RegulationBreakthrough Therapy (United States), Orphan Drug (United States), Orphan Drug (European Union), PRIME (European Union), Orphan Drug (Japan), Orphan Drug (South Korea), Paediatric investigation plan (European Union)

Structure/Sequence

Molecular FormulaC26H23BrFN7O3

InChIKeyPIBARDGJJAGJAJ-NQIIRXRSSA-N

CAS Registry1903768-17-1

Clinical Trials associated with Danicopan

NCT07413250

/ Active, not recruitingNot Applicable

A Study to Assess Long-Term Safety of Danicopan Add-on Therapy in Participants With Paroxysmal Nocturnal Hemoglobinuria: Analysis of International PNH Interest Group (IPIG)-Registry Data

This is a noninterventional registry-based cohort study utilizing data collected on danicopan-treated patients with paroxysmal nocturnal hemoglobinuria (PNH) through the International PNH Interest Group (IPIG) PNH registry. The primary objectives seek to characterize the long-term safety profile of danicopan as add-on therapy to ravulizumab/eculizumab in participants with PNH.

Start Date14 Jan 2026

Sponsor / Collaborator

Alexion Pharmaceuticals, Inc.

NCT07413679

/ Active, not recruitingNot Applicable

An Observational Study to Assess Long-Term Safety of Danicopan Add-on Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuria: Analysis of IPIG-Registry Data

This is a noninterventional cohort study using primary and secondary data from the International PNH Interest Group (IPIG) PNH registry. It is designed to characterize the long-term safety and tolerability of danicopan as add-on therapy to eculizumab or ravulizumab in adult participants with PNH.

Start Date18 Aug 2025

Sponsor / Collaborator

Alexion Pharmaceuticals, Inc.

NCT06449001

/ RecruitingPhase 3

A Phase 3 Open-Label Study of Danicopan as Add-on Treatment to Ravulizumab or Eculizumab in Pediatric Participants With Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Significant Extravascular Hemolysis

The primary objective of this study is to evaluate efficacy of danicopan as add-on treatment to ravulizumab or eculizumab as assessed by hemoglobin (Hgb) change from Baseline at Week 12 in pediatric participants with paroxysmal nocturnal hemoglobinuria (PNH) and clinically significant extravascular hemolysis (CS-EVH).

Start Date11 Aug 2025

Sponsor / Collaborator

Alexion Pharmaceuticals, Inc.

[+1]

100 Clinical Results associated with Danicopan

100 Translational Medicine associated with Danicopan

100 Patents (Medical) associated with Danicopan

Literatures (Medical) associated with Danicopan

01 Jul 2026·HemaSphere

Switching between complement inhibitors in paroxysmal nocturnal hemoglobinuria: Analysis of strategy, efficacy, and safety

Article

Author: Kulasekararaj, Austin ; Fattizzo, Bruno ; Álvarez, Miguel Gómez ; Kelly, Richard J. ; Griffin, Morag ; Patriquin, Christopher J. ; Lee, Jong Wook ; Panse, Jens ; Röth, Alexander ; Trikha, Roochi ; Munir, Talha ; Gandhi, Shreyans ; Pike, Alexandra ; Muus, Petra ; Nishimura, Jun‐ichi ; Ueda, Yasutaka

Abstract:

Paroxysmal nocturnal hemoglobinuria (PNH) is an ultra‐orphan disease. In 2026, approved complement inhibitors (CIs) for PNH include three C5 inhibitors (C5i) and three proximal inhibitors (PIs). Clinical trials for the approved PI pegcetacoplan, iptacopan, and C5i + danicopan had clear protocols for changing from terminal to PI, extrapolated into real‐world practice. There is no guidance for patients changing from a PI to a different CI. We present the largest international cohort to date. The inclusion criteria were as follows: patients established on a PI, who have changed treatment to a different CI. Sixty‐five patients with a median age at PNH diagnosis of 40 years were included. Indications for CI switch were as follows: hemolysis (extravascular/recurrent breakthrough/not specified), side effects, trial termination, patient choice, and other. CI changes (149) were classified as (1) terminal‐to‐proximal (75/149), 6 hemolytic events reported within 14 days of CI change; (2) proximal‐to‐proximal (45/149), 2 hemolytic events reported within 14 days of CI change; and (3) proximal‐to‐terminal (29/149), 13 hemolytic events within 14 days of CI change. This is the largest cohort of PNH patients facing multiple CI changes. With the increasing availability of different CI classes, patients may experience several treatment changes over their disease course. CI should not be interrupted. The cohort would suggest that overlapping treatment for patients changing from PI to another CI is not required. Tapering of treatment does not seem to be effective at preventing hemolysis complications. Patients should be monitored closely for hemolysis, especially when changing from proximal‐to‐terminal inhibition. Prospective clinical/laboratory analysis is recommended for further clarification management for this patient group.

01 Jun 2026·CURRENT THERAPEUTIC RESEARCH-CLINICAL AND EXPERIMENTAL

Understanding Pharmacokinetic-Drug Interactions With Drugs Approved by the US Food and Drug Administration in 2024 to Better Manage the Risk of Drug Interactions With Concomitant Medications: A Review of Clinical Data From New Drug Applications

Review

Author: Owens, Katie ; Argon, Sophie ; Yu, Jingjing ; Ragueneau-Majlessi, Isabelle ; Wang, Yan

Objective:

This analysis aimed to provide a mechanistic understanding and an evaluation of the clinical relevance of pharmacokinetic drug-drug interactions (DDIs) associated with drugs approved by the Food and Drug Administration in 2024.

Methods:

Drug metabolism, transport, and absorption-based DDI data available in New Drug Applications (NDAs) of small molecular drugs approved (n = 34) was analyzed. The mechanism and clinical outcome of these interactions were characterized based on in vitro, in silico, and clinical data.

Results:

As objects, 11 drugs were identified as clinical substrates. Of these, 7 drugs were substrates of CYP3A, 3 of CYP2C9, one of CYP1A2, and one of CYP2C8, including the sensitive substrates vanzacaftor (CYP3A) and vorasidenib (CYP1A2). As precipitants, 6 drugs (acoramidis, cefepime/enmetazobactam, givinostat, lazertinib, mavorixafor, and resmetirom) were clinical inhibitors of CYP enzymes (2C8, 2C9, 2D6, 2E1, and 3A), with mavorixafor being a CYP2D6 strong inhibitor. Two drugs (elafibranor and tovorafenib) showed weak induction of CYP3A. Regarding transporter data, 3 drugs were substrates of transporters, including seladelpar (BCRP and OAT3), sulopenem (OAT3), and vadadustat (OAT1/3), and 8 drugs (arimoclomol, danicopan, givinostat, lazertinib, mavorixafor, resmetirom, vadadustat, and vazacaftor/tezacaftor/deutivacaftor) were inhibitors of transporters. All clinical DDIs with AUC changes ≥ 2-fold triggered labeling recommendations. Several DDIs with an AUC change <2 also had labeling recommendations, pertaining most often to the concomitant use of drugs with a narrow therapeutic index.

Conclusions:

Mechanistic DDI insights from newly approved therapies can be extrapolated to inform the management of commonly co-administered drugs, supporting a safer and more effective use of new drug products in the context of polypharmacy.

01 May 2026·CURRENT OPINION IN OPHTHALMOLOGY

Systemic medications and their impact on age-related macular degeneration development and progression: a review of current evidence

Review

Author: Yuan, Melissa ; Alshaikhsalama, Ahmed ; Rahimy, Ehsan ; Alsoudi, Amer

Purpose of review:

This review examines commonly prescribed systemic medications and their possible associations with age-related macular degeneration (AMD) development and progression. With the limitations and risks of current intravitreal therapies, there is growing interest in oral pharmacotherapy for AMD management. The following review synthesizes observational studies, meta-analyses, and ongoing clinical trials to evaluate the potential effects of commonly used systemic medications on AMD.

Recent findings:

Metformin demonstrates conflicting evidence, with several meta-analyses and large cohort study showing reduced AMD odds, while a recent randomized phase II trial found no effect on geographic atrophy progression. For statins, emerging evidence suggests that treatment duration exceeding 2 years and medium-intensity to high-intensity dosing may confer protection against AMD development. Aspirin demonstrates discordant results between different study designs: two large randomized controlled trials showed no benefit for AMD, while a 10-year observational study suggested protective effects.Fenofibrates show promise in preclinical models but require additional clinical investigation. Danicopan also shows modest effects in complement-related disorders and is currently undergoing a phase 2 trial to evaluate efficacy in patients with geographic atrophy. Finally, dopamine agonists appear to improve visual acuity and reduce subretinal fluid and central retinal thickness in newly diagnosed exudative AMD, as shown in an open-label pilot study, but require further investigation.

Summary:

Multiple systemic medications have highlighted mixed or stage-dependent benefits on AMD development and progression. Some agents such as metformin and aspirin have shown conflicting findings, having been evaluated in randomized trials and large observational studies. Other medications including GLP-1 agonists, dopamine agonists, statins, fenofibrates, and danicopan show early promise in more limited studies, but require further clinical validation.

