Hematology Today—November 24, 2025

This week’s hematology update spotlights key developments, regulatory milestones, and the latest progress shaping the biopharma landscape.

In Today’s Newsletter

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💡 Soligenix hits FLASH2 interim-analysis enrollment in CTCL [1] [US • 19 Nov 2025]

https://ir.soligenix.com/2025-11-19-Soligenix-Achieves-Enrollment-Milestone-for-Planned-Interim-Analysis-in-Confirmatory-Phase-3-Clinical-Trial-of-HyBryte-TM-for-the-Treatment-of-Cutaneous-T-Cell-Lymphoma
Context: Confirmatory Phase 3, randomized, double-blind, placebo-controlled, ~80 early-stage CTCL; interim at ~60% completing Week 18.
Key point: 50 of 80 enrolled for interim; blinded response rate to date reported above trial estimate; company guides topline in 2H26.
Implication: May influence prescriber choice and payer reviews pending full data.

🧬 EMA positive opinion for ODD on BI-1808 in CTCL [2] [EU • 17 Nov 2025]

https://www.bioinvent.com/en/press/ema-positive-opinion-orphan-drug-designation-bioinvents-bi-1808-treatment-cutaneous-t-cell
Context: Anti-TNFR2 antibody; ongoing Phase 2a shows disease control with responses in advanced CTCL (n not stated here).
Key point: COMP adopted positive opinion for Orphan Drug Designation; EC decision pending.
Implication: Regulatory/generics: Introduces competition that may affect pricing and formulary access.

💉 Lunsumio SC gets EU nod for 3L+ follicular lymphoma [3] [EU • 19 Nov 2025]

https://www.roche.com/media/releases/med-cor-2025-11-19
Context: GO29781 Phase I/II showed SC PK non-inferior to IV; ORR 74.5% and CR 58.5% in 3L+ FL; low-grade CRS profile.
Key point: Conditional marketing authorisation for 3L+ FL; ~1-minute SC injection offers faster administration versus IV.
Implication: Regulatory/generics: Introduces competition that may affect pricing and formulary access.

✅ FDA approves EPKINLY + R2 for R/R FL after ≥1 line [4] [US • 18 Nov 2025]

https://news.abbvie.com/2025-11-18-AbbVie-Announces-U-S-FDA-Approval-of-EPKINLY-R-epcoritamab-bysp-in-Combination-with-Rituximab-and-Lenalidomide-for-Relapsed-or-Refractory-Follicular-Lymphoma
Context: Phase 3 EPCORE FL-1, EPKINLY + R2 vs R2; PFS HR 0.21; ORR 89% vs 74%; CR 74% vs 43%; outpatient SC dosing.
Key point: First bispecific combination therapy approved in lymphoma; converts FL monotherapy to full approval.
Implication: Regulatory/generics: Introduces competition that may affect pricing and formulary access.

🇯🇵 Gazyva label in Japan adds combo with venetoclax for untreated CLL [5] [20 Nov 2025]

https://www.chugai-pharm.co.jp/english/news/detail/20251120150000_1202.html
Context: Based on Japan Phase II M20-353 and global Phase III CLL14; updates electronic package insert.
Key point: Obinutuzumab can be used with venetoclax in previously untreated CD20-positive CLL, including SLL.
Implication: Regulatory/generics: Introduces competition that may affect pricing and formulary access.

🩺 Medunik USA maintains Siklos supply and support in SCD [6] [US • 18 Nov 2025]

https://www.newswire.ca/news-releases/medunik-usa-reaffirms-support-for-sickle-cell-disease-community-with-uninterrupted-supply-and-patient-centered-services-833716365.html
Context: Siklos is FDA-approved dissolvable hydroxyurea for SCD ≥2 years; expanded Medicaid coverage and assistance programs.
Key point: Company affirms uninterrupted Siklos availability amid constraints for some hydroxyurea products; patient services broadened.
Implication: Access programs: May expand screening, initiation, and follow-up at scale.

🧪 Agios mitapivat Phase 3 in SCD meets Hb response, misses SCPC primary [7] [US • 19 Nov 2025]

https://investor.agios.com/news-releases/news-release-details/agios-announces-topline-results-rise-phase-3-trial-mitapivat
Context: RISE UP Phase 3, 207 patients, 52-week double-blind; 2 primary endpoints, 5 key secondary endpoints.
Key point: Significant hemoglobin response and hemolysis biomarker improvements; SCPC rate reduction trend not statistically significant; US filing planned.
Implication: Clinical topline/efficacy: May influence prescriber choice and payer reviews pending full data.

