Hematology Today—May 18, 2026

This week’s Hematology update highlights regulatory progress, clinical advancements, registrational planning, and emerging in vivo genetic medicine platforms.

In Today’s Newsletter

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🩸 Agios mitapivat sNDA in sickle cell disease [1] [US • 12 May 2026]

https://investor.agios.com/news-releases/news-release-details/agios-submits-snda-fda-us-accelerated-approval-mitapivat-sickle
Context: The confirmatory trial is designed to test transfusion burden reduction, with transfusion-free status from Week 4 through Week 52 as the primary endpoint.
Key point: Agios submitted an FDA sNDA seeking accelerated approval for mitapivat, an oral PK activator, in sickle cell disease.
Implication: May influence prescriber choice and payer reviews pending full data.

💉 Pfizer HYMPAVZI EC approval in hemophilia with inhibitors [2] [EU • 13 May 2026]

https://www.pfizer.com/news/press-release/press-release-detail/european-commission-approves-pfizers-hympavzi-treatment
Context: Phase 3 BASIS data showed superior bleed reduction versus on-demand therapy, with continued benefit in open-label extension.
Key point: The European Commission expanded HYMPAVZI (marstacimab) for patients 12 years and older with hemophilia A or B with inhibitors.
Implication: May influence prescriber choice and payer reviews pending full data.

💊 Taiho INQOVI plus venetoclax FDA approval in AML [3] [US • 14 May 2026]

https://www.taiho.co.jp/en/release/2026/20260514.html
Context: Approval was supported by ASCERTAIN-V, which evaluated complete remission and duration of complete remission.
Key point: FDA approved INQOVI (decitabine and cedazuridine) plus venetoclax for newly diagnosed AML patients who are older or ineligible for intensive induction chemotherapy.
Implication: Could streamline initiation and adherence via an all-oral regimen.

🧬 Mirai Bio CD8-targeted LNP NHP proof-of-concept [4] [US • 12 May 2026]

https://www.miraibio.com/news/mirai-bio-presents-data-showing-rapid-advancement-of-novel-cd8-t-cell-targeted-lnps-to-functional-nhp-proof-of-concept-at-asgct-2026
Context: Systemic CAR mRNA delivery generated CAR-expressing CD8+ T cells and drove B-cell depletion in non-human primates.
Key point: Mirai Bio reported functional NHP proof-of-concept for CD8-targeted LNPs designed for in vivo RNA CAR-T programs.
Implication: Signals pipeline investment and modality expansion.

🧪 Moleculin MIRACLE trial unblinding update in R/R AML [5] [US • 13 May 2026]

https://moleculin.com/moleculin-announces-imminent-miracle-trial-unblinding-as-blinded-data-continue-to-significantly-outperform-historical-benchmarks/
Context: MIRACLE is evaluating Annamycin plus cytarabine versus cytarabine plus placebo in relapsed or refractory AML. Key point: Moleculin said first 45-subject data unblinding is expected before 30 Jun 2026.
Implication: May influence prescriber choice and payer reviews pending full data.

🧫 Sana SG293 in vivo CAR-T NHP data [6] [US • 12 May 2026]

https://ir.sana.com/news-releases/news-release-details/sana-biotechnology-presents-preclinical-data-vivo-car-t-cell
Context: SG293 is a CD8-targeted fusosome that delivers genetic material to make CD19-directed CAR T cells, without lymphodepleting chemotherapy.
Key point: Sana presented NHP data for surrogate SG293 showing CD8-targeted in vivo CAR-T generation and B-cell depletion.
Implication: Signals pipeline investment and modality expansion.

✍️ Tessera Gene Writing updates in SCD and CAR-T [7] [US • 14 May 2026]

https://www.tesseratherapeutics.com/news/tessera-therapeutics-showcases-new-preclinical-data-advancing-in-vivo-program-in-sickle-cell-disease-and-car-t-applications-at-the-american-society-of-gene-and-cell-therapy-29th-annual-meeting
Context: Data included HBB editing in LT-HSCs and CD20 CAR-T generation after LNP-delivered Gene Writer dosing.
Key point: Tessera reported NHP data for in vivo Gene Writing in sickle cell disease and CAR-T applications.
Implication: Signals pipeline investment and modality expansion.

