AstraZeneca advances haematology and cell therapy ambition with largest-ever presence at ASH

On December 4, 2025 AstraZeneca reported its ambition to redefine haematology care with new data from its diverse pipeline and portfolio at the 67th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition, 6-9 December 2025.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

This year’s ASH (Free ASH Whitepaper) congress will feature the Company’s largest presence to date, with 65 abstracts across eight approved and investigational medicines, including 15 oral presentations.

Key presentations include:

Phase I trial of surovatamig: Updated results at three-year follow up from the ongoing first-in-human trial of surovatamig, a CD19xCD3 T-cell engager, in relapsed/refractory (R/R) follicular lymphoma (FL) (Oral Abstract #1005).
DURGA-1 Phase Ib/II study: Initial data for AZD0120 in patients with R/R multiple myeloma (MM). AZD0120 is an investigational BCMAxCD19 chimeric antigen receptor T-cell (CAR T) therapy (Oral Abstract #269).
ECHO Phase III trial: Results after 50 months of follow up evaluating Calquence (acalabrutinib) plus bendamustine and rituximab in the first-line treatment of mantle cell lymphoma (MCL) (Oral Abstract #885).
ALXN1210-TMA-314 Phase III trial: Additional results from the open-label trial evaluating Ultomiris (ravulizumab) in paediatric patients with haematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA) (Oral Abstract #1052).
Anas Younes, Senior Vice President, Haematology R&D and Chief Medical Officer, AstraZeneca, said: "We are advancing a broad pipeline of investigational therapies with the potential to redefine patient care across multiple types of blood cancer. At ASH (Free ASH Whitepaper), we are sharing meaningful progress with early efficacy and safety data for AZD0120, our first cell therapy, in multiple myeloma, and for surovatamig, a novel T-cell engager, in B-cell malignancies."

Christophe Hotermans, Senior Vice President, Head of Global Medical Affairs, Alexion, said: "At ASH (Free ASH Whitepaper), we look forward to demonstrating how pioneering science can drive meaningful advances for people living with rare haematologic conditions. New data on Ultomiris, including Phase III results in paediatric patients with HSCT-TMA, will show clinically meaningful overall survival and improved outcomes, highlighting our ongoing pursuit to realise the full potential of our medicines and their impact on treating rare conditions."

Additional highlights include:

SYRUS Phase I/II trial: Updated safety and efficacy data for surovatamig in adolescent and adult patients with R/R B-cell acute lymphoblastic leukaemia (Abstract #3345)
Phase I trial of surovatamig: Initial efficacy and safety data for surovatamig in R/R diffuse large B-cell lymphoma (Abstract #5514)
AZD0120: Follow-up data from two investigator-initiated trials (IIT) in China evaluating AZD0120 as a first-line therapy in high-risk, newly diagnosed MM (Oral Abstract #258).
TrAVeRse Phase II trial: Preliminary results evaluating Calquence plus venetoclax and rituximab in treatment-naïve MCL patients (Oral Abstract #884).
AMPLIFY Phase III trial: Exploratory analyses supporting the safety and efficacy profile of Calquence in first-line chronic lymphocytic leukaemia, with new subgroup data evaluating the impact of prognostic mutations on clinical outcomes (Poster Abstract #3898).
ALPHA Phase III trial: Sub-analysis of results evaluating Voydeya (danicopan) as add-on to Ultomiris or Soliris (eculizumab) in adults with paroxysmal nocturnal haemoglobinuria (PNH) and clinically significant extravascular haemolysis, including in patients with advanced age (Oral Abstract #949).
Ultomiris: Real-world evidence highlighting the impact of Ultomiris in certain patient subgroups across approved indications, including in pregnant patients (Poster Abstracts #6238 and #4458).
Key presentations during the 67th ASH (Free ASH Whitepaper) Annual Meeting and Exposition

Lead Author

Abstract Title

Presentation Details (ET)

Awan, F et al.

Budget Impact of Fixed Duration Acalabrutinib in Combination with Venetoclax in Previously Untreated Chronic Lymphocytic Leukemia Patients in the United States

Abstract #2627

Poster Abstract Session

Session 902. Health Services and Quality Improvement: Lymphoid Malignancies: Poster I

6 December 2025
5:30 PM – 7:30 PM

Cheah, C et al.

Analysis of predictive factors for POD24 in patients with previously untreated mantle cell lymphoma receiving bendamustine-rituximab with or without acalabrutinib in the Phase 3 ECHO trial

Abstract #3578

Poster Abstract Session

Session 623. Mantle Cell, Follicular, Waldenstrom’s, and Other Indolent B Cell Lymphomas: Clinical and Epidemiological: Poster II

7 December 2025
6:00 PM – 8:00 PM

Ghia, P et al.

