AstraZeneca will present 47 abstracts showcasing new data from across its hematology portfolio and clinical pipeline, demonstrating its commitment to redefining care for hard-to-treat blood diseases at the 64th American Society of Hematology (ASH) Annual Meeting and Exposition, December 10 to 13, 2022.

A total of eight approved and potential new medicines will be featured across more than 10 types of blood cancers and rare diseases, including data in chronic lymphocytic leukemia (CLL), follicular lymphoma (FL), diffuse large B-cell lymphoma (DLBCL), mantle cell lymphoma (MCL), paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS) and amyloid light chain (AL) amyloidosis.

Anas Younes, Senior Vice President, Oncology R&D, AstraZeneca, said: 'At this year's ASH Annual Meeting, our data demonstrate the broad potential of our hematology pipeline and the continued strength of our approved medicines. Data are being highlighted from many of our early-stage molecules, including clinical trials of TNB-486 (AZD0486), a B-cell targeting T-cell engager, and presentations of long-term follow-up data will show the consistent safety and efficacy profile of CALQUENCE.'

Gianluca Pirozzi, Senior Vice President, Head of Development and Safety, Alexion, AstraZeneca Rare Disease said: 'The depth and breadth of Alexion data at this year's ASH Annual Meeting reinforce the importance of earlier diagnosis and disease management for rare diseases that are often not well-understood. We will share research across several therapy areas - including an oral presentation demonstrating the potential of vemircopan, an investigational, second-generation factor D inhibitor as monotherapy treatment of paroxysmal nocturnal hemoglobinuria - underscoring our leadership and unwavering commitment to driving critical innovations in rare disease.'

CALQUENCE (acalabrutinib) real-world evidence and long-term follow-up data support consistent efficacy and safety profile

A post-hoc safety analysis from the head-to-head ELEVATE-RR Phase III trial of CALQUENCE versus ibrutinib will further support tolerability differences of CALQUENCE in relapsed or refractory CLL.1

Final long-term follow-up results of the Phase I/II trials evaluating CALQUENCE monotherapy in front-line and relapsed or refractory CLL will further support the continued efficacy and safety CALQUENCE demonstrated in both settings.2,3

An oral presentation of Phase II research sponsored by the Dana-Farber Cancer Institute will show the efficacy and tolerability of CALQUENCE combined with venetoclax and obinutuzumab in a front-line, high-risk CLL population.4

A retrospective pooled analysis will show the benefit of adding obinutuzumab to CALQUENCE in the front-line CLL setting in patients with select genomic characteristics.5

An oral presentation of preliminary Phase II results sponsored by Weill Cornell Medicine will show that CALQUENCE combined with lenalidomide and rituximab is generally well-tolerated, highly effective and produces high rates of minimal residual disease-negative complete remission in front-line MCL.6

Novel treatment strategies with emerging pipeline molecules exhibit therapeutic potential

An oral presentation of interim Phase I results evaluating TNB-486 (AZD0486), a CD19/CD3 next generation bispecific T-cell engager, will show the potential of targeting CD19/CD3, leading to an increase in anti-cancer activity in heavily pretreated patients with B-cell non-Hodgkin lymphoma (NHL).7

Results from Phase I and II trials of CDK9 inhibitor AZD4573 alone and with CALQUENCE will exhibit data on tolerability across a broad range of hematologic malignancies, including relapsed or refractory DLBCL.8,9

Preliminary results from an ongoing Phase I trial will demonstrate that Bcl-2/Bcl-xl inhibitor AZD0466 has been well-tolerated in patients with advanced hematologic malignancies.10

Innovating to help address the treatment needs of all patients with PNH

An oral presentation detailing interim results from a Phase II open-label trial of vemircopan (ALXN2050) will highlight efficacy and safety data from the treatment-naive patient group, establishing proof-of-concept as a monotherapy for PNH.11

An interim analysis from an ongoing Phase IV trial assessing the impact of switching to standard, weight-based intravenous (i.v.) ULTOMIRIS (ravulizumab-cwvz) from high-dose i.v. SOLIRIS (eculizumab) in adults with PNH will be presented.12

Improving diagnosis and management of life-threatening rare diseases

An analysis of data from the Global aHUS Registry, which contains information on patients across more than 100 sites in more than 20 countries, will highlight the importance of considering aHUS as a diagnosis even in the presence of a triggering condition or associated event.13

An analysis of real-world patient data from the US Premier Healthcare Database will expand on the potential of the PLASMIC scoring system to aid in identifying people with aHUS and making earlier treatment decisions.14

An analysis of pediatric patients with hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA) will provide insights on the correlation between complement activation and endothelial damage in HSCT-TMA and the potential for useful biomarkers indicative of this damage to inform diagnosis.15

Results through one year on safety, tolerability and biomarker data will be presented from a Phase II trial evaluating CAEL-101, a potentially first-in-class monoclonal antibody, in adults with AL amyloidosis.16

A real-world analysis in a current population with AL amyloidosis using Komodo Health US claims data will highlight the need for greater awareness and understanding to accelerate time to diagnosis.17

AstraZeneca in hematology

AstraZeneca is pushing the boundaries of science to redefine care in hematology. We have expanded our commitment to patients with hematologic conditions, not only in oncology but also in rare diseases with the acquisition of Alexion, allowing us to reach more patients with high unmet needs. By applying our deep understanding of blood cancers, leveraging our strength in solid tumor oncology and delivering on Alexion's pioneering legacy in complement science to provide innovative medicines for rare diseases, we are pursuing the end-to-end development of novel therapies designed to target underlying drivers of disease.

By targeting hematologic conditions with high unmet medical needs, we aim to deliver innovative medicines and approaches to improve patient outcomes. Our goal is to help transform the lives of patients living with malignant, rare and other related hematologic diseases, shaped by insights from patients, caregivers and physicians to have the most meaningful impact.

AstraZeneca in oncology

AstraZeneca is leading a revolution in oncology with the ambition to provide cures for cancer in every form, following the science to understand cancer and all its complexities to discover, develop and deliver life-changing medicines to patients.

The Company's focus is on some of the most challenging cancers. It is through persistent innovation that AstraZeneca has built one of the most diverse portfolios and pipelines in the industry, with the potential to catalyze changes in the practice of medicine and transform the patient experience.

AstraZeneca has the vision to redefine cancer care and, one day, eliminate cancer as a cause of death.

About Alexion, AstraZeneca Rare Disease

Alexion, AstraZeneca Rare Disease, is the group within AstraZeneca focused on rare diseases, created following the 2021 acquisition of Alexion Pharmaceuticals, Inc. As a leader in rare diseases for 30 years, Alexion is focused on serving patients and families affected by rare diseases and devastating conditions through the discovery, development and commercialization of life-changing medicines. Alexion focuses its research efforts on novel molecules and targets in the complement cascade and its development efforts on hematology, nephrology, neurology, metabolic disorders, cardiology and ophthalmology. Headquartered in Boston, Massachusetts, Alexion has offices around the globe and serves patients in more than 50 countries.

About AstraZeneca

AstraZeneca is a global, science-led biopharmaceutical company that focuses on the discovery, development and commercialization of prescription medicines in Oncology, Rare Diseases and BioPharmaceuticals, including Cardiovascular, Renal & Metabolism, and Respiratory & Immunology. Based in Cambridge, UK, AstraZeneca operates in over 100 countries, and its innovative medicines are used by millions of patients worldwide.

Contact:

Brendan McEvoy

Tel: +1 302 885 2677

(C) 2022 Electronic News Publishing, source ENP Newswire