Hematology Today—January 19, 2026

This week’s Hematology update spans regulatory milestones, late-stage clinical readouts, and early-phase progress across hematologic cancers and bleeding disorders. Themes include expedited designations, pivotal efficacy signals, and real-world evidence with potential to reduce treatment burden.

In Today’s Newsletter

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💉 BESREMi sBLA for ET gets PDUFA date [1] [US • 13 Jan 2026]

https://us.pharmaessentia.com/fda-confirms-a-pdufa-goal-date-of-august-30-2026-for-the-sbla-submission-of-ropeginterferon-alfa-2b-njft-in-essential-thrombocythemia-et/
Context: FDA accepted sBLA for ropeginterferon alfa-2b-njft in adult ET, Standard review; supported by Phase 3 SURPASS-ET and Phase 2b EXCEED-ET.
Key point: PDUFA goal date is 30 Aug 2026; FDA identified no filing review issues.
Implication: Regulatory/generics: Introduces competition that may affect pricing and formulary access.

🧪 EXCEED-ET Phase 2b topline shows efficacy in ET [2] [US • 12 Jan 2026]

https://us.pharmaessentia.com/pharmaessentia-announces-positive-topline-phase-2b-data-from-exceed-et-study-evaluating-ropeginterferon-alfa-2b-njft-for-essential-thrombocythemia/
Context: Single-arm Phase 2b, North America; n=91; modified ELN composite response; allele burden decline in JAK2V617F subgroup.
Key point: ITT response ≈60.2% at months 10 and 13; higher in treatment-naïve (~68%) vs HU-experienced (~33.4%).
Implication: Clinical topline/efficacy: May influence prescriber choice and payer reviews pending full data.

🧬 FDA Breakthrough for IPN60340 + Ven-Aza in unfit AML [3] [EU/US • 13 Jan 2026]

https://www.ipsen.com/press-release/u-s-fda-grants-ipsens-ipn60340-ict01-breakthrough-therapy-designation-in-first-line-unfit-acute-myeloid-leukemia-3218133/
Context: IPN60340 (ICT01) is an anti-BTN3A mAb; BTD based on Phase I/II EVICTION single-arm data with encouraging responses vs historical SOC.
Key point: Designation covers first-line unfit AML in combination with venetoclax and azacitidine.
Implication: Partnerships/BD: Signals pipeline investment and modality expansion.

🧯 Ficlatuzumab combo starts dosing in front-line AML [4] [US • 14 Jan 2026]

https://www.aveooncology.com/aveo-oncology-an-lg-chem-company-announces-first-patient-dosed-in-front-line-aml-combination-study-of-ficlatuzumab/
Context: Beat AML Master Trial sub-study; Phase 1b/2 of ficlatuzumab + azacitidine + venetoclax in untreated AML ≥60.
Key point: First patient dosed; study will assess safety, PK/PD, and preliminary efficacy of HGF-ligand blockade with Ven-Aza.
Implication: Partnerships/BD: Signals pipeline investment and modality expansion.

❤️ Annamycin independent review finds no cardiotoxicity [5] [US/EU • 13 Jan 2026]

https://moleculin.com/moleculin-reports-independent-assessment-confirms-no-cardiotoxicity-of-annamycin-in-90-subjects/
Context: Five trials in AML and STS; review included ECGs, echo with GLS, and central troponins; many exceeded typical anthracycline lifetime dose.
Key point: Independent expert concluded no evidence of cardiotoxicity in 90 annamycin-treated subjects.
Implication: Clinical topline/efficacy: May influence prescriber choice and payer reviews pending full data.

🔁 Ficlatuzumab combo starts dosing in front-line AML (duplicate) [6] [US • 14 Jan 2026]

https://www.aveooncology.com/aveo-oncology-an-lg-chem-company-announces-first-patient-dosed-in-front-line-aml-combination-study-of-ficlatuzumab/
Context: Same Beat AML Phase 1b/2 sub-study and design as above.
Key point: Confirms first-patient-dosed milestone for ficlatuzumab + azacitidine + venetoclax in untreated AML ≥60.
Implication: Partnerships/BD: Signals pipeline investment and modality expansion.

🦠 Epcoritamab hits PFS in R/R DLBCL, OS neutral [7] [US • 16 Jan 2026]

https://news.abbvie.com/2026-01-16-AbbVie-Announces-Topline-Results-for-Epcoritamab-DuoBody-R-CD3xCD20-from-Phase-3-EPCORE-R-DLBCL-1-Trial-in-Patients-with-Relapsed-Refractory-Diffuse-Large-B-cell-Lymphoma-DLBCL
Context: Phase 3 open-label, n=483, transplant-ineligible; epcoritamab vs R-GemOx or BR.
Key point: Improved PFS (HR 0.74; 95% CI 0.60–0.92); OS not statistically significant (HR 0.96; 95% CI 0.77–1.20); safety consistent.
Implication: Clinical topline/efficacy: May influence prescriber choice and payer reviews pending full data.

