Hematology Today — October 27, 2025

LucidQuest

2 weeks ago

This week in hematology: the latest clinical updates, industry progress, and developments from leading biopharma companies.

In Today’s Newsletter

🧬 Blenrep BVd approved for 3L+ multiple myeloma [1] [US • 23 Oct 2025]

https://www.gsk.com/en-gb/media/press-releases/blenrep-approved-by-us-fda-for-use-in-treatment-of-relapsedrefractory-multiple-myeloma/
Context: DREAMM-7 Phase 3, 494 patients, BVd vs DVd.
Key point: BVd cut risk of death by 51% and tripled median PFS vs DVd in indicated 3L+ population.
Implication: May influence prescriber choice and payer reviews pending full data.

🧪 HDP-101 (pamlectabart tismanitin) gets FDA Fast Track in MM [2] [US • 23 Oct 2025]

https://heidelberg-pharma.com/en/press-investors/announcements/press-releases-and-ad-hoc-announcements/pr-heidelberg-pharmas-lead-adc-candidate-hdp-101-granted-fast-track-designation-by-us-fda-for-the-treatment-of-multiple-myeloma
Context: Supported by nonclinical data and ongoing Phase I/IIa clinical data in R/R MM.
Key point: Fast Track grants enhanced FDA interactions and rolling review eligibility.
Implication: Signals pipeline investment and modality expansion.

💊 Revuforj (revumenib) FDA-approved for R/R NPM1m AML [3] [US • 24 Oct 2025]

https://ir.syndax.com/news-releases/news-release-details/syndax-announces-fda-approval-revuforjr-revumenib-adult-and
Context: Label expansion built on AUGMENT-101 Phase 2; CR+CRh 23% (n stated in source).
Key point: First therapy approved for both R/R AML with NPM1 mutation and R/R acute leukemia with KMT2A translocation.
Implication: May influence prescriber choice and payer reviews pending full data.

🔗 Nurix starts DAYBreak pivotal bexobrutideg study in r/r CLL [4] [US • 22 Oct 2025]

https://ir.nurixtx.com/news-releases/news-release-details/nurix-initiates-daybreaktm-pivotal-study-bexobrutideg-relapsed
Context: Single-arm Phase 2 at 600 mg QD, alignment with FDA, MHRA, EMA; Phase 3 confirmatory planned.
Key point: Study may support Accelerated Approval in post-cBTKi, BCL-2i, ncBTKi population.
Implication: Signals pipeline investment and modality expansion.

🧯 Ofirnoflast (HT-6184) gets FDA ODD for MDS [5] [US • 23 Oct 2025]

https://finance.yahoo.com/news/ofirnoflast-ht-6184-receives-orphan-121500565.html
Context: NEK7 modulator targeting NLRP3 inflammasome; company cites completed Phase 2 in MDS (details not provided).
Key point: Orphan Drug Designation confers incentives, including potential 7-year exclusivity upon approval.
Implication: Signals pipeline investment and modality expansion.

🧷 GTB-3650 TriKE® Phase 1 advances to Cohort 4 [6] [US • 23 Oct 2025]

https://www.gtbiopharma.com/news-media/press-releases/detail/301/gt-biopharma-provides-enrollment-update-on-gtb-3650-phase-1
Context: Six patients treated across Cohorts 1–3, no safety/tolerability issues; dose-escalation up to 100 μg/kg/day allowed.
Key point: Proceeding to 10 μg/kg/day, described as nearer a potential efficacy threshold.
Implication: Signals pipeline investment and modality expansion.

🩸 UK approves concizumab (Alhemo) for haemophilia with inhibitors [7] [UK • 24 Oct 2025]

https://www.gov.uk/government/news/concizumab-approved-to-prevent-or-reduce-the-frequency-of-bleeding-episodes-in-people-aged-12-years-and-older-with-haemophilia-a-or-b-with-inhibitors
Context: MHRA approval via International Recognition Procedure; SC daily dosing.
Key point: Prophylaxis approved for patients 12+ with haemophilia A or B with inhibitors.
Implication: Introduces competition that may affect pricing and formulary access.

