This biweekly hematology video covers regulatory actions, clinical progress in hematologic cancers, market updates, and access or policy changes, including new U.S. product availability and draft guidance that may reshape trial endpoints.
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- Week 12–18 January 2026
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Top Stories Covered in This Video
Chapters
0:00 Introduction
0:08 FDA accepts sBLA for ropeginterferon alfa-2b-njft in essential thrombocythemia, PDUFA August 30, 2026
0:45 FDA grants Breakthrough Therapy to IPN60340 plus venetoclax and azacitidine in first-line unfit AML
1:19 Epcoritamab Phase 3 in R/R DLBCL improves PFS, OS not significant
2:05 VLX-1005 12-LOX inhibitor highlighted for heparin-induced thrombocytopenia, FDA engagement planned
2:34 FDA draft guidance proposes MRD negativity and CR as surrogate endpoints for accelerated approval in multiple myeloma
3:08 DARZALEX 2025 net sales USD 14.351 billion reported by J&J
3:42 Zavabresib OPN-2853 receives Orphan Drug Designation for myelofibrosis
4:16 Takeda launches GAMMAGARD LIQUID ERC low-IgA 10 percent IG in the United States
4:56 How to reach us
Transcript
Welcome to the latest edition of Hematology Updates, covering breakthroughs in the past two weeks. Brought to you by LucidQuest.
PharmaEssentia reports the FDA accepted the supplemental BLA for ropeginterferon alfa-2b-njft in essential thrombocythemia with a Standard review, setting a PDUFA target of August 30, 2026. This submission is supported by the Phase 3 SURPASS-ET study and the confirmatory Phase 2b EXCEED-ET, with endpoints not specified in the announcement. Expect potential shifts in prescriber and payer evaluations once full data are available.
Ipsen’s BTN3A antibody IPN60340 received FDA Breakthrough Therapy Designation in first-line unfit acute myeloid leukemia when combined with venetoclax and azacitidine. The designation is based on EVICTION Phase I/II data, including a single-arm subset with the venetoclax and azacitidine regimen that showed encouraging response rates versus historical standards of care. This supports expedited development and broader modality investment.
AbbVie and Genmab announced topline Phase 3 results in relapsed or refractory diffuse large B-cell lymphoma. In EPCORE DLBCL-1, epcoritamab improved progression-free survival versus physician’s choice of R-GemOx or bendamustine and rituximab, with a hazard ratio of 0.74, while overall survival did not reach statistical significance, hazard ratio 0.96. The trial enrolled 483 transplant-ineligible patients and reported a safety profile consistent with prior experience. These results may shape treatment decisions after full data disclosure.
Cadrenal highlighted VLX-1005, a selective 12-lipoxygenase inhibitor for heparin-induced thrombocytopenia. Emerging Phase 2 data in suspected HIT suggest reduced thrombotic complications, with details not yet specified. The program holds Orphan Drug Designation and Fast Track, and FDA engagement is planned regarding a potential Phase 3.
The FDA issued draft guidance proposing minimal residual disease negativity after complete response, and complete response or stringent complete response, as surrogate endpoints for accelerated approval in multiple myeloma. The draft emphasizes validated MRD assays, confirmatory trials, and a preference for randomized designs, while allowing single-arm approaches in defined situations. This may influence study design, clinical practice interpretation, and payer discussions.
Genmab reported 2025 DARZALEX net sales of 14.351 billion US dollars as disclosed by Johnson and Johnson, with 8.266 billion from the United States and 6.085 billion from the rest of world. Royalty revenues to Genmab are governed by the global license. Continued growth across intravenous and subcutaneous formulations underscores the product’s market momentum and competitive impact.
Opna Bio announced Orphan Drug Designation for zavabresib, also known as OPN-2853, in myelofibrosis. This BET inhibitor has been tested with ruxolitinib in the Phase 1 PROMise study, where data presented at ASH 2025 showed at least 50 percent spleen length reduction in 16 of 26 evaluable patients. The designation provides development incentives and potential exclusivity upon approval.
Takeda introduced GAMMAGARD LIQUID ERC in the United States, a ready-to-use 10 percent immune globulin with low IgA content of no more than 2 micrograms per milliliter. It is indicated for primary immunodeficiency in patients two years of age and older, can be administered intravenously or subcutaneously, and allows room-temperature storage for up to 24 months. The label includes thrombosis and renal warnings consistent with the IG class, and the product shares a manufacturing platform with Takeda’s immunoglobulin portfolio.
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Why it matters
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MRD/CR guidance, if finalized, could reshape myeloma trial endpoints and timelines.
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Epcoritamab’s PFS win without OS benefit will focus discussions on value, sequencing, and payer criteria.
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New designations for IPN60340 and zavabresib reinforce momentum in AML and MF beyond JAK inhibition.
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Low-IgA IG availability provides a differentiated option within PI care pathways.
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DARZALEX sales underscore entrenched anti-CD38 use, influencing competitive strategies.
🗓️ Explore weekly details and sources
- Week 12–18 January 2026
- Week 19–25 January 2025
📚 See the full Hematology archive on our research hub page.
FAQ
What is the PDUFA date for ropeginterferon alfa-2b-njft in essential thrombocythemia?
The PDUFA date is 30 August 2026, following FDA acceptance of the supplemental biologics license application under a Standard Review classification.
Why did IPN60340 receive Breakthrough Therapy Designation?
Breakthrough Therapy Designation was granted based on early clinical signals from the EVICTION trial, showing potential substantial improvement when IPN60340 is combined with venetoclax and azacitidine in first-line, unfit acute myeloid leukemia, although data remain preliminary.
How did epcoritamab perform in the EPCORE DLBCL-1 study?
The study demonstrated improvements in progression-free survival versus chemoimmunotherapy, along with higher complete response rate and duration of response, while overall survival was not statistically significant; the safety profile was consistent with prior experience.
What is VLX-1005 and what is its status in heparin-induced thrombocytopenia?
VLX-1005 is a first-in-class selective 12-lipoxygenase inhibitor with emerging Phase 2 signals in suspected heparin-induced thrombocytopenia; it holds Orphan Drug and Fast Track designations, with FDA discussions planned regarding a potential Phase 3 program.
What does the FDA’s draft guidance change for multiple myeloma trials?
The draft guidance supports minimal residual disease negativity and complete response as surrogate endpoints for accelerated approval, provided assays are validated and confirmatory trials are planned, with a preference for randomized study designs.
How large were DARZALEX sales in 2025 and who benefits?
Global DARZALEX sales reached 14.351 billion US dollars in 2025, as reported by Johnson & Johnson, with Genmab receiving royalties under its existing license agreement.
Entities / Keywords
PharmaEssentia, BESREMi, ropeginterferon alfa-2b-njft; Ipsen, IPN60340, ICT01, BTN3A, AML; AbbVie, Genmab, epcoritamab, EPKINLY, DLBCL; Cadrenal, VLX-1005, 12-LOX, HIT; FDA, MRD, complete response, accelerated approval, multiple myeloma; Genmab, DARZALEX, daratumumab; Opna Bio, zavabresib, OPN-2853, BET inhibitor, myelofibrosis; Takeda, GAMMAGARD LIQUID ERC, immunoglobulin, low IgA, PI.