Orca Bio has jumped into the commercial market with an FDA approval for Tregzi, a first-of-its-kind engineered cell therapy for blood cancer patients requiring transplants.
Tuesday, the FDA approved the allogeneic regulatory T cell (Treg)-based immunotherapy, also known as Orca-T, for use as an alternative stem cell transplant to improve survival free of chronic graft versus host disease (GVHD) in adults with hematological malignancies.
At its core, Tregzi is a re-engineered hematopoietic stem cell transplant (HSCT) graft. Rather than infusing a mix of stem and immune cells from a matched healthy donor directly into a patient, Tregzi reformulates the blood material to include purified Tregs to suppress GVHD, a common and often debilitating complication of transplants.
It also leverages hematopoietic stem and progenitor cells that help patients rebuild their immune system to fight cancer, followed by infusion of conventional T cells to wipe out any residual cancer cells and accelerate recovery against infections.
The FDA’s approval for Tregzi is “a significant milestone for the field, for the company, [and] for leukemia patients,” Orca Bio co-founder and CEO, Nate Fernhoff, Ph.D., said in an interview with Fierce Pharma.
“Historically, a stem cell transplant for these leukemia patients can be curative, but that cure comes with the compromise of complications,” Fernhoff said. “With Tregzi, we’ve now shown that we can reduce that trade-off and really offer a more fully restorative treatment to the blood cancer patients.”
The FDA approval is based on the phase 3 Precision-T trial, which randomized 187 patients with acute myeloid leukemia, acute lymphoblastic leukemia, myelodysplastic syndrome and mixed-phenotype acute leukemia to receive either Tregzi or unmanipulated allograft. All patients also received additional GVHD prophylaxis; for Tregzi, it was single-agent tacrolimus; for control, tacrolimus and methotrexate were used.
At one year, 78% of patients who received Tregzi were alive and free from chronic GVHD, compared to 38% with regular transplants, translating into a 74% improvement in chronic GVHD-free survival.
For the incidence of moderate-to-severe chronic GVHD alone, the rate was 13% and 44% for Tregzi and control, respectively, after one year. And overall survival was 94% with the Orca drug and 83% with unedited allograft.
Tuesday’s approval comes after a nearly three-month extension from the FDA’s original target decision date. The agency took additional time after asking for extra manufacturing-related data from Orca.
“Production of each cell therapy is very unique, and so each one requires bi-directional education campaign between the FDA and the sponsor, especially when it comes to the manufacturing process,” Fernhoff said.
Recognizing the industry adage that in cell therapies, “the process is the product,” Orca is now focused on delivering Tregzi reliably to patients. While production of Tregzi is for now concentrated at a 100,000-square-foot facility in Sacramento, California, Orca is committed to maintaining a vein-to-vein timeline between collecting donor cells and having the final product available at the hospital for infusion within 72 hours.
“There’s a tremendous amount of planning and coordination,” Fernhoff said. “We have dedicated teams set up to do this. And importantly, we have now produced […] over 500 products for patients, and these are products with donors from all around the country and patients all around the country, and we’re able to meet this time window time and time again.”
Tregzi avoids the manufacturing failure issue that has plagued existing autologous CAR-T therapies, which depend on the fitness of a patient’s own T cells. By comparison, Tregzi uses healthy donor cells that are more reliable. As a result, Fernhoff said Orca expects to keep manufacturing failure rate at single digit to low single digit.
Orca is launching Tregzi at “a handful of” treatment centers and has “strategically planned” a production ramp and an onboarding ramp to have around 25 centers by the end of the year, Fernhoff said.
Further down the line, the California company a few days ago announced a new manufacturing facility in Princeton, New Jersey, to further reduce vein-to-vein time across the country and meet additional demand.
Orca is charging Tregzi at a wholesale acquisition cost of $428,000.
“We believe this price reflects the clinical and economic value Tregzi delivers, not just as a one-time therapy, but as an intervention that may substantially reduce the serious, costly downstream complications of allogeneic transplant, particularly chronic GVHD,” the company said in a statement to Fierce.
Orca has established pathways to reimbursement across both commercial and government programs, and it’s committed to providing support to ensure access for eligible patients, the company added.
With the FDA approval, Orca Bio has become one of very few U.S.-based commercial drugmakers that remains privately held. At the beginning of the year, the company unveiled $250 million in new capital from two recent financing rounds, including a series F that closed in December. Most of those proceeds are dedicated to support Tregzi’s launch.
Now halfway into 2026, the biotech IPO market appears to be opening up with record-breaking listings.
“Over the coming months, we’ll see what else develops there,” Fernhoff said. “But there does seem like there’s a window [for IPOs].”
The chief executive argued that Orca is “well capitalized” for the launch of Tregzi, which is “a huge priority” for the company right now, while calling additional fundraising “a bit of a distraction for the team.”
“We’re going to be really focused on running the launch and establishing ourselves in the market,” he said. “Looking a little bit further down the road, there are additional priorities and prerogatives that the company could undertake with additional financing. But we’re going to make it through launch before we start going after some of them.”