Press release

Menlo Park, CA

October 14, 2020

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, 2020Orca Bio, a clinical-stage biotechnology company, today announced that the U.S. 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 U.S. If a therapy with ODD receives the first FDA approval for the designated condition, it is eligible for 7-year market exclusivity.

"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.” (Ivan Dimov, Ph.D.)

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, Ph.D., Chief Executive Officer and Co-Founder 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.

About Orca Bio

Orca Bio is a clinical-stage biotechnology company developing a pipeline of high-precision allogeneic cell therapy products. Orca Bio’s products are designed with the goal of safely and effectively replacing a patient’s diseased blood and immune system with a healthy one. The company’s proprietary therapeutic and manufacturing platforms are exclusively licensed from Stanford University. For more information, visit www.orcabio.com. Follow Orca Bio on Twitter: @orcabio.

Bitnami