AstraZeneca will present 47 abstracts showcasing new data from across its haematology 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, 10 to 13 December 2022.

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

Anas Younes, Senior Vice President, Haematology R&D, AstraZeneca, said: "At this year's ASH Annual Meeting, our data demonstrate the broad potential of our haematology 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 haemoglobinuria - underscoring our leadership and unwavering commitment to driving critical innovations in rare disease."

• 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

• 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 haematologic 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 haematologic malignancies.10

• 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-naïve 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) from high-dose i.v. Soliris (eculizumab) in adults with PNH will be presented.12

• 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 paediatric patients with haematopoietic 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

Key presentations during the 64th ASH Annual Meeting and Exposition

Lead author Abstract title Presentation details
Calquence
(acalabrutinib)
Byrd, J Final Results of Abstract # 4431
the Phase 1/2
Study of Poster Session: 642. Chronic Lymphocytic
Acalabrutinib Leukemia: Clinical and Epidemiological:
Monotherapy in Poster III
Treatment-Naive
Chronic 12 December 2022
Lymphocytic
Leukemia with >6 18:00-20:00 CST
Years of Follow
-Up Location: Hall D (Ernest N. Morial
Convention Center)
Davids, MS Contribution of Abstract # 1815
Obinutuzumab to
Acalabrutinib Poster Session: 642. Chronic Lymphocytic
Therapy in Leukemia: Clinical and Epidemiological:
Patients with Poster I
Treatment-Naive
Chronic 10 December 2022
Lymphocytic
Leukemia: 17:30-19:30 CST
Analysis of
Survival Outcomes Location: Hall D (Ernest N. Morial
by Genomic Convention Center)
Features
Davies, AJ Durable Responses Abstract # 4265
from
Acalabrutinib in Poster Session: 626. Aggressive Lymphomas:
Combination with Prospective Therapeutic Trials: Poster III
Rituximab,
Cyclophosphamide, 12 December 2022
Doxorubicin,
Vincristine and 18:00-20:00 CST
Prednisolone (R
-CHOP) as First Location: Hall D (Ernest N. Morial
Line Therapy for Convention Center)
Patients with
Diffuse Large B
-Cell Lymphoma
(DLBCL): The
ACCEPT Phase
Ib/II Single Arm
Study
Furman, R Phase 1/2 Study Abstract # 4434
of Acalabrutinib
Monotherapy in Poster Session: 642. Chronic Lymphocytic
Patients with Leukemia: Clinical and Epidemiological:
Relapsed/Refractor Poster III
y Chronic
Lymphocytic 12 December 2022
Leukemia: Final
Results with >4 18:00-20:00 CST
Years of Follow
-Up Location: Hall D (Ernest N. Morial
Convention Center)
Ruan, J Phase 2 Trial of Abstract # 73
Acalabrutinib
-Lenalidomide Oral Session: 623. Mantle Cell,
-Rituximab (ALR) Follicular, and Other Indolent B Cell
with Real-Time Lymphomas: Clinical and Epidemiological I
Monitoring of MRD
in Patients with 10 December 2022
Treatment-Naïve
Mantle Cell 9:30 CST
Lymphoma
Location: La Nouvelle Orleans Ballroom C
(Ernest N. Morial Convention Center)
Ryan, CE Updated Results Abstract # 344
from a
Multicenter, Oral Session: 642. Chronic Lymphocytic
Phase 2 Study of Leukemia: Clinical and Epidemiological:
Acalabrutinib, Targeted Triplet Combinations and
Venetoclax, Richter's Transformation
Obinutuzumab
(AVO) in a 10 December 2022
Population of
Previously 16:15 CST
Untreated
Patients with CLL Location: R06-R09 (Ernest N. Morial
Enriched for High Convention Center)
-Risk Disease
Seymour, JF Assessing the Abstract # 3133
Burden of Adverse
Events in a Head Poster Session: 642. Chronic Lymphocytic
-to-Head Trial of Leukemia: Clinical and Epidemiological:
Acalabrutinib Poster II
Versus Ibrutinib
in Previously 11 December 2022
Treated Chronic
Lymphocytic 18:00-20:00 CST
Leukemia (CLL)
Location: Hall D (Ernest N. Morial
Convention Center)
AZD0486
(CD19/CD3 T
-cell engager)
Hou, JZ Interim Results Abstract # 612
of the Phase 1
Study of Tnb-486, Oral Session: 623. Mantle Cell,
a Novel CD19xCD3 Follicular, and Other Indolent B Cell
T-Cell Engager, Lymphomas: Clinical and Epidemiological IV
in Patients with
Relapsed/Refractor 11 December 2022
y (R/R) B-NHL
17:45 CST