News (Medical) associated with Danicopan

09 Mar 2026

·www.biospace.com

NovelMed Therapeutics Announces Regulatory Clearance for Subcutaneous Ruxoprubart and Reports Positive Phase II Intravenous Monotherapy Results in PNH

--- Precision Bb Targeting, Robust Efficacy, Transfusion Independence, Excellent Safety in Phase II PNH Study, and Subcutaneous Administration Key Highlights: Monotherapy and Clinical Outcomes The 12-16-week open-label study evaluated Ruxoprubart as monotherapy in treatment-naïve PNH patients. Key findings include: Hemoglobin (Hb) Improvement: Hemoglobin levels increased by 1.5–2.7 g/dL, with an average rise of ~2.13 g/dL during weekly dosing, reflecting meaningful correction from the severely depressed baseline common in untreated PNH. Complete Transfusion Independence: Throughout the weekly dosing period, 100% of patients required no blood transfusions, demonstrating effective control of hemolysis and clinically meaningful correction of anemia. PNH Red Blood Cells (RBCs) Survival & Expansion: PNH RBC populations expanded to up to 94% of their maximum level. This is consistent with near-complete suppression of ongoing complement-mediated hemolysis, a core driver of anemia and fatigue. Biochemical Control (LDH): Lactate Dehydrogenase (LDH) levels were significantly reduced, reflecting biochemical control of intravascular hemolysis (IVH). Quality-of-Life (QoL) Gains: Patient‑reported outcomes, including the Functional Assessment of Chronic Illness Therapy (FACIT) and the European Organization for Research and Treatment of Cancer (EORTC) questionnaires, showed meaningful improvements across fatigue, pain, and overall functional capacity. Safety and Tolerability: The therapy was well-tolerated with no drug-related Adverse Events (AEs) or Serious Adverse Events (SAEs) observed. Orphan Drug Designation: Ruxoprubart holds U.S. FDA Orphan Drug Designation (ODD) for PNH, highlighting its potential to address this rare and life-threatening condition. CLEVELAND, March 09, 2026 (GLOBE NEWSWIRE) -- NovelMed Therapeutics Inc., a clinical-stage biopharmaceutical company specializing in alternative pathway (AP) complement-mediated therapies, today announced two major milestones in the clinical development of Ruxoprubart (NM8074) for Paroxysmal Nocturnal Hemoglobinuria (PNH). The company has received regulatory clearance to initiate a Phase II study for the subcutaneous (SC) route of administration, designed as a convenient, weekly, self-administered injection. Concurrently, NovelMed reported the full results of its Phase II intravenous (IV) monotherapy study in treatment-naïve adult patients with PNH. The study met all efficacy endpoints, including significant hemoglobin improvement and complete transfusion avoidance, with an exceptional safety profile. “These milestones validate both our clinical progress and our development strategy,” said Dr. Rekha Bansal, Chief Executive Officer of NovelMed. “Regulatory clearance of the SC route represents the next logical step toward a patient-friendly treatment option, while our monotherapy data demonstrate the strong, consistent efficacy and tolerability of targeting Bb. We are now progressing into the next phase of clinical development.” Advancing Two Complementary Tracks in PNH NovelMed is progressing Ruxoprubart through two distinct delivery methods to maximize patient access and convenience: Subcutaneous (SC) Administration: Cleared for Phase II Development The regulatory agency has cleared the SC route of administration, enabling a planned, weekly self-injection regimen at home. SC trials are planned but have not yet been initiated. This approach is expected to reduce patient and caregiver burden by eliminating the need for IV infusions in a clinical setting. Intravenous (IV) Monotherapy: Treatment-Naïve PNH Patients The successful completion of the intravenous Phase II study establishes Ruxoprubart as a potent monotherapy capable of addressing the core drivers of PNH without adjunctive treatment. “Ruxoprubart has proven its potential as a definitive monotherapy, delivering exceptional efficacy without compromise,” said Mr. Alex Kumar, Chief Strategy Advisor. “Coupled with FDA Orphan Drug Designation status and regulatory clearance for SC dosing, we have a clear, accelerated path forward. NovelMed is now uniquely positioned to lead the next generation of precision complement therapeutics on a global scale.” Precision Targeting: The Bb Mechanism and Differentiation PNH is an AP-driven hemolytic disorder. Ruxoprubart (NM8074) is a first-in-class monoclonal antibody engineered for precision targeting of Bb, the proteolytically activated catalytic fragment generated upon AP activation. By binding Bb rather than native Factor B, Ruxoprubart delivers precise AP-selective inhibition while preserving the Classical Pathway (CP), which remains essential for protecting patients from infections. “The Phase II intravenous results demonstrate a clinical efficacy and safety pro aligns well with FDA expectations for monotherapy development in PNH,” said Mr. Robert Bard. “With the SC route now cleared for evaluation under our Phase II program, we are advancing along a well-defined regulatory path. This progress brings us closer to enabling patient-friendly, self-administered treatment options supported by a mechanism designed for pathway-selective precision.” Competitive Landscape at a Glance Ruxoprubart (NovelMed): Targets activated Bb (FDA Orphan Drug Designation Holder). A precision monotherapy that provides selective AP inhibition while preserving the CP for host defense. Soliris® / Ultomiris® (AstraZeneca): Targets C5. A distal blocker of both AP and CP that fails to prevent C3b deposition, which leads to persistent anemia. Empaveli® / Syfovre® (Apellis): Targets C3. A proximal blocker that provides broad inhibition of both AP and CP, addressing C3b-mediated EVH but impacting overall complement surveillance. Fabhalta® (Novartis): Targets native Factor B. A proximal blocker that lacks precision by targeting the native protein rather than the proteolytically activated Bb fragment. Voydeya® (AstraZeneca): Targets Factor D. A proximal blocker, strictly approved as an add-on therapy to C5 inhibitors rather than a standalone monotherapy. Key Pillars of the Regulatory Strategy Monotherapy Treatment: Ruxoprubart has demonstrated the ability to meet all primary endpoints as a standalone treatment. This allows for a more direct regulatory path, as the drug does not need to be tested or approved alongside existing C5 inhibitors to prove its value. Subcutaneous (SC) Administration: The regulatory clearance for the SC route is a major milestone. Having passed rigorous safety and manufacturing reviews, Ruxoprubart is now cleared for human trials using at-home injections. This shifts the treatment model from clinical IV infusions to a more convenient, patient-controlled approach. Precision Targeting of the Bb: Ruxoprubart selectively targets the AP while keeping the CP intact. This "selective precision" is designed to maintain the body’s natural ability to fight infections, a differentiated safety pro supports the case for expedited regulatory designations like Fast Track or Breakthrough Therapy. Figure. Complement-mediated destruction of red blood cells in paroxysmal nocturnal hemoglobinuria (PNH). About Paroxysmal Nocturnal Hemoglobinuria (PNH) Paroxysmal Nocturnal Hemoglobinuria (PNH) is a rare, acquired, and life-threatening hematologic disorder caused by a somatic mutation in the PIGA gene. This mutation results in the deficiency of GPI-anchored complement regulatory proteins, specifically CD55 and CD59, which act as protective shields for RBCs. Without these proteins, RBCs are highly vulnerable to persistent attack by the AP of the complement system, leading to chronic intravascular and extravascular hemolysis. This ongoing destruction manifests clinically as severe anemia, dangerously elevated Lactate Dehydrogenase (LDH) levels, and a significantly increased risk of life-threatening thrombosis and renal impairment. Effective treatment is measured by stabilization of hemoglobin and the expansion of the PNH RBC clone, indicating that these cells are successfully protected from complement-mediated attack. About NovelMed Therapeutics NovelMed Therapeutics Inc. is a clinical-stage biopharmaceutical company dedicated to developing next-generation immunotherapies that selectively target the complement AP. The company has pioneered a site-specific approach by engineering monoclonal antibodies, such as its lead candidates Ruxoprubart (NM8074) and NM5072, to target proteolytically activated fragments, such as Bb, rather than native proteins. This precision enables potent blockade of the disease-driving AP while intentionally preserving the CP’s critical role in host defense and immune surveillance. Supported by a robust portfolio of intellectual property and promising Phase II data, NovelMed is advancing a differentiated pipeline of first-in-class biologics designed to achieve durable clinical outcomes without compromising the patient’s ability to fight infections. Strategic Partnership Opportunity Following the achievement of 100% transfusion avoidance in its Phase II monotherapy study and the recent regulatory clearance for SC administration, NovelMed is actively pursuing a strategic partnership to propel Ruxoprubart through late-stage global development. This program is exceptionally well-positioned for expansion, supported by U.S. FDA Orphan Drug Designation (ODD) and a validated, precision mechanism that addresses significant unmet needs in the PNH market. NovelMed seeks a partner with extensive clinical and commercial expertise to accelerate the regulatory path towards approval and support the global launch of this patient-friendly, self-administered therapy. By combining NovelMed’s innovative science with a partner's scale, the company aims to redefine the treatment landscape for rare complement-mediated diseases. Media Contact: Ya Gao, MS NovelMed Therapeutics, Inc. media@novelmed.com (216) 440 2696 Forward-Looking Statements This press release contains forward-looking statements that reflect NovelMed’s current expectations and projections about future events. Ruxoprubart (NM8074) is an investigational therapy and has not been approved by any regulatory authority for commercial use. The subcutaneous administration route has received regulatory clearance for further study; no subcutaneous clinical trials have been initiated. Forward-looking statements include expectations about clinical development, regulatory interactions, future study plans, and potential outcomes. NovelMed is a privately held, clinical stage biotechnology company which undertakes no obligation to update these statements except as required by law. A photo accompanying this announcement is available at

