This is a Phase II, open-label study designed to evaluate the safety, efficacy, and immunogenicity of NM8074 in PNH patients undergoing complement-inhibitor therapy with Soliris.

Start Date01 Mar 2025

Sponsor / Collaborator

NovelMed Therapeutics, Inc.

NCT06673394 / Not yet recruitingPhase 2IIT

Eculizumab for Acute Attack of Neuromyelitis Optica Spectrum Disorder (NMOSD): a Multi-Center, Phase 2, Open Label Trial (EASE-NMO)

Neuromyelitis optica spectrum disorder (NMOSD) is a relapsing, inflammatory autoimmune disorder of the central nervous system characterized by the pathogenic anti-aquaporin 4 antibody (AQP4-IgG). The objectives of this study are to assess the efficacy and safety of eculizumab for treatment of patients with neuromyelitis optica spectrum disorders during acute phase who are anti-aquaporin-4 (AQP4) antibody-positive. Eculizumab, a humanized monoclonal antibody, inhibits the terminal complement protein C5 and prevents its cleavage into C5a and the formation of C5b-9 (MAC), has approved for preventive treatment of NMOSD. Given the high efficacy of C5 inhibition, eculizumab is proposed to potentially provide rapid relief from astrocyte destruction by reducing MAC formation, which could contribute to the fast alleviation of neurological deficit during NMO acute attack. The potential of eculizumab warrants further investigation as a treatment for acute neuromyelitis optica spectrum disorders attacks.

Start Date01 Dec 2024

Sponsor / Collaborator

Tianjin Medical University General Hospital

NCT06593938 / Not yet recruitingPhase 3

A Multicenter, Randomized, Open-label, Active-comparator Controlled Phase III Study to Evaluate the Efficacy and Safety of HRS-5965 Capsule in Patients With Paroxysmal Nocturnal Hemoglobinuria

A study of the efficacy and safety of HRS-5965 capsules compared to eculizumab for 24 weeks in patients with PNH.

Start Date30 Sep 2024

Sponsor / Collaborator

Chengdu Suncadia Pharmaceuticals Co., Ltd.

100 Clinical Results associated with Eculizumab

100 Translational Medicine associated with Eculizumab

100 Patents (Medical) associated with Eculizumab

2,508

Literatures (Medical) associated with Eculizumab

01 Dec 2024·PEDIATRIC NEPHROLOGY

Kidney transplantation in children and adolescents with C3 glomerulopathy or immune complex membranoproliferative glomerulonephritis: a real-world study within the CERTAIN research network

Article

Author: Fichtner, Alexander ; Shenoy, Mohan ; Meier, Matthias ; Webb, Nicholas J A ; Becker, Jan ; Ozkaya, Ozan ; Büscher, Anja ; Bilge, Ilmay ; Patry, Christian ; Tönshoff, Burkhard ; Krupka, Kai ; Antoniello, Benedetta ; Gulhan, Bora ; Feißt, Manuel ; Hogan, Julien ; Höcker, Britta ; Weber, Lutz T ; Bald, Martin ; Sellier-Leclerc, Anne-Laure ; Kanzelmeyer, Nele

Abstract:

Background:

Complement 3 glomerulopathy (C3G) and immune complex membranoproliferative glomerulonephritis (IC-MPGN) are ultra-rare chronic kidney diseases with an overall poor prognosis, with approximately 40–50% of patients progressing to kidney failure within 10 years of diagnosis. C3G is characterized by a high rate of disease recurrence in the transplanted kidney. However, there is a lack of published data on clinical outcomes in the pediatric population following transplantation.

Methods:

In this multicenter longitudinal cohort study of the Cooperative European Paediatric Renal Transplant Initiative (CERTAIN) registry, we compared the post-transplant outcomes of pediatric patients with C3G (n = 17) or IC-MPGN (n = 3) with a matched case–control group (n = 20).

Results:

Eleven of 20 children (55%) with C3G or IC-MPGN experienced a recurrence within 5 years post-transplant. Patients with C3G or IC-MPGN had a 5-year graft survival of 61.4%, which was significantly (P = 0.029) lower than the 5-year graft survival of 90% in controls; five patients with C3G or IC-MPGN lost their graft due to recurrence during this observation period. Both the 1-year (20%) and the 5-year (42%) rates of biopsy-proven acute rejection episodes were comparable between patients and controls. Complement-targeted therapy with eculizumab, either as prophylaxis or treatment, did not appear to be effective.

Conclusions:

These data in pediatric patients with C3G or IC-MPGN show a high risk of post-transplant disease recurrence (55%) and a significantly lower 5-year graft survival compared to matched controls with other primary kidney diseases. These data underscore the need for post-transplant patients for effective and specific therapies that target the underlying disease mechanism.

Graphical abstract:

01 Dec 2024·NEUROLOGICAL SCIENCES

Therapeutic challenges and unmet needs in the management of myasthenia gravis: an Italian expert opinion

Review

Author: Liguori, Rocco ; Habetswallner, Francesco ; Rodolico, Carmelo ; Mantegazza, Renato ; Pegoraro, Elena ; Evoli, Amelia ; Sgarzi, Manlio ; Saccà, Francesco ; Bonifati, Domenico Marco ; Pappagallo, Giovanni ; Schenone, Angelo ; Antonini, Giovanni

Myasthenia gravis (MG) is a rare, autoimmune, neurological disorder. Most MG patients have autoantibodies against acetylcholine receptors (AChRs). Some have autoantibodies against muscle-specific tyrosine kinase (MuSK) or lipoprotein-receptor-related protein 4 (LRP4), and some are seronegative. Standard of care, which includes anti-cholinesterase drugs, thymectomy, corticosteroids (CS), and off-label use of non-steroidal immunosuppressive drugs (NSISTs), is bounded by potential side effects and limited efficacy in refractory generalized MG (gMG) patients. This highlights the need for new therapeutic approaches for MG. Eculizumab, a monoclonal antibody that inhibits the complement system, has been recently approved in Italy for refractory gMG. A panel of 11 experts met to discuss unmet therapeutic needs in the acute and chronic phases of the disease, as well as the standard of care for refractory patients. Survival was emphasized as an acute phase outcome. In the chronic phase, persistent remission and early recognition of exacerbations to prevent myasthenic crisis and respiratory failure were considered crucial. Refractory patients require treatments with fast onset of action, improved tolerability, and the ability to slow disease progression and increase life expectancy. The Panel agreed that eculizumab would presumably meet the therapeutic needs of many refractory gMG patients. The panel concluded that the unmet needs of current standard of care treatments for gMG are significant. Evaluating new therapeutic options accurately is essential to find the best balance between efficacy and tolerability for each patient. Collecting real-world data on novel molecules in routine clinical practice is necessary to address unmet needs.

01 Dec 2024·PEDIATRIC NEPHROLOGY

Protein-losing enteropathy as a new phenotype in atypical hemolytic uremic syndrome caused by CD46 gene mutation

Review

Author: Liu, Jiaojiao ; Zhai, Yihui ; Tang, Xiaoshan ; Fang, Xiaoyan ; Sun, Manqing ; Chen, Jing ; Shen, Qian ; Miao, Qianfan ; Han, Xinli ; Zhang, Zhiqing ; Wang, Chunyan ; Xu, Hong

BACKGROUND:

Atypical hemolytic uremic syndrome (aHUS) is a life-threatening thrombotic microangiopathy. Genetic defects in the alternative complement (AP) pathway have been identified in 60-70% of individuals. Eculizumab is recommended as a first-line therapy.

METHODS:

We collected the clinical data of a pediatric patient with aHUS accompanied by protein-losing enteropathy (PLE). Genetic testing was performed. Related literature on aHUS combined with PLE was reviewed.

RESULTS:

A 15-year-old Chinese girl was diagnosed with aHUS at 3.7 years of age and experienced five episodes; her symptoms completely resolved with plasma treatment. Severe gastrointestinal symptoms and hypoalbuminemia presented after the first episode, and PLE was diagnosed. A novel homozygous CD46 variant was identified, and FACS revealed significantly decreased CD46 expression. She presented at a recent relapse with persistent GI symptoms and headache and progressed to chronic kidney failure; peritoneal dialysis was initiated. Eculizumab was given 8 months after the last recurrence. Surprisingly, PLE was cured. Afterward, dialysis was discontinued, and eGFR recovered to 44.8 ml/min/1.73 m². A review of the literature indicated that PLE with thrombosis was caused by CD55 variants via hyperactivation of the AP system. We report an aHUS patient with PLE caused by CD46 variants. Symptoms of both PLE and aHUS were significantly alleviated in our patient and patients with CD55 variants treated with eculizumab, indicating that PLE was a new symptom of aHUS in our patient with a CD46 variant.

CONCLUSIONS:

Our case expands the phenotype of aHUS caused by a CD46 mutation and provides evidence of the efficacy of eculizumab after a long phase of chronic kidney failure.

192

News (Medical) associated with Eculizumab

12 Nov 2024

·medcitynews.com

Neurogene Gene Therapy Shows Signs of Efficacy in Small Study, But an Adverse Event Spooks Investors - MedCity News

A Neurogene gene therapy in development for the rare neurodevelopmental disorder Rett syndrome has encouraging early data in a small group of patients, an interim readout that comes a week after a private placement infused the biotech with $200 million to last it the next two years. But investors are focusing on a late-breaking development — a complication experienced by a patient who received the high dose of the experimental one-time treatment. The data reported after Monday’s market close are from seven total pediatric Rett syndrome patients treated with the Neurogene therapy, NGN-401. As of an Oct. 17 cutoff date, five patients had received the low dose and two received the high dose. For these patients, Neurogene said the therapy was safe and well tolerated. No treatment-related adverse events were reported. The adverse event happened in a third patient who received the high dose of NGN-401 after the data cutoff. Neurogene said it learned of the complication on Monday. The company said this complication was consistent with known risks of gene therapies delivered by adeno-associated viruses (AAV). NGN-401 uses the AAV9 viral vector for delivery. High doses of AAV are associated with inflammation and excessive activity of the complement system, a part of the immune system. The complication could keep Neurogene from pursuing development of the high dose of its gene therapy. The New York-based company’s stock price opened Tuesday at $48.28, down more than 32% from Monday’s closing price. Consumer / Employer Health Executives on Digital Transformation in Healthcare Hear executives from Quantum Health, Surescripts, EY, Clinical Architecture and Personify Health share their views on digital transformation in healthcare. By MedCity News In Rett syndrome, a mutation in one copy of the MECP2 gene leads to deficiency of a protein that’s important for normal brain function. The disease, which mainly affects girls, leads to delay in an infant’s early development. As the child gets older, the disease leads to a loss of the motor and communication skills that were attained. The lone FDA-approved Rett treatment is Acadia Pharmaceutical’s Daybue, a twice daily oral drug that reduces symptoms from the inherited disorder. The Neurogene therapy delivers to cells a full-length version of the MECP2 gene that is intended to replace the expression of the key protein, but not so much that the therapy causes complications. Neurogene accomplishes this with its EXACT transgene regulation technology, which acts as a “genetic thermostat” to control gene expression and void variability in expression that can lead to toxicity. In addition to the safety data reported for NGN-401, the early results also show some signs of efficacy in four of the patients in the low-dose group. According to measures taken with widely used psychiatric and Rett assessments, Neurogene reported a clinically meaningful gain of skills and developmental milestones compared to the natural history of the disease. William Blair analyst Sami Corwin, who spoke with Neurogene management, said the adverse event reported by the company appears to be a systemic inflammatory response that is a known side effect of high-dose AAV gene therapies. In a note sent to investors Tuesday, she said this complication can managed or resolved with corticosteroids or Soliris, an antibody drug designed to block a particular complement system protein. “Given the event does not appear to be CNS-localized, we do not think it is related to the [intracerebral ventricular] injection procedure or EXACT technology,” Corwin said. “Importantly, we do not see the event as impacting the continued development of the low dose.” Exclusive Heard at HLTH 2024: Insights from Innovative Healthcare Executives Executives from Imagine360, Verily, BrightInsight, Lantern, and Rhapsody shared their approaches to reducing healthcare costs and facilitating digital transformation. By MedCity News Leering Partners analyst Mani Foroohar said in a research note that the efficacy data were impressive enough even at the low dose to offer consistent differentiation compared to Rett natural history. Regardless of the adverse event, he expects the program will move into a pivotal study at least in the low dose. The low-dose cohort has a targeted enrollment of eight patients. In an investor presentation, Neurogene said it expects to complete enrollment in this group by the end of the year. In the first half of next year, the company will provide an update on the pivotal trial’s design. Additional Phase 1/2 data will be reported in the second half of 2025. Neurogene has also started enrolling an adolescent and adult group of patients, which it said could support expanding use of the drug to a broader swath of the Rett patient population. Neurogene’s competition in Rett includes Taysha Gene Therapies, which is in Phase 1/2 testing with TSHA-102. This gene therapy is made with a Taysha technology that mediates MECP2 expression. The company expects to report data from the first of two cohorts in the first half of 2025. Separately, Neurogene said it does not expect to proceed with its gene therapy for Batten disease. Given the rarity of this disease of the central nervous system, the company said it needs a streamlined regulatory pathway. Neurogene applied for a Regenerative Medicine Advanced Therapy (RMAT) designation for this program. Companies that receive this designation for a therapeutic candidate benefit from earlier and more frequent interactions with the agency and the possibility of an accelerated approval. The FDA turned down Neurogene’s RMAT application for its Batten program. Public domain image by Flickr user SciTechTrend

