s second-generation investigational allogeneic T-cell immunotherapy, Orca-Q, in patients with hematologic malignancies without the use of any post-treatment graft versus host disease (GvHD) prophylaxis.

“We are pleased to present exciting new data from our lead clinical programs for patients with blood cancers, including three-year follow-up with Orca-T, our lead cell therapy candidate currently being evaluated in a pivotal Phase 3 clinical trial,” said Ivan Dimov, Ph.D., co-founder and chief executive officer of Orca Bio. “We also look forward to debuting the results of patients treated with Orca-Q without the use of any GvHD prophylaxis. Overall, these findings represent important progress toward our goal of delivering our high-precision approach to more patients who could potentially benefit.”

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

Oral Session: 732. Allogeneic Transplantation: Disease Response and Comparative Treatment Studies: Finding the Ideal Donor and Graft: Going Beyond HLA

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

Abstract Number: 694

Date and Time: Sunday, December 8, 2024 at 5:15 PM PT

Location: San Diego Convention Center, Room 6A

Oral Session: 704. Cellular Immunotherapies: Early Phase Clinical Trials and Toxicities: B Cell-directed CAR T Cell Therapies for ALL and for Autoimmunity

Title: Safe and Effective Combination of Donor-Derived, Allogeneic CD19/CD22-CAR T Cells with Myeloablative Graft-Engineered Allo-HCT for High-Risk B-ALL 

Abstract Number: 679

Date and Time: Sunday, December 8, 2024 at 4:30 PM PT

Location: Marriott Marquis San Diego Marina, Marriott Grand Ballroom 2-4


Oral Session: 722. Allogeneic Transplantation: Acute and Chronic GVHD and Immune Reconstitution: Monitoring, Understanding and Optimizing GVHD Interventions

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

Abstract Number: 382

Date and Time: Saturday, December 7, 2024 at 4:45 PM PT

Location: San Diego Convention Center, Room 6CF

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 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-Q

Orca-Q is Orca Bio’s second-generation investigational allogeneic T-cell immunotherapy being evaluated in clinical trials for the treatment of multiple hematologic malignancies, including 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. 

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 investigational products are designed to replace a patient’s diseased blood and immune system with a healthy one, with the goal of improving outcomes with 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.

Contact:

Corporate Communications

Kelsey Grossman

media@orcabio.com

Investor Relations

Joshua Murray

ir@orcabio.com


Orca Bio Presents Three-Year Survival Data with Orca-T® in Patients with Hematological Malignancies at the 66th ASH Annual Meeting

Press release

Menlo Park, CA

December 8, 2024

Orca Bio Presents Three-Year Survival Data with Orca-T® in Patients with Hematological Malignancies at the 66th ASH Annual Meeting

Three-year overall survival with Orca-T allogeneic T-cell immunotherapy was 86% for patients with AML, ALL and MDS compared to 67% in a non-randomized historical cohort of post-transplant cyclophosphamide (PTCy)
Reduced non-relapse mortality and increased relapse-free survival seen with Orca-T compared to PTCy at one-year
In a second oral presentation, early feasibility data were presented on the combination of Orca-T and allogeneic CAR-T cells in patients with high-risk B-ALL
Orca-T is being evaluated in a pivotal Phase 3 clinical trial versus a conventional allogeneic stem cell transplant with data expected in the first half of 2025

MENLO PARK, CA, December 8, 2024  Orca Bio, a late-stage biotechnology company committed to transforming the lives of patients through high-precision cell therapy, today presented positive long-term data on its lead investigational allogeneic T-cell immunotherapy, Orca-T, at the 66th American Society of Hematology (ASH) Annual Meeting.

Three-Year Survival Data with Orca-T vs PTCy

The results of a three-year follow-up analysis with Orca-T plus single-agent tacrolimus (TAC) showed encouraging overall survival (OS) in patients with acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL) and high-risk myelodysplastic syndrome (MDS). These results also showed an improvement when compared to a historical cohort of patients receiving an allogeneic stem cell transplant (alloHSCT) with post-transplant cyclophosphamide (PTCy)-based graft versus host disease (GvHD) prophylaxis.

“With the current standard of care, physicians must carefully balance the risk of relapse with the risk of toxicities when treating patients with serious hematological malignancies, as both impact overall survival,” said presenting author Caspian Oliai, MD, medical director of the UCLA Bone Marrow Transplantation Stem Cell Processing Center. “The preliminary results with Orca-T have been promising, and now observational outcomes show sustained improvement in comparison to a similar cohort of patients who received a conventional alloHSCT with PTCy at one, two and three years. These data add to the growing body of evidence suggesting Orca-T could potentially offer an important new treatment option for patients.”

This analysis from the multicenter Phase 1b clinical trial evaluated a subset of patients (n=77) who received Orca-T with TAC. These outcomes were compared to patients from a CIBMTR literature-based cohort (n=293) who received alloHSCT with a PTCy-based GvHD prophylaxis regimen. All patients across both groups received myeloablative conditioning (MAC) and used a related or unrelated fully matched donor. These subsets of patients were selected based on disease, disease status and conditioning regimens that most closely resemble the patients enrolled in the ongoing pivotal Phase 3 clinical trial evaluating Orca-T.

