Hematology Update: Jaypirca CLL win, BTK degrader data, WHO EML update and More
From Phase 3 CLL PFS superiority (Jaypirca) and promising BTK degrader responses to myeloma dose-optimization and pivotal AML trial starts—this week’s signals are strong.
We span CLL, multiple myeloma, AML/MDS, myelofibrosis, and hemophilia A, with WHO EML updates and assay advances underscoring access and measurement progress.
In Today’s Newsletter
🧬 CellCentric completes Phase 2 enrollment for inobrodib in myeloma [1] [UK/US • 09 Sep 2025]
Context: 60 patients across 3 dose cohorts of inobrodib+pomalidomide+dexamethasone.
Key point: Enrollment completed in randomized dose-optimization; data expected end 2025 to guide registration studies.
Implication: May influence prescriber choice and payer reviews pending full data.
💊 Lilly’s Jaypirca tops Phase 3 CLL trial [2] [US • 08 Sep 2025]
Context: BRUIN CLL-313 enrolled 282 treatment-naïve CLL/SLL patients, pirtobrutinib vs bendamustine+rituximab.
Key point: Statistically significant, clinically meaningful PFS benefit; OS not yet mature but trending positive.
Implication: May influence prescriber choice and payer reviews pending full data.
🔄 BTK degrader bexobrutideg shows high ORR in CLL [3] [US • Sep 2025]
Context: Phase 1a NX-5948-301, 48 relapsed/refractory CLL patients (81% double-refractory).
Key point: ORR 81% with durable responses, including patients resistant to both BTK and BCL2 inhibitors.
Implication: May influence prescriber choice and payer reviews pending full data.
🔬 Biosplice opens NCI trial of cirtuvivint in AML/MDS [4] [09 Sep 2025]
Context: Phase 1 trial led by Dana-Farber, NCI-sponsored; cirtuvivint alone or with ASTX727.
Key point: First patient dosed under CRADA with NCI in relapsed/refractory AML/MDS.
Implication: May influence prescriber choice and payer reviews pending full data.
🌍 Moleculin begins EU dosing in pivotal AML trial [5] [EU • 08 Sep 2025]
Context: MIRACLE Phase 2B/3 trial of Annamycin+cytarabine in relapsed/refractory AML; global sites active.
Key point: First EU patient dosed; early unblinding of 45 subjects expected late 2025.
Implication: May influence prescriber choice and payer reviews pending full data.
📊 Karyopharm completes Phase 3 SENTRY enrollment [6] [US • 10 Sep 2025]
Context: 353 JAKi-naïve myelofibrosis patients randomized to selinexor+ruxolitinib vs placebo+ruxolitinib.
Key point: Enrollment completed; topline SVR35 and symptom data due Mar 2026.
Implication: May influence prescriber choice and payer reviews pending full data.
📅 Oxcia highlights Blood Cancer Awareness Month [7] [Sweden • 10 Sep 2025]
https://oxcia.com/blood-cancer-awareness-month/
Context: Campaign to raise awareness of leukemia, lymphoma, myeloma; first national Blood Cancer Day in Sweden.
Key point: Oxcia emphasized AML program OXC-101 (Orphan Drug status, Phase 1/2 expansion ongoing).
Implication: May expand screening, initiation, and follow-up at scale.
🏃 Hemlibra reduces bleeds, burden in hemophilia A [8] [EU • 12 Sep 2025]
https://hemophilianewstoday.com/news/hemlibra-help-active-men-hemophilia-a-stay-game/
Context: Phase 4 HemiNorth 2, 28 severe hemophilia A patients switching from FVIII prophylaxis.
Key point: Annual treated bleeds dropped (mean 5.9→1.6); 92% preferred Hemlibra.
Implication: May influence prescriber choice and payer reviews pending full data.
🌐 WFH drives WHO EML hemophilia updates [9] [Global • 12 Sep 2025]
Key point: WHO added emicizumab, recombinant FVIII/FIX, desmopressin to core list; removed cryoprecipitate and PCC.
Implication: Introduces competition that may affect pricing and formulary access.
