Sodium Phenylbutyrate

THOUSAND OAKS, Calif., Aug. 5, 2025 /PRNewswire/ -- Amgen (NASDAQ:AMGN) today announced financial results for the second quarter of 2025. "We're delivering strong performance and reaching more patients with innovative medicines and biosimilars that address serious diseases. We continue to invest in science that enables longer, healthier lives and supports sustainable, long-term growth," said Robert A. Bradway, chairman and chief executive officer. Key results include: For the second quarter, total revenues increased 9% to $9.2 billion in comparison to the second quarter of 2024. Product sales grew 9%, driven by 13% volume growth, partially offset by 3% lower net selling price. Fifteen products delivered at least double-digit sales growth in the second quarter, including Repatha® (evolocumab), EVENITY® (romosozumab-aqqg), IMDELLTRA® (tarlatamab-dlle)/IMDYLLTRA™ (tarlatamab), BLINCYTO® (blinatumomab), TEZSPIRE® (tezepelumab-ekko), UPLIZNA® (inebilizumab-cdon) and TAVNEOS® (avacopan). GAAP earnings per share (EPS) increased 92% from $1.38 to $2.65, primarily driven by higher revenues. GAAP operating income increased from $1.9 billion to $2.7 billion, and GAAP operating margin increased 6.6 percentage points to 30.3%. Non-GAAP EPS increased 21% from $4.97 to $6.02, primarily driven by higher revenues, partially offset by higher operating expenses. Non-GAAP operating income increased from $3.9 billion to $4.3 billion, and non-GAAP operating margin increased 0.7 percentage points to 48.9%. The Company generated $1.9 billion of free cash flow in the second quarter of 2025 versus $2.2 billion in the second quarter of 2024, driven by 2024 tax payments deferred to 2025 and higher capital expenditures, partially offset by business performance. 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 in the attached reconciliations. Refer to Non-GAAP Financial Measures below for further discussion. Product Sales Performance General Medicine Repatha® (evolocumab) sales increased 31% year-over-year to $696 million in the second quarter, driven by 36% volume growth, partially offset by unfavorable changes to estimated sales deductions. EVENITY® (romosozumab-aqqg) sales increased 32% year-over-year to $518 million in the second quarter, primarily driven by volume growth. Prolia® (denosumab) sales decreased 4% year-over-year to $1.1 billion in the second quarter, driven by lower net selling price. For 2025, we expect sales erosion driven by biosimilar competition in the second half of the year, as biosimilars have now launched in the U.S. market. Rare Disease TEPEZZA® (teprotumumab-trbw) sales increased 5% year-over-year to $505 million in the second quarter, primarily driven by higher inventory levels. KRYSTEXXA® (pegloticase) sales increased 19% year-over-year to $349 million in the second quarter, of which 12% was derived from higher inventory levels and 6% from volume growth. UPLIZNA® (inebilizumab-cdon) sales increased 91% year-over-year to $176 million in the second quarter, driven by 79% volume growth, with 16% derived from higher inventory levels. Year-over-year sales benefited from the timing of shipments to our ex-U.S. partner that occurred in the third quarter of 2024. Excluding these shipments, sales grew by 56% year-over-year in the second quarter. TAVNEOS® (avacopan) sales increased 55% year-over-year to $110 million in the second quarter, driven by volume growth. Ultra-Rare products, which consist of RAVICTI® (glycerol phenylbutyrate), PROCYSBI® (cysteamine bitartrate), ACTIMMUNE® (interferon gamma-1b), QUINSAIR® (levofloxacin) and BUPHENYL® (sodium phenylbutyrate), generated $183 million of sales in the second quarter. Sales decreased 2% year-over-year for the second quarter, driven by lower net selling price. Inflammation TEZSPIRE® (tezepelumab-ekko) sales increased 46% year-over-year to $342 million in the second quarter, driven by volume growth. Otezla® (apremilast) sales increased 14% year-over-year to $618 million in the second quarter, driven by 12% favorable changes to estimated sales deductions and 4% volume growth. Enbrel® (etanercept) sales