Orca Bio Presents Positive Data Reinforcing Clinical Profile of Orca-T and Orca-Q at 2023 Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR

Press release

Menlo Park, CA

February 16, 2023

    Orca Bio Presents Positive Data Reinforcing Clinical Profile of Orca-T and Orca-Q at 2023 Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR

Three oral presentations showcase clinical evidence for Orca Bio’s promising pipeline of early- and late-stage high-precision cell therapies

Orca-T demonstrated high relapse-free survival in recipients with fully matched donors and early potential in patients with mismatched donors

Orca-Q showed improved graft-versus-host disease-free, relapse-free survival in patients with haploidentical donors

MENLO PARK, CA, February 16, 2023 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 data from the company’s investigational high-precision cell therapy programs, Orca-T and Orca-Q, were presented in three oral presentations at the 2023 Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR.

Positive data on Orca-T was presented from the single-center Phase 2 and multi-center Phase 1b trials of patients with acute myeloid leukemia (AML), acute lymphocytic leukemia (ALL), myelodysplastic syndromes (MDS) and other hematological malignancies. Outcomes with Orca-T appeared to be further enhanced in patients who received a conditioning regimen of busulfan, fludarabine and thiotepa (BFT). At 12 months, the 71 patients in the Orca-T BFT subgroup reported no non-relapse mortality (0%), and high rates of relapse-free survival (87%), graft-versus-host disease-free, relapse-free survival (GRFS) (81%) and overall survival (94%). These data were previously presented at the 64th American Society of Hematology (ASH) Annual Meeting in December 2022.

In a second oral presentation, new data highlighted how expanding the donor pool may help address disparities in access to transplant. Today, approximately 70% of blood cancer patients do not have access to a fully matched related donor, and the likelihood of a patient finding a matched unrelated donor in the U.S. is as low as 29% for some ethnic backgrounds. While the use of human leukocyte antigen (HLA) mismatched donors has increased access to treatment for these patients undergoing allogeneic hematopoietic stem cell transplant (alloHSCT), clinical outcomes have also been associated with significantly worse overall survival, disease-free survival, treatment-related mortality and acute graft versus host disease (aGvHD). In a small cohort (n=8), Orca-T demonstrated promising results in patients with HLA mismatched (7/8) donors, including no cases of severe GvHD or non-relapse mortality and 100% overall survival to date. While this early data is encouraging, more studies of Orca-T in this patient population are ongoing and will provide additional clinical data in this setting.

"As we continue to enroll patients in our pivotal Phase 3 trial of Orca-T, we are extremely encouraged by its continued potential to improve relapse-free and overall survival rates,” said Ivan Dimov, Ph.D., co-founder and chief executive officer of Orca Bio. “Beyond its promise to enhance outcomes and reduce deadly, transplant-related risks, this early evidence that Orca-T may improve clinical outcomes in patients who can’t find a fully matched donor is an important step toward our ultimate goal of offering a safe and effective treatment to all patients who may benefit.”

Orca Bio also presented findings from a Phase 1 multi-center trial of its second investigational cell therapy, Orca-Q, showing that patients with haploidentical – or half-matched – allogeneic hematopoietic stem cell donors, experienced no moderate-to-severe chronic GvHD (0%) and an improved GRFS rate (75%). Orca-Q is a proprietary composition of enriched CD34+ stem cells combined with specific T cell subsets derived from haploidentical donors. Unlike standard of care haploidentical alloHSCT, Orca-Q does not require post-transplant cyclophosphamide (PTCy). These data were also previously presented at the ASH Annual Meeting.

“A haploidentical stem cell transplant is currently the only curative treatment option available for the majority of blood cancer patients who lack a matched donor, but providers often find themselves balancing the risk of relapse and the risk of debilitating side effects like GvHD, infections and other toxicities,” said Samer Srour, M.D., Department of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer Center. “These interim findings show patients treated with Orca-Q experienced improved outcomes with significantly fewer transplant-related toxicities, suggesting this novel cell therapy could help to address this critical unmet medical need.”

Links to the abstracts follow:

Oral Presentation Title: Precision-Engineered Cell Therapy Orca-T Demonstrates High Relapse-Free Survival at 1 Year While Reducing Graft-Versus-Host Disease and Toxicity

Oral Presentation Title: Orca-T, a High-Precision Cell Therapy, for the Treatment of Hematologic Malignancies in Patients with 7/8 Mismatch Donors

Oral Presentation Title: Orca-Q Demonstrates Favorable GvHD-and-Relapse-Free Survival with Haploidentical Donors without Post-Transplant Cyclophosphamide

Poster Presentation Title: Estimating the Current and Future Costs and Health Outcomes of Patients Undergoing Allogeneic Hematopoietic Stem Cell Transplantation (Allo-HCT)


About Orca-T

Orca-T is an investigational high-precision allogeneic cell 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 hematological malignancies.