🧪 Precision BioLogic expands FVIII assay utility [10] [Canada • 10 Sep 2025]
Key point: CRYOcheck FVIII assay validated against interference from emicizumab and Mim8; FDA-cleared.
Implication: Could inform practice and payer discussions; interpretation depends on study design and confounding control.
Why it matters
- Multiple myeloma and AML trial progress signal momentum in next-gen small molecules and combinations.
- BTK pipeline diversification (Jaypirca vs bexobrutideg) reflects strong innovation in CLL resistance management.
- Myelofibrosis combo strategy may create first-in-class dual therapy standard.
- Blood cancer awareness campaigns and WHO EML updates highlight global push for access and earlier diagnosis.
- Hemophilia field shows parallel advances in therapies, diagnostics, and global health policy.
📢 Stay Ahead in Hematology Research!
✅ Contact LucidQuest at info@lqventures.com for strategic guidance on Hematology innovations and clinical research.
FAQ
What is inobrodib’s status in multiple myeloma?
CellCentric [1] completed Phase 2 dose-optimization enrollment; data due by end of 2025 to support registration studies.
How does Jaypirca compare to chemoimmunotherapy in CLL?
In BRUIN CLL-313, Lilly’s Jaypirca [2] significantly improved PFS over bendamustine+rituximab in front-line CLL/SLL.
What are the latest data on bexobrutideg?
At SOHO 2025, bexobrutideg [3] showed 81% ORR and durable benefit in heavily pretreated CLL, including BTK- and BCL2-refractory patients.
What new AML/MDS program did Biosplice launch?
Biosplice [4] dosed the first patient in an NCI-sponsored trial of cirtuvivint, alone or with ASTX727, in relapsed/refractory AML/MDS.
When will Moleculin report AML trial data?
Moleculin [5] expects first MIRACLE trial unblinding (45 patients) in late 2025; larger Part B to follow.
What change did WHO make for hemophilia treatment?
WFH [9] achieved WHO EML revisions: emicizumab and recombinant clotting factors moved to core list; cryoprecipitate removed.
Entities / Keywords
CellCentric; inobrodib; Eli Lilly; Jaypirca; pirtobrutinib; bexobrutideg (NX-5948); Biosplice; cirtuvivint; ASTX727; Moleculin; Annamycin (naxtarubicin); AnnAraC; Karyopharm; selinexor; ruxolitinib; Oxcia; OXC-101; Hemlibra; emicizumab; Roche; World Federation of Hemophilia (WFH); WHO; Essential Medicines List; Precision BioLogic; CRYOcheck; Mim8; multiple myeloma; CLL; SLL; AML; MDS; myelofibrosis; hemophilia A.
References
- https://www.cellcentric.com/press-release/cellcentric-announces-completion-of-enrollment-for-dose-optimization-cohorts-in-phase-2-study-for-multiple-myeloma/
- https://investor.lilly.com/news-releases/news-release-details/lillys-jaypirca-pirtobrutinib-first-and-only-approved-non-0
- https://www.bloodcancerstoday.com/post/novel-btk-degrader-bexobrutideg-shows-high-response-rates-in-relapsed-or-refractory-cll
- https://www.globenewswire.com/news-release/2025/09/09/3146697/0/en/Biosplice-Therapeutics-Announces-First-Patient-Dosed-in-NCI-Sponsored-Clinical-Trial-of-Cirtuvivint-in-Acute-Myeloid-Leukemia-and-Myelodysplastic-Syndromes.html
- https://moleculin.com/moleculin-doses-first-eu-patient-in-phase-3-pivotal-miracle-trial-for-treatment-of-acute-myeloid-leukemia/
- Karyopharm
- https://oxcia.com/blood-cancer-awareness-month/
- https://hemophilianewstoday.com/news/hemlibra-help-active-men-hemophilia-a-stay-game/
- WFHhttps://www.prnewswire.com/news-releases/wfh-mobilizes-global-community-to-achieve-milestone-revisions-to-who-eml-302555143.html
- https://www.prnewswire.com/news-releases/precision-biologic-leads-the-way-in-hemophilia-testing-302551622.htmlPrecision BioLogic