Location: 278-282 (Ernest N. Morial
Convention Center)
AZD0466 (Bcl
-2/Bcl-xL
inhibitor)
Arslan, S Safety and Abstract # 4094
Tolerability of
AZD0466 as Poster Session: 616. Acute Myeloid
Monotherapy for Leukemias: Investigational Therapies,
Patients with Excluding Transplantation and Cellular
Advanced Immunotherapies: Poster III
Hematological
Malignancies. 12 December 2022
Preliminary
Results from an 18:00-20:00 CST
Ongoing Phase
I/II Trial Location: Hall D (Ernest N. Morial
Convention Center)
AZD4573 (CDK9
inhibitor)
Brümmendorf, T Safety, Abstract # 1353
Tolerability,
Pharmacokinetics Poster Session: 605. Molecular
(PK) and Pharmacology and Drug Resistance: Lymphoid
Preliminary Neoplasms: Poster I
Antitumor
Activity of the 10 December 2022
Cyclin-Dependent
Kinase-9 (CDK9) 17:30-19:30 CST
Inhibitor AZD4573
in Location: Hall D (Ernest N. Morial
Relapsed/Refractor Convention Center)
y Hematological
Malignancies: A
Phase 1 First-in
-Human Study
Strati, P Phase 1b/2a Study Abstract # 2962
of AZD4573
(CDK9i) and Poster Session: 627. Aggressive Lymphomas:
Acalabrutinib in Clinical and Epidemiological: Poster II
Patients with
Relapsed/Refractor 11 December 2022
y Diffuse Large B
-Cell Lymphoma 18:00-20:00 CST
(r/r DLBCL):
Results from Dose Location: Hall D (Ernest N. Morial
-Escalation Convention Center)
Vemircopan
(ALXN2050)
Browett, P Vemircopan Abstract # 294
(ALXN2050)
Monotherapy in Oral Session: 508. Bone Marrow Failure:
Paroxysmal Acquired: Clinical Studies
Nocturnal
Hemoglobinuria: 10 December 2022
Interim Data from
a Phase 2 Open 17:15 CST
-Label Proof-of
-Concept Study Location: 260-262 (Ernest N. Morial
Convention Center)
Ultomiris
(ravulizumab)
Griffin, M Terminal Abstract # 1251
Complement
Inhibition and Poster Session: 508. Bone Marrow Failure:
Control of Acquired: Poster I
Hemolysis in
Paroxysmal 10 December 2022
Nocturnal
Hemoglobinuria 17:30-19:30 CST
Following
Switching from Location: Hall D (Ernest N. Morial
High-Dose Convention Center)
Eculizumab to
Ravulizumab: An
Interim Analysis
ALXN1820
Dai, Y A Phase 2a, Abstract # 3713
Randomized, Open
-Label Study to Poster Session: 114. Hemoglobinopathies,
Evaluate Multiple Excluding Thalassemia: Clinical and
Dosing Regimens Epidemiological: Poster III
of Subcutaneous
ALXN1820 in Adult 12 December 2022
Patients with
Sickle Cell 18:00-20:00 CST
Disease
Location: Hall D (Ernest N. Morial
Convention Center)
CAEL-101
Valent, J 1-Year Results Abstract # 4550
from a Phase 2
Study to Poster Session: 653. Myeloma and Plasma
Determine Safety Cell Dyscrasias: Prospective Therapeutic
and Tolerability Trials: Poster III
of Treating
Patients with 12 December 2022
Light-Chain (AL)
Amyloidosis with 18:00-20:00 CST
CAEL-101, an Anti
-Amyloid Location: Hall D (Ernest N. Morial
Monoclonal Convention Center)
Antibody,
Combined with
Anti-Plasma Cell
Dyscrasia
AL Amyloidosis
Catini, J Evaluation of the Abstract # 1887
Path to Diagnosis
and Time to Poster Session: 652. Multiple Myeloma and
Treatment in Plasma Cell Dyscrasias: Clinical and
Patients with Epidemiological: Poster I
Light-Chain
Amyloidosis Using 10 December 2022
the Komodo Claims
Database 17:30-19:30 CST

