The 2025 annual meeting of the American Society of Hematology (ASH) is underway in Orlando, Florida, from December 6 to 9. The event highlights recent advancements in blood-related diseases.
BioSpace has summarized some notable presentations from the conference so far.
CAR-T therapies are a major focus at ASH 2025. Gilead and its partner Arcellx presented updated results for their CAR-T candidate anitocabtagene autoleucel (anito-cel), which is being tested in patients with relapsed or refractory multiple myeloma. In the Phase II iMMagine-1 study, anito-cel achieved a 96% overall response rate and a 74% complete or stringent complete response rate. At 18 months, progression-free survival was reported at 66%, while overall survival was at 90%. The companies said that anito-cel showed a “manageable” toxicity profile without delayed neurotoxicities such as Parkinsonism, cranial nerve palsies, or Guillain-Barré syndrome.
BMO Capital Markets analysts commented on these findings: “The full iMMagine-1 data presented this weekend at ASH solidify anito-cel’s differentiated profile vs. key competitors,” specifically mentioning Johnson & Johnson and Legend Biotech’s Carvykti therapy. The analysts added that these results “clear the path to a potential 2026 approval/launch.”
Johnson & Johnson and Legend Biotech also presented long-term outcomes for Carvykti in the Phase III CARTITUDE-4 study. Among standard-risk patients with relapsed or refractory multiple myeloma who received Carvykti, progression-free survival at 30 months was reported at 71%, compared to 43.2% among those on standard care.
Both anito-cel and Carvykti target the BCMA protein in treating multiple myeloma.
Fulcrum Therapeutics introduced new data for its oral fetal hemoglobin inducer pociredir in sickle cell disease during the conference. In one cohort of its Phase Ib PIONEER study, Fulcrum observed an average increase of 8.6% in fetal hemoglobin over twelve weeks; forty-four percent of patients reached hemoglobin levels above twenty percent. Lactate dehydrogenase levels—a marker for red blood cell destruction—decreased significantly after treatment with pociredir.
Fulcrum reported five serious adverse events but noted all were consistent with pre-existing conditions related to sickle cell disease and assessed pociredir’s safety profile as “favorable.” Truist Securities analysts wrote after Fulcrum’s presentation that pociredir’s performance “more than reassures” and will help establish it as a promising asset for Fulcrum Therapeutics. Following this update, Fulcrum shares rose by sixty-three percent to $14.53 in pre-market trading on Monday.
Fulcrum is preparing for a meeting with the FDA next year regarding possible advancement toward registrational studies for pociredir.
Incyte featured its monoclonal antibody INCA033989 for myelofibrosis treatment targeting mutant calreticulin protein (mutCALR). According to two Phase I studies, forty-one point seven percent of patients achieved SVR25—defined as at least a twenty-five percent reduction in spleen volume—after twenty-four weeks; thirty-three point three percent achieved SVR35. Outcomes were better among those who had not previously received JAK inhibitor therapy, reaching seventy-one point four percent (SVR25) and fifty-seven point one percent (SVR35). Anemia symptoms improved in fifty-six percent of treated patients; forty percent showed major anemia responses.
With these findings, Incyte plans to advance mutCALR antibody into registrational development next year, according to President Pablo Cagnoni.
Disc Medicine shared initial data from its ongoing Phase II trial evaluating DISC-0974—an anti-hemojuvelin antibody—for anemia associated with myelofibrosis. Half of the patients receiving JAK inhibitor therapy experienced major hematologic responses; seventy-one percent of those with low transfusion burden became transfusion independent over sixteen weeks.
Disc Medicine described DISC-0974 as “generally well-tolerated,” noting that most adverse events were unrelated to treatment except diarrhea and urinary tract infections. Truist Securities analysts commented: “DISC-0974 represents a differentiated and potentially superior treatment option for MF-related anemia compared to currently available therapies.” Additional data are expected later next year before discussions with regulators about further clinical trials.
