Explore this week’s Hematology highlights, including FDA updates, partnerships and acquisitions, new developments, pipeline updates, and progress from industry players.
In Today’s Newsletter
💡 Sonrotoclax gets FDA Breakthrough, joins Project Orbis [1] [US • 13 Oct 2025]
Context: Based on Phase 1/2 BGB-11417-201 topline, Phase 3 CELESTIAL-RRMCL underway. Additional Fast Track and Orphan designations noted in source.
Key point: FDA granted Breakthrough Therapy Designation to sonrotoclax for adult R/R MCL after BTKi and anti-CD20. FDA accepted participation in Project Orbis.
Implication: May influence prescriber choice and payer reviews pending full data.
🧬 Minovia nabs FDA Orphan Drug Designation for MNV-201 in MDS [2] [US • 15 Oct 2025]
https://finance.yahoo.com/news/minovia-therapeutics-receives-fda-orphan-113000608.html
Context: Program also holds Fast Track and Rare Pediatric Disease designations in Pearson syndrome. Company disclosed a planned SPAC transaction.
Key point: FDA granted Orphan Drug Designation to MNV-201 in myelodysplastic syndrome, adding to existing Fast Track in MDS.
Implication: Regulatory incentives could accelerate development and access.
🧪 Amphista nominates AMX-883, oral BRD9 degrader, for AML [3] [UK • 15 Oct 2025]
Context: Preclinical data show selective, rapid BRD9 degradation and in vivo activity. IND planned early 2026, first-in-human H2 2026.
Key point: AMX-883 selected as Amphista’s first clinical candidate for AML, a Targeted Glue degrader that recruits DCAF16 rather than CRBN or VHL.
Implication: Signals pipeline investment and modality expansion.
🤝 Alanis partners with Blood Cancer United’s Beat AML on Notch-1/Jagged-1 [4] [US • 15 Oct 2025]
Context: ATI-D1, an anti-Jagged-1 antibody, is Alanis’s lead for MDS/AML, internal analyses suggest pathway-linked activity.
Key point: Collaboration will profile Notch-1/Jagged-1 signaling across AML molecular subtypes using Beat AML Master Trial samples.
Implication: Signals pipeline investment and modality expansion.
🧫 Rocket’s KRESLADI BLA resubmission accepted, PDUFA set [5] [US • 14 Oct 2025]
Context: Global Phase 1/2 met all primary and secondary endpoints, with 100% overall survival at 12 months post-infusion, and was well tolerated (per source).
Key point: FDA accepted the resubmitted BLA for KRESLADI (marne-cel) in severe LAD-I, with a PDUFA target action date of 28 Mar 2026.
Implication: May influence prescriber choice and payer reviews pending full data.
🧑⚕️ Soligenix refreshes US CTCL Medical Advisory Board for HyBryte [6] [US • 14 Oct 2025]
Context: Board comprises dermatology and oncology KOLs with HyBryte experience, advising on clinical strategy, potential home-use, and reimbursement.
Key point: Company updated its US Medical Advisory Board to guide Phase 3 HyBryte clinical development in CTCL.
Implication: Signals pipeline investment and modality expansion.
💼 Novo Nordisk to acquire and license zaltenibart from Omeros, up to $2.1B [7] [15 Oct 2025]
Context: Novo plans a global PNH program and will explore other rare blood and kidney disorders per source.
Key point: Novo Nordisk agreed to an asset purchase and license for Omeros’s MASP-3 inhibitor zaltenibart, up to $2.1B in payments including $340M upfront and near-term milestones.
Implication: Signals pipeline investment and modality expansion.
🧲 AstraZeneca adds a second Phase 3 trial for surovatamig in first-line LBCL [8] [16 Oct 2025]
https://www.oncologypipeline.com/apexonco/astra-pulls-further-ahead-mercks-curon-project
Context: Joins an existing Phase 3 in first-line FL. Phase 1 showed dose-dependent ORRs with noted neutropenia and ICANS.
Key point: A second Phase 3 trial will start in Nov 2025 in first-line LBCL (elderly or unfit), testing surovatamig + R-mini-CHOP versus R-mini-CHOP.
Implication: May influence prescriber choice and payer reviews pending full data.
🩸 Galleri multi-cancer blood test, Pathfinder 2 prospective study results [9] [UK • 18 Oct 2025]
https://news.sky.com/story/new-blood-test-for-more-than-50-cancers-could-transform-outcomes-13452132
Context: Observational analysis in more than 23,000 adults in the US and Canada, experts call for mortality impact evidence before screening adoption.
Key point: Pathfinder 2, a large prospective study in asymptomatic adults, reported 61.6% confirmation among signal-positive cases, 99.6% specificity, and 92% tissue-of-origin accuracy.
