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2016 Biomedtracker / Datamonitor Healthcare Post-ASH Report
January 27, 2017
The 58th American Society of Hematology (ASH) Annual Meeting and Exposition was held in San Diego, CA from December 3-6, 2016.
The bullets below provide a summary of the conference.
For our disclosures, please read the Biomedtracker Research Standards.
- CAR-T cell therapies generated excitement at ASH 2016 due to major data releases from leading developers. In particular, Kite presented highly anticipated interim data from its pivotal ZUMA-1 trial which is expected to support US FDA approval of KTE-C19 in refractory DLBCL. Novartis also made a strong case for accelerated approval of CTL-019 with promising data from its ongoing Phase II trial in pediatric and young adult patients with R/R B-cell ALL.
- SGN-CD33A (vadastuximab talirine, SGEN) was used in combination with HMAs to speed up the responses and to increase the response rate in AML patients not suitable for intensive chemotherapy. Data presented at ASH showed high remission rates even in patients with underlying myelodysplasia or who were positive for FLT3/ITD.
- Topline data from a Phase Ib trial evaluating 7+3 in combination with SGN-CD33A in AML were presented. The combination showed a CR/CRi of 76% which is higher than historical rates for the 7+3 regimen (approximately 40-50%).
- In CLL patients intolerant of ibrutinib therapy, acalabrutinib (AZN) was well tolerated with no major hemorrhages. Efficacy was good for these hard to treat patients with a 79% ORR.
- BPDCN is an indication with no standard of care. Updated results from a Phase I/II trial of SL-401 (STML) showed a 100% overall response rate, including an 81% complete response rate in first-line BPDCN patients.
- Detailed Phase III data from a myelofibrosis trial showed that pacritinib (CTIC) was clearly better than BAT in terms of spleen response and improvement of symptoms in patients with platelet counts less than 100,000.
- Positive Phase II data for crizanlizumab (NVS) in sickle cell anemia patients showed a substantial reduction in the rate of pain crises compared to placebo.
- Updated data from the Phase I/II study of GBT440 (GBT), a modifier of hemoglobin structure that prevents deoxygenated sickle hemoglobin from aggregating, showed an increase in hemoglobin responders compared to placebo, along with a reduction in irreversibly sickled cells.
- Updated data from the Phase I/II study of the hemophilia B gene therapy, SPK-9001 (PFE), showed that a single infusion of SPK-9001 achieves sustained factor IX expression.
- LentiGlobin (BLUE) gene therapy showed roughly 2-4 fold increases in vector copy number (VCN) with the new manufacturing process compared to the old, in cells from patients with beta-thalassemia and sickle cell disease. In sickle cell, however, there is still significant uncertainty how much an impact this and other measures will have on later declines in VCN.
- Updated results from ongoing Phase I and Phase II trials of fitusiran (ALNY) continue to support its safety and efficacy in hemophilia A and B patients with and without inhibitors. Monthly infusions of the siRNA inhibitor were associated with significant reductions in bleeding events.
- Chronic GVHD (cGVHD) is the most common cause of morbidity in patients who have undergone allogeneic transplant and there are no approved therapies for patients with cGVHD who fail corticosteroids. Updated results from a Phase I/II trial of ibrutinib (Imbruvica, ABBV) in patients who have failed at least one prior treatment for cGVHD, showed an ORR of 67%.
For our disclosures, please read the Biomedtracker Research Standards.
| Indications Covered: |
Acute Lymphoblastic Leukemia (ALL)
Acute Myelogenous Leukemia (AML) Amyloidosis Anaplastic Large Cell Lymphoma (ALCL) - NHL Anemia Anemia Due to Chronic Kidney Disease, Dialysis-Dependent Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) Bone Marrow Transplant and Stem Cell Transplant Cancer Cardiovascular Disease Chronic Lymphocytic Leukemia (CLL)/Small Cell Lymphocytic Lymphoma (SLL) - NHL Chronic Myelogenous Leukemia (CML) Chronic Myelomonocytic Leukemia (CMML) Complement Deficiencies / Abnormalities Cutaneous T-Cell Lymphoma (CTCL) - NHL Cytomegalovirus (CMV) Infection (Antiviral) Diffuse Large B-Cell Lymphoma (DLBCL) - NHL Drug Toxicity Fungal Infections - Systemic Graft vs. Host Disease (GVHD) - Treatment Hematologic Cancer Hemophilia A Hemophilia A and B - General Clotting Products Hemophilia B Hodgkin's Lymphoma Hypereosinophilic Syndrome (HES) Immune Thrombocytopenic Purpura (ITP) Indolent Non-Hodgkin's Lymphoma - iNHL Inflammatory Disorders Marginal Zone Lymphoma - NHL Mastocytosis Melanoma Meningococcal Vaccines and Other Meningococcus-Specific Agents (Antibacterial) Metabolic - General Multiple Myeloma (MM) Myelodysplastic Syndrome (MDS) Myelofibrosis (MF) Non-Hodgkin's Lymphoma (NHL) Non-Small Cell Lung Cancer (NSCLC) Paroxysmal Nocturnal Hemoglobinuria (PNH) Peripheral T-Cell Lymphoma (PTCL) - NHL Polycythemia Vera (PV) Porphyria Sickle Cell Disease Stroke Prevention in Atrial Fibrillation (SPAF) Thrombotic Thrombocytopenic Purpura (TTP) Venous Thromboembolism (VTE) Waldenstrom Macroglobulinemia (WM) / Lymphoplasmacytic Lymphoma (LPL) - NHL |
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