decreased 34% year-over-year to $604 million in the second quarter, driven by 20% unfavorable changes to estimated sales deductions and 19% lower net selling price resulting from increased 340B Program mix and the impact of the U.S. Medicare Part D redesign, partially offset by 3% volume growth. AMJEVITA® (adalimumab-atto)/AMGEVITA™ (adalimumab) sales were flat year-over-year at $133 million in the second quarter. PAVBLU® (aflibercept-ayyh) generated $130 million of sales in the second quarter. WEZLANA™ (ustekinumab-auub)/WEZENLA™ (ustekinumab) generated $35 million of sales in the second quarter. Oncology BLINCYTO® (blinatumomab) sales increased 45% year-over-year to $384 million in the second quarter, driven by volume growth. Vectibix® (panitumumab) sales increased 13% year-over-year to $305 million in the second quarter, driven by volume growth. KYPROLIS® (carfilzomib) sales were flat year-over-year at $378 million in the second quarter. LUMAKRAS®/LUMYKRAS™ (sotorasib) sales increased 6% year-over-year to $90 million in the second quarter, driven by 16% volume growth, partially offset by 10% lower net selling price. XGEVA® (denosumab) sales decreased 5% year-over-year to $532 million in the second quarter, driven by 2% unfavorable changes to estimated sales deductions and volume decline. For 2025, we expect sales erosion driven by biosimilar competition in the second half of the year, as biosimilars have now launched in the U.S. market. Nplate® (romiplostim) sales increased 7% year-over-year to $369 million in the second quarter, driven by volume growth. IMDELLTRA® (tarlatamab-dlle)/IMDYLLTRA™ (tarlatamab) generated $134 million of sales in the second quarter. Sales increased 65% quarter-over-quarter, driven by volume growth. MVASI® (bevacizumab-awwb) sales increased 22% year-over-year to $191 million in the second quarter, driven by 16% favorable changes to estimated sales deductions, 6% volume growth and 5% higher net selling price, partially offset by lower inventory levels. In the quarter, MVASI benefited from competitor bevacizumab product shortages, and going forward, we expect to return to continued sales erosion driven by competition. Established Products Our established products, which consist of Aranesp® (darbepoetin alfa), Parsabiv® (etelcalcetide) and Neulasta® (pegfilgrastim), generated $533 million of sales in the second quarter. Sales decreased 5% year-over-year for the second quarter, driven by 14% lower net selling price and 4% lower volume, partially offset by favorable changes to estimated sales deductions. Product Sales Detail by Product and Geographic Region Operating Expense, Operating Margin and Tax Rate Analysis On a GAAP basis: Total Operating Expenses increased 1% year-over-year for the second quarter. Cost of Sales as a percentage of product sales decreased 5.9 percentage points driven by lower amortization expense from the fair value step-up of inventory acquired from Horizon and lower manufacturing costs, partially offset by higher profit share expense and changes in our sales mix. Research & Development (R&D) expenses increased 21% driven by investments in later-stage clinical programs, including those related to MariTide, for which four Phase 3 studies are underway. Selling, General & Administrative (SG&A) expenses decreased 5% driven by lower commercial product-related expenses and lower Horizon acquisition-related expenses. Other operating expenses include litigation expenses. Operating Margin as a percentage of product sales increased 6.6 percentage points in the second quarter to 30.3%. Tax Rate increased 2.7 percentage points in the second quarter due to the change in earnings mix, including lower amortization expense from the fair value step-up of inventory acquired from Horizon and current year net unfavorable items, as compared to the prior year. On a non-GAAP basis: Total Operating Expenses increased 8% year-over-year for the second quarter. Cost of Sales as a percentage of product sales increased 0.2 percentage points driven by higher profit share expense and changes in our sales mix, partially offset by lower manufacturing