Implication: Could inform practice and payer discussions, interpretation depends on study design and confounding control.
Why it matters
- Parallel advances in apoptosis, targeted protein degradation, and T-cell engagement broaden hematology options.
- FDA designations and a gene therapy BLA milestone mark clearer regulatory paths in rare disorders.
- Complement-pathway BD signals sustained investment in PNH and renal–hematologic disease.
- Advisory and collaboration updates aim to tighten trial design, stratification, and real-world translation.
- Early detection blood tests may alter screening paradigms if outcome benefits are proven.
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FAQ
What exactly did FDA grant for sonrotoclax?
Breakthrough Therapy Designation for adult R/R mantle cell lymphoma after BTKi and anti-CD20, with Project Orbis participation approved based on Phase 1/2 data [1].
Which FDA designations does MNV-201 hold?
In MDS, Orphan Drug Designation and Fast Track. For Pearson syndrome, Fast Track and Rare Pediatric Disease designations are noted [2].
When is FDA action expected for KRESLADI?
PDUFA target action date is 28 Mar 2026 for severe LAD-I following acceptance of the resubmitted BLA [5].
What is zaltenibart and how will Novo Nordisk develop it?
Zaltenibart is a MASP-3 inhibitor for the alternative complement pathway. Novo plans a global PNH program and may explore other rare indications per the agreement [7].
What is distinct about AMX-883?
AMX-883 is a DCAF16-recruiting Targeted Glue degrader of BRD9, avoiding CRBN or VHL, with selective degradation and preclinical efficacy in AML models [3].
What is changing in AstraZeneca’s surovatamig program?
A second Phase 3 trial will evaluate surovatamig plus R-mini-CHOP in first-line LBCL for elderly or unfit patients, adding to the existing Phase 3 in FL [8].
What is Pathfinder 2 in the Galleri news?
Pathfinder 2 is a prospective, real-world study in >23,000 asymptomatic adults assessing Galleri’s signal performance and workflow integration, reporting specificity of 99.6% and tissue-of-origin accuracy of 92% [9].
Entities / Keywords
BeOne Medicines, sonrotoclax, BCL2 inhibitor, mantle cell lymphoma, Project Orbis; Minovia Therapeutics, MNV-201, mitochondrial cell therapy, myelodysplastic syndrome, Pearson syndrome; Amphista Therapeutics, AMX-883, BRD9, DCAF16, Targeted Glue, AML; Alanis Therapeutics, ATI-D1, Jagged-1, Notch-1, Beat AML Master Trial; Rocket Pharmaceuticals, KRESLADI, marne-cel, LAD-I, gene therapy, Priority Review Voucher; Soligenix, HyBryte, synthetic hypericin, CTCL, Medical Advisory Board; Novo Nordisk, Omeros, zaltenibart, OMS906, MASP-3, PNH, complement pathway; AstraZeneca, surovatamig, AZD0486, CD19 T-cell engager, LBCL, FL, R-mini-CHOP; Grail, Galleri, multi-cancer early detection, Pathfinder 2, specificity, tissue of origin.
References
- https://www.businesswire.com/news/home/20251013125479/en/BeOne-Medicines-Sonrotoclax-Granted-Breakthrough-Therapy-Designation-by-U.S.-FDA
- https://finance.yahoo.com/news/minovia-therapeutics-receives-fda-orphan-113000608.html
- https://amphista.com/amphista-therapeutics-nominates-amx-883-an-orally-available-targeted-glue-degrader-of-brd9-as-its-first-clinical-development-candidate-for-the-treatment-of-acute-myeloid-leukaemia/
- https://lifestyle.utv.ie/story/185549/alanis-therapeutics-announces-collaboration-with-blood-cancer-uniteds-beat-aml-to-advance-therapeutic-for-mdsaml/
- https://ir.rocketpharma.com/news-releases/news-release-details/rocket-pharmaceuticals-announces-fda-acceptance-bla-resubmission
- https://ir.soligenix.com/2025-10-14-Soligenix-Updates-United-States-Medical-Advisory-Board-for-Cutaneous-T-Cell-Lymphoma
- https://www.globenewswire.com/news-release/2025/10/15/3167091/0/en/Novo-Nordisk-and-Omeros-announce-asset-purchase-and-license-agreement-for-Omeros-clinical-stage-MASP-3-inhibitor-zaltenibart-OMS906.html
- https://www.oncologypipeline.com/apexonco/astra-pulls-further-ahead-mercks-curon-project
- https://news.sky.com/story/new-blood-test-for-more-than-50-cancers-could-transform-outcomes-13452132