Phase 2Orphan DrugClinical ResultImmunotherapyFast Track

04 Dec 2025

·www.businesswire.com

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

WILMINGTON, Del.--(BUSINESS WIRE)--AstraZeneca advances its ambition to redefine hematology care with new data from its diverse pipeline and portfolio at the 67th American Society of Hematology (ASH) Annual Meeting and Exposition, December 6-9, 2025. This year’s ASH 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-cwvz) in pediatric patients with hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA) (Oral Abstract #1052). Anas Younes, Senior Vice President, Hematology 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, 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, we look forward to demonstrating how pioneering science can drive meaningful advances for people living with rare hematologic conditions. New data on ULTOMIRIS, including Phase III results in pediatric patients with HSCT-TMA, will show clinically meaningful overall survival and improved outcomes, highlighting our ongoing pursuit to realize 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 leukemia (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 leukemia, 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 hemoglobinuria (PNH) and clinically significant extravascular hemolysis, 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 Annual Meeting and Exposition Lead Author Abstract Title Presentation Details (ET) CALQUENCE® (acalabrutinib) 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 December 6, 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 December 7, 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 December 7, 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 December 8, 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 December 7, 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 December 6, 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 December 8, 2025 3:15 PM – 3:30 PM ULTOMIRIS® (ravulizumab-cwvz) 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 December 8, 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 December 7, 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 December 7, 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 December 8, 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 December 6, 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 December 7, 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 December 8, 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 December 7, 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 December 7, 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 December 8, 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 December 8, 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 December 6, 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 December 6, 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 December 6, 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 November 3, 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 December 7, 2025 6:00 PM – 8:00 PM INDICATIONS AND USAGE CALQUENCE is a Bruton tyrosine kinase (BTK) inhibitor indicated: In combination with bendamustine and rituximab (BR) for the treatment of adult patients with previously untreated mantle cell lymphoma (MCL) who are ineligible for autologous hematopoietic stem cell transplantation (HSCT). For the treatment of adult patients with MCL who have received at least one prior therapy. For the treatment of adult patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL). IMPORTANT SAFETY INFORMATION ABOUT CALQUENCE® (acalabrutinib) tablets Serious and Opportunistic Infections Fatal and serious infections, including opportunistic infections, have occurred in patients with hematologic malignancies treated with CALQUENCE. Serious or Grade 3 or higher infections (bacterial, viral, or fungal) occurred in 32% of 1,764 patients exposed to CALQUENCE in clinical trials, most often due to respiratory tract infections (19% of all patients, including pneumonia in 9%). These infections predominantly occurred in the absence of Grade 3 or 4 neutropenia, with neutropenic infection reported in 2.7% of all patients. Opportunistic infections in recipients of CALQUENCE have included, but are not limited to, hepatitis B virus reactivation, fungal pneumonia, Pneumocystis jirovecii pneumonia, Epstein-Barr virus reactivation, cytomegalovirus, and progressive multifocal leukoencephalopathy (PML). Consider prophylaxis in patients who are at increased risk for opportunistic infections. Monitor patients for signs and symptoms of infection and treat promptly. Hemorrhage Fatal and serious hemorrhagic events have occurred in patients treated with CALQUENCE. Major hemorrhage (serious or Grade 3 or higher bleeding or any central nervous system bleeding) occurred in 4.4% of patients, with fatal hemorrhage occurring in 0.2% of 1,764 patients exposed to CALQUENCE in clinical trials. Bleeding events of any grade, excluding bruising and petechiae, occurred in 40% of patients. Use of antithrombotic agents concomitantly with CALQUENCE may further increase the risk of hemorrhage. In clinical trials, major hemorrhage occurred in 7% of patients taking CALQUENCE without antithrombotic agents and 4% of patients taking CALQUENCE with antithrombotic agents. Consider the risks and benefits of antithrombotic agents when co-administered with CALQUENCE. Monitor patients for signs of bleeding. Consider the benefit-risk of withholding CALQUENCE for 3 to 7 days pre- and post-surgery depending upon the type of surgery and the risk of bleeding. Cytopenias CALQUENCE can cause Grade 3 or 4 cytopenias. Grade 3 or 4 cytopenias included absolute neutrophil count decreased (26%), platelets decreased (10%), hemoglobin decreased (10%), and absolute lymphocyte count decreased (10%) in patients treated with CALQUENCE alone or in combination with obinutuzumab; Grade 4 neutropenia developed in 14% of patients. Monitor complete blood counts regularly during treatment. Interrupt treatment, reduce the dose, or discontinue treatment as warranted. Second Primary Malignancies Second primary malignancies, including skin cancers and other solid tumors, occurred in 18% of 1,764 patients exposed to CALQUENCE in clinical trials. The most frequent second primary malignancy was non-melanoma skin cancer, reported in 10% of patients, followed by other solid tumors in 9% (including melanoma, lung cancer, gastrointestinal cancers, and genitourinary cancers) and hematologic malignancies (1%). Monitor patients for the development of second cancers and advise protection from sun exposure. Cardiac Arrhythmias Fatal and serious cardiac arrhythmias have occurred in patients treated with CALQUENCE. Grade 3 or 4 atrial fibrillation or flutter was reported in 2.6% of 1,764 patients treated with CALQUENCE, with all grades of atrial fibrillation or flutter reported in 7% of all patients. Grade 3 or higher ventricular arrhythmia events were reported in 0.6% of patients, including fatal cases in 0.3% of all patients. The risk of arrhythmias may be increased in patients with cardiac risk factors, hypertension, previous arrhythmias, and acute infection. Monitor for symptoms of arrhythmia (e.g., palpitations, dizziness, syncope, dyspnea) and manage as appropriate. Hepatotoxicity, Including Drug-Induced Liver Injury Hepatotoxicity, including severe, life-threatening, and potentially fatal cases of drug-induced liver injury (DILI), has occurred in patients treated with Bruton tyrosine kinase inhibitors, including CALQUENCE. Evaluate bilirubin and transaminases at baseline and throughout treatment with CALQUENCE. For patients who develop abnormal liver tests after CALQUENCE, monitor more frequently for liver test abnormalities and clinical signs and symptoms of hepatic toxicity. If DILI is suspected, withhold CALQUENCE. Upon confirmation of DILI, discontinue CALQUENCE. ADVERSE REACTIONS Previously Untreated Mantle Cell Lymphoma The most common adverse reactions (≥15%) of any grade in patients with previously untreated MCL who received CALQUENCE plus BR were rash (47%), COVID-19 (38%), fatigue (37%), diarrhea (37%), pneumonia (31%), headache (31%), upper respiratory tract infection (30%), pyrexia (29%), cough (27%), vomiting (26%), constipation (25%), hemorrhage (20%), edema (20%), secondary primary malignancy (19%), dizziness (18%), arthralgia (18%), and dyspnea (17%). Grade 4 laboratory abnormalities in >15% of patients treated with CALQUENCE plus BR include lymphocytes decreased (26%), absolute neutrophils decreased (36%), and uric acid increased (17%). Serious adverse reactions occurred in 69% of patients who received CALQUENCE plus BR. Serious adverse reactions reported in ≥2% of patients were pneumonia (23%; includes COVID-19 pneumonia), COVID-19 (20%; includes COVID-19 pneumonia), second primary malignancy (7%), pyrexia (6%), rash (3.4%), febrile neutropenia (3.4%), atrial fibrillation (3%), sepsis (2.7%), and anemia (2.4%). Fatal adverse reactions that occurred within 30 days of the last study treatment were reported in 12% who received CALQUENCE plus BR including COVID-19 (6%; includes COVID-19 pneumonia), pneumonia (1%), second primary malignancy (0.7%), sepsis (0.3%), and pneumonitis (0.3%). Adverse reactions led to permanent discontinuation of CALQUENCE in 43%, dosage interruptions in 74%, and dosage reductions in 10% of patients. Adverse reactions that resulted in dosage modification in >10% included infections, cytopenias, rashes, and gastrointestinal toxicity. Adverse reactions which resulted in permanent discontinuation of CALQUENCE in ≥4% of patients included COVID-19 (includes COVID-19 pneumonia) and neutropenia. Previously Treated Mantle Cell Lymphoma The most common adverse reactions (≥20%) of any grade in patients with relapsed or refractory MCL exposed to CALQUENCE were anemia,* thrombocytopenia,* headache (39%), neutropenia,* diarrhea (31%), fatigue (28%), myalgia (21%), and bruising (21%). The most common Grade ≥3 non-hematological adverse reaction (reported in at least 2% of patients) was diarrhea (3.2%). *Treatment-emergent decreases (all grades) of hemoglobin (46%), platelets (44%), and neutrophils (36%) were based on laboratory measurements and adverse reactions. Dose reductions or discontinuations due to any adverse reaction were reported in 1.6% and 6.5% of patients, respectively. Increases in creatinine to 1.5 to 3 times the upper limit of normal (ULN) occurred in 4.8% of patients. Chronic Lymphocytic Leukemia The most common adverse reactions (≥30%) of any grade in patients with CLL exposed to CALQUENCE were anemia,* neutropenia,* thrombocytopenia,* headache, upper respiratory tract infection, and diarrhea. *Treatment-emergent decreases (all grades) of hemoglobin, platelets, and neutrophils were based on laboratory measurements and adverse reactions. In patients with previously untreated CLL exposed to CALQUENCE, fatal adverse reactions that occurred in the absence of disease progression and with onset within 30 days of the last study treatment were reported in 2% for each treatment arm, most often from infection. Serious adverse reactions were reported in 39% of patients in the CALQUENCE plus obinutuzumab arm and 32% in the CALQUENCE monotherapy arm, most often due to events of pneumonia (2.8% to 7%). Adverse reactions led to CALQUENCE dose reduction in 7% and 4% of patients in the CALQUENCE plus obinutuzumab arm (N=178) and CALQUENCE monotherapy arm (N=179), respectively. Adverse events led to discontinuation in 11% and 10% of patients, respectively. Increases in