costs. R&D expenses increased 18% driven by investments in later-stage clinical programs, including those related to MariTide, for which four Phase 3 studies are underway. SG&A expenses decreased 2% primarily driven by lower commercial product-related expenses. Operating Margin as a percentage of product sales increased 0.7 percentage points in the second quarter to 48.9%. Tax Rate decreased 0.7 percentage points in the second quarter due to the change in earnings mix. Cash Flow and Balance Sheet The Company generated $1.9 billion of free cash flow in the second quarter of 2025 versus $2.2 billion in the second quarter of 2024, driven by timing of 2024 tax payments deferred to 2025 and higher capital expenditures, partially offset by business performance. The Company declared a second quarter 2025 dividend on March 4, 2025 of $2.38 per share that was paid on June 6, 2025 to all stockholders of record as of May 16, 2025, representing a 6% increase from the same period in 2024. The Company retired $1.4 billion of debt during the second quarter of 2025, and $4.3 billion year to date. During the second quarter of 2025, there were no repurchases of shares of common stock. Cash and cash equivalents totaled $8.0 billion and debt outstanding totaled $56.2 billion as of June 30, 2025. For the full year 2025, the Company expects: Total revenues in the range of $35.0 billion to $36.0 billion. On a GAAP basis, EPS in the range of $10.97 to $12.11, and a tax rate in the range of 11.0% to 12.5%. On a non-GAAP basis, EPS in the range of $20.20 to $21.30, and a tax rate in the range of 14.5% to 16.0%. Capital expenditures to be approximately $2.3 billion. Share repurchases not to exceed $500 million. This guidance includes the estimated impact of implemented tariffs, but does not account for any tariffs or potential pricing actions announced or described but not implemented as well as any tariffs, sector specific tariffs, or pricing actions that could be implemented in the future. Second Quarter Product and Pipeline Update The Company provided the following updates on selected product and pipeline programs: General Medicine MariTide (maridebart cafraglutide, AMG 133) MariTide is a differentiated peptide-antibody conjugate that activates the glucagon like peptide 1 (GLP-1) receptor and antagonizes the glucose-dependent insulinotropic polypeptide receptor (GIPR). In June, the underlying details from Part 1 of the Phase 2 study of MariTide and complete results from the primary analysis of the Phase 1 pharmacokinetics low dose initiation (PK-LDI) study evaluating lower starting doses of MariTide were presented at the American Diabetes Association 85th Scientific Sessions and simultaneously published in the New England Journal of Medicine. In the Phase 2 study per the efficacy estimand1, MariTide demonstrated: up to ~20% average weight loss in people living with obesity without Type 2 diabetes (T2D). up to ~17% average weight loss in people living with obesity with T2D. no weight loss plateau by 52 weeks, indicating the potential for further weight reduction. robust and sustained reduction in hemoglobin A1c (HbA1c) of up to 2.2% in people living with obesity and T2D. improvements across pre-specified cardiometabolic measures, including waist circumference, blood pressure, high-sensitivity C-reactive protein (hs-CRP) and select lipid parameters. No new safety signals were identified in the Phase 2 study and tolerability was consistent with the GLP-1 class. The most frequently reported adverse events (AEs) were gastrointestinal (GI) related, and most were mild to moderate, were predominantly limited to initial dosing and less frequent when dose escalation was used without compromising efficacy. Discontinuation rates of MariTide due to GI AEs in the dose escalation arms (up to 7.8%) were lower than non-dose escalation arms. The Phase 1 PK-LDI study showed participants that received 21 mg/70 mg/350 mg had an overall incidence of vomiting of 24.4% and participants that received 35 mg/70 mg/350 mg had an overall incidence of vomiting of 22.5%. There were no discontinuations due to GI AEs at any time