The 2025 meeting of the American Society of Hematology features some of the newest developments in blood cancers and rare diseases.
The 2025 annual meeting of the American Society of Hematology (2025) is in full swing—the conference runs from Dec. 6 to 9 in Orlando, Florida—featuring some of the latest and potentially practice-changing developments in blood-related diseases.
Here, BioSpace reviews some of the most interesting and impactful presentations at the meeting so far.
CAR-T Leaders Gilead, J&J/Legend Tout Promising Multiple Myeloma Data
Taking center stage at ASH 2025 are CAR-T therapies, a burgeoning class of treatments with difficult business prospects.
One company at the leading edge of this modality is Gilead, which has partnered with California’s Arcellx to advance anitocabtagene autoleucel (anito-cel). In a Saturday presentation, the companies provided updated data from the registrational Phase II iMMagine-1 study, touting a 96% overall response rate in treating relapsed or refractory multiple myeloma (RRMM), including a 74% complete response/stringent complete response rate.
Progression-free and overall survival rates at 18 months were 66% and 90%, respectively.
The partners also claimed that anito-cel had a “manageable” toxicity profile in these patients, with no delayed neurotoxicities such as Parkinsonism, cranial nerve palsies and Guillain-Barré syndrome.
“The full iMMagine-1 data presented this weekend at ASH solidify anito-cel’s differentiated profile vs. key competitors,” analysts at BMO Capital Markets said in a Sunday afternoon note, pointing specifically to Johnson & Johnson and Legend Biotech’s Carvykti. These full data, the analysts added, “clear the path to a potential 2026 approval/launch.”
J&J and Legend are also at ASH, with an oral session on Saturday that looked at the long-term survival outcomes of Carvykti-treated patients in the Phase III CARTITUDE-4 study. In the intent-to-treat cohort, standard-risk RRMM patients on Carvykti saw a 71% progression-free survival rate at 30 months, versus 43.2% in comparators on standard of care. Survival remained high in the as-treated analysis and when looking at patients with a specific genetic mutation.
Like anito-cel, Carvykti targets the BCMA protein for the treatment of RMM.
Fulcrum’s Pill Makes Headway into Sickle Cell Disease
Heading into ASH 2025, analysts were excited about Fulcrum Therapeutics and its oral fetal hemoglobin inducer pociredir, being tested for sickle cell disease. In a Dec. 3 note ahead of the meeting, Truist Securities said a readout expected for pociredir is “shaping up to be a large mover for the stock.”
On Saturday, Fulcrum presented findings from one cohort of its Phase Ib PIONEER study, touting a mean 8.6% increase in fetal hemoglobin over 12 weeks. 44% of patients reached hemoglobin levels above 20%. Lactate dehydrogenase, a marker of the destruction of red blood cells, significantly dropped after pociredir treatment.
As for safety, Fulcrum detected five serious adverse events, all of which were consistent with pre-existing conditions associated with sickle cell disease. The company found pociredir’s safety profile to be “favorable” overall.
In a Sunday note after Fulcrum’s presentation, Truist analysts said that pociredir’s performance “more than reassures” and will help the biotech “further establish the early but promising profile of [its] lead asset.”
Shares of Fulcrum are up 63% to $14.53 in pre-market trading Monday.
Fulcrum is gearing up for an FDA meeting in the first half of next year, Truist continued, adding that pociredir has a “viable path to a potential registrational study, sooner rather than later.”
Incyte Pushes Antibody Into Late-Stage Development for Myelofibrosis
Incyte’s showing at ASH was centered around INCA033989, a monoclonal antibody that treats the rare blood marrow and blood cancer myelofibrosis by targeting mutant calreticulin protein (mutCALR), touting “rapid and robust” improvements in spleen volume and anemia.
With these findings, the mutCALR antibody could represent a meaningful step for Incyte as it tries to fill a loss of exclusivity gap left by previous myelofibrosis drug ruxolitinib, analysts at BMO Capital Markets said in a Sunday note.
Two Phase I readouts on Sunday showed that 41.7% of patients treated with INCA033989 achieved an outcome called SVR25, referring to at least a 25% reduction in spleen volume, at 24 weeks. At the same time point, 33.3% hit SVR35. These figures were better in those who had not undergone previous treatment with a JAK inhibitor, reaching 71.4% and 57.1%, respectively.
In addition, INCA033989 treatment improved anemia symptoms in 56% of evaluable treated patients, with 40% showing major anemia response.
With these data, Incyte plans to push its mutCALR antibody into registrational development next year, President Pablo Cagnoni said in a statement.
Calreticulin is a molecule that helps maintain calcium balance in cells and promote proper protein folding. Alternations in calreticulin has been linked with abnormal protein function and the development of myeloproliferative neoplasms.
Disc Presents ‘Differentiated’ Mid-Stage Efficacy for Anti-Anemia Antibody
Also playing in the blood disease arena is Disc Medicine, which is developing the anti-hemojuvelin antibody DISC-0974 for the treatment of anemia related to myelofibrosis.
At ASH, the Massachusetts biotech presented initial data from an ongoing Phase II study touting a major hematologic response of 50% in patients also undergoing JAK inhibitor therapy. Additionally, 71% of patients with low transfusion burden at baseline reached transfusion independence over a 16-week period.
As for safety, Disc reported that its therapeutic antibody was “generally well-tolerated,” with most adverse events deemed unrelated to the drug. The only treatment-related toxicities were diarrhea and urinary tract infections.
Reacting to these data in a Sunday note, analysts at Truist Securities said that “DISC-0974 represents a differentiated and potentially superior treatment option for MF-related anemia compared to currently available therapies.”
Disc will release more data for the antibody in the back half of next year, after which the biotech will have an end-of-Phase II meeting with the FDA, Truist added. Pending the agency’s feedback, a pivotal trial for DISC-0974 could come as soon as the first half of 2027.
