This week’s Hematology update spotlights key developments, regulatory milestones, and the latest progress shaping the biopharma landscape.
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In Today’s Newsletter
🧬 TECVAYLI + DARZALEX SC EMA submission in earlier RRMM [1] [EU • 06 Jan 2026]
Context: Phase 3 MajesTEC-3 in RRMM after 1–3 prior lines compared Tec-Dara vs DPd/DVd (n=587).
Key point: Statistically significant PFS and OS benefit, HR for progression or death 0.17; safety showed high Grade 3/4 AEs in both arms with more infections on combo.
Implication: May influence prescriber choice and payer reviews pending full data.
🧪 First US multi-center IND for in vivo BCMA CAR-T, KLN-1010 [2] [US • 07 Jan 2026]
Context: Enables US expansion of Phase 1 inMMyCAR in RRMM; Australia enrolling.
Key point: Single-infusion, no lymphodepletion manufacturing; initial 4 pts in AU reported MRD-negative at 1 month (durability to 3 months in longest-followed).
Implication: Signals pipeline investment and modality expansion.
🌍 Revuforj (revumenib) managed access beyond US [3] [07 Jan 2026]
Context: Syndax + WODA program spans parts of Eurasia, CEE, Israel, Middle East/Turkey, LATAM, Africa.
Key point: Enables named-patient access where permitted; US approvals cover KMT2Ar R/R acute leukemia and R/R AML with susceptible NPM1 mutation.
Implication: May expand screening, initiation, and follow-up at scale.
🧫 AB8939 + venetoclax: 4/4 early responses in high-risk R/R AML [4] [EU • 07 Jan 2026]
https://www.ab-science.com/wp-content/uploads/2026/01/CP-AB8939-JAN-26-VENG-VF.pdf
Context: Phase 1 combo stage; current dose AB8939 21.3 mg/m² + venetoclax for 14 days.
Key point: Fourth consecutive responder included TP53-mutated, complex karyotype; combination well-tolerated without DLTs in reported cases.
Implication: May influence prescriber choice and payer reviews pending full data.
🧪 CER-1236 AML update adds MDS/MF cohorts [5] [US • 07 Jan 2026]
https://www.cero.bio/press-release?storyId=5313479179734752
Context: CertainT-1 Phase 1/1b completed DLT window in first cohort.
Key point: Observed T-cell expansion with no CRS/ICANS; in one MDS→AML patient, 61-day platelet transfusion-free interval noted.
Implication: Could inform practice and payer discussions; interpretation depends on study design and confounding control.
📈 ELVN-001 shows encouraging MMR in pretreated CML [6] [US • 08 Jan 2026]
Context: Phase 1b ENABLE; 60 pts, heavily pretreated, many with prior asciminib/ponatinib.
Key point: By 24 weeks, 53% achieved MMR in randomized 60/120 mg cohorts; favorable tolerability, no MTD.
Implication: May influence prescriber choice and payer reviews pending full data.
✅ Sonrotoclax approved in China for R/R MCL and R/R CLL/SLL [7] [CN • 06 Jan 2026]
Context: Approval supported by Phase 1/2 MCL (ORR 52.4%) and Phase 2 CLL/SLL (ORR 77%) single-arm data.
Key point: First-in-world approval; US Priority Review for MCL monotherapy noted.
Implication: Introduces competition that may affect pricing and formulary access.
🧩 frontMIND hits PFS in first-line DLBCL with tafasitamab regimen [8] [US • 05 Jan 2026]
Context: Global Phase 3, tafasitamab + lenalidomide + R-CHOP vs R-CHOP in untreated DLBCL.
Key point: Primary endpoint met, HR 0.75 for PFS; EFS also met; no new safety signals. sBLA planned H1 2026.
Implication: May influence prescriber choice and payer reviews pending full data.
🎗️ CK0804 Treg therapy receives Orphan Drug Designation in MF [9] [US • 06 Jan 2026]
Context: Allogeneic CXCR4^hi Tregs aim to home to marrow/spleen and secrete IL-10.
Key point: In a 13-patient MF study, signals included spleen volume reduction and symptom improvement in evaluable subsets.
Implication: Signals pipeline investment and modality expansion.
🩸 Takeda/Protagonist file NDA for rusfertide in PV [10] [US • 05 Jan 2026]
https://www.takeda.com/newsroom/newsreleases/2025/new-drug-application-pv/
Context: NDA includes VERIFY 52-week data meeting primary and key secondary endpoints; rusfertide is weekly SC hepcidin mimetic.
Key point: Aims to deliver durable hematocrit control and reduce phlebotomy burden; BTD, ODD, Fast Track granted.
Implication: May influence prescriber choice and payer reviews pending full data.