during the study. Part 2 of the Phase 2 chronic weight management study is ongoing in adults living with obesity or overweight, with or without T2D. Data readout is anticipated in Q4 2025. A Phase 2 study investigating MariTide for the treatment of T2D is ongoing in adults living with and without obesity. Data readout is anticipated in Q4 2025. MARITIME-1, a Phase 3 study of MariTide is enrolling adults living with obesity or overweight, without T2D. MARITIME-2, a Phase 3 study of MariTide is enrolling adults living with obesity or overweight, with T2D. MARITIME-CV, a Phase 3 study of MariTide on cardiovascular (CV) outcomes was initiated and is enrolling adults living with established atherosclerotic cardiovascular disease and obesity or overweight. MARITIME-HF, a Phase 3 study of MariTide on reduction of heart failure events and cardiovascular risk, was initiated and is enrolling adults living with heart failure with preserved or mildly reduced ejection fraction and obesity. The Company is planning to initiate Phase 3 study of obstructive sleep apnea in H2 2025. AMG 513 A Phase 1 study of AMG 513 is enrolling adults living with obesity. Repatha VESALIUS-CV, a Phase 3 CV outcomes study of Repatha, is ongoing in patients at high CV risk without prior myocardial infarction or stroke. Data readout is event driven and anticipated in H2 2025. EVOLVE-MI, a Phase 4 study of Repatha administered within 10 days of an acute myocardial infarction to reduce the risk of CV events, is ongoing. Olpasiran (AMG 890) Olpasiran is a potentially best-in-class small interfering ribonucleic acid (siRNA) molecule that reduces lipoprotein(a) (Lp(a)) synthesis in the liver. The OCEAN(a)-outcomes trial, a Phase 3 secondary prevention CV outcomes study, is ongoing in patients with atherosclerotic CV disease and elevated Lp(a). A Phase 3 CV outcomes study in patients with elevated Lp(a) and at high risk for a first CV event is expected to be initiated in H2 2025/H1 2026. Rare Disease UPLIZNA U.S. Food and Drug Administration (FDA) review of the MINT Phase 3 data in patients with generalized myasthenia gravis is ongoing, with a PDUFA date of December 14, 2025. TEPEZZA In June, the European Commission granted marketing authorization approval of TEPEZZA for the treatment of adults with moderate to severe thyroid eye disease (TED). Regulatory review is underway in multiple additional geographies. A Phase 3 study of TEPEZZA in Japan is enrolling patients with chronic/low clinical activity score TED. A Phase 3 study evaluating the subcutaneous route of administration of teprotumumab has completed enrollment of patients with TED. TAVNEOS A Phase 3, open-label study of TAVNEOS in combination with rituximab or a cyclophosphamide-containing regimen, is enrolling patients from 6 years to < 18 years of age with active ANCA-associated vasculitis (Granulomatosis with Polyangiitis (GPA)/Microscopic Polyangiitis (MPA)). Dazodalibep Dazodalibep is a fusion protein that inhibits CD40L. Two Phase 3 studies of dazodalibep in Sjögren's disease are underway. The first study has completed enrollment of patients with moderate-to-severe systemic disease activity. The second study is enrolling patients with moderate-to-severe symptomatic burden and low systemic disease activity. Daxdilimab Daxdilimab is a fully human monoclonal antibody targeting immunoglobulin-like transcript 7 (ILT7). A Phase 2 study of daxdilimab is ongoing in patients with moderate-to-severe active primary discoid lupus erythematosus refractory to standard of care. A Phase 2 study of daxdilimab is ongoing in patients with dermatomyositis and antisynthetase inflammatory myositis. AMG 329 AMG 329 is a fully human monoclonal antibody targeting FMS-like tyrosine kinase 3 (FLT3) ligand. A Phase 2 study of AMG 329 is ongoing in patients with Sjögren's disease. AMG 732 AMG 732 is an insulin-like growth factor-1 receptor (IGF-1R) targeting monoclonal antibody. A Phase 2 study of AMG 732 is enrolling patients with moderate-to-severe active TED. Inflammation TEZSPIRE Two Phase 3 studies of TEZSPIRE are enrolling adults with moderate to very severe chronic