Location: Hall D (Ernest N. Morial
Convention Center)
HSCT-TMA
Jacobi, P Complement Abstract # 2431
Activation is
Associated Poster Session: 301. Vasculature,
Endothelium, Thrombosis and Platelets:
with Endothelial Basic and Translational: Poster II
Damage in
11 December 2022
Hematopoietic
Stem Cell 18:00-20:00 CST
Transplant
Location: Hall D (Ernest N. Morial
Associated Convention Center)
-Thrombotic
Microangiopathy
aHUS
Gasteyger, C Use of PLASMIC Abstract # 1178
Scores to Aid
Diagnosis of Poster Session: 331. Thrombotic
aHUS: A Real Microangiopathies/Thrombocytopenias and
-World Analysis COVID-19-related Thrombotic/Vascular
of Hospitalized Disorders: Clinical and Epidemiological:
Patients from the Poster I
Premier
Healthcare 10 December 2022
Database
17:30-19:30 CST

Location: Hall D (Ernest N. Morial
Convention Center)
Siedlecki, A Characterization Abstract # 1173
of Patients with
aHUS and Poster Session: 331. Thrombotic
Triggering/Associa Microangiopathies/Thrombocytopenias and
ted Events, with COVID-19-related Thrombotic/Vascular
and without Disorders: Clinical and Epidemiological:
Complement Poster I
Pathogenic
Variants or anti 10 December 2022
-CFH Antibodies:
A Global aHUS 17:30-19:30 CST
Registry Analysis
Location: Hall D (Ernest N. Morial
Convention Center)

Notes

AstraZeneca in haematology

AstraZeneca is pushing the boundaries of science to redefine care in haematology. We have expanded our commitment to patients with haematologic 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 tumour 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 haematologic 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 haematologic 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 catalyse 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.

Alexion
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 commercialisation of life-changing medicines. Alexion focuses its research efforts on novel molecules and targets in the complement cascade and its development efforts on haematology, 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.

AstraZeneca
AstraZeneca (LSE/STO/Nasdaq: AZN) is a global, science-led biopharmaceutical company that focuses on the discovery, development, and commercialisation 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. Please visit astrazeneca.com (http://www.astrazeneca.com/) and follow the Company on Twitter @AstraZeneca (https://twitter.com/AstraZeneca).

Contacts
For details on how to contact the Investor Relations Team, please click here (https://www.astrazeneca.com/investor-relations.html#Contacts). For media contacts, click here (https://www.astrazeneca.com/media-centre/contacts.html).