creatinine to 1.5 to 3 times ULN occurred in 3.9% and 2.8% of patients in the CALQUENCE combination arm and monotherapy arm, respectively. In patients with relapsed/refractory CLL exposed to CALQUENCE, serious adverse reactions occurred in 29% of patients. Serious adverse reactions in >5% of patients who received CALQUENCE included lower respiratory tract infection (6%). Fatal adverse reactions within 30 days of the last dose of CALQUENCE occurred in 2.6% of patients, including from second primary malignancies and infection. Adverse reactions led to CALQUENCE dose reduction in 3.9% of patients (N=154), dose interruptions in 34% of patients, most often due to respiratory tract infections followed by neutropenia, and discontinuation in 10% of patients, most frequently due to second primary malignancies followed by infection. Increases in creatinine to 1.5 to 3 times ULN occurred in 1.3% of patients who received CALQUENCE. DRUG INTERACTIONS Strong CYP3A Inhibitors: Avoid co-administration of CALQUENCE with a strong CYP3A inhibitor. If these inhibitors will be used short-term, interrupt CALQUENCE. After discontinuation of strong CYP3A inhibitor for at least 24 hours, resume previous dosage of CALQUENCE. Moderate CYP3A Inhibitors: Reduce the dosage of CALQUENCE to 100 mg once daily when co-administered with a moderate CYP3A inhibitor. Strong CYP3A Inducers: Avoid co-administration of CALQUENCE with a strong CYP3A inducer. If co-administration is unavoidable, increase the dosage of CALQUENCE to 200 mg approximately every 12 hours. SPECIFIC POPULATIONS Based on findings in animals, CALQUENCE may cause fetal harm and dystocia when administered to a pregnant woman. There are no available data in pregnant women to inform the drug-associated risk. Advise pregnant women of the potential risk to a fetus. Pregnancy testing is recommended for females of reproductive potential prior to initiating CALQUENCE therapy. Advise female patients of reproductive potential to use effective contraception during treatment with CALQUENCE and for 1 week following the last dose of CALQUENCE. It is not known if CALQUENCE is present in human milk. Advise lactating women not to breastfeed while taking CALQUENCE and for 2 weeks after the last dose. Avoid use of CALQUENCE in patients with severe hepatic impairment (Child-Pugh class C). No dosage adjustment of CALQUENCE is recommended in patients with mild (Child-Pugh class A) or moderate (Child-Pugh class B) hepatic impairment. Please see full Prescribing Information, including Patient Information. INDICATIONS & IMPORTANT SAFETY INFORMATION for ULTOMIRIS INDICATIONS Paroxysmal Nocturnal Hemoglobinuria (PNH) ULTOMIRIS is indicated for the treatment of adult and pediatric patients one month of age and older with paroxysmal nocturnal hemoglobinuria (PNH). Atypical Hemolytic Uremic Syndrome (aHUS) ULTOMIRIS is indicated for the treatment of adult and pediatric patients one month of age and older with atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy (TMA). Limitation of Use: ULTOMIRIS is not indicated for the treatment of patients with Shiga toxin E. coli related hemolytic uremic syndrome (STEC-HUS). Generalized Myasthenia Gravis (gMG) ULTOMIRIS is indicated for the treatment of adult patients with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody-positive. Neuromyelitis Optica Spectrum Disorder (NMOSD) ULTOMIRIS is indicated for the treatment of adult patients with neuromyelitis optica spectrum disorder (NMOSD) who are anti-aquaporin-4 (AQP4) antibody positive. IMPORTANT SAFETY INFORMATION WARNING: SERIOUS MENINGOCOCCAL INFECTIONS ULTOMIRIS, a complement inhibitor, increases the risk of serious infections caused by Neisseria meningitidis [see Warnings and Precautions (5.1)] Life-threatening and fatal meningococcal infections have occurred in patients treated with complement inhibitors. These infections may become rapidly life-threatening or fatal if not recognized and treated early. Because of the risk of serious meningococcal infections, ULTOMIRIS is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called ULTOMIRIS and SOLIRIS REMS [see Warnings and Precautions (5.2)]. CONTRAINDICATIONS Initiation in patients with unresolved serious Neisseria meningitidis infection. WARNINGS AND PRECAUTIONS Serious Meningococcal Infections ULTOMIRIS, a complement inhibitor, increases a patient’s susceptibility to serious, life-threatening, or fatal infections caused by meningococcal bacteria (septicemia and/or meningitis) in any serogroup, including non-groupable strains. Life-threatening and fatal meningococcal infections have occurred in both vaccinated and unvaccinated patients treated with complement inhibitors. Revaccinate patients in accordance with ACIP recommendations considering the duration of ULTOMIRIS therapy. Note that ACIP recommends an administration schedule in patients receiving complement inhibitors that differs from the administration schedule in the vaccine prescribing information. If urgent ULTOMIRIS therapy is indicated in a patient who is not up to date with meningococcal vaccines according to ACIP recommendations, provide antibacterial drug prophylaxis and administer meningococcal vaccines as soon as possible. Various durations and regimens of antibacterial drug prophylaxis have been considered, but the optimal durations and drug regimens for prophylaxis and their efficacy have not been studied in unvaccinated or vaccinated patients receiving complement inhibitors, including ULTOMIRIS. The benefits and risks of treatment with ULTOMIRIS, as well as those associated with antibacterial drug prophylaxis in unvaccinated or vaccinated patients, must be considered against the known risks for serious infections caused by Neisseria meningitidis. Vaccination does not eliminate the risk of serious meningococcal infections, despite development of antibodies following vaccination. Closely monitor patients for early signs and symptoms of meningococcal infection and evaluate patients immediately if infection is suspected. Inform patients of these signs and symptoms and instruct patients to seek immediate medical care if they occur. Promptly treat known infections. Meningococcal infection may become rapidly life-threatening or fatal if not recognized and treated early. Consider interruption of ULTOMIRIS in patients who are undergoing treatment for serious meningococcal infection depending on the risks of interrupting treatment in the disease being treated. ULTOMIRIS and SOLIRIS REMS Due to the risk of serious meningococcal infections, ULTOMIRIS is available only through a restricted program called ULTOMIRIS and SOLIRIS REMS. Prescribers must enroll in the REMS, counsel patients about the risk of serious meningococcal infection, provide patients with the REMS educational materials, assess patient vaccination status for meningococcal vaccines (against serogroups A, C, W, Y, and B) and vaccinate if needed according to current ACIP recommendations two weeks prior to the first dose of ULTOMIRIS. Antibacterial drug prophylaxis must be prescribed if treatment must be started urgently, and the patient is not up to date with both meningococcal vaccines according to current ACIP recommendations at least two weeks prior to the first dose of ULTOMIRIS. Patients must receive counseling about the need to receive meningococcal vaccines and to take antibiotics as directed, signs and symptoms of meningococcal infection, and be instructed to carry the Patient Safety Card at all times during and for 8 months following ULTOMIRIS treatment. Further information is available at www.UltSolREMS.com or 1-888-765-4747. Other Infections Serious infections with Neisseria species (other than Neisseria meningitidis), including disseminated gonococcal infections, have been reported. ULTOMIRIS blocks terminal complement activation; therefore, patients may have increased susceptibility to infections, especially with encapsulated bacteria, such as infections caused by Neisseria meningitidis but also Streptococcus pneumoniae, Haemophilus influenzae, and to a lesser extent, Neisseria gonorrhoeae. Children treated with ULTOMIRIS may be at increased risk of developing serious infections due to Streptococcus pneumoniae and Haemophilus influenzae type b (Hib). Administer vaccinations for the prevention of Streptococcus pneumoniae and Haemophilus influenzae type b (Hib) infections according to ACIP recommendations. Patients receiving ULTOMIRIS are at increased risk for infections due to these organisms, even if they develop antibodies following vaccination. Monitoring Disease Manifestations after ULTOMIRIS Discontinuation Treatment Discontinuation for PNH After discontinuing treatment with ULTOMIRIS, closely monitor for signs and symptoms of hemolysis, identified by elevated LDH along with sudden decrease in PNH clone size or hemoglobin, or re-appearance of symptoms such as fatigue, hemoglobinuria, abdominal pain, shortness of breath (dyspnea), major adverse vascular event (including thrombosis), dysphagia, or erectile dysfunction. Monitor any patient who discontinues ULTOMIRIS for at least 16 weeks to detect hemolysis and other reactions. If signs and symptoms of hemolysis occur after discontinuation, including elevated LDH, consider restarting treatment with ULTOMIRIS. Treatment Discontinuation for aHUS ULTOMIRIS treatment of aHUS should be a minimum duration of 6 months. Due to heterogeneous nature of aHUS events and patient-specific risk factors, treatment duration beyond the initial 6 months should be individualized. There are no specific data on ULTOMIRIS discontinuation. After discontinuing treatment with ULTOMIRIS, patients should be monitored for clinical symptoms and laboratory signs of TMA complications for at least 12 months. TMA complications post-discontinuation can be identified if any of the following is observed: Clinical symptoms of TMA include changes in mental status, seizures, angina, dyspnea, thrombosis or increasing blood pressure. In addition, at least two of the following laboratory signs observed concurrently and results should be confirmed by a second measurement 28 days apart with no interruption: a decrease in platelet count of 25% or more as compared to either baseline or to peak platelet count during ULTOMIRIS treatment; an increase in serum creatinine of 25% or more as compared to baseline or to nadir during ULTOMIRIS treatment; or, an increase in serum LDH of 25% or more as compared to baseline or to nadir during ULTOMIRIS treatment. If TMA complications occur after discontinuation, consider reinitiation of ULTOMIRIS treatment or appropriate organ-specific supportive measures. Thromboembolic Event Management The effect of withdrawal of anticoagulant therapy during treatment with ULTOMIRIS has not been established. Treatment should not alter anticoagulant management. Infusion-Related Reactions Administration of ULTOMIRIS may result in systemic infusion-related reactions, including anaphylaxis and hypersensitivity reactions. In clinical trials, infusion-related reactions occurred in approximately 1 to 7% of patients, including lower back pain, abdominal pain, muscle spasms, drop or elevation in blood pressure, rigors, limb discomfort, drug hypersensitivity (allergic reaction), and dysgeusia (bad taste). These reactions did not require discontinuation of ULTOMIRIS. If signs of cardiovascular instability or respiratory compromise occur, interrupt ULTOMIRIS and institute appropriate supportive measures. ADVERSE REACTIONS Adverse Reactions for PNH Adverse reactions reported in ≥10% or more of patients with PNH were upper respiratory tract