Gene TherapyDrug ApprovalClinical Result

05 Nov 2024

·www.businesswire.com

Genentech to Present New and Encouraging Long-term Follow-up Data Across Broad Hematology Portfolio at ASH 2024

SOUTH SAN FRANCISCO, Calif.--( BUSINESS WIRE )--Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY) announced today that it will present more than 40 abstracts across nine blood disorders at the 66th American Society of Hematology (ASH) Annual Meeting and Exposition, held December 7-10, 2024 in San Diego, California. The data underscore Genentech’s commitment to advance patient outcomes in lymphoma with long-term follow-up of its approved medicines Polivy ® (polatuzumab vedotin-piiq), Lunsumio ® (mosunetuzumab-axgb) and Columvi ® (glofitamab-gxbm) as well as new investigational combination data. Key presentations include: Five-year data from the Phase III POLARIX study (abstract #469) reinforce the potential of Polivy in combination with Rituxan ® (rituximab), cyclophosphamide, doxorubicin and prednisone (R-CHP) to provide durable and lasting remissions and for the first time show a positive trend in overall survival (OS) for people with first-line diffuse large B-cell lymphoma (DLBCL), an area that previously had little advancement in nearly two decades. Extended follow-up data of up to four years from the pivotal GO29781 study of Lunsumio (abstract #4407) and NP30179 study of Columvi (abstract #865) show long-lasting remissions and immune system recovery after the end of treatment, supporting the use of fixed-duration bispecific antibodies for third-line or later (3L+) follicular lymphoma (FL) and DLBCL, respectively. First presentation of data for a subcutaneous formulation of Lunsumio monotherapy from the pivotal Phase II GO29781 study (abstract #1645) show high rates of deep and durable responses and low rates and severity of cytokine release syndrome in people with 3L+ FL. Subcutaneously administered Lunsumio could further improve the patient experience by combining shorter administration time with the existing benefits of a fixed-duration and outpatient therapy. New patient-reported outcomes data from the Phase III STARGLO study (abstract #5132) indicate comparable health-related quality of life between treatment arms, despite higher median number of cycles received with the Columvi combination (11 versus 4). Together with the significant improvement in OS observed in the study, these data support the potential benefit for patients with second-line or later DLBCL. New and updated data from investigational study combinations of Polivy with bispecific antibodies Lunsumio and Columvi in relapsed or refractory DLBCL, including the Phase Ib/II NP39488 (abstract #988) and Phase II GO40516 (abstract #989) studies, add to the growing body of evidence demonstrating the potential of novel bispecific antibody/Polivy combinations in earlier treatment lines, and support their ongoing Phase III development. Overview of key presentations featuring Genentech medicines Medicine Abstract title Abstract number/presentation details Polivy Five-Year Analysis of the POLARIX Study: Prolonged Follow-up Confirms Positive Impact of Polatuzumab Vedotin Plus Rituximab, Cyclophosphamide, Doxorubicin, and Prednisone (Pola-R-CHP) on Outcomes #469 oral presentation Session: 626. Aggressive Lymphomas: Clinical and Epidemiological: CARs, Bispecifics, and ADCs: Progress and Challenges in Aggressive B Cell Lymphoma Sunday, December 8, 2024 9:30 AM PST A Multicenter, Prospective, Observational Study of Pola-R-CHP in Patients With Previously Untreated Diffuse Large B-Cell Lymphoma (POLASTAR): A Preliminary Analysis #4475 poster presentation Session: 626. Aggressive Lymphomas: Clinical and Epidemiological: Poster III Monday, December 9, 2024 6:00 - 8:00 PM PST Lunsumio A Randomized Phase II Study of Mosunetuzumab SC Plus Polatuzumab Vedotin Demonstrates Improved Outcomes Versus Rituximab Plus Polatuzumab Vedotin in Patients (Pts) with Relapsed or Refractory (R/R) Large B-Cell Lymphoma (LBCL) #989 oral presentation Session: 627. Aggressive Lymphomas: Pharmacologic Therapies: Chemotherapy-free Combinations for Relapsed Aggressive Lymphomas Monday, December 9, 2024 5:30 PM PST Subcutaneous Mosunetuzumab Leads to High Rates of Durable Responses, Low Rates of Cytokine Release Syndrome, and Non-Inferior Exposure Compared with Intravenous Administration in Patients with Relapsed/Refractory Follicular Lymphoma: Primary Analysis of a Pivotal Phase II Study #1645 poster presentation Session: 623. Mantle Cell, Follicular, Waldenstrom’s, and Other Indolent B Cell Lymphomas: Clinical and Epidemiological: Poster I Saturday, December 7, 2024 5:30 - 7:30 PM PST Mosunetuzumab Continues to Demonstrate Clinically Meaningful Outcomes in Patients with Relapsed and/or Refractory Follicular Lymphoma after ≥2 Prior Therapies Including Those with a History of POD24: 4-Year Follow-up of a Pivotal Phase II Study #4407 poster presentation Session: 623. Mantle Cell, Follicular, Waldenstrom’s, and Other Indolent B Cell Lymphomas: Clinical and Epidemiological: Poster III Monday, December 9, 2024 6:00 - 8:00 PM PST Mosunetuzumab Monotherapy Demonstrates Encouraging Activity and a Manageable Safety Profile in Patients with Heavily Pre-Treated Relapsed or Refractory Mantle Cell Lymphoma #1646 poster presentation Session: 623. Mantle Cell, Follicular, Waldenstrom’s, and Other Indolent B Cell Lymphomas: Clinical and Epidemiological: Poster I Saturday, December 7, 2024 5:30 - 7:30 PM PST Fixed-Duration Subcutaneous Mosunetuzumab is Active and has a Manageable Safety Profile in Patients with Previously Untreated, Low-Tumor Burden Follicular Lymphoma: Updated Results from the Phase II MorningSun Study #3008 poster presentation Session: 623. Mantle Cell, Follicular, Waldenstrom’s, and Other Indolent B Cell Lymphomas: Clinical and Epidemiological: Poster II Sunday, December 8, 2024 6:00 - 8:00 PM PST Travel Burden and Travel Costs of Bispecific Antibodies in Patients with Relapsed/Refractory Diffuse Large B-Cell Lymphoma and Relapsed/Refractory Follicular Lymphoma #782 oral presentation Session: 902. Health Services and Quality Improvement: Lymphoid Malignancies: For a Better Tomorrow - Improving Access to Blood Cancer Treatments and Trials Monday, December 9, 2024 10:45 AM PST Columvi Fixed-duration Glofitamab Monotherapy Continues to Demonstrate Durable Responses in Patients with Relapsed or Refractory Large B-Cell Lymphoma: 3-year Follow-Up From a Pivotal Phase II Study #865 oral presentation Session: 627. Aggressive Lymphomas: Pharmacologic Therapies: New R-CHOP Combinations for Treatment Naïve DLBCL Sunday, December 8, 2024 Midday PST Glofitamab in Combination with Polatuzumab Vedotin Maintains Durable Responses and a Manageable Safety Profile in Patients with Heavily Pre-treated Relapsed/Refractory (R/R) Large B-Cell Lymphoma (LBCL) Including High-Grade B-Cell Lymphoma (HGBCL): Extended Follow-Up of a Phase Ib/II Study #988 oral presentation Session: 627. Aggressive Lymphomas: Pharmacologic Therapies: Chemotherapy-free Combinations for Relapsed Aggressive Lymphomas Monday, December 9, 2024 5:15 PM PST Glofitamab in Combination with Rituximab plus Ifosfamide, Carboplatin, and Etoposide shows Favorable Efficacy and Manageable Safety in Patients with Relapsed or Refractory Diffuse Large B-cell Lymphoma, Eligible for Stem Cell Transplant or Chimeric Antigen Receptor T-cell Therapy: Results from a Phase Ib Study #987 oral presentation Session: 627. Aggressive Lymphomas: Pharmacologic Therapies: Chemotherapy-free Combinations for Relapsed Aggressive Lymphomas Monday, December 9, 2024 5:00 PM PST Primary Results of Patient-Reported Outcomes in Patients with Relapsed/Refractory Diffuse Large B-cell Lymphoma Treated with Glofitamab plus Gemcitabine and Oxaliplatin (Glofit-GemOx) Versus Rituximab plus GemOx (R-GemOx) from the Phase III STARGLO Study #5132 poster presentation Session: 906. Outcomes Research: Lymphoid Malignancies Excluding Plasma Cell Disorders: Poster III Monday, December 9, 2024 6:00 - 8:00 PM PST Glofitamab Induces High Response Rates and Durable Remissions in Patients (Pts) with Heavily Pretreated Relapsed/Refractory (R/R) Mantle Cell Lymphoma (MCL), including those with a Poor Prognosis: Subgroup Results from a Phase I/II Study #1631 poster presentation Session: 623. Mantle Cell, Follicular, Waldenstrom’s, and Other Indolent B Cell Lymphomas: Clinical and Epidemiological: Poster I Saturday, December 7, 2024 5:30 - 7:30 PM PST A Healthcare Utilization Model Comparing Time Toxicity Between Glofitamab and Epcoritamab Treatment Regimens #3647 poster presentation Session: 902. Health Services and Quality Improvement: Lymphoid Malignancies: Poster II Sunday, December 8, 2024 6:00 - 8:00 PM PST Englumafusp alfa Englumafusp Alfa (CD19-4-1BBL) Combined with Glofitamab is Safe and Efficacious in Patients with R/R B-NHL: Extended Follow Up Analysis of the Dose-Escalation Part of Phase I Trial BP41072 #990 oral presentation Session: 627. Aggressive Lymphomas: Pharmacologic Therapies: Chemotherapy-free Combinations for Relapsed Aggressive Lymphomas Monday, December 9, 2024 5:45 PM PST Cevostamab Cevostamab in Patients with Heavily Pretreated Relapsed/Refractory Multiple Myeloma (RRMM): Updated Results from an Ongoing Phase I Study Demonstrate Clinically Meaningful Activity and Manageable Safety and Inform the Doses and Regimen for Combination Studies #1021 oral presentation Session: 654. Multiple Myeloma: Pharmacologic Therapies: Into the Future: New Drugs and Combinations in Multiple Myeloma Monday, December 9, 2024 4:30 PM PST Venclexta ® (venetoclax)* CRISTALLO: Results from a Phase III Trial of Venetoclax–Obinutuzumab versus Fludarabine, Cyclophosphamide and Rituximab or Bendamustine–Rituximab in Patients with Untreated Chronic Lymphocytic Leukemia Without Del(17p) or TP53 Mutations #3237 poster presentation Session: 642. Chronic Lymphocytic Leukemia: Clinical and Epidemiological: Poster II Sunday, December 8, 2024 6:00 - 8:00 PM PST P-CD19CD20-ALLO1 (in collaboration with Poseida Therapeutics) P-CD19CD20-ALLO1: Potent Fully Allogeneic CAR-T Therapy Targeting CD19 and CD20 with Superior Efficacy Over Single-Target Products #4805 poster presentation Session: 702. CAR-T Cell Therapies: Basic and Translational: Poster III Monday, December 9, 2024 6:00 - 8:00 PM PST P-BCMA-ALLO1 (in collaboration with Poseida Therapeutics) A Phase 1 Study of P-BCMA-ALLO1, a Non-viral, Allogeneic BCMA Directed CAR-T in Relapsed/Refractory Multiple Myeloma (RRMM): Results from Optimized Lymphodepletion Cohort #4828 poster presentation Session: 704. Cellular Immunotherapies: Early Phase Clinical Trials and Toxicities: Poster III Monday, December 9, 2024 6:00 - 8:00 PM PST Hemlibra ® (emicizumab) Bleed Patterns in Infants, From Birth to 12 Months of Age, with Hemophilia A Treated with Emicizumab: Exploratory Analysis of the HAVEN 7 Study #1214 poster presentation Session: 322. Hemophilia A and B: Clinical and Epidemiological: Poster I Saturday, December 7, 2024 5:30 - 7:30 PM PST Real-World Experience With Emicizumab for Hemophilia A From the Physician Perspective Based on Survey Data #5078 poster presentation Session: 905. Outcomes Research: Non-Malignant Conditions Excluding Hemoglobinopathies: Poster III Monday, December 9, 2024 6:00 - 8:00 PM PST PiaSky ® (crovalimab) Phase III Randomized COMMODORE 2 Trial: 2-Year Efficacy and Safety of Crovalimab in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Naive to Complement Inhibition #2687 poster presentation Session: 508. Bone Marrow Failure: Acquired: Poster II Sunday, December 8, 2024 6:00 - 8:00 PM PST Phase III COMMODORE 1 Trial: 2-Year Efficacy and Safety of Crovalimab in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Switched from Ravulizumab #4078 poster presentation Session: 508. Bone Marrow Failure: Acquired: Poster III Monday, December 9, 2024 6:00 - 8:00 PM PST *Venclexta is being developed by AbbVie and Genentech, a member of the Roche Group. It is jointly commercialized by the companies in the United States and commercialized by AbbVie outside of the United States. About Polivy ® (polatuzumab vedotin-piiq) Polivy is a first-in-class anti-CD79b antibody-drug conjugate (ADC). The CD79b protein is expressed specifically in the majority of B cells, an immune cell impacted in some types of non-Hodgkin’s lymphoma (NHL), making it a promising target for the development of new therapies. Polivy binds to cancer cells such as CD79b and destroys these B cells through the delivery of an anti-cancer agent, which is thought to minimize the effects on normal cells. Polivy is being developed by Genentech using Seagen ADC technology and is currently being investigated for the treatment of several types of NHL. Polivy U.S. Indication Polivy is a prescription medicine used with other medicines (a rituximab product, cyclophosphamide, doxorubicin, and prednisone) as a first treatment for adults who have moderate to high risk diffuse large B-cell lymphoma (DLBCL), not otherwise specified (NOS) or high-grade B-cell lymphoma (HGBL). Polivy is a prescription medicine