Hematology Video Recap—November 17, 2025

LucidQuest

1 day ago

This biweekly Hematology video recap spans regulatory filings, late-stage trial initiations, expedited designations, access decisions, and rare-disease approvals—plus early program moves.

🎯 Watch Our Video Summary Capturing Hematology News from the Last Two Weeks

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Week 3–9 November 2025
Week 10–16 November 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.

In Canada, the Canadian Drug Agency recommended conditional public reimbursement for belantamab mafodotin in combination regimens BPd and BVd for relapsed or refractory multiple myeloma after referencing outcomes from DREAMM-7 and DREAMM-8. The recommendation follows Health Canada’s July 2025 approval and urges progression to pCPA negotiations, which could expand access, screening, and real-world uptake.

CHARM Therapeutics has nominated CHM-029, a next generation pan-menin inhibitor discovered through AI DragonFold. Pre clinical AML models demonstrated dose dependent tumor regression and activity against known resistance mutations, and forced mutation assays did not generate resistance. First in human evaluation is targeted for Q2 2026.

Kura Oncology has dosed the first patient in the second global randomized double blind Phase 3 KOMET 017 trial in newly diagnosed NPM1 mutated or KMT2A rearranged AML across intensive and non intensive regimens. This milestone triggered a 30 million dollar payment from Kyowa Kirin, bringing total milestones received to 105 million dollars.

Leukogene Therapeutics received FDA Orphan Drug Designation for M2T CD33, also called LTI 214, a CD33 targeting MHCII engager directed at blasts and leukemic stem cells in AML. The designation enables incentives including tax credits, fee waivers, and potential US market exclusivity upon approval.

Otsuka has filed an EMA marketing authorization application for all oral decitabine–cedazuridine, Inaqovi, plus venetoclax in newly diagnosed AML ineligible for intensive induction. The filing is supported by Phase 1 Phase 2 ASCERTAIN V, which reported a 46.5 percent complete remission rate at a median of 2.4 months and sustained remissions at 6 and 12 months per source report.

March Biosciences has received FDA Regenerative Medicine Advanced Therapy designation for MB 105, an autologous CD5 targeted CAR-T therapy in relapsed or refractory CD5 positive T cell lymphoma. Early data from the Phase 2 trial NCT06534060 indicate clinical activity with a manageable safety profile, and RMAT may enable expedited review and enhanced FDA interactions.

In Germany, the Paul Ehrlich Institut has granted the first approval for Prufibry, a fibrinogen concentrate for congenital and acquired fibrinogen deficiency across age groups. Austria and Spain are expected to follow via a decentralized procedure, while the US BLA review is ongoing and a regulatory decision is anticipated by year end.

Zydus Lifesciences has received FDA Orphan Drug Designation for desidustat in beta thalassemia, a HIF prolyl hydroxylase inhibitor program. The designation enables development incentives and potential 7 year exclusivity upon US approval.

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🗓️ Explore weekly details and sources

Week 3–9 November 2025
Week 10–16 November 2025

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

FAQ

What did the CDA recommend for Blenrep in RRMM?

Reimburse-with-conditions for belantamab mafodotin combinations BPd and BVd, referencing DREAMM-7/8 outcomes, with pCPA negotiations urged next [1].

How is CHM-029 positioned versus first-gen menin inhibitors?

Per CHARM, CHM-029 retained potency against known resistance mutations and showed dose-dependent tumor regression in pre-clinical models; first-in-human is targeted for Q2 2026 [2].

What is Kura’s KOMET-017 testing, and why the milestone?

Two global, randomized, double-blind Phase 3 trials in newly diagnosed NPM1-m or KMT2A-r AML across intensive and non-intensive chemo; dosing the first patient in the second trial triggered a $30M Kyowa Kirin payment [3].

What does FDA ODD confer to Leukogene’s LTI-214?

Incentives such as tax credits, fee waivers, and potential market exclusivity upon approval for AML, supporting the MHCII-engager platform approach [4].

What supports Otsuka’s EMA filing for Inaqovi plus venetoclax?

Phase I/II ASCERTAIN-V data in newly diagnosed, unfit AML, with a reported 46.5% CR and sustained remissions at 6 and 12 months per source report [5].

What does RMAT mean for March Bio’s MB-105?

Breakthrough-like benefits including enhanced FDA interactions and eligibility for rolling or priority review, based on early signs of activity in R/R CD5-positive TCL [6].

What exactly was approved for Grifols/Biotest in Europe?

Germany approved Prufibry for congenital and acquired fibrinogen deficiency, with Austria and Spain expected to follow via a decentralized procedure; US BLA review ongoing per company [7].

What indication did Zydus’ desidustat receive ODD for?

Beta-thalassemia, enabling orphan incentives and potential 7-year exclusivity if approved in the US [8].

Entities / Keywords

Belantamab mafodotin (Blenrep; GSK); BCMA ADC; DREAMM-7; DREAMM-8; pCPA; CHARM Therapeutics; CHM-029; menin inhibitor; Kura Oncology; ziftomenib; KOMET-017; Kyowa Kirin; Leukogene Therapeutics; M2T-CD33; LTI-214; Otsuka; Inaqovi (decitabine–cedazuridine); venetoclax; ASCERTAIN-V; March Biosciences; MB-105; CD5 CAR-T; Grifols; Biotest; Prufibry; fibrinogen concentrate; Zydus Lifesciences; desidustat; beta-thalassemia