Nicola Gökbuget’s Post

The Hematology Round Up from #ASCO24 (Multiple Myeloma) Phase 3 study results of isatuximab, bortezomib, lenalidomide, and dexamethasone (Isa-VRd) versus VRd for transplant-ineligible patients with newly diagnosed multiple myeloma (IMROZ). https://lnkd.in/d2dRh6Hp Phase 3 randomized study of isatuximab (Isa) plus lenalidomide and dexamethasone (Rd) with bortezomib versus isard in patients with newly diagnosed transplant ineligible multiple myeloma (NDMM TI). https://lnkd.in/dSjVvk7X Daratumumab (DARA) + bortezomib/lenalidomide/dexamethasone (VRd) in transplant-eligible (TE) patients (pts) with newly diagnosed multiple myeloma (NDMM): Analysis of minimal residual disease (MRD) in the PERSEUS trial. Thank you for your attention and enjoy ASCO Disclosure: This Hematology Round Up was supported by Sanofi https://lnkd.in/d9ic5mpy

Regeneron touts success with bispecific antibody in multiple myeloma trial  Regeneron has released encouraging trial results for its multiple myeloma antibody, #linvoseltamab. The data from the Phase I/II LINKER-MM1 (NCT03761108) study was presented at the European Hematology Association (EHA) Congress 2024 in Madrid, Spain, on 13–16 June.  The 14-month follow up data demonstrated a 71% objective response rate, with 50% of patients achieving a complete response or better and 63% achieving a very good partial response or better, as determined by an independent review committee.  The median overall survival (OS) was 31 months, while the median progression free survival (PFS) and median duration of response (DoR) were not reached, as patients had not progressed yet. The median DoR for all responders was 29 months. https://lnkd.in/gsz5frwg

Pegcetacoplan is a novel anti-complement drug used to treat the rare and complement-mediated hematological disease paroxysmal nocturnal hemoglobinuria (PNH). In Annals of Hematology Mendy Boersma-ter Avest presents her findings that personalized dosing has the potential to (cost-neutrally) improve patient friendliness and effectiveness using pharmacokinetically-guided dosing. Read it here: https://lnkd.in/ed9GrY_d

Daratumumab is increasingly incorporated into the standard treatment regimens for patients with plasma cell dyscrasias in Asia, especially with cost-containment measures implemented by various regional health authorities. This analysis aimed to study daratumumab’s tolerability amongst Asian patients. This is a retrospective medical records review of patients who received daratumumab between November 2016 and August 2021 as part of routine clinical care. Sixty-two patients were included in the study: 62.9% had renal impairment, and 27.4% had creatinine clearance (CrCl) <30ml/min. Forty-five patients (72.6%) received daratumumab combination therapy, with a median 1 line of prior therapy. The median duration of follow-up was 12.4 months, and the median duration patients were on treatment with daratumumab was 12.3 months. Twenty-one of 62 (33.9%) patients experienced infusion-related reactions (IRRs) after the first dose of intravenous daratumumab. Seven developed mostly grades 1 and 2 respiratory events, and 14 showed grades 1 and 2 non-respiratory IRRs. Only one patient experienced a grade 1 IRR with the second infusion, with none developing any IRRs in the third or subsequent infusions. Eight (12.9%) patients were affected by hematological adverse events (AEs), mostly grades 2 and 3, with one experiencing grade-4 neutropenia without sepsis. Six (9.7%) patients experienced non-hematological AEs, the commonest being pneumonia and other infections, with one developing Nocardia pneumonia (grade 4) 14 months after the initiation of daratumumab. In conclusion, daratumumab is tolerable amongst Asian patients, including the elderly, and patients with severe renal impairment and chronic lung diseases.