used with other medicines, bendamustine and a rituximab product, to treat DLBCL in adults who have progressed after at least 2 prior therapies. Important Safety Information Possible serious side effects Everyone reacts differently to Polivy therapy, so it’s important to know what the side effects are. Some people who have been treated with Polivy have experienced serious to fatal side effects. Your doctor may stop or adjust your treatment if any serious side effects occur. Be sure to contact your healthcare team if there are any signs of these side effects. Nerve problems in your arms and legs: This may happen as early as after your first dose and may worsen with every dose. Your doctor will monitor for signs and symptoms, such as changes in your sense of touch, numbness or tingling in your hands or feet, nerve pain, burning sensation, any muscle weakness, or changes to your walking pattern Infusion-related reactions: You may experience fever, chills, rash, breathing problems, low blood pressure, or hives within 24 hours of your infusion Low blood cell counts: Treatment with Polivy can cause severe low blood cell counts. Your doctor will monitor your blood counts throughout treatment with Polivy Infections: If you have a fever of 100.4°F (38°C) or higher, chills, cough, or pain during urination, contact your healthcare team. Your doctor may also give you medication before giving you Polivy, which may prevent some infections Rare and serious brain infections: Your doctor will monitor closely for signs and symptoms of these types of infections. Contact your doctor if you experience confusion, dizziness or loss of balance, trouble talking or walking, or vision changes Tumor lysis syndrome: Caused by the fast breakdown of cancer cells. Signs include nausea, vomiting, diarrhea, and lack of energy Potential harm to liver: Some signs include tiredness, weight loss, pain in the abdomen, dark urine, and yellowing of your skin or the white part of your eyes. You may be at higher risk if you already had liver problems or you are taking other medication Side effects seen most often The most common side effects during treatment were Nerve problems in arms and legs Nausea Tiredness or lack of energy Diarrhea Constipation Hair loss Redness and sores of the lining of the mouth, lips, throat, and digestive tract Polivy may lower your red or white blood cell counts and increase uric acid levels. Polivy may not be for everyone. Talk to your doctor if you are Pregnant or think you are pregnant: Data have shown that Polivy may harm your unborn baby Planning to become pregnant: Women should avoid getting pregnant while taking Polivy. Women should use effective contraception during treatment and for 3 months after their last Polivy treatment. Men taking Polivy should use effective contraception during treatment and for 5 months after their last Polivy treatment Breastfeeding: Women should not breastfeed while taking Polivy and for 2 months after the last dose These may not be all the side effects. Talk to your healthcare provider for more information about the benefits and risks of Polivy treatment. You may report side effects to the FDA at (800) FDA-1088 or http://www.fda.gov/medwatch . You may also report side effects to Genentech at (888) 835-2555. Please see the full Prescribing Information and visit https://www.Polivy.com for additional Important Safety Information. About Lunsumio ® (mosunetuzumab-axgb) Lunsumio is a first-in-class CD20xCD3 T-cell engaging bispecific antibody designed to target CD20 on the surface of B cells and CD3 on the surface of T cells. This dual targeting activates and redirects a patient’s existing T cells to engage and eliminate target B cells by releasing cytotoxic proteins into the B cells. A robust clinical development program for Lunsumio is ongoing, investigating the molecule as a monotherapy and in combination with other medicines, for the treatment of people with B-cell non-Hodgkin’s lymphomas, including follicular lymphoma and diffuse large B-cell lymphoma, and other blood cancers. Lunsumio U.S. Indication Lunsumio (mosunetuzumab-axgb) is a prescription medicine used to treat adults with follicular lymphoma whose cancer has come back or did not respond to previous treatment, and who have already received two or more treatments for their cancer. It is not known if Lunsumio is safe and effective in children. The conditional approval of Lunsumio is based on response rate. There are ongoing studies to establish how well the drug works. What is the most important information I should know about Lunsumio? Lunsumio may cause Cytokine Release Syndrome (CRS), a serious side effect that is common during treatment with Lunsumio and can also be severe or life-threatening. Get medical help right away if you develop any signs or symptoms of CRS at any time, including: fever of 100.4°F (38°C) or higher chills low blood pressure fast or irregular heartbeat tiredness or weakness difficulty breathing headache confusion feeling anxious dizziness or light-headedness nausea vomiting Due to the risk of CRS, you will receive Lunsumio on a “step-up dosing schedule.” The step-up dosing schedule is when you receive smaller “step-up” doses of Lunsumio on Day 1 and Day 8 of your first cycle of treatment You will receive a higher dose of Lunsumio on Day 15 of your first cycle of treatment If your dose of Lunsumio is delayed for any reason, you may need to repeat the step-up dosing schedule Before each dose in Cycle 1 and Cycle 2, you will receive medicines to help reduce your risk of CRS Your healthcare provider will check you for CRS during treatment with Lunsumio and may treat you in a hospital if you develop signs and symptoms of CRS. Your healthcare provider may temporarily stop or completely stop your treatment with Lunsumio, if you have severe side effects. What are the possible side effects of Lunsumio? Lunsumio may cause serious side effects, including: Neurologic problems. Your healthcare provider will check you for neurologic problems during treatment with Lunsumio. Your healthcare provider may also refer you to a healthcare provider who specializes in neurologic problems. Tell your healthcare provider right away if you develop any signs or symptoms of neurologic problems during or after treatment with Lunsumio, including: headache numbness and tingling of the arms, legs, hands, or feet dizziness confusion and disorientation difficulty paying attention or understanding things forgetting things or forgetting who or where you are trouble speaking, reading, or writing sleepiness or trouble sleeping tremors loss of consciousness seizures muscle problems or muscle weakness loss of balance or trouble walking Serious infections. Lunsumio can cause serious infections that may lead to death. Your healthcare provider will check you for signs and symptoms of infection before and during treatment. Tell your healthcare provider right away if you develop any signs or symptoms of infection during treatment with Lunsumio, including: fever of 100.4° F (38° C) or higher chest pain tiredness shortness of breath painful rash sore throat pain during urination feeling weak or generally unwell Low blood cell counts. Low blood cell counts are common during treatment with Lunsumio and can also be severe. Your healthcare provider will check your blood cell counts during treatment with Lunsumio. Lunsumio may cause the following low blood cell counts: low white blood cell counts (neutropenia). Low white blood cells can increase your risk for infection low red blood cell counts (anemia). Low red blood cells can cause tiredness and shortness of breath low platelet counts (thrombocytopenia). Low platelet counts can cause bruising or bleeding problems Growth in your tumor or worsening of tumor related problems (Tumor flare). Lunsumio may cause serious or severe worsening of your tumor. Tell your healthcare provider if you develop any of these signs or symptoms of tumor flare during your treatment with Lunsumio: tender or swollen lymph nodes, chest pain, cough, trouble breathing, and pain or swelling at the site of the tumor Your healthcare provider may temporarily stop or permanently stop treatment with Lunsumio if you develop severe side effects. The most common side effects of Lunsumio include: tiredness, rash, fever, and headache. The most common severe abnormal lab test results with Lunsumio include: decreased phosphate, increased glucose, and increased uric acid levels. Before receiving Lunsumio, tell your healthcare provider about all of your medical conditions, including if you: have ever had an infusion reaction after receiving Lunsumio have an infection, or have had an infection in the past which lasted a long time or keeps coming back have or have had Epstein-Barr Virus are pregnant or plan to become pregnant. Lunsumio may harm your unborn baby. Tell your healthcare provider right away if you become pregnant or think you may be pregnant during treatment with Lunsumio Females who are able to become pregnant: your healthcare provider should do a pregnancy test before you start treatment with Lunsumio you should use an effective method of birth control during your treatment and for 3 months after the last dose of Lunsumio are breastfeeding or plan to breastfeed. It is not known if Lunsumio passes into your breast milk. Do not breastfeed during treatment and for 3 months after the last dose of Lunsumio Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. What should I avoid while receiving Lunsumio? Do not drive, operate heavy machinery, or do other dangerous activities if you develop dizziness, confusion, tremors, sleepiness, or any other symptoms that impair consciousness until your signs and symptoms go away. These may be signs and symptoms of CRS or neurologic problems. These are not all the possible side effects of Lunsumio. Talk to your healthcare provider for more information about the benefits and risks of Lunsumio. You may report side effects to the FDA at (800) FDA-1088 or http://www.fda.gov/medwatch . You may also report side effects to Genentech at (888) 835-2555. Please see Important Safety Information, including Serious Side Effects, as well as the Lunsumio full Prescribing Information and Medication Guide or visit https://www.Lunsumio.com . About Columvi ® (glofitamab-gxbm) Columvi is a CD20xCD3 T-cell engaging bispecific antibody designed to target CD3 on the surface of T cells and CD20 on the surface of B cells. Columvi was designed with a novel 2:1 structural format. This T-cell engaging bispecific antibody is engineered to have one region that binds to CD3, a protein on T cells, a type of immune cell, and two regions that bind to CD20, a protein on B cells, which can be healthy or malignant. This dual-targeting brings the T cell in close proximity to the B cell, activating the release of cancer cell-killing proteins from the T cell. A clinical development program for Columvi is ongoing, investigating the molecule as a monotherapy and in combination with other medicines for the treatment of people with B-cell non-Hodgkin’s lymphomas, including diffuse large B-cell lymphoma and other blood cancers. Columvi U.S. Indication Columvi (glofitamab-gxbm) is a prescription medicine to treat adults with certain types of diffuse large B-cell lymphoma (DLBCL) or large B-cell lymphoma (LBCL) that has come back (relapsed) or that did not respond to previous treatment (refractory), and who have received 2 or more prior treatments for their cancer. It is not known if Columvi is safe and effective in children. The conditional approval of Columvi is based on response rate and durability of response. There are ongoing studies to establish how well the drug works. What is the most important information I should know about Columvi? Columvi can cause Cytokine Release Syndrome (CRS), a serious side effect that is common during treatment with Columvi, and can also be serious and lead to death. Call your healthcare provider or get emergency medical help right away if you develop any signs or symptoms of CRS, including: fever of 100.4°F (38°C) or higher chills or shaking fast or irregular heartbeat dizziness or light-headedness trouble breathing shortness of breath Due to the risk of CRS, you will receive Columvi on a “step-up dosing schedule”. A single dose of a medicine called obinutuzumab will be given to you on the first day of your first treatment cycle (Day 1 of Cycle 1). You will start the Columvi step-up dosing schedule a week after the obinutuzumab dose. The step-up dosing schedule is when you receive smaller “step-up” doses of Columvi on Day 8 and Day 15 of Cycle 1. This is to help reduce your risk of CRS. You should be hospitalized during your infusion and for 24 hours after receiving the first step-up dose on Day 8. You should be hospitalized during your infusion and for 24 hours after receiving the second step-up dose on Day 15 if you experienced CRS during the first step-up dose. You will receive your first full dose of Columvi a week after the second step-up dose (this will be Day 1 of Cycle 2). If your dose of Columvi is delayed for any