🧬 Senti Bio SENTI-202 RMAT and registrational strategy [8] [US • 14 May 2026]

https://investors.sentibio.com/news-releases/news-release-details/senti-biosciences-holdings-announces-positive-fda-rmat-meeting
Context: SENTI-202 is an off-the-shelf Logic Gated CAR-NK cell therapy, and future manufacturing will use Donor X material.
Key point: Senti Bio completed an FDA RMAT Type B meeting and plans a single-arm, multi-center registrational trial for SENTI-202 in relapsed or refractory AML.
Implication: May influence prescriber choice and payer reviews pending full data.

🛡️ BioInvent BI-1808 CTCL immune-activation data [9] [Sweden • 12 May 2026]

https://www.bioinvent.com/en/press/bioinvents-tnfr2-antibody-bi-1808-shows-strong-activity-and-immune-activation-single-agent
Context: BI-1808 showed objective responses as monotherapy and with pembrolizumab, with immune activation and mostly mild to moderate treatment-related adverse events.
Key point: BioInvent reported Phase 2a activity for BI-1808, an anti-TNFR2 antibody, in advanced CTCL.
Implication: May influence prescriber choice and payer reviews pending full data.

Why it matters

Hematology remains active across regulatory submissions, approvals, and registrational planning in sickle cell disease, hemophilia, AML, and CTCL.
In vivo cell and genetic medicine platforms are moving toward functional NHP readouts, including T-cell targeting and B-cell depletion.
AML remains a major development focus, spanning approved oral regimens, anthracycline combinations, and off-the-shelf CAR-NK approaches.
Delivery is a central theme, with LNP, fusosome, Gene Writing, and CAR-NK platforms all positioned around faster or more scalable treatment models.

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FAQ

What did Agios submit for mitapivat in sickle cell disease?

Agios submitted an FDA sNDA seeking accelerated approval for mitapivat, an oral PK activator. The confirmatory trial is designed around transfusion burden reduction. [1]

What changed for Pfizer’s HYMPAVZI in Europe?

The EC expanded HYMPAVZI’s indication to include patients 12 years and older with hemophilia A or B with inhibitors. The approval relied on Phase 3 BASIS data. [2]

Why is Taiho’s INQOVI plus venetoclax approval notable?

Taiho described INQOVI plus venetoclax as the first all-oral combination approved for newly diagnosed AML patients who are older or ineligible for intensive induction chemotherapy. [3]

What is the common thread across Mirai Bio, Sana, and Tessera?

All three reported preclinical in vivo delivery or CAR-T generation data, including NHP evidence of T-cell targeting, CAR-T generation, or B-cell depletion. [4], [6], [7]

What is Senti Bio changing for SENTI-202 manufacturing?

Senti Bio said future SENTI-202 manufacturing, including pivotal study use, will use Donor X material after exploratory analysis linked that phenotype with higher response. [8]

What did BioInvent report for BI-1808 in CTCL?

BioInvent reported Phase 2a activity for BI-1808 as monotherapy and with pembrolizumab, plus immune activation markers in advanced CTCL. [9]

Entities / Keywords

Agios Pharmaceuticals, mitapivat, PK activator, sickle cell disease, sNDA, FDA accelerated approval, RISE UP
Pfizer, HYMPAVZI, marstacimab, hemophilia A, hemophilia B, inhibitors, European Commission, BASIS
Taiho Pharmaceutical, Taiho Oncology, INQOVI, decitabine, cedazuridine, venetoclax, AML, ASCERTAIN-V
Mirai Bio, CD8-targeted LNP, mRNA, CD20 CAR, NHP, in vivo RNA CAR-T, ASGCT
Moleculin Biotech, Annamycin, naxtarubicin, cytarabine, MIRACLE, R/R AML
Sana Biotechnology, SG293, SG227, fusosome, CD19 CAR T, BCMA, NHL, multiple myeloma
Tessera Therapeutics, Gene Writing, HBB, LT-HSC, sickle cell disease, CD20 CAR-T, targeted LNP
Senti Biosciences, SENTI-202, CAR-NK, Gene Circuit, RMAT, Donor X, R/R AML
BioInvent, BI-1808, TNFR2, CTCL, mycosis fungoides, Sézary syndrome, pembrolizumab, KEYTRUDA