infection and headache. Serious adverse reactions were reported in 15 (6.8%) patients receiving ULTOMIRIS. The serious adverse reactions in patients treated with ULTOMIRIS included hyperthermia and pyrexia. No serious adverse reaction was reported in more than 1 patient treated with ULTOMIRIS. One fatal case of sepsis was identified in a patient treated with ULTOMIRIS. In clinical studies, clinically relevant adverse reactions in 1% of adult patients include infusion-related reactions. Adverse reactions reported in ≥10% of pediatric patients treated with ULTOMIRIS who were treatment-naïve vs. Eculizumab-experienced were anemia (20% vs. 25%), abdominal pain (0% vs. 38%), constipation (0% vs. 25%), pyrexia (20% vs. 13%), upper respiratory tract infection (20% vs. 75%), pain in extremity (0% vs. 25%), and headache (20% vs. 25%). Adverse Reactions for aHUS Most common adverse reactions in patients with aHUS (incidence ≥20%) were upper respiratory tract infection, diarrhea, nausea, vomiting, headache, hypertension and pyrexia. Serious adverse reactions were reported in 42 (57%) patients with aHUS receiving ULTOMIRIS. The most frequent serious adverse reactions reported in more than 2 patients (2.7%) treated with ULTOMIRIS were hypertension, pneumonia and abdominal pain. Adverse reactions reported in ≥20% of pediatric patients treated with ULTOMIRIS were diarrhea, constipation, vomiting, pyrexia, upper respiratory tract infection, decreased vitamin D, headache, cough, rash, and hypertension. Adverse Reactions for gMG Most common adverse reactions in adult patients with gMG (incidence ≥10%) were diarrhea and upper respiratory tract infection. Serious adverse reactions were reported in 20 (23%) of patients treated with ULTOMIRIS and in 14 (16%) patients receiving placebo. The most frequent serious adverse reactions were infections reported in at least 8 (9%) patients treated with ULTOMIRIS and in 4 (4%) patients treated with placebo. Of these infections, one fatal case of COVID-19 pneumonia was identified in a patient treated with ULTOMIRIS and one case of infection led to discontinuation of ULTOMIRIS. Adverse Reactions for NMOSD Most common adverse reactions in adult patients with NMOSD (incidence ≥10%) were COVID-19, headache, back pain, arthralgia, and urinary tract infection. Serious adverse reactions were reported in 8 (13.8%) patients with NMOSD receiving ULTOMIRIS. DRUG INTERACTIONS Plasma Exchange, Plasmapheresis, and Intravenous Immunoglobulins Concomitant use of ULTOMIRIS with plasma exchange (PE), plasmapheresis (PP), or intravenous immunoglobulin (IVIg) treatment can reduce serum ravulizumab concentrations and requires a supplemental dose of ULTOMIRIS. Neonatal Fc Receptor Blockers Concomitant use of ULTOMIRIS with neonatal Fc receptor (FcRn) blockers (e.g., efgartigimod) may lower systemic exposures and reduce effectiveness of ULTOMIRIS. Closely monitor for reduced effectiveness of ULTOMIRIS. USE IN SPECIFIC POPULATIONS Pregnancy Exposure Registry There is a pregnancy exposure registry that monitors pregnancy outcomes in women exposed to ULTOMIRIS during pregnancy. Healthcare providers and patients may call 1-833-793-0563 or go to www.UltomirisPregnancyStudy.com to enroll in or to obtain information about the registry. To report SUSPECTED ADVERSE REACTIONS, contact Alexion Pharmaceuticals, Inc. at 1-844-259-6783 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. Please see accompanying full Prescribing Information for ULTOMIRIS, including Boxed WARNING regarding serious and life-threatening or fatal meningococcal infections. INDICATION & IMPORTANT SAFETY INFORMATION FOR VOYDEYA™ (danicopan) INDICATION VOYDEYA is indicated as an add-on therapy to ravulizumab or eculizumab for the treatment of extravascular hemolysis (EVH) in adults with paroxysmal nocturnal hemoglobinuria (PNH). Limitation of Use: VOYDEYA has not been shown to be effective as monotherapy and should only be prescribed as an add-on to ravulizumab or eculizumab. IMPORTANT SAFETY INFORMATION WARNING: SERIOUS INFECTIONS CAUSED BY ENCAPSULATED BACTERIA VOYDEYA, a complement inhibitor, increases the risk of serious infections, especially those caused by encapsulated bacteria, such as Neisseria meningitidis, Streptococcus pneumoniae, and Haemophilus influenzae type B [see Warnings and Precautions (5.1)]. Life-threatening and fatal infections with encapsulated bacteria have occurred in patients treated with complement inhibitors. These infections may become rapidly life-threatening or fatal if not recognized and treated early. Because of the risk of serious infections caused by encapsulated bacteria, VOYDEYA is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the VOYDEYA REMS [see Warnings and Precautions (5.2)]. CONTRAINDICATIONS Initiation in patients with unresolved serious infection caused by encapsulated bacteria, including Neisseria meningitidis, Streptococcus pneumoniae, or Haemophilus influenzae type B. WARNINGS AND PRECAUTIONS Serious Infections Caused by Encapsulated Bacteria VOYDEYA, a complement inhibitor, increases a patient’s susceptibility to serious, life-threatening, or fatal infections caused by encapsulated bacteria, including Neisseria meningitidis (caused by any serogroup, including non-groupable strains), Streptococcus pneumoniae, and Haemophilus influenzae type B. Life-threatening and fatal infections with encapsulated bacteria have occurred in both vaccinated and unvaccinated patients treated with complement inhibitors. Complete, update, or revaccinate patients in accordance with ACIP recommendations considering the duration of VOYDEYA therapy. Note that ACIP recommends an administration schedule in patients receiving complement inhibitors that differs from the administration schedule in the vaccine prescribing information. If urgent VOYDEYA therapy is indicated in a patient who is not up to date with vaccines against encapsulated bacteria according to ACIP recommendations, provide antibacterial drug prophylaxis and administer these vaccines as soon as possible. Various durations and regimens of antibacterial drug prophylaxis have been considered, but the optimal durations and drug regimens for prophylaxis and their efficacy have not been studied in unvaccinated or vaccinated patients receiving complement inhibitors, including VOYDEYA. The benefits and risks of treatment with VOYDEYA, as well as those associated with antibacterial drug prophylaxis in unvaccinated or vaccinated patients, must be considered against the known risks for serious infections caused by encapsulated bacteria. Vaccination does not eliminate the risk of serious encapsulated bacterial infections, despite development of antibodies following vaccination. Closely monitor patients for early signs and symptoms of serious infection and evaluate patients immediately if an infection is suspected. Inform patients of these signs and symptoms and instruct patients to seek immediate medical care if they occur. Promptly treat known infections. Serious infection may become rapidly life-threatening or fatal if not recognized and treated early. Consider interruption of VOYDEYA in patients who are undergoing treatment for serious infections. VOYDEYA REMS Due to the risk of serious infections caused by encapsulated bacteria, VOYDEYA is available only through a restricted program called VOYDEYA REMS. Per the REMS requirements: Prescribers must enroll in the REMS, counsel patients about the risk of serious infections caused by encapsulated bacteria, provide patients with the REMS educational materials, assess patient vaccination status for vaccines against encapsulated bacteria, and vaccinate if needed according to current ACIP recommendations 2 weeks prior to the first dose of VOYDEYA. Antibacterial drug prophylaxis must be prescribed if treatment must be started urgently and the patient is not up to date with vaccines against encapsulated bacteria according to current ACIP recommendations at least 2 weeks prior to the first dose of VOYDEYA. Pharmacies that dispense VOYDEYA must be certified in the VOYDEYA REMS and must verify prescribers are certified. Patients must receive counseling from the prescriber about the need to receive vaccinations against encapsulated bacteria per ACIP recommendations, to take antibiotics as directed, the early signs and symptoms of serious infection, and be instructed to carry the Patient Safety Card at all times during and for 1 week following the last dose of VOYDEYA. Further information is available at www.voydeyarems.com or 1-888-765-4747. Hepatic Enzyme Increases Hepatic enzyme elevations have been observed in patients treated with VOYDEYA. A total of 14% of patients receiving VOYDEYA had elevations in serum alanine aminotransferase (ALT). ALT elevations >3× the upper limit of normal (ULN) and ≤5× ULN occurred in 9% of VOYDEYA-treated patients, and ALT elevations >5× ULN and ≤10× ULN occurred in 5% of VOYDEYA-treated patients. Assess liver enzyme test results prior to the initiation of VOYDEYA and periodically during treatment. Consider treatment interruption or discontinuation if elevations are clinically significant or if the patient becomes symptomatic. VOYDEYA has not been studied in patients with severe hepatic impairment. Monitoring of PNH Manifestations After VOYDEYA Discontinuation After discontinuing treatment with VOYDEYA, closely monitor patients for at least 2 weeks after the last dose for signs and symptoms of hemolysis. If discontinuation of VOYDEYA is necessary, continue background treatment with ravulizumab or eculizumab or consider alternative therapy if necessary. The signs and symptoms of hemolysis may include sudden decrease in hemoglobin or fatigue. If hemolysis occurs after discontinuation of VOYDEYA, consider restarting treatment with VOYDEYA, if appropriate. Hyperlipidemia VOYDEYA increases total cholesterol and LDL-cholesterol. Of the 50 VOYDEYA-treated patients who had a normal total cholesterol level at baseline, 30% developed Grade 1 hypercholesterolemia. Of the 6 VOYDEYA-treated patients who had Grade 1 hypercholesterolemia at baseline, 1 patient experienced increased total cholesterol that worsened to Grade 2. Of the 54 VOYDEYA-treated patients who had LDL-cholesterol ≤130 mg/dL at baseline, 13% developed LDL-cholesterol >130-160 mg/dL, and 9% developed LDL-cholesterol >160-190 mg/dL. Some patients required cholesterol-lowering medications. Monitor serum lipid parameters periodically during treatment with VOYDEYA and initiate cholesterol-lowering medication, if indicated. ADVERSE REACTIONS The most common adverse reaction reported in ≥10% of patients treated with VOYDEYA was headache. Serious adverse reactions were reported in 5% of patients who received VOYDEYA and included pancreatitis, cholecystitis, and increased blood bilirubin. No specific serious adverse reaction was reported in more than 1 patient treated with VOYDEYA. Adverse reactions reported in ≥5% of patients treated with VOYDEYA and greater than placebo in the randomized, controlled period included vomiting, pyrexia, increased alanine aminotransferase, hypertension, and pain in the extremities. Clinically relevant adverse reactions in <5% of patients included increased serum triglycerides. DRUG INTERACTIONS BCRP Substrates Danicopan is a Breast Cancer Resistance Protein (BCRP) inhibitor. Concomitant use of VOYDEYA with a BCRP substrate increases the plasma concentrations of the BCRP substrate, which may increase the risk for adverse reactions associated with the BCRP substrate. If used together, monitor patients more frequently for adverse reactions, associated with the