reason, you may need to repeat the “step-up dosing schedule”. If you had more than mild CRS with your previous dose of Columvi, you should be hospitalized during and for 24 hours after receiving your next dose of Columvi. Before each dose of Columvi, you will receive medicines to help reduce your risk of CRS and infusion-related reactions. Your healthcare provider will monitor you for CRS during treatment with Columvi and may treat you in a hospital if you develop signs and symptoms of CRS. Your healthcare provider may temporarily stop or completely stop your treatment with Columvi if you have severe side effects. Carry the Columvi Patient Wallet Card with you at all times and show it to all of your healthcare providers. The Columvi Patient Wallet Card lists the signs and symptoms of CRS you should get emergency medical help for right away. What are the possible side effects of Columvi? Columvi may cause serious side effects, including: Cytokine Release Syndrome. Neurologic problems. Columvi can cause serious neurologic problems that may lead to death. Your healthcare provider will monitor you for neurologic problems during treatment with Columvi. Your healthcare provider may also refer you to a healthcare provider who specializes in neurologic problems. Tell your healthcare provider right away if you develop any signs or symptoms of neurologic problems, including: headache confusion and disorientation difficulty paying attention or understanding things trouble speaking sleepiness memory problems numbness, tingling, or weakness of the hands or feet dizziness shaking (tremors) Serious Infections. Columvi can cause serious infections that may lead to death. Your healthcare provider will monitor you for signs and symptoms of infection and treat you as needed. Tell your healthcare provider right away if you develop any signs of an infection, including: fever, chills, weakness, cough, shortness of breath, or sore throat. Growth in your tumor or worsening of tumor related problems (tumor flare). Tell your healthcare provider if you get any of these signs or symptoms of tumor flare: tender or swollen lymph nodes pain or swelling at the site of the tumor chest pain cough trouble breathing The most common side effects of Columvi include: CRS, muscle and bone pain, rash, and tiredness. The most common severe abnormal lab test results with Columvi include: decreased white blood cells, decreased phosphate (an electrolyte), increased uric acid levels, and decreased fibrinogen (a protein that helps with blood clotting). Your healthcare provider may temporarily stop or completely stop treatment with Columvi if you develop certain side effects. Before receiving Columvi, tell your healthcare provider about all of your medical conditions, including if you: have an infection have kidney problems are pregnant or plan to become pregnant. Columvi may harm your unborn baby Females who are able to become pregnant: Your healthcare provider should do a pregnancy test before you start treatment with Columvi. You should use effective birth control (contraception) during treatment and for 1 month after your last dose of Columvi. Talk to your healthcare provider about what birth control method is right for you during this time. Tell your healthcare provider right away if you become pregnant or think you may be pregnant during treatment with Columvi. are breastfeeding or plan to breastfeed. Columvi may pass into your breast milk. Do not breastfeed during treatment and for 1 month after your last dose of Columvi. Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. What should I avoid while receiving Columvi? Do not drive, operate heavy machinery, or do other dangerous activities if you develop dizziness, confusion, shaking (tremors), sleepiness, or any other symptoms that impair consciousness until your signs and symptoms go away. These may be signs and symptoms of neurologic problems. These are not all the possible side effects of Columvi. Talk to your health care provider for more information about the benefits and risks of Columvi. You may report side effects to the FDA at (800) FDA-1088 or http://www.fda.gov/medwatch . You may also report side effects to Genentech at (888) 835-2555. Please see Important Safety Information, including Serious Side Effects , as well as the Columvi full Prescribing Information and Medication Guide or visit https://www.Columvi.com . About Venclexta ® (venetoclax) Venclexta is a first-in-class targeted medicine designed to selectively bind and inhibit the B-cell lymphoma-2 (BCL-2) protein. In some blood cancers and other tumors, BCL-2 builds up and prevents cancer cells from dying or self-destructing, a process called apoptosis. Venclexta blocks the BCL-2 protein and works to help restore the process of apoptosis. Venclexta is being developed by AbbVie and Genentech, a member of the Roche Group. It is jointly commercialized by the companies in the United States and commercialized by AbbVie outside of the United States. Together, the companies are committed to research with Venclexta, which is currently being studied in clinical trials across several types of blood cancers. Venclexta U.S. Indications Venclexta is a prescription medicine used: to treat adults with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL). in combination with azacitidine, or decitabine, or low-dose cytarabine to treat adults with newly-diagnosed acute myeloid leukemia (AML) who: ‒ are 75 years of age or older, or ‒ have other medical conditions that prevent the use of standard chemotherapy. It is not known if Venclexta is safe and effective in children. Important Safety Information What is the most important information patients should know about Venclexta? Venclexta can cause serious side effects, including: Tumor lysis syndrome (TLS). TLS is caused by the fast breakdown of cancer cells. TLS can cause kidney failure, the need for dialysis treatment, and may lead to death. The patient’s doctor will do tests to check their risk of getting TLS before they start taking Venclexta. The patient will receive other medicines before starting and during treatment with Venclexta to help reduce the risk of TLS. The patient may also need to receive intravenous (IV) fluids into their vein. The patient’s doctor will do blood tests to check for TLS when the patient first starts treatment and during treatment with Venclexta. It is important for patients to keep appointments for blood tests. Patients should tell their doctor right away if they have any symptoms of TLS during treatment with Venclexta, including fever, chills, nausea, vomiting, confusion, shortness of breath, seizures, irregular heartbeat, dark or cloudy urine, unusual tiredness, or muscle or joint pain. Patients should drink plenty of water during treatment with Venclexta to help reduce the risk of getting TLS. Patients should drink 6 to 8 glasses (about 56 ounces total) of water each day, starting 2 days before the first dose on the day of the first dose of Venclexta, and each time a dose is increased. The patient’s doctor may delay, decrease the dose, or stop treatment with Venclexta if the patient has side effects. When restarting Venclexta after stopping for 1 week or longer, the patient’s doctor may again check for the risk of TLS and change the patient’s dose. What patients should not take Venclexta? Certain medicines must not be taken when the patient first starts taking Venclexta and while the dose is being slowly increased because of the risk of increased TLS. Patients should tell their doctor about all the medicines they take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Venclexta and other medicines may affect each other causing serious side effects. Patients must not start new medicines during treatment with Venclexta without first talking with their doctor. Before taking Venclexta, patients must tell their doctor about all of their medical conditions, including if they: Have kidney or liver problems. Have problems with body salts or electrolytes, such as potassium, phosphorus, or calcium. Have a history of high uric acid levels in the blood or gout. Are scheduled to receive a vaccine. Patients should not receive a “live vaccine” before, during, or after treatment with Venclexta, until the patient’s doctor tells them it is okay. If the patient is not sure about the type of immunization or vaccine, the patient should ask their doctor. These vaccines may not be safe or may not work as well during treatment with Venclexta. Are pregnant or plan to become pregnant. Venclexta may harm an unborn baby. If the patient is able to become pregnant, the patient’s doctor should do a pregnancy test before the patient starts treatment with Venclexta, and the patient should use effective birth control during treatment and for at least 30 days after the last dose of Venclexta. If the patient becomes pregnant or thinks they are pregnant, the patient should tell their doctor right away. Are breastfeeding or plan to breastfeed. It is not known if Venclexta passes into the patient’s breast milk. Patients are instructed to not breastfeed during treatment with Venclexta and for 1 week after the last dose. What to avoid while taking Venclexta: Patients should not drink grapefruit juice or eat grapefruit, Seville oranges (often used in marmalades), or starfruit while they are taking Venclexta. These products may increase the amount of Venclexta in the patient’s blood. What are the possible side effects of Venclexta? Venclexta can cause serious side effects, including: Low white blood cell counts (neutropenia). Low white blood cell counts are common with Venclexta, but can also be severe. The patient’s doctor will do blood tests to check their blood counts during treatment with Venclexta and may pause dosing. Infections. Death and serious infections such as pneumonia and blood infection (sepsis) have happened during treatment with Venclexta. The patient’s doctor will closely monitor and treat the patient right away if they have a fever or any signs of infection during treatment with Venclexta. Patients should tell their doctor right away if they have a fever or any signs of an infection during treatment with Venclexta. The most common side effects of Venclexta when used in combination with obinutuzumab or rituximab or alone in people with CLL or SLL include low white blood cell count; low platelet count; low red blood cell count; diarrhea; nausea; upper respiratory tract infection; cough; muscle and joint pain; tiredness; and swelling of arms, legs, hands, and feet. The most common side effects of Venclexta in combination with azacitidine or decitabine or low-dose cytarabine in people with AML include nausea; diarrhea; low platelet count; constipation; low white blood cell count; fever with low white blood cell count; tiredness; vomiting; swelling of arms, legs, hands, or feet; fever; infection in lungs; shortness of breath; bleeding; low red blood cell count; rash; stomach (abdominal) pain; infection in your blood; muscle and joint pain; dizziness; cough; sore throat; and low blood pressure. Venclexta may cause fertility problems in males. This may affect the ability to father a child. Patients should talk to their doctor if they have concerns about fertility. These are not all the possible side effects of Venclexta. Patients should call their doctor for medical advice about side effects. Report side effects to the FDA at 1-800-FDA-1088 or http://www.fda.gov/medwatch . Report side effects to Genentech at 1-888-835-2555. Please see the Venclexta full Prescribing Information , including the Medication Guide , for additional Important Safety Information or visit https://www.venclexta.com/ . About Hemlibra Hemlibra is a bispecific factor IXa- and factor X-directed antibody. It is designed to bring together factor IXa and factor X, proteins required to activate the natural coagulation cascade and restore the blood clotting process for hemophilia A patients. Hemlibra is a prophylactic (preventative) treatment that can be administered by an injection of a ready-to-use solution under the skin (subcutaneously) once weekly, every two weeks or every four weeks. Hemlibra was created by Chugai Pharmaceutical Co., Ltd. and is being co-developed globally by Chugai, Roche and Genentech . Hemlibra U.S. Indication Hemlibra is a prescription medicine used for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children, ages newborn and older, with hemophilia A with or without factor VIII inhibitors. Important Safety Information What is the most important information to know about Hemlibra? Hemlibra increases the potential for blood to clot. People who use activated prothrombin complex concentrate (aPCC; Feiba ® ) to treat breakthrough bleeds while taking Hemlibra may be at risk of serious side effects related to blood clots. These serious side effects include: Thrombotic microangiopathy (TMA), a condition involving blood clots and injury to small blood vessels that may cause harm to one’s kidneys, brain, and other organs Blood clots (thrombotic events), which may form in blood vessels in the arm, leg, lung, or head