Impact of prognostic mutations on outcomes with fixed-duration acalabrutinib-venetoclax combinations versus chemoimmunotherapy: An exploratory analysis from AMPLIFY

Abstract #3898

Poster Abstract Session

Session 642. Chronic Lymphocytic Leukemia: Clinical and Epidemiological: Poster II

7 December 2025
6:00 PM – 8:00 PM

Hawkes, E et al.

Acalabrutinib plus venetoclax and rituximab in patients with treatment-naive (TN) mantle cell lymphoma (MCL): Results from the phase 2 TrAVeRse study

Abstract #884

Oral Abstract Session

Session 623. Mantle Cell, Follicular, Waldenstrom’s, and Other Indolent B Cell Lymphomas: Clinical and Epidemiological: Single Agent and Combination therapy for MCL

8 December 2025
3:00 PM – 03:15 PM

Hou, J-Z et al.

Real-world incidence of treatment-emergent (TE) cardiovascular (CV) events among chronic lymphocytic (CLL)/small lymphocytic lymphoma (SLL) patients receiving acalabrutinib (acala) or zanubrutinib (zanu) monotherapy

Abstract #4506

Poster Abstract Session

Session 906. Outcomes Research: Lymphoid Malignancies Excluding Plasma Cell Disorders: Poster II

7 December 2025
6:00 PM – 8:00 PM

Seymour, JF et al.

A post hoc safety analysis of fixed-duration acalabrutinib-venetoclax combinations vs chemoimmunotherapy: Results from the Phase 3 AMPLIFY trial

Abstract #2118

Poster Abstract Session

Session 642. Chronic Lymphocytic Leukemia: Clinical and Epidemiological: Poster I

6 December 2025
5:30 PM – 7:30 PM

Wang, ML et al.

Time to third-line treatment after bendamustine-rituximab with or without acalabrutinib in patients with previously untreated mantle cell lymphoma: Updated analysis of the phase 3 ECHO trial after 50 months of follow-up

Abstract #885

Oral Abstract Session

Session 623. Mantle Cell, Follicular, Waldenstrom’s, and Other Indolent B Cell Lymphomas: Clinical and Epidemiological: Single Agent and Combination therapy for MCL

8 December 2025
3:15 PM – 3:30 PM

Ultomiris (ravulizumab)

Schoettler, M et al.

Outcomes in pediatric patients with HSCT-TMA treated with ravulizumab

Abstract #1052

Oral Abstract Session

Session 723. Allogeneic Transplantation: Long-term Follow-up, Complications, and Disease Recurrence: Outcomes, toxicities and late effects

8 December 2025
4:45 PM – 05:00 PM

Chaudhury, S et al.

Organ dysfunction in pediatric patients with HSCT-TMA treated with ravulizumab

Abstract #4266

Poster Abstract Session

Session 723. Allogeneic Transplantation: Long-term Follow-up, Complications, and Disease Recurrence: Poster II

7 December 2025
6:00 PM – 8:00 PM

Sherrard, H et al.

Safety of ravulizumab use in pregnancy: Insights from a global pharmacovigilance analysis

Abstract #4458

Poster Abstract Session

Session 905. Outcomes Research: Non-Malignant Conditions Excluding Hemoglobinopathies: Poster II

7 December 2025
6:00 PM – 8:00 PM

Gandhi, S et al.

Real-world analysis of ravulizumab safety and effectiveness in advanced age patients with paroxysmal nocturnal hemoglobinuria: Insights from the international PNH registry

Abstract #6238

Poster Abstract Session

Session 905. Outcomes Research: Non-Malignant Conditions Excluding Hemoglobinopathies: Poster III

8 December 2025
6:00 PM – 8:00 PM

Fasenra (benralizumab)

Klion et al.

Efficacy and safety of benralizumab in patients with hypereosinophilic syndrome: Results from the Phase 3 natron study

Abstract #79

Oral Abstract Session

Session 634. Myeloproliferative Syndromes: Clinical and Epidemiological: Expanding the Therapeutic and Prognostic Landscape in Myeloproliferative Neoplasms, Mastocytosis and Hypereosinophilic Syndrome
06 December, 2025
9:30 AM – 9:45 AM

Klion et al.

Patient perspectives on the burden of hypereosinophilic syndrome: Results from the Phase 3 natron interview sub-study

Abstract #4465

Poster Presentation

Session 905. Outcomes Research: Non-Malignant Conditions Excluding Hemoglobinopathies: Poster II
07 December, 2025
6:00 PM – 8:00 PM

Voydeya (danicopan)

Kulasekararaj, A et al.

