Watch Our Video Summary Capturing Top Hematology News from the Last Two Weeks
From Heidelberg Pharma’s HDP-101 advancement and Novo Nordisk’s Mim8 filing to AbbVie’s Pivekimab submission—this episode’s signals are robust. We span multiple myeloma, hemophilia, and BPDCN, with regulatory milestones and clinical advances across hematology, underscoring therapeutic diversification and accelerating innovation.
Top Stories Covered in This Video
🧬 Heidelberg Pharma advances HDP-101 to Cohort 9 [1] [EU • 25 Sep 2025]
Context: Phase I/IIa dose-escalation in relapsed/refractory multiple myeloma (anti-BCMA ATAC).
Key point: No dose-limiting toxicities at 140 µg/kg; one very good partial response in Cohort 8; one complete remission ongoing from Cohort 5.
Implication: May influence prescriber choice and payer reviews pending full data.
💵 Aptose & Hanmi extend loan for tuspetinib program [2] [US • 22 Sep 2025]
Context: Amended US $11.9 million facility to fund tuspetinib (multi-kinase inhibitor) in triplet AML therapy.
Key point: Supports ongoing TUSCANY Phase 1/2 combining tuspetinib + azacitidine + venetoclax; early CR/CRh responses reported.
Implication: Signals pipeline investment and modality expansion.
🧪 Kura Oncology & Kyowa Kirin launch FLT3/NPM1 trial [3] [US • 01 Oct 2025]
Context: KOMET-007 trial arm testing ziftomenib + quizartinib + 7+3 chemotherapy in newly diagnosed FLT3/NPM1 AML.
Key point: Builds on Breakthrough-designated ziftomenib’s activity and safety profile; aims to improve outcomes in high-relapse population.
Implication: May influence prescriber choice and payer reviews pending full data.
🧫 AbbVie submits FDA BLA for Pivekimab sunirine (PVEK) [4] [US • 30 Sep 2025]
Context: Based on global Phase 1/2 CADENZA in BPDCN (CD123-targeting ADC).
Key point: First AbbVie ADC in blood cancer; addresses unmet need in aggressive BPDCN.
Implication: May influence prescriber choice and payer reviews pending full data.
💉 Novo Nordisk files FDA BLA for Mim8 (denecimig) [5] [US • 29 Sep 2025]
Context: FRONTIER studies underpin submission of FVIIIa-mimetic bispecific antibody for hemophilia A prophylaxis.
Key point: First potential once-monthly to weekly subcutaneous prophylaxis via prefilled pen, for patients with or without inhibitors.
Implication: May influence prescriber choice and payer reviews pending full data.
🧫 CRISPR Tx & Sirius Tx begin Phase 2 of SRSD107 (siRNA FXI inhibitor) [6] [EU • 22 Sep 2025]
Context: Global trial in patients undergoing total knee arthroplasty for prevention of venous thromboembolism.
Key point: First patient dosed; prior Phase 1 showed > 93 % FXI reduction with sustained effect up to 6 months.
Implication: Could inform practice and payer discussions; interpretation depends on study design and confounding control.
💊 Chugai launches PiaSky in Taiwan [7] [Taiwan • 01 Oct 2025]
https://www.chugai-pharm.co.jp/english/news/detail/20251001153000_1188.html
Context: PiaSky (anti-C5 recycling antibody) approved for PNH ≥13 years, ≥40 kg.
Key point: First subcutaneous PNH therapy in Taiwan; allows dosing every 4 weeks with low volume.
Implication: Introduces competition that may affect pricing and formulary access.
🧬 Long-term durability of Valoctocogene Roxaparvovec gene therapy [8]
https://bioengineer.org/long-term-durability-of-valoctocogene-roxaparvovec-in-hemophilia-a/
Context: Analysis in Advances in Therapy extrapolated trial data for sustained Factor VIII production post-infusion.
Key point: Stable FVIII levels observed for multiple years; minimal adverse effects reported.
Implication: Could inform practice and payer discussions; interpretation depends on study design and confounding control.
Why It Matters
- Multiple antibody-drug conjugates (HDP-101, PVEK) and gene/RNA-based approaches (Valoctocogene Roxaparvovec, SRSD107) underscore continued innovation in hematologic disorders.
- Dual and triplet AML regimens (tuspetinib triplet, ziftomenib combination) reflect a shift toward precision combinations.
- BLA submissions from AbbVie and Novo Nordisk highlight regulatory momentum for ADCs and bispecific antibodies.
- New regional approvals (PiaSky Taiwan) demonstrate geographic diffusion of next-generation biologics.
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FAQ
What is HDP-101 and who develops it?
HDP-101 is an anti-BCMA antibody–amanitin conjugate (ATAC) by Heidelberg Pharma [1], in Phase I/IIa for relapsed/refractory multiple myeloma.
How is tuspetinib positioned in AML treatment?
Tuspetinib (Aptose Biosciences [2]) is a multi-kinase inhibitor studied in triplet combination with azacitidine + venetoclax for newly diagnosed AML unfit for induction chemotherapy.
What differentiates ziftomenib in AML therapy?
Ziftomenib (Kura Oncology [3]) is an oral menin inhibitor, now tested with quizartinib and intensive chemo for FLT3/NPM1-mutated AML, targeting a high-relapse subset.
Which ADCs advanced this week?
AbbVie’s Pivekimab sunirine (PVEK) [4] entered FDA review for BPDCN, while Heidelberg’s HDP-101 [1] progressed to higher dosing after early efficacy signals.
What hemophilia therapies are in focus?
Novo Nordisk’s Mim8 [5] (denecimig) filed for FDA approval as a flexible-dosing prophylaxis, and Valoctocogene Roxaparvovec [8] showed sustained benefit in gene-therapy analysis.
What is SRSD107 and its significance?
SRSD107 (CRISPR Tx & Sirius Tx [6]) is a siRNA targeting FXI, designed for long-acting anticoagulation with potentially lower bleeding risk.
How does PiaSky expand PNH options?
Chugai’s PiaSky [7] is Taiwan’s first subcutaneous C5 inhibitor, reducing infusion burden for PNH patients.
Entities / Keywords
Heidelberg Pharma • Aptose Biosciences • Hanmi Pharmaceutical • Kura Oncology • Kyowa Kirin • AbbVie • Novo Nordisk • CRISPR Therapeutics • Sirius Therapeutics • Chugai Pharmaceutical • Valoctocogene Roxaparvovec • HDP-101 • Tuspetinib • Ziftomenib • Pivekimab Sunirine (PVEK) • Mim8 (Denecimig) • SRSD107 • PiaSky • Hemophilia A • Multiple Myeloma • Acute Myeloid Leukemia • Paroxysmal Nocturnal Hemoglobinuria
References
https://www.chugai-pharm.co.jp/english/news/detail/20251001153000_1188.html
https://bioengineer.org/long-term-durability-of-valoctocogene-roxaparvovec-in-hemophilia-a/