This weekly Hematology update covers key developments including late-stage clinical progress, regulatory actions, and new data disclosures. Highlights include pivotal trial activity, designation and review outcomes, and policy efforts to expand access and equity.
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🧬 Senti Biosciences completes Phase 1 enrollment for SENTI-202 in R/R AML [1] [US • 11 Feb 2026]
Context: Phase 1 trial of SENTI-202 (CD33/FLT3-targeting logic-gated CAR-NK); RMAT designation noted (trial identifier cited as NCT06325748).
Key point: Senti Bio says enrollment is complete and it plans FDA discussions in H1 2026 on a potential pivotal program and possible indication expansion (endpoint details not specified).
Implication: May influence prescriber choice and payer reviews pending full data.
🧪 Yatiri Bio reports blinded prediction concordance for Foghorn’s FHD-286 in R/R AML or MDS [2] [10 Feb 2026]
https://finance.yahoo.com/news/yatiri-bio-accurately-predicts-aml-110000821.html
Context: Blinded retrospective validation using eight pre-treatment PBMC samples from a Phase 1 trial of FHD-286 (camibirstat) (trial identifier cited as NCT04891757).
Key point: Yatiri Bio reports 100% concordance between ProteoCharts™ predictions and observed clinical responses in this small sample set and says a collaboration agreement with Foghorn was signed (terms partly disclosed).
Implication: Could inform practice and payer discussions; interpretation depends on study design and confounding control.
🧫 Gyala Therapeutics initiates Phase I/IIa GYA01 (CD84 CAR-T) in R/R AML and T-ALL [3] [EU • 10 Feb 2026]
Context: Open-label, single-arm, interventional study with dose-escalation then expansion, authorized by Spain’s AEMPS; sites include Hospital La Fe (Valencia) and Hospital Clínic Barcelona.
Key point: Gyala says GYA01, a CD84-targeting CAR-T, will be evaluated for safety, tolerability, and preliminary efficacy in acute leukemias with limited options.
Implication: Signals pipeline investment and modality expansion.
🧪 Lyell Immunopharma doses first patient in Phase 3 head-to-head CAR T trial in 2L aggressive LBCL [4] [US/Canada/Australia • 12 Feb 2026]
Context: PiNACLE – H2H randomizes rondecabtagene autoleucel (ronde-cel, LYL314; CD19/CD20) vs investigator’s choice of lisocabtagene maraleucel (liso-cel) or axicabtagene ciloleucel (axi-cel); primary endpoint is event-free survival.
Key point: Lyell reports first patient dosing and frames the trial as the first to randomize patients between different approved CAR T therapies (per company statement).
Implication: May influence prescriber choice and payer reviews pending full data.
🧫 Elicera Therapeutics: ISCT 2026 abstract accepted, early CARMA signal for ELC-301 [5] [EU • 11 Feb 2026]
Context: CARMA Phase I/IIa evaluates ELC-301 (CD20 CAR T) “iTANK-armed”; ISCT 2026 Dublin runs 06–09 May 2026.
Key point: Elicera reports complete metabolic response in four of six treated patients across the first two cohorts (additional efficacy and safety details not specified in the release).
Implication: May influence prescriber choice and payer reviews pending full data.
🧾 Sanofi: rilzabrutinib gets FDA Breakthrough Therapy (US) and orphan designation (Japan) for wAIHA [6] [US/Japan • 09 Feb 2026]
https://www.sanofi.com/en/media-room/press-releases/2026/2026-02-09-06-00-00-3234232
Context: Based on data from the ongoing LUMINA 2 Phase 2b study (NCT05002777); LUMINA 3 Phase 3 is described (NCT07086976).
Key point: Sanofi says rilzabrutinib (Wayrilz, where approved for ITP) received US Breakthrough Therapy designation and Japan orphan designation for warm autoimmune hemolytic anemia.
Implication: Introduces competition that may affect pricing and formulary access.
📩 Disc Medicine: FDA Complete Response Letter for bitopertin NDA in EPP, APOLLO Phase 3 results requested [7] [US • 13 Feb 2026]
Context: NDA was for accelerated approval; FDA agreed AURORA and BEACON showed lowered metal-free PPIX, but did not see evidence of association with sunlight exposure-based endpoints as measured.
Key point: Disc says FDA issued a CRL and indicated it needs Phase 3 APOLLO results before making a decision; company cites topline data anticipated Q4 2026.