The analysis included patients who had a median follow-up time of 33 months (range 5-54 months) and 24 months (range 0-53 months) in the Orca-T Phase 1b and PTCy cohorts, respectively. The OS at 12, 24 and 36 months (p=0.0021) are summarized in the table below:

Phase 1b Orca-T plus TAC OS AlloHSCT with PTCy OS
1 Year 96% (range 88%-99%; 95% CI) 82% (range 78%-87%; 95% CI)
2 Year 88% (range 78%-94%; 95% CI) 73% (range 68%-79%; 95% CI)
3 Year 86% (range 73-92%; 95% CI) 67% (range 61%-74%; 95% CI)

Further analyses of these patient subsets found Orca-T may reduce non-relapse mortality (NRM) and increase relapse-free survival (RFS) compared to PTCy. At one year, NRM was 1.4% in the Orca-T group and 7.4% in the PTCy cohort. The rate of RFS at one year was 83% and 71% for patients receiving Orca-T and PTCy, respectively. Results also found that age had no significant impact on OS at one year. For patients receiving Orca-T, OS was 95% (88%-100%) in patients under the age of 50 and 97% (92%-100%) in patients over the age of 50. For patients in the PTCy cohort, OS was 86% (81%-92%) in patients under the age of 50 and 77% (70%-85%) in patients over the age of 50. 

Across all patients in the Phase 1b trial, 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 positive long-term follow-up data for Orca-T as these findings provide valuable insight into how the cell therapy performs over extended periods of time,” said Scott McClellan, MD, PhD, chief medical officer at Orca Bio. “As we eagerly await the results of our pivotal Phase 3 clinical trial evaluating Orca-T in a similar patient population, we are one step closer to gathering the information needed for BLA submission and toward potentially making a significant difference for patients.”

Combining Orca Bio Approach with CAR-T Technology

A second oral presentation highlighted initial feasibility, safety and efficacy data from an investigator-sponsored Phase 1 clinical trial evaluating OrCAR-T, a platform that combines the Orca Bio technology with CAR-T technology. This presentation highlighted data following treatment with Orca-T and allogeneic CD19/CD22 CAR-T cells in patients with high-risk B-cell acute lymphoblastic leukemia (B-ALL).

B-ALL remains incurable for many patients despite treatment with an alloHSCT or autologous CAR-T cells, which can also lead to serious complications including GvHD, infections and CAR-related toxicities. Preliminary results for 10 patients treated with OrCAR-T who reached the primary endpoint of 42 days post-treatment showed neutrophil and platelet engraftment at 14 days. At a median follow up of 381 days (range 61-762 days, including 6 patients with >12 months of follow-up) across three dose escalation levels (1x106 CAR+ cells/kg; 2x106 CAR+ cells/kg; 3x106 CAR+ cells/kg), there were no cases of acute or chronic GvHD, no grade 3 or greater cytokine release syndrome and no neurotoxicity observed. 

These early findings suggest a single treatment that combines a high-precision cell therapy like Orca-T with specific CAR-T cells has the potential to benefit patients with a range of hematologic diseases.


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 investigational products are designed to replace a patient’s diseased blood and immune system with a healthy one, with the goal of improving outcomes with 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.

Contact:

Corporate Communications

Kelsey Grossman

media@orcabio.com

Investor Relations

Joshua Murray

ir@orcabio.com

Orca Bio to Present at the 43rd Annual J.P. Morgan Healthcare Conference

Press release

Menlo Park, CA

January 6, 2025

Orca Bio to Present at the 43rd Annual J.P. Morgan Healthcare Conference

MENLO PARK, CA, January 6, 2025  Orca Bio, a late-stage biotechnology company committed to transforming the lives of patients through high-precision cell therapy, today announced that Ivan Dimov, Ph.D., Orca Bio’s co-founder and chief executive officer, will present at the 43rd Annual J.P. Morgan Healthcare Conference in San Francisco, CA.

The company presentation will take place on Monday, January 13, 2025, at 8:00AM PST at the Westin St. Francis in the Golden Gate Room on the 32nd floor.

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 investigational products are designed to replace a patient’s diseased blood and immune system with a healthy one, with the goal of improving outcomes with 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.