BCRP substrate and consider dose reduction of the BCRP substrate according to its prescribing information. Rosuvastatin Danicopan significantly increased rosuvastatin exposure. The dose of rosuvastatin should not exceed 10mg once daily when concomitantly used with VOYDEYA. P-glycoprotein Substrates Danicopan is an inhibitor of P-glycoprotein (P-gp). Concomitant administration of VOYDEYA with P-gp substrates may increase the plasma concentrations of the P-gp substrates. Dose adjustment might be necessary for P-gp substrates where minimal concentration changes may lead to serious adverse reactions. USE IN SPECIFIC POPULATIONS Pregnancy There are no available data on VOYDEYA use in pregnant individuals to evaluate for a drug-associated risk of major birth defects, miscarriage, or adverse maternal or fetal outcomes. There are risks to the mother and fetus associated with untreated PNH in pregnancy. The use of VOYDEYA in pregnant women or women planning to become pregnant may be considered following an assessment of the risks and benefits. Lactation There are no data on the presence of VOYDEYA in human milk, the effects on the breastfed child, or the effect on milk production. VOYDEYA is present in animal milk. When a drug is present in animal milk, it is likely that the drug will be present in human milk. Because of the potential for serious adverse reactions in the breastfed child, including serious infections with encapsulated bacteria and liver enzyme increases, advise patients not to breastfeed during treatment with VOYDEYA and for 3 days after the last dose. Hepatic Impairment No dose adjustment is required in patients with mild to moderate hepatic impairment. Studies have not been conducted in patients with severe hepatic impairment, therefore, avoid use of VOYDEYA in this patient population. To report SUSPECTED ADVERSE REACTIONS, contact Alexion Pharmaceuticals, Inc. at 1-844-259-6783 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. Please see accompanying full Prescribing Information for VOYDEYA (danicopan), including Boxed WARNING regarding serious and life-threatening or fatal infections. IMPORTANT SAFETY INFORMATION CONTRAINDICATIONS Known hypersensitivity to benralizumab or excipients. WARNINGS AND PRECAUTIONS Hypersensitivity Reactions Hypersensitivity reactions (e.g., anaphylaxis, angioedema, urticaria, rash) have occurred after administration of FASENRA. These reactions generally occur within hours of administration, but in some instances have a delayed onset (i.e., days). Discontinue in the event of a hypersensitivity reaction. Acute Asthma Symptoms or Deteriorating Disease FASENRA should not be used to treat acute asthma symptoms, acute exacerbations, or acute bronchospasm. Reduction of Corticosteroid Dosage Do not discontinue systemic or inhaled corticosteroids abruptly upon initiation of therapy with FASENRA. Reductions in corticosteroid dose, if appropriate, should be gradual and performed under the direct supervision of a physician. Reduction in corticosteroid dose may be associated with systemic withdrawal symptoms and/or unmask conditions previously suppressed by systemic corticosteroid therapy. Parasitic (Helminth) Infection It is unknown if FASENRA will influence a patient’s response against helminth infections. Treat patients with pre-existing helminth infections before initiating therapy with FASENRA. If patients become infected while receiving FASENRA and do not respond to anti-helminth treatment, discontinue FASENRA until infection resolves. ADVERSE REACTIONS The most common adverse reactions (incidence ≥ 5%) include headache and pharyngitis. Injection site reactions (e.g., pain, erythema, pruritus, papule) occurred at a rate of 2.2% in patients treated with FASENRA compared with 1.9% in patients treated with placebo in asthma exacerbation studies. USE IN SPECIFIC POPULATIONS The data on pregnancy exposure from the clinical trials are insufficient to inform on drug-associated risk. Monoclonal antibodies such as benralizumab are transported across the placenta during the third trimester of pregnancy; therefore, potential effects on a fetus are likely to be greater during the third trimester of pregnancy. INDICATIONS FASENRA is indicated for: the add-on maintenance treatment of patients with severe asthma aged 6 years and older and with an eosinophilic phenotype. FASENRA is not indicated for the relief of acute bronchospasm or status asthmaticus the treatment of adult patients with eosinophilic granulomatosis with polyangiitis (EGPA) Please read Prescribing Information, including Patient Information. CALQUENCE CALQUENCE (acalabrutinib) is a second-generation, selective inhibitor of Bruton’s tyrosine kinase (BTK). CALQUENCE binds covalently to BTK, thereby inhibiting its activity. In B-cells, BTK signaling results in activation of pathways necessary for B-cell proliferation, trafficking, chemotaxis and adhesion. CALQUENCE has been used to treat more than 85,000 patients worldwide and is approved for the treatment of chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL) in the US and Japan, approved for CLL in the EU and many other countries worldwide and approved in China for relapsed or refractory CLL and SLL. CALQUENCE is also approved for the treatment of adult patients with previously untreated MCL in the US, and in China and several other countries for the treatment of adult patients with mantle cell lymphoma (MCL) who have received at least one prior therapy. CALQUENCE is not currently approved for the treatment of MCL in Japan or the EU. As part of an extensive clinical development program, CALQUENCE is currently being evaluated as a single treatment and in combination with standard-of-care chemoimmunotherapy for patients with multiple B-cell blood cancers, including CLL, MCL and diffuse large B-cell lymphoma. ULTOMIRIS® (ravulizumab-cwvz) ULTOMIRIS® (ravulizumab-cwvz), the longest-acting C5 complement inhibitor, provides immediate, complete and sustained complement inhibition. The medication works by inhibiting the C5 protein in the terminal complement cascade, a part of the body’s immune system. When activated in an uncontrolled manner, the complement cascade over-responds, leading the body to attack its own healthy cells. Following a loading dose, ULTOMIRIS is administered intravenously every eight weeks in adults, or every four or eight weeks in pediatric patients (based on body weight). ULTOMIRIS is approved in the US, EU, Japan and other countries for the treatment of certain adults with paroxysmal nocturnal hemoglobinuria (PNH) and is also approved for certain children with PNH in the US, EU and other countries. ULTOMIRIS is also approved in the US, EU, Japan and other countries for the treatment of certain adults and children with atypical hemolytic uremic syndrome (aHUS). Additionally, ULTOMIRIS is approved in the US, EU, Japan, China and other countries for the treatment of certain adults with generalized myasthenia gravis (gMG). Further, ULTOMIRIS is approved in the US, EU, Japan, China and other countries for the treatment of certain adults with neuromyelitis optica spectrum disorder (NMOSD). ULTOMIRIS is being assessed as a treatment for additional indications as part of a broad development program. VOYDEYA™ (danicopan) VOYDEYA™ (danicopan) is a first-in-class oral Factor D inhibitor. The medication works by selectively inhibiting Factor D, a complement system protein that plays a key role in the amplification of the complement system response. When activated in an uncontrolled manner, the complement cascade over-responds, leading the body to attack its own healthy cells. VOYDEYA has been granted Breakthrough Therapy designation by the US Food and Drug Administration and PRIority MEdicines (PRIME) status by the European Medicines Agency. VOYDEYA has also been granted Orphan Drug Designation in the US, EU and Japan for the treatment of PNH. VOYDEYA is approved in the US, EU, Japan and other countries as add-on therapy to ravulizumab or eculizumab for the treatment of certain adults with PNH. FASENRA FASENRA (benralizumab) is currently approved in more than 80 countries, including the US, EU, Japan, and China. FASENRA has been prescribed to over 150,000 patients globally. FASENRA was developed by AstraZeneca and is in-licensed from BioWa, Inc., a wholly owned subsidiary of Kyowa Kirin Co., Ltd., Japan. AstraZeneca in hematology AstraZeneca is pushing the boundaries of science to redefine care in hematology. Our goal is to help transform the lives of patients living with malignant, rare and other related hematologic diseases through innovative medicines and approaches that are shaped by insights from patients, caregivers and physicians. In addition to our marketed products, we are spearheading the development of novel therapies designed to target underlying drivers of disease across multiple scientific platforms. Our acquisitions of Alexion, with expertise in rare, non-malignant blood disorders, and Gracell Biotechnologies Inc., pioneers of autologous cell therapies, expand our hematology pipeline and enable us to reach more patients with high unmet needs through the end-to-end discovery, development and delivery of novel therapies. AstraZeneca in cell therapy AstraZeneca’s ambition is to realize the full potential of cell therapies. It is focused on empowering the immune system to attack cancers, reset the underlying drivers of immune-mediated diseases to return patients to health, and provide transformative solutions with curative potential for people living with rare diseases. To achieve this, the Company is building world-class cell therapy capabilities and advancing a broad pipeline of cell therapies, enabled by technologies including chimeric antigen receptor T-cells (CAR T), T-cell receptor therapies (TCR T) and CAR T regulatory (CAR Tregs) cells. 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. AstraZeneca in respiratory & immunology Respiratory & Immunology, part of AstraZeneca BioPharmaceuticals, is a key disease area and growth driver to the Company. AstraZeneca is an established leader in respiratory care with a 50-year heritage and a growing portfolio of medicines in immune-mediated diseases. The Company is committed to addressing the vast unmet needs of these chronic, often debilitating, diseases with a pipeline and portfolio of inhaled medicines, biologics and new modalities aimed at previously unreachable biologic targets. Our ambition is to deliver life-changing medicines that help eliminate COPD as a leading cause of death, eliminate asthma attacks and achieve clinical remission in immune-mediated diseases. Alexion Alexion, AstraZeneca Rare Disease, is focused on serving patients and families affected by rare diseases and devastating conditions through the discovery, development and delivery of life-changing medicines. A pioneering leader in rare disease for more than three decades, Alexion was the first to translate the complex biology of the complement system into transformative medicines, and today it continues to build a diversified pipeline across disease areas with significant unmet need, using an array of innovative modalities. As part of AstraZeneca, Alexion is continually expanding its global geographic footprint to serve more rare disease patients around the world. It is headquartered in Boston, US. 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’s innovative medicines are sold in more than 125 countries and used by millions of patients worldwide. Please www.astrazeneca-us.com and follow the Company on social media @AstraZeneca. US-107372 Last Updated 12/25