Patients should talk to their doctor about the signs and symptoms of these serious side effects, which can include Confusion Stomach, chest, or back pain Weakness Nausea or vomiting Swelling, pain, or redness Feeling sick or faint Decreased urination Swelling of arms and legs Yellowing of skin and eyes Eye pain, swelling, or trouble seeing Fast heart rate Numbness in your face Headache Shortness of breath Coughing up blood If patients experience any of these symptoms during or after treatment with Hemlibra, they should get medical help right away. Patients should carefully follow their healthcare provider’s instructions regarding when to use an on demand bypassing agent or factor VIII, and the dose and schedule to use for breakthrough bleed treatment. If aPCC (Feiba ® ) is needed, patients should talk to their healthcare provider in case they feel they need more than 100 U/kg of aPCC (Feiba ® ) total. Patients’ bodies may make antibodies against Hemlibra, which may stop Hemlibra from working properly. Patients should contact their healthcare provider immediately if they notice that Hemlibra has stopped working for them (e.g., increase in bleeds). The most common side effects of Hemlibra include: injection site reactions (redness, tenderness, warmth, or itching at the site of injection), headache, and joint pain. These are not all of the possible side effects of Hemlibra. Patients can speak with their healthcare provider for more information. What else should patients know about Hemlibra? Patients should see the detailed “Instructions for Use” that comes with Hemlibra for information on how to prepare and inject a dose of Hemlibra, and how to properly throw away (dispose of) used needles and syringes. Patients should stop taking their prophylactic bypassing therapy the day before they start Hemlibra Patients may continue taking their prophylactic factor VIII for the first week of Hemlibra Hemlibra may interfere with laboratory tests that measure how well blood is clotting and create an inaccurate result. Patients should speak with their healthcare provider about how this may affect their care. Medicines are sometimes prescribed for purposes other than those listed in a Medication Guide. Patients should only use Hemlibra for the condition it was prescribed. Patients should not give Hemlibra to other people, even if they have the same symptoms that they have. It may harm them. Patients should tell their healthcare provider about all the medicines they take , including prescription medicines, over-the-counter medicines, vitamins, or herbal supplements. Patients should keep a list of them to show their healthcare provider and pharmacist. Before using Hemlibra, patients should tell their healthcare provider about all of their medical conditions , including if they are pregnant, plan to become pregnant, are breastfeeding, or plan to breastfeed. Since Hemlibra was tested in males, there is no information on whether Hemlibra may impact an unborn baby or breast milk. Females who are able to become pregnant should use birth control during treatment. Side effects may be reported to the FDA at (800) FDA-1088 or www.fda.gov/medwatch . Side effects may also be reported to Genentech at (888) 835-2555. Please see Important Safety Information, including Serious Side Effects, as well as the Hemlibra full Prescribing Information and Medication Guide or visit https://www.hemlibra.com/ . Indications & Important Safety Information What is PIASKY? PIASKY is a prescription medicine used to treat a disease called paroxysmal nocturnal hemoglobinuria (PNH) in adults and children 13 years of age or older who weigh at least 88 pounds (40 kg). It is not known if PIASKY is safe and effective in children under 13 years of age and in people who weigh less than 88 pounds (40kg). What is the most important information I should know about PIASKY? PIASKY is a medicine that can affect your immune system. PIASKY may lower the ability of your immune system to fight infections PIASKY increases your chance of getting serious infections caused by Neisseria meningitidis . Meningococcal infections may quickly become life-threatening or cause death if not recognized and treated early. You must complete or update your meningococcal vaccines at least 2 weeks before your first dose of PIASKY. If your healthcare provider decides that immediate treatment with PIASKY is needed and your meningococcal vaccination is not up to date, you should receive meningococcal vaccination as soon as possible, and receive antibiotics for as long as your healthcare provider tells you. If you have been given a meningococcal vaccine in the past, you might need additional vaccines before starting PIASKY. Your healthcare provider will decide if you need additional meningococcal vaccine. Meningococcal vaccines do not prevent all meningococcal infections. Call your healthcare provider or get emergency medical care right away if you get any of these signs and symptoms of a serious meningococcal infection: fever fever and a rash fever with a high heart rate fever with a headache headache with nausea or vomiting headache with a stiff neck or stiff back confusion muscle aches, with flu-like symptoms eyes sensitive to light Your healthcare provider will give you a Patient Safety Card about the risk of serious meningococcal infection. Carry it with you at all times during treatment and for 11 months after your last dose of PIASKY. Your risk of meningococcal infection may continue for several months after your last dose of PIASKY. It is important to show this card to any healthcare provider who treats you. This will help them diagnose and treat you quickly. PIASKY is only available through a program called the PIASKY Risk Evaluation and Mitigation Strategy (PIASKY REMS). Before you can receive PIASKY, your healthcare provider must: enroll in the PIASKY REMS program. counsel you about the risk of serious meningococcal infection. give you information about the signs and symptoms of serious meningococcal infection. make sure that you are vaccinated with a meningococcal vaccine and that you receive antibiotics if you need to start PIASKY right away if you are not up to date on your vaccines. give you a Patient Safety Card about your risk of meningococcal infection. Immune system reactions called Type III hypersensitivity reactions are common during treatment with PIASKY and can be serious. If you are currently being treated with or have been treated with another C5 inhibitor medicine and you switch to PIASKY , PIASKY may cause Type III hypersensitivity reactions. People may also develop Type III hypersensitivity reactions when they switch from PIASKY to another C5 inhibitor medicine. If you have been treated with another C5 inhibitor medicine and you switch to PIASKY, or if you have been treated with PIASKY and you switch to another C5 inhibitor medicine, your healthcare provider should monitor you for 30 days after you switch medicines. Call your healthcare provider or go to the nearest emergency room right away if you have any signs or symptoms of Type III hypersensitivity reaction including: joint pain muscle or bone pain rash or skin problems itching headache kidney problems numbness and tingling or a feeling of pins and needles especially of the hands and feet fever weakness, tiredness, or lack of energy stomach trouble or pain PIASKY may also increase the risk of other types of serious infections , including infections caused by Neisseria spp. , Streptococcus pneumoniae , Haemophilus influenzae , and Neisseria gonorrhoeae . If you receive treatment with PIASKY, you should receive vaccines against Streptococcus pneumoniae . If your child receives treatment with PIASKY, your child should receive vaccines against Streptococcus pneumoniae and may receive vaccines against Haemophilus influenzae , depending on their age. Call your healthcare provider right away if you have any new signs or symptoms of infection such as: fever of 100.4°F (38°C) or higher cough chest pain tiredness feeling short of breath painful rash sore throat burning pain when passing urine feeling weak or generally unwell Who should not receive PIASKY? Do not receive PIASKY if you: Have a serious meningococcal infection caused by Neisseria meningitidis when you are starting PIASKY treatment. Are allergic to crovalimab or any of the ingredients in PIASKY. Before receiving PIASKY tell your healthcare provider about all of your medical conditions, including if you: have an infection or fever. are pregnant or plan to become pregnant. It is not known if PIASKY may harm your unborn baby. are breastfeeding or plan to breastfeed. It is not known if PIASKY passes into your breast milk. Tell your healthcare provider about all the medicines you take , including prescription and over-the-counter medicines, vitamins, and herbal supplements. PIASKY and other medicines can affect each other, causing side effects. Especially tell your healthcare provider if you are currently being treated with or have ever been treated with any other complementary C5 inhibitor (C5 inhibitor) medicine. PIASKY is a C5 inhibitor medicine. Know the medicines you take and the vaccines you receive. Keep a list of them to show your healthcare provider and pharmacist when you get a new medicine. How should I receive PIASKY? Your healthcare provider will give you your PIASKY treatment. Your first dose will be given through a vein by intravenous (IV) infusion on Day 1 by your healthcare provider. This is the first loading dose. Another loading dose will be given as an injection under the skin (subcutaneous) on Days 2, 8, 15, and 22. Your maintenance doses will begin on Day 29 and then will be given every 4 weeks as a subcutaneous injection. Your healthcare provider will prescribe the dose based on your weight. If your weight changes, tell your healthcare provider. Talk to your healthcare provider if you miss receiving your dose of PIASKY. If you are changing treatment from another C5 inhibitor such as eculizumab or ravulizumab to PIASKY , you should receive your first loading dose of PIASKY no sooner than the time you would have received your next scheduled dose of eculizumab or ravulizumab. If you stop taking PIASKY and do not switch to another treatment for your PNH, your healthcare provider will need to monitor you closely for at least 20 weeks after stopping PIASKY. Stopping treatment with PIASKY may cause a breakdown of red blood cells due to PNH. Symptoms or problems that can happen due to red blood cell breakdown include: a lower number of red blood cells (anemia) blood in your urine or dark urine feeling short of breath feeling tired or low energy (fatigue) stomach pain blood clotting (thrombosis) difficulty swallowing difficulty getting or keeping an erection (erectile dysfunction) kidneys not working properly What are the possible side effects of PIASKY? PIASKY can cause serious side effects including: Infusion- and injection-related reactions. Infusion- or injection-related reactions may happen during or after your PIASKY administration. Symptoms may include headache, pain at infusion or injection site or in other parts of your body, swelling, bruising or bleeding, red skin, itching and rash. PIASKY can also cause serious allergic reactions. Tell your healthcare provider right away or go to the nearest emergency room if you get any of the following symptoms or symptoms of a serious allergic reaction: shortness of breath or trouble breathing pain or tightness in your chest wheezing feeling dizzy or lightheaded swelling of the throat, lips, tongue, or face skin itching, hives, or rash fever or chills The most common side effects of PIASKY are: infusion-related reactions respiratory tract infections including infections of the lungs, cold symptoms, and pain or swelling of the nose or throat viral infections Type III hypersensitivity reactions Tell your healthcare provider about any side effect that bothers you or that does not go away. These are not all the possible side effects of PIASKY. Call your healthcare provider for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088 or www.fda.gov/medwatch . You may also report side effects to Genentech at (888) 835-2555. Please see the full Prescribing Information and Medication Guide for additional Important Safety Information, including Serious Side Effects , or visit https://www.piasky.com/ . About Genentech in Hematology For more than 20 years, Genentech has been developing medicines with the goal to redefine treatment in hematology. Today, we’re investing more than ever in our effort to bring innovative treatment options to people with diseases of the blood. For more information visit http://www.gene.com/hematology . About Genentech Founded more than 40 years ago, Genentech is a leading biotechnology company that discovers, develops, manufactures and commercializes medicines to treat patients with serious and life-threatening medical conditions. The company, a member of the Roche Group, has headquarters in South San Francisco, California. For additional information about the company, please visit http://www.gene.com .

