Axatilimab-csfr

Orca Bio has announced that the US FDA extended the review timeline for its Biologics License Application (BLA) for Orca-T, an investigational allogeneic T-cell immunotherapy targeting hematologic malignancies including acute leukemias and myelodysplastic syndromes. The revised PDUFA target action date is July 6, 2026, three months beyond the original deadline, following the FDA’s classification of a recent company submission as a Major Amendment to the BLA. The extension was triggered by Orca Bio’s submission of updated chemistry, manufacturing and controls (CMC) information provided in response to FDA requests during the standard review process, the company said. The agency has not requested additional clinical data, and Orca Bio stated it believes the updated CMC information does not affect the benefit-risk conclusions of the BLA. The filing carries Priority Review designation, and Orca-T has previously received Regenerative Medicine Advanced Therapy (RMAT) and Orphan Drug Designation for the prevention of graft-versus-host disease (GVHD) or death in patients eligible for hematopoietic stem cell transplant (HSCT). Orca-T is composed of highly purified hematopoietic stem cells, regulatory T-cells, and conventional T-cells derived from matched related or unrelated donors. The inclusion of regulatory T-cells is central to the product’s design rationale: by modulating the post-transplant alloreactive immune response, the approach aims to reduce GVHD without eliminating the graft-versus-leukemia effect that makes allogeneic transplantation therapeutically relevant. This positions Orca-T not as an adjunctive drug but as a redefined graft — intended to replace conventional unmanipulated cell products at the transplant step itself. The clinical program supporting the BLA includes the Phase III Precision-T trial (NCT05316701), which enrolled patients with hematologic malignancies and used moderate or severe chronic GVHD-free survival at 12 months as its primary endpoint, assessed by an independent Endpoint Adjudication Committee. An earlier Phase Ib study (NCT04013685) focused on safety endpoints including the incidence of grade 3 or 4 acute GVHD and primary graft failure. The filing enters review at a time when GVHD remains a substantial source of morbidity and non-relapse mortality in the allogeneic transplant setting. Approved prophylactic agents include abatacept, which received FDA approval in 2021 for use in unrelated-donor transplants, while treatment options for established GVHD include ruxolitinib, belumosudil, and axatilimab, the latter approved in 2024 for recurrent or refractory chronic GVHD. Despite these additions, no approved intervention has demonstrated the capacity to simultaneously reduce GVHD and preserve the graft-versus-leukemia effect — the core biological tension that precision graft engineering approaches such as Orca-T are designed to address. Your email address will not be published. Required fields are marked * Comment * Name * Email * Website

Always be in the clinic — those are the “ABCs” of biotech, drug development veteran Briggs Morrison tells Endpoints News. And that’s precisely where Morrison’s Crossbow Therapeutics has been for the past few quarters with its first T cell engager. The company now has plans to test a second cancer drug in patients this summer thanks to a fresh $77 million financing round. That Series B marks the second major financing in the TCE space to be announced Wednesday morning, with Shanghai-based Excalipoint earlier expanding its seed round to $68.7 million. Crossbow formed in 2021 and broke onto the scene in July 2023 with an $80 million Series A . Since then, the Cambridge, MA-based startup has “basically hit everything” that cofounder and corporate development leader Geraldine Paulus laid out in its business plan years ago, CEO Morrison said in an interview. Morrison formed Crossbow after spending eight years in the roles of CEO and head of R&D at Syndax Pharmaceuticals, which developed the medicines Revuforj and Niktimvo. His industry roots run deep, having served as CMO for a stint at AstraZeneca in the early 2010s after building street cred at Pfizer and Merck. By the end of this year, Crossbow should know whether its initial T cell engager works, Morrison said. That drug, CBX-250, is in Phase 1 testing in relapsed and refractory myeloid malignancies such as acute myeloid leukemia, chronic myeloid leukemia, myelodysplastic syndromes and chronic myelomonocytic leukemia. Positive data from the medicine, which goes after the cathepsin G peptide-HLA complex, could help launch the company’s expansion into other areas. Its second asset, CBX-663, recognizes a peptide that comes from telomerase, also in the context of pHLA, Morrison said. It will be tested for multiple hematologic and solid tumors, the company said. Crossbow hopes the two medicines will obliterate tumors and be safe for the rest of the body. (The company’s name traces to the Middle Ages, when the Catholic Church banned crossbows because of their lethality, Morrison previously told Endpoints.) While Crossbow has been meeting its deadlines, getting CBX-663 to an IND filing before this summer was no small feat. “It’s turned out, I’d say, to be harder than we thought” to make the TCR mimetic antibodies, Morrison said. The company’s R&D efforts could lead to more than just T cell engagers down the road. The bulk of the Series B will focus on the two lead assets, but the 38-person startup has some early work to explore potential expansion into areas such as in vivo CAR-T and antibody-drug conjugates. Crossbow’s entry into those other areas depends on whether the company can prove its first clinical-stage medicines have no off-target concerns and are effective at killing bad actors. Morrison will have some well-known names to help him answer those questions. His startup’s investor base includes Taiho Ventures, Arkin Bio Capital, Eli Lilly, Pfizer Ventures, MPM BioImpact, Polaris Partners and others. Editor’s note: This story was updated to correct that Crossbow was founded in 2021, not 2022.