obstructive pulmonary disease (COPD) and a BEC ≥ 150 cells/µl. In May, primary results from the Phase 3b WAYFINDER study were presented, showing that TEZSPIRE reduces or eliminates oral cortical steroid (OCS) use in OCS-dependent patients with severe uncontrolled asthma. FDA review of the WAYPOINT Phase 3 data in patients with chronic rhinosinusitis with nasal polyps is ongoing with a PDUFA date of October 19, 2025. A Phase 3 study of TEZSPIRE has completed enrollment of patients with eosinophilic esophagitis. Rocatinlimab (AMG 451/KHK4083) Rocatinlimab is a first-in-class T-cell rebalancing monoclonal antibody that inhibits and reduces OX40-positive pathogenic T-cells. The eight study ROCKET Phase 3 program evaluating rocatinlimab in patients with moderate-to-severe atopic dermatitis (AD) has enrolled over 3,300 patients. Enrollment is now complete in seven studies. Key upcoming milestones from the ROCKET Phase 3 program: ROCKET ASCEND is a study evaluating rocatinlimab maintenance therapy in adult and adolescent patients with moderate-to-severe AD. Data readout is anticipated in H2 2025. ROCKET ASTRO is a 52-week study of rocatinlimab in adolescent patients with moderate-to-severe AD. Data readout is anticipated in H2 2025. A Phase 2 study of rocatinlimab is enrolling patients with moderate-to-severe asthma. A Phase 3 study of rocatinlimab is enrolling patients with prurigo nodularis. Blinatumomab Blinatumomab is a bispecific T-cell engager (BiTE®) molecule targeting CD19. A Phase 2 study of blinatumomab in autoimmune disease was initiated in adults with systemic lupus erythematosus (SLE) and in adults with refractory rheumatoid arthritis. Inebilizumab Inebilizumab is a B-cell depleting monoclonal antibody targeting CD19. A Phase 2 study of inebilizumab in autoimmune disease is enrolling adults with SLE with nephritis. AMG 104 (AZD8630) AMG 104 is an inhaled anti-thymic stromal lymphopoietin (TSLP) fragment antigen-binding (Fab). A Phase 2 study is enrolling patients with asthma. Oncology BLINCYTO / blinatumomab In June, Phase 1b/2 subcutaneous blinatumomab data were presented at the European Hematology Association Congress and simultaneously published in The Lancet Haematology demonstrating 89–92% remission rates (complete remission/complete remission with partial hematological recovery rates/complete remission with incomplete count recovery) and manageable safety in adults with relapsed/refractory CD19-positive Philadelphia chromosome (Ph)-negative B-cell precursor acute lymphoblastic leukemia (B-ALL). The dose-expansion and optimization phase of a Phase 1/2 study of subcutaneous blinatumomab is ongoing in adult patients with relapsed or refractory CD19-positive Ph-negative B-ALL. The Company is planning to initiate a potentially registration-enabling Phase 2 portion of this study in both adults and adolescents in H2 2025. Golden Gate, a Phase 3 study of BLINCYTO alternating with low-intensity chemotherapy, is enrolling older adult patients with newly diagnosed CD19-positive Ph-negative B-ALL. IMDELLTRA / tarlatamab IMDELLTRA is the first and only FDA-approved delta-like ligand 3 (DLL3) targeting BiTE® molecule. In June, interim results from the global Phase 3 DeLLphi-304 trial were presented at the American Society of Clinical Oncology annual meeting (ASCO) and simultaneously published in the New England Journal of Medicine. The data showed that IMDELLTRA reduced the risk of death by 40% and significantly extended median overall survival (OS) by more than five months compared to standard-of-care (SOC) chemotherapy in patients with small cell lung cancer (SCLC) who progressed on or after one line of platinum-based chemotherapy. Imdelltra significantly improved patient-reported outcomes of dyspnea and cough compared to SOC chemotherapy. The safety profile of IMDELLTRA was consistent with its known profile. Regulatory filing activities are underway. The Company is advancing a comprehensive, global clinical development program across extensive-stage (ES) and limited-stage (LS) SCLC: DeLLphi-303, a Phase 1b study of IMDELLTRA in combination with a programmed cell death