🧯 IV iron dextran modeled as most cost-effective for IDA in HMB [11] [08 Jan 2026]
https://medicalxpress.com/news/2026-01-iv-iron-effective-treatment-women.html
Context: Blood Advances modeling across a menstrual lifetime compared IV iron vs oral ferrous sulfate.
Key point: Single-dose IV iron dextran had favorable ICER and net monetary benefit vs oral; assumptions noted.
Implication: Could inform practice and payer discussions; interpretation depends on study design and confounding control.
Why it matters
- Earlier-line BCMA/CD38 combinations and in vivo CAR-T approaches aim to broaden myeloma access and durability.
- New B-cell malignancy entrants, from CD19 combos to next-gen BCL2 inhibitors, signal competitive shifts in front-line and R/R settings.
- Access programs and ODD designations may accelerate availability in underserved geographies and rare diseases.
- Cost-effectiveness analyses for supportive care, like IV iron, can reshape first-line practice and coverage.
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FAQ
What did MajesTEC-3 show for teclistamab + daratumumab?
Significant PFS and OS improvements versus daratumumab-based standards in RRMM after 1–3 prior lines, with HR 0.17 for progression or death and higher infection rates on combo. EMA variation filed on 06 Jan 2026. [1]
How is KLN-1010 different from traditional CAR-T?
It is an in vivo anti-BCMA approach aiming to generate CAR-T cells inside the patient after a single infusion, without preparative chemotherapy or bespoke manufacturing. US IND cleared on 07 Jan 2026. [2]
Where will revumenib be available via managed access?
WODA will administer named-patient access across parts of Eurasia, Central/Southeast Europe, Israel, Middle East/Turkey, Latin America, and Africa, where regulations permit. [3]
What is the regulatory status of sonrotoclax?
It received first-in-world approval in China for R/R MCL and R/R CLL/SLL, with US Priority Review ongoing for MCL monotherapy. [7]
What did frontMIND change in first-line DLBCL?
Adding tafasitamab + lenalidomide to R-CHOP met PFS and EFS endpoints vs R-CHOP alone. Incyte plans an sBLA in H1 2026. [8]
Why pursue rusfertide in PV?
VERIFY showed sustained hematocrit control and reduced phlebotomy burden. An NDA was submitted to FDA on 05 Jan 2026. [10]
Entities / Keywords
Teclistamab (TECVAYLI; bispecific BCMA×CD3), Daratumumab (DARZALEX; CD38), MajesTEC-3; Kelonia KLN-1010 (in vivo BCMA CAR-T); Revuforj (revumenib; menin inhibitor; Syndax; WODA); AB8939 (microtubule destabilizer; ALDH1A1/ALDH2), Venetoclax (BCL2); CER-1236 (CER-T; phagocytic T cells); ELVN-001 (BCR::ABL1 active-site TKI); Sonrotoclax (BGB-11417; BCL2 inhibitor); Tafasitamab (Monjuvi/Minjuvi; CD19 mAb); CK0804 (CXCR4^hi Treg); Rusfertide (hepcidin mimetic; PV); IV iron dextran, IDA, heavy menstrual bleeding.
References
- https://www.jnj.com/media-center/press-releases/johnson-johnson-submits-application-to-the-european-medicines-agency-for-tecvayli-teclistamab-in-combination-with-darzalex-daratumumab-subcutaneous-formulation-for-patients-with-relapsed-refractory-multiple-myeloma
- https://keloniatx.com/kelonia-therapeutics-announces-fda-clearance-of-investigational-new-drug-ind-application-for-kln-1010-an-in-vivo-bcma-car-t-therapy-for-r/
- https://ir.syndax.com/news-releases/news-release-details/syndax-and-world-orphan-drug-alliance-launch-multi-regional
- https://www.ab-science.com/wp-content/uploads/2026/01/CP-AB8939-JAN-26-VENG-VF.pdf
- https://www.cero.bio/press-release?storyId=5313479179734752
- https://www.prnewswire.com/news-releases/enliven-reports-positive-initial-phase-1b-data-for-elvn-001-in-cml-and-outlines-2026-clinical-milestones-302655863.html
- https://ir.beonemedicines.com/news/beone-medicines-novel-bcl2-inhibitor-sonrotoclax-achieves-first-in-world-approval-in-rr-mcl-and-rr-cllsll/562da165-f6a9-48d0-a357-aca27ac112ca
- https://www.biospace.com/press-releases/incyte-announces-positive-topline-results-from-pivotal-study-of-tafasitamab-monjuvi-minjuvi-as-a-first-line-treatment-for-diffuse-large-b-cell-lymphoma
- https://www.prnewswire.com/news-releases/fda-grants-orphan-drug-designation-to-cellenkos-ck0804-treg-therapy-for-treatment-of-myelofibrosis-302653827.html
- https://www.takeda.com/newsroom/newsreleases/2025/new-drug-application-pv/
- https://medicalxpress.com/news/2026-01-iv-iron-effective-treatment-women.html