Phase 2Clinical ResultPhase 3Phase 1ASH

30 Oct 2024

·www.amgen.com

AMGEN REPORTS THIRD QUARTER 2024 FINANCIAL RESULTS

THOUSAND OAKS, Calif. , Oct. 30, 2024 /PRNewswire/ -- Amgen (NASDAQ:AMGN) today announced financial results for the third quarter of 2024. "Strong growth in sales and earnings this quarter reflects the momentum we're building throughout our business. We continue to invest heavily in our rapidly advancing pipeline, with a focus on delivering innovative therapies across our core therapeutic areas," said Robert A. Bradway , chairman and chief executive officer. Key results include: References in this release to "non-GAAP" measures, measures presented "on a non-GAAP basis" and "free cash flow" (computed by subtracting capital expenditures from operating cash flow) refer to non-GAAP financial measures. Adjustments to the most directly comparable GAAP financial measures and other items are presented on the attached reconciliations. Refer to Non-GAAP Financial Measures below for further discussion. Product Sales Performance General Medicine Oncology Inflammation Rare Disease Except for TAVNEOS ® , the products listed below were added through the acquisition of Horizon on Oct. 6, 2023 . Established Products Product Sales Detail by Product and Geographic Region $Millions, except percentages Q3 '24 Q3 '23 YOY Δ U.S. ROW TOTAL TOTAL TOTAL Repatha ® $ 281 $ 286 $ 567 $ 406 40 % EVENITY ® 289 110 399 307 30 % Prolia ® 683 362 1,045 986 6 % BLINCYTO ® 237 90 327 220 49 % Vectibix ® 132 150 282 252 12 % KYPROLIS ® 238 140 378 349 8 % LUMAKRAS ® /LUMYKRAS ™ 53 45 98 52 88 % XGEVA ® 373 168 541 519 4 % Nplate ® 345 111 456 419 9 % IMDELLTRA ™ 36 — 36 — N/A MVASI ® 136 59 195 213 (8 %) TEZSPIRE ® 269 — 269 161 67 % Otezla ® 460 104 564 567 (1 %) Enbrel ® 817 8 825 1,035 (20 %) AMJEVITA ® /AMGEVITA ™ 28 138 166 152 9 % TEPEZZA ®(1) 482 6 488 — N/A KRYSTEXXA ®(1) 310 — 310 — N/A UPLIZNA ®(1) 74 32 106 — N/A TAVNEOS ® 74 6 80 37 * Ultra-Rare products (1) 180 8 188 — N/A EPOGEN ® 33 — 33 50 (34 %) Aranesp ® 105 232 337 323 4 % Parsabiv ® 32 38 70 95 (26 %) Neulasta ® 84 26 110 124 (11 %) Other products (2) 228 53 281 281 — % Total product sales $ 5,979 $ 2,172 $ 8,151 $ 6,548 24 % N/A = not applicable *Change in excess of 100% (1) Horizon-acquired products, and the Ultra-Rare products consist of RAVICTI ® , PROCYSBI ® , ACTIMMUNE ® , QUINSAIR ® and BUPHENYL ® (2) Consists of (i) Aimovig ® , RIABNI ® , KANJINTI ® , AVSOLA ® , NEUPOGEN ® , IMLYGIC ® , BEKEMV ™ , Corlanor ® , WEZLANA ™ /WEZENLA ™ and Sensipar ® /Mimpara ™ , where Biosimilars total $148 million in Q3 '24 and $104 million in Q3 '23; and (ii) Horizon-acquired products including RAYOS ® and PENNSAID ® Operating Expense, Operating Margin and Tax Rate Analysis On a GAAP basis for the third quarter: On a non-GAAP basis for the third quarter: $Millions, except percentages GAAP Non-GAAP Q3 '24 Q3 '23 YOY Δ Q3 '24 Q3 '23 YOY Δ Cost of Sales $ 3,310 $ 1,806 83 % $ 1,454 $ 1,137 28 % % of product sales 40.6 % 27.6 % 13.0 pts 17.8 % 17.4 % 0.4 pts Research & Development $ 1,450 $ 1,079 34 % $ 1,440 $ 1,070 35 % % of product sales 17.8 % 16.5 % 1.3 pts 17.7 % 16.3 % 1.4 pts Selling, General & Administrative $ 1,625 $ 1,353 20 % $ 1,565 $ 1,293 21 % % of product sales 19.9 % 20.7 % (0.8) pts 19.2 % 19.7 % (0.5) pts Other $ 71 $ 644 (89 %) $ — $ — N/A Total Operating Expenses $ 6,456 $ 4,882 32 % $ 4,459 $ 3,500 27 % Operating Margin operating income as % of product sales 25.1 % 30.9 % (5.8) pts 49.6 % 52.0 % (2.4) pts Tax Rate 8.7 % 11.1 % (2.4) pts 13.4 % 16.1 % (2.7) pts pts: percentage points N/A = not applicable Cash Flow and Balance Sheet $Billions, except shares Q3 '24 Q3 '23 YOYΔ Operating Cash Flow $ 3.6 $ 2.8 $ 0.8 Capital Expenditures $ 0.3 $ 0.2 $ 0.0 Free Cash Flow $ 3.3 $ 2.5 $ 0.8 Dividends Paid $ 1.2 $ 1.1 $ 0.1 Share Repurchases $ 0.0 $ — $ 0.0 Average Diluted Shares (millions) 542 538 4 Note: Numbers may not add due to rounding $Billions 9/30/24 12/31/23 YTD Δ Cash and Investments $ 9.0 $ 10.9 $ (1.9) Debt Outstanding $ 60.4 $ 64.6 $ (4.2) Note: Numbers may not add due to rounding 2024 Guidance For the full year 2024, the Company now expects: Third Quarter Product and Pipeline Update The Company provided the following updates on selected product and pipeline programs: General Medicine MariTide (maridebart cafraglutide, AMG 133) AMG 513 Olpasiran (AMG 890) Repatha Oncology IMDELLTRA BLINCYTO Xaluritamig (AMG 509) AMG 193 Bemarituzumab Nplate LUMAKRAS/LUMYKRAS Inflammation TEZSPIRE Rocatinlimab (AMG 451/KHK4083) Otezla Blinatumomab Inebilizumab Efavaleukin alfa (AMG 592) Ordesekimab (AMG 714/PRV-015) AMG 104 (AZD8630) Rare Disease TAVNEOS TEPEZZA KRYSTEXXA UPLIZNA Dazodalibep Daxdilimab Fipaxalparant Biosimilars TEZSPIRE is being developed in collaboration with AstraZeneca. AMG 104 is being developed in collaboration with AstraZeneca. Rocatinlimab, formerly AMG 451/KHK4083, is being developed in collaboration with Kyowa Kirin. Ordesekimab, formerly AMG 714 and also known as PRV-015, is being developed in collaboration with Provention Bio , a Sanofi Company . For the purposes of the collaboration, Provention Bio conducts a clinical trial and leads certain development and regulatory activities for the program. Xaluritamig, formerly AMG 509, is being developed pursuant to a research collaboration with Xencor, Inc. IDE397 is an investigational MAT2A inhibitor from IDEAYA Biosciences. EYLEA is a registered trademark of Regeneron Pharmaceuticals, Inc. OPDIVO is a registered trademark of Bristol-Myers Squibb Company. KEYTRUDA is a registered trademark of Merck & Co., Inc. Non-GAAP Financial Measures In this news release, management has presented its operating results for the third quarters of 2024 and 2023, in accordance with U.S. Generally Accepted Accounting Principles (GAAP) and on a non-GAAP basis. In addition, management has presented its full year 2024 EPS and tax guidance in accordance with GAAP and on a non-GAAP basis. These non-GAAP financial measures are computed by excluding certain items related to acquisitions, divestitures, restructuring and certain other items from the related GAAP financial measures. Management has presented Free Cash Flow (FCF), which is a non-GAAP financial measure, for the third quarters of 2024 and 2023. FCF is computed by subtracting capital expenditures from operating cash flow, each as determined in accordance with GAAP. The Company believes that its presentation of non-GAAP financial measures provides useful supplementary information to and facilitates additional analysis by investors. The Company uses certain non-GAAP financial measures to enhance an investor's overall understanding of the financial performance and prospects for the future of the Company's normal and recurring business activities by facilitating comparisons of results of normal and recurring business operations among current, past and future periods. The Company believes that FCF provides a further measure of the Company's liquidity. The Company uses the non-GAAP financial measures set forth in the news release in connection with its own budgeting and financial planning internally to evaluate the performance of the business, including to allocate resources and to evaluate results relative to incentive compensation targets. The non-GAAP financial measures are in addition to, not a substitute for, or superior to, measures of financial performance prepared in accordance with GAAP. About Amgen Amgen discovers, develops, manufactures and delivers innovative medicines to help millions of patients in their fight against some of the world's toughest diseases. More than 40 years ago, Amgen helped to establish the biotechnology industry and remains on the cutting-edge of innovation, using technology and human genetic data to push beyond what's known today. Amgen is advancing a broad and deep pipeline that builds on its existing portfolio of medicines to treat cancer, heart disease, osteoporosis, inflammatory diseases and rare diseases. In 2024, Amgen was named one of the "World's Most Innovative Companies" by Fast Company and one of "America's Best Large Employers" by Forbes, among other external recognitions . Amgen is one of the 30 companies that comprise the Dow Jones Industrial Average ® , and it is also part of the Nasdaq-100 Index ® , which includes the largest and most innovative non-financial companies listed on the Nasdaq Stock Market based on market capitalization. For more information, visit Amgen.com and follow Amgen on X , LinkedIn , Instagram , TikTok , YouTube and Threads . Forward-Looking Statements This news release contains forward-looking statements that are based on the current expectations and beliefs of Amgen . All statements, other than statements of historical fact, are statements that could be deemed forward-looking statements, including any statements on the outcome, benefits and synergies of collaborations, or potential collaborations, with any other company (including BeiGene, Ltd. or Kyowa Kirin Co., Ltd.), the performance of Otezla ® (apremilast) (including anticipated Otezla sales growth and the timing of non-GAAP EPS accretion), our acquisitions of Teneobio, Inc. , ChemoCentryx, Inc. , or Horizon (including the prospective performance and outlook of Horizon's business, performance and opportunities and any potential strategic benefits, synergies or opportunities expected as a result of such acquisition, and any projected impacts from the Horizon acquisition on our acquisition-related expenses going forward), as well as estimates of revenues, operating margins, capital expenditures, cash, other financial metrics, expected legal, arbitration, political, regulatory or clinical results or practices, customer and prescriber patterns or practices, reimbursement activities and outcomes, effects of pandemics or other widespread health problems on our business, outcomes, progress, and other such estimates and results. Forward-looking statements involve significant risks and uncertainties, including those discussed below and more fully described in the Securities and Exchange Commission reports filed by Amgen , including our most recent annual report on Form 10-K and any subsequent periodic reports on Form 10-Q and current reports on Form 8-K. Unless otherwise noted, Amgen is providing this information as of the date of this news release and does not undertake any obligation to update any forward-looking statements contained in this document as a result of new information, future events or otherwise. No forward-looking statement can be guaranteed and actual results may differ materially from those we project. Our results may be affected by our ability to successfully market both new and existing products domestically and internationally, clinical and regulatory developments involving current and future products, sales growth of recently launched products, competition from other products including biosimilars, difficulties or delays in manufacturing our products and global economic conditions. In addition, sales of our products are affected by pricing pressure, political and public scrutiny and reimbursement policies imposed by third-party payers, including governments, private insurance plans and managed care providers and may be affected by regulatory, clinical and guideline developments and domestic and international trends toward managed care and healthcare cost containment. Furthermore, our research, testing, pricing, marketing and other operations are subject to extensive regulation by domestic and foreign government regulatory authorities. We or others could identify safety, side effects or manufacturing problems with our products, including our devices, after they are on the market. Our business may be impacted by government investigations, litigation and product liability claims. In addition, our business may be impacted by the adoption of new tax legislation or exposure to additional tax liabilities. If we fail to meet the compliance obligations in the corporate integrity agreement between us and the U.S. government, we could become subject to significant sanctions. Further, while we routinely obtain patents for our products and technology, the protection offered by our patents and patent applications may be challenged, invalidated or circumvented by our competitors, or we may fail to prevail in present and future intellectual property litigation. We perform a substantial amount of our commercial manufacturing activities at a few key facilities, including in Puerto Rico , and also depend on third parties for a portion of our manufacturing activities, and limits on supply may constrain sales of certain of our current products and product candidate development. An outbreak of disease or similar public health threat, such as COVID-19, and the public and governmental effort to mitigate against the spread of such disease, could have a significant adverse effect on the supply of materials for our manufacturing activities, the distribution of our products, the commercialization of our product candidates, and our clinical trial operations, and any such events may have a material adverse effect on our product development, product sales, business and results of operations. We rely on collaborations with third parties for the development of some of our product candidates and for the commercialization and sales of some of our commercial products. In addition, we compete with other companies with respect to many of our marketed products as well as for the discovery and development of new products. Discovery or identification of new product candidates or development of new indications for existing products cannot be guaranteed and movement from concept to product is uncertain; consequently, there can be no guarantee that any particular product candidate or development of a new indication for an existing product will be successful and become a commercial product. Further, some raw materials, medical devices and component parts for our products are supplied by sole third-party suppliers. Certain of our distributors, customers and payers have substantial purchasing leverage in their dealings with us. The discovery of significant problems with a product similar to one of our products that implicate an entire class of products could have a material adverse effect on sales of the affected products and on our business and results of operations. Our efforts to collaborate with or acquire other companies, products or technology, and to integrate the operations of companies or to support the products or technology we have acquired, may not be successful. There can be no guarantee that we will be able to realize any of the strategic benefits, synergies or opportunities arising from the Horizon acquisition, and such benefits, synergies or opportunities may take longer to realize than expected. We