Hematology Update: Cemsidomide–Elranatamab Combo, Ziftomenib AML Trial, Fast Track CAR T & More
From C4–Pfizer’s cemsidomide–elranatamab partnership in myeloma to Kura–Kyowa Kirin’s first AML patient on ziftomenib plus quizartinib, this week’s update spans key milestones. Verismo treated its first follicular lymphoma patient with SynKIR-310, Umoja gained Fast Track for UB-VV111 CAR T, and AbbVie filed a BLA for PVEK in BPDCN. Added highlights include Novo Nordisk’s Mim8 filing in hemophilia A, Chugai’s PiaSky launch in Taiwan, and Star Therapeutics raising $125M to advance VGA039.
In Today’s Newsletter
🧩 C4 Therapeutics & Pfizer collaborate on multiple myeloma combo trial [1] [US • 01 Oct 2025]
https://ir.c4therapeutics.com/news-releases/news-release-details/c4-therapeutics-announces-clinical-trial-collaboration-and
Context: Phase 1b study (launch Q2 2026) to evaluate cemsidomide + elranatamab + dexamethasone in relapsed/refractory multiple myeloma.
Key point: Pfizer will supply elranatamab (ELREXFIO®) free of charge; C4 Therapeutics sponsors the trial.
Implication: Signals pipeline partnership and modality expansion.
🧬 Kura Oncology and Kyowa Kirin launch FLT3/NPM1 AML trial [2] [US • 01 Oct 2025]
https://ir.kuraoncology.com/news-releases/news-release-details/kura-oncology-and-kyowa-kirin-launch-clinical-trial-evaluating
Context: KOMET-007 trial tests ziftomenib + quizartinib with chemotherapy in newly diagnosed FLT3/NPM1-mutated AML.
Key point: Ziftomenib is the only menin inhibitor with FDA Breakthrough Therapy designation in relapsed/refractory NPM1-mutated AML.
Implication: May influence prescriber choice and payer reviews pending full data.
🧫 Verismo Therapeutics treats first follicular lymphoma patient in CELESTIAL-301 [3] [US • 02 Oct 2025]
https://www.prnewswire.com/news-releases/verismo-therapeutics-treats-first-follicular-lymphoma-patient-in-celestial-301-trial-strengthening-collaboration-with-ifli-302573330.html
Context: Phase 1 trial of SynKIR-310 (KIR-CAR T) for relapsed/refractory B-cell lymphomas.
Key point: First FL patient treated; study includes post-CAR T and CAR-naïve cohorts; supported by IFLI.
Implication: May influence prescriber choice and payer reviews pending full data.
💡 Soligenix expands European advisory board for HyBryte™ program [4] [EU • 30 Sep 2025]
https://ir.soligenix.com/2025-09-30-Soligenix-Announces-Expansion-of-European-Medical-Advisory-Board-for-Cutaneous-T-Cell-Lymphoma
Context: Supports confirmatory Phase 3 FLASH2 study of HyBryte™ (synthetic hypericin) for cutaneous T-cell lymphoma.
Key point: Board expands to five European CTCL experts for clinical and regulatory guidance.
Implication: Signals pipeline investment and modality expansion.
🧠 Umoja Biopharma wins FDA Fast Track for in vivo CAR T therapy UB-VV111 [5] [US • 30 Sep 2025]
https://www.umoja-biopharma.com/news/#Press-Releases
Context: UB-VV111 targets CD19+ B-cell malignancies via in vivo CAR T generation; Phase 1 ongoing (NCT06528301).
Key point: Designation covers relapsed/refractory LBCL and CLL after ≥ 2 lines of therapy; AbbVie holds option to license.
Implication: May influence prescriber choice and payer reviews pending full data.
💉 AbbVie submits BLA for Pivekimab sunirine (PVEK) in BPDCN [6] [US • 30 Sep 2025]
https://news.abbvie.com/2025-09-30-AbbVie-Submits-Biologics-License-Application-BLA-to-U-S-FDA-for-Pivekimab-sunirine-PVEK-an-Investigational-Antibody-Drug-Conjugate-ADC-to-Treat-Rare-Cancer-with-Limited-Treatment-Options
Context: Submission based on Phase 1/2 CADENZA trial in blastic plasmacytoid dendritic cell neoplasm.
Key point: PVEK targets CD123 and has FDA Breakthrough designation for relapsed/refractory BPDCN.
Implication: May influence prescriber choice and payer reviews pending full data.
💠 Chugai launches PiaSky in Taiwan for PNH [7] [TW • 01 Oct 2025]
https://www.chugai-pharm.co.jp/english/news/detail/20251001153000_1188.html
Context: PiaSky (anti-C5 Recycling Antibody™) approved for patients ≥ 13 years old with PNH.
Key point: First subcutaneous PNH therapy in Taiwan; administered every 4 weeks.
Implication: May expand screening, initiation and follow-up at scale.
🩸 Novo Nordisk submits BLA for Mim8 (denecimig) in hemophilia A [8] [US • 29 Sep 2025]
https://www.prnewswire.com/news-releases/novo-nordisk-submits-biologics-license-application-bla-to-fda-for-mim8-an-investigational-prophylaxis-treatment-for-people-living-with-hemophilia-a-with-or-without-inhibitors-302568838.html
Context: BLA covers prophylaxis for hemophilia A with or without inhibitors; data from FRONTIER program.
Key point: Denecimig is a next-generation FVIIIa mimetic bispecific antibody with flexible dosing (weekly–monthly).