👶 THROMBATE III US label expanded to pediatrics with hATd [8] [US • 18 Nov 2025]

https://www.grifols.com/en/view-news/-/news/grifols-receives-expanded-indication-for-thrombate-iii-antithrombin-iii-human-label-in-us-strengthening-treatment-options-for-pediatric-patients
Context: FDA approval based on extrapolated adult data from two trials; first AT concentrate approved for both adults and children with hATd.
Key point: Addresses treatment gap for pediatric hereditary antithrombin deficiency.
Implication: Regulatory/generics: Introduces competition that may affect pricing and formulary access.

🏭 CSL to invest ~US$1.5b in US plasma-therapy manufacturing [9] [US • 18 Nov 2025]

https://newsroom.csl.com/2025-11-18-CSL-to-invest-approximately-1-5b-in-U-S-to-manufacture-plasma-derived-therapies
Context: Five-year US build-out, subject to board approval; builds on >US$3b invested since 2018.
Key point: Expansion to strengthen onshore plasma-derived therapy capacity and supply chain, with new US jobs.
Implication: Partnerships/BD: Signals pipeline investment and modality expansion.

🧒 Hemlibra maintains joint health over 3 years in pediatric HemA, Japan study [10] [21 Nov 2025]

https://hemophilianewstoday.com/news/3-years-hemlibra-keeps-joints-healthy-kids-hemophilia-a/
Context: AOZORA post-marketing study interim, 30 boys ≤11 years, Japan; MRI and clinical joint assessments.
Key point: Safety consistent; many showed maintained or improved joint health, with reduced treated bleeds over 3 years.
Implication: Observational/RWE: Could inform practice and payer discussions; interpretation depends on study design and confounding control.

Why it matters

Bispecifics in FL advanced in parallel: US combo approval for EPKINLY + R2 and EU SC formulation approval for Lunsumio, adding options and convenience.
CTCL momentum spans photodynamic therapy milestone at Soligenix and anti-TNFR2 ODD progress at BioInvent.
Japan frontline CLL options modernize with venetoclax + obinutuzumab reflected in labeling.
SCD landscape mixes access and efficacy signals: Siklos supply reinforced while mitapivat shows anti-hemolytic gains without SCPC significance.
Pediatric access strengthened via THROMBATE III label expansion and Hemlibra long-term joint health data.

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FAQ

What did the EPCORE FL-1 trial show for EPKINLY + R2?

It significantly improved PFS and ORR versus R2, with CR in about three-quarters of patients; now FDA-approved for R/R FL after ≥1 prior line [4].

How does Lunsumio SC differ from IV in EU-approved 3L+ FL?

SC demonstrated PK non-inferiority and high ORR/CR, delivered via ~1-minute injection, reducing chair time versus 2–4 hour infusions [3].

What is the status of HyBryte in CTCL?

Soligenix reached 50 of 80 patients for the confirmatory Phase 3 interim; blinded response to date exceeds estimate; company guides topline in 2H26 [1].

What does EMA’s positive opinion on BI-1808 mean?

COMP issued a positive opinion for ODD in CTCL; EC decision pending, which could unlock incentives if granted [2].

Did mitapivat work in the RISE UP SCD trial?

It met the hemoglobin-response primary and improved hemolysis markers; SCPC reduction was a non-significant trend. Agios plans US filing after FDA discussion [7].

What label changes landed this week?

Japan added obinutuzumab + venetoclax in untreated CLL, and the US expanded THROMBATE III to pediatrics with hereditary antithrombin deficiency [5][8].

Entities / Keywords

Soligenix; HyBryte, SGX301; CTCL; FLASH2. BioInvent; BI-1808; TNFR2; Orphan Drug Designation; EMA. Roche; Lunsumio, mosunetuzumab; subcutaneous; follicular lymphoma. AbbVie; EPKINLY, epcoritamab; rituximab; lenalidomide; EPCORE FL-1. Chugai; Nippon Shinyaku; Gazyva, obinutuzumab; Venclexta, venetoclax; CLL14. Medunik USA; Duchesnay; Siklos, hydroxyurea; SCD. Agios; mitapivat; RISE UP; SCPC; PROMIS Fatigue. Grifols; THROMBATE III; hereditary antithrombin deficiency. CSL; plasma-derived therapies. Hemlibra, emicizumab; AOZORA; pediatric HemA.