may not be able to successfully integrate Horizon, and such integration may take longer, be more difficult or cost more than expected. A breakdown, cyberattack or information security breach of our information technology systems could compromise the confidentiality, integrity and availability of our systems and our data. Our stock price is volatile and may be affected by a number of events. Our business and operations may be negatively affected by the failure, or perceived failure, of achieving our environmental, social and governance objectives. The effects of global climate change and related natural disasters could negatively affect our business and operations. Global economic conditions may magnify certain risks that affect our business. Our business performance could affect or limit the ability of our Board of Directors to declare a dividend or our ability to pay a dividend or repurchase our common stock. We may not be able to access the capital and credit markets on terms that are favorable to us, or at all. CONTACT: Amgen , Thousand Oaks Elissa Snook , 609-251-1407 (media) Justin Claeys , 805-313-9775 (investors) Amgen Inc. Consolidated Statements of Income - GAAP (In millions, except per-share data) (Unaudited) Three months ended September 30 , Nine months ended September 30 , 2024 2023 2024 2023 Revenues: Product sales $ 8,151 $ 6,548 $ 23,310 $ 19,077 Other revenues 352 355 1,028 917 Total revenues 8,503 6,903 24,338 19,994 Operating expenses: Cost of sales 3,310 1,806 9,746 5,339 Research and development 1,450 1,079 4,240 3,250 Selling, general and administrative 1,625 1,353 5,218 3,905 Other 71 644 187 874 Total operating expenses 6,456 4,882 19,391 13,368 Operating income 2,047 2,021 4,947 6,626 Other income (expense): Interest expense, net (776) (759) (2,408) (2,054) Other income, net 1,830 685 1,288 2,431 Income before income taxes 3,101 1,947 3,827 7,003 Provision for income taxes 271 217 364 1,053 Net income $ 2,830 $ 1,730 $ 3,463 $ 5,950 Earnings per share: Basic $ 5.27 $ 3.23 $ 6.45 $ 11.12 Diluted $ 5.22 $ 3.22 $ 6.40 $ 11.06 Weighted-average shares used in calculation of earnings per share: Basic 537 535 537 535 Diluted 542 538 541 538 Amgen Inc. Consolidated Balance Sheets - GAAP (In millions) September 30 , December 31 , 2024 2023 (Unaudited) Assets Current assets: Cash and cash equivalents $ 9,011 $ 10,944 Trade receivables, net 7,317 7,268 Inventories 7,362 9,518 Other current assets 3,076 2,602 Total current assets 26,766 30,332 Property, plant and equipment, net 6,156 5,941 Intangible assets, net 28,920 32,641 Goodwill 18,658 18,629 Other noncurrent assets 10,383 9,611 Total assets $ 90,883 $ 97,154 Liabilities and Stockholders' Equity Current liabilities: Accounts payable and accrued liabilities $ 16,768 $ 16,949 Current portion of long-term debt 3,544 1,443 Total current liabilities 20,312 18,392 Long-term debt 56,854 63,170 Long-term deferred tax liabilities 1,711 2,354 Long-term tax liabilities 2,280 4,680 Other noncurrent liabilities 2,199 2,326 Total stockholders' equity 7,527 6,232 Total liabilities and stockholders' equity $ 90,883 $ 97,154 Shares outstanding 538 535 Amgen Inc. GAAP to Non-GAAP Reconciliations (Dollars in millions) (Unaudited) Three months ended September 30 , Nine months ended September 30 , 2024 2023 2024 2023 GAAP cost of sales $ 3,310 $ 1,806 $ 9,746 $ 5,339 Adjustments to cost of sales: Acquisition-related expenses (a) (1,856) (668) (5,546) (2,008) Certain net charges pursuant to our restructuring and cost-savings initiatives — (1) — (36) Total adjustments to cost of sales (1,856) (669) (5,546) (2,044) Non-GAAP cost of sales $ 1,454 $ 1,137 $ 4,200 $ 3,295 GAAP cost of sales as a percentage of product sales 40.6 % 27.6 % 41.8 % 28.0 % Acquisition-related expenses (a) (22.8) (10.2) (23.8) (10.5) Certain net charges pursuant to our restructuring and cost-savings initiatives 0.0 0.0 0.0 (0.2) Non-GAAP cost of sales as a percentage of product sales 17.8 % 17.4 % 18.0 % 17.3 % GAAP research and development expenses $ 1,450 $ 1,079 $ 4,240 $ 3,250 Adjustments to research and development expenses: Acquisition-related expenses (b) (10) (9) (60) (27) Certain net charges pursuant to our restructuring and cost-savings initiatives — — — (17) Total adjustments to research and development expenses (10) (9) (60) (44) Non-GAAP research and development expenses $ 1,440 $ 1,070 $ 4,180 $ 3,206 GAAP research and development expenses as a percentage of product sales 17.8 % 16.5 % 18.2 % 17.0 % Acquisition-related expenses (b) (0.1) (0.2) (0.3) (0.1) Certain net charges pursuant to our restructuring and cost-savings initiatives 0.0 0.0 0.0 (0.1) Non-GAAP research and development expenses as a percentage of product sales 17.7 % 16.3 % 17.9 % 16.8 % GAAP selling, general and administrative expenses $ 1,625 $ 1,353 $ 5,218 $ 3,905 Adjustments to selling, general and administrative expenses: Acquisition-related expenses (c) (60) (47) (255) (138) Certain net charges pursuant to our restructuring and cost-savings initiatives — (13) — (13) Total adjustments to selling, general and administrative expenses (60) (60) (255) (151) Non-GAAP selling, general and administrative expenses $ 1,565 $ 1,293 $ 4,963 $ 3,754 GAAP selling, general and administrative expenses as a percentage of product sales 19.9 % 20.7 % 22.4 % 20.5 % Acquisition-related expenses (c) (0.7) (0.8) (1.1) (0.7) Certain net charges pursuant to our restructuring and cost-savings initiatives 0.0 (0.2) 0.0 (0.1) Non-GAAP selling, general and administrative expenses as a percentage of product sales 19.2 % 19.7 % 21.3 % 19.7 % GAAP operating expenses $ 6,456 $ 4,882 $ 19,391 $ 13,368 Adjustments to operating expenses: Adjustments to cost of sales (1,856) (669) (5,546) (2,044) Adjustments to research and development expenses (10) (9) (60) (44) Adjustments to selling, general and administrative expenses (60) (60) (255) (151) Certain net charges pursuant to our restructuring and cost-savings initiatives (d) — (16) 4 (183) Certain other expenses (e) (71) (628) (191) (691) Total adjustments to operating expenses (1,997) (1,382) (6,048) (3,113) Non-GAAP operating expenses $ 4,459 $ 3,500 $ 13,343 $ 10,255 Three months ended September 30 , Nine months ended September 30 , 2024 2023 2024 2023 GAAP operating income $ 2,047 $ 2,021 $ 4,947 $ 6,626 Adjustments to operating expenses 1,997 1,382 6,048 3,113 Non-GAAP operating income $ 4,044 $ 3,403 $ 10,995 $ 9,739 GAAP operating income as a percentage of product sales 25.1 % 30.9 % 21.2 % 34.7 % Adjustments to cost of sales 22.8 10.2 23.8 10.7 Adjustments to research and development expenses 0.1 0.2 0.3 0.2 Adjustments to selling, general and administrative expenses 0.7 1.0 1.1 0.8 Certain net charges pursuant to our restructuring and cost-savings initiatives (d) 0.0 0.2 0.0 1.0 Certain other expenses (e) 0.9 9.5 0.8 3.7 Non-GAAP operating income as a percentage of product sales 49.6 % 52.0 % 47.2 % 51.1 % GAAP interest expense, net $ (776) $ (759) $ (2,408) $ (2,054) Adjustments to interest expense, net: Interest expense on acquisition-related debt (f) — 332 — 788 Non-GAAP interest expense, net $ (776) $ (427) $ (2,408) $ (1,266) GAAP other income, net $ 1,830 $ 685 $ 1,288 $ 2,431 Adjustments to other income, net Interest income and other expenses on acquisition-related debt (f) — (313) — (607) Net gains from equity investments (g) (1,608) (170) (693) (1,305) Total adjustments to other income, net (1,608) (483) (693) (1,912) Non-GAAP other income, net $ 222 $ 202 $ 595 $ 519 GAAP income before income taxes $ 3,101 $ 1,947 $ 3,827 $ 7,003 Adjustments to income before income taxes: Adjustments to operating expenses 1,997 1,382 6,048 3,113 Adjustments to interest expense, net — 332 — 788 Adjustments to other income, net (1,608) (483) (693) (1,912) Total adjustments to income before income taxes 389 1,231 5,355 1,989 Non-GAAP income before income taxes $ 3,490 $ 3,178 $ 9,182 $ 8,992 GAAP provision for income taxes $ 271 $ 217 $ 364 $ 1,053 Adjustments to provision for income taxes: Income tax effect of the above adjustments (h) 228 271 1,007 442 Other income tax adjustments (i) (33) 23 (44) 6 Total adjustments to provision for income taxes 195 294 963 448 Non-GAAP provision for income taxes $ 466 $ 511 $ 1,327 $ 1,501 GAAP tax as a percentage of income before taxes 8.7 % 11.1 % 9.5 % 15.0 % Adjustments to provision for income taxes: Income tax effect of the above adjustments (h) 5.6 4.2 5.4 1.6 Other income tax adjustments (i) (0.9) 0.8 (0.4) 0.1 Total adjustments to provision for income taxes 4.7 5.0 5.0 1.7 Non-GAAP tax as a percentage of income before taxes 13.4 % 16.1 % 14.5 % 16.7 % GAAP net income $ 2,830 $ 1,730 $ 3,463 $ 5,950 Adjustments to net income: Adjustments to income before income taxes, net of the income tax effect 161 960 4,348 1,547 Other income tax adjustments (i) 33 (23) 44 (6) Total adjustments to net income 194 937 4,392 1,541 Non-GAAP net income $ 3,024 $ 2,667 $ 7,855 $ 7,491 Note: Numbers may not add due to rounding Amgen Inc. GAAP to Non-GAAP Reconciliations (In millions, except per-share data) (Unaudited) The following table presents the computations for GAAP and non-GAAP diluted earnings per share: Three months ended September 30, 2024 Three months ended September 30, 2023 GAAP Non-GAAP GAAP Non-GAAP Net income $ 2,830 $ 3,024 $ 1,730 $ 2,667 Weighted-average shares for diluted EPS 542 542 538 538 Diluted EPS $ 5.22 $ 5.58 $ 3.22 $ 4.96 Nine months ended September 30, 2024 Nine months ended September 30, 2023 GAAP Non-GAAP GAAP Non-GAAP Net income $ 3,463 $ 7,855 $ 5,950 $ 7,491 Weighted-average shares for diluted EPS 541 541 538 538 Diluted EPS $ 6.40 $ 14.52 $ 11.06 $ 13.92 (a) The adjustments related primarily to noncash amortization of intangible assets and fair value step-up of inventory acquired from business acquisitions. (b) For the three months ended September 30, 2024 , the adjustments related primarily to noncash amortization of intangible assets from business acquisitions. For the nine months ended September 30, 2024 , the adjustments related primarily to acquisition-related costs related to our Horizon acquisition. For the three and nine months ended September 30, 2023 , the adjustments related primarily to noncash amortization of intangible assets from business acquisitions. (c) For the three and nine months ended September 30, 2024 and 2023, the adjustments related primarily to acquisition-related costs related to our Horizon acquisition. (d) For the three and nine months ended September 30, 2023 , the adjustments related primarily to separation costs associated with our restructuring plan initiated in early 2023. (e) For the three and nine months ended September 30, 2024 , the adjustments related primarily to impairment charges for in-process R&D assets and changes in the fair values of contingent consideration liabilities, both related to our Teneobio, Inc. acquisition from 2021. For the three and nine months ended September 30, 2023 , the adjustments related primarily to a net impairment charge for AMG 340. (f) For the three and nine months ended September 30, 2023 , the adjustments included (i) interest expense and income on senior notes issued in March 2023 and (ii) debt issuance costs and other fees related to our bridge credit and term loan credit agreements, incurred prior to the closing of our acquisition of Horizon. (g) For the three and nine months ended September 30, 2024 , the adjustments related primarily to our BeiGene equity fair value adjustment. For the three and nine months ended September 30, 2023 , the adjustments related primarily to our Neumora Therapeutics, Inc. and BeiGene equity fair value adjustments, respectively. (h) The tax effect of the adjustments between our GAAP and non-GAAP results takes into account the tax treatment and related tax rate(s) that apply to each adjustment in the applicable tax jurisdiction(s). Generally, the tax impact of adjustments, including the amortization of intangible assets and acquired inventory, gains and losses on our investments in equity securities and expenses related to restructuring and cost-savings initiatives, depends on whether the amounts are deductible in the respective tax jurisdictions and the applicable tax rate(s) in those jurisdictions. Due to these factors, the effective tax rate for the adjustments to our GAAP income before income taxes for the three and nine months ended September 30, 2024 , was 58.6% and 18.8%, respectively, compared to 22.0% and 22.2% for the corresponding periods of the prior year. (i) The adjustments related to certain acquisition items, prior period and other items excluded from GAAP earnings. Amgen Inc. Reconciliations of Cash Flows (In millions) (Unaudited) Three months ended September 30 , Nine months ended September 30 , 2024 2023 2024 2023 Net cash provided by operating activities $ 3,571 $ 2,760 $ 6,719 $ 7,933 Net cash (used in) provided by investing activities (210) (262) (644) 885 Net cash (used in) provided by financing activities (3,651) (2,005) (8,008) 18,294 (Decrease) increase in cash and cash equivalents (290) 493 (1,933) 27,112 Cash and cash equivalents at beginning of period 9,301 34,248 10,944 7,629 Cash and cash equivalents at end of period $ 9,011 $ 34,741 $ 9,011 $ 34,741 Three months ended September 30 , Nine months ended September 30 , 2024 2023 2024 2023 Net cash provided by operating activities $ 3,571 $ 2,760 $ 6,719 $ 7,933 Capital expenditures (257) (248) (725) (863) Free cash flow $ 3,314 $ 2,512 $ 5,994 $ 7,070 Amgen Inc. Reconciliation of GAAP EPS Guidance to Non-GAAP EPS Guidance for the Year Ending December 31, 2024 (Unaudited) GAAP diluted EPS guidance $ 8.71 — $ 9.56 Known adjustments to arrive at non-GAAP*: Acquisition-related expenses (a) 11.33 — 11.38 Net gains from equity investments (1.01) Other 0.12 Non-GAAP diluted EPS guidance $ 19.20 — $ 20.00 * The known adjustments are presented net of their related tax impact, which amount to approximately $2.39 per share. (a) The adjustments primarily include noncash amortization of intangible assets and fair value step-up of inventory acquired in business acquisitions. Our GAAP diluted EPS guidance does not include the effect of GAAP adjustments triggered by events that may occur subsequent to this press release such as acquisitions, asset impairments, litigation, changes in fair value of our contingent consideration obligations and changes in fair value of our equity investments. Reconciliation of GAAP Tax Rate Guidance to Non-GAAP Tax Rate Guidance for the Year Ending December 31, 2024 (Unaudited) GAAP tax rate guidance 9.0 % — 10.5 % Tax rate of known adjustments discussed above 4.5 % — 5.0 % Non-GAAP tax rate guidance 14.0 % — 15.0 % View original content to download multimedia: https://www.prnewswire.com/news-releases/amgen-reports-third-quarter-2024-financial-results-302291966.html SOURCE Amgen

