Hematology Update: Advanced HDP-101 Cohort, Pivotal Ziftomenib Data, Pediatric AML Funding & More
From HDP-101’s next-phase safety confirmation in myeloma to landmark ziftomenib data ahead of FDA review, this week’s update spans pivotal progress: new triplet funding for Aptose, revumenib’s CytoCell CDx authorization, major pediatric leukemia trial support, and global advances in hemophilia, AML, and VTE.
In Today’s Newsletter
🧬 Heidelberg Pharma pushes HDP-101 into Cohort 9 [1] [EU • 25 Sep 2025]
https://heidelberg-pharma.com/en/press-investors/announcements/press-releases-and-ad-hoc-announcements/pr-heidelberg-pharma-advances-to-cohort-9-in-phase-i-iia-trial-of-lead-atac-candidate-hdp-101-in-multiple-myeloma
Context: Phase I/IIa, open-label, non-randomized trial in relapsed/refractory multiple myeloma.
Key point: Cohort 8 (n=8) at 140 µg/kg showed favorable safety; one patient achieved VGPR; earlier CR also noted.
Implication: May influence prescriber choice and payer reviews pending full data.
📑 Ziftomenib pivotal data published in JCO [2] [US/EU • 25 Sep 2025]
https://ir.kuraoncology.com/news-releases/news-release-details/kura-oncology-and-kyowa-kirin-announce-publication-pivotal
Context: Phase 2 KOMET-001 in 92 adults with R/R NPM1-m AML.
Key point: CR/CRh rate 23%; 61% MRD-negative; manageable safety; FDA PDUFA set for 30 Nov 2025.
Implication: May influence prescriber choice and payer reviews pending full data.
💰 Aptose extends Hanmi loan for tuspetinib AML program [3] [22 Sep 2025]
https://www.aptose.com/news-media/press-releases/detail/333/aptose-and-hanmi-pharmaceutical-extend-loan-agreement-to
Context: $11.9M amended facility through Dec 2025; builds on prior $8.5M drawn.
Key point: Financing supports tuspetinib triplet (TUS+VEN+AZA) in newly diagnosed AML.
Implication: Signals pipeline investment and modality expansion.
🧪 FDA clears OGT’s CytoCell KMT2A CDx [4] [US • 22 Sep 2025]
https://www.ogt.com/us/about-us/news/u-s-food-and-drug-administration-authorizes-cytocell-kmt2a-breakapart-fish-probe-kit-pdx/
Context: Detects rearrangements in AML/ALL; Class II CDx with rapid turnaround.
Key point: CytoCell KMT2A Breakapart FISH Probe Kit PDx authorized as CDx for revumenib in KMT2Ar acute leukemia.
Implication: Introduces competition that may affect pricing and formulary access.
🎗️ Big Nova Foundation grants $8M to Blood Cancer United [5] [29 Sep 2025]
https://philanthropynewsdigest.org/news/blood-cancer-united-receives-8-million-from-big-nova-foundation
Context: Funding offsets federal research cuts; expands patient access.
Key point: Philanthropic award supports pediatric acute leukemia trials and early-career researchers.
Implication: May expand screening, initiation, and follow-up at scale.
💉 Rituximab dose strategies in acquired hemophilia A [6] [26 Sep 2025]
https://hemophilianewstoday.com/news/standard-low-dose-rituximab-effective-acquired-hemophilia-a/
Context: Retrospective study of 11 adults; rituximab with cyclophosphamide + steroids.
Key point: 9/11 achieved complete remission; standard dose acted faster than low dose.
Implication: Could inform practice and payer discussions; interpretation depends on study design and confounding control.
🧬 Valoctocogene Roxaparvovec shows durable effect [7]
https://bioengineer.org/long-term-durability-of-valoctocogene-roxaparvovec-in-hemophilia-a/
Context: Modeling from hemophilia A trials.
Key point: Data suggest sustained FVIII activity for years post-infusion; favorable safety noted.
Implication: May influence prescriber choice and payer reviews pending full data.
🩸 Azercell blood donation drive in Azerbaijan [8] [23 Sep 2025]
https://azertag.az/en/xeber/__azercell_conducts_blood_donation_campaign_under_for_a_thalassemia_free_life_program-3763627
Context: Organized with Azerbaijan Thalassemia Federation and Republic Blood Bank.
Key point: CSR event collected blood to support children with thalassemia, hemophilia, leukemia.
Implication: May expand screening, initiation, and follow-up at scale.
🇯🇵 VarmX gains rare PMDA Phase 1 waiver [9] [JP • 25 Sep 2025]
https://varmx.com/news/varmx-receives-phase-1-waiver-from-japanese-regulator-pmda-for-lead-asset-vmx-c001/
Context: Bypass agent for FXa DOAC patients needing urgent surgery; FDA IND and Fast Track already secured.