protein ligand-1 (PD-L1) inhibitor, carboplatin and etoposide or separately in combination with a PD-L1 inhibitor alone, is ongoing in patients with first-line ES-SCLC. DeLLphi-305, a Phase 3 study of IMDELLTRA and durvalumab, is enrolling patients with first-line ES-SCLC in the maintenance setting. DeLLphi-306, a Phase 3 study of IMDELLTRA following concurrent chemoradiation therapy, is enrolling patients with LS-SCLC. DeLLphi-308, a Phase 1b study evaluating subcutaneous tarlatamab, is enrolling patients with second line or later ES-SCLC. DeLLphi-309, a Phase 2 study evaluating alternative intravenous dosing regimens of IMDELLTRA in second-line ES-SCLC, is enrolling patients. DeLLphi-310, a Phase 1b study of IMDELLTRA in combination with YL201, a B7-H3 targeting antibody-drug conjugate, with or without a PD-L1 inhibitor, is enrolling patients with ES-SCLC. DeLLphi-311, a Phase 1b study of IMDELLTRA in combination with etakafusp alfa (AB248), a novel CD8+ T-cell selective interleukin-2 (IL-2), was initiated in patients with ES-SCLC. DeLLphi-312, a Phase 3 study of first-line IMDELLTRA in combination with carboplatin, etoposide and durvalumab, was initiated in patients with ES-SCLC. Xaluritamig (AMG 509) Xaluritamig is a first-in-class BiTE® targeting six-transmembrane epithelial antigen of prostate 1 (STEAP1). XALute, a Phase 3 study of xaluritamig, is enrolling patients with metastatic castrate resistant prostate cancer (mCRPC) who have previously been treated with taxane-based chemotherapy. A Phase 1 study of xaluritamig monotherapy is ongoing in patients with mCRPC who have not yet received taxane-based chemotherapy and is ongoing in patients with mCPRC who have previously received taxane-based chemotherapy in a fully outpatient treatment setting to further improve administration convenience. This study continues to enroll mCRPC patients into a combination treatment of xaluritamig and abiraterone. A Phase 1b study of neoadjuvant xaluritamig therapy prior to radical prostatectomy is enrolling patients with newly diagnosed localized intermediate or high‐risk prostate cancer. A Phase 1b study of xaluritamig is enrolling patients with high-risk biochemically recurrent prostate cancer after definitive therapy. Bemarituzumab Bemarituzumab is a first-in-class fibroblast growth factor receptor 2b (FGFR2b) targeting monoclonal antibody. In June, the Company announced the Phase 3 FORTITUDE-101 clinical trial of first-line bemarituzumab plus chemotherapy (mFOLFOX6) met its primary endpoint of OS at a pre-specified interim analysis: Bemarituzumab plus chemotherapy demonstrated a statistically significant and clinically meaningful improvement in OS as compared to placebo plus chemotherapy in patients with unresectable locally advanced or metastatic gastric or gastroesophageal junction cancer with FGFR2b overexpression and who are non-HER2 positive. FGFR2b overexpression was defined as 2+/3+ staining in ≥10% of tumor cells by centrally performed immunohistochemistry testing. The most common treatment-emergent adverse events (>25%) in patients treated with bemarituzumab plus chemotherapy were reduced visual acuity, punctate keratitis, anemia, neutropenia, nausea, corneal epithelium defect and dry eye. While ocular events were consistent with the Phase 2 experience and observed in both arms, they occurred with greater frequency and severity in the Phase 3 bemarituzumab arm. Detailed results from FORTITUDE 101 will be shared at a future medical meeting. FORTITUDE-102, a Phase 1b/3 study of bemarituzumab plus chemotherapy and nivolumab, is ongoing in patients with first-line gastric cancer. Phase 3 data readout is anticipated in H2 2025/H1 2026. FORTITUDE-103, a Phase 1b/2 study of bemarituzumab plus oral chemotherapy regimens with or without nivolumab, is enrolling patients with first-line gastric cancer. FORTITUDE-301, a Phase 1b/2 basket study of bemarituzumab monotherapy, is ongoing in patients with solid tumors with FGFR2b overexpression. AMG 193 AMG 193 is a first-in-class small molecule methylthioadenosine (MTA)-cooperative protein arginine methyltransferase 5 (PRMT5) inhibitor. A