Implication: May influence prescriber choice and payer reviews pending full data.
🩸 Chugai: NXT007 Phase I/II data in hemophilia A switching from emicizumab without washout [8] [Japan • 09 Feb 2026]
https://www.chugai-pharm.co.jp/english/news/detail/20260209170000_1225.html
Context: NXTAGE Part C evaluated multiple ascending doses in people aged 12 to <65 years switching from emicizumab; interim analysis described as 14 participants with ≥16 weeks exposure; presented at EAHAD 2026.
Key point: Chugai reports favorable tolerability during direct switching, and no bleeds requiring treatment in the highest-dose cohorts (as reported); Phase 3 studies are planned in 2026.
Implication: May influence prescriber choice and payer reviews pending full data.
🌍 WHO executive board resolution aims to reduce hemophilia care gaps, ahead of May vote [9] [Global • 13 Feb 2026]
https://hemophilianewstoday.com/news/who-resolution-aims-address-gaps-hemophilia-care/
Context: Article describes a resolution to be presented to the World Health Assembly for adoption in May, including focus on underdiagnosis and access inequities, especially for women and girls.
Key point: The resolution calls on member states to strengthen awareness, diagnosis, treatment access, and care systems for hemophilia and other bleeding disorders, with a detailed plan targeted by 2028.
Implication: Access programs: May expand screening, initiation, and follow-up at scale.
Why it matters
- Multiple cell therapy programs are advancing, including enrollment completion, new trial starts, and pivotal head-to-head positioning.
- The Disc CRL highlights ongoing scrutiny of surrogate endpoints and the need to show linkage to clinical benefit as measured.
- Hemophilia updates span both next-gen switching strategies (NXT007) and global policy moves (WHO), with potential downstream effects on care delivery.
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FAQ
What is SENTI-202 (Senti Bio) and what is the update?
SENTI-202 is a logic-gated CAR-NK targeting CD33 and FLT3 in AML. Senti says Phase 1 enrollment is complete and FDA discussions are planned in H1 2026. [1]
What exactly did Yatiri Bio claim about FHD-286 prediction?
Yatiri Bio reports 100% concordance between predictions from blinded pre-treatment samples and observed outcomes in eight Phase 1 trial samples for FHD-286, in a retrospective analysis. [2]
What is Gyala’s GYA01 trial testing?
Gyala says GYA01 is a CD84-targeting CAR-T being evaluated in an open-label Phase I/IIa trial for relapsed or refractory AML and T-ALL in Spain. [3]
Why did the FDA issue a CRL for bitopertin in EPP (Disc)?
Disc says FDA agreed bitopertin lowered metal-free PPIX but did not see evidence linking PPIX changes to sunlight exposure-based endpoints as measured, and requested APOLLO Phase 3 results. [7]
What did Chugai report about switching from emicizumab to NXT007?
Chugai reports favorable tolerability when switching without washout, and no bleeds requiring treatment in the highest-dose cohorts (per the company’s Part C summary). [8]
Entities / Keywords
Senti Biosciences (Senti Bio), SENTI-202, CAR-NK, CD33, FLT3, AML, RMAT
Yatiri Bio, ProteoCharts™, biomarkers, AI proteomics, Foghorn Therapeutics, FHD-286 (camibirstat), AML, MDS
Gyala Therapeutics, GYA01, CD84, CAR-T, AML, T-ALL, AEMPS
Lyell Immunopharma, rondecabtagene autoleucel (ronde-cel, LYL314), CD19/CD20, PiNACLE – H2H, liso-cel, axi-cel, LBCL
Elicera Therapeutics, ELC-301, iTANK, CARMA, ISCT 2026
Sanofi, rilzabrutinib (Wayrilz), BTK inhibitor, wAIHA, LUMINA 2, LUMINA 3
Disc Medicine, bitopertin, EPP, CRL, APOLLO, PPIX
Chugai Pharmaceutical, NXT007, emicizumab, NXTAGE, hemophilia A
WHO, World Health Assembly, bleeding disorders, access equity
References
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https://finance.yahoo.com/news/yatiri-bio-accurately-predicts-aml-110000821.html
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https://www.sanofi.com/en/media-room/press-releases/2026/2026-02-09-06-00-00-3234232
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https://www.chugai-pharm.co.jp/english/news/detail/20260209170000_1225.html
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https://hemophilianewstoday.com/news/who-resolution-aims-address-gaps-hemophilia-care/