Implication: May influence prescriber choice and payer reviews pending full data.
🌍 Kenya, Uganda and Côte d’Ivoire partner with Terumo BCT on sickle cell care [9] [AF • 30 Sep 2025]
https://www.terumobct.com/en/gl/our-company/news-and-events/news-releases/kenya–uganda-and-cote-d-ivoire-partner-with-terumo-blood-and-ce.html
Context: Three MoUs signed to create coordinated national frameworks for sickle cell disease management.
Key point: Partnerships span public health policy, research and training to reduce child mortality.
Implication: May expand screening, initiation and follow-up at scale.
💰 Star Therapeutics raises $125 M Series D to advance VGA039 for VWD [10] [US • 30 Sep 2025]
https://star-therapeutics.com/star-therapeutics-announces-oversubscribed-125-million-series-d-financing/
Context: Funding co-led by Sanofi Ventures and Viking Global Investors.
Key point: Proceeds support Phase 3 study of VGA039, a Protein S-targeting antibody for von Willebrand disease.
Implication: Signals pipeline investment and modality expansion.
Why it matters
- Targeted-degrader and menin-inhibitor partnerships show momentum in precision oncology.
- First-in-class cell and antibody therapies are advancing toward regulatory milestones (Fast Track, BLA).
- Asia launches (PiaSky) and Africa frameworks (Terumo BCT) underscore regional expansion of rare-disease care.
- Hematology remains a magnet for venture capital with $125 M financing at Star Therapeutics.
- Multiple Phase 3 programs expected to report in 2026 (Soligenix, C4T, Star).
📢 Stay Ahead in Hematology Research!
✅ Contact LucidQuest at info@lqventures.com for strategic guidance on Hematology innovations and clinical research.
FAQ
What is the goal of C4 Therapeutics’ trial with Pfizer?
A Phase 1b study will assess safety and dose of cemsidomide plus elranatamab for relapsed/refractory multiple myeloma [1].
How does Kura’s ziftomenib fit into AML therapy?
Ziftomenib is a menin inhibitor targeting NPM1 and KMT2A mutations; the new trial tests it with FLT3 inhibition to improve frontline outcomes [2].
What makes Umoja’s UB-VV111 distinct from traditional CAR T therapies?
It creates CAR T cells directly in vivo using the VivoVec™ system, avoiding ex vivo manufacturing delays [5].
Why is Chugai’s PiaSky launch notable?
It is Taiwan’s first subcutaneous therapy for PNH, offering less frequent dosing and potential coverage for C5 mutations [7].
What is Star Therapeutics’ VGA039 designed to do?
VGA039 targets Protein S to restore clotting balance in von Willebrand disease and is now in Phase 3 evaluation [10].
How will Terumo BCT’s Africa agreements impact sickle cell care?
They aim to embed SCD services into national health systems through policy, training and technology partnerships [9].
Entities / Keywords
C4 Therapeutics (CCCC); Pfizer; cemsidomide; elranatamab; multiple myeloma; Kura Oncology (KURA); Kyowa Kirin; ziftomenib; AML; FLT3; NPM1; Verismo Therapeutics; SynKIR-310; IFLI; follicular lymphoma; Soligenix (SNGX); HyBryte™; CTCL; synthetic hypericin; Umoja Biopharma; UB-VV111; CAR T; AbbVie option; AbbVie (ABBV); Pivekimab sunirine (PVEK); BPDCN; ADC; CD123; Chugai Pharma; PiaSky; PNH; anti-C5 Recycling Antibody™; Novo Nordisk; Mim8 (denecimig); hemophilia A; FVIIIa mimetic; Terumo BCT; Kenya; Uganda; Côte d’Ivoire; sickle cell disease; Star Therapeutics; VGA039; von Willebrand disease; Sanofi Ventures; Viking Global.
References
- https://ir.c4therapeutics.com/news-releases/news-release-details/c4-therapeutics-announces-clinical-trial-collaboration-and
- https://ir.kuraoncology.com/news-releases/news-release-details/kura-oncology-and-kyowa-kirin-launch-clinical-trial-evaluating
- https://www.prnewswire.com/news-releases/verismo-therapeutics-treats-first-follicular-lymphoma-patient-in-celestial-301-trial-strengthening-collaboration-with-ifli-302573330.html
- https://ir.soligenix.com/2025-09-30-Soligenix-Announces-Expansion-of-European-Medical-Advisory-Board-for-Cutaneous-T-Cell-Lymphoma
- https://www.umoja-biopharma.com/news/#Press-Releases
- https://news.abbvie.com/2025-09-30-AbbVie-Submits-Biologics-License-Application-BLA-to-U-S-FDA-for-Pivekimab-sunirine-PVEK-an-Investigational-Antibody-Drug-Conjugate-ADC-to-Treat-Rare-Cancer-with-Limited-Treatment-Options
- https://www.chugai-pharm.co.jp/english/news/detail/20251001153000_1188.html
- https://www.prnewswire.com/news-releases/novo-nordisk-submits-biologics-license-application-bla-to-fda-for-mim8-an-investigational-prophylaxis-treatment-for-people-living-with-hemophilia-a-with-or-without-inhibitors-302568838.html
- https://www.terumobct.com/en/gl/our-company/news-and-events/news-releases/kenya–uganda-and-cote-d-ivoire-partner-with-terumo-blood-and-ce.html
- https://star-therapeutics.com/star-therapeutics-announces-oversubscribed-125-million-series-d-financing/