Danicopan add-on therapy demonstrates positive efficacy and safety outcomes in advanced age adults with paroxysmal nocturnal hemoglobinuria and clinically significant extravascular hemolysis: A sub-analysis of the phase 3 ALPHA trial

Abstract #949

Oral Abstract Session

Session 905. Outcomes Research: Non-Malignant Conditions Excluding Hemoglobinopathies: Antithrombotic Roulette: Balancing Risk, Cost, and Care

8 December 2025
2:45 PM – 03:00 PM

Surovatamig

Aldoss, I et al.

Safety and efficacy of surovatamig (AZD0486) in adolescent and adult patients with relapsed or refractory (R/R) B-cell acute lymphoblastic leukemia (B-ALL): Updated results from the Phase 1/2 SYRUS study

Abstract #3345

Poster Abstract Session

Session 613. Acute Lymphoblastic Leukemias: Therapies Excluding Allogeneic Transplantation: Poster II

7 December 2025
6:00 PM – 8:00 PM

Cheah, C et al.

SOUNDTRACK-B: A Phase 2 single-arm study to evaluate the efficacy and safety of surovatamig (AZD0486) in relapsed or refractory B-cell Non-Hodgkin lymphoma

Abstract #3747 (TiP)

Poster Abstract Session

Session 629. Aggressive Lymphomas, Immunotherapy including Bispecific Antibodies: Poster II

7 December 2025
6:00 PM – 8:00 PM

Hou, JZ et al.

Three-year follow-up of the Phase 1 first-in-human study investigating surovatamig, a novel CD19xCD3 T-cell engager, in patients with relapsed/refractory (R/R) follicular lymphoma (FL)

Abstract #1005

Oral Abstract Session

Session 623. Mantle Cell, Follicular, Waldenstrom’s, and Other Indolent B Cell Lymphomas: Clinical and Epidemiological – Follicular Lymphoma

8 December 2025
5:00 PM – 5:15 PM

Kim, TM et al.

Surovatamig (AZD0486), a CD19xCD3 T-cell engager (TCE), demonstrates high rate of minimal residual disease (MRD)-negative complete responses in relapsed/refractory (R/R) diffuse large B-cell lymphoma (DLBCL), including in patients who previously progressed on CD20 TCE and CD19 CAR T-cell therapies

Abstract #5514

Poster Abstract Session

Session 629. Aggressive Lymphomas, Immunotherapy including Bispecific Antibodies: Poster III

8 December 2025
6:00 PM – 8:00 PM

AZD0120

Du, J et al.

A dual targeting BCMA and CD19 FasTCAR-T (GC012F/AZD0120) as first-line therapy for newly diagnosed multiple myeloma

Abstract #258

Oral Abstract Session

Session 655. Multiple Myeloma: Cellular Therapies: Clinical Trial Advances in CAR T-Cell Therapy for Multiple Myeloma

6 December 2025
2:15 PM – 2:30 PM

Richard, S et al.

Safety and efficacy of AZD0120, a BCMA/CD19 dual-targeting CAR T-cell therapy, in relapsed/refractory multiple myeloma: Preliminary results from the DURGA-1 Phase 1b/2 study

Abstract #269

Oral Abstract Session

Session 704. Multiple Myeloma: Cellular Therapies: Clinical Trial Advances in CAR T-Cell Therapy for Multiple Myeloma

6 December 2025
3:00 PM – 3:15 PM

Feng, J et al.

One-year follow-up of CD19/BCMA dual-targeting FasTCAR-T GC012F (AZD0120) therapy in patients with refractory systemic lupus erythematosus

Abstract #2384

Poster Abstract Session

Session 704. Cellular Immunotherapies: Early Phase Clinical Trials and Toxicities: Poster I

6 December 2025
5:30 PM – 7:30 PM

Lentzsch, S et al.

ALACRITY: A Phase 1b/2 Study of AZD0120 (BCMA/CD19 CAR-T cell therapy) in Participants with Relapsed or Refractory Light Chain Amyloidosis (AL)

Abstract #8236

ePublication

3 November 2025

AZD4512

Han, H et al.