Phase 2 study of AMG 193 is enrolling patients with methylthioadenosine phosphorylase (MTAP)-null previously treated advanced non-small cell lung cancer (NSCLC). A Phase 1/1b/2 study of AMG 193 is enrolling patients with advanced MTAP-null solid tumors in the dose-expansion portion of the study. A Phase 1b study of AMG 193 alone or in combination with other therapies is enrolling patients with advanced MTAP-null thoracic malignancies. A Phase 1b study of AMG 193 in combination with other therapies is enrolling patients with advanced MTAP-null gastrointestinal, biliary tract or pancreatic cancers. Kyprolis In May, the FDA granted pediatric exclusivity to Kyprolis for studies conducted under a Written Request and approved updates to the "Pediatric Use" subsection of the Kyprolis prescribing information. LUMAKRAS/LUMYKRAS In May, the FDA granted Breakthrough Therapy Designation to LUMAKRAS in combination with Vectibix and FOLFIRI for the first-line treatment of patients with KRAS G12C-mutated metastatic colorectal cancer (CRC), as determined by an FDA-approved test. This is the third Breakthrough Therapy Designation granted by the FDA for LUMAKRAS. CodeBreaK 301, a Phase 3 study of LUMAKRAS in combination with Vectibix and FOLFIRI vs. FOLFIRI with or without bevacizumab-awwb, is enrolling patients with first-line KRAS G12C–mutated metastatic CRC. CodeBreaK 202, a Phase 3 study of LUMAKRAS plus chemotherapy vs. pembrolizumab plus chemotherapy, is enrolling patients with first-line KRAS G12C–mutated and PD-L1 negative advanced NSCLC. Nplate In June, data were presented at ASCO from the final analysis of RECITE, a Phase 3 study of Nplate as supportive care for chemotherapy-induced thrombocytopenia (CIT) in gastrointestinal cancers: the study met its primary endpoint; more patients on Nplate had no chemotherapy dose modifications due to CIT compared to placebo (84.4% vs. 35.7%, Odds Ratio 10.2; P<0.001). Nplate was well tolerated in a highly comorbid population, with no treatment-related serious adverse events or treatment-related adverse events leading to death or discontinuation of Nplate or chemotherapy. PROCLAIM, a Phase 3 study of Nplate for the treatment of CIT, is enrolling patients with NSCLC, ovarian cancer, or breast cancer. Biosimilars A randomized, double-blind pharmacokinetic similarity study of ABP 206 compared with OPDIVO® (nivolumab) is ongoing in patients with resected stage III or stage IV melanoma in the adjuvant setting. Data readout is anticipated in H2 2025. A randomized, double-blind comparative clinical study of ABP 206 compared with OPDIVO is enrolling patients with treatment-naïve unresectable or metastatic melanoma. A randomized, double-blind pharmacokinetic similarity study of ABP 234 compared with KEYTRUDA® (pembrolizumab) is enrolling patients with early-stage non-squamous NSCLC as adjuvant treatment. A randomized, double-blind combined pharmacokinetic/comparative clinical study of ABP 234 compared to KEYTRUDA is enrolling patients with advanced or metastatic non-squamous NSCLC. A randomized, double-blind pharmacokinetic similarity/comparative clinical study of ABP 692 compared to OCREVUS® (ocrelizumab) is enrolling patients with relapsing-remitting multiple sclerosis. 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. Xaluritamig, formerly AMG 509, is being developed pursuant to a research collaboration with Xencor, Inc. YL201 is an investigational B7-H3 targeting antibody-drug conjugate being developed by MediLink. Etakafusp alfa (AB248) is a novel CD8+ T cell selective interleukin-2 (IL-2) being developed by Asher Biotherapeutics. OPDIVO is a registered trademark of Bristol-Myers Squibb Company. KEYTRUDA is a registered trademark of Merck & Co., Inc. OCREVUS is a registered trademark of Genentech, Inc. 1 The efficacy estimand represents the efficacy as if treated participants had adhered to MariTide for the entire 52-week study period. The efficacy estimand includes endpoint data so long as study drug is taken. Where endpoint data is missing with early discontinuation, the endpoint results for the patient are estimated using individual patient response and predicted performance after drug discontinuation. Non-GAAP Financial Measures In this news release, management has presented its operating results for the second quarters of 2025 and 2024, in accordance with U.S. Generally Accepted Accounting Principles (GAAP) and on a non-GAAP basis. In addition, management has presented its full year 2025 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 second quarters of 2025 and 2024. 