Hematology Video Recap—December 15, 2025

This biweekly hematology update covers major regulatory decisions, late-stage clinical trial readouts, and next-generation cell therapy advances shaping treatment strategies across blood cancers and rare hematologic disorders.

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Week 1–7 December 2025
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Transcript

Welcome to the latest edition of Hematology Updates, covering breakthroughs in the past two weeks. Brought to you by LucidQuest.

Lilly reported BRUIN CLL-314, which randomized 662 BTKi-naïve CLL or SLL patients to pirtobrutinib versus ibrutinib with a data cutoff of 10 Jun 2025, meeting non-inferiority on overall response rate in the intent-to-treat population, showing a numerically higher ORR for pirtobrutinib, and an immature progression-free survival trend favoring pirtobrutinib.

Disc Medicine’s open-label Phase 2 RALLY-MF enrolled 47 patients with myelofibrosis and anemia who received DISC-0974 50 mg subcutaneously every four weeks for up to six doses, yielding consistent hepcidin reductions, anemia responses across non–transfusion-dependent and transfusion-dependent cohorts, and transfusion independence in a subset.

The FDA approved Breyanzi for adults with relapsed or refractory marginal zone lymphoma after at least two prior lines of therapy, based on the single-arm TRANSCEND FL MZL cohort, with safety consistent with prior indications.

In multiple myeloma, early DURGA-1 Phase 1b Phase 2 data for dual-target FasTCAR AZD0120 showed a 96 percent overall response rate across two dose levels with a median follow-up of 3.9 months, deepening responses, and manufacturing timelines compatible with outpatient administration, alongside manageable cytokine release syndrome and limited neurotoxicity.

Kura Oncology and Kyowa Kirin reported that KOMZIFTI, ziftomenib, combined with venetoclax and azacitidine in the KOMET-007 Phase 1a Phase 1b trial presented at ASH 2025, produced high composite complete response rates with measurable residual disease negativity in a subset of newly diagnosed, chemo-ineligible NPM1-mutated AML, and encouraging overall response rates in relapsed or refractory NPM1-mutated and KMT2A-rearranged cohorts, with tolerability aligned to venetoclax and azacitidine.

Senti Bio received RMAT designation for the allogeneic CAR-NK therapy SENTI-202 in relapsed or refractory AML, supported by Phase 1 data in CD33 and or FLT3-positive malignancies showing early overall and complete response rates with durability at the recommended Phase 2 dose, enabling enhanced FDA interaction and expedited pathways.

Bristol Myers Squibb announced FDA priority review for Opdivo plus AVD in previously untreated stage III–IV classical Hodgkin lymphoma for patients aged 12 and older, supported by the Phase 3 SWOG S1826 trial, with a PDUFA target date of 08 Apr 2026.

In China, the NMPA approved Qfitlia, fitusiran, for prophylaxis in hemophilia A and B, and Cablivi, caplacizumab, for acquired or immune-mediated thrombotic thrombocytopenic purpura, offering low-frequency subcutaneous dosing and a targeted option alongside plasma exchange and immunosuppression.

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Why it matters

BTK landscape: First covalent vs non-covalent BTK head-to-head in CLL may reset frontline dynamics [1].
Anemia in MF: Non-erythropoietic, iron-mobilizing approach shows cross-subgroup signal [2].
Cell therapy breadth: Breyanzi adds a fifth B-cell indication, expanding eligible population [3].
Faster CAR manufacturing: FasTCAR timelines could ease access bottlenecks in myeloma if durability holds [4].
Menin combinations: Ziftomenib triplet activity supports ongoing registrational strategy [5].
Regulatory acceleration: RMAT could shorten path for an allogeneic CAR-NK in AML [6].
Frontline cHL: Checkpoint + AVD seeks to shift standard of care pending approval [7].
China access: Dual approvals expand rare-heme options within a large patient base [8].

🗓️ Explore weekly details and sources

Week 1–7 December 2025
Week 8–14 December 2025

📚 See the full Hematology archive on our research hub page.

FAQ

What did BRUIN CLL-314 show for pirtobrutinib vs ibrutinib?

Non-inferiority on ORR in ITT with numerically higher ORR for pirtobrutinib, and immature PFS trending in its favor across populations [1]. May inform use earlier in CLL/SLL pending full PFS data.

How broad were the MF anemia responses with DISC-0974?

Responses were observed regardless of transfusion status or background JAK inhibitor use, with hepcidin reductions and improved fatigue reported in the open-label Phase 2 study [2].

What supports Breyanzi’s new MZL approval?

Single-arm TRANSCEND FL MZL cohort showed high ORR assessed by IRC in 3L+ patients, with a safety profile consistent with prior indications [3].

How mature are the AZD0120 myeloma data?

Early Phase 1b/2 results with median 3.9-month follow-up showed 96% ORR and high CR/sCR, with manageable CRS and limited neurotoxicity, enabling outpatient use in some patients [4].

What is the case for ziftomenib + ven/aza in AML?

In ASH 2025 presentations, newly diagnosed NPM1-m had high CRc with MRD negativity in a subset; R/R NPM1-m and KMT2A-r cohorts showed directional ORR benefits, with tolerability aligned to ven/aza [5].

What does RMAT mean for SENTI-202?

RMAT reflects preliminary clinical benefit potential and enables closer FDA guidance, rolling review eligibility, and expedited interactions for the off-the-shelf CAR-NK in R/R AML [6].

What exactly is under FDA priority review for Opdivo?

The sBLA for Opdivo + AVD in previously untreated stage III–IV cHL for patients 12+ has a PDUFA goal date of 08 Apr 2026 [7].

What did China approve for Sanofi?

NMPA approved Qfitlia (fitusiran) for prophylaxis in hemophilia A/B and Cablivi (caplacizumab) for aTTP/iTTP, expanding rare-heme options with low-frequency dosing and targeted microthrombi inhibition [8].

Entities / Keywords

Pirtobrutinib (Jaypirca); Ibrutinib (Imbruvica); CLL/SLL; BRUIN CLL-314; DISC-0974; Myelofibrosis anemia; Breyanzi (lisocabtagene maraleucel); Marginal zone lymphoma; AZD0120; FasTCAR; Multiple myeloma; KOMZIFTI (ziftomenib); Venetoclax; Azacitidine; NPM1-m; KMT2A-r; Senti Bio; SENTI-202; CAR-NK; RMAT; Opdivo (nivolumab); AVD; Classical Hodgkin lymphoma; Qfitlia (fitusiran); Cablivi (caplacizumab); NMPA China.