AZD4512: A novel CD22-directed antibody-drug conjugate for the treatment of b-cell malignancies

Abstract #3296

Poster Abstract Session

Session 605. Molecular Pharmacology and Drug Resistance: Lymphoid Neoplasms: Poster II

7 December 2025
6:00 PM – 8:00 PM

(Press release, AstraZeneca, DEC 4, 2025, View Source [SID1234661131])

AvenCell Therapeutics Announces IND Clearance and EMA Approval of Clinical Trial Application (CTA) for the QUADvance Study, a Phase I/II trial evaluating AVC-203, a Novel Allogeneic CD19/CD20 CAR-T Investigational Therapy for the Treatment of Relapsed/Refractory B-cell malignancies

On December 4, 2025 AvenCell Therapeutics, Inc., a leading clinical-stage cell therapy company focused on advancing both switchable and allogeneic CAR-T cell therapies, reported that the FDA and the European Medicines Agency (EMA) have cleared the company’s IND and approved the Clinical Trial Application (CTA), respectively, for QUADvance (AVC-203-01), a Phase II/II trial of AVC-203 for the treatment of relapsed/refractory B-cell malignancies.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

AVC-203 is a CRISPR-engineered allogeneic CAR-T candidate designed to target and eliminate cells expressing receptors CD19 and/or CD20, which are known to be expressed in nearly all B-cell malignancies.

"We are excited to build on the early success and promising activity and safety of our ongoing clinical program of allogeneic CAR-T in AML (NCT05949125) by now advancing into B Cell Lymphoma with what we believe to be the most scientifically compelling allogeneic technology in the industry," said Andrew Schiermeier, AvenCell’s President & CEO. "To date, attempts at solving the key scalability and cost issues of autologous CAR-T therapy by engineering cells from unrelated donors have fallen short. Nevertheless, the obvious benefits of such an approach, including consistency and potency of an actual drug product – something in vivo approaches cannot provide – remain as compelling as ever. AvenCell remains focused on ensuring that any patient who can benefit from CAR-T therapy can receive it, through the massive scaling of supply and dramatic reductions in cost of goods, with a product that is as good or better than best-in-class autologous CAR-Ts."

About AVC-203

AVC-203 is a CRISPR-engineered allogeneic CAR-T therapy incorporating four key innovations:

Dual antigen targeting: AVC-203 CAR-T cells contain a receptor that simultaneously targets CD19 and CD20.

Immune evasion: CRISPR/Cas9 engineering enables donor cells to avoid both Graft-versus-Host Disease (GvHD) and rejection by the patient’s immune system.

Improved T-cell fitness and off-the-shelf availability: Allogeneic manufacturing from healthy donors leverages better T-cell fitness and eliminates patient-specific production, enabling immediate treatment

Switchable targeting: A RevCAR receptor dimerized to the CD19/CD20 CAR enables flexible targeting of additional tumor antigens through bi- or tri-specific bridging proteins, allowing future target expansion beyond CD19/CD20.
Trial Designs and Objectives

The phase I/II study will be conducted at multiple sites in the US and Europe and will evaluate the safety, tolerability, efficacy, and pharmacokinetics of AVC-203 in adults with relapsed or refractory B-cell malignancies. The phase Ia dose escalation study is expected to be followed by a phase Ib dose expansion study and a Phase II pivotal trial.

B-cell malignancies

B-cell malignancies — including non-Hodgkin lymphomas, multiple myeloma, and B-cell acute lymphoblastic leukemia (B-ALL) — account for the majority of blood cancers. Approximately 120,000 and 150,000 new cases are diagnosed annually in the United States and Europe, respectively.

(Press release, AvenCell Therapeutics, DEC 4, 2025, View Source;302632517.html [SID1234661145])

AstraZeneca advances haematology and cell therapy ambition with largest-ever presence at ASH

On December 4, 2025 AstraZeneca reported its ambition to redefine haematology care with new data from its diverse pipeline and portfolio at the 67th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition, 6-9 December 2025.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

This year’s ASH (Free ASH Whitepaper) congress will feature the Company’s largest presence to date, with 65 abstracts across eight approved and investigational medicines, including 15 oral presentations.

Key presentations include:

Phase I trial of surovatamig: Updated results at three-year follow up from the ongoing first-in-human trial of surovatamig, a CD19xCD3 T-cell engager, in relapsed/refractory (R/R) follicular lymphoma (FL) (Oral Abstract #1005).
DURGA-1 Phase Ib/II study: Initial data for AZD0120 in patients with R/R multiple myeloma (MM). AZD0120 is an investigational BCMAxCD19 chimeric antigen receptor T-cell (CAR T) therapy (Oral Abstract #269).
ECHO Phase III trial: Results after 50 months of follow up evaluating Calquence (acalabrutinib) plus bendamustine and rituximab in the first-line treatment of mantle cell lymphoma (MCL) (Oral Abstract #885).
ALXN1210-TMA-314 Phase III trial: Additional results from the open-label trial evaluating Ultomiris (ravulizumab) in paediatric patients with haematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA) (Oral Abstract #1052).
Anas Younes, Senior Vice President, Haematology R&D and Chief Medical Officer, AstraZeneca, said: "We are advancing a broad pipeline of investigational therapies with the potential to redefine patient care across multiple types of blood cancer. At ASH (Free ASH Whitepaper), we are sharing meaningful progress with early efficacy and safety data for AZD0120, our first cell therapy, in multiple myeloma, and for surovatamig, a novel T-cell engager, in B-cell malignancies."