Key point: VMX-C001 cleared to enter Phase 3 in Japan without bridging trial; inclusion in global EquilibriX-S.
Implication: May influence prescriber choice and payer reviews pending full data.
🧬 CRISPR Tx & Sirius dose first EU patient in SRSD107 Phase 2 [10] [EU • 22 Sep 2025]
https://ir.crisprtx.com/news-releases/news-release-details/crispr-therapeutics-and-sirius-therapeutics-announce-first
Context: Randomized Phase 2 in TKA patients; SRSD107 is long-acting siRNA targeting FXI.
Key point: First patient dosed; Phase 1 showed >93% FXI knockdown with prolonged effect.
Implication: May influence prescriber choice and payer reviews pending full data.
Why it matters
- BCMA- and menin-targeting programs [1,2,4] highlight momentum in precision hematology.
- Philanthropic funding [5] helps offset policy-driven research gaps.
- Multiple hemophilia innovations [6,7,8,9,10] show diversification across antibodies, gene therapy, bypass agents, and siRNA.
- Loan-based financing [3] underscores reliance on strategic partnerships for AML pipeline progress.
📢 Stay Ahead in Hematology Research!
✅ Contact LucidQuest at info@lqventures.com for strategic guidance on Hematology innovations and clinical research.
FAQ
What is HDP-101 (Heidelberg Pharma)?
A BCMA-targeting ATAC ADC in Phase I/IIa for relapsed/refractory multiple myeloma. Early cohorts showed CR and VGPR with favorable safety [1].
How effective is ziftomenib (Kura/Kyowa Kirin)?
In KOMET-001, CR/CRh rate reached 23% with 61% MRD negativity; safety was manageable. FDA review deadline is 30 Nov 2025 [2].
What is tuspetinib (Aptose/Hanmi)?
An oral kinase inhibitor in a triplet regimen (with azacitidine + venetoclax) for AML. A $11.9M loan facility extension funds ongoing Phase 1/2 [3].
Why is OGT’s KMT2A FISH probe important?
It’s FDA-authorized as a CDx for revumenib in KMT2A-rearranged acute leukemia, enabling rapid patient selection [4].
What is VMX-C001 (VarmX)?
A recombinant FX bypass agent for DOAC patients facing urgent surgery or bleeding. Japan waived Phase 1, allowing direct Phase 3 entry [9].
What is SRSD107 (CRISPR Tx/Sirius)?
A siRNA against FXI for thrombosis prevention. Phase 1 showed >93% sustained FXI knockdown; Phase 2 dosing has begun in EU [10].
Entities / Keywords
Heidelberg Pharma; HDP-101; BCMA; multiple myeloma; Kura Oncology; Kyowa Kirin; ziftomenib; NPM1-m AML; KOMET-001; Aptose Biosciences; Hanmi Pharmaceutical; tuspetinib; AML triplet; OGT; CytoCell; KMT2A; revumenib (REVUFORJ®); CDx; Blood Cancer United; Big Nova Foundation; pediatric leukemia; Rituximab; acquired hemophilia A; corticosteroids; cyclophosphamide; Valoctocogene Roxaparvovec; hemophilia A; FVIII gene therapy; Azercell; thalassemia; hemophilia; leukemia CSR; VarmX; VMX-C001; FXa DOAC; urgent surgery; CRISPR Therapeutics; Sirius Therapeutics; SRSD107; FXI siRNA; VTE
References
- https://heidelberg-pharma.com/en/press-investors/announcements/press-releases-and-ad-hoc-announcements/pr-heidelberg-pharma-advances-to-cohort-9-in-phase-i-iia-trial-of-lead-atac-candidate-hdp-101-in-multiple-myeloma
- https://ir.kuraoncology.com/news-releases/news-release-details/kura-oncology-and-kyowa-kirin-announce-publication-pivotal
- https://www.aptose.com/news-media/press-releases/detail/333/aptose-and-hanmi-pharmaceutical-extend-loan-agreement-to
- https://www.ogt.com/us/about-us/news/u-s-food-and-drug-administration-authorizes-cytocell-kmt2a-breakapart-fish-probe-kit-pdx/
- https://philanthropynewsdigest.org/news/blood-cancer-united-receives-8-million-from-big-nova-foundation
- https://hemophilianewstoday.com/news/standard-low-dose-rituximab-effective-acquired-hemophilia-a/
- https://bioengineer.org/long-term-durability-of-valoctocogene-roxaparvovec-in-hemophilia-a/
- https://azertag.az/en/xeber/__azercell_conducts_blood_donation_campaign_under_for_a_thalassemia_free_life_program-3763627
- https://varmx.com/news/varmx-receives-phase-1-waiver-from-japanese-regulator-pmda-for-lead-asset-vmx-c001/
- https://ir.crisprtx.com/news-releases/news-release-details/crispr-therapeutics-and-sirius-therapeutics-announce-first