Innovations in Haematology Cell Therapy at ASH 2025
Advancing Haematology Cell Therapy Innovations at ASH 2025
AstraZeneca is significantly advancing haematology cell therapy with its largest-ever presence at the 67th American Society of Hematology (ASH) Annual Meeting and Exposition, held from December 6-9, 2025. The company will present data from 65 abstracts across eight approved and investigational medicines, including 15 oral presentations. The core focus highlights early efficacy and safety results for investigational therapies like surovatamig, a CD19xCD3 T-cell engager, and AZD0120, a BCMA x CD19 chimeric antigen receptor T-cell (CAR T) therapy, in various relapsed/refractory blood cancers such as follicular lymphoma (FL), diffuse large B-cell lymphoma (DLBCL), and multiple myeloma (MM).
This research highlights sustained benefits of established therapies like Calquence (acalabrutinib) in mantle cell lymphoma (MCL) and chronic lymphocytic leukaemia (CLL), and Ultomiris (ravulizumab) in paroxysmal nocturnal haemoglobinuria (PNH) and paediatric hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA), positioning these developments as steps toward redefining haematology care through innovative cell-based and targeted approaches.
Promising Early Signals from Dual-Target Cell Therapies
Astrazeneca is presenting promising initial data from Phase I and Ib/II trials of surovatamig and AZD0120, demonstrating their potential to address unmet needs in relapsed/refractory B-cell malignancies. For instance, updated three-year follow-up results from the Phase I first-in-human trial of surovatamig in R/R FL, presented as Oral Abstract #1005, reveal sustained efficacy with high rates of minimal residual disease (MRD)-negative complete responses, even in patients previously progressed on CD20 T-cell engagers (TCEs) or CD19 CAR T therapies.
Similarly, preliminary results from the DURGA-1 Phase Ib/II study of AZD0120 in R/R MM, featured in Oral Abstract #269, show encouraging safety and efficacy profiles, including deep responses in heavily pretreated patients. These findings are further supported by data from the SYRUS Phase I/II trial (Abstract #3345) for surovatamig in R/R B-cell acute lymphoblastic leukaemia (B-ALL) and investigator-initiated trials in China (Oral Abstract #258) evaluating AZD0120 as first-line therapy in high-risk, newly diagnosed MM, where one-year follow-up indicates durable responses.
Collectively, these results highlight a trend toward dual-targeting strategies in haematology cell therapy, offering improved durability and reduced resistance compared to single-antigen approaches, which could shift the treatment paradigm for aggressive hematologic cancers.
Sustained Outcomes in BTK and Complement Inhibition
Building on early-stage innovations, Astrazeneca is also presenting robust long-term data for Calquence and Ultomiris, reinforcing their roles in improving patient outcomes across haematologic indications. In the ECHO Phase III trial (Oral Abstract #885), after 50 months of follow-up, Calquence combined with bendamustine and rituximab in first-line MCL treatment demonstrated delayed time to third-line therapy, with exploratory analyses from the TrAVeRse Phase II trial (Oral Abstract #884) showing preliminary efficacy in treatment-naïve MCL when paired with venetoclax and rituximab.
For Ultomiris, the ALXN1210-TMA-314 Phase III trial (Oral Abstract #1052) reports clinically meaningful overall survival and organ function improvements in paediatric HSCT-TMA patients, while the ALPHA Phase III sub-analysis (Oral Abstract #949) of Voydeya (danicopan) as an add-on to Ultomiris or Soliris in PNH with extravascular haemolysis confirms positive outcomes, particularly in advanced-age subgroups. Real-world evidence from international registries (Poster Abstracts #6238 and #4458) further validates Ultomiris’s safety in pregnant patients and those with advanced PNH, with no new signals of concern.
These outcomes highlight the value of extended follow-up in establishing real-world applicability, particularly for rare conditions, and align with broader trends in precision haematology where combination regimens enhance progression-free survival.
Economic Value of Next-Gen Haematology Cell Therapy
The durable responses from surovatamig and AZD0120 could justify premium pricing in market access negotiations for R/R blood cancers, where current CAR T options like those targeting BCMA alone often require costly bridging therapies; however, their dual-targeting design may reduce relapse rates, potentially lowering long-term hospitalization costs and improving reimbursement under frameworks like the U.S. Medicare system.
Similarly, extended data for Calquence in MCL and CLL (e.g., delayed third-line treatment in ECHO) support fixed-duration regimens that minimize chronic therapy expenses compared to indefinite BTK inhibitor use. For Ultomiris and Voydeya in PNH and HSCT-TMA, paediatric and real-world outcomes enhance evidence for orphan drug designations, facilitating faster reimbursement pathways while addressing equity in rare disease access.