Christophe Hotermans, Senior Vice President, Head of Global Medical Affairs, Alexion, said: "At ASH (Free ASH Whitepaper), we look forward to demonstrating how pioneering science can drive meaningful advances for people living with rare haematologic conditions. New data on Ultomiris, including Phase III results in paediatric patients with HSCT-TMA, will show clinically meaningful overall survival and improved outcomes, highlighting our ongoing pursuit to realise the full potential of our medicines and their impact on treating rare conditions."

Additional highlights include:

SYRUS Phase I/II trial: Updated safety and efficacy data for surovatamig in adolescent and adult patients with R/R B-cell acute lymphoblastic leukaemia (Abstract #3345)
Phase I trial of surovatamig: Initial efficacy and safety data for surovatamig in R/R diffuse large B-cell lymphoma (Abstract #5514)
AZD0120: Follow-up data from two investigator-initiated trials (IIT) in China evaluating AZD0120 as a first-line therapy in high-risk, newly diagnosed MM (Oral Abstract #258).
TrAVeRse Phase II trial: Preliminary results evaluating Calquence plus venetoclax and rituximab in treatment-naïve MCL patients (Oral Abstract #884).
AMPLIFY Phase III trial: Exploratory analyses supporting the safety and efficacy profile of Calquence in first-line chronic lymphocytic leukaemia, with new subgroup data evaluating the impact of prognostic mutations on clinical outcomes (Poster Abstract #3898).
ALPHA Phase III trial: Sub-analysis of results evaluating Voydeya (danicopan) as add-on to Ultomiris or Soliris (eculizumab) in adults with paroxysmal nocturnal haemoglobinuria (PNH) and clinically significant extravascular haemolysis, including in patients with advanced age (Oral Abstract #949).
Ultomiris: Real-world evidence highlighting the impact of Ultomiris in certain patient subgroups across approved indications, including in pregnant patients (Poster Abstracts #6238 and #4458).
Key presentations during the 67th ASH (Free ASH Whitepaper) Annual Meeting and Exposition

Lead Author

Abstract Title

Presentation Details (ET)

Awan, F et al.

Budget Impact of Fixed Duration Acalabrutinib in Combination with Venetoclax in Previously Untreated Chronic Lymphocytic Leukemia Patients in the United States

Abstract #2627

Poster Abstract Session

Session 902. Health Services and Quality Improvement: Lymphoid Malignancies: Poster I

6 December 2025
5:30 PM – 7:30 PM

Cheah, C et al.

Analysis of predictive factors for POD24 in patients with previously untreated mantle cell lymphoma receiving bendamustine-rituximab with or without acalabrutinib in the Phase 3 ECHO trial

Abstract #3578

Poster Abstract Session

Session 623. Mantle Cell, Follicular, Waldenstrom’s, and Other Indolent B Cell Lymphomas: Clinical and Epidemiological: Poster II

7 December 2025
6:00 PM – 8:00 PM

Ghia, P et al.

Impact of prognostic mutations on outcomes with fixed-duration acalabrutinib-venetoclax combinations versus chemoimmunotherapy: An exploratory analysis from AMPLIFY

Abstract #3898

Poster Abstract Session

Session 642. Chronic Lymphocytic Leukemia: Clinical and Epidemiological: Poster II

7 December 2025
6:00 PM – 8:00 PM

Hawkes, E et al.

Acalabrutinib plus venetoclax and rituximab in patients with treatment-naive (TN) mantle cell lymphoma (MCL): Results from the phase 2 TrAVeRse study

Abstract #884

Oral Abstract Session

Session 623. Mantle Cell, Follicular, Waldenstrom’s, and Other Indolent B Cell Lymphomas: Clinical and Epidemiological: Single Agent and Combination therapy for MCL

8 December 2025
3:00 PM – 03:15 PM

Hou, J-Z et al.

Real-world incidence of treatment-emergent (TE) cardiovascular (CV) events among chronic lymphocytic (CLL)/small lymphocytic lymphoma (SLL) patients receiving acalabrutinib (acala) or zanubrutinib (zanu) monotherapy

Abstract #4506

Poster Abstract Session

Session 906. Outcomes Research: Lymphoid Malignancies Excluding Plasma Cell Disorders: Poster II

7 December 2025
6:00 PM – 8:00 PM

Seymour, JF et al.