References

1. Seymour JF, Byrd JC, Munir T, et al. Assessing the Burden of Adverse Events in a Head-to-Head Trial of Acalabrutinib Versus Ibrutinib in Previously Treated Chronic Lymphocytic Leukemia (CLL) [abstract and poster]. Presented at: American Society of Hematology (ASH) Congress; December 10-13, 2022; New Orleans, Louisiana. Abs 3133.
2. Byrd JC, Woyach JA, Furman RR, et al. Final Results of the Phase 1/2 Study of Acalabrutinib Monotherapy in Treatment-Naive Chronic Lymphocytic Leukemia with >6 Years of Follow-up [abstract and poster]. Presented at: American Society of Hematology (ASH) Congress; December 10-13, 2022; New Orleans, Louisiana. Abs 4431.
3. Furman RR, Wierda WG, Schuh A, et al. Phase 1/2 Study of Acalabrutinib Monotherapy in Patients with Relapsed/Refractory Chronic Lymphocytic Leukemia: Final Results with >4 Years of Follow-up [abstract and poster]. Presented at: American Society of Hematology (ASH) Congress; December 10-13, 2022; New Orleans, Louisiana. Abs 4434.
4. Ryan CE, Lampson BL, Tyekucheva, S, et al. Updated Results from a Multicenter, Phase 2 Study of Acalabrutinib, Venetoclax, Obinutuzumab (AVO) in a Population of Previously Untreated Patients with CLL Enriched for High-Risk Disease [abstract and oral]. Presented at: American Society of Hematology (ASH) Congress; December 10-13, 2022; New Orleans, Louisiana. Abs 344.
5. Davids MS, Sharman JP, Eyre TA, et al. Contribution of Obinutuzumab to Acalabrutinib Therapy in Patients with Treatment-Naive Chronic Lymphocytic Leukemia: Analysis of Survival Outcomes by Genomic Features [abstract and poster]. Presented at: American Society of Hematology (ASH) Congress; December 10-13, 2022; New Orleans, Louisiana. Abs 1815.
6. Ruan J, Leonard JP, Chen GZ, et al. Phase 2 Trial of Acalabrutinib-Lenalidomide-Rituximab (ALR) with Real-Time Monitoring of MRD in Patients with Treatment-Naïve Mantle Cell Lymphoma [abstract and oral]. Presented at: American Society of Hematology (ASH) Congress; December 10-13, 2022; New Orleans, Louisiana. Abs 73.
7. Hou JZ, Jacobs R, Cho SG, et al. Interim Results of the Phase 1 Study of Tnb-486, a Novel CD19xCD3 T-Cell Engager, in Patients with Relapsed/Refractory (R/R) B-NHL [abstract and oral]. Presented at: American Society of Hematology (ASH) Congress; December 10-13, 2022; New Orleans, Louisiana. Abs 612.
8. Brümmendorf T, Medd P, Koch R, et al. Safety, Tolerability, Pharmacokinetics (PK) and Preliminary Antitumor Activity of the Cyclin-Dependent Kinase-9 (CDK9) Inhibitor AZD4573 in Relapsed/Refractory Hematological Malignancies: A Phase 1 First-in-Human Study [abstract and poster]. Presented at: American Society of Hematology (ASH) Congress; December 10-13, 2022; New Orleans, Louisiana. Abs 1353.
9. Strati P, Kim TM, Danilov A, et al. Phase 1b/2a Study of AZD4573 (CDK9i) and Acalabrutinib in Patients with Relapsed/Refractory Diffuse Large B-Cell Lymphoma (r/r DLBCL): Results from Dose-Escalation [abstract and poster]. Presented at: American Society of Hematology (ASH) Congress; December 10-13, 2022; New Orleans, Louisiana. Abs 2962.
10. Arslan S, Fleming S, Jain N, et al. Safety and Tolerability of AZD0466 as Monotherapy for Patients with Advanced Hematological Malignancies. Preliminary Results from an Ongoing Phase I/II Trial [abstract and poster]. Presented at: American Society of Hematology (ASH) Congress; December 10-13, 2022; New Orleans, Louisiana. Abs 4094.
11. Browett P, Kulasekararaj A, Notaro R, et al. Vemircopan (ALXN2050) Monotherapy in Paroxysmal Nocturnal Hemoglobinuria: Interim Data from a Phase 2 Open-Label Proof-of-Concept Study [abstract and oral]. Presented at: American Society of Hematology (ASH) Congress; December 10-13, 2022; New Orleans, Louisiana. Abs 294.
12. Griffin M, Gandhi S, Hicks E, et al. Terminal Complement Inhibition and Control of Hemolysis in Paroxysmal Nocturnal Hemoglobinuria Following Switching from High-Dose Eculizumab to Ravulizumab: An Interim Analysis [abstract and poster]. Presented at: American Society of Hematology (ASH) Congress; December 10-13, 2022; New Orleans, Louisiana. Abs 1251.
13. Siedlecki A, Al-Dakkak I, Anokhina K, et al. Characterization of Patients with aHUS and Triggering/Associated Events, with and without Complement Pathogenic Variants or Anti-CFH Antibodies: A Global aHUS Registry Analysis [abstract and poster]. Presented at: American Society of Hematology (ASH) Congress; December 10-13, 2022; New Orleans, Louisiana. Abs 1173.
14. Gasteyger C, Uriol-Rivera M, Ávila A, et al. Use of PLASMIC Scores to Aid Diagnosis of aHUS: A Real-World Analysis of Hospitalized Patients from the Premier Healthcare Database [abstract and poster]. Presented at: American Society of Hematology (ASH) Congress; December 10-13, 2022; New Orleans, Louisiana. Abs 1178.
15. Jacobi P, Cofiell R, Chang CH, et. al. Complement Activation is Associated with Endothelial Damage in Hematopoietic Stem Cell Transplant Associated-Thrombotic Microangiopathy [abstract and poster]. Presented at: American Society of Hematology (ASH) Congress; December 10-13, 2022; New Orleans, Louisiana. Abs 2431.
16. Valent J, Liedtke M, Zonder J, et al. 1-Year Results from a Phase 2 Study to Determine Safety and Tolerability of Treating Patients with Light-Chain (AL) Amyloidosis with CAEL-101, an Anti-Amyloid Monoclonal Antibody, Combined with Anti-Plasma Cell Dyscrasia [abstract and poster]. Presented at: American Society of Hematology (ASH) Congress; December 10-13, 2022; New Orleans, Louisiana. Abs 4550.
17. Catini J, Doan Q, Evans J, et al. Evaluation of the Path to Diagnosis and Time to Treatment in Patients with Light-Chain Amyloidosis Using the Komodo Claims Database [abstract and poster]. Presented at: American Society of Hematology (ASH) Congress; December 10-13, 2022; New Orleans, Louisiana. Abs 1887.