AcquisitionFinancial StatementBiosimilarDrug Approval

100 Deals associated with Eculizumab

External Link

KEGG	Wiki	ATC	Drug Bank
D03940	Eculizumab	L04AA25	DB01257

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
AQP4-IgG positive Neuromyelitis optica spectrum disorder	AU	Alexion Pharmaceuticals, Inc.	20 Mar 2009
Atypical Hemolytic Uremic Syndrome	EU	Alexion Europe SAS	20 Jun 2007
Atypical Hemolytic Uremic Syndrome	IS	Alexion Europe SAS	20 Jun 2007
Atypical Hemolytic Uremic Syndrome	LI	Alexion Europe SAS	20 Jun 2007
Atypical Hemolytic Uremic Syndrome	NO	Alexion Europe SAS	20 Jun 2007
Myasthenia Gravis	EU	Alexion Europe SAS	20 Jun 2007
Myasthenia Gravis	IS	Alexion Europe SAS	20 Jun 2007
Myasthenia Gravis	LI	Alexion Europe SAS	20 Jun 2007
Myasthenia Gravis	NO	Alexion Europe SAS	20 Jun 2007
Neuromyelitis Optica	EU	Alexion Europe SAS	20 Jun 2007
Neuromyelitis Optica	IS	Alexion Europe SAS	20 Jun 2007
Neuromyelitis Optica	LI	Alexion Europe SAS	20 Jun 2007
Neuromyelitis Optica	NO	Alexion Europe SAS	20 Jun 2007
Hemoglobinuria, Paroxysmal	US	Alexion Pharmaceuticals, Inc.	16 Mar 2007

Developing

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Indication	Highest Phase	Country/Location	Organization	Date
Delayed Graft Function	Phase 3	US	Alexion Pharmaceuticals, Inc.	21 Aug 2014
Delayed Graft Function	Phase 3	AU	Alexion Pharmaceuticals, Inc.	21 Aug 2014
Delayed Graft Function	Phase 3	BR	Alexion Pharmaceuticals, Inc.	21 Aug 2014
Delayed Graft Function	Phase 3	CA	Alexion Pharmaceuticals, Inc.	21 Aug 2014
Delayed Graft Function	Phase 3	CZ	Alexion Pharmaceuticals, Inc.	21 Aug 2014
Delayed Graft Function	Phase 3	FR	Alexion Pharmaceuticals, Inc.	21 Aug 2014
Delayed Graft Function	Phase 3	DE	Alexion Pharmaceuticals, Inc.	21 Aug 2014
Delayed Graft Function	Phase 3	IT	Alexion Pharmaceuticals, Inc.	21 Aug 2014
Delayed Graft Function	Phase 3	ES	Alexion Pharmaceuticals, Inc.	21 Aug 2014
AQP4-IgG positive Optic Neuritis	Phase 3	US	Alexion Pharmaceuticals, Inc.	11 Apr 2014

Clinical Result

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04320602 (CTgov)	Phase 4	Hemoglobinuria, Paroxysmal	18	Eculizumab+Ravulizumab	pqlilzwaxo(loffngwbkq) = vjbirrmtme fpqlahxhvx (huqgbgskdx, pruzvuduhc - ahrmrvnxba) View more	-	12 Aug 2024
NCT04085601 \| NCT03500549 \| NCT02588833 (PADDOCK \| PRINCE \| PEGASUS, Pubmed)	Not Applicable	Anemia \| Hemoglobinuria, Paroxysmal	25	Pegcetacoplan	yavzquimpo(plxuvnzgfv) = increased mqjsjnskex (dappcarhhc ) View more	Positive	29 Jul 2024
				Eculizumab
NCT00844428 \| NCT00838513 (C08-003B, FDA_CDER) Manual	Phase 2	Atypical Hemolytic Uremic Syndrome	20	Eculizumab (at 26 wks)	lwzuepaott(sxqqqlwcxc) = ywhyfqmqkm rwqlmyjbxd (iywdntfgli ) View more	Positive	19 Jul 2024
				Eculizumab (at 2 yrs)	lwzuepaott(sxqqqlwcxc) = ypxifcahyo rwqlmyjbxd (iywdntfgli ) View more
NCT01770951 (C09-001r, FDA_CDER) Manual	Not Applicable	Atypical Hemolytic Uremic Syndrome	19	Eculizumab (<2 yrs)	ztojwervcy(haknqmsdpi) = hjurbjbboq lijqzeonlk (rlbjgcnsyx ) View more	Positive	19 Jul 2024
				Eculizumab (2 to <12 yrs)	ztojwervcy(haknqmsdpi) = ttqjcxjvxt lijqzeonlk (rlbjgcnsyx ) View more
NCT00844844 \| NCT00844545 (C08-002B, FDA_CDER) Manual	Phase 2	Atypical Hemolytic Uremic Syndrome	17	Eculizumab (at 26 wks)	qjkynossqm(aiwozelvts) = kjkzydxfcb sxgxgtdvqr (wwvybabtsd ) View more	Positive	19 Jul 2024
				Eculizumab (at 2 yrs)	qjkynossqm(aiwozelvts) = rwaqflpavl sxgxgtdvqr (wwvybabtsd ) View more
NCT01193348 (C10-003, FDA_CDER) Manual	Phase 2	Atypical Hemolytic Uremic Syndrome	22	Eculizumab (Patients 1 month to <12 years)	dxgacspknf(dgzegvpjcj) = gpraxhajrr uomdvyjppr (izmsgmshdc, 36 - 83) View more	Positive	19 Jul 2024
				Eculizumab (All Patients)	dxgacspknf(dgzegvpjcj) = lxwgqugamz uomdvyjppr (izmsgmshdc, 41 - 83) View more
NCT01194973 (C10-004, FDA_CDER) Manual	Phase 2	Atypical Hemolytic Uremic Syndrome	41	Eculizumab	tkwbgvmeya(lrozfvporg) = kijmmrxlku jbxbzemoxk (yaujeilkwv, 40 - 72) View more	Positive	19 Jul 2024
NCT00122330 (FDA_CDER) Manual	Phase 3	Hemoglobinuria, Paroxysmal	87	Placebo	wwisiknysw(dgweetrkyf) = wperbwbcuw fnapycdmek (xzfjkrlals ) View more	Positive	19 Jul 2024
				Eculizumab	wwisiknysw(dgweetrkyf) = ywmvwlnlra fnapycdmek (xzfjkrlals ) View more
NCT03500549 (PEGASUS, EHA2024) Manual	Phase 3	Hemoglobinuria, Paroxysmal	68	pegcetacoplan +eculizumab	-	Positive	14 May 2024
				pegcetacoplan +eculizumab (patients with baseline IO)	avywfvcqsf(schacipjmo) = snpshbpdmx wnjoofruvi (zmizqiwwzu, 8.0) View more
EHA2024	Not Applicable	Hemoglobinuria, Paroxysmal	200	Eculizumab	cdtnguawhk(causyfhzsa) = cijuwaljzo loyywtwduk (xpxdyotjbk ) View more	Positive	14 May 2024
				Ravulizumab	cdtnguawhk(causyfhzsa) = urgiqagvqt loyywtwduk (xpxdyotjbk ) View more

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