A post hoc safety analysis of fixed-duration acalabrutinib-venetoclax combinations vs chemoimmunotherapy: Results from the Phase 3 AMPLIFY trial

Abstract #2118

Poster Abstract Session

Session 642. Chronic Lymphocytic Leukemia: Clinical and Epidemiological: Poster I

6 December 2025
5:30 PM – 7:30 PM

Wang, ML et al.

Time to third-line treatment after bendamustine-rituximab with or without acalabrutinib in patients with previously untreated mantle cell lymphoma: Updated analysis of the phase 3 ECHO trial after 50 months of follow-up

Abstract #885

Oral Abstract Session

Session 623. Mantle Cell, Follicular, Waldenstrom’s, and Other Indolent B Cell Lymphomas: Clinical and Epidemiological: Single Agent and Combination therapy for MCL

8 December 2025
3:15 PM – 3:30 PM

Ultomiris (ravulizumab)

Schoettler, M et al.

Outcomes in pediatric patients with HSCT-TMA treated with ravulizumab

Abstract #1052

Oral Abstract Session

Session 723. Allogeneic Transplantation: Long-term Follow-up, Complications, and Disease Recurrence: Outcomes, toxicities and late effects

8 December 2025
4:45 PM – 05:00 PM

Chaudhury, S et al.

Organ dysfunction in pediatric patients with HSCT-TMA treated with ravulizumab

Abstract #4266

Poster Abstract Session

Session 723. Allogeneic Transplantation: Long-term Follow-up, Complications, and Disease Recurrence: Poster II

7 December 2025
6:00 PM – 8:00 PM

Sherrard, H et al.

Safety of ravulizumab use in pregnancy: Insights from a global pharmacovigilance analysis

Abstract #4458

Poster Abstract Session

Session 905. Outcomes Research: Non-Malignant Conditions Excluding Hemoglobinopathies: Poster II

7 December 2025
6:00 PM – 8:00 PM

Gandhi, S et al.

Real-world analysis of ravulizumab safety and effectiveness in advanced age patients with paroxysmal nocturnal hemoglobinuria: Insights from the international PNH registry

Abstract #6238

Poster Abstract Session

Session 905. Outcomes Research: Non-Malignant Conditions Excluding Hemoglobinopathies: Poster III

8 December 2025
6:00 PM – 8:00 PM

Fasenra (benralizumab)

Klion et al.

Efficacy and safety of benralizumab in patients with hypereosinophilic syndrome: Results from the Phase 3 natron study

Abstract #79

Oral Abstract Session

Session 634. Myeloproliferative Syndromes: Clinical and Epidemiological: Expanding the Therapeutic and Prognostic Landscape in Myeloproliferative Neoplasms, Mastocytosis and Hypereosinophilic Syndrome
06 December, 2025
9:30 AM – 9:45 AM

Klion et al.

Patient perspectives on the burden of hypereosinophilic syndrome: Results from the Phase 3 natron interview sub-study

Abstract #4465

Poster Presentation

Session 905. Outcomes Research: Non-Malignant Conditions Excluding Hemoglobinopathies: Poster II
07 December, 2025
6:00 PM – 8:00 PM

Voydeya (danicopan)

Kulasekararaj, A et al.

Danicopan add-on therapy demonstrates positive efficacy and safety outcomes in advanced age adults with paroxysmal nocturnal hemoglobinuria and clinically significant extravascular hemolysis: A sub-analysis of the phase 3 ALPHA trial

Abstract #949

Oral Abstract Session

Session 905. Outcomes Research: Non-Malignant Conditions Excluding Hemoglobinopathies: Antithrombotic Roulette: Balancing Risk, Cost, and Care

8 December 2025
2:45 PM – 03:00 PM

Surovatamig

Aldoss, I et al.

Safety and efficacy of surovatamig (AZD0486) in adolescent and adult patients with relapsed or refractory (R/R) B-cell acute lymphoblastic leukemia (B-ALL): Updated results from the Phase 1/2 SYRUS study

Abstract #3345

Poster Abstract Session

Session 613. Acute Lymphoblastic Leukemias: Therapies Excluding Allogeneic Transplantation: Poster II

7 December 2025
6:00 PM – 8:00 PM

Cheah, C et al.

SOUNDTRACK-B: A Phase 2 single-arm study to evaluate the efficacy and safety of surovatamig (AZD0486) in relapsed or refractory B-cell Non-Hodgkin lymphoma

Abstract #3747 (TiP)

Poster Abstract Session

Session 629. Aggressive Lymphomas, Immunotherapy including Bispecific Antibodies: Poster II

7 December 2025
6:00 PM – 8:00 PM

Hou, JZ et al.