Contact:

Corporate Communications

Kelsey Grossman

media@orcabio.com

Investor Relations

Joshua Murray

ir@orcabio.com

Orca Bio Announces U.S. FDA Regenerative Medicine Advanced Therapy (RMAT) Designation Granted to Orca-Q® for the Treatment of High-Risk Hematologic Malignancies

Press release

Menlo Park, CA

April 28, 2026

Orca Bio Announces U.S. FDA Regenerative Medicine Advanced Therapy (RMAT) Designation Granted to Orca-Q® for the Treatment of High-Risk Hematologic Malignancies 

RMAT designation based on promising preliminary Phase 1 clinical data, including positive outcomes across overall survival, acute and chronic GVHD and non-relapse mortality 
Phase 1 trial was recently expanded to include additional cohorts of patients receiving reduced intensity or non-myeloablative conditioning with either matched, 7/8 mismatched or haploidentical donors  
New clinical findings and longer follow-up data expected in 2026 

MENLO PARK, CA, April 28, 2026  Orca Bio, a late-stage biotechnology company committed to transforming the lives of patients through high-precision cell therapy, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to Orca-Q, Orca Bio’s second-generation investigational allogeneic T-cell immunotherapy for the treatment of high-risk hematologic malignancies.

"The FDA’s RMAT designation for Orca-Q recognizes the significant unmet need for patients with serious hematologic malignancies and validates the promising clinical findings from our ongoing Phase 1 study,” said Nate Fernhoff, Ph.D., co-founder and chief executive officer at Orca Bio. “As this is our second cell therapy candidate to receive RMAT status, we have experienced firsthand how this program can support accelerated development and enhanced communication with the FDA. With our newly expanded Phase 1 study continuing to enroll patients and additional data expected later this year, we remain focused on advancing our high-precision approach to deliver an important new treatment to patients with blood cancer.”  

 

The RMAT application included data from the ongoing Phase 1 clinical trial (NCT03802695) evaluating Orca-Q for the treatment of hematologic malignancies across six treatment cohorts, including in patients with haploidentical donors, and in patients receiving non-myeloablative or reduced intensity conditioning. Clinical findings demonstrated durability and encouraging outcomes across key metrics, including overall survival, acute and chronic graft versus host disease (GVHD) and non-relapse mortality. Enrollment is ongoing, with new data expected to be presented at upcoming scientific congresses. Additional details can be found on www.ClinicalTrials.gov.  

 

RMAT designation is a specialized FDA program created to accelerate the development and review of promising new therapies, including cell therapies, intended to treat serious or life-threatening conditions. To qualify, a therapy must demonstrate preliminary clinical evidence suggesting the potential to address unmet medical needs. This designation provides important opportunities during the drug development process, including increased FDA guidance and eligibility for priority and rolling reviews, as well as accelerated approval pathways. By streamlining these regulatory milestones, the program aims to bring transformative innovations to patients more quickly. 

About Orca-Q

Orca-Q is Orca Bio’s second-generation investigational allogeneic T-cell immunotherapy being evaluated 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

media@orcabio.com

Investor Relations

Joshua Murray

ir@orcabio.com

Orca Bio to Present at the 42nd Annual J.P. Morgan Healthcare Conference

Press release

Menlo Park, CA

January 3, 2024

Orca Bio to Present at the 42nd Annual J.P. Morgan Healthcare Conference

MENLO PARK, CA, Jan. 3 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 that Ivan Dimov, Ph.D., Orca Bio’s co-founder and chief executive officer, will present at the 42nd Annual J.P. Morgan Healthcare Conference.

The company presentation will take place on Monday, January 8, 2024, at 2:00PM PST at the Westin St. Francis in the Golden Gate Room on the 32nd floor.

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

media@orcabio.com

Investor Relations

Joshua Murray

ir@orcabio.com

Orca Bio Announces FDA Review Extension of BLA for Orca-T for the Treatment of Hematologic Malignancies

Press release

Menlo Park, CA

April 1, 2026

Orca Bio Announces FDA Review Extension of BLA for Orca-T® for the Treatment of Hematologic Malignancies 

New PDUFA Target Action Date of July 6, 2026 allows FDA additional time to complete its review 

MENLO PARK, CA, April 1, 2026  Orca Bio, a late-stage biotechnology company committed to transforming the lives of patients through high-precision cell therapy, today announced that the U.S. Food and Drug Administration (FDA) has extended the review timeline of its Biologics License Application (BLA) for Orca-T for the treatment of patients with hematologic malignancies. The new Prescription Drug User Fee Act (PDUFA) target action date is July 6, 2026. 

 

The extension follows Orca Bio's recent submission of updated chemistry, manufacturing and controls (CMC) information in response to requests from the FDA as part of the standard review process. The FDA classified the submission as a Major Amendment (MA) to the BLA, which extends the review by three months. Importantly, the FDA has not requested any additional clinical data. Orca Bio believes that the updated information submitted in the amendment does not affect the benefit-risk conclusions of the BLA. 

 

"We appreciate the frequent engagement with the FDA throughout the review process,” said Nate Fernhoff, co-founder and chief executive officer at Orca Bio. “Our continued focus is on preparing for the potential approval and commercial launch of Orca-T. We remain committed to working with the Agency, physicians and the broader blood cancer community to deliver this important therapy to patients with hematologic malignancies as quickly as possible.”  

 

The BLA for Orca-T was granted Priority Review by the FDA and Orca-T previously received Regenerative Medicine Advanced Therapy (RMAT) and Orphan Drug Designation. 

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 hematopoietic stem cells, regulatory T-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).  

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

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