About Orca-Q

Orca-Q is Orca Bio’s second investigational high-precision allogeneic cell therapy to enter clinical development for hematological malignancies. Orca-Q is a proprietary composition of enriched CD34+ stem cells combined with specific T cell subsets derived from haploidentical donors that are purified through Orca Bio’s high-precision platform. Orca-Q is a therapy that has the potential to improve patient outcomes and reduce the risks of graft versus host disease, without the use of post-transplant cyclophosphamide (PTCy) in patients for whom a full human leukocyte antigen (HLA) match cannot be found.

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

Kelsey Grossman

media@orcabio.com

Investor Relations

Joshua Murray

ir@orcabio.com


Orca Bio to Present New Clinical Data on Orca-T at the 63rd American Society of Hematology Annual Meeting

Oral and poster presentations will include one-year follow-up data from an expanded group of patients and an analysis of treatment outcomes in patients with myelofibrosis

MENLO PARK, CA, November 4, 2021 – Orca Bio, a clinical-stage biotechnology company developing purified, high-precision cell therapies for cancer, autoimmune and genetic blood disorders, today announced that new data will be presented from a single-center Phase 2 trial and a multi-center Phase 1b trial at the American Society of Hematology (ASH) Annual Meeting. Specifically, pooled data from patients treated with Orca-T, an investigational high-precision cell therapy product being studied in patients with hematologic malignancies, will be shared in an oral presentation. Additionally, results of Orca-T in a subset of patients with myelofibrosis from those same trials will be shared in a poster presentation. “Our clinical experience with Orca-T has grown significantly over the past year and we are pleased to present data on an expanded group of patients who now have one year of follow-up with Orca-T,” said Ivan Dimov, Ph.D., cofounder and chief executive officer of Orca Bio. “We look forward to presenting these important findings at ASH as we continue to work to deliver on our promise of maximizing patient survival while minimizing the compromise of toxicities to enable better outcomes.” The ASH abstracts are now available at www.hematology.org. Details of the Orca Bio presentations follow: Oral Presentation: Orca-T Results in High GVHD-Free and Relapse-Free Survival Following Myeloablative Conditioning for Hematological Malignancies: Results of a Single Center Phase 2 and a Multicenter Phase 1b Study Abstract Number: 98 Session: 722. Allogeneic Transplantation: Acute and Chronic GVHD, Immune Reconstitution: Prevention of graft vs. host disease after allogeneic hematopoietic cell transplantation Presentation Date: Saturday, December 11, 2021, at 9:45 AM EST Location: Georgia World Congress Center, B302-B303 Poster Presentation: Outcomes for Myelofibrosis Patients Following Myeloablative Allogeneic Stem Cell Transplantation Using the Orca-T Graft from HLA-Matched Related and Unrelated Donors Abstract Number: 1819 Session: 722. Allogeneic Transplantation: Acute and Chronic GVHD, Immune Reconstitution: Poster I Presentation Date: Saturday, December 11, 2021, at 5:30 PM – 7:30 PM EST Location: Georgia World Congress Center, Hall B5
Orca Bio Announces Updated Orca-T Clinical Data Presented at the 2021 TCT (Transplantation & Cellular Therapy) Meetings of the American Society of Transplantation and Cellular Therapy (ASTCT) and Center for International Blood & Marrow Transplant Research (CIBMTR)Orca-T (n=50) with single-agent graft versus host disease (GvHD) prophylaxis when compared with a cohort of patients who received a standard-of-care (SOC) hematopoietic stem cell transplant (HSCT) (n=144) and a 2-agent GvHD prophylaxis demonstrated: MENLO PARK, CA, February 11, 2021 – Orca Bio, a clinical-stage biotechnology company developing high-precision investigational cell therapies, today announced updated clinical data from its first-generation investigational therapy Orca-T, which was presented at the 2021 TCT Meetings of ASTCT and CIBMTR by Everett Meyer, MD, PhD, Assistant Professor of Medicine, Blood and Marrow Transplantation at Stanford University. Results from multicenter (NCT04013685) and single-center (NCT01660607) phase I/II trials showed that patients receiving Orca-T (n=50) with single-agent GvHD prophylaxis demonstrated improved 1-year GvHD-free and Relapse-Free Survival (GRFS) (75% vs 31%) when compared with a cohort of patients who received a standard-of-care (SOC) hematopoietic stem cell transplant (HSCT) (n=144) and 2-agent GvHD prophylaxis. As of the data cut-off, Orca-T plus single-agent GvHD prophylaxis was well-tolerated in the clinical trial, with a decreased incidence of grade 2+ acute GvHD (at 100 days: 10% vs 30%) and chronic GvHD (at 365 days: 3% vs 46%). The high-precision investigational cellular products continue to be successfully manufactured and scaled in a central facility and were delivered to patients at multiple clinical sites with a vein-to-vein time of less than 72 hours. The presentation can be viewed on the Orca Bio website. “Orca-T continues its groundbreaking performance in our phase 1b clinical trial where it is under investigation to improve survival by preventing transplant-related mortality, aGvHD, and cGvHD,” said Nate Fernhoff, PhD, Chief Scientific Officer at Orca Bio.
Orca Bio to Present New Clinical Data on Orca-T at the European Hematology Association 2022 Congress