Three-year follow-up of the Phase 1 first-in-human study investigating surovatamig, a novel CD19xCD3 T-cell engager, in patients with relapsed/refractory (R/R) follicular lymphoma (FL)

Abstract #1005

Oral Abstract Session

Session 623. Mantle Cell, Follicular, Waldenstrom’s, and Other Indolent B Cell Lymphomas: Clinical and Epidemiological – Follicular Lymphoma

8 December 2025
5:00 PM – 5:15 PM

Kim, TM et al.

Surovatamig (AZD0486), a CD19xCD3 T-cell engager (TCE), demonstrates high rate of minimal residual disease (MRD)-negative complete responses in relapsed/refractory (R/R) diffuse large B-cell lymphoma (DLBCL), including in patients who previously progressed on CD20 TCE and CD19 CAR T-cell therapies

Abstract #5514

Poster Abstract Session

Session 629. Aggressive Lymphomas, Immunotherapy including Bispecific Antibodies: Poster III

8 December 2025
6:00 PM – 8:00 PM

AZD0120

Du, J et al.

A dual targeting BCMA and CD19 FasTCAR-T (GC012F/AZD0120) as first-line therapy for newly diagnosed multiple myeloma

Abstract #258

Oral Abstract Session

Session 655. Multiple Myeloma: Cellular Therapies: Clinical Trial Advances in CAR T-Cell Therapy for Multiple Myeloma

6 December 2025
2:15 PM – 2:30 PM

Richard, S et al.

Safety and efficacy of AZD0120, a BCMA/CD19 dual-targeting CAR T-cell therapy, in relapsed/refractory multiple myeloma: Preliminary results from the DURGA-1 Phase 1b/2 study

Abstract #269

Oral Abstract Session

Session 704. Multiple Myeloma: Cellular Therapies: Clinical Trial Advances in CAR T-Cell Therapy for Multiple Myeloma

6 December 2025
3:00 PM – 3:15 PM

Feng, J et al.

One-year follow-up of CD19/BCMA dual-targeting FasTCAR-T GC012F (AZD0120) therapy in patients with refractory systemic lupus erythematosus

Abstract #2384

Poster Abstract Session

Session 704. Cellular Immunotherapies: Early Phase Clinical Trials and Toxicities: Poster I

6 December 2025
5:30 PM – 7:30 PM

Lentzsch, S et al.

ALACRITY: A Phase 1b/2 Study of AZD0120 (BCMA/CD19 CAR-T cell therapy) in Participants with Relapsed or Refractory Light Chain Amyloidosis (AL)

Abstract #8236

ePublication

3 November 2025

AZD4512

Han, H et al.

AZD4512: A novel CD22-directed antibody-drug conjugate for the treatment of b-cell malignancies

Abstract #3296

Poster Abstract Session

Session 605. Molecular Pharmacology and Drug Resistance: Lymphoid Neoplasms: Poster II

7 December 2025
6:00 PM – 8:00 PM

(Press release, AstraZeneca, DEC 4, 2025, View Source [SID1234661131])

OTR Therapeutics Raises $100 Million in Series A Financing to Advance Early Innovation into Global Transformative Therapies

On December 4, 2025 OTR Therapeutics, a biotechnology company dedicated to transforming early-stage innovations into globally impactful therapies, emerged from stealth and reported the successful completion of a $100 million Series A financing closed in June 2025. The round was backed by True Light Capital, a wholly-owned subsidiary of Temasek, LAV, Pfizer Ventures, and Sirona Capital.

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Founded in March 2025, OTR Therapeutics is establishing a novel, scalable, and capital-efficient model that synergizes internal R&D with strategic curation of high-potential external innovation. This integrated approach enables the company to focus on scientific rigor and operational agility, leveraging its deep regional insights and the development efficiency of the local ecosystem to accelerate innovative therapies to patients worldwide.

The proceeds of the financing will advance OTR’s pipeline of differentiated programs that target significant treatment gaps in immunology & inflammation, oncology, and other disease areas. The funds will also expand OTR’s R&D hub capability, which emphasizes both scientific excellence and strategic partnership agility to foster expedited development of high-impact therapies.

In line with its strategy, OTR also announced the acquisition of a preclinical program with best-in-class potential for neurological diseases with high unmet needs. This acquisition is part of the company’s strategic and ongoing efforts to identify and advance promising early-stage assets into global clinical development, alongside its internal proprietary discovery programs to address a broader range of critical patient needs.