🧒 Nivolumab + AVD benefits adolescents with advanced HL [8] [US • 12 Jan 2026]

https://www.roswellpark.org/newsroom/202601-roswell-park-childrens-healthcare-atlanta-analysis-confirms-benefit-nivolumab-combo
Context: Subset analysis of a Phase 3 trial; adolescents 12–17, n=240 within total n=994.
Key point: Three-year PFS 93% with nivolumab-AVD vs 82% with brentuximab-AVD; radiation use dropped to less than 1%.
Implication: Observational/RWE: Could inform practice and payer discussions; interpretation depends on study design and confounding control.

🧵 VLX-1005 targets HIT via selective 12-LOX inhibition [9] [US • 12 Jan 2026]

https://www.cadrenal.com/cadrenal-therapeutics-highlights-high-incidence-of-thrombotic-complications-in-heparin-induced-thrombocytopenia-hit-at-the-j-p-morgan-healthcare-conference-and-vlx-1005-as-a-potential-therapeutic-s/
Context: Company highlights HIT thrombotic burden; early Phase 2 findings suggest reduced thrombotic complications; ODD and Fast Track noted.
Key point: Planning FDA discussions for a potential pivotal Phase 3 in HIT with selective 12-LOX inhibitor VLX-1005.
Implication: Clinical topline/efficacy: May influence prescriber choice and payer reviews pending full data.

🩸 EHL-FIX cuts bleeds and infusions in pediatric hemophilia B [10] [16 Jan 2026]

https://hemophilianewstoday.com/extended-half-life-products-reduce-bleeds-hemophilia-b-children-study/
Context: Retrospective real-world study in Germany; switch from SHL-FIX to EHL-FIX.
Key point: Lower ABR and factor consumption with fewer infusions; joint health and quality of life maintained.
Implication: Observational/RWE: Could inform practice and payer discussions; interpretation depends on study design and confounding control.

Why it matters

Ropeginterferon could become a new ET option in the US, supported by Phase 3 and Phase 2b data.
Front-line unfit AML is seeing diversification across BTN3A activation and HGF pathway blockade.
A non-cardiotoxic anthracycline would ease lifetime dose constraints across oncology.
PFS-positive, OS-neutral DLBCL data may still shift treatment sequences and payer views for bispecifics.
Adolescent HL data support earlier immunotherapy access with less radiation exposure.
RWE supports EHL FIX adoption to reduce burden and potentially costs in pediatric hemophilia B.

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FAQ

What decision date did FDA set for BESREMi in ET?

FDA set a PDUFA goal of 30 Aug 2026 for the sBLA in adult ET, Standard review [1].

How were EXCEED-ET responses by prior HU exposure?

Topline ITT response ≈60.2% at months 10 and 13, higher in treatment-naïve (~68%) vs HU-experienced (~33.4%) using a modified ELN composite [2].

What does Ipsen’s Breakthrough Therapy cover?

IPN60340 (ICT01) with venetoclax and azacitidine for first-line unfit AML, based on Phase I/II EVICTION data showing encouraging responses vs historical SOC [3].

What milestone did AVEO report in AML, and why list twice?

First patient dosed in a Beat AML Phase 1b/2 sub-study of ficlatuzumab + azacitidine + venetoclax; included twice to preserve your original sequence [4][6].

What were EPCORE DLBCL-1 topline outcomes?

Epcoritamab improved PFS vs chemo (HR 0.74) with OS not statistically significant (HR 0.96); safety aligned with known profile [7].

What did Moleculin’s review conclude on annamycin?

No evidence of cardiotoxicity in 90 subjects across five trials, including many above typical anthracycline lifetime exposure [5].

What pediatric HL change is suggested?

Support to lower the minimum age to 12 for nivolumab + AVD, with higher 3-year PFS and minimal radiation use in adolescents [8].

How does VLX-1005 propose to help HIT?

Selective 12-LOX inhibition aims to modulate immune-mediated platelet activation, with early Phase 2 signal and plans for pivotal design discussions [9].

Entities / Keywords

PharmaEssentia, BESREMi, ropeginterferon alfa-2b-njft, ET, SURPASS-ET, EXCEED-ET; Ipsen, IPN60340, ICT01, BTN3A, EVICTION, AML; AVEO Oncology, LG Chem, ficlatuzumab, HGF, c-Met, Beat AML; Moleculin, annamycin, naxtarubicin, cardiotoxicity; AbbVie, Genmab, epcoritamab, EPKINLY, TEPKINLY, DLBCL, bispecific; Roswell Park, Children’s Healthcare of Atlanta, nivolumab, Opdivo, AVD, brentuximab vedotin, Adcetris, Hodgkin lymphoma; Cadrenal Therapeutics, VLX-1005, 12-LOX, HIT; hemophilia B, extended half-life FIX, Idelvion, Alprolix, Rebinyn/Refixia.