🧵 Anemia in myelofibrosis, outcomes and QoL, expert view [8] [24 Oct 2025]

https://www.curetoday.com/view/anemia-management-in-myelofibrosis-survival-and-quality-of-life
Context: Interview with Dr. Palandri, SIMPLIFY-1 author; discusses anemia burden and momelotinib potential.
Key point: Severe anemia associated with markedly shorter survival, high system burden; momelotinib positioned to address anemia plus symptoms.
Implication: Could inform practice and payer discussions, interpretation depends on study design and confounding control.

🤰 PlGF biomarker predicts preeclampsia risk in SCD pregnancies [9] [21 Oct 2025]

https://www.hematology.org/newsroom/press-releases/2025/biomarker-can-help-predict-preeclampsia-risk-in-women-with-sickle-cell-disease
Context: Blood Advances study, retrospective, single center; n=83 SCD vs 149 controls, 20–36 weeks gestation.
Key point: Low PlGF thresholds predicted early and late preeclampsia in SCD with high sensitivity/specificity.
Implication: Could inform practice and payer discussions; interpretation depends on study design and confounding control.

🧭 ASH launches Blood Health Information Hub [10] [24 Oct 2025]

https://www.hematology.org/newsroom/press-releases/2025/ash-launches-blood-health-information-hub
Context: Public resource on common blood topics, expert reviewed.
Key point: Aims to counter hematology misinformation with vetted content.
Implication: May expand screening, initiation, and follow-up at scale.

Why it matters

BCMA and menin advances show rapid evolution of targeted options across MM and AML.
Regulatory designations and pivotal starts indicate momentum toward broader access in hematologic cancers.
UK concizumab approval expands inhibitor-population prophylaxis choices in haemophilia.
Real-world burden of anemia in MF underscores value of therapies addressing anemia alongside disease control.
Biomarker-guided obstetric care in SCD may reduce maternal-fetal complications.

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FAQ

What did FDA approve for Blenrep and in whom?

FDA approved Blenrep with bortezomib and dexamethasone for adult RRMM after at least two prior lines including a PI and IMiD, supported by DREAMM-7 showing OS and PFS benefits [1]. May inform prescriber and payer decisions pending full data.

Is HDP-101 close to approval with Fast Track?

No. Fast Track enables more frequent FDA engagement and rolling review, but safety and efficacy are not yet established and the product remains investigational in an ongoing Phase I/IIa study [2].

What is new about Revuforj’s label?

Revuforj is now FDA-approved for R/R AML with a susceptible NPM1 mutation, adding to its KMT2A-rearranged acute leukemia indication, based on AUGMENT-101 Phase 2 data (endpoint specifics provided by the company) [3].

What is Nurix testing in DAYBreak?

Bexobrutideg, an oral BTK degrader, in a pivotal single-arm Phase 2 for r/r CLL after cBTKi, BCL-2i, and ncBTKi, aligned with multiple regulators and intended to support Accelerated Approval [4].

What benefit does Orphan Drug Designation give ofirnoflast?

It provides incentives such as tax credits and potential 7-year US exclusivity upon approval; it is not an approval. Ofirnoflast targets NEK7–NLRP3 inflammasome biology in MDS per the company [5].

How far along is GTB-3650?

Phase 1 dose escalation advanced to Cohort 4 without safety/tolerability issues in prior cohorts, aiming to explore higher doses; clinical activity not detailed yet [6].

Entities / Keywords

Blenrep (belantamab mafodotin-blmf); BCMA; DREAMM-7; multiple myeloma; HDP-101 (pamlectabart tismanitin); amanitin ADC; Fast Track; Revuforj (revumenib); menin inhibitor; NPM1m AML; KMT2A; bexobrutideg (NX-5948); BTK degrader; CLL; Ofirnoflast (HT-6184); NEK7; NLRP3; MDS; GTB-3650; TriKE; CD33; concizumab (Alhemo); haemophilia inhibitors; PlGF; preeclampsia; sickle cell disease; momelotinib (Ojjaara); myelofibrosis anemia.