"The rapidly evolving global pharmaceutical R&D landscape demands greater novelty, speed and efficiency," said Dr. Zhui Chen, Founder and CEO of OTR Therapeutics. "At OTR, we aim to build a next-generation biotech model that delivers unprecedented R&D and capital efficiency. It enables us to stay agile while remaining rigorously focused on propelling novel, differentiated drug candidates through global clinical translation in a disciplined and efficient manner. We are grateful for the strong support from our investors and their confidence in our vision and capability to deliver transformative therapies for patients."

Co-founded by Zhui Chen, Ph.D., Shannon Chuai, Ph.D., and Yuan Shi, Ph.D., OTR Therapeutics is led by a team of seasoned drug hunters and entrepreneurs in the biotech and pharmaceutical industries with a proven track record of delivering breakthrough innovation, operational excellence, and financial success.

Yi Shi, Managing Director, LAV: "We believe the biopharma industry is shifting towards more capital-efficient and specialized R&D models. OTR Therapeutics is at the forefront of this evolution, demonstrating how a focused, integrated framework can expedite the journey of translating early-stage innovation into global clinical development."

Michael Baran, Partner, Pfizer Ventures: "Pfizer Ventures identifies and invests in emerging companies who are developing innovative medicines and technologies that have the potential to shape the future of our industry. To this end, we’re pleased to be able to support OTR Therapeutics as it scales its R&D capabilities and builds a portfolio of potentially transformative therapies."

(Press release, OTR Therapeutics, DEC 4, 2025, View Source [SID1234661146])

TransThera Publishes Clinical Studies of Tinengotinib (TT-00420) against Cholangiocarcinoma on Lancet

On December 4, 2025 TransThera Sciences Inc. ("TransThera") reported the publication of clinical results from a US-based Phase 2 trial evaluating tinengotinib in patients with Cholangiocarcinoma (CCA) on The Lancet Gastroenterology and Hepatology (Impact Factor: 38.6).

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Cholangiocarcinoma (CCA) is an aggressive malignancy of the bile ducts, frequently driven by FGFR2 fusions-genomic alterations that are targetable by FGFR inhibitors such as pemigatinib and futibatinib. However, resistance to these agents commonly arises due to secondary FGFR2 mutations.

In a multicenter, open-label Phase 2 trial (NCT04919642), patients with FGFR2 fusion-positive CCA who had either primary resistance or developed acquired resistance to prior FGFR inhibitor (FGFRi) therapy were enrolled, along with patients harboring other FGFR alterations or FGFR wiled-type tumors. Tinengotinib demonstrated clinical activity in patients with FGFR2 fusion-positive CCA with acquired FGFRi resistance, as well as in those with other FGFR-altered subtypes.

Dr. Milind Javle of The University of Texas MD Anderson Cancer Center, corresponding author of the publication, stated: "We currently have two FDA-approved therapies targeting FGFR2 fusions in CCA. But resistance remains a major clinical challenge. As such, next generation FGFR inhibitors capable of overcoming resistance are urgently needed. Tinengotinib, as a multi-kinase FGFR inhibitor, is designed to inhibit both FGFR and compensatory pathways contributing to resistance. In this phase 2 study, tinengotinib demonstrated durable responses and meaningful clinical benefit. These promising results provide a strong rationale to proceed with a Phase 3 registration study".

Dr. Jean Fan, Chief Medical Officer of TransThera, also commented: "We are very pleased that the clinical trial results have gained peer recognition and were published in such a prestigious journal. This study provides important insights into treatment strategies for patients with FGFR-altered, chemotherapy- and FGFR inhibitor–refractory or relapsed CCA, including the comparison of tinengotinib versus physician’s choice. We are committed to advancing global enrollment and delivering new treatment options for patients with metastatic cholangiocarcinoma."

Disclaimer: This article serves as a press release by TransThera to disclose the company’s latest developments. It is not intended as a product promotion advertisement and does not constitute the company’s or investment advice.

About Tinengotinib

Tinengotinib is an internally discovered, registrational clinical stage, multi-kinase inhibitor that exerts antitumor effects by targeting FGFRs and VEGFRs, mitotic kinases Aurora A/B and Janus kinases (JAK). Ongoing clinical trials in the US and China have revealed the potential of tinengotinib to be efficacious in various solid tumors. It was granted the Orphan Drug Designation (ODD) and Fast Track Designation (FTD) by the FDA for the treatment of CCA, the Breakthrough Therapy Designation (BTD) by the National Medical Products Administration (NMPA) in China, the Orphan Drug Designation (ODD) for the treatment of biliary tract cancer by the European Medicines Agency (EMA). It was also approved for inclusion in the Priority Review and Approval Procedure by the NMPA for the treatment of CCA.

(Press release, TransThera Biosciences, DEC 4, 2025, View Source [SID1234661132])