Clinical ResultPhase 3Phase 2Cell TherapyImmunotherapy

04 Dec 2025

·www.astrazeneca.com

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

AstraZeneca advances its ambition to redefine haematology care with new data from its diverse pipeline and portfolio at the 67th American Society of Hematology (ASH) Annual Meeting and Exposition, 6-9 December 2025. This year’s ASH 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,aCD19xCD3 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: Additionalresults 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, 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, 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 Annual Meeting and Exposition Lead Author Abstract Title Presentation Details (ET) Lead Author Awan, F et al. Abstract Title Budget Impact of Fixed Duration Acalabrutinib in Combination with Venetoclax in Previously Untreated Chronic Lymphocytic Leukemia Patients in the United States Presentation Details (ET) 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 Lead Author Cheah, C et al. Abstract Title 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 Presentation Details (ET) 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 Lead Author Ghia, P et al. Abstract Title Impact of prognostic mutations on outcomes with fixed-duration acalabrutinib-venetoclax combinations versus chemoimmunotherapy: An exploratory analysis from AMPLIFY Presentation Details (ET) Abstract #3898 Poster Abstract Session Session 642. Chronic Lymphocytic Leukemia: Clinical and Epidemiological: Poster II 7 December 2025 6:00 PM – 8:00 PM Lead Author Hawkes, E et al. Abstract Title Acalabrutinib plus venetoclax and rituximab in patients with treatment-naive (TN) mantle cell lymphoma (MCL): Results from the phase 2 TrAVeRse study Presentation Details (ET) 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 Lead Author Hou, J-Z et al. Abstract Title 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 Presentation Details (ET) 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 Lead Author Seymour, JF et al. Abstract Title A post hoc safety analysis of fixed-duration acalabrutinib-venetoclax combinations vs chemoimmunotherapy: Results from the Phase 3 AMPLIFY trial Presentation Details (ET) Abstract #2118 Poster Abstract Session Session 642. Chronic Lymphocytic Leukemia: Clinical and Epidemiological: Poster I 6 December 2025 5:30 PM – 7:30 PM Lead Author Wang, ML et al. Abstract Title 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 Presentation Details (ET) 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) Ultomiris (ravulizumab) Schoettler, M et al. Ultomiris (ravulizumab) Outcomes in pediatric patients with HSCT-TMA treated with ravulizumab Ultomiris (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 Ultomiris (ravulizumab) Chaudhury, S et al. Ultomiris (ravulizumab) Organ dysfunction in pediatric patients with HSCT-TMA treated with ravulizumab Ultomiris (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 Ultomiris (ravulizumab) Sherrard, H et al. Ultomiris (ravulizumab) Safety of ravulizumab use in pregnancy: Insights from a global pharmacovigilance analysis Ultomiris (ravulizumab) 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 Ultomiris (ravulizumab) Gandhi, S et al. Ultomiris (ravulizumab) Real-world analysis of ravulizumab safety and effectiveness in advanced age patients with paroxysmal nocturnal hemoglobinuria: Insights from the international PNH registry Ultomiris (ravulizumab) 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) Fasenra (benralizumab) Klion et al. Fasenra (benralizumab) Efficacy and safety of benralizumab in patients with hypereosinophilic syndrome: Results from the Phase 3 natron study Fasenra (benralizumab) 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 Fasenra (benralizumab) Klion et al. Fasenra (benralizumab) Patient perspectives on the burden of hypereosinophilic syndrome: Results from the Phase 3 natron interview sub-study Fasenra (benralizumab) 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) Voydeya (danicopan) Kulasekararaj, A et al. Voydeya (danicopan) 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 Voydeya (danicopan) 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 Surovatamig Aldoss, I et al. Surovatamig 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 Surovatamig 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 Surovatamig Cheah, C et al. Surovatamig 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 Surovatamig 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 Surovatamig Hou, JZ et al. Surovatamig 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) Surovatamig 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 Surovatamig Kim, TM et al. Surovatamig 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 Surovatamig 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 AZD0120 Du, J et al. AZD0120 A dual targeting BCMA and CD19 FasTCAR-T (GC012F/AZD0120) as first-line therapy for newly diagnosed multiple myeloma AZD0120 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 AZD0120 Richard, S et al. AZD0120 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 AZD0120 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 AZD0120 Feng, J et al. AZD0120 One-year follow-up of CD19/BCMA dual-targeting FasTCAR-T GC012F (AZD0120) therapy in patients with refractory systemic lupus erythematosus AZD0120 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 AZD0120 Lentzsch, S et al. AZD0120 ALACRITY: A Phase 1b/2 Study of AZD0120 (BCMA/CD19 CAR-T cell therapy) in Participants with Relapsed or Refractory Light Chain Amyloidosis (AL) AZD0120 Abstract #8236 ePublication 3 November 2025 AZD4512 AZD4512 Han, H et al. AZD4512 AZD4512: A novel CD22-directed antibody-drug conjugate for the treatment of b-cell malignancies AZD4512 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 Notes AstraZeneca in haematology AstraZeneca is pushing the boundaries of science to redefine care in haematology. Our goal is to help transform the lives of patients living with malignant, rare and other related haematologic diseases through innovative medicines and approaches that are shaped by insights from patients, caregivers and physicians. In addition to our marketed products, we are spearheading the development of novel therapies designed to target underlying drivers of disease across multiple scientific platforms. Our acquisitions of Alexion, with expertise in rare, non-malignant blood disorders, and Gracell Biotechnologies Inc., pioneers of autologous cell therapies, expand our haematology pipeline and enable us to reach more patients with high unmet needs through the end-to-end discovery, development and delivery of novel therapies. AstraZeneca in cell therapy AstraZeneca’s ambition is to realise the full potential of cell therapies. It is focused on empowering the immune system to attack cancers, reset the underlying drivers of immune-mediated diseases to return patients to health, and provide transformative solutions with curative potential for people living with rare diseases. To achieve this, the Company is building world-class cell therapy capabilities and advancing a broad pipeline of cell therapies, enabled by technologies including chimeric antigen receptor T-cells (CAR T), T-cell receptor therapies (TCR T) and CAR T regulatory (CAR Tregs) cells. 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 focused on serving patients and families affected by rare diseases and devastating conditions through the discovery, development and delivery of life-changing medicines. A pioneering leader in rare disease for more than three decades, Alexion was the first to translate the complex biology of the complement system into transformative medicines, and today it continues to build a diversified pipeline across disease areas with significant unmet need, using an array of innovative modalities. As part of AstraZeneca, Alexion is continually expanding its global geographic footprint to serve more rare disease patients around the world. It is headquartered in Boston, US. 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’s innovative medicines are sold in more than 125 countries and used by millions of patients worldwide. Please visit astrazeneca.com and follow the Company on Social Media @AstraZeneca. Contacts For details on how to contact the Investor Relations Team, please click here. For Media contacts, click here. Oncology Corporate and financial