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, 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 BeOne Medicines Ltd. or Kyowa Kirin Co., Ltd.), the performance of Otezla® (apremilast), our acquisitions of ChemoCentryx, Inc. or Horizon Therapeutics plc (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), 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, including those resulting from geopolitical relations and government actions. 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. 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CONTACT: Amgen, Thousand Oaks Elissa Snook, 609-251-1407 (media) Justin Claeys, 805-313-9775 (investors) 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. This guidance includes the estimated impact of implemented tariffs, but does not account for any tariffs or potential pricing actions announced or described but not implemented as well as any tariffs, sector specific tariffs, or pricing actions that could be implemented in the future. SOURCE Amgen WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

10岁的小杰（化名）从小就是个“体质差”的孩子，小时候常常因为发烧、呕吐住院，还特别不喜欢吃肉，稍微吃点高蛋白的食物就胃口差、犯恶心。家人以为他只是肠胃功能不好，也没太当回事。直到最近，小杰突然在一天早晨出现神志模糊、频繁呕吐、极度嗜睡，被紧急送往医院。化验发现，他的血氨值高得惊人，已达高氨血症昏迷的边缘！医生立即为他进行了氨的快速清除和低蛋白支持治疗，并进一步进行代谢病筛查。结果发现他血液中的瓜氨酸水平显著升高，尿素循环中断。最终通过基因检测证实，小杰患有瓜氨酸血症I型（CTLN1）——一种由于ASS1基因突变导致的罕见代谢性疾病。概述瓜氨酸血症I型（CTLN1）是一种罕见的常染色体隐性遗传病，包括新生儿急性（典型）型、较轻的晚发型、妊娠期或妊娠后开始的型以及无症状型。CTLN1是由精氨酸琥珀酸酯合成酶（ASS）的缺陷或缺失引起的。ASS是六种酶中的一种，在清除体内氮的过程中起作用，这一过程被称为尿素循环。这种酶的缺乏导致血液和所有体液中的氮以氨的形式过度积累。患有典型形式的婴儿可能会出现呕吐、拒绝进食、进行性昏睡，并出现颅内压升高的迹象。及时治疗可以延长生存期，但通常会出现神经功能障碍。晚发型的病程有时较轻，但高氨血症的发作与典型型相似。临床表现CTLN1 是瓜氨酸血症I型（Citrullinemia Type I），属于一种尿素循环障碍（UCD），由 ASS1 基因突变导致精氨基琥珀酸合成酶（ASS）缺乏所致。以下是经纠正后的瓜氨酸血症I型（CTLN1）症状与体征总结：CTLN1的临床表现严重程度因个体而异。典型的新生儿型最为严重，通常在出生后24到72小时内发病，常在首次进食蛋白质后出现症状。早期表现包括拒奶、嗜睡、呕吐、烦躁和食欲减退，随后可迅速进展为肌张力减弱、呼吸困难、癫痫发作、脑积水、肝功能衰竭，若未及时治疗，可能在短时间内因高氨血症昏迷而危及生命。若昏迷持续超过三天，患儿极易出现神经系统不可逆损伤，如发育迟缓、智力障碍和脑瘫。颅内压升高还可引起肌张力亢进、痉挛、病理反射及反复癫痫发作。部分患儿由于ASS酶残余活性存在，在婴儿期或儿童期才逐渐发病。临床表现相对较轻，包括生长发育迟缓、体重增加不良、厌恶高蛋白食物、进行性嗜睡、活动协调障碍、反复呕吐。这些患儿常在健康期与高氨血症发作期之间反复波动，但即便是轻型个体，也有可能突发高氨血症昏迷，并出现严重的神经损伤甚至危及生命。还有一种罕见的晚发型CTLN1发生在女性妊娠期间或产后。患者可突然出现剧烈呕吐、嗜睡、精神错乱、幻觉、癫痫、躁狂甚至昏迷。部分患者还可能出现脑积水。这类成人型瓜氨酸血症常因未曾诊断的轻型病程在应激状态下失代偿，症状以急性神经精神改变为主，易被误诊为产后精神病或其他神经系统疾病，延误治疗。诊断瓜氨酸血症I型（CTLN1）的诊断总结如下：瓜氨酸血症的诊断依赖于详细的家族病史、临床表现以及一系列特征性的生化和分子检测。血液中氨和瓜氨酸的显著升高是CTLN1的重要诊断线索。确诊可通过ASS1基因的分子遗传学检测来实现。新生儿筛查是目前最常见的早期诊断方式，使用串联质谱法检测血斑中的瓜氨酸水平，在美国已成为所有州标准筛查项目的一部分。早期识别对于预防高氨血症所致的脑损伤至关重要。若家族中已确认有致病性ASS1基因突变，可提供携带者筛查和产前诊断选择。对已知患者的同胞，应在出生后立即进行检查，尤其注意氨或瓜氨酸是否升高，必要时应尽早开始限制蛋白摄入的治疗策略，以降低潜在神经系统损伤的风险。治疗CTLN1的治疗需要由生化遗传学家、儿科医生、神经科医生和营养师组成的多学科团队协作，以确保全面管理。治疗的核心目标是防止体内氨的积聚，并在高氨血症发作时迅速清除过量氨，从而避免神经系统损伤和危及生命的并发症。急性发作期间的治疗重点在于迅速降低血氨水平。包括住院治疗、严格限制蛋白摄入、静脉给予精氨酸、苯甲酸钠和苯乙酸钠等氮清除药物，同时提供非蛋白热量支持（如葡萄糖）。若氨水平过高或病情恶化，可通过血液透析清除体内氨，尤其适用于高氨血症昏迷或首次确诊的严重病例。长期管理包括维持低蛋白、高热量的饮食，同时确保婴儿和儿童有足够的营养以促进正常生长发育。食物中通常采用高生物价值的天然蛋白质（如母乳或配方奶）、必需氨基酸配方，以及不含蛋白的热量补充剂，另辅以多种维生素和钙补充剂。药物方面，可持续使用Buphenyl、Ammonul、Raviciti和精氨酸等帮助氮的排出。监测在管理中至关重要。需定期检测血氨水平及血浆氨基酸浓度，以评估病情稳定性和饮食疗效。高氨血症的预警信号包括嗜睡、情绪改变、头痛、呕吐、拒食和踝关节挛缩等，应及时识别并干预。为了防止颅内压升高，也需对患者进行神经系统状况的密切监控。对于某些反复发作或控制困难的患者，肝移植可能是改善预后、延长寿命的有效手段。肝移植可恢复尿素循环功能，从而根治氨代谢异常，提高患者生活质量。参考资料：1.Potter MA, Zeesman S, Brennan B, et al. Pregnancy in a healthy woman with untreated citrullinemia. Am J Med Genet A. 2004; 129A: 77, 82.2.Ruitenbeek W, Kobayashi K, Iijima M, et al. Moderate citrullinaemia without hyperammonaemia in a child with mutated and deficient argininosuccinate synthetase. Ann Clin Biochem. 2003; 40: 102, 7.3.Häberle J, Pauli S, Schmidt E,et al. Mild citrullinemia in Caucasians is an allelic variant of argininosuccinate synthetase deficiency (citrullinemia type 1). Mol Genet Metab. 2003; 80: 302, 6.来源 | 梅斯医学编辑 | wanny神经系统罕见病交流群↓点击下方“阅读原文”，下载梅斯医学APP吧！