Interesting and crucial to have more follow up... novel therapies are promising but we should be careful 

HaemaLogiX Ltd Chief Scientific Officer Dr Rosanne Dunn explains the key highlights of the abstract that was recently published in the European Hematology Association (EHA)’s official open access journal, HemaSphere Journal. Dr Dunn highlights that the data have shown that 100% of amyloidosis patient bone marrow plasma cells express either LMA or KMA. It is also highlighted that LMA and KMA define treatment-resistant, disease-causing plasma cells – in keeping with our published KappaMab Phase 2b clinical results which have also shown that our immunotherapies can deplete these persistent aberrant cells without affecting normal immune cells. Watch the full video below from Dr Dunn on the key highlights of this published data. For more details on this pre-clinical data and its publication, visit our website via this link: https://bit.ly/4b1QgNg

We are pleased to announce that our new pre-clinical data has been published in the Abstract Book, under the European Hematology Association (EHA)’s official open access journal, HemaSphere Journal. Published under the title “Novel Antigens LMA and KMA Are Expressed on Bone Marrow Plasma Cells from Patients with AL Amyloidosis but They Are Not Detected on Normal Plasma Cells”, the pre-clinical data shows that 100% of amyloidosis patient bone marrow plasma cells express either LMA or KMA. The range of antigen densities was higher for LMA and KMA compared to BCMA. LMA and KMA were detected on CD38-positive cells in all patient samples treated with daratumumab, indicating that HaemaLogiX immunotherapies could be used to treat patients who are not responding to daratumumab. Currently attending the EHA Congress (#EHA2024) in Madrid, Spain, CEO/Managing Director Damian Clarke-Bruce commented: "This research reinforces earlier findings that our novel antigens, KMA and LMA, are predominantly found on malignant plasma cells. This offers a new clinical pathway for addressing AL amyloidosis, multiple myeloma, and some autoimmune diseases.” A sincere thank you to Professor Jesus San Miguel at Clínica Universidad de Navarra and his team in Spain, as well as our research collaborators at The Westmead Institute for Medical Research led by Professor David Gottlieb, for your tremendous research work on this study. For more details on this pre-clinical data and its publication, visit our website via this link: https://bit.ly/4b1QgNg #amyloidosis #novelantigen 

Last week I presented the first results of the phase 2 ICON study at the European Hematology Association (EHA) conference in Madrid. In the ICON study, relapsed/refractory multiple myeloma patients were treated with iberdomide, low-dose cyclophosphamide and dexamethasone. Iberdomide is a novel cereblon E3 ligase modulator (CELMoD™) with improved direct anti-myeloma activity and immune-stimulatory effects. This treatment regimen resulted in a ORR of 82% and a median PFS of 17.8 months in patients with 2-4 prior lines of therapy. This fully oral, well tolerated, and active combination warrants further evaluation in RRMM. The full abstract is available online: https://lnkd.in/eUqb4Vaq What an interesting conference the #EHA2024 has been! Thanks to all co-authors and my supervisors Niels Van De Donk, Sonja Zweegman and Tuna Mutis.

#JIPO Research article by Nguyen and Van discusses the "Treatment of Stage IV #NSCLC with #Pembrolizumab in Combination with Platinum-Based Doublet #Chemotherapy in Vietnam" https://lnkd.in/eFPZzYuU

We are excited to provide updated clinical safety and efficacy data from 7 transfusion-dependent beta thalassemia patients treated with renizgamglogene autogedtemcel (reni-cel) in the Phase 1/2 EdiThal clinical trial at European Hematology Association (EHA) annual congress. Treated patients had early and robust increases of total hemoglobin (Hb) and fetal hemoglobin, with total Hb rising to or above the transfusion independence threshold of 9.0 g/dL. All patients have been transfusion free for a range of 4.1 to 12.8 months after receiving the last red blood cell transfusion at 0.5–2.2 months post-reni-cel infusion. Reni-cel was well-tolerated and continues to demonstrate a safety profile consistent with myeloablative conditioning with busulfan and autologous hematopoietic stem cell transplant. #BetaThalassemia #Biotechnology #Cas12a #CellTherapy #ClinicalTrials #CRISPR #EHA #GeneEditing #Hematology