Clinical ResultPhase 3Phase 2Cell TherapyImmunotherapy

100 Deals associated with Danicopan

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KEGG	Wiki	ATC	Drug Bank
D11641	-	-	DB15401

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Hemoglobinuria, Paroxysmal	Japan	Alexion Pharmaceuticals, Inc.	18 Jan 2024
Hemoglobinuria, Paroxysmal	Japan	Alexion Pharma GK	18 Jan 2024

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Hemolysis	Phase 3	Canada	Alexion Pharmaceuticals, Inc.	11 Aug 2025
Hemolysis	Phase 3	France	Alexion Pharmaceuticals, Inc.	11 Aug 2025
Hemolysis	Phase 3	United Kingdom	Alexion Pharmaceuticals, Inc.	11 Aug 2025
Age Related Macular Degeneration	Phase 2	United States	Alexion Pharmaceuticals, Inc.	23 Aug 2021
Age Related Macular Degeneration	Phase 2	Japan	Alexion Pharmaceuticals, Inc.	23 Aug 2021
Age Related Macular Degeneration	Phase 2	Australia	Alexion Pharmaceuticals, Inc.	23 Aug 2021
Age Related Macular Degeneration	Phase 2	Czechia	Alexion Pharmaceuticals, Inc.	23 Aug 2021
Age Related Macular Degeneration	Phase 2	France	Alexion Pharmaceuticals, Inc.	23 Aug 2021
Age Related Macular Degeneration	Phase 2	Germany	Alexion Pharmaceuticals, Inc.	23 Aug 2021
Age Related Macular Degeneration	Phase 2	Hungary	Alexion Pharmaceuticals, Inc.	23 Aug 2021

Clinical Result

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


ISTH2026	Not Applicable	Hemoglobinuria, Paroxysmal	240	Proximal complement inhibitors	qhbbywwekd(zjjilmceky) = nfqaxvljyr tadgbyqvjq (jqesheqang ) View more	Positive	13 Jul 2026
				C5 inhibitors	-
EHA2026	Not Applicable	Hemoglobinuria, Paroxysmal	34	DanicopanEculizumab) + Danicopantor (Ravulizumab or Eculizumab)	maeshiscdt(kdhizpvavl) = rdxfbvufth xitcftcedw (glkvpkiauw ) View more	Positive	12 May 2026
EHA2026	Not Applicable	Hemoglobinuria, Paroxysmal	10	EculizumabDanicopanRavulizumabPegcetacoplan + EculizumabDanicopanRavulizumabPegcetacoplan + EculizumabDanicopanRavulizumabPegcetacoplan + EculizumabDanicopanRavulizumabPegcetacoplan	dsfuenoxvg(xyaibpjfin) = During a median follow-up of 49.5 months, 3/10 patients required therapy modification due to persistent anemia compatible with EVH. ewdsdoehfu (ddmvnunkpn )	Positive	12 May 2026
NCT04469465 (ALPHA, EBMT2026)	Phase 3	Hemoglobinuria, Paroxysmal	86	Danicopan (aged ≥65 years)	mfyhlhlzxj(kztpgsnybw) = tbyoumnffg xvouudgpuw (sfhvcubgry, 74.4) View more	Positive	22 Mar 2026
				Danicopan (aged <65 years)	mfyhlhlzxj(kztpgsnybw) = jtbhcctnhi xvouudgpuw (sfhvcubgry, 97.7) View more
ASH2025	Not Applicable	Hemoglobinuria, Paroxysmal	10	Danicopan+C5i	mvdsyfzkli(yacgxufswu) = mild abdominal pain and diarrhea ebqpqstsga (rwaxsjezla )	Positive	06 Dec 2025
ASH2025	Not Applicable	Hemoglobinuria, Paroxysmal	87	eculizumab	zmhpeoffff(gusklfalkn) = ikzjjlypkf bujlpholwe (jtfcabfnav ) View more	Positive	06 Dec 2025
				ravulizumab	zmhpeoffff(gusklfalkn) = rbfbinsmln bujlpholwe (jtfcabfnav ) View more
NCT04469465 (ALPHA, EHA2025)	Phase 3	Hemolysis \| Hemoglobinuria, Paroxysmal \| Bone Marrow Failure Disorders Add-on	86	Danicopan	evouoozbzt(jourrccszc) = boozbmkayr qweobohcwt (ddbcwmyllf, 55.3) View more	Positive	14 May 2025
NCT02946463 \| NCT03056040 \| NCT04469465 (ALPHA, EHA2025)	Phase 3	Hemoglobinuria, Paroxysmal Add-on	-	Ravulizumab	xnlewpoapg(wwuplngbac) = dqbccfbcdw twgxplcddp (ongwgsjfwo, per 10 person - years) View more	Positive	14 May 2025
				Danicopan	xqhvssltsp(ipjdahmjcj) = tgjfxsdvjs tgzvjzmzqv (yqbzmzexae )
NCT04469465 (ALPHA, EBMT2025)	Phase 3	Hemoglobinuria, Paroxysmal	86	Danicopan add-on to ravulizumab or eculizumab	ciehuburen(tnzyfjmajk) = EORTC fatigue subscale scores in the danicopan arm improved from 43.3 (27.2) at baseline to scores comparable with the general population norm of 29.5; in the placebo-danicopan arm scores showed improvement from baseline. zlniohdkfo (blrfultufz ) View more	Positive	30 Mar 2025
				Placebo add-on to ravulizumab or eculizumab
NCT04469465 (ALPHA, EHA 12024 \| EHA 22024) Manual	Phase 3	Hemoglobinuria, Paroxysmal	86	Danicopan + Ravulizumab/Eculizumab	aphcaznqen(aprbohhpll) = The proportion of pts with increased Hb (≥2 g/dL) without transfusion was maintained in the Dan–Dan arm and improved in the Pbo–Dan arm from wk 12 to 24 (Dan–Dan, 54.4% to 41.8%; Pbo–Dan, 0% to 33.3%). At wks 24 and 48, mean Hb levels and ARC were maintained in the Dan–Dan arm andimproved from wk 12 in the Pbo–Dan arm rtoydirtdo (kpeurzaohz ) View more	Positive	14 May 2024

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