MENLO PARK, CA, May 12, 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 new clinical data on its lead investigational high-precision cell therapy, Orca-T, will be shared in an oral presentation at the hybrid European Hematology Association (EHA) Congress from June 9-17, 2022, in Vienna, Austria.

Oral Session: Clinical Studies in Transplantation

Title: Orca-T, an Engineered Allograft, Results in High GVHD-Free and Relapse-Free Survival Following Myeloablative Conditioning for Hematological Malignancies

Abstract Number: S237

Date and Time: Sunday, June 12, at 11:30–12:45 CEST / 5:30AM–6:45AM EDT

Location: Hall Stolz 1-2

The presentation will highlight results from the single-center Phase 2 and multi-center Phase 1b trials of Orca-T in patients with acute myeloid leukemia (AML), acute lymphocytic leukemia (ALL) and myelodysplastic syndromes (MDS). Data included in the abstract will be updated at the time of presentation.

The oral session will take place in Vienna and will be livestreamed on the EHA Congress platform.

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

Positive Clinical Results from More than 100 Patients Treated with Orca Bio’s Lead Investigational High Precision Cell Therapy Presented at 63rd ASH Annual Meeting

Pooled study results showed significantly higher graft-versus-host disease-free, relapse-free survival rates and improved overall survival rates with Orca-T compared to standard of care

Data from Phase 1b/2 trials include 109 patients with acute myeloid leukemia, acute lymphocytic leukemia, myelodysplastic syndromes, myelofibrosis and other hematological malignancies

Data from eight patients with myelofibrosis who received Orca-T showed regression of marrow fibrosis

Orca-T was manufactured reliably and delivered with vein-to-vein times of 72 hours or less

Company plans to commence Phase 3 study in early 2022

MENLO PARK, CA, Dec. 11, 2021 – Orca Bio, a clinical-stage biotechnology company developing purified, high-precision cell therapies for the treatment of cancer, autoimmune diseases and genetic blood disorders, today announced positive clinical data on its lead investigational cell therapy, Orca-T, were presented at the American Society of Hematology (ASH) Annual Meeting. The data presented on 109 patients with at least 90 days of follow-up in combined data from the Phase 1b and Phase 2 trials showed significantly higher graft-versus-host disease-free, relapse-free survival (GRFS) rates compared to patients who received standard of care. “The lower rates of graft versus host disease and improved relapse-free survival suggest Orca-T has the potential to become a safer and more effective treatment option for patients living with serious blood cancers,” said Ivan Dimov, Ph.D., cofounder and chief executive officer of Orca Bio. “We are further encouraged by the validation of our platform as we advance Orca-T into our pivotal Phase 3 trial, a significant step toward potential commercialization and, most importantly, to helping more patients in need.” Findings presented today in an oral presentation included pooled results from the single-center Phase 2 and multi-center Phase 1b trials from 109 patients with acute myeloid leukemia, acute lymphocytic leukemia, myelodysplastic syndromes, myelofibrosis and other hematological malignancies. Median follow-up for these patients was 617 days (single-center) and 209 days (multi-center). For comparison purposes, a contemporary, propensity-matched cohort of 95 matched patients undergoing standard of care allogeneic hematopoietic stem cell transplant (alloHSCT) served as the standard of care cohort (“SOC cohort”). Results demonstrated that: In a poster presentation at ASH, Orca Bio also shared an analysis of treatment impact in patients with myelofibrosis. The analysis compared eight patients with myelofibrosis who were treated with Orca-T with six patients who underwent standard of care (SOC) alloHSCT. Regression of marrow fibrosis to myelofibrosis grade 0 or 1 was observed by Day 100 post-transplant in all eight Orca-T recipients, but was observed in only one of the six SOC patients. Additionally, Orca-T recipients had lower incidence of acute and chronic GvHD. Along with the positive Phase 1b/2 results, Orca Bio announced that it has completed a successful end of Phase 2 meeting with the U.S. Food and Drug Administration and plans to commence a Phase 3 trial for Orca